In the opinion of Neil H. Shear, MD, a stepwise approach is the best way to diagnose possible drug-induced skin disease and determine the root cause.

“Often, we need to think of more than one cause,” he said during the annual meeting of the Society for Pediatric Dermatology. “It could be drug X. It could be drug Y. It could be contrast media. We must think broadly and pay special attention to skin of color, overlapping syndromes, and the changing diagnostic assessment over time.”

His suggested diagnostic triangle includes appearance of the rash or lesion(s), systemic impact, and histology. “The first is the appearance,” said Dr. Shear, professor emeritus of dermatology, clinical pharmacology and toxicology, and medicine at the University of Toronto. “Is it exanthem? Is it blistering? Don’t just say drug ‘rash.’ That doesn’t work. You need to know if there are systemic features, and sometimes histologic information can change your approach or diagnosis, but not as often as one might think,” he said, noting that, in his view, the two main factors are appearance and systemic impact.

The presence of fever is a hallmark of systemic problems, he continued, “so if you see fever, you know you’re probably going to be dealing with a complex reaction, so we need to know the morphology.” Consider whether it is simple exanthem (a mild, uncomplicated rash) or complex exanthem (drug rash with eosinophilia and systemic symptoms or fever, malaise, and adenopathy).

As for other morphologies, urticarial lesions could be urticaria or a serum sickness-like reaction, pustular lesions could be acneiform or acute generalized exanthematous pustulosis, while blistering lesions could suggest a fixed drug response or Stevens-Johnson syndrome (SJS)/toxic epidermal necrolysis (TEN).

Dr. Shear considers SJS/TEN as a spectrum of blistering disease, “because there’s not a single diagnosis,” he said. “There’s a spectrum, if you will, depending on how advanced people are in their disease.” He coauthored a 1991 report describing eight cases of mycoplasma and Stevens-Johnson syndrome. “I was surprised at how long that stood up as about the only paper in that area,” he said. “But there’s much more happening now with a proliferation of terms,” he added, referring to MIRM (Mycoplasma pneumonia–induced rash and mucositis), RIME (reactive infectious mucocutaneous eruption); and Fuchs syndrome, or SJS without skin lesions.

What was not appreciated in the early classification of SJS, he continued, was a “side basket” of bullous erythema multiforme. “We didn’t know what to call it,” he said. “At one point we called it bullous erythema multiforme. At another point we called it erythema multiforme major. We just didn’t know what it was.”

The appearance and systemic effects of SJS comprise what he termed SJS type 2 – or the early stages of TEN. Taken together, he refers to these two conditions as TEN Spectrum, or TENS. “One of the traps is that TENS can look like varicella, and vice versa, especially in very dark brown or black skin,” Dr. Shear said. “You have to be careful. A biopsy might be worthwhile. Acute lupus has the pathology of TENS but the patients are not as systemically ill as true TENS.”

In 2011, Japanese researchers reported on 38 cases of SJS associated with M. pneumoniae, and 78 cases of drug-induced SJS. They found that 66% of adult patients with M. pneumoniae–associated SJS developed mucocutaneous lesions and fever/respiratory symptoms on the same day, mostly shortness of breath and cough. In contrast, most of the patients aged under 20 years developed fever/respiratory symptoms before mucocutaneous involvement.

“The big clinical differentiator between drug-induced SJS and mycoplasma-induced SJS was respiratory disorder,” said Dr. Shear, who was not affiliated with the study. “That means you’re probably looking at something that’s mycoplasma related [when respiratory problems are present]. Even if you can’t prove it’s mycoplasma related, that probably needs to be the target of your therapy. The idea ... is to make sure it’s clear at the end. One, so they get better, and two, so that we’re not giving drugs needlessly when it was really mycoplasma.”

Noting that HLA-B*15:02 is a marker for carbamazepine-induced SJS and TEN, he said, “a positive HLA test can support the diagnosis, confirm the suspected offending drug, and is valuable for familial genetic counseling.”

As for treatment of SJS, TEN, and other cytotoxic T-lymphocyte–mediated severe cutaneous adverse reactions, a randomized Japanese clinical trial evaluating prednisolone 1-1.5 mg/kg/day IV versus etanercept 25-50 mg subcutaneously twice per week in 96 patients with SJS-TEN found that etanercept decreased the mortality rate by 8.3%. In addition, etanercept reduced skin healing time, when compared with prednisolone (a median of 14 vs. 19 days, respectively; P = .010), and was associated with a lower incidence of GI hemorrhage (2.6% vs. 18.2%, respectively; P = .03).

Dr. Shear said that he would like to see better therapeutics for severe, complex patients. “After leaving the hospital, people with SJS or people with TEN need to have ongoing care, consultation, and explanation so they and their families know what drugs are safe in the future.”

Dr. Shear disclosed that he has been a consultant to AbbVie, Amgen, Bausch Medicine, Novartis, Sanofi-Genzyme, UCB, LEO Pharma, Otsuka, Janssen, Alpha Laboratories, Lilly, ChemoCentryx, Vivoryon, Galderma, Innovaderm, Chromocell, and Kyowa Kirin.

dbrunk@mdedge.com

In the opinion of Neil H. Shear, MD, a stepwise approach is the best way to diagnose possible drug-induced skin disease and determine the root cause.

“Often, we need to think of more than one cause,” he said during the annual meeting of the Society for Pediatric Dermatology. “It could be drug X. It could be drug Y. It could be contrast media. We must think broadly and pay special attention to skin of color, overlapping syndromes, and the changing diagnostic assessment over time.”

His suggested diagnostic triangle includes appearance of the rash or lesion(s), systemic impact, and histology. “The first is the appearance,” said Dr. Shear, professor emeritus of dermatology, clinical pharmacology and toxicology, and medicine at the University of Toronto. “Is it exanthem? Is it blistering? Don’t just say drug ‘rash.’ That doesn’t work. You need to know if there are systemic features, and sometimes histologic information can change your approach or diagnosis, but not as often as one might think,” he said, noting that, in his view, the two main factors are appearance and systemic impact.

The presence of fever is a hallmark of systemic problems, he continued, “so if you see fever, you know you’re probably going to be dealing with a complex reaction, so we need to know the morphology.” Consider whether it is simple exanthem (a mild, uncomplicated rash) or complex exanthem (drug rash with eosinophilia and systemic symptoms or fever, malaise, and adenopathy).

As for other morphologies, urticarial lesions could be urticaria or a serum sickness-like reaction, pustular lesions could be acneiform or acute generalized exanthematous pustulosis, while blistering lesions could suggest a fixed drug response or Stevens-Johnson syndrome (SJS)/toxic epidermal necrolysis (TEN).

Dr. Shear considers SJS/TEN as a spectrum of blistering disease, “because there’s not a single diagnosis,” he said. “There’s a spectrum, if you will, depending on how advanced people are in their disease.” He coauthored a 1991 report describing eight cases of mycoplasma and Stevens-Johnson syndrome. “I was surprised at how long that stood up as about the only paper in that area,” he said. “But there’s much more happening now with a proliferation of terms,” he added, referring to MIRM (Mycoplasma pneumonia–induced rash and mucositis), RIME (reactive infectious mucocutaneous eruption); and Fuchs syndrome, or SJS without skin lesions.

What was not appreciated in the early classification of SJS, he continued, was a “side basket” of bullous erythema multiforme. “We didn’t know what to call it,” he said. “At one point we called it bullous erythema multiforme. At another point we called it erythema multiforme major. We just didn’t know what it was.”

The appearance and systemic effects of SJS comprise what he termed SJS type 2 – or the early stages of TEN. Taken together, he refers to these two conditions as TEN Spectrum, or TENS. “One of the traps is that TENS can look like varicella, and vice versa, especially in very dark brown or black skin,” Dr. Shear said. “You have to be careful. A biopsy might be worthwhile. Acute lupus has the pathology of TENS but the patients are not as systemically ill as true TENS.”

In 2011, Japanese researchers reported on 38 cases of SJS associated with M. pneumoniae, and 78 cases of drug-induced SJS. They found that 66% of adult patients with M. pneumoniae–associated SJS developed mucocutaneous lesions and fever/respiratory symptoms on the same day, mostly shortness of breath and cough. In contrast, most of the patients aged under 20 years developed fever/respiratory symptoms before mucocutaneous involvement.

“The big clinical differentiator between drug-induced SJS and mycoplasma-induced SJS was respiratory disorder,” said Dr. Shear, who was not affiliated with the study. “That means you’re probably looking at something that’s mycoplasma related [when respiratory problems are present]. Even if you can’t prove it’s mycoplasma related, that probably needs to be the target of your therapy. The idea ... is to make sure it’s clear at the end. One, so they get better, and two, so that we’re not giving drugs needlessly when it was really mycoplasma.”

Noting that HLA-B*15:02 is a marker for carbamazepine-induced SJS and TEN, he said, “a positive HLA test can support the diagnosis, confirm the suspected offending drug, and is valuable for familial genetic counseling.”

As for treatment of SJS, TEN, and other cytotoxic T-lymphocyte–mediated severe cutaneous adverse reactions, a randomized Japanese clinical trial evaluating prednisolone 1-1.5 mg/kg/day IV versus etanercept 25-50 mg subcutaneously twice per week in 96 patients with SJS-TEN found that etanercept decreased the mortality rate by 8.3%. In addition, etanercept reduced skin healing time, when compared with prednisolone (a median of 14 vs. 19 days, respectively; P = .010), and was associated with a lower incidence of GI hemorrhage (2.6% vs. 18.2%, respectively; P = .03).

Dr. Shear said that he would like to see better therapeutics for severe, complex patients. “After leaving the hospital, people with SJS or people with TEN need to have ongoing care, consultation, and explanation so they and their families know what drugs are safe in the future.”

Dr. Shear disclosed that he has been a consultant to AbbVie, Amgen, Bausch Medicine, Novartis, Sanofi-Genzyme, UCB, LEO Pharma, Otsuka, Janssen, Alpha Laboratories, Lilly, ChemoCentryx, Vivoryon, Galderma, Innovaderm, Chromocell, and Kyowa Kirin.

dbrunk@mdedge.com

In the opinion of Neil H. Shear, MD, a stepwise approach is the best way to diagnose possible drug-induced skin disease and determine the root cause.

“Often, we need to think of more than one cause,” he said during the annual meeting of the Society for Pediatric Dermatology. “It could be drug X. It could be drug Y. It could be contrast media. We must think broadly and pay special attention to skin of color, overlapping syndromes, and the changing diagnostic assessment over time.”

His suggested diagnostic triangle includes appearance of the rash or lesion(s), systemic impact, and histology. “The first is the appearance,” said Dr. Shear, professor emeritus of dermatology, clinical pharmacology and toxicology, and medicine at the University of Toronto. “Is it exanthem? Is it blistering? Don’t just say drug ‘rash.’ That doesn’t work. You need to know if there are systemic features, and sometimes histologic information can change your approach or diagnosis, but not as often as one might think,” he said, noting that, in his view, the two main factors are appearance and systemic impact.

The presence of fever is a hallmark of systemic problems, he continued, “so if you see fever, you know you’re probably going to be dealing with a complex reaction, so we need to know the morphology.” Consider whether it is simple exanthem (a mild, uncomplicated rash) or complex exanthem (drug rash with eosinophilia and systemic symptoms or fever, malaise, and adenopathy).

As for other morphologies, urticarial lesions could be urticaria or a serum sickness-like reaction, pustular lesions could be acneiform or acute generalized exanthematous pustulosis, while blistering lesions could suggest a fixed drug response or Stevens-Johnson syndrome (SJS)/toxic epidermal necrolysis (TEN).

Dr. Shear considers SJS/TEN as a spectrum of blistering disease, “because there’s not a single diagnosis,” he said. “There’s a spectrum, if you will, depending on how advanced people are in their disease.” He coauthored a 1991 report describing eight cases of mycoplasma and Stevens-Johnson syndrome. “I was surprised at how long that stood up as about the only paper in that area,” he said. “But there’s much more happening now with a proliferation of terms,” he added, referring to MIRM (Mycoplasma pneumonia–induced rash and mucositis), RIME (reactive infectious mucocutaneous eruption); and Fuchs syndrome, or SJS without skin lesions.

What was not appreciated in the early classification of SJS, he continued, was a “side basket” of bullous erythema multiforme. “We didn’t know what to call it,” he said. “At one point we called it bullous erythema multiforme. At another point we called it erythema multiforme major. We just didn’t know what it was.”

The appearance and systemic effects of SJS comprise what he termed SJS type 2 – or the early stages of TEN. Taken together, he refers to these two conditions as TEN Spectrum, or TENS. “One of the traps is that TENS can look like varicella, and vice versa, especially in very dark brown or black skin,” Dr. Shear said. “You have to be careful. A biopsy might be worthwhile. Acute lupus has the pathology of TENS but the patients are not as systemically ill as true TENS.”

In 2011, Japanese researchers reported on 38 cases of SJS associated with M. pneumoniae, and 78 cases of drug-induced SJS. They found that 66% of adult patients with M. pneumoniae–associated SJS developed mucocutaneous lesions and fever/respiratory symptoms on the same day, mostly shortness of breath and cough. In contrast, most of the patients aged under 20 years developed fever/respiratory symptoms before mucocutaneous involvement.

“The big clinical differentiator between drug-induced SJS and mycoplasma-induced SJS was respiratory disorder,” said Dr. Shear, who was not affiliated with the study. “That means you’re probably looking at something that’s mycoplasma related [when respiratory problems are present]. Even if you can’t prove it’s mycoplasma related, that probably needs to be the target of your therapy. The idea ... is to make sure it’s clear at the end. One, so they get better, and two, so that we’re not giving drugs needlessly when it was really mycoplasma.”

Noting that HLA-B*15:02 is a marker for carbamazepine-induced SJS and TEN, he said, “a positive HLA test can support the diagnosis, confirm the suspected offending drug, and is valuable for familial genetic counseling.”

As for treatment of SJS, TEN, and other cytotoxic T-lymphocyte–mediated severe cutaneous adverse reactions, a randomized Japanese clinical trial evaluating prednisolone 1-1.5 mg/kg/day IV versus etanercept 25-50 mg subcutaneously twice per week in 96 patients with SJS-TEN found that etanercept decreased the mortality rate by 8.3%. In addition, etanercept reduced skin healing time, when compared with prednisolone (a median of 14 vs. 19 days, respectively; P = .010), and was associated with a lower incidence of GI hemorrhage (2.6% vs. 18.2%, respectively; P = .03).

Dr. Shear said that he would like to see better therapeutics for severe, complex patients. “After leaving the hospital, people with SJS or people with TEN need to have ongoing care, consultation, and explanation so they and their families know what drugs are safe in the future.”

Dr. Shear disclosed that he has been a consultant to AbbVie, Amgen, Bausch Medicine, Novartis, Sanofi-Genzyme, UCB, LEO Pharma, Otsuka, Janssen, Alpha Laboratories, Lilly, ChemoCentryx, Vivoryon, Galderma, Innovaderm, Chromocell, and Kyowa Kirin.

dbrunk@mdedge.com

Background: Acid suppressants inhibit gastric acid secretion and disrupt the intestinal microbiome, but whether that facilitates colonization and infection with MDROs is unclear.

Dr. Li is assistant professor of medicine, section of hospital medicine, at the University of Virginia School of Medicine, Charlottesville.

Background: Acid suppressants inhibit gastric acid secretion and disrupt the intestinal microbiome, but whether that facilitates colonization and infection with MDROs is unclear.

Dr. Li is assistant professor of medicine, section of hospital medicine, at the University of Virginia School of Medicine, Charlottesville.

Background: Acid suppressants inhibit gastric acid secretion and disrupt the intestinal microbiome, but whether that facilitates colonization and infection with MDROs is unclear.

Dr. Li is assistant professor of medicine, section of hospital medicine, at the University of Virginia School of Medicine, Charlottesville.

Five of the more uncommon dermatologic disorders – sarcoidosis, Rosai-Dorfman disease, Erdheim-Chester disease, eosinophilic fasciitis, and cutaneous Crohn disease – are linked to internal diseases and may spawn misdiagnoses, a dermatologist told colleagues.

“Proper diagnosis can lead to an effective management in our patients,” said Jeffrey Callen, MD, professor of medicine and chief of dermatology at the University of Louisville (Ky.), who spoke at the Inaugural Symposium for Inflammatory Skin Disease.
 

Sarcoidosis

The cause of sarcoidosis, an inflammatory disease that tends to affect the lungs, “is unknown, but it’s probably an immunologic disorder,” Dr. Callen said, “and there probably is a genetic predisposition.” About 20%-25% of patients with sarcoidosis have skin lesions that are either “specific” (a biopsy that reveals a noncaseating – “naked” – granuloma) or “nonspecific” (most commonly, erythema nodosum, or EN).

The specific lesions in sarcoidosis may occur in parts of the body, such as the knees, which were injured earlier in life and may have taken in foreign bodies, Dr. Callen said. As for nonspecific lesions, about 20% of patients with EN have an acute, self-limiting form of sarcoidosis. “These patients will have bilateral hilar lymphadenopathy, anterior uveitis, and polyarthritis. It’s generally treated symptomatically because it goes away on its own.”

He cautioned colleagues to beware of indurated, infiltrative facial lesions known as lupus pernio that are commonly found on the nose. They’re more prevalent in Black patients and possibly women, who are at higher risk of manifestations outside the skin, he said. “If you have it along the nasal rim, you should look into the upper respiratory tract for involvement.”

Dr. Callen recommends an extensive workup in patients with suspected sarcoidosis, including biopsy (with the exception of EN lesions), cultures and special stains, and screening when appropriate, for disease in organs such as the eyes, lungs, heart, and kidneys.

As for treatment, “the disease is in the dermis, and some topical therapies are not highly effective,” he said. There are injections that can be given, including corticosteroids, and there are a variety of oral treatments that are all off label.” These include corticosteroids, antimalarials, allopurinol, and tetracyclines, among several others. Subcutaneous and intravenous treatments are also options, along with surgery and laser therapy to treat specific lesions.

Rosai-Dorfman disease

This rare disorder is caused by overproduction of certain white blood cells in the lymph nodes, which can cause nodular lesions. The disease most often appears in children and young adults, often Black individuals and males. It is fatal in as many as 11% of patients, justifying aggressive treatment in patients with aggressive disease, Dr. Callen said. When it’s limited to the skin, however, “nothing may need to be done.”

Dr. Callen highlighted consensus recommendations about diagnosis and treatment of Rosai-Dorfman disease published in 2018.

He also noted the existence of cutaneous Rosai-Dorfman disease, a “solitary process” that appears more commonly in females, and in people of Asian heritage, compared with White individuals. It is characterized by single, clustered or widespread lesions: They can be xanthomatous, erythematous, or red-brown papules, nodules, and plaques. They’re acneiform, pustular, giant granuloma annulare–like, subcutaneous, and vasculitis-like, he said.

While Rosai-Dorfman disease can be linked to lymphoma, hypothyroidism, and lupus erythematosus, “nothing necessarily needs to be done when it’s skin-limited since it can be self-resolving,” he noted. Other treatments include radiotherapy, cryotherapy, excision, topical and oral corticosteroids, thalidomide, and methotrexate.

The disease can be serious, and is fatal in 5% of cases. When a vital organ is threatened, Dr. Callen suggested surgery, chemotherapy, or radiation.
 

Erdheim-Chester disease

This disease – which is extremely rare, with just 500 cases noted before 2014 – occurs when the body overproduces macrophages. It’s most common in middle-aged people and in men, who make up 75% of cases. About a quarter of patients develop skin lesions: Red-brown to yellow nodules and xanthelasma-like indurated plaques on the eyelids, scalp, neck, trunk, and axillae, and “other cutaneous manifestations have been reported in patients,” Dr. Callen said.

The disease also frequently affects the bones, large vessels, heart, lungs, and central nervous system. Interferon-alpha is the first-line treatment, and there are several other alternative therapies, although 5-year survival (68%) is poor, and it is especially likely to be fatal in those with central nervous system involvement.
 

Eosinophilic fasciitis

Eosinophilic fasciitis (EF) “is a disorder of unknown etiology that causes sclerosis of the skin” without Raynaud’s phenomenon, Dr. Callen said. Look for erythema, swelling, and induration of the extremities that is accompanied by peripheral eosinophilia, and if necessary, confirm the diagnosis with full skin-to-muscle biopsy or MRI.

There are many possible triggers, including strenuous exercise, initiation with hemodialysis, radiation therapy and burns, and graft-versus-host disease. Other potential causes include exposure to medications such as statins, phenytoin, ramipril, subcutaneous heparin, and immune checkpoint inhibitor therapy. The disorder is also linked to autoimmune and hematologic disorders.

Dr. Callen, who highlighted EF guidelines published in 2018, said treatments include physical therapy, prednisone, methotrexate, mycophenolate, and hydroxychloroquine.
 

Metastatic Crohn’s disease

This is a rare granulomatous inflammation of skin that often affects the genitals, especially in children. It is noncontiguous with the GI tract, and severity of skin involvement does not always parallel the severity of the disease in the GI tract, Dr. Callen said. However, the condition can occur before or simultaneously with the development of GI disease, or after GI surgery.

He highlighted a review of metastatic Crohn’s disease, published in 2014, and noted that there are multiple treatments, including systemic corticosteroids, tumor necrosis factor–alpha inhibitors, and topical therapies.

Dr. Callen reported no relevant disclosures.

Five of the more uncommon dermatologic disorders – sarcoidosis, Rosai-Dorfman disease, Erdheim-Chester disease, eosinophilic fasciitis, and cutaneous Crohn disease – are linked to internal diseases and may spawn misdiagnoses, a dermatologist told colleagues.

“Proper diagnosis can lead to an effective management in our patients,” said Jeffrey Callen, MD, professor of medicine and chief of dermatology at the University of Louisville (Ky.), who spoke at the Inaugural Symposium for Inflammatory Skin Disease.
 

Sarcoidosis

The cause of sarcoidosis, an inflammatory disease that tends to affect the lungs, “is unknown, but it’s probably an immunologic disorder,” Dr. Callen said, “and there probably is a genetic predisposition.” About 20%-25% of patients with sarcoidosis have skin lesions that are either “specific” (a biopsy that reveals a noncaseating – “naked” – granuloma) or “nonspecific” (most commonly, erythema nodosum, or EN).

The specific lesions in sarcoidosis may occur in parts of the body, such as the knees, which were injured earlier in life and may have taken in foreign bodies, Dr. Callen said. As for nonspecific lesions, about 20% of patients with EN have an acute, self-limiting form of sarcoidosis. “These patients will have bilateral hilar lymphadenopathy, anterior uveitis, and polyarthritis. It’s generally treated symptomatically because it goes away on its own.”

He cautioned colleagues to beware of indurated, infiltrative facial lesions known as lupus pernio that are commonly found on the nose. They’re more prevalent in Black patients and possibly women, who are at higher risk of manifestations outside the skin, he said. “If you have it along the nasal rim, you should look into the upper respiratory tract for involvement.”

Dr. Callen recommends an extensive workup in patients with suspected sarcoidosis, including biopsy (with the exception of EN lesions), cultures and special stains, and screening when appropriate, for disease in organs such as the eyes, lungs, heart, and kidneys.

As for treatment, “the disease is in the dermis, and some topical therapies are not highly effective,” he said. There are injections that can be given, including corticosteroids, and there are a variety of oral treatments that are all off label.” These include corticosteroids, antimalarials, allopurinol, and tetracyclines, among several others. Subcutaneous and intravenous treatments are also options, along with surgery and laser therapy to treat specific lesions.

Rosai-Dorfman disease

This rare disorder is caused by overproduction of certain white blood cells in the lymph nodes, which can cause nodular lesions. The disease most often appears in children and young adults, often Black individuals and males. It is fatal in as many as 11% of patients, justifying aggressive treatment in patients with aggressive disease, Dr. Callen said. When it’s limited to the skin, however, “nothing may need to be done.”

Dr. Callen highlighted consensus recommendations about diagnosis and treatment of Rosai-Dorfman disease published in 2018.

He also noted the existence of cutaneous Rosai-Dorfman disease, a “solitary process” that appears more commonly in females, and in people of Asian heritage, compared with White individuals. It is characterized by single, clustered or widespread lesions: They can be xanthomatous, erythematous, or red-brown papules, nodules, and plaques. They’re acneiform, pustular, giant granuloma annulare–like, subcutaneous, and vasculitis-like, he said.

While Rosai-Dorfman disease can be linked to lymphoma, hypothyroidism, and lupus erythematosus, “nothing necessarily needs to be done when it’s skin-limited since it can be self-resolving,” he noted. Other treatments include radiotherapy, cryotherapy, excision, topical and oral corticosteroids, thalidomide, and methotrexate.

The disease can be serious, and is fatal in 5% of cases. When a vital organ is threatened, Dr. Callen suggested surgery, chemotherapy, or radiation.
 

Erdheim-Chester disease

This disease – which is extremely rare, with just 500 cases noted before 2014 – occurs when the body overproduces macrophages. It’s most common in middle-aged people and in men, who make up 75% of cases. About a quarter of patients develop skin lesions: Red-brown to yellow nodules and xanthelasma-like indurated plaques on the eyelids, scalp, neck, trunk, and axillae, and “other cutaneous manifestations have been reported in patients,” Dr. Callen said.

The disease also frequently affects the bones, large vessels, heart, lungs, and central nervous system. Interferon-alpha is the first-line treatment, and there are several other alternative therapies, although 5-year survival (68%) is poor, and it is especially likely to be fatal in those with central nervous system involvement.
 

Eosinophilic fasciitis

Eosinophilic fasciitis (EF) “is a disorder of unknown etiology that causes sclerosis of the skin” without Raynaud’s phenomenon, Dr. Callen said. Look for erythema, swelling, and induration of the extremities that is accompanied by peripheral eosinophilia, and if necessary, confirm the diagnosis with full skin-to-muscle biopsy or MRI.

There are many possible triggers, including strenuous exercise, initiation with hemodialysis, radiation therapy and burns, and graft-versus-host disease. Other potential causes include exposure to medications such as statins, phenytoin, ramipril, subcutaneous heparin, and immune checkpoint inhibitor therapy. The disorder is also linked to autoimmune and hematologic disorders.

Dr. Callen, who highlighted EF guidelines published in 2018, said treatments include physical therapy, prednisone, methotrexate, mycophenolate, and hydroxychloroquine.
 

Metastatic Crohn’s disease

This is a rare granulomatous inflammation of skin that often affects the genitals, especially in children. It is noncontiguous with the GI tract, and severity of skin involvement does not always parallel the severity of the disease in the GI tract, Dr. Callen said. However, the condition can occur before or simultaneously with the development of GI disease, or after GI surgery.

He highlighted a review of metastatic Crohn’s disease, published in 2014, and noted that there are multiple treatments, including systemic corticosteroids, tumor necrosis factor–alpha inhibitors, and topical therapies.

Dr. Callen reported no relevant disclosures.

Five of the more uncommon dermatologic disorders – sarcoidosis, Rosai-Dorfman disease, Erdheim-Chester disease, eosinophilic fasciitis, and cutaneous Crohn disease – are linked to internal diseases and may spawn misdiagnoses, a dermatologist told colleagues.

“Proper diagnosis can lead to an effective management in our patients,” said Jeffrey Callen, MD, professor of medicine and chief of dermatology at the University of Louisville (Ky.), who spoke at the Inaugural Symposium for Inflammatory Skin Disease.
 

Sarcoidosis

The cause of sarcoidosis, an inflammatory disease that tends to affect the lungs, “is unknown, but it’s probably an immunologic disorder,” Dr. Callen said, “and there probably is a genetic predisposition.” About 20%-25% of patients with sarcoidosis have skin lesions that are either “specific” (a biopsy that reveals a noncaseating – “naked” – granuloma) or “nonspecific” (most commonly, erythema nodosum, or EN).

The specific lesions in sarcoidosis may occur in parts of the body, such as the knees, which were injured earlier in life and may have taken in foreign bodies, Dr. Callen said. As for nonspecific lesions, about 20% of patients with EN have an acute, self-limiting form of sarcoidosis. “These patients will have bilateral hilar lymphadenopathy, anterior uveitis, and polyarthritis. It’s generally treated symptomatically because it goes away on its own.”

He cautioned colleagues to beware of indurated, infiltrative facial lesions known as lupus pernio that are commonly found on the nose. They’re more prevalent in Black patients and possibly women, who are at higher risk of manifestations outside the skin, he said. “If you have it along the nasal rim, you should look into the upper respiratory tract for involvement.”

Dr. Callen recommends an extensive workup in patients with suspected sarcoidosis, including biopsy (with the exception of EN lesions), cultures and special stains, and screening when appropriate, for disease in organs such as the eyes, lungs, heart, and kidneys.

As for treatment, “the disease is in the dermis, and some topical therapies are not highly effective,” he said. There are injections that can be given, including corticosteroids, and there are a variety of oral treatments that are all off label.” These include corticosteroids, antimalarials, allopurinol, and tetracyclines, among several others. Subcutaneous and intravenous treatments are also options, along with surgery and laser therapy to treat specific lesions.

Rosai-Dorfman disease

This rare disorder is caused by overproduction of certain white blood cells in the lymph nodes, which can cause nodular lesions. The disease most often appears in children and young adults, often Black individuals and males. It is fatal in as many as 11% of patients, justifying aggressive treatment in patients with aggressive disease, Dr. Callen said. When it’s limited to the skin, however, “nothing may need to be done.”

Dr. Callen highlighted consensus recommendations about diagnosis and treatment of Rosai-Dorfman disease published in 2018.

He also noted the existence of cutaneous Rosai-Dorfman disease, a “solitary process” that appears more commonly in females, and in people of Asian heritage, compared with White individuals. It is characterized by single, clustered or widespread lesions: They can be xanthomatous, erythematous, or red-brown papules, nodules, and plaques. They’re acneiform, pustular, giant granuloma annulare–like, subcutaneous, and vasculitis-like, he said.

While Rosai-Dorfman disease can be linked to lymphoma, hypothyroidism, and lupus erythematosus, “nothing necessarily needs to be done when it’s skin-limited since it can be self-resolving,” he noted. Other treatments include radiotherapy, cryotherapy, excision, topical and oral corticosteroids, thalidomide, and methotrexate.

The disease can be serious, and is fatal in 5% of cases. When a vital organ is threatened, Dr. Callen suggested surgery, chemotherapy, or radiation.
 

Erdheim-Chester disease

This disease – which is extremely rare, with just 500 cases noted before 2014 – occurs when the body overproduces macrophages. It’s most common in middle-aged people and in men, who make up 75% of cases. About a quarter of patients develop skin lesions: Red-brown to yellow nodules and xanthelasma-like indurated plaques on the eyelids, scalp, neck, trunk, and axillae, and “other cutaneous manifestations have been reported in patients,” Dr. Callen said.

The disease also frequently affects the bones, large vessels, heart, lungs, and central nervous system. Interferon-alpha is the first-line treatment, and there are several other alternative therapies, although 5-year survival (68%) is poor, and it is especially likely to be fatal in those with central nervous system involvement.
 

Eosinophilic fasciitis

Eosinophilic fasciitis (EF) “is a disorder of unknown etiology that causes sclerosis of the skin” without Raynaud’s phenomenon, Dr. Callen said. Look for erythema, swelling, and induration of the extremities that is accompanied by peripheral eosinophilia, and if necessary, confirm the diagnosis with full skin-to-muscle biopsy or MRI.

There are many possible triggers, including strenuous exercise, initiation with hemodialysis, radiation therapy and burns, and graft-versus-host disease. Other potential causes include exposure to medications such as statins, phenytoin, ramipril, subcutaneous heparin, and immune checkpoint inhibitor therapy. The disorder is also linked to autoimmune and hematologic disorders.

Dr. Callen, who highlighted EF guidelines published in 2018, said treatments include physical therapy, prednisone, methotrexate, mycophenolate, and hydroxychloroquine.
 

Metastatic Crohn’s disease

This is a rare granulomatous inflammation of skin that often affects the genitals, especially in children. It is noncontiguous with the GI tract, and severity of skin involvement does not always parallel the severity of the disease in the GI tract, Dr. Callen said. However, the condition can occur before or simultaneously with the development of GI disease, or after GI surgery.

He highlighted a review of metastatic Crohn’s disease, published in 2014, and noted that there are multiple treatments, including systemic corticosteroids, tumor necrosis factor–alpha inhibitors, and topical therapies.

Dr. Callen reported no relevant disclosures.

Two chronic gynecologic conditions notably affect a woman’s quality of life (QoL), including fertility – one is endometriosis, and the other is a fibroid uterus. For a benign tumor, fibroids have an impressive prevalence found in approximately 50%-60% of women during their reproductive years. By menopause, it is estimated that 70% of woman have a fibroid, yet the true incidence is unknown given that only 25% of women experience symptoms bothersome enough to warrant intervention. This month’s article reviews the burden of fibroids and the latest management options that may potentially avoid surgery.

Background

Fibroids are monoclonal tumors of uterine smooth muscle that originate from the myometrium. Risk factors include family history, being premenopausal, increasing time since last delivery, obesity, and hypertension (ACOG Practice Bulletin no. 228 Jun 2021: Obstet Gynecol. 2021 Jun 1;137[6]:e100-e15) but oral hormonal contraception, depot medroxyprogesterone acetate (MPA), and increased parity reduce the risk of fibroids. Compared with White women, Black women have a 2-3 times higher prevalence of fibroids, develop them at a younger age, and present with larger fibroids.

The FIGO leiomyoma classification is the agreed upon system for identifying fibroid location. Symptoms are all too familiar to gynecologists, with life-threatening hemorrhage with severe anemia being the most feared, particularly for FIGO types 1-5. Transvaginal ultrasound is the simplest imaging tool for evaluation.

Adapted from Int J Gynecol Obstet. 2011;113:3-13.

Fibroids and fertility

Fibroids can impair fertility in several ways: alteration of local anatomy, including the detrimental effects of abnormal uterine bleeding; functional changes by increasing uterine contractions and impairing endometrium and myometrial blood supply; and changes to the local hormonal environment that could impair egg/sperm transport, or embryo implantation (Hum Reprod Update. 2017;22:665-86).

Prior to consideration of surgery, saline infusion sonogram can determine the degree of impact on the endometrium, which is most applicable to the infertility patient, but can also allow guidance toward the appropriate surgical approach.
 

Treatment options – medical

Management of fibroids is based on a woman’s age, desire for fertility, symptoms, and location of the fibroid(s). Expectant observation of a woman with fibroids may be a reasonable approach, provided the lack of symptoms impairing QoL and of anemia. Typically, there is no change in fibroid size during the short term, considered less than 1 year. Regarding fertility, studies are heterogeneous so there is no definitive conclusion that fibroids impair natural fertility (Reprod Biomed Online. 2021;43:100-10). Spontaneous regression, defined by a reduction in fibroid volume of greater than 20%, has been noted to occur in 7.0% of fibroids (Curr Obstet Gynecol Rep. 2018;7[3]:117-21).

When fertility is not desired, medical management of fibroids is the initial conservative approach. GnRH agonists have been utilized for temporary relief of menometrorrhagia because of fibroids and to reduce their volume, particularly preoperatively. However, extended treatment can induce bone mineral density loss. Add-back therapy (tibolone, raloxifene, estriol, and ipriflavone) is of value in reducing bone loss while MPA and tibolone may manage vasomotor symptoms. More recently, the use of a GnRH antagonist (elagolix) along with add-back therapy has been approved for up to 24 months by the Food and Drug Administration and has demonstrated a more than 50% amenorrhea rate at 12 months (Obstet Gynecol. 2020;135:1313-26).

Progesterone plays an important role in fibroid growth, but the mechanism is unclear. Although not FDA approved, selective progesterone receptor modulators (SPRM) act directly on fibroid size reduction at the level of the pituitary to induce amenorrhea through inhibition of ovulation. Also, more than one course of SPRMs can provide benefit for bleeding control and volume reduction. The SPRM ulipristal acetate for four courses of 3 months demonstrated 73.5% of patients experienced a fibroid volume reduction of greater than 25% and were amenorrheic (Fertil Steril. 2017;108:416-25). GnRH agonists or SPRMs may benefit women if the fibroid is larger than 3 cm or anemia exists, thereby precluding immediate surgery.

Other medication options include the levonorgestrel IUD, combined hormonal contraceptives, and tranexamic acid – all of which have limited data on effective results of treating abnormal uterine bleeding.
 

Treatment options – surgical

Fibroids are the most common reason for hysterectomy as they are the contributing indication in approximately one-third of surgeries. When future fertility is desired, current surgical options include hysteroscopic and laparoscopic (including robotic) myomectomy. Hysteroscopy is the standard approach for FIGO type 1 fibroids and can also manage some type 2 fibroids provided they are less than 3 cm and the latter is greater than 5 mm from the serosa. Type 2 fibroids may benefit from a “two-step” removal to allow the myometrium to contract and extrude the fibroid. In light of the risk of fluid overload with nonelectrolyte solutions that enable the use of monopolar cautery, many procedures are now performed with bipolar cautery or morcellators.

Laparoscopy (including robotic) has outcomes similar to those of laparotomy although the risk of uterine rupture with the former requires careful attention to thorough closure of the myometrial defect. Robotic myomectomy has outcomes similar to those of standard laparoscopy with less blood loss, but operating times may be prolonged (Best Pract Res Clin Obstet Gynaecol. 2018;46:113-9).

The rate of myomectomy is reported to be 9.2 per 10,000 woman-years in Black women and 1.3 per 10,000 woman years in White women (Fertil Steril 2017;108;416-25). The rate of recurrence after myomectomy can be as great as 60% when patients are followed up to 5 years. Intramural fibroids greater than 2.85 cm and not distorting the uterine cavity may decrease in vitro fertilization (IVF) success (Fertil Steril 2014;101:716-21).
 

Noninvasive treatment modalities

Uterine artery embolization (UAE) is the most popular minimally invasive alternative to surgical myomectomy. Risks include postembolization syndrome (pain, fever, nausea, leukocytosis, and occasionally malaise), infection, and damage to fertility. Rarely, loss of ovarian function can occur, particularly in women above age 45. Because of the disruption of uterine blood flow, UAE increases the risk of accelerating ovarian aging and infertility as well as atrophic endometrium. In addition, pregnancy complications are increased including miscarriage, preterm labor, and postpartum hemorrhage. There is debate regarding the need for cesarean section at time of delivery given the potential for weakening of the uterine wall following UAE.

High-intensity focused ultrasound (HIFU) is guided by ultrasound or MRI and involves a high-energy-density ultrasound wave passing through the skin. The wave is absorbed and transformed into heat, causing the tissue protein to coagulate, and to be absorbed by the body. The procedure is scarless, carries a minimal risk of infection, and offers less pain compared with traditional approaches. However, HIFU is time consuming, and skin burns and unintentional tissue injury are a risk. A meta-analysis demonstrated improved symptoms of fibroids at 6 and 12 months (J Min Invasive Gynecol. 2021 in press).

Ultrasound-guided microwave ablation (MWA) uses an ablative electrode that is directly inserted into the target tissue via transcutaneous or transcervical approach via ultrasound guidance using microwave to produce heat for tissue coagulation necrosis. The advantages of MWA compared with HIFU and RFA are a higher tissue temperature, larger ablation volume, shorter operating time, less pain and no adverse major events (J Min Invasive Gynecol. 2021, in press).
 

Conclusion

The current literature cannot conclude that fibroids reduce the likelihood of achieving pregnancy with or without fertility treatment, based on a specific size, number, or location (not including submucosal or cavity-distorting intramural fibroids). Definitive evidence on the efficacy of myomectomy to improve fertility remains limited. Hysteroscopic myomectomy presumably improves pregnancy rates, but there is uncertainty as to its role in reducing miscarriage. Novel nonsurgical modalities are available and are expected to continue being developed but clarity on fertility outcomes is needed.
 

Dr. Trolice is director of Fertility CARE – The IVF Center in Winter Park, Fla., and professor of obstetrics and gynecology at the University of Central Florida, Orlando. He has no conflicts of interests. Please contact him at obnews@mdedge.com.

Two chronic gynecologic conditions notably affect a woman’s quality of life (QoL), including fertility – one is endometriosis, and the other is a fibroid uterus. For a benign tumor, fibroids have an impressive prevalence found in approximately 50%-60% of women during their reproductive years. By menopause, it is estimated that 70% of woman have a fibroid, yet the true incidence is unknown given that only 25% of women experience symptoms bothersome enough to warrant intervention. This month’s article reviews the burden of fibroids and the latest management options that may potentially avoid surgery.

Background

Fibroids are monoclonal tumors of uterine smooth muscle that originate from the myometrium. Risk factors include family history, being premenopausal, increasing time since last delivery, obesity, and hypertension (ACOG Practice Bulletin no. 228 Jun 2021: Obstet Gynecol. 2021 Jun 1;137[6]:e100-e15) but oral hormonal contraception, depot medroxyprogesterone acetate (MPA), and increased parity reduce the risk of fibroids. Compared with White women, Black women have a 2-3 times higher prevalence of fibroids, develop them at a younger age, and present with larger fibroids.

The FIGO leiomyoma classification is the agreed upon system for identifying fibroid location. Symptoms are all too familiar to gynecologists, with life-threatening hemorrhage with severe anemia being the most feared, particularly for FIGO types 1-5. Transvaginal ultrasound is the simplest imaging tool for evaluation.

Adapted from Int J Gynecol Obstet. 2011;113:3-13.

Fibroids and fertility

Fibroids can impair fertility in several ways: alteration of local anatomy, including the detrimental effects of abnormal uterine bleeding; functional changes by increasing uterine contractions and impairing endometrium and myometrial blood supply; and changes to the local hormonal environment that could impair egg/sperm transport, or embryo implantation (Hum Reprod Update. 2017;22:665-86).

Prior to consideration of surgery, saline infusion sonogram can determine the degree of impact on the endometrium, which is most applicable to the infertility patient, but can also allow guidance toward the appropriate surgical approach.
 

Treatment options – medical

Management of fibroids is based on a woman’s age, desire for fertility, symptoms, and location of the fibroid(s). Expectant observation of a woman with fibroids may be a reasonable approach, provided the lack of symptoms impairing QoL and of anemia. Typically, there is no change in fibroid size during the short term, considered less than 1 year. Regarding fertility, studies are heterogeneous so there is no definitive conclusion that fibroids impair natural fertility (Reprod Biomed Online. 2021;43:100-10). Spontaneous regression, defined by a reduction in fibroid volume of greater than 20%, has been noted to occur in 7.0% of fibroids (Curr Obstet Gynecol Rep. 2018;7[3]:117-21).

When fertility is not desired, medical management of fibroids is the initial conservative approach. GnRH agonists have been utilized for temporary relief of menometrorrhagia because of fibroids and to reduce their volume, particularly preoperatively. However, extended treatment can induce bone mineral density loss. Add-back therapy (tibolone, raloxifene, estriol, and ipriflavone) is of value in reducing bone loss while MPA and tibolone may manage vasomotor symptoms. More recently, the use of a GnRH antagonist (elagolix) along with add-back therapy has been approved for up to 24 months by the Food and Drug Administration and has demonstrated a more than 50% amenorrhea rate at 12 months (Obstet Gynecol. 2020;135:1313-26).

Progesterone plays an important role in fibroid growth, but the mechanism is unclear. Although not FDA approved, selective progesterone receptor modulators (SPRM) act directly on fibroid size reduction at the level of the pituitary to induce amenorrhea through inhibition of ovulation. Also, more than one course of SPRMs can provide benefit for bleeding control and volume reduction. The SPRM ulipristal acetate for four courses of 3 months demonstrated 73.5% of patients experienced a fibroid volume reduction of greater than 25% and were amenorrheic (Fertil Steril. 2017;108:416-25). GnRH agonists or SPRMs may benefit women if the fibroid is larger than 3 cm or anemia exists, thereby precluding immediate surgery.

Other medication options include the levonorgestrel IUD, combined hormonal contraceptives, and tranexamic acid – all of which have limited data on effective results of treating abnormal uterine bleeding.
 

Treatment options – surgical

Fibroids are the most common reason for hysterectomy as they are the contributing indication in approximately one-third of surgeries. When future fertility is desired, current surgical options include hysteroscopic and laparoscopic (including robotic) myomectomy. Hysteroscopy is the standard approach for FIGO type 1 fibroids and can also manage some type 2 fibroids provided they are less than 3 cm and the latter is greater than 5 mm from the serosa. Type 2 fibroids may benefit from a “two-step” removal to allow the myometrium to contract and extrude the fibroid. In light of the risk of fluid overload with nonelectrolyte solutions that enable the use of monopolar cautery, many procedures are now performed with bipolar cautery or morcellators.

Laparoscopy (including robotic) has outcomes similar to those of laparotomy although the risk of uterine rupture with the former requires careful attention to thorough closure of the myometrial defect. Robotic myomectomy has outcomes similar to those of standard laparoscopy with less blood loss, but operating times may be prolonged (Best Pract Res Clin Obstet Gynaecol. 2018;46:113-9).

The rate of myomectomy is reported to be 9.2 per 10,000 woman-years in Black women and 1.3 per 10,000 woman years in White women (Fertil Steril 2017;108;416-25). The rate of recurrence after myomectomy can be as great as 60% when patients are followed up to 5 years. Intramural fibroids greater than 2.85 cm and not distorting the uterine cavity may decrease in vitro fertilization (IVF) success (Fertil Steril 2014;101:716-21).
 

Noninvasive treatment modalities

Uterine artery embolization (UAE) is the most popular minimally invasive alternative to surgical myomectomy. Risks include postembolization syndrome (pain, fever, nausea, leukocytosis, and occasionally malaise), infection, and damage to fertility. Rarely, loss of ovarian function can occur, particularly in women above age 45. Because of the disruption of uterine blood flow, UAE increases the risk of accelerating ovarian aging and infertility as well as atrophic endometrium. In addition, pregnancy complications are increased including miscarriage, preterm labor, and postpartum hemorrhage. There is debate regarding the need for cesarean section at time of delivery given the potential for weakening of the uterine wall following UAE.

High-intensity focused ultrasound (HIFU) is guided by ultrasound or MRI and involves a high-energy-density ultrasound wave passing through the skin. The wave is absorbed and transformed into heat, causing the tissue protein to coagulate, and to be absorbed by the body. The procedure is scarless, carries a minimal risk of infection, and offers less pain compared with traditional approaches. However, HIFU is time consuming, and skin burns and unintentional tissue injury are a risk. A meta-analysis demonstrated improved symptoms of fibroids at 6 and 12 months (J Min Invasive Gynecol. 2021 in press).

Ultrasound-guided microwave ablation (MWA) uses an ablative electrode that is directly inserted into the target tissue via transcutaneous or transcervical approach via ultrasound guidance using microwave to produce heat for tissue coagulation necrosis. The advantages of MWA compared with HIFU and RFA are a higher tissue temperature, larger ablation volume, shorter operating time, less pain and no adverse major events (J Min Invasive Gynecol. 2021, in press).
 

Conclusion

The current literature cannot conclude that fibroids reduce the likelihood of achieving pregnancy with or without fertility treatment, based on a specific size, number, or location (not including submucosal or cavity-distorting intramural fibroids). Definitive evidence on the efficacy of myomectomy to improve fertility remains limited. Hysteroscopic myomectomy presumably improves pregnancy rates, but there is uncertainty as to its role in reducing miscarriage. Novel nonsurgical modalities are available and are expected to continue being developed but clarity on fertility outcomes is needed.
 

Dr. Trolice is director of Fertility CARE – The IVF Center in Winter Park, Fla., and professor of obstetrics and gynecology at the University of Central Florida, Orlando. He has no conflicts of interests. Please contact him at obnews@mdedge.com.

Two chronic gynecologic conditions notably affect a woman’s quality of life (QoL), including fertility – one is endometriosis, and the other is a fibroid uterus. For a benign tumor, fibroids have an impressive prevalence found in approximately 50%-60% of women during their reproductive years. By menopause, it is estimated that 70% of woman have a fibroid, yet the true incidence is unknown given that only 25% of women experience symptoms bothersome enough to warrant intervention. This month’s article reviews the burden of fibroids and the latest management options that may potentially avoid surgery.

Background

Fibroids are monoclonal tumors of uterine smooth muscle that originate from the myometrium. Risk factors include family history, being premenopausal, increasing time since last delivery, obesity, and hypertension (ACOG Practice Bulletin no. 228 Jun 2021: Obstet Gynecol. 2021 Jun 1;137[6]:e100-e15) but oral hormonal contraception, depot medroxyprogesterone acetate (MPA), and increased parity reduce the risk of fibroids. Compared with White women, Black women have a 2-3 times higher prevalence of fibroids, develop them at a younger age, and present with larger fibroids.

The FIGO leiomyoma classification is the agreed upon system for identifying fibroid location. Symptoms are all too familiar to gynecologists, with life-threatening hemorrhage with severe anemia being the most feared, particularly for FIGO types 1-5. Transvaginal ultrasound is the simplest imaging tool for evaluation.

Adapted from Int J Gynecol Obstet. 2011;113:3-13.

Fibroids and fertility

Fibroids can impair fertility in several ways: alteration of local anatomy, including the detrimental effects of abnormal uterine bleeding; functional changes by increasing uterine contractions and impairing endometrium and myometrial blood supply; and changes to the local hormonal environment that could impair egg/sperm transport, or embryo implantation (Hum Reprod Update. 2017;22:665-86).

Prior to consideration of surgery, saline infusion sonogram can determine the degree of impact on the endometrium, which is most applicable to the infertility patient, but can also allow guidance toward the appropriate surgical approach.
 

Treatment options – medical

Management of fibroids is based on a woman’s age, desire for fertility, symptoms, and location of the fibroid(s). Expectant observation of a woman with fibroids may be a reasonable approach, provided the lack of symptoms impairing QoL and of anemia. Typically, there is no change in fibroid size during the short term, considered less than 1 year. Regarding fertility, studies are heterogeneous so there is no definitive conclusion that fibroids impair natural fertility (Reprod Biomed Online. 2021;43:100-10). Spontaneous regression, defined by a reduction in fibroid volume of greater than 20%, has been noted to occur in 7.0% of fibroids (Curr Obstet Gynecol Rep. 2018;7[3]:117-21).

When fertility is not desired, medical management of fibroids is the initial conservative approach. GnRH agonists have been utilized for temporary relief of menometrorrhagia because of fibroids and to reduce their volume, particularly preoperatively. However, extended treatment can induce bone mineral density loss. Add-back therapy (tibolone, raloxifene, estriol, and ipriflavone) is of value in reducing bone loss while MPA and tibolone may manage vasomotor symptoms. More recently, the use of a GnRH antagonist (elagolix) along with add-back therapy has been approved for up to 24 months by the Food and Drug Administration and has demonstrated a more than 50% amenorrhea rate at 12 months (Obstet Gynecol. 2020;135:1313-26).

Progesterone plays an important role in fibroid growth, but the mechanism is unclear. Although not FDA approved, selective progesterone receptor modulators (SPRM) act directly on fibroid size reduction at the level of the pituitary to induce amenorrhea through inhibition of ovulation. Also, more than one course of SPRMs can provide benefit for bleeding control and volume reduction. The SPRM ulipristal acetate for four courses of 3 months demonstrated 73.5% of patients experienced a fibroid volume reduction of greater than 25% and were amenorrheic (Fertil Steril. 2017;108:416-25). GnRH agonists or SPRMs may benefit women if the fibroid is larger than 3 cm or anemia exists, thereby precluding immediate surgery.

Other medication options include the levonorgestrel IUD, combined hormonal contraceptives, and tranexamic acid – all of which have limited data on effective results of treating abnormal uterine bleeding.
 

Treatment options – surgical

Fibroids are the most common reason for hysterectomy as they are the contributing indication in approximately one-third of surgeries. When future fertility is desired, current surgical options include hysteroscopic and laparoscopic (including robotic) myomectomy. Hysteroscopy is the standard approach for FIGO type 1 fibroids and can also manage some type 2 fibroids provided they are less than 3 cm and the latter is greater than 5 mm from the serosa. Type 2 fibroids may benefit from a “two-step” removal to allow the myometrium to contract and extrude the fibroid. In light of the risk of fluid overload with nonelectrolyte solutions that enable the use of monopolar cautery, many procedures are now performed with bipolar cautery or morcellators.

Laparoscopy (including robotic) has outcomes similar to those of laparotomy although the risk of uterine rupture with the former requires careful attention to thorough closure of the myometrial defect. Robotic myomectomy has outcomes similar to those of standard laparoscopy with less blood loss, but operating times may be prolonged (Best Pract Res Clin Obstet Gynaecol. 2018;46:113-9).

The rate of myomectomy is reported to be 9.2 per 10,000 woman-years in Black women and 1.3 per 10,000 woman years in White women (Fertil Steril 2017;108;416-25). The rate of recurrence after myomectomy can be as great as 60% when patients are followed up to 5 years. Intramural fibroids greater than 2.85 cm and not distorting the uterine cavity may decrease in vitro fertilization (IVF) success (Fertil Steril 2014;101:716-21).
 

Noninvasive treatment modalities

Uterine artery embolization (UAE) is the most popular minimally invasive alternative to surgical myomectomy. Risks include postembolization syndrome (pain, fever, nausea, leukocytosis, and occasionally malaise), infection, and damage to fertility. Rarely, loss of ovarian function can occur, particularly in women above age 45. Because of the disruption of uterine blood flow, UAE increases the risk of accelerating ovarian aging and infertility as well as atrophic endometrium. In addition, pregnancy complications are increased including miscarriage, preterm labor, and postpartum hemorrhage. There is debate regarding the need for cesarean section at time of delivery given the potential for weakening of the uterine wall following UAE.

High-intensity focused ultrasound (HIFU) is guided by ultrasound or MRI and involves a high-energy-density ultrasound wave passing through the skin. The wave is absorbed and transformed into heat, causing the tissue protein to coagulate, and to be absorbed by the body. The procedure is scarless, carries a minimal risk of infection, and offers less pain compared with traditional approaches. However, HIFU is time consuming, and skin burns and unintentional tissue injury are a risk. A meta-analysis demonstrated improved symptoms of fibroids at 6 and 12 months (J Min Invasive Gynecol. 2021 in press).

Ultrasound-guided microwave ablation (MWA) uses an ablative electrode that is directly inserted into the target tissue via transcutaneous or transcervical approach via ultrasound guidance using microwave to produce heat for tissue coagulation necrosis. The advantages of MWA compared with HIFU and RFA are a higher tissue temperature, larger ablation volume, shorter operating time, less pain and no adverse major events (J Min Invasive Gynecol. 2021, in press).
 

Conclusion

The current literature cannot conclude that fibroids reduce the likelihood of achieving pregnancy with or without fertility treatment, based on a specific size, number, or location (not including submucosal or cavity-distorting intramural fibroids). Definitive evidence on the efficacy of myomectomy to improve fertility remains limited. Hysteroscopic myomectomy presumably improves pregnancy rates, but there is uncertainty as to its role in reducing miscarriage. Novel nonsurgical modalities are available and are expected to continue being developed but clarity on fertility outcomes is needed.
 

Dr. Trolice is director of Fertility CARE – The IVF Center in Winter Park, Fla., and professor of obstetrics and gynecology at the University of Central Florida, Orlando. He has no conflicts of interests. Please contact him at obnews@mdedge.com.

An experimental gene therapy produced marked clinical improvement in children with aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disorder that affects the synthesis of key neurotransmitters to cause severe developmental and motor disability.

In an article published July 12, 2021, in Nature Communications, a group of researchers based at the University of California, San Francisco, and Ohio State University, Columbus, described results from seven children ages 4-9 with AADC deficiency who underwent a novel form of surgery to deliver a viral vector expressing the human AADC gene to the midbrain.

Previous trials of this gene therapy in children with AADC deficiency targeted a different region of the brain, the putamen, with only slight clinical improvement. Here, investigators chose two midbrain regions – the substantia nigra pars compacta and the ventral tegmental area – in the hope of restoring healthy AADC enzyme activity in those neurons.

The study’s corresponding author, Krystof Bankiewicz, MD, PhD, professor and vice chair of research at Ohio State University, director of the Brain Health and Performance Center at Ohio State University, and professor emeritus and vice chair for research at UCSF, said in an interview that the brain regions chosen for this trial resulted from years of efforts to identify an ideal target in this disease.

“This particular vector undergoes axonal transport,” he said. “If you inject it into specific regions of the brain it will be transported into the terminals [of the nerve fibers]. And by looking at the imaging of these patients, we found that they still have the wiring in the brain that’s so critical. So we decided to aim at a much more difficult target, going directly to the source of the problem, which is the substantia nigra and the ventral tegmental area. This targets two critical pathways in the brain: one that drives motor responses and another that controls emotions.”
 

‘Surprising’ improvement seen

The children in the study – four girls and three boys – underwent surgery from 2016 to the end of 2018, and were divided into two dose cohorts, with one receiving three times the amount of vector as the other. Both groups, however, saw similar levels of improvement.

All but one child saw complete resolution of a hallmark symptom of the disease – oculogyric crises, or prolonged spasms of muscles controlling eye movement – within 3 months of surgery. Of the children followed at least 18 months, six attained head control within a year, two became able to eat and drink by mouth, and four gained the ability to sit up unaided in that time. At 18 months one child had learned to speak 50 words using an augmentative communication device.

One child died unexpectedly 7 months after the procedure, Dr. Bankiewicz said in an interview. This death appeared to be caused by cardiac complications of his disease, Dr. Bankiewicz said, which are common in AADC deficiency.

While the investigators are now looking at delivering the AADC gene therapy in younger children – who were excluded from this trial because of safety concerns surrounding the complex procedure – investigators were surprised by the level of improvement seen in older children.

“We initially didn’t believe – at least not all of us – that we could actually make an impact in the older patients, and that is not the case,” said Dr. Bankiewicz, who has since used the same gene therapy on a compassionate-use basis in Europe and seen durable clinical improvement in patients as old as 26. “The fact that we saw a response in that patient tells us something about how incredibly plastic the brain is.”

While the new study does not detail improvements in the children’s social and emotional well-being, Dr. Bankiewicz said these, too, were pronounced. “Kids fall into oculogyric crises in stress-inducing situation. They might be in a stroller being taken for a walk, and something in the environment would stress them. Sometimes they had to be kept in a dark room isolated from stress.” Following the gene therapy, “they’re laughing, they’re social, they can interact with their environment. It’s really touching to see them able to develop a bond now with their caregivers.”
 

Implication for other disorders

Dr. Bankiewicz and colleagues have previously used the same gene to boost AADC activity in patients with Parkinson’s disease. The group is also in trials to deliver a neuroprotective gene to the brains of people with early-stage Alzheimer’s disease, and a gene-silencing therapy in patients with Huntington’s disease. They will also continue recruiting pediatric patients for trials of the AADC gene therapy.

“We have been developing a method for safely treating younger children, so now we will go to 3 years old and maybe even below,” Dr. Bankiewicz said. “Earlier is probably better, but for technical and safety considerations we needed to be conservative first. It is hugely stressful to go into very sick patients with that type of therapy in that part of the brain. We had to get it right the first time, and it looks like we did.”

The study was funded by the National Institutes of Health, the AADC Research Trust, the Pediatric Neurotransmitter Disease Association, and Ohio State University, with materials and technical support donated by ClearPoint Neuro. Several coauthors disclosed financial relationships with producers of diagnostic tests or biotechnology firms. Dr. Bankiewicz is a founder and shareholder of Brain Neurotherapy Bio, a company that develops gene therapies for Parkinson’s and other diseases.


 

An experimental gene therapy produced marked clinical improvement in children with aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disorder that affects the synthesis of key neurotransmitters to cause severe developmental and motor disability.

In an article published July 12, 2021, in Nature Communications, a group of researchers based at the University of California, San Francisco, and Ohio State University, Columbus, described results from seven children ages 4-9 with AADC deficiency who underwent a novel form of surgery to deliver a viral vector expressing the human AADC gene to the midbrain.

Previous trials of this gene therapy in children with AADC deficiency targeted a different region of the brain, the putamen, with only slight clinical improvement. Here, investigators chose two midbrain regions – the substantia nigra pars compacta and the ventral tegmental area – in the hope of restoring healthy AADC enzyme activity in those neurons.

The study’s corresponding author, Krystof Bankiewicz, MD, PhD, professor and vice chair of research at Ohio State University, director of the Brain Health and Performance Center at Ohio State University, and professor emeritus and vice chair for research at UCSF, said in an interview that the brain regions chosen for this trial resulted from years of efforts to identify an ideal target in this disease.

“This particular vector undergoes axonal transport,” he said. “If you inject it into specific regions of the brain it will be transported into the terminals [of the nerve fibers]. And by looking at the imaging of these patients, we found that they still have the wiring in the brain that’s so critical. So we decided to aim at a much more difficult target, going directly to the source of the problem, which is the substantia nigra and the ventral tegmental area. This targets two critical pathways in the brain: one that drives motor responses and another that controls emotions.”
 

‘Surprising’ improvement seen

The children in the study – four girls and three boys – underwent surgery from 2016 to the end of 2018, and were divided into two dose cohorts, with one receiving three times the amount of vector as the other. Both groups, however, saw similar levels of improvement.

All but one child saw complete resolution of a hallmark symptom of the disease – oculogyric crises, or prolonged spasms of muscles controlling eye movement – within 3 months of surgery. Of the children followed at least 18 months, six attained head control within a year, two became able to eat and drink by mouth, and four gained the ability to sit up unaided in that time. At 18 months one child had learned to speak 50 words using an augmentative communication device.

One child died unexpectedly 7 months after the procedure, Dr. Bankiewicz said in an interview. This death appeared to be caused by cardiac complications of his disease, Dr. Bankiewicz said, which are common in AADC deficiency.

While the investigators are now looking at delivering the AADC gene therapy in younger children – who were excluded from this trial because of safety concerns surrounding the complex procedure – investigators were surprised by the level of improvement seen in older children.

“We initially didn’t believe – at least not all of us – that we could actually make an impact in the older patients, and that is not the case,” said Dr. Bankiewicz, who has since used the same gene therapy on a compassionate-use basis in Europe and seen durable clinical improvement in patients as old as 26. “The fact that we saw a response in that patient tells us something about how incredibly plastic the brain is.”

While the new study does not detail improvements in the children’s social and emotional well-being, Dr. Bankiewicz said these, too, were pronounced. “Kids fall into oculogyric crises in stress-inducing situation. They might be in a stroller being taken for a walk, and something in the environment would stress them. Sometimes they had to be kept in a dark room isolated from stress.” Following the gene therapy, “they’re laughing, they’re social, they can interact with their environment. It’s really touching to see them able to develop a bond now with their caregivers.”
 

Implication for other disorders

Dr. Bankiewicz and colleagues have previously used the same gene to boost AADC activity in patients with Parkinson’s disease. The group is also in trials to deliver a neuroprotective gene to the brains of people with early-stage Alzheimer’s disease, and a gene-silencing therapy in patients with Huntington’s disease. They will also continue recruiting pediatric patients for trials of the AADC gene therapy.

“We have been developing a method for safely treating younger children, so now we will go to 3 years old and maybe even below,” Dr. Bankiewicz said. “Earlier is probably better, but for technical and safety considerations we needed to be conservative first. It is hugely stressful to go into very sick patients with that type of therapy in that part of the brain. We had to get it right the first time, and it looks like we did.”

The study was funded by the National Institutes of Health, the AADC Research Trust, the Pediatric Neurotransmitter Disease Association, and Ohio State University, with materials and technical support donated by ClearPoint Neuro. Several coauthors disclosed financial relationships with producers of diagnostic tests or biotechnology firms. Dr. Bankiewicz is a founder and shareholder of Brain Neurotherapy Bio, a company that develops gene therapies for Parkinson’s and other diseases.


 

An experimental gene therapy produced marked clinical improvement in children with aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic disorder that affects the synthesis of key neurotransmitters to cause severe developmental and motor disability.

In an article published July 12, 2021, in Nature Communications, a group of researchers based at the University of California, San Francisco, and Ohio State University, Columbus, described results from seven children ages 4-9 with AADC deficiency who underwent a novel form of surgery to deliver a viral vector expressing the human AADC gene to the midbrain.

Previous trials of this gene therapy in children with AADC deficiency targeted a different region of the brain, the putamen, with only slight clinical improvement. Here, investigators chose two midbrain regions – the substantia nigra pars compacta and the ventral tegmental area – in the hope of restoring healthy AADC enzyme activity in those neurons.

The study’s corresponding author, Krystof Bankiewicz, MD, PhD, professor and vice chair of research at Ohio State University, director of the Brain Health and Performance Center at Ohio State University, and professor emeritus and vice chair for research at UCSF, said in an interview that the brain regions chosen for this trial resulted from years of efforts to identify an ideal target in this disease.

“This particular vector undergoes axonal transport,” he said. “If you inject it into specific regions of the brain it will be transported into the terminals [of the nerve fibers]. And by looking at the imaging of these patients, we found that they still have the wiring in the brain that’s so critical. So we decided to aim at a much more difficult target, going directly to the source of the problem, which is the substantia nigra and the ventral tegmental area. This targets two critical pathways in the brain: one that drives motor responses and another that controls emotions.”
 

‘Surprising’ improvement seen

The children in the study – four girls and three boys – underwent surgery from 2016 to the end of 2018, and were divided into two dose cohorts, with one receiving three times the amount of vector as the other. Both groups, however, saw similar levels of improvement.

All but one child saw complete resolution of a hallmark symptom of the disease – oculogyric crises, or prolonged spasms of muscles controlling eye movement – within 3 months of surgery. Of the children followed at least 18 months, six attained head control within a year, two became able to eat and drink by mouth, and four gained the ability to sit up unaided in that time. At 18 months one child had learned to speak 50 words using an augmentative communication device.

One child died unexpectedly 7 months after the procedure, Dr. Bankiewicz said in an interview. This death appeared to be caused by cardiac complications of his disease, Dr. Bankiewicz said, which are common in AADC deficiency.

While the investigators are now looking at delivering the AADC gene therapy in younger children – who were excluded from this trial because of safety concerns surrounding the complex procedure – investigators were surprised by the level of improvement seen in older children.

“We initially didn’t believe – at least not all of us – that we could actually make an impact in the older patients, and that is not the case,” said Dr. Bankiewicz, who has since used the same gene therapy on a compassionate-use basis in Europe and seen durable clinical improvement in patients as old as 26. “The fact that we saw a response in that patient tells us something about how incredibly plastic the brain is.”

While the new study does not detail improvements in the children’s social and emotional well-being, Dr. Bankiewicz said these, too, were pronounced. “Kids fall into oculogyric crises in stress-inducing situation. They might be in a stroller being taken for a walk, and something in the environment would stress them. Sometimes they had to be kept in a dark room isolated from stress.” Following the gene therapy, “they’re laughing, they’re social, they can interact with their environment. It’s really touching to see them able to develop a bond now with their caregivers.”
 

Implication for other disorders

Dr. Bankiewicz and colleagues have previously used the same gene to boost AADC activity in patients with Parkinson’s disease. The group is also in trials to deliver a neuroprotective gene to the brains of people with early-stage Alzheimer’s disease, and a gene-silencing therapy in patients with Huntington’s disease. They will also continue recruiting pediatric patients for trials of the AADC gene therapy.

“We have been developing a method for safely treating younger children, so now we will go to 3 years old and maybe even below,” Dr. Bankiewicz said. “Earlier is probably better, but for technical and safety considerations we needed to be conservative first. It is hugely stressful to go into very sick patients with that type of therapy in that part of the brain. We had to get it right the first time, and it looks like we did.”

The study was funded by the National Institutes of Health, the AADC Research Trust, the Pediatric Neurotransmitter Disease Association, and Ohio State University, with materials and technical support donated by ClearPoint Neuro. Several coauthors disclosed financial relationships with producers of diagnostic tests or biotechnology firms. Dr. Bankiewicz is a founder and shareholder of Brain Neurotherapy Bio, a company that develops gene therapies for Parkinson’s and other diseases.


 

Although prostate cancer is the second most common cancer in men worldwide, it is not a lethal cancer: Improvements in early detection and treatment have boosted the 10-year relative survival rate to 98%.

But for many men, life after treatment is an ongoing struggle.

One of the first qualitative survey studies of long-term prostate cancer survivorship in Australia found that many survivors are living with adverse effects such as urinary incontinence and sexual dysfunction and that they continue to have heightened feelings of distress.

Many of the patients surveyed said that they had not received follow-up care and felt they had been “abandoned” by their health care professionals.

One man called it “the survivorship abyss.”

“As the prevalence of prostate cancer survivors continues to grow globally, the absence of integrated shared survivorship care models into clinical practice, such as survivorship care guidelines/plans and/or interventions, will perpetuate the sense of abandonment and the overwhelming burden of care of men lost in the PC [prostate cancer] ‘survivorship abyss,’” say the authors.

The report was published online in Psycho-Oncology.

“The good news is that more men than ever before are surviving prostate cancer,” commented lead author Carolyn G. Mazariego, PhD, a research fellow at the Daffodil Center of the Cancer Council New South Wales and the University of Sydney.

“As our population grows and ages, an increasing number of men are facing these survivorship challenges, and the need for clear survivorship care guidelines becomes increasingly vital,” Dr. Mazariego told this news organization.
 

Details of the survey findings

To find participants for their study, the team drew upon a cohort of 578 men who were included in the 15-year follow-up phase of the longitudinal New South Wales Prostate Cancer Care and Outcomes Study (PCOS).

The researchers interviewed 37 men for the study. The majority (88.6%) had been diagnosed with localized disease, and just over half (54%) had undergone radical prostatectomy as their primary treatment.

Some expressed regret over having had surgery. One respondent, a 15-year survivor, commented: “I really didn’t know how intrusive [surgery] was as far as losing the length of my penis and its functions. To this day, I sometimes can’t believe it’s happened. It’s devastating.”

The survey also found that most survivors viewed active treatment as the only way to avoid death.

In hindsight, some questioned whether radical treatment was necessary and whether they might have been better served by active surveillance.

Many said they were never given a chance to discuss sexual dysfunction, and others said their questions sparked an awkward, limited conversation. Many said they suffered in silence.

“We know that there is a sort of ‘cycle of silence’ between patients and health care providers about sexual issues, and that was particularly true for the men we spoke to,” Dr. Mazariego said. “This cycle of silence can stem from confusion and ambiguity as to who men should speak with regarding these ongoing issues.”

“It’s just like being part of a secret society,” said one survivor. “Like you don’t know about it until you’re in it ... I don’t think people want to know if they have it [prostate cancer] or want it known. It’s all hush hush.”

Prostate cancer patients often feel uncomfortable talking about sexual function, Dr. Mazariego pointed out. “Many men instead internalized these thoughts or concerns and just ‘got on’ with life.”

One survey participant said: “Well, you just gotta deal with it [issues relating to prostate cancer]. Just got to try and wipe out the thoughts, that’s all you can do. Suck it up and carry on.”
 

Discussions must be part of standard of care

Dr. Mazariego emphasized that for prostate cancer survivorship to improve, conversations about the psychosocial needs of patients must be normalized and made routine. Discussions about post-treatment side effects that cause functional impairments and are stressful to relationships must be embedded into the standards of care, she added. This will allow physicians to use these kinds of conversations “as a springboard for referrals to appropriate care.”

“Patients of all ages will have concerns related to sex and sexual function, whether you ask about them or not,” said Brad Zebrack, PhD, MSW, MPH, professor at the University of Michigan School of Social Work, Ann Arbor, who was approached for comment.

At the very least, physicians should be prepared to “acknowledge and describe possible effects of therapy on sex and sexual function and follow up by describing available supports for education and either individual or couples counseling,” said Dr. Zebrack, who is also a member of the health behavior and outcomes research program at the Rogel Cancer Center.

Physicians can also refer patients to evidence-based resources and toolkits, he said. He noted that Will2Love offers self-help programs for cancer-related sexual problems and is “an outstanding resource.”
 

Distress screening needed

Universal psychosocial distress screening is needed to identify men with prostate cancer who have high levels of distress, argues Jeff Dunn, AO, PhD, who is CEO of the Prostate Cancer Foundation of Australia (PCFA), in Sydney.

Without this, men will not seek help for their unmet needs, Dr. Dunn noted in a 2019 editorial in the European Journal of Cancer Care.

The unmet psychosocial needs of prostate cancer patients and their partners “are highly prevalent,” said Dr. Dunn and co-author Suzanne K. Chambers, AO, PhD, dean of the Faculty of Health at the University of Technology Sydney. “...[F]or many prostate cancer survivors, the physical, social, psychological and relationship challenges will be long term, if not lifelong,” they write.

Even in Australia, where 1 in every 6 men are expected to be diagnosed with prostate cancer by age 85, efforts to improve survivorship care have not produced the desired results, they note.

In 2019, the PCFA published a monograph in which the organization recommended routine distress screenings and referrals to evidence-based psychosocial care for prostate cancer survivors. The foundation predicted that it would be “a game-changer for every Australian man impacted by the disease.”

“Yet, we still do not have a national survivorship care plan for prostate cancer survivors in Australia,” Dr. Mazariego said.

Left untreated, the psychosocial effects of prostate cancer “are considerable and certainly factors that should be considered as a threat to well-being,” she emphasized.

“Certainly, accumulation of unmet psychosocial needs increases risks for depression and suicide, which is true in both cancer and noncancer populations,” said Dr. Zebrack.

Data back this up. One study of patients with prostate cancer found that after diagnosis, 1 in 4 experienced anxiety, and 1 in 5 experienced depression. Another study found that the risk for suicide was higher among men with prostate cancer during the first year after diagnosis than among men with other solid-organ malignancies.

More recently, a 2018 population-based Australian cohort study of men in New South Wales revealed that the risk for death by suicide was 70% higher among men who had been diagnosed with prostate cancer compared with men in the general population. This risk was higher within the first year following diagnosis and was higher in men with nonlocalized disease, those who were single, those who were living in a major city, and those who were unmarried.

Dr. Mazariego and the team reporting the survey found that men who received supportive care had a greater chance of reconciling living with functional impairments. “Receiving adequate survivorship care and trusting patient-clinician relationships appeared to be associated with greater resilience and positivity in the men’s acceptance of cancer-related, long-term challenges and personal limitations,” they note.
 

Developing postcancer identity

Survivors also appeared to have developed a post-cancer identity that was more invested in personal relationships or in taking a more active role in their own health.

“Treating my prostate cancer gave me a second chance,” said one survivor. “Yeah, I’m a survivor, but more importantly, I’m a loving husband now. I know I wasn’t as giving back then.”

Another man admitted that before his diagnosis, he never went to the doctor. “I go much more often now,” he said. “You need to check up on yourself, at least once a year. Could have caught the cancer earlier if I was doing that.”

These comments appear to be borne out by data released last year by the American Cancer Society (ACS), which found that the number of number of cancer-related suicides in the United States was on the decline, as reported by this news organization.

Some of the biggest decreases occurred in men with prostate cancer, although prostate cancer was associated with 15% of cancer-related suicides. Only lung cancer was associated with more cancer-related suicides, at 18%.

A factor in the decrease in cancer-related suicides was likely to be an increase in the use of supportive care services, the ACS researchers commented.

“Although no causal relationship can be established, our findings suggest an evolving role of psycho-oncology care and palliative and hospice care given the promotion and increased utilization of these services among cancer patients during this period,” they commented.

The study was funded by the Cancer Institute NSW. Dr. Mazariego and colleagues have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Although prostate cancer is the second most common cancer in men worldwide, it is not a lethal cancer: Improvements in early detection and treatment have boosted the 10-year relative survival rate to 98%.

But for many men, life after treatment is an ongoing struggle.

One of the first qualitative survey studies of long-term prostate cancer survivorship in Australia found that many survivors are living with adverse effects such as urinary incontinence and sexual dysfunction and that they continue to have heightened feelings of distress.

Many of the patients surveyed said that they had not received follow-up care and felt they had been “abandoned” by their health care professionals.

One man called it “the survivorship abyss.”

“As the prevalence of prostate cancer survivors continues to grow globally, the absence of integrated shared survivorship care models into clinical practice, such as survivorship care guidelines/plans and/or interventions, will perpetuate the sense of abandonment and the overwhelming burden of care of men lost in the PC [prostate cancer] ‘survivorship abyss,’” say the authors.

The report was published online in Psycho-Oncology.

“The good news is that more men than ever before are surviving prostate cancer,” commented lead author Carolyn G. Mazariego, PhD, a research fellow at the Daffodil Center of the Cancer Council New South Wales and the University of Sydney.

“As our population grows and ages, an increasing number of men are facing these survivorship challenges, and the need for clear survivorship care guidelines becomes increasingly vital,” Dr. Mazariego told this news organization.
 

Details of the survey findings

To find participants for their study, the team drew upon a cohort of 578 men who were included in the 15-year follow-up phase of the longitudinal New South Wales Prostate Cancer Care and Outcomes Study (PCOS).

The researchers interviewed 37 men for the study. The majority (88.6%) had been diagnosed with localized disease, and just over half (54%) had undergone radical prostatectomy as their primary treatment.

Some expressed regret over having had surgery. One respondent, a 15-year survivor, commented: “I really didn’t know how intrusive [surgery] was as far as losing the length of my penis and its functions. To this day, I sometimes can’t believe it’s happened. It’s devastating.”

The survey also found that most survivors viewed active treatment as the only way to avoid death.

In hindsight, some questioned whether radical treatment was necessary and whether they might have been better served by active surveillance.

Many said they were never given a chance to discuss sexual dysfunction, and others said their questions sparked an awkward, limited conversation. Many said they suffered in silence.

“We know that there is a sort of ‘cycle of silence’ between patients and health care providers about sexual issues, and that was particularly true for the men we spoke to,” Dr. Mazariego said. “This cycle of silence can stem from confusion and ambiguity as to who men should speak with regarding these ongoing issues.”

“It’s just like being part of a secret society,” said one survivor. “Like you don’t know about it until you’re in it ... I don’t think people want to know if they have it [prostate cancer] or want it known. It’s all hush hush.”

Prostate cancer patients often feel uncomfortable talking about sexual function, Dr. Mazariego pointed out. “Many men instead internalized these thoughts or concerns and just ‘got on’ with life.”

One survey participant said: “Well, you just gotta deal with it [issues relating to prostate cancer]. Just got to try and wipe out the thoughts, that’s all you can do. Suck it up and carry on.”
 

Discussions must be part of standard of care

Dr. Mazariego emphasized that for prostate cancer survivorship to improve, conversations about the psychosocial needs of patients must be normalized and made routine. Discussions about post-treatment side effects that cause functional impairments and are stressful to relationships must be embedded into the standards of care, she added. This will allow physicians to use these kinds of conversations “as a springboard for referrals to appropriate care.”

“Patients of all ages will have concerns related to sex and sexual function, whether you ask about them or not,” said Brad Zebrack, PhD, MSW, MPH, professor at the University of Michigan School of Social Work, Ann Arbor, who was approached for comment.

At the very least, physicians should be prepared to “acknowledge and describe possible effects of therapy on sex and sexual function and follow up by describing available supports for education and either individual or couples counseling,” said Dr. Zebrack, who is also a member of the health behavior and outcomes research program at the Rogel Cancer Center.

Physicians can also refer patients to evidence-based resources and toolkits, he said. He noted that Will2Love offers self-help programs for cancer-related sexual problems and is “an outstanding resource.”
 

Distress screening needed

Universal psychosocial distress screening is needed to identify men with prostate cancer who have high levels of distress, argues Jeff Dunn, AO, PhD, who is CEO of the Prostate Cancer Foundation of Australia (PCFA), in Sydney.

Without this, men will not seek help for their unmet needs, Dr. Dunn noted in a 2019 editorial in the European Journal of Cancer Care.

The unmet psychosocial needs of prostate cancer patients and their partners “are highly prevalent,” said Dr. Dunn and co-author Suzanne K. Chambers, AO, PhD, dean of the Faculty of Health at the University of Technology Sydney. “...[F]or many prostate cancer survivors, the physical, social, psychological and relationship challenges will be long term, if not lifelong,” they write.

Even in Australia, where 1 in every 6 men are expected to be diagnosed with prostate cancer by age 85, efforts to improve survivorship care have not produced the desired results, they note.

In 2019, the PCFA published a monograph in which the organization recommended routine distress screenings and referrals to evidence-based psychosocial care for prostate cancer survivors. The foundation predicted that it would be “a game-changer for every Australian man impacted by the disease.”

“Yet, we still do not have a national survivorship care plan for prostate cancer survivors in Australia,” Dr. Mazariego said.

Left untreated, the psychosocial effects of prostate cancer “are considerable and certainly factors that should be considered as a threat to well-being,” she emphasized.

“Certainly, accumulation of unmet psychosocial needs increases risks for depression and suicide, which is true in both cancer and noncancer populations,” said Dr. Zebrack.

Data back this up. One study of patients with prostate cancer found that after diagnosis, 1 in 4 experienced anxiety, and 1 in 5 experienced depression. Another study found that the risk for suicide was higher among men with prostate cancer during the first year after diagnosis than among men with other solid-organ malignancies.

More recently, a 2018 population-based Australian cohort study of men in New South Wales revealed that the risk for death by suicide was 70% higher among men who had been diagnosed with prostate cancer compared with men in the general population. This risk was higher within the first year following diagnosis and was higher in men with nonlocalized disease, those who were single, those who were living in a major city, and those who were unmarried.

Dr. Mazariego and the team reporting the survey found that men who received supportive care had a greater chance of reconciling living with functional impairments. “Receiving adequate survivorship care and trusting patient-clinician relationships appeared to be associated with greater resilience and positivity in the men’s acceptance of cancer-related, long-term challenges and personal limitations,” they note.
 

Developing postcancer identity

Survivors also appeared to have developed a post-cancer identity that was more invested in personal relationships or in taking a more active role in their own health.

“Treating my prostate cancer gave me a second chance,” said one survivor. “Yeah, I’m a survivor, but more importantly, I’m a loving husband now. I know I wasn’t as giving back then.”

Another man admitted that before his diagnosis, he never went to the doctor. “I go much more often now,” he said. “You need to check up on yourself, at least once a year. Could have caught the cancer earlier if I was doing that.”

These comments appear to be borne out by data released last year by the American Cancer Society (ACS), which found that the number of number of cancer-related suicides in the United States was on the decline, as reported by this news organization.

Some of the biggest decreases occurred in men with prostate cancer, although prostate cancer was associated with 15% of cancer-related suicides. Only lung cancer was associated with more cancer-related suicides, at 18%.

A factor in the decrease in cancer-related suicides was likely to be an increase in the use of supportive care services, the ACS researchers commented.

“Although no causal relationship can be established, our findings suggest an evolving role of psycho-oncology care and palliative and hospice care given the promotion and increased utilization of these services among cancer patients during this period,” they commented.

The study was funded by the Cancer Institute NSW. Dr. Mazariego and colleagues have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Although prostate cancer is the second most common cancer in men worldwide, it is not a lethal cancer: Improvements in early detection and treatment have boosted the 10-year relative survival rate to 98%.

But for many men, life after treatment is an ongoing struggle.

One of the first qualitative survey studies of long-term prostate cancer survivorship in Australia found that many survivors are living with adverse effects such as urinary incontinence and sexual dysfunction and that they continue to have heightened feelings of distress.

Many of the patients surveyed said that they had not received follow-up care and felt they had been “abandoned” by their health care professionals.

One man called it “the survivorship abyss.”

“As the prevalence of prostate cancer survivors continues to grow globally, the absence of integrated shared survivorship care models into clinical practice, such as survivorship care guidelines/plans and/or interventions, will perpetuate the sense of abandonment and the overwhelming burden of care of men lost in the PC [prostate cancer] ‘survivorship abyss,’” say the authors.

The report was published online in Psycho-Oncology.

“The good news is that more men than ever before are surviving prostate cancer,” commented lead author Carolyn G. Mazariego, PhD, a research fellow at the Daffodil Center of the Cancer Council New South Wales and the University of Sydney.

“As our population grows and ages, an increasing number of men are facing these survivorship challenges, and the need for clear survivorship care guidelines becomes increasingly vital,” Dr. Mazariego told this news organization.
 

Details of the survey findings

To find participants for their study, the team drew upon a cohort of 578 men who were included in the 15-year follow-up phase of the longitudinal New South Wales Prostate Cancer Care and Outcomes Study (PCOS).

The researchers interviewed 37 men for the study. The majority (88.6%) had been diagnosed with localized disease, and just over half (54%) had undergone radical prostatectomy as their primary treatment.

Some expressed regret over having had surgery. One respondent, a 15-year survivor, commented: “I really didn’t know how intrusive [surgery] was as far as losing the length of my penis and its functions. To this day, I sometimes can’t believe it’s happened. It’s devastating.”

The survey also found that most survivors viewed active treatment as the only way to avoid death.

In hindsight, some questioned whether radical treatment was necessary and whether they might have been better served by active surveillance.

Many said they were never given a chance to discuss sexual dysfunction, and others said their questions sparked an awkward, limited conversation. Many said they suffered in silence.

“We know that there is a sort of ‘cycle of silence’ between patients and health care providers about sexual issues, and that was particularly true for the men we spoke to,” Dr. Mazariego said. “This cycle of silence can stem from confusion and ambiguity as to who men should speak with regarding these ongoing issues.”

“It’s just like being part of a secret society,” said one survivor. “Like you don’t know about it until you’re in it ... I don’t think people want to know if they have it [prostate cancer] or want it known. It’s all hush hush.”

Prostate cancer patients often feel uncomfortable talking about sexual function, Dr. Mazariego pointed out. “Many men instead internalized these thoughts or concerns and just ‘got on’ with life.”

One survey participant said: “Well, you just gotta deal with it [issues relating to prostate cancer]. Just got to try and wipe out the thoughts, that’s all you can do. Suck it up and carry on.”
 

Discussions must be part of standard of care

Dr. Mazariego emphasized that for prostate cancer survivorship to improve, conversations about the psychosocial needs of patients must be normalized and made routine. Discussions about post-treatment side effects that cause functional impairments and are stressful to relationships must be embedded into the standards of care, she added. This will allow physicians to use these kinds of conversations “as a springboard for referrals to appropriate care.”

“Patients of all ages will have concerns related to sex and sexual function, whether you ask about them or not,” said Brad Zebrack, PhD, MSW, MPH, professor at the University of Michigan School of Social Work, Ann Arbor, who was approached for comment.

At the very least, physicians should be prepared to “acknowledge and describe possible effects of therapy on sex and sexual function and follow up by describing available supports for education and either individual or couples counseling,” said Dr. Zebrack, who is also a member of the health behavior and outcomes research program at the Rogel Cancer Center.

Physicians can also refer patients to evidence-based resources and toolkits, he said. He noted that Will2Love offers self-help programs for cancer-related sexual problems and is “an outstanding resource.”
 

Distress screening needed

Universal psychosocial distress screening is needed to identify men with prostate cancer who have high levels of distress, argues Jeff Dunn, AO, PhD, who is CEO of the Prostate Cancer Foundation of Australia (PCFA), in Sydney.

Without this, men will not seek help for their unmet needs, Dr. Dunn noted in a 2019 editorial in the European Journal of Cancer Care.

The unmet psychosocial needs of prostate cancer patients and their partners “are highly prevalent,” said Dr. Dunn and co-author Suzanne K. Chambers, AO, PhD, dean of the Faculty of Health at the University of Technology Sydney. “...[F]or many prostate cancer survivors, the physical, social, psychological and relationship challenges will be long term, if not lifelong,” they write.

Even in Australia, where 1 in every 6 men are expected to be diagnosed with prostate cancer by age 85, efforts to improve survivorship care have not produced the desired results, they note.

In 2019, the PCFA published a monograph in which the organization recommended routine distress screenings and referrals to evidence-based psychosocial care for prostate cancer survivors. The foundation predicted that it would be “a game-changer for every Australian man impacted by the disease.”

“Yet, we still do not have a national survivorship care plan for prostate cancer survivors in Australia,” Dr. Mazariego said.

Left untreated, the psychosocial effects of prostate cancer “are considerable and certainly factors that should be considered as a threat to well-being,” she emphasized.

“Certainly, accumulation of unmet psychosocial needs increases risks for depression and suicide, which is true in both cancer and noncancer populations,” said Dr. Zebrack.

Data back this up. One study of patients with prostate cancer found that after diagnosis, 1 in 4 experienced anxiety, and 1 in 5 experienced depression. Another study found that the risk for suicide was higher among men with prostate cancer during the first year after diagnosis than among men with other solid-organ malignancies.

More recently, a 2018 population-based Australian cohort study of men in New South Wales revealed that the risk for death by suicide was 70% higher among men who had been diagnosed with prostate cancer compared with men in the general population. This risk was higher within the first year following diagnosis and was higher in men with nonlocalized disease, those who were single, those who were living in a major city, and those who were unmarried.

Dr. Mazariego and the team reporting the survey found that men who received supportive care had a greater chance of reconciling living with functional impairments. “Receiving adequate survivorship care and trusting patient-clinician relationships appeared to be associated with greater resilience and positivity in the men’s acceptance of cancer-related, long-term challenges and personal limitations,” they note.
 

Developing postcancer identity

Survivors also appeared to have developed a post-cancer identity that was more invested in personal relationships or in taking a more active role in their own health.

“Treating my prostate cancer gave me a second chance,” said one survivor. “Yeah, I’m a survivor, but more importantly, I’m a loving husband now. I know I wasn’t as giving back then.”

Another man admitted that before his diagnosis, he never went to the doctor. “I go much more often now,” he said. “You need to check up on yourself, at least once a year. Could have caught the cancer earlier if I was doing that.”

These comments appear to be borne out by data released last year by the American Cancer Society (ACS), which found that the number of number of cancer-related suicides in the United States was on the decline, as reported by this news organization.

Some of the biggest decreases occurred in men with prostate cancer, although prostate cancer was associated with 15% of cancer-related suicides. Only lung cancer was associated with more cancer-related suicides, at 18%.

A factor in the decrease in cancer-related suicides was likely to be an increase in the use of supportive care services, the ACS researchers commented.

“Although no causal relationship can be established, our findings suggest an evolving role of psycho-oncology care and palliative and hospice care given the promotion and increased utilization of these services among cancer patients during this period,” they commented.

The study was funded by the Cancer Institute NSW. Dr. Mazariego and colleagues have disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

The U.S. Food and Drug Administration has approved odevixibat (Bylvay, Albireo Pharma), the first treatment for pruritus associated with all types of progressive familial intrahepatic cholestasis (PFIC).

PFIC is a rare disorder affecting an estimated one to two people per 100,000. The disorder usually appears within the first few months of life and causes progressive, life-threatening liver disease, often leading to cirrhosis and liver failure before age 10.

In PFIC, liver cells are unable to drain bile acids, leading to the buildup of toxic substances in the liver. While the precise cause of severe itching in patients with PFIC is unknown, it may involve increased levels of bile acids in the body and skin.

Odevixibat is a potent, nonsystemic ileal bile acid transport inhibitor that does not need refrigeration and is given as a once-daily capsule or opened and sprinkled onto soft foods, the company said in a news release announcing the approval.

There are at least three types of PFIC; all are inherited genetic conditions caused by gene mutations. Odevixibat is indicated to treat all subtypes.

“Treating children with PFIC can be difficult and frustrating given the current treatment options. Bylvay gives us a nonsurgical option and will change how we treat PFIC,” Richard Thompson, MD, principal investigator for the two trials that led to the approval, said in the news release.

“With this approval, my colleagues and I now have the opportunity to revisit how PFIC patients are being managed, and we are hopeful for better outcomes for these children,” said Dr. Thompson, professor of molecular hepatology at King’s College London.

The approval of odevixibat was supported by data from the PEDFIC 1 and PEDFIC 2 trials.

PEDFIC 1 enrolled 62 children with PFIC and severe itching, with 20 assigned to placebo and 42 to odevixibat, given once daily with a meal in the morning. Odevixibat met both of its primary endpoints, with the drug improving pruritus (P = .004) and reducing serum bile acid responses (P = .003).

In PEDFIC 2, a long-term, open-label extension study, the effects of odevixibat on pruritis and serum bile acids were sustained up to 48 weeks.

Odevixibat was well tolerated in both trials, with the most common treatment-related gastrointestinal adverse events being diarrhea/frequent stools. There were no serious treatment-related adverse events.

Children taking the drug should undergo liver test monitoring periodically during treatment, the FDA said when announcing the approval. Odevixibat may affect absorption of fat-soluble vitamins such as A, D, E, and K. Patients should be monitored for fat-soluble vitamin deficiency while taking the drug.

Full prescribing information is available online.

“Until now, invasive surgery was the only approved treatment option. With the approval of Bylvay, parents may find hope in having a less invasive treatment option available,” Emily Ventura, leader of the PFIC Advocacy and Resource Network and mother to a child with PFIC, said in the news release.

The company said it will launch odevixibat “immediately” to accelerate availability for patients and families affected by PFIC.

Odevixibat is also being studied in other rare pediatric cholestatic liver diseases, including biliary atresia and Alagille syndrome.

A version of this article first appeared on Medscape.com.

The U.S. Food and Drug Administration has approved odevixibat (Bylvay, Albireo Pharma), the first treatment for pruritus associated with all types of progressive familial intrahepatic cholestasis (PFIC).

PFIC is a rare disorder affecting an estimated one to two people per 100,000. The disorder usually appears within the first few months of life and causes progressive, life-threatening liver disease, often leading to cirrhosis and liver failure before age 10.

In PFIC, liver cells are unable to drain bile acids, leading to the buildup of toxic substances in the liver. While the precise cause of severe itching in patients with PFIC is unknown, it may involve increased levels of bile acids in the body and skin.

Odevixibat is a potent, nonsystemic ileal bile acid transport inhibitor that does not need refrigeration and is given as a once-daily capsule or opened and sprinkled onto soft foods, the company said in a news release announcing the approval.

There are at least three types of PFIC; all are inherited genetic conditions caused by gene mutations. Odevixibat is indicated to treat all subtypes.

“Treating children with PFIC can be difficult and frustrating given the current treatment options. Bylvay gives us a nonsurgical option and will change how we treat PFIC,” Richard Thompson, MD, principal investigator for the two trials that led to the approval, said in the news release.

“With this approval, my colleagues and I now have the opportunity to revisit how PFIC patients are being managed, and we are hopeful for better outcomes for these children,” said Dr. Thompson, professor of molecular hepatology at King’s College London.

The approval of odevixibat was supported by data from the PEDFIC 1 and PEDFIC 2 trials.

PEDFIC 1 enrolled 62 children with PFIC and severe itching, with 20 assigned to placebo and 42 to odevixibat, given once daily with a meal in the morning. Odevixibat met both of its primary endpoints, with the drug improving pruritus (P = .004) and reducing serum bile acid responses (P = .003).

In PEDFIC 2, a long-term, open-label extension study, the effects of odevixibat on pruritis and serum bile acids were sustained up to 48 weeks.

Odevixibat was well tolerated in both trials, with the most common treatment-related gastrointestinal adverse events being diarrhea/frequent stools. There were no serious treatment-related adverse events.

Children taking the drug should undergo liver test monitoring periodically during treatment, the FDA said when announcing the approval. Odevixibat may affect absorption of fat-soluble vitamins such as A, D, E, and K. Patients should be monitored for fat-soluble vitamin deficiency while taking the drug.

Full prescribing information is available online.

“Until now, invasive surgery was the only approved treatment option. With the approval of Bylvay, parents may find hope in having a less invasive treatment option available,” Emily Ventura, leader of the PFIC Advocacy and Resource Network and mother to a child with PFIC, said in the news release.

The company said it will launch odevixibat “immediately” to accelerate availability for patients and families affected by PFIC.

Odevixibat is also being studied in other rare pediatric cholestatic liver diseases, including biliary atresia and Alagille syndrome.

A version of this article first appeared on Medscape.com.

The U.S. Food and Drug Administration has approved odevixibat (Bylvay, Albireo Pharma), the first treatment for pruritus associated with all types of progressive familial intrahepatic cholestasis (PFIC).

PFIC is a rare disorder affecting an estimated one to two people per 100,000. The disorder usually appears within the first few months of life and causes progressive, life-threatening liver disease, often leading to cirrhosis and liver failure before age 10.

In PFIC, liver cells are unable to drain bile acids, leading to the buildup of toxic substances in the liver. While the precise cause of severe itching in patients with PFIC is unknown, it may involve increased levels of bile acids in the body and skin.

Odevixibat is a potent, nonsystemic ileal bile acid transport inhibitor that does not need refrigeration and is given as a once-daily capsule or opened and sprinkled onto soft foods, the company said in a news release announcing the approval.

There are at least three types of PFIC; all are inherited genetic conditions caused by gene mutations. Odevixibat is indicated to treat all subtypes.

“Treating children with PFIC can be difficult and frustrating given the current treatment options. Bylvay gives us a nonsurgical option and will change how we treat PFIC,” Richard Thompson, MD, principal investigator for the two trials that led to the approval, said in the news release.

“With this approval, my colleagues and I now have the opportunity to revisit how PFIC patients are being managed, and we are hopeful for better outcomes for these children,” said Dr. Thompson, professor of molecular hepatology at King’s College London.

The approval of odevixibat was supported by data from the PEDFIC 1 and PEDFIC 2 trials.

PEDFIC 1 enrolled 62 children with PFIC and severe itching, with 20 assigned to placebo and 42 to odevixibat, given once daily with a meal in the morning. Odevixibat met both of its primary endpoints, with the drug improving pruritus (P = .004) and reducing serum bile acid responses (P = .003).

In PEDFIC 2, a long-term, open-label extension study, the effects of odevixibat on pruritis and serum bile acids were sustained up to 48 weeks.

Odevixibat was well tolerated in both trials, with the most common treatment-related gastrointestinal adverse events being diarrhea/frequent stools. There were no serious treatment-related adverse events.

Children taking the drug should undergo liver test monitoring periodically during treatment, the FDA said when announcing the approval. Odevixibat may affect absorption of fat-soluble vitamins such as A, D, E, and K. Patients should be monitored for fat-soluble vitamin deficiency while taking the drug.

Full prescribing information is available online.

“Until now, invasive surgery was the only approved treatment option. With the approval of Bylvay, parents may find hope in having a less invasive treatment option available,” Emily Ventura, leader of the PFIC Advocacy and Resource Network and mother to a child with PFIC, said in the news release.

The company said it will launch odevixibat “immediately” to accelerate availability for patients and families affected by PFIC.

Odevixibat is also being studied in other rare pediatric cholestatic liver diseases, including biliary atresia and Alagille syndrome.

A version of this article first appeared on Medscape.com.

Family medicine has evolved in many ways since its inception in 1969, especially in terms of the people who are practicing it, but it still needs more diversity to represent the faces of patients.

The specialty has been on the path to a racially diverse workforce over the past few decades. Since family medicine is a discipline where doctors look not just at individual patients, but also at the health of the community, some family physicians see working in their specialty as a way to integrate public health with medicine to curb health inequities.

Maria Harsha Wusu, MD, MSEd, is an example of such a family physician.

Dr. Wusu, who is Black and of Japanese descent, chose to practice family medicine to address health in communities of color “which we know in the United States experience health inequities due to structural racism,” she said in an interview.

“It’s a discipline where you look at the health of the entire family and the community and you really look at the environmental, the political, the historical factors that are influencing the health of the community,” explained Dr. Wusu, who is currently the director of health equity at Morehouse School of Medicine, Atlanta. Family physicians are not just asking: “Does a patient have hypertension?” but also whether a patient has access to healthy food, green space and other things.

While the field of family medicine is more diverse in the 21st century than it was at its beginning, Dr. Wusu, who completed her residency in 2016, still faced challenges to achieving her goal of helping communities of color. These specifically stemmed from a lack of diversity among the people in the places where she studied to become a doctor and family physician.

There were moments when Dr. Wusu felt isolated while in medical school and residency, because so few students and faculty members she saw looked like her.

Plus, studies have shown that a racially and ethnically diverse physician workforce is necessary to address health inequities. Research published in 2018 in the Journal of Health Care for the Poor and Underserved, for example, found that underrepresented physicians are more likely to practice in underserved areas than their White peers.

“I went to medical school at a historically White institution, and so there were very few people who identified as what we would say are underrepresented minorities in medicine,” Dr. Wusu explained. “There are issues with both implicit and explicit racism, which I think could be echoed by colleagues across the country that are additional challenges that I think medical students, particularly students of color, experience during what is an already kind of challenging time of medical school and the rigorous training of residency.”

Ada Stewart, MD, FAAFP, president of the American Academy of Family Physicians, echoed Dr. Wusu’s medical school experience. Dr. Stewart, who finished her residency in 2003, said that, out of the 120 students in her graduating medical class, only 10 were Black.

Marginalized groups are still underrepresented in residences. According to data compiled by the Association of American Medical Colleges, only 9.3% of Black people, 10% of Latino, and 0.3% of Native Hawaiian or other Pacific Islanders are residents in family medicine residency programs in 2019-2020. Meanwhile, White residents make up 50.8% of the residency program.

“We really need to do all that we can to increase diversity within our medical schools and residencies,” Dr. Stewart said.

In regards to gender, there has been an increasing number of women in the family medicine specialty. A 2021 study found that the proportion of female physicians in family medicine has grown from 33.9% 2010 to 41.9% in 2020.

“There’s still room for growth and we have to change the system and those structures that created these problems,” noted Dr. Stewart.
 

The social responsibility of family medicine

The family medicine specialty was born during an era of protest of social change, alongside the Civil Rights Movement, the peace movement, and counterculture protests. In April 1966, 3 years before American Boards approved family practice as a new specialty; the National Commission on Community Health stated that every person should have a personal physician who is the “central point for the integration and continuity of all medical services to his patient.” They also said such physicians should be aware of the “many and varied social, emotional and environmental factors that influence the health of his patient and family.”

While the diversity of family medicine specialty has significantly increased since its beginnings, it continues to lag behind the general U.S. population. A 2018 study published in the Journal of the American Board of Family Medicine, which aggregated data from 1987 to 2017, found that the proportion of Black and Latino board certified family physicians increased from 1.3% to 7.8% and 2.3% to 9.1%, respectively, in 30 years.

A 2014 study included 2 decades worth of data from the U.S. Census and the Association of American Medical Colleges to examine trends in racial and ethnic composition among family medicine residents. The U.S. population increased from 9% to 17% for Latinos, 11.7% to 12.2% for Blacks/African Americans, and 0.87% to 0.89% for Native Americans from 1990 to 2012. Meanwhile, minority representation in family residencies increased 4.9% to 9.4% for Hispanics/Latinos, from 4.2% to 7.9% for Blacks/African Americans, and from 0.7% to 0.9% for Native Americans.

Furthermore, 13.4% of the U.S. population is Black and 18.5% of the population is Latino, while only 7.8% of family medicine residents in 2019 were Black and 9.1% of family medicine residents were Latino, according to a recent study published in Family Medicine.
 

Recruiting a diverse physician workforce

The AAFP has launched a few initiatives to increase diversity within the specialty. In 2017, the AAFP established the Center for Diversity and Health Equity, a center to address social determinants of health. The EveryONE Project, an initiative that’s part of the AAFP’s center, offers members education and resources to promote workforce diversity. Some of those resources include “The Ladder Program,” an initiative founded by an AAFP member which involves monthly meetings and events for students as young as 9 years old to introduce them to medicine at a young age.

“You can’t see what you don’t see,” noted Dr. Stewart, who is the first Black, female president of the AAFP, and the fourth woman in the role. “I really have seen how important it is to be a mentor and to be out there so that individuals who look like me can see that they too can become a family physician and be that member of their community.”

In addition to The Ladder Program, some other resources aimed at increasing diversity among family physicians include Tour for Diversity in Medicine and the Doctors Back to School Program.

The Tour for Diversity in Medicine involves a team of physicians, other clinicians and students hosting events nationwide for minority students to help them see a path to medicine and other health professions. Meanwhile, the AAFP’s Doctors Back to School Program involves family physicians visiting children at schools, clubs, community organizations, and other places to raise childhood awareness of family medicine and help them see their own potential in health care careers.

Dr. Stewart said these programs have been successful in increasing underrepresented groups.

“We are trying to see how best to measure their success,” the AAFP president said. “Looking at the high numbers of individuals who chose the specialty of family medicine last year is what I would deem a success.”

Dr. Wusu also believes outreach to children in elementary schools is important when it comes to increasing diversity in the family medicine specialty.

One organization that’s proving such outreach is the Student National Medical Association, a branch of the National Medical Association, which is a professional organization of Black physicians. This group’s initiative, the Health Professions Recruitment Exposure Programs, exposes teens to science-related activities while introducing them to careers in health professions. Another SMNA program, called Youth Science Enrichment Program, targets elementary and junior high school students.

Dr. Wusu led a 2019 project that focused on creating a more diverse family medicine residency program by developing and implementing a strategic plan for diversity recruitment, which involved increasing outreach to marginalized groups and revising interviews to minimize bias. In a paper published on the results of the initiative, Dr. Wusu and coauthors noted that, between 2014 and 2017, the total number of underrepresented minority applicants to the Boston Medical Center Family Medicine Residency Program increased by 80%. Before the intervention, the percentage of applicants who were part of an underrepresented group ranged from 0% to 20%. During the intervention, that range jumped to 25% to 50%, according to the paper.

While Dr. Wusu considers these programs to be beneficial for the specialty, she doesn’t believe they should be done in isolation. There should also be efforts to tackle lack of opportunities and structural racism.

“With any inequity, you have to address it on multiple levels,” Dr. Wusu explained. “It’s great that there has been recognition for a need for diversity in family medicine, but my hope is that the call for equity would reach beyond that.”
 

Fostering an inclusive environment

While family medicine has come a long way with regards to increasing the amount of underrepresented groups in its specialty, Stephen Richmond, MD, MPH, believes there also needs to be a focus on the infrastructure of support to help retain these physicians.

“The problem of diversity within family medicine and largely in other specialties cannot be summed up simply as a matter of poor representation. We must do be better to understand the lived experience of physicians of color, in particular Black physicians, once they arrive in the medical professional environment. Doing so will help institutions to provide that infrastructure of support – including antiracism policies and practices – that will enhance wellness and representation,” said Dr. Richmond, clinical assistant professor of medicine in the division of primary care and population health at Stanford (Calif.) University.

Dr. Wusu also suggested establishing an explicitly antiracist environment. This can be done in multiple ways, including by holding programs that acknowledge the impact of structural racism on both the patients, medical students and faculty, and by educating staff about the history of racism, she said.

Dr. Stewart and Dr. Wusu think the specialty has improved in its representation of minorities, but that it has a “long way to go.”

“I think family medicine is really one of the more inclusive specialties. I mean, it has higher numbers of Black and Brown residents and physicians than other specialties,” Dr. Wusu said. “We still have a very long way to go to be at numbers that match the general population. But I think, in that way, family medicine is a place where a lot of Black and Brown physicians can find a home.”
 

Family medicine has evolved in many ways since its inception in 1969, especially in terms of the people who are practicing it, but it still needs more diversity to represent the faces of patients.

The specialty has been on the path to a racially diverse workforce over the past few decades. Since family medicine is a discipline where doctors look not just at individual patients, but also at the health of the community, some family physicians see working in their specialty as a way to integrate public health with medicine to curb health inequities.

Maria Harsha Wusu, MD, MSEd, is an example of such a family physician.

Dr. Wusu, who is Black and of Japanese descent, chose to practice family medicine to address health in communities of color “which we know in the United States experience health inequities due to structural racism,” she said in an interview.

“It’s a discipline where you look at the health of the entire family and the community and you really look at the environmental, the political, the historical factors that are influencing the health of the community,” explained Dr. Wusu, who is currently the director of health equity at Morehouse School of Medicine, Atlanta. Family physicians are not just asking: “Does a patient have hypertension?” but also whether a patient has access to healthy food, green space and other things.

While the field of family medicine is more diverse in the 21st century than it was at its beginning, Dr. Wusu, who completed her residency in 2016, still faced challenges to achieving her goal of helping communities of color. These specifically stemmed from a lack of diversity among the people in the places where she studied to become a doctor and family physician.

There were moments when Dr. Wusu felt isolated while in medical school and residency, because so few students and faculty members she saw looked like her.

Plus, studies have shown that a racially and ethnically diverse physician workforce is necessary to address health inequities. Research published in 2018 in the Journal of Health Care for the Poor and Underserved, for example, found that underrepresented physicians are more likely to practice in underserved areas than their White peers.

“I went to medical school at a historically White institution, and so there were very few people who identified as what we would say are underrepresented minorities in medicine,” Dr. Wusu explained. “There are issues with both implicit and explicit racism, which I think could be echoed by colleagues across the country that are additional challenges that I think medical students, particularly students of color, experience during what is an already kind of challenging time of medical school and the rigorous training of residency.”

Ada Stewart, MD, FAAFP, president of the American Academy of Family Physicians, echoed Dr. Wusu’s medical school experience. Dr. Stewart, who finished her residency in 2003, said that, out of the 120 students in her graduating medical class, only 10 were Black.

Marginalized groups are still underrepresented in residences. According to data compiled by the Association of American Medical Colleges, only 9.3% of Black people, 10% of Latino, and 0.3% of Native Hawaiian or other Pacific Islanders are residents in family medicine residency programs in 2019-2020. Meanwhile, White residents make up 50.8% of the residency program.

“We really need to do all that we can to increase diversity within our medical schools and residencies,” Dr. Stewart said.

In regards to gender, there has been an increasing number of women in the family medicine specialty. A 2021 study found that the proportion of female physicians in family medicine has grown from 33.9% 2010 to 41.9% in 2020.

“There’s still room for growth and we have to change the system and those structures that created these problems,” noted Dr. Stewart.
 

The social responsibility of family medicine

The family medicine specialty was born during an era of protest of social change, alongside the Civil Rights Movement, the peace movement, and counterculture protests. In April 1966, 3 years before American Boards approved family practice as a new specialty; the National Commission on Community Health stated that every person should have a personal physician who is the “central point for the integration and continuity of all medical services to his patient.” They also said such physicians should be aware of the “many and varied social, emotional and environmental factors that influence the health of his patient and family.”

While the diversity of family medicine specialty has significantly increased since its beginnings, it continues to lag behind the general U.S. population. A 2018 study published in the Journal of the American Board of Family Medicine, which aggregated data from 1987 to 2017, found that the proportion of Black and Latino board certified family physicians increased from 1.3% to 7.8% and 2.3% to 9.1%, respectively, in 30 years.

A 2014 study included 2 decades worth of data from the U.S. Census and the Association of American Medical Colleges to examine trends in racial and ethnic composition among family medicine residents. The U.S. population increased from 9% to 17% for Latinos, 11.7% to 12.2% for Blacks/African Americans, and 0.87% to 0.89% for Native Americans from 1990 to 2012. Meanwhile, minority representation in family residencies increased 4.9% to 9.4% for Hispanics/Latinos, from 4.2% to 7.9% for Blacks/African Americans, and from 0.7% to 0.9% for Native Americans.

Furthermore, 13.4% of the U.S. population is Black and 18.5% of the population is Latino, while only 7.8% of family medicine residents in 2019 were Black and 9.1% of family medicine residents were Latino, according to a recent study published in Family Medicine.
 

Recruiting a diverse physician workforce

The AAFP has launched a few initiatives to increase diversity within the specialty. In 2017, the AAFP established the Center for Diversity and Health Equity, a center to address social determinants of health. The EveryONE Project, an initiative that’s part of the AAFP’s center, offers members education and resources to promote workforce diversity. Some of those resources include “The Ladder Program,” an initiative founded by an AAFP member which involves monthly meetings and events for students as young as 9 years old to introduce them to medicine at a young age.

“You can’t see what you don’t see,” noted Dr. Stewart, who is the first Black, female president of the AAFP, and the fourth woman in the role. “I really have seen how important it is to be a mentor and to be out there so that individuals who look like me can see that they too can become a family physician and be that member of their community.”

In addition to The Ladder Program, some other resources aimed at increasing diversity among family physicians include Tour for Diversity in Medicine and the Doctors Back to School Program.

The Tour for Diversity in Medicine involves a team of physicians, other clinicians and students hosting events nationwide for minority students to help them see a path to medicine and other health professions. Meanwhile, the AAFP’s Doctors Back to School Program involves family physicians visiting children at schools, clubs, community organizations, and other places to raise childhood awareness of family medicine and help them see their own potential in health care careers.

Dr. Stewart said these programs have been successful in increasing underrepresented groups.

“We are trying to see how best to measure their success,” the AAFP president said. “Looking at the high numbers of individuals who chose the specialty of family medicine last year is what I would deem a success.”

Dr. Wusu also believes outreach to children in elementary schools is important when it comes to increasing diversity in the family medicine specialty.

One organization that’s proving such outreach is the Student National Medical Association, a branch of the National Medical Association, which is a professional organization of Black physicians. This group’s initiative, the Health Professions Recruitment Exposure Programs, exposes teens to science-related activities while introducing them to careers in health professions. Another SMNA program, called Youth Science Enrichment Program, targets elementary and junior high school students.

Dr. Wusu led a 2019 project that focused on creating a more diverse family medicine residency program by developing and implementing a strategic plan for diversity recruitment, which involved increasing outreach to marginalized groups and revising interviews to minimize bias. In a paper published on the results of the initiative, Dr. Wusu and coauthors noted that, between 2014 and 2017, the total number of underrepresented minority applicants to the Boston Medical Center Family Medicine Residency Program increased by 80%. Before the intervention, the percentage of applicants who were part of an underrepresented group ranged from 0% to 20%. During the intervention, that range jumped to 25% to 50%, according to the paper.

While Dr. Wusu considers these programs to be beneficial for the specialty, she doesn’t believe they should be done in isolation. There should also be efforts to tackle lack of opportunities and structural racism.

“With any inequity, you have to address it on multiple levels,” Dr. Wusu explained. “It’s great that there has been recognition for a need for diversity in family medicine, but my hope is that the call for equity would reach beyond that.”
 

Fostering an inclusive environment

While family medicine has come a long way with regards to increasing the amount of underrepresented groups in its specialty, Stephen Richmond, MD, MPH, believes there also needs to be a focus on the infrastructure of support to help retain these physicians.

“The problem of diversity within family medicine and largely in other specialties cannot be summed up simply as a matter of poor representation. We must do be better to understand the lived experience of physicians of color, in particular Black physicians, once they arrive in the medical professional environment. Doing so will help institutions to provide that infrastructure of support – including antiracism policies and practices – that will enhance wellness and representation,” said Dr. Richmond, clinical assistant professor of medicine in the division of primary care and population health at Stanford (Calif.) University.

Dr. Wusu also suggested establishing an explicitly antiracist environment. This can be done in multiple ways, including by holding programs that acknowledge the impact of structural racism on both the patients, medical students and faculty, and by educating staff about the history of racism, she said.

Dr. Stewart and Dr. Wusu think the specialty has improved in its representation of minorities, but that it has a “long way to go.”

“I think family medicine is really one of the more inclusive specialties. I mean, it has higher numbers of Black and Brown residents and physicians than other specialties,” Dr. Wusu said. “We still have a very long way to go to be at numbers that match the general population. But I think, in that way, family medicine is a place where a lot of Black and Brown physicians can find a home.”
 

Family medicine has evolved in many ways since its inception in 1969, especially in terms of the people who are practicing it, but it still needs more diversity to represent the faces of patients.

The specialty has been on the path to a racially diverse workforce over the past few decades. Since family medicine is a discipline where doctors look not just at individual patients, but also at the health of the community, some family physicians see working in their specialty as a way to integrate public health with medicine to curb health inequities.

Maria Harsha Wusu, MD, MSEd, is an example of such a family physician.

Dr. Wusu, who is Black and of Japanese descent, chose to practice family medicine to address health in communities of color “which we know in the United States experience health inequities due to structural racism,” she said in an interview.

“It’s a discipline where you look at the health of the entire family and the community and you really look at the environmental, the political, the historical factors that are influencing the health of the community,” explained Dr. Wusu, who is currently the director of health equity at Morehouse School of Medicine, Atlanta. Family physicians are not just asking: “Does a patient have hypertension?” but also whether a patient has access to healthy food, green space and other things.

While the field of family medicine is more diverse in the 21st century than it was at its beginning, Dr. Wusu, who completed her residency in 2016, still faced challenges to achieving her goal of helping communities of color. These specifically stemmed from a lack of diversity among the people in the places where she studied to become a doctor and family physician.

There were moments when Dr. Wusu felt isolated while in medical school and residency, because so few students and faculty members she saw looked like her.

Plus, studies have shown that a racially and ethnically diverse physician workforce is necessary to address health inequities. Research published in 2018 in the Journal of Health Care for the Poor and Underserved, for example, found that underrepresented physicians are more likely to practice in underserved areas than their White peers.

“I went to medical school at a historically White institution, and so there were very few people who identified as what we would say are underrepresented minorities in medicine,” Dr. Wusu explained. “There are issues with both implicit and explicit racism, which I think could be echoed by colleagues across the country that are additional challenges that I think medical students, particularly students of color, experience during what is an already kind of challenging time of medical school and the rigorous training of residency.”

Ada Stewart, MD, FAAFP, president of the American Academy of Family Physicians, echoed Dr. Wusu’s medical school experience. Dr. Stewart, who finished her residency in 2003, said that, out of the 120 students in her graduating medical class, only 10 were Black.

Marginalized groups are still underrepresented in residences. According to data compiled by the Association of American Medical Colleges, only 9.3% of Black people, 10% of Latino, and 0.3% of Native Hawaiian or other Pacific Islanders are residents in family medicine residency programs in 2019-2020. Meanwhile, White residents make up 50.8% of the residency program.

“We really need to do all that we can to increase diversity within our medical schools and residencies,” Dr. Stewart said.

In regards to gender, there has been an increasing number of women in the family medicine specialty. A 2021 study found that the proportion of female physicians in family medicine has grown from 33.9% 2010 to 41.9% in 2020.

“There’s still room for growth and we have to change the system and those structures that created these problems,” noted Dr. Stewart.
 

The social responsibility of family medicine

The family medicine specialty was born during an era of protest of social change, alongside the Civil Rights Movement, the peace movement, and counterculture protests. In April 1966, 3 years before American Boards approved family practice as a new specialty; the National Commission on Community Health stated that every person should have a personal physician who is the “central point for the integration and continuity of all medical services to his patient.” They also said such physicians should be aware of the “many and varied social, emotional and environmental factors that influence the health of his patient and family.”

While the diversity of family medicine specialty has significantly increased since its beginnings, it continues to lag behind the general U.S. population. A 2018 study published in the Journal of the American Board of Family Medicine, which aggregated data from 1987 to 2017, found that the proportion of Black and Latino board certified family physicians increased from 1.3% to 7.8% and 2.3% to 9.1%, respectively, in 30 years.

A 2014 study included 2 decades worth of data from the U.S. Census and the Association of American Medical Colleges to examine trends in racial and ethnic composition among family medicine residents. The U.S. population increased from 9% to 17% for Latinos, 11.7% to 12.2% for Blacks/African Americans, and 0.87% to 0.89% for Native Americans from 1990 to 2012. Meanwhile, minority representation in family residencies increased 4.9% to 9.4% for Hispanics/Latinos, from 4.2% to 7.9% for Blacks/African Americans, and from 0.7% to 0.9% for Native Americans.

Furthermore, 13.4% of the U.S. population is Black and 18.5% of the population is Latino, while only 7.8% of family medicine residents in 2019 were Black and 9.1% of family medicine residents were Latino, according to a recent study published in Family Medicine.
 

Recruiting a diverse physician workforce

The AAFP has launched a few initiatives to increase diversity within the specialty. In 2017, the AAFP established the Center for Diversity and Health Equity, a center to address social determinants of health. The EveryONE Project, an initiative that’s part of the AAFP’s center, offers members education and resources to promote workforce diversity. Some of those resources include “The Ladder Program,” an initiative founded by an AAFP member which involves monthly meetings and events for students as young as 9 years old to introduce them to medicine at a young age.

“You can’t see what you don’t see,” noted Dr. Stewart, who is the first Black, female president of the AAFP, and the fourth woman in the role. “I really have seen how important it is to be a mentor and to be out there so that individuals who look like me can see that they too can become a family physician and be that member of their community.”

In addition to The Ladder Program, some other resources aimed at increasing diversity among family physicians include Tour for Diversity in Medicine and the Doctors Back to School Program.

The Tour for Diversity in Medicine involves a team of physicians, other clinicians and students hosting events nationwide for minority students to help them see a path to medicine and other health professions. Meanwhile, the AAFP’s Doctors Back to School Program involves family physicians visiting children at schools, clubs, community organizations, and other places to raise childhood awareness of family medicine and help them see their own potential in health care careers.

Dr. Stewart said these programs have been successful in increasing underrepresented groups.

“We are trying to see how best to measure their success,” the AAFP president said. “Looking at the high numbers of individuals who chose the specialty of family medicine last year is what I would deem a success.”

Dr. Wusu also believes outreach to children in elementary schools is important when it comes to increasing diversity in the family medicine specialty.

One organization that’s proving such outreach is the Student National Medical Association, a branch of the National Medical Association, which is a professional organization of Black physicians. This group’s initiative, the Health Professions Recruitment Exposure Programs, exposes teens to science-related activities while introducing them to careers in health professions. Another SMNA program, called Youth Science Enrichment Program, targets elementary and junior high school students.

Dr. Wusu led a 2019 project that focused on creating a more diverse family medicine residency program by developing and implementing a strategic plan for diversity recruitment, which involved increasing outreach to marginalized groups and revising interviews to minimize bias. In a paper published on the results of the initiative, Dr. Wusu and coauthors noted that, between 2014 and 2017, the total number of underrepresented minority applicants to the Boston Medical Center Family Medicine Residency Program increased by 80%. Before the intervention, the percentage of applicants who were part of an underrepresented group ranged from 0% to 20%. During the intervention, that range jumped to 25% to 50%, according to the paper.

While Dr. Wusu considers these programs to be beneficial for the specialty, she doesn’t believe they should be done in isolation. There should also be efforts to tackle lack of opportunities and structural racism.

“With any inequity, you have to address it on multiple levels,” Dr. Wusu explained. “It’s great that there has been recognition for a need for diversity in family medicine, but my hope is that the call for equity would reach beyond that.”
 

Fostering an inclusive environment

While family medicine has come a long way with regards to increasing the amount of underrepresented groups in its specialty, Stephen Richmond, MD, MPH, believes there also needs to be a focus on the infrastructure of support to help retain these physicians.

“The problem of diversity within family medicine and largely in other specialties cannot be summed up simply as a matter of poor representation. We must do be better to understand the lived experience of physicians of color, in particular Black physicians, once they arrive in the medical professional environment. Doing so will help institutions to provide that infrastructure of support – including antiracism policies and practices – that will enhance wellness and representation,” said Dr. Richmond, clinical assistant professor of medicine in the division of primary care and population health at Stanford (Calif.) University.

Dr. Wusu also suggested establishing an explicitly antiracist environment. This can be done in multiple ways, including by holding programs that acknowledge the impact of structural racism on both the patients, medical students and faculty, and by educating staff about the history of racism, she said.

Dr. Stewart and Dr. Wusu think the specialty has improved in its representation of minorities, but that it has a “long way to go.”

“I think family medicine is really one of the more inclusive specialties. I mean, it has higher numbers of Black and Brown residents and physicians than other specialties,” Dr. Wusu said. “We still have a very long way to go to be at numbers that match the general population. But I think, in that way, family medicine is a place where a lot of Black and Brown physicians can find a home.”
 

Like most American doctors, I take a variety of insurances and insurance plans.

Some of these, particularly HMOs, require a referring physician to send me a written, insurance-approved, referral (AKA authorization) before the visit, to submit with my bill.

Medical visits of all kinds are generally billed on a scale from 1 (brief/simple issue) to 5 (lots of time needed/very complicated).

After 23 years, I’m used to this.

But recently a new wrinkle has emerged.

In the last month I’ve gotten two referrals (both from the same internist), except these state, very clearly, that charges for any visit cannot exceed level 3.

And they’re telling me this before I’ve ever seen the patients, or have any idea how complicated they are, or how long a list of questions they and/or their families will have.

No.

I faxed them back asking for a referral allowing me to bill up to level 5 if needed. I might charge less than that, but none of us know how complicated or long a visit will be until someone comes in. There’s no crystal ball in medicine.

I’m sure someone will say I’m a money-grubbing doctor who couldn’t care less about the patient.

That’s far from the truth. I’m here for the patients. I like helping them. It’s why I do this.

But I can’t help anyone if I can’t afford to keep the office lights on, either.

I never heard back from them. Maybe they decided the patients didn’t need me that much. Maybe they sent them to another neurologist and took my name off their referral list. Maybe they never even noticed my return fax.

What will happen now, I have no idea. Maybe this was something that office tried, to see if I noticed. Maybe it’s the start of the next wave of medical cutbacks. Maybe it was a staff error at the other end.

But either way, none of us can see patients at a loss and hope to make it up on quantity. This isn’t an amusement park or thrift store. People with problems need time, and time costs money. I need to pay my staff, my rent, and my mortgage. If I can’t do those things, I won’t be able to help anyone.

That’s just, for better or worse, the way it is.

Dr. Block has a solo neurology practice in Scottsdale, Ariz.

Like most American doctors, I take a variety of insurances and insurance plans.

Some of these, particularly HMOs, require a referring physician to send me a written, insurance-approved, referral (AKA authorization) before the visit, to submit with my bill.

Medical visits of all kinds are generally billed on a scale from 1 (brief/simple issue) to 5 (lots of time needed/very complicated).

After 23 years, I’m used to this.

But recently a new wrinkle has emerged.

In the last month I’ve gotten two referrals (both from the same internist), except these state, very clearly, that charges for any visit cannot exceed level 3.

And they’re telling me this before I’ve ever seen the patients, or have any idea how complicated they are, or how long a list of questions they and/or their families will have.

No.

I faxed them back asking for a referral allowing me to bill up to level 5 if needed. I might charge less than that, but none of us know how complicated or long a visit will be until someone comes in. There’s no crystal ball in medicine.

I’m sure someone will say I’m a money-grubbing doctor who couldn’t care less about the patient.

That’s far from the truth. I’m here for the patients. I like helping them. It’s why I do this.

But I can’t help anyone if I can’t afford to keep the office lights on, either.

I never heard back from them. Maybe they decided the patients didn’t need me that much. Maybe they sent them to another neurologist and took my name off their referral list. Maybe they never even noticed my return fax.

What will happen now, I have no idea. Maybe this was something that office tried, to see if I noticed. Maybe it’s the start of the next wave of medical cutbacks. Maybe it was a staff error at the other end.

But either way, none of us can see patients at a loss and hope to make it up on quantity. This isn’t an amusement park or thrift store. People with problems need time, and time costs money. I need to pay my staff, my rent, and my mortgage. If I can’t do those things, I won’t be able to help anyone.

That’s just, for better or worse, the way it is.

Dr. Block has a solo neurology practice in Scottsdale, Ariz.

Like most American doctors, I take a variety of insurances and insurance plans.

Some of these, particularly HMOs, require a referring physician to send me a written, insurance-approved, referral (AKA authorization) before the visit, to submit with my bill.

Medical visits of all kinds are generally billed on a scale from 1 (brief/simple issue) to 5 (lots of time needed/very complicated).

After 23 years, I’m used to this.

But recently a new wrinkle has emerged.

In the last month I’ve gotten two referrals (both from the same internist), except these state, very clearly, that charges for any visit cannot exceed level 3.

And they’re telling me this before I’ve ever seen the patients, or have any idea how complicated they are, or how long a list of questions they and/or their families will have.

No.

I faxed them back asking for a referral allowing me to bill up to level 5 if needed. I might charge less than that, but none of us know how complicated or long a visit will be until someone comes in. There’s no crystal ball in medicine.

I’m sure someone will say I’m a money-grubbing doctor who couldn’t care less about the patient.

That’s far from the truth. I’m here for the patients. I like helping them. It’s why I do this.

But I can’t help anyone if I can’t afford to keep the office lights on, either.

I never heard back from them. Maybe they decided the patients didn’t need me that much. Maybe they sent them to another neurologist and took my name off their referral list. Maybe they never even noticed my return fax.

What will happen now, I have no idea. Maybe this was something that office tried, to see if I noticed. Maybe it’s the start of the next wave of medical cutbacks. Maybe it was a staff error at the other end.

But either way, none of us can see patients at a loss and hope to make it up on quantity. This isn’t an amusement park or thrift store. People with problems need time, and time costs money. I need to pay my staff, my rent, and my mortgage. If I can’t do those things, I won’t be able to help anyone.

That’s just, for better or worse, the way it is.

Dr. Block has a solo neurology practice in Scottsdale, Ariz.

The risk of in-hospital complications and poor birth outcomes were greater in pregnant women with current or historical cancer diagnoses, new research suggests.

The study, published in Mayo Clinic Proceedings, found that women with current and historical cancer diagnoses had an increased risk of death, kidney injury, and stroke during delivery hospitalizations, compared with those with no cancer. When it came to delivery outcomes, this group also had a higher risk for preterm birth and postpartum hemorrhage. Those with a current cancer diagnoses had a 1.7-fold increase in odds for a preterm birth, compared with women without cancer.

“Our study found that metastases increased the odds of mortality, cesarean delivery, preterm birth, and stillbirth,” the researchers noted. “Coupled with previous research reporting that pregnant women are more likely to be diagnosed with advanced disease, this implies that pregnant women with newly diagnosed cancer have poor prognoses.”

However, although women with prior cancer had increased odds of mortality, the researchers said it was not statistically significant.

“The study really did not show an increase of mortality [for women with prior cancer diagnosis],” said Justin Chura, MD, a specialist in gynecologic oncology who was not involved in the study. “And the reason might be because there is not or the reason might be because it’s such a rare event. You would need 100 million births to assess that. So I would actually use caution in that interpretation.”

Researchers analyzed more than 43 million delivery hospitalizations of women with or without current or historical cancer diagnoses between January 2004 and December 2014. They found that the most common cancer diagnoses were hematologic, thyroid, cervical, skin, and breast.

Of the five most common cancers, the prevalence of all maternal complications and negative delivery outcomes was the highest among women with hematologic cancers. They were more likely to experience peripartum cardiomyopathy, acute kidney injury, and arrhythmia, compared with other cancers. Postpartum hemorrhage, maternal mortality, and placental abruption was also more likely to occur in those with this type of cancer.

“I was surprised that it was the hematologic cancers that were worse when they did it by cancer type,” said Dr. Chura, who is the chief of surgery and the director of gynecologic oncology and robotic surgery at the Cancer Treatment Centers of America’s Eastern Regional Medical Center in Philadelphia. “I think this is a useful bit of information for counseling our patients and also to identify the cohort with the highest risk.”

The findings also suggested that those with skin cancer had the highest odds for stroke, while women with cervical and breast cancers were more likely to experience acute kidney injury and preterm birth.

Dr. Chura said cancer treatments can have an impact on a woman’s health when she’s giving birth. For example, if a woman is diagnosed with cervical cancer, doctors may perform a cone biopsy on her where they remove a large portion of the cervix and still leave them with the ability to conceive and become pregnant. However, those patients are left with a higher risk of a preterm delivery.

For women with a hematologic cancer like non-Hodgkin’s lymphoma, chest radiation may cause some subsequent damage to their heart muscles “and now the stress of pregnancy puts more demand on the heart that can lead to cardiac complications for that patient,” Dr. Chura said.

“There are potential long-term effects from radiation and chemotherapy,” Dr. Chura said.

Previous studies have shown that chemotherapy may affect pregnancy and delivery. A 2019 study published in the Journal of Cancer also found that 59 pregnant women with cancer had increased mortality compared with those without the long-term illness. Meanwhile, another 2018 study published in Cancer found that women who conceived less than a year after starting chemotherapy had higher risks of preterm birth in comparison with those who conceived more than a year after starting chemotherapy. The study also found that cancer survivors who conceived more than a year after finishing chemotherapy with or without radiation had no higher risk of a preterm birth than those without cancer.

Dr. Chura said the new study could force doctors to think about the long-term effects of their cancer therapies and make them more apt to think about how to make cancer therapy less toxic with less long-term health consequences, while still curing patients.

“Most oncologists, when dealing with younger patients, are very focused on curing the cancer at hand, but not necessarily thinking 5 or 10 years down the road,” Dr. Chura said. “[This study] could help inform or at least make us aware of the long-term consequences of our cancer therapies.”

Dr. Chura had no relevant financial disclosures.

The risk of in-hospital complications and poor birth outcomes were greater in pregnant women with current or historical cancer diagnoses, new research suggests.

The study, published in Mayo Clinic Proceedings, found that women with current and historical cancer diagnoses had an increased risk of death, kidney injury, and stroke during delivery hospitalizations, compared with those with no cancer. When it came to delivery outcomes, this group also had a higher risk for preterm birth and postpartum hemorrhage. Those with a current cancer diagnoses had a 1.7-fold increase in odds for a preterm birth, compared with women without cancer.

“Our study found that metastases increased the odds of mortality, cesarean delivery, preterm birth, and stillbirth,” the researchers noted. “Coupled with previous research reporting that pregnant women are more likely to be diagnosed with advanced disease, this implies that pregnant women with newly diagnosed cancer have poor prognoses.”

However, although women with prior cancer had increased odds of mortality, the researchers said it was not statistically significant.

“The study really did not show an increase of mortality [for women with prior cancer diagnosis],” said Justin Chura, MD, a specialist in gynecologic oncology who was not involved in the study. “And the reason might be because there is not or the reason might be because it’s such a rare event. You would need 100 million births to assess that. So I would actually use caution in that interpretation.”

Researchers analyzed more than 43 million delivery hospitalizations of women with or without current or historical cancer diagnoses between January 2004 and December 2014. They found that the most common cancer diagnoses were hematologic, thyroid, cervical, skin, and breast.

Of the five most common cancers, the prevalence of all maternal complications and negative delivery outcomes was the highest among women with hematologic cancers. They were more likely to experience peripartum cardiomyopathy, acute kidney injury, and arrhythmia, compared with other cancers. Postpartum hemorrhage, maternal mortality, and placental abruption was also more likely to occur in those with this type of cancer.

“I was surprised that it was the hematologic cancers that were worse when they did it by cancer type,” said Dr. Chura, who is the chief of surgery and the director of gynecologic oncology and robotic surgery at the Cancer Treatment Centers of America’s Eastern Regional Medical Center in Philadelphia. “I think this is a useful bit of information for counseling our patients and also to identify the cohort with the highest risk.”

The findings also suggested that those with skin cancer had the highest odds for stroke, while women with cervical and breast cancers were more likely to experience acute kidney injury and preterm birth.

Dr. Chura said cancer treatments can have an impact on a woman’s health when she’s giving birth. For example, if a woman is diagnosed with cervical cancer, doctors may perform a cone biopsy on her where they remove a large portion of the cervix and still leave them with the ability to conceive and become pregnant. However, those patients are left with a higher risk of a preterm delivery.

For women with a hematologic cancer like non-Hodgkin’s lymphoma, chest radiation may cause some subsequent damage to their heart muscles “and now the stress of pregnancy puts more demand on the heart that can lead to cardiac complications for that patient,” Dr. Chura said.

“There are potential long-term effects from radiation and chemotherapy,” Dr. Chura said.

Previous studies have shown that chemotherapy may affect pregnancy and delivery. A 2019 study published in the Journal of Cancer also found that 59 pregnant women with cancer had increased mortality compared with those without the long-term illness. Meanwhile, another 2018 study published in Cancer found that women who conceived less than a year after starting chemotherapy had higher risks of preterm birth in comparison with those who conceived more than a year after starting chemotherapy. The study also found that cancer survivors who conceived more than a year after finishing chemotherapy with or without radiation had no higher risk of a preterm birth than those without cancer.

Dr. Chura said the new study could force doctors to think about the long-term effects of their cancer therapies and make them more apt to think about how to make cancer therapy less toxic with less long-term health consequences, while still curing patients.

“Most oncologists, when dealing with younger patients, are very focused on curing the cancer at hand, but not necessarily thinking 5 or 10 years down the road,” Dr. Chura said. “[This study] could help inform or at least make us aware of the long-term consequences of our cancer therapies.”

Dr. Chura had no relevant financial disclosures.

The risk of in-hospital complications and poor birth outcomes were greater in pregnant women with current or historical cancer diagnoses, new research suggests.

The study, published in Mayo Clinic Proceedings, found that women with current and historical cancer diagnoses had an increased risk of death, kidney injury, and stroke during delivery hospitalizations, compared with those with no cancer. When it came to delivery outcomes, this group also had a higher risk for preterm birth and postpartum hemorrhage. Those with a current cancer diagnoses had a 1.7-fold increase in odds for a preterm birth, compared with women without cancer.

“Our study found that metastases increased the odds of mortality, cesarean delivery, preterm birth, and stillbirth,” the researchers noted. “Coupled with previous research reporting that pregnant women are more likely to be diagnosed with advanced disease, this implies that pregnant women with newly diagnosed cancer have poor prognoses.”

However, although women with prior cancer had increased odds of mortality, the researchers said it was not statistically significant.

“The study really did not show an increase of mortality [for women with prior cancer diagnosis],” said Justin Chura, MD, a specialist in gynecologic oncology who was not involved in the study. “And the reason might be because there is not or the reason might be because it’s such a rare event. You would need 100 million births to assess that. So I would actually use caution in that interpretation.”

Researchers analyzed more than 43 million delivery hospitalizations of women with or without current or historical cancer diagnoses between January 2004 and December 2014. They found that the most common cancer diagnoses were hematologic, thyroid, cervical, skin, and breast.

Of the five most common cancers, the prevalence of all maternal complications and negative delivery outcomes was the highest among women with hematologic cancers. They were more likely to experience peripartum cardiomyopathy, acute kidney injury, and arrhythmia, compared with other cancers. Postpartum hemorrhage, maternal mortality, and placental abruption was also more likely to occur in those with this type of cancer.

“I was surprised that it was the hematologic cancers that were worse when they did it by cancer type,” said Dr. Chura, who is the chief of surgery and the director of gynecologic oncology and robotic surgery at the Cancer Treatment Centers of America’s Eastern Regional Medical Center in Philadelphia. “I think this is a useful bit of information for counseling our patients and also to identify the cohort with the highest risk.”

The findings also suggested that those with skin cancer had the highest odds for stroke, while women with cervical and breast cancers were more likely to experience acute kidney injury and preterm birth.

Dr. Chura said cancer treatments can have an impact on a woman’s health when she’s giving birth. For example, if a woman is diagnosed with cervical cancer, doctors may perform a cone biopsy on her where they remove a large portion of the cervix and still leave them with the ability to conceive and become pregnant. However, those patients are left with a higher risk of a preterm delivery.

For women with a hematologic cancer like non-Hodgkin’s lymphoma, chest radiation may cause some subsequent damage to their heart muscles “and now the stress of pregnancy puts more demand on the heart that can lead to cardiac complications for that patient,” Dr. Chura said.

“There are potential long-term effects from radiation and chemotherapy,” Dr. Chura said.

Previous studies have shown that chemotherapy may affect pregnancy and delivery. A 2019 study published in the Journal of Cancer also found that 59 pregnant women with cancer had increased mortality compared with those without the long-term illness. Meanwhile, another 2018 study published in Cancer found that women who conceived less than a year after starting chemotherapy had higher risks of preterm birth in comparison with those who conceived more than a year after starting chemotherapy. The study also found that cancer survivors who conceived more than a year after finishing chemotherapy with or without radiation had no higher risk of a preterm birth than those without cancer.

Dr. Chura said the new study could force doctors to think about the long-term effects of their cancer therapies and make them more apt to think about how to make cancer therapy less toxic with less long-term health consequences, while still curing patients.

“Most oncologists, when dealing with younger patients, are very focused on curing the cancer at hand, but not necessarily thinking 5 or 10 years down the road,” Dr. Chura said. “[This study] could help inform or at least make us aware of the long-term consequences of our cancer therapies.”

Dr. Chura had no relevant financial disclosures.