Online reviews most important factor in choosing a doctor: Survey

Article Type
Changed
Wed, 12/08/2021 - 09:46

Online reviews and star ratings are the most important factor in choosing a new health care provider, according to a new survey from Press Ganey, a provider of patient satisfaction surveys. According to the data, this online information is more important to consumers in selecting a physician than another doctor’s referral and is more than twice as important when choosing a primary care physician.

m-imagephotography/Thinkstock.com

In fact, 83% of respondents said they went online to read reviews of a physician after receiving a referral from another provider.

The online research trend reflects not only the increased familiarity of all generations with the internet but also the growing consumerization of health care, Thomas Jeffrey, president of the Sullivan/Luallin Group, a patient experience consulting firm, told this news organization.

“According to patient satisfaction surveys, people are becoming health care consumers more than in the past,” he noted. “Historically, we didn’t look at health care as a consumer product. But, with high deductibles and copays, doctor visits can represent a pretty significant out-of-pocket expense. As it begins to hit folks’ pocketbooks, they become more savvy shoppers.”

Digital preferences for providers were gaining “positive momentum” even before the COVID-19 pandemic, but the crisis “drove upticks in some consumer digital behaviors,” the Press Ganey report pointed out.

Mr. Jeffrey agreed, noting that this finding matches what Sullivan/Luallin has discovered in its research. “I think the pandemic pushed people to engage more online,” he said. “The highest net promoter score [likelihood to recommend in market surveys] for a pharmacy is the Amazon pharmacy, which is an online-based delivery service. Then you have telehealth visits, which are more convenient in many ways.”
 

How patients search online

In choosing a new primary care doctor, 51.1% go on the web first, 23.8% seek a referral from another health care provider, and 4.4% get information from an insurer or a benefits manager, according to the survey.

The factors that matter most to consumers when they pick any provider, in order, are online ratings and reviews of the physician, referral from a current doctor, ratings and reviews of the facility, and the quality and completeness of a doctor’s profile on a website or online directory. The doctor’s online presence and the quality of their website are also important.

According to Press Ganey, search engines like Google are the most used digital resources, with 65.4% of consumers employing them to find a doctor. However, consumers now use an average of 2.7 sites in their search. The leading destinations are a hospital or a clinic site, WebMD, Healthgrades, and Facebook. (This news organization is owned by WebMD.)

Compared with 2019, the report said, there has been a 22.8% decline in the use of search engines for seeking a doctor and a 53.7% increase in the use of health care review sites such as Healthgrades and Vitals.

When reading provider reviews, consumers look for more recent reviews and want the reviews to be “authentic and informative.” They also value the star ratings. About 84%of respondents said they wouldn’t book an appointment with a referred provider that had a rating of less than four stars.

Overall, the top reasons why people are deterred from making an appointment are difficulty contacting the office, the poor quality of online reviews, and an average online rating of less than four stars.

The vast majority of respondents (77%) said they believe internet reviews reflect their own experience with a provider organization, and only 2.6% said the reviews were inaccurate. Another finding of the survey indicates that this attention of patients to reviews of their own provider doesn’t represent idle curiosity: About 57% of Baby Boomers and 45% of millennials/Gen Z’ers said they’d written online reviews of a doctor or a hospital.
 

 

 

Factors in patient loyalty

The Press Ganey survey asked which of several factors, besides excellent care, patients weighed when giving a five-star review to a health care provider.

Quality of customer service was rated first by 70.8% of respondents, followed by cleanliness of facilities (67.5%), communication (63.4%), the provider’s bedside manner (63%), ease of appointment booking (58.8%), ease of patient intake/registration (52.3%), quality and accuracy of information (40.1%), availability of telehealth services (21.7%), and waiting room amenities (21.8%).

The report explained that “quality of customer service” means “demeanor, attentiveness, and helpfulness of staff and practitioners.” “Communication” refers to things like follow-up appointment reminders and annual checkup reminders.

According to Mr. Jeffrey, these factors were considered more important than a doctor’s bedside manner because of the team care approach in most physician offices. “We see a lot more folks derive their notion of quality from continuity of care. And if they feel the physician they love is being supported by a less than competent team, that can impact significantly their sense of the quality of care,” he said.
 

Online appointment booking is a must

To win over the online consumer, Press Ganey emphasized, practices should ensure that provider listings are accurate and complete. In addition, offering online appointment booking can avoid the top challenge in making a new appointment, which is getting through to the office.

Mr. Jeffrey concurred, although he notes that practices have to be careful about how they enable patients to select appointment slots online. He suggests that an appointment request form on a patient portal first ask what the purpose of the visit is and that it offer five or so options. If the request fits into a routine visit category, the provider’s calendar pops up and the patient can select a convenient time slot. If it’s something else, an appointment scheduler calls the patient back.

“There needs to be greater access to standard appointments online,” he said. “While privacy is an issue, you can use the patient portal that most EHRs have to provide online booking. If you want to succeed going forward, that’s going to be a major plus.”

Of course, to do any of this, including reading provider reviews, a consumer needs a good internet connection and a mobile or desktop device. While broadband internet access is still not available in some communities, the breakdown of the survey respondents by demographics shows that low-income people were included.

Mr. Jeffrey doesn’t believe that a lack of internet access or digital devices prevents many Americans from going online today. “Even in poor communities, most people have internet access through their smartphones. Even baby boomers are familiar with smartphones. I haven’t seen internet access be a big barrier for low-income households, because they all have access to phones.”

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

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Online reviews and star ratings are the most important factor in choosing a new health care provider, according to a new survey from Press Ganey, a provider of patient satisfaction surveys. According to the data, this online information is more important to consumers in selecting a physician than another doctor’s referral and is more than twice as important when choosing a primary care physician.

m-imagephotography/Thinkstock.com

In fact, 83% of respondents said they went online to read reviews of a physician after receiving a referral from another provider.

The online research trend reflects not only the increased familiarity of all generations with the internet but also the growing consumerization of health care, Thomas Jeffrey, president of the Sullivan/Luallin Group, a patient experience consulting firm, told this news organization.

“According to patient satisfaction surveys, people are becoming health care consumers more than in the past,” he noted. “Historically, we didn’t look at health care as a consumer product. But, with high deductibles and copays, doctor visits can represent a pretty significant out-of-pocket expense. As it begins to hit folks’ pocketbooks, they become more savvy shoppers.”

Digital preferences for providers were gaining “positive momentum” even before the COVID-19 pandemic, but the crisis “drove upticks in some consumer digital behaviors,” the Press Ganey report pointed out.

Mr. Jeffrey agreed, noting that this finding matches what Sullivan/Luallin has discovered in its research. “I think the pandemic pushed people to engage more online,” he said. “The highest net promoter score [likelihood to recommend in market surveys] for a pharmacy is the Amazon pharmacy, which is an online-based delivery service. Then you have telehealth visits, which are more convenient in many ways.”
 

How patients search online

In choosing a new primary care doctor, 51.1% go on the web first, 23.8% seek a referral from another health care provider, and 4.4% get information from an insurer or a benefits manager, according to the survey.

The factors that matter most to consumers when they pick any provider, in order, are online ratings and reviews of the physician, referral from a current doctor, ratings and reviews of the facility, and the quality and completeness of a doctor’s profile on a website or online directory. The doctor’s online presence and the quality of their website are also important.

According to Press Ganey, search engines like Google are the most used digital resources, with 65.4% of consumers employing them to find a doctor. However, consumers now use an average of 2.7 sites in their search. The leading destinations are a hospital or a clinic site, WebMD, Healthgrades, and Facebook. (This news organization is owned by WebMD.)

Compared with 2019, the report said, there has been a 22.8% decline in the use of search engines for seeking a doctor and a 53.7% increase in the use of health care review sites such as Healthgrades and Vitals.

When reading provider reviews, consumers look for more recent reviews and want the reviews to be “authentic and informative.” They also value the star ratings. About 84%of respondents said they wouldn’t book an appointment with a referred provider that had a rating of less than four stars.

Overall, the top reasons why people are deterred from making an appointment are difficulty contacting the office, the poor quality of online reviews, and an average online rating of less than four stars.

The vast majority of respondents (77%) said they believe internet reviews reflect their own experience with a provider organization, and only 2.6% said the reviews were inaccurate. Another finding of the survey indicates that this attention of patients to reviews of their own provider doesn’t represent idle curiosity: About 57% of Baby Boomers and 45% of millennials/Gen Z’ers said they’d written online reviews of a doctor or a hospital.
 

 

 

Factors in patient loyalty

The Press Ganey survey asked which of several factors, besides excellent care, patients weighed when giving a five-star review to a health care provider.

Quality of customer service was rated first by 70.8% of respondents, followed by cleanliness of facilities (67.5%), communication (63.4%), the provider’s bedside manner (63%), ease of appointment booking (58.8%), ease of patient intake/registration (52.3%), quality and accuracy of information (40.1%), availability of telehealth services (21.7%), and waiting room amenities (21.8%).

The report explained that “quality of customer service” means “demeanor, attentiveness, and helpfulness of staff and practitioners.” “Communication” refers to things like follow-up appointment reminders and annual checkup reminders.

According to Mr. Jeffrey, these factors were considered more important than a doctor’s bedside manner because of the team care approach in most physician offices. “We see a lot more folks derive their notion of quality from continuity of care. And if they feel the physician they love is being supported by a less than competent team, that can impact significantly their sense of the quality of care,” he said.
 

Online appointment booking is a must

To win over the online consumer, Press Ganey emphasized, practices should ensure that provider listings are accurate and complete. In addition, offering online appointment booking can avoid the top challenge in making a new appointment, which is getting through to the office.

Mr. Jeffrey concurred, although he notes that practices have to be careful about how they enable patients to select appointment slots online. He suggests that an appointment request form on a patient portal first ask what the purpose of the visit is and that it offer five or so options. If the request fits into a routine visit category, the provider’s calendar pops up and the patient can select a convenient time slot. If it’s something else, an appointment scheduler calls the patient back.

“There needs to be greater access to standard appointments online,” he said. “While privacy is an issue, you can use the patient portal that most EHRs have to provide online booking. If you want to succeed going forward, that’s going to be a major plus.”

Of course, to do any of this, including reading provider reviews, a consumer needs a good internet connection and a mobile or desktop device. While broadband internet access is still not available in some communities, the breakdown of the survey respondents by demographics shows that low-income people were included.

Mr. Jeffrey doesn’t believe that a lack of internet access or digital devices prevents many Americans from going online today. “Even in poor communities, most people have internet access through their smartphones. Even baby boomers are familiar with smartphones. I haven’t seen internet access be a big barrier for low-income households, because they all have access to phones.”

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

Online reviews and star ratings are the most important factor in choosing a new health care provider, according to a new survey from Press Ganey, a provider of patient satisfaction surveys. According to the data, this online information is more important to consumers in selecting a physician than another doctor’s referral and is more than twice as important when choosing a primary care physician.

m-imagephotography/Thinkstock.com

In fact, 83% of respondents said they went online to read reviews of a physician after receiving a referral from another provider.

The online research trend reflects not only the increased familiarity of all generations with the internet but also the growing consumerization of health care, Thomas Jeffrey, president of the Sullivan/Luallin Group, a patient experience consulting firm, told this news organization.

“According to patient satisfaction surveys, people are becoming health care consumers more than in the past,” he noted. “Historically, we didn’t look at health care as a consumer product. But, with high deductibles and copays, doctor visits can represent a pretty significant out-of-pocket expense. As it begins to hit folks’ pocketbooks, they become more savvy shoppers.”

Digital preferences for providers were gaining “positive momentum” even before the COVID-19 pandemic, but the crisis “drove upticks in some consumer digital behaviors,” the Press Ganey report pointed out.

Mr. Jeffrey agreed, noting that this finding matches what Sullivan/Luallin has discovered in its research. “I think the pandemic pushed people to engage more online,” he said. “The highest net promoter score [likelihood to recommend in market surveys] for a pharmacy is the Amazon pharmacy, which is an online-based delivery service. Then you have telehealth visits, which are more convenient in many ways.”
 

How patients search online

In choosing a new primary care doctor, 51.1% go on the web first, 23.8% seek a referral from another health care provider, and 4.4% get information from an insurer or a benefits manager, according to the survey.

The factors that matter most to consumers when they pick any provider, in order, are online ratings and reviews of the physician, referral from a current doctor, ratings and reviews of the facility, and the quality and completeness of a doctor’s profile on a website or online directory. The doctor’s online presence and the quality of their website are also important.

According to Press Ganey, search engines like Google are the most used digital resources, with 65.4% of consumers employing them to find a doctor. However, consumers now use an average of 2.7 sites in their search. The leading destinations are a hospital or a clinic site, WebMD, Healthgrades, and Facebook. (This news organization is owned by WebMD.)

Compared with 2019, the report said, there has been a 22.8% decline in the use of search engines for seeking a doctor and a 53.7% increase in the use of health care review sites such as Healthgrades and Vitals.

When reading provider reviews, consumers look for more recent reviews and want the reviews to be “authentic and informative.” They also value the star ratings. About 84%of respondents said they wouldn’t book an appointment with a referred provider that had a rating of less than four stars.

Overall, the top reasons why people are deterred from making an appointment are difficulty contacting the office, the poor quality of online reviews, and an average online rating of less than four stars.

The vast majority of respondents (77%) said they believe internet reviews reflect their own experience with a provider organization, and only 2.6% said the reviews were inaccurate. Another finding of the survey indicates that this attention of patients to reviews of their own provider doesn’t represent idle curiosity: About 57% of Baby Boomers and 45% of millennials/Gen Z’ers said they’d written online reviews of a doctor or a hospital.
 

 

 

Factors in patient loyalty

The Press Ganey survey asked which of several factors, besides excellent care, patients weighed when giving a five-star review to a health care provider.

Quality of customer service was rated first by 70.8% of respondents, followed by cleanliness of facilities (67.5%), communication (63.4%), the provider’s bedside manner (63%), ease of appointment booking (58.8%), ease of patient intake/registration (52.3%), quality and accuracy of information (40.1%), availability of telehealth services (21.7%), and waiting room amenities (21.8%).

The report explained that “quality of customer service” means “demeanor, attentiveness, and helpfulness of staff and practitioners.” “Communication” refers to things like follow-up appointment reminders and annual checkup reminders.

According to Mr. Jeffrey, these factors were considered more important than a doctor’s bedside manner because of the team care approach in most physician offices. “We see a lot more folks derive their notion of quality from continuity of care. And if they feel the physician they love is being supported by a less than competent team, that can impact significantly their sense of the quality of care,” he said.
 

Online appointment booking is a must

To win over the online consumer, Press Ganey emphasized, practices should ensure that provider listings are accurate and complete. In addition, offering online appointment booking can avoid the top challenge in making a new appointment, which is getting through to the office.

Mr. Jeffrey concurred, although he notes that practices have to be careful about how they enable patients to select appointment slots online. He suggests that an appointment request form on a patient portal first ask what the purpose of the visit is and that it offer five or so options. If the request fits into a routine visit category, the provider’s calendar pops up and the patient can select a convenient time slot. If it’s something else, an appointment scheduler calls the patient back.

“There needs to be greater access to standard appointments online,” he said. “While privacy is an issue, you can use the patient portal that most EHRs have to provide online booking. If you want to succeed going forward, that’s going to be a major plus.”

Of course, to do any of this, including reading provider reviews, a consumer needs a good internet connection and a mobile or desktop device. While broadband internet access is still not available in some communities, the breakdown of the survey respondents by demographics shows that low-income people were included.

Mr. Jeffrey doesn’t believe that a lack of internet access or digital devices prevents many Americans from going online today. “Even in poor communities, most people have internet access through their smartphones. Even baby boomers are familiar with smartphones. I haven’t seen internet access be a big barrier for low-income households, because they all have access to phones.”

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

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Metabolites implicated in CHD development in African Americans

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Wed, 12/08/2021 - 14:01

Selected metabolic biomarkers may influence disease risk and progression in African American and White persons in different ways, a cohort study of the landmark Jackson Heart Study has found.

Dr. Daniel E. Cruz

The investigators identified 22 specific metabolites that seem to influence incident CHD risk in African American patients – 13 metabolites that were also replicated in a multiethnic population and 9 novel metabolites that include N-acylamides and leucine, a branched-chain amino acid.

“To our knowledge, this is the first time that an N-acylamide as a class of molecule has been shown to be associated with incident coronary heart disease,” lead study author Daniel E. Cruz, MD, an instructor at Harvard Medical School in the division of cardiovascular medicine at Beth Israel Deaconess Medical Center in Boston, said in an interview.

The researchers analyzed targeted plasma metabolomic profiles of 2,346 participants in the Jackson Heart Study, a prospective population-based cohort study in the Mississippi city that included 5,306 African American patients evaluated over 15 years. They then performed a replication analysis of CHD-associated metabolites among 1,588 multiethnic participants in the Women’s Health Initiative, another population-based cohort study that included 161,808 postmenopausal women, also over 15 years. In all, the study, published in JAMA Cardiology, identified 46 metabolites that were associated with incident CHD up to 16 years before the incident event

Dr. Cruz said the “most interesting” findings were the roles of the N-acylamide linoleoyl ethanolamide and leucine. The former is of interest “because it is a lipid-signaling molecule that has been shown to have anti-inflammatory effects on macrophages; the influence and effects on macrophages are of particular interest because of macrophages’ central role in atherosclerosis and coronary heart disease,” he said.

Leucine draws interest because, in this study population, it was linked to a reduced risk of incident CHD. The researchers cited four previous studies in predominantly non-Hispanic White populations that found no association between branched-chain amino acids and incident CHD in Circulation, Stroke Circulation: Genomic and Precision Medicine, and Atherosclerosis. Other branched-amino acids included in the analysis trended toward a decreased risk of CHD, but those didn’t achieve the same statistical significance as that of leucine, Dr. Cruz said.

“In some of the analyses we did, there was a subset of metabolites that the associations with CHD appeared to be different between self-identified African Americans in the Jackson cohort vs. self-identified non-Hispanic Whites, and leucine was one of them,” Dr. Cruz said.

He emphasized that this study “is not a genetic analysis” because the participants self-identified their race. “So our next step is to figure out why this difference appears between these self-identified groups,” Dr. Cruz said. “We suspect environmental factors play a role – psychological stress, diet, income level, to name a few – but we are also interested to see if there are genetic causes.”

The results “are not clinically applicable,” Dr. Cruz said, but they do point to a need for more ethnically and racially diverse study populations. “The big picture is that, before we go implementing novel biomarkers into clinical practice, we need to make sure that they are accurate across different populations of people,” he said. “The only way to do this is to study different groups with the same rigor and vigor and thoughtfulness as any other group.”

These findings fall in line with other studies that found other nonmetabolomic biomarkers have countervailing effects on CHD risk in African Americans and non-Hispanic Whites, said Christie M. Ballantyne, MD, chief of cardiology at Baylor College of Medicine in Houston. For example, African Americans have been found to have lower triglyceride and HDL cholesterol levels than those of Whites.

Dr. Christie M. Ballantyne

The study “points out that there may be important biological differences in the metabolic pathways and abnormalities in the development of CHD between races,” Dr. Ballantyne said. “This further emphasizes both the importance and challenge of testing therapies in multiple racial/ethnic groups and with more even representation between men and women.”

Combining metabolomic profiling along with other biomarkers and possibly genetics may be helpful to “personalize” therapies in the future, he added.

Dr. Cruz and Dr. Ballantyne have no relevant relationships to disclose.

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Selected metabolic biomarkers may influence disease risk and progression in African American and White persons in different ways, a cohort study of the landmark Jackson Heart Study has found.

Dr. Daniel E. Cruz

The investigators identified 22 specific metabolites that seem to influence incident CHD risk in African American patients – 13 metabolites that were also replicated in a multiethnic population and 9 novel metabolites that include N-acylamides and leucine, a branched-chain amino acid.

“To our knowledge, this is the first time that an N-acylamide as a class of molecule has been shown to be associated with incident coronary heart disease,” lead study author Daniel E. Cruz, MD, an instructor at Harvard Medical School in the division of cardiovascular medicine at Beth Israel Deaconess Medical Center in Boston, said in an interview.

The researchers analyzed targeted plasma metabolomic profiles of 2,346 participants in the Jackson Heart Study, a prospective population-based cohort study in the Mississippi city that included 5,306 African American patients evaluated over 15 years. They then performed a replication analysis of CHD-associated metabolites among 1,588 multiethnic participants in the Women’s Health Initiative, another population-based cohort study that included 161,808 postmenopausal women, also over 15 years. In all, the study, published in JAMA Cardiology, identified 46 metabolites that were associated with incident CHD up to 16 years before the incident event

Dr. Cruz said the “most interesting” findings were the roles of the N-acylamide linoleoyl ethanolamide and leucine. The former is of interest “because it is a lipid-signaling molecule that has been shown to have anti-inflammatory effects on macrophages; the influence and effects on macrophages are of particular interest because of macrophages’ central role in atherosclerosis and coronary heart disease,” he said.

Leucine draws interest because, in this study population, it was linked to a reduced risk of incident CHD. The researchers cited four previous studies in predominantly non-Hispanic White populations that found no association between branched-chain amino acids and incident CHD in Circulation, Stroke Circulation: Genomic and Precision Medicine, and Atherosclerosis. Other branched-amino acids included in the analysis trended toward a decreased risk of CHD, but those didn’t achieve the same statistical significance as that of leucine, Dr. Cruz said.

“In some of the analyses we did, there was a subset of metabolites that the associations with CHD appeared to be different between self-identified African Americans in the Jackson cohort vs. self-identified non-Hispanic Whites, and leucine was one of them,” Dr. Cruz said.

He emphasized that this study “is not a genetic analysis” because the participants self-identified their race. “So our next step is to figure out why this difference appears between these self-identified groups,” Dr. Cruz said. “We suspect environmental factors play a role – psychological stress, diet, income level, to name a few – but we are also interested to see if there are genetic causes.”

The results “are not clinically applicable,” Dr. Cruz said, but they do point to a need for more ethnically and racially diverse study populations. “The big picture is that, before we go implementing novel biomarkers into clinical practice, we need to make sure that they are accurate across different populations of people,” he said. “The only way to do this is to study different groups with the same rigor and vigor and thoughtfulness as any other group.”

These findings fall in line with other studies that found other nonmetabolomic biomarkers have countervailing effects on CHD risk in African Americans and non-Hispanic Whites, said Christie M. Ballantyne, MD, chief of cardiology at Baylor College of Medicine in Houston. For example, African Americans have been found to have lower triglyceride and HDL cholesterol levels than those of Whites.

Dr. Christie M. Ballantyne

The study “points out that there may be important biological differences in the metabolic pathways and abnormalities in the development of CHD between races,” Dr. Ballantyne said. “This further emphasizes both the importance and challenge of testing therapies in multiple racial/ethnic groups and with more even representation between men and women.”

Combining metabolomic profiling along with other biomarkers and possibly genetics may be helpful to “personalize” therapies in the future, he added.

Dr. Cruz and Dr. Ballantyne have no relevant relationships to disclose.

Selected metabolic biomarkers may influence disease risk and progression in African American and White persons in different ways, a cohort study of the landmark Jackson Heart Study has found.

Dr. Daniel E. Cruz

The investigators identified 22 specific metabolites that seem to influence incident CHD risk in African American patients – 13 metabolites that were also replicated in a multiethnic population and 9 novel metabolites that include N-acylamides and leucine, a branched-chain amino acid.

“To our knowledge, this is the first time that an N-acylamide as a class of molecule has been shown to be associated with incident coronary heart disease,” lead study author Daniel E. Cruz, MD, an instructor at Harvard Medical School in the division of cardiovascular medicine at Beth Israel Deaconess Medical Center in Boston, said in an interview.

The researchers analyzed targeted plasma metabolomic profiles of 2,346 participants in the Jackson Heart Study, a prospective population-based cohort study in the Mississippi city that included 5,306 African American patients evaluated over 15 years. They then performed a replication analysis of CHD-associated metabolites among 1,588 multiethnic participants in the Women’s Health Initiative, another population-based cohort study that included 161,808 postmenopausal women, also over 15 years. In all, the study, published in JAMA Cardiology, identified 46 metabolites that were associated with incident CHD up to 16 years before the incident event

Dr. Cruz said the “most interesting” findings were the roles of the N-acylamide linoleoyl ethanolamide and leucine. The former is of interest “because it is a lipid-signaling molecule that has been shown to have anti-inflammatory effects on macrophages; the influence and effects on macrophages are of particular interest because of macrophages’ central role in atherosclerosis and coronary heart disease,” he said.

Leucine draws interest because, in this study population, it was linked to a reduced risk of incident CHD. The researchers cited four previous studies in predominantly non-Hispanic White populations that found no association between branched-chain amino acids and incident CHD in Circulation, Stroke Circulation: Genomic and Precision Medicine, and Atherosclerosis. Other branched-amino acids included in the analysis trended toward a decreased risk of CHD, but those didn’t achieve the same statistical significance as that of leucine, Dr. Cruz said.

“In some of the analyses we did, there was a subset of metabolites that the associations with CHD appeared to be different between self-identified African Americans in the Jackson cohort vs. self-identified non-Hispanic Whites, and leucine was one of them,” Dr. Cruz said.

He emphasized that this study “is not a genetic analysis” because the participants self-identified their race. “So our next step is to figure out why this difference appears between these self-identified groups,” Dr. Cruz said. “We suspect environmental factors play a role – psychological stress, diet, income level, to name a few – but we are also interested to see if there are genetic causes.”

The results “are not clinically applicable,” Dr. Cruz said, but they do point to a need for more ethnically and racially diverse study populations. “The big picture is that, before we go implementing novel biomarkers into clinical practice, we need to make sure that they are accurate across different populations of people,” he said. “The only way to do this is to study different groups with the same rigor and vigor and thoughtfulness as any other group.”

These findings fall in line with other studies that found other nonmetabolomic biomarkers have countervailing effects on CHD risk in African Americans and non-Hispanic Whites, said Christie M. Ballantyne, MD, chief of cardiology at Baylor College of Medicine in Houston. For example, African Americans have been found to have lower triglyceride and HDL cholesterol levels than those of Whites.

Dr. Christie M. Ballantyne

The study “points out that there may be important biological differences in the metabolic pathways and abnormalities in the development of CHD between races,” Dr. Ballantyne said. “This further emphasizes both the importance and challenge of testing therapies in multiple racial/ethnic groups and with more even representation between men and women.”

Combining metabolomic profiling along with other biomarkers and possibly genetics may be helpful to “personalize” therapies in the future, he added.

Dr. Cruz and Dr. Ballantyne have no relevant relationships to disclose.

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FROM JAMA CARDIOLOGY

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Treating ALL: Asparaginase enzyme levels linked to toxicities

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Tue, 12/07/2021 - 10:04

Key toxicities related to treating acute lymphoblastic leukemia (ALL) with asparaginase, specifically pancreatitis and osteonecrosis, are associated with increases in asparaginase enzyme activity, suggesting that patients at risk for those toxicities would benefit from therapeutic drug monitoring, according to new research.

In the study, published Oct. 8 in Blood Advances, increased asparaginase enzyme activity was not significantly associated with overall asparaginase toxicity. However,“ a significant association between increasing asparaginase enzyme activity levels and pancreatitis and osteonecrosis was found, which should be taken into consideration when future treatment protocols are designed,” the authors concluded.

“The [findings are] new, and we have included patients from a quite big cohort, which is unique,” coauthor Birgitte Klug Albertsen, MD, PhD, associate clinical professor with Aarhus (Denmark) University Hospital, told this news organization.

Therapeutic drug monitoring already is widely used during treatment with asparaginase, the standard of care treatment for ALL; however, the focus of this monitoring has typically been on clinical effectiveness, as levels of asparaginase enzyme activity can indicate hypersensitivity reactions, while the absence of such activity can suggest inferior outcomes.

Meanwhile, drug monitoring is not normally used to assess the risk of treatment-related toxicities. This has been due to a lack of evidence regarding asparaginase enzyme activity and toxicity risk, which, if severe enough, can prevent further treatment.

To investigate the relationship with toxicities, Dr. Albertsen and colleagues evaluated data from seven countries in Europe on 1,155 children between the ages of 1 and 17.9 who were diagnosed with ALL and treated with asparaginase, according to the Nordic Society of Paediatric Haematology and Oncology (NOPHO) ALL2008 protocol between July 2008 and March 2016.

Blood samples drawn approximately 14 days after asparaginase administration showed that some level of asparaginase enzyme activity was measurable in 955 patients (82.7%), while 200 patients (17.3%) had asparaginase inactivation. Overall, there were 159 asparaginase-associated toxicities of pancreatitis, thromboembolism, or osteonecrosis among the 955 patients with measurable asparaginase enzyme activity.

There were no significant differences in median asparaginase enzyme activity levels between those who did and did not experience toxicities (224 IU/L vs. 221 IU/L, respectively; P = .1), and the results did not change after adjustment for age and sex. However, the risk of pancreatitis was found to increase with a hazard ratio (HR) of 1.40 for each 100 IU/L increase in the median asparaginase enzyme activity level (P = .002).

Likewise, an increase in risk was observed for osteonecrosis (HR 1.36; P = .02) per 100 IU/L increase in median asparaginase enzyme activity. However, the HR for the risk of thromboembolism, the most common of asparaginase-related toxicities, was not significant (HR 0.99; P = .96).

Dr. Albertsen said the etiology behind the occurrence of osteonecrosis is not well understood.

“We know that steroids, especially dexamethasone, are a risk factor,” she said. “We believe that asparaginase may play a role too, but a clear association has not been demonstrated.”

In the NOPHO ALL2008 protocol used in the study, dexamethasone is used in the same time periods as PEG-asparaginase treatment for patients receiving 15 doses.

The finding of only a nonsignificant trend between asparaginase enzyme activity with overall toxicities may have reflected the low dose that was used, Dr. Albertsen added.

“In the NOPHO ALL2008 protocol, we used quite a low dose of PEG-asparaginase, and the risk may be higher in protocols using higher doses,” she said.
 

Relapse reduction

Notably, the study showed that asparaginase enzyme elevations were, in fact, not significantly associated with a reduction in the risk of leukemic relapse (HR .88 per 100 IU/L increase; P = .35).

Those findings suggest that measurable asparaginase enzyme activity levels, and thus asparaginase depletion, “may be sufficient to ensure an antileukemic effect,” the authors noted.

“Therapeutic drug monitoring of asparaginase enzyme activity is indicated mainly to detect inactivation, but [it] may be further explored for dose reduction to reduce some specific toxicities,” they concluded.

Dr. Albertsen disclosed being sponsor of the investigator-initiatied NOR-GRASPALL 2016 trial.

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Key toxicities related to treating acute lymphoblastic leukemia (ALL) with asparaginase, specifically pancreatitis and osteonecrosis, are associated with increases in asparaginase enzyme activity, suggesting that patients at risk for those toxicities would benefit from therapeutic drug monitoring, according to new research.

In the study, published Oct. 8 in Blood Advances, increased asparaginase enzyme activity was not significantly associated with overall asparaginase toxicity. However,“ a significant association between increasing asparaginase enzyme activity levels and pancreatitis and osteonecrosis was found, which should be taken into consideration when future treatment protocols are designed,” the authors concluded.

“The [findings are] new, and we have included patients from a quite big cohort, which is unique,” coauthor Birgitte Klug Albertsen, MD, PhD, associate clinical professor with Aarhus (Denmark) University Hospital, told this news organization.

Therapeutic drug monitoring already is widely used during treatment with asparaginase, the standard of care treatment for ALL; however, the focus of this monitoring has typically been on clinical effectiveness, as levels of asparaginase enzyme activity can indicate hypersensitivity reactions, while the absence of such activity can suggest inferior outcomes.

Meanwhile, drug monitoring is not normally used to assess the risk of treatment-related toxicities. This has been due to a lack of evidence regarding asparaginase enzyme activity and toxicity risk, which, if severe enough, can prevent further treatment.

To investigate the relationship with toxicities, Dr. Albertsen and colleagues evaluated data from seven countries in Europe on 1,155 children between the ages of 1 and 17.9 who were diagnosed with ALL and treated with asparaginase, according to the Nordic Society of Paediatric Haematology and Oncology (NOPHO) ALL2008 protocol between July 2008 and March 2016.

Blood samples drawn approximately 14 days after asparaginase administration showed that some level of asparaginase enzyme activity was measurable in 955 patients (82.7%), while 200 patients (17.3%) had asparaginase inactivation. Overall, there were 159 asparaginase-associated toxicities of pancreatitis, thromboembolism, or osteonecrosis among the 955 patients with measurable asparaginase enzyme activity.

There were no significant differences in median asparaginase enzyme activity levels between those who did and did not experience toxicities (224 IU/L vs. 221 IU/L, respectively; P = .1), and the results did not change after adjustment for age and sex. However, the risk of pancreatitis was found to increase with a hazard ratio (HR) of 1.40 for each 100 IU/L increase in the median asparaginase enzyme activity level (P = .002).

Likewise, an increase in risk was observed for osteonecrosis (HR 1.36; P = .02) per 100 IU/L increase in median asparaginase enzyme activity. However, the HR for the risk of thromboembolism, the most common of asparaginase-related toxicities, was not significant (HR 0.99; P = .96).

Dr. Albertsen said the etiology behind the occurrence of osteonecrosis is not well understood.

“We know that steroids, especially dexamethasone, are a risk factor,” she said. “We believe that asparaginase may play a role too, but a clear association has not been demonstrated.”

In the NOPHO ALL2008 protocol used in the study, dexamethasone is used in the same time periods as PEG-asparaginase treatment for patients receiving 15 doses.

The finding of only a nonsignificant trend between asparaginase enzyme activity with overall toxicities may have reflected the low dose that was used, Dr. Albertsen added.

“In the NOPHO ALL2008 protocol, we used quite a low dose of PEG-asparaginase, and the risk may be higher in protocols using higher doses,” she said.
 

Relapse reduction

Notably, the study showed that asparaginase enzyme elevations were, in fact, not significantly associated with a reduction in the risk of leukemic relapse (HR .88 per 100 IU/L increase; P = .35).

Those findings suggest that measurable asparaginase enzyme activity levels, and thus asparaginase depletion, “may be sufficient to ensure an antileukemic effect,” the authors noted.

“Therapeutic drug monitoring of asparaginase enzyme activity is indicated mainly to detect inactivation, but [it] may be further explored for dose reduction to reduce some specific toxicities,” they concluded.

Dr. Albertsen disclosed being sponsor of the investigator-initiatied NOR-GRASPALL 2016 trial.

Key toxicities related to treating acute lymphoblastic leukemia (ALL) with asparaginase, specifically pancreatitis and osteonecrosis, are associated with increases in asparaginase enzyme activity, suggesting that patients at risk for those toxicities would benefit from therapeutic drug monitoring, according to new research.

In the study, published Oct. 8 in Blood Advances, increased asparaginase enzyme activity was not significantly associated with overall asparaginase toxicity. However,“ a significant association between increasing asparaginase enzyme activity levels and pancreatitis and osteonecrosis was found, which should be taken into consideration when future treatment protocols are designed,” the authors concluded.

“The [findings are] new, and we have included patients from a quite big cohort, which is unique,” coauthor Birgitte Klug Albertsen, MD, PhD, associate clinical professor with Aarhus (Denmark) University Hospital, told this news organization.

Therapeutic drug monitoring already is widely used during treatment with asparaginase, the standard of care treatment for ALL; however, the focus of this monitoring has typically been on clinical effectiveness, as levels of asparaginase enzyme activity can indicate hypersensitivity reactions, while the absence of such activity can suggest inferior outcomes.

Meanwhile, drug monitoring is not normally used to assess the risk of treatment-related toxicities. This has been due to a lack of evidence regarding asparaginase enzyme activity and toxicity risk, which, if severe enough, can prevent further treatment.

To investigate the relationship with toxicities, Dr. Albertsen and colleagues evaluated data from seven countries in Europe on 1,155 children between the ages of 1 and 17.9 who were diagnosed with ALL and treated with asparaginase, according to the Nordic Society of Paediatric Haematology and Oncology (NOPHO) ALL2008 protocol between July 2008 and March 2016.

Blood samples drawn approximately 14 days after asparaginase administration showed that some level of asparaginase enzyme activity was measurable in 955 patients (82.7%), while 200 patients (17.3%) had asparaginase inactivation. Overall, there were 159 asparaginase-associated toxicities of pancreatitis, thromboembolism, or osteonecrosis among the 955 patients with measurable asparaginase enzyme activity.

There were no significant differences in median asparaginase enzyme activity levels between those who did and did not experience toxicities (224 IU/L vs. 221 IU/L, respectively; P = .1), and the results did not change after adjustment for age and sex. However, the risk of pancreatitis was found to increase with a hazard ratio (HR) of 1.40 for each 100 IU/L increase in the median asparaginase enzyme activity level (P = .002).

Likewise, an increase in risk was observed for osteonecrosis (HR 1.36; P = .02) per 100 IU/L increase in median asparaginase enzyme activity. However, the HR for the risk of thromboembolism, the most common of asparaginase-related toxicities, was not significant (HR 0.99; P = .96).

Dr. Albertsen said the etiology behind the occurrence of osteonecrosis is not well understood.

“We know that steroids, especially dexamethasone, are a risk factor,” she said. “We believe that asparaginase may play a role too, but a clear association has not been demonstrated.”

In the NOPHO ALL2008 protocol used in the study, dexamethasone is used in the same time periods as PEG-asparaginase treatment for patients receiving 15 doses.

The finding of only a nonsignificant trend between asparaginase enzyme activity with overall toxicities may have reflected the low dose that was used, Dr. Albertsen added.

“In the NOPHO ALL2008 protocol, we used quite a low dose of PEG-asparaginase, and the risk may be higher in protocols using higher doses,” she said.
 

Relapse reduction

Notably, the study showed that asparaginase enzyme elevations were, in fact, not significantly associated with a reduction in the risk of leukemic relapse (HR .88 per 100 IU/L increase; P = .35).

Those findings suggest that measurable asparaginase enzyme activity levels, and thus asparaginase depletion, “may be sufficient to ensure an antileukemic effect,” the authors noted.

“Therapeutic drug monitoring of asparaginase enzyme activity is indicated mainly to detect inactivation, but [it] may be further explored for dose reduction to reduce some specific toxicities,” they concluded.

Dr. Albertsen disclosed being sponsor of the investigator-initiatied NOR-GRASPALL 2016 trial.

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New AKI risk score for PCI patients passes validation

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A pair of updated scoring models for estimating a patient’s risk for contrast-associated acute kidney injury during and immediately after percutaneous coronary intervention worked better than a widely used prior version in initial validation testing using data collected at a single U.S. tertiary-care hospital.

Mount Sinai Medical Center
Dr. Roxana Mehran

While the two new risk scores looked promising, they need further, external validation with additional, diverse patient cohorts, Roxana Mehran, MD, cautioned at the American Heart Association scientific sessions.

“Don’t change anything until we externally validate this,” urged Dr. Mehran, professor and director of the Center for Interventional Cardiovascular Research and Clinical Trials at Icahn School of Medicine at Mount Sinai in New York. External validation of the two new risk scores is in progress with planned reporting of the results in 2022, she said in an interview.

One of the two new algorithms, which both predict a patient’s risk for developing acute kidney injury (AKI) as a result of receiving iodinated contrast media within 48 hours of a percutaneous coronary intervention (PCI), relies on eight easily available variables taken from a patient’s medical record just prior to undergoing PCI: age, type of coronary disease (ranging from asymptomatic or stable angina to ST-segment elevation MI), estimated glomerular filtration rate, left ventricular ejection fraction, diabetes, hemoglobin, basal glucose, and heart failure.

This risk score accounted for 72% (a C-statistic of 0.72) of the observed AKI episodes during the derivation phase, which used data from 14,616 consecutive Mount Sinai patients who underwent PCI during January 2012–December 2017.

Internal validation using data from 5,606 consecutive Mount Sinai patients who underwent PCI during January 2018–December 2020 showed that the eight-item formula accounted for 84% of all incident AKI events occurring during or within 48 hours of a PCI procedure.
 

Accounting for periprocedural variables

A second risk score included the eight preprocedural variables plus four additional periprocedural variables: complex PCI anatomy, contrast volume during the procedure, development of a periprocedural bleed, and having slow or no reflow into affected coronaries (less than TIMI grade 3 flow) immediately after the procedure. The second model produced a C-statistic of 0.74 during derivation and accounted for 86% of incident AKI events in the validation analysis.

The data Dr. Mehran reported appeared in The Lancet .

She and her coauthors designed these two new algorithms to replace a “widely used” and externally validated risk score that Dr. Mehran and associates introduced in 2004. Despite its merits, the 17-year-old scoring formula has limitations including “low discrimination” with a C-statistic of 0.67, derivation from data that’s now 20 years old, and exclusion of patients with ST-elevation MIs, the authors said in the new report.

Dr. Mehran encouraged interventional cardiologists to use both new risk scores (once externally validated) when possible.

The eight-item preprocedural model “gives clinicians an idea about a patient’s risk [for incident AKI] before they go into the catheterization laboratory,” and then they can further refine the risk assessment during the procedure based on the four periprocedural risk factors, she explained. The goal is to target “tailored preventive strategies” to patients identified by the scoring algorithms as being at high risk for AKI.
 

 

 

A role for preventive measures

Preventive strategies to consider for higher-risk patients include limiting the administered volume of iodinated contrast media, increasing hydration, and avoiding nephrotoxic agents, Dr. Mehran said. The two new risk-assessment tools will “allow for better evaluation of PCI patients” when testing “innovative strategies and treatments” designed to help avoid contrast-associated AKI.

“The focus to date has been on measures to protect renal function from contrast media, based on indirect data,” Estelle C. Nijssen, MSc, and Joachim E. Wildberger, MD, wrote in an editorial that accompanied the published report. “The effect of prophylactic measures on longer-term averse outcomes is still unclear,” they noted. “Perhaps our focus should shift from contrast and renal function to the heart, the role of which has probably been undervalued in this setting,” wrote Ms. Nijssen, a researcher at Maastricht (The Netherlands) University, and Dr. Wildberger, professor and chairman of the department of radiology at Maastricht University.

The editorial’s authors noted that the two new risk scores have the advantage of relying on variables that are “readily available in clinical practice.” But they also noted several limitations, such as the model’s development from largely low-risk patients who had a low, roughly 30% prevalence of chronic kidney disease. During 9 full years studied, 2012-2020, the annual incidence of AKI showed a downward trend, with an incidence of just over 3% in 2020.

Dr. Mehran attributed this decline in AKI to “great work identifying high-risk patients” and using the prophylactic measures she cited. But even when occurring at relatively low incidence, “AKI is still an important complication that is associated with mortality post PCI,” she stressed.

Establishing a safe contrast dose

“The study is great, and helps reinforce the risk factors that are most important to consider when risk stratifying patients prior to PCI,” said Neal Yuan, MD, a cardiologist at the University of California, San Francisco, who has studied contrast-associated AKI in patients who undergo PCI. The report from Dr. Mehran also “confirms in a large cohort the association between contrast-associated AKI and death,” and describes “an easy method for calculating risk,” he said in an interview.

Dr. Yuan agreed on the need for external validation, and once adequately validated he called for incorporation of the risk score into EHRs. Another important issue for future study is “how much [AKI] risk is too much risk,” he said.

The risk factors identified in Dr. Mehran’s report “are some of the same ones identified in previous studies. Even though this was a more contemporary dataset, there is not a ton of new [findings]; it mainly strengthens findings from prior studies.”

Results published by Dr. Yuan and his associates in 2020 used data from more than 20,000 U.S. patients who underwent PCI to try to identify a generally safe upper limit for the dose of iodinated contrast.

The main purpose for performing AKI risk stratification on PCI patients is to “identify high-risk patients and use preventive strategies when treating these patients.” Current AKI preventive strategies “mainly fall into intravascular volume expansion, and reduced contrast.” What’s less clear is “how to operationalize reduced contrast,” he said.

The report by Dr. Yuan showed that “about 10% of PCI patients were at very high risk” for contrast-associated AKI “no matter what is done.” In contrast, about two-thirds of PCI patients “could receive lots of contrast and still be very unlikely to develop AKI,” Dr. Yuan said.

He voiced some skepticism about the willingness of many clinicians to routinely use a formal risk score to assess their patients scheduled for PCI.

Most operators “approximate AKI risk based on variables such as age and creatinine level, but few take time to put the variables into a calculator to get an exact risk number.” In a “small survey” he ran, he found that these rough approximations often ignore important risk factors like hemoglobin level. This inertia by clinicians against routinely using a risk score could be addressed, at least in part, by integrating the risk score into an EHR for automatic calculation, Dr. Yuan suggested.

Dr. Mehran noted that the risk score that she introduced in 2004 is used “in many EHRs to identify high-risk patients.”

The current study received no commercial or external funding. Dr. Mehran has been a consultant to Boston Scientific, Cine-Med Research, CIRM, and Janssen, and she holds equity in Applied Therapeutics, Elixir Medical, and STEL. Dr. Wildberger had no relevant disclosures. Ms. Nijssen and Dr. Yuan had no disclosures.

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A pair of updated scoring models for estimating a patient’s risk for contrast-associated acute kidney injury during and immediately after percutaneous coronary intervention worked better than a widely used prior version in initial validation testing using data collected at a single U.S. tertiary-care hospital.

Mount Sinai Medical Center
Dr. Roxana Mehran

While the two new risk scores looked promising, they need further, external validation with additional, diverse patient cohorts, Roxana Mehran, MD, cautioned at the American Heart Association scientific sessions.

“Don’t change anything until we externally validate this,” urged Dr. Mehran, professor and director of the Center for Interventional Cardiovascular Research and Clinical Trials at Icahn School of Medicine at Mount Sinai in New York. External validation of the two new risk scores is in progress with planned reporting of the results in 2022, she said in an interview.

One of the two new algorithms, which both predict a patient’s risk for developing acute kidney injury (AKI) as a result of receiving iodinated contrast media within 48 hours of a percutaneous coronary intervention (PCI), relies on eight easily available variables taken from a patient’s medical record just prior to undergoing PCI: age, type of coronary disease (ranging from asymptomatic or stable angina to ST-segment elevation MI), estimated glomerular filtration rate, left ventricular ejection fraction, diabetes, hemoglobin, basal glucose, and heart failure.

This risk score accounted for 72% (a C-statistic of 0.72) of the observed AKI episodes during the derivation phase, which used data from 14,616 consecutive Mount Sinai patients who underwent PCI during January 2012–December 2017.

Internal validation using data from 5,606 consecutive Mount Sinai patients who underwent PCI during January 2018–December 2020 showed that the eight-item formula accounted for 84% of all incident AKI events occurring during or within 48 hours of a PCI procedure.
 

Accounting for periprocedural variables

A second risk score included the eight preprocedural variables plus four additional periprocedural variables: complex PCI anatomy, contrast volume during the procedure, development of a periprocedural bleed, and having slow or no reflow into affected coronaries (less than TIMI grade 3 flow) immediately after the procedure. The second model produced a C-statistic of 0.74 during derivation and accounted for 86% of incident AKI events in the validation analysis.

The data Dr. Mehran reported appeared in The Lancet .

She and her coauthors designed these two new algorithms to replace a “widely used” and externally validated risk score that Dr. Mehran and associates introduced in 2004. Despite its merits, the 17-year-old scoring formula has limitations including “low discrimination” with a C-statistic of 0.67, derivation from data that’s now 20 years old, and exclusion of patients with ST-elevation MIs, the authors said in the new report.

Dr. Mehran encouraged interventional cardiologists to use both new risk scores (once externally validated) when possible.

The eight-item preprocedural model “gives clinicians an idea about a patient’s risk [for incident AKI] before they go into the catheterization laboratory,” and then they can further refine the risk assessment during the procedure based on the four periprocedural risk factors, she explained. The goal is to target “tailored preventive strategies” to patients identified by the scoring algorithms as being at high risk for AKI.
 

 

 

A role for preventive measures

Preventive strategies to consider for higher-risk patients include limiting the administered volume of iodinated contrast media, increasing hydration, and avoiding nephrotoxic agents, Dr. Mehran said. The two new risk-assessment tools will “allow for better evaluation of PCI patients” when testing “innovative strategies and treatments” designed to help avoid contrast-associated AKI.

“The focus to date has been on measures to protect renal function from contrast media, based on indirect data,” Estelle C. Nijssen, MSc, and Joachim E. Wildberger, MD, wrote in an editorial that accompanied the published report. “The effect of prophylactic measures on longer-term averse outcomes is still unclear,” they noted. “Perhaps our focus should shift from contrast and renal function to the heart, the role of which has probably been undervalued in this setting,” wrote Ms. Nijssen, a researcher at Maastricht (The Netherlands) University, and Dr. Wildberger, professor and chairman of the department of radiology at Maastricht University.

The editorial’s authors noted that the two new risk scores have the advantage of relying on variables that are “readily available in clinical practice.” But they also noted several limitations, such as the model’s development from largely low-risk patients who had a low, roughly 30% prevalence of chronic kidney disease. During 9 full years studied, 2012-2020, the annual incidence of AKI showed a downward trend, with an incidence of just over 3% in 2020.

Dr. Mehran attributed this decline in AKI to “great work identifying high-risk patients” and using the prophylactic measures she cited. But even when occurring at relatively low incidence, “AKI is still an important complication that is associated with mortality post PCI,” she stressed.

Establishing a safe contrast dose

“The study is great, and helps reinforce the risk factors that are most important to consider when risk stratifying patients prior to PCI,” said Neal Yuan, MD, a cardiologist at the University of California, San Francisco, who has studied contrast-associated AKI in patients who undergo PCI. The report from Dr. Mehran also “confirms in a large cohort the association between contrast-associated AKI and death,” and describes “an easy method for calculating risk,” he said in an interview.

Dr. Yuan agreed on the need for external validation, and once adequately validated he called for incorporation of the risk score into EHRs. Another important issue for future study is “how much [AKI] risk is too much risk,” he said.

The risk factors identified in Dr. Mehran’s report “are some of the same ones identified in previous studies. Even though this was a more contemporary dataset, there is not a ton of new [findings]; it mainly strengthens findings from prior studies.”

Results published by Dr. Yuan and his associates in 2020 used data from more than 20,000 U.S. patients who underwent PCI to try to identify a generally safe upper limit for the dose of iodinated contrast.

The main purpose for performing AKI risk stratification on PCI patients is to “identify high-risk patients and use preventive strategies when treating these patients.” Current AKI preventive strategies “mainly fall into intravascular volume expansion, and reduced contrast.” What’s less clear is “how to operationalize reduced contrast,” he said.

The report by Dr. Yuan showed that “about 10% of PCI patients were at very high risk” for contrast-associated AKI “no matter what is done.” In contrast, about two-thirds of PCI patients “could receive lots of contrast and still be very unlikely to develop AKI,” Dr. Yuan said.

He voiced some skepticism about the willingness of many clinicians to routinely use a formal risk score to assess their patients scheduled for PCI.

Most operators “approximate AKI risk based on variables such as age and creatinine level, but few take time to put the variables into a calculator to get an exact risk number.” In a “small survey” he ran, he found that these rough approximations often ignore important risk factors like hemoglobin level. This inertia by clinicians against routinely using a risk score could be addressed, at least in part, by integrating the risk score into an EHR for automatic calculation, Dr. Yuan suggested.

Dr. Mehran noted that the risk score that she introduced in 2004 is used “in many EHRs to identify high-risk patients.”

The current study received no commercial or external funding. Dr. Mehran has been a consultant to Boston Scientific, Cine-Med Research, CIRM, and Janssen, and she holds equity in Applied Therapeutics, Elixir Medical, and STEL. Dr. Wildberger had no relevant disclosures. Ms. Nijssen and Dr. Yuan had no disclosures.

A pair of updated scoring models for estimating a patient’s risk for contrast-associated acute kidney injury during and immediately after percutaneous coronary intervention worked better than a widely used prior version in initial validation testing using data collected at a single U.S. tertiary-care hospital.

Mount Sinai Medical Center
Dr. Roxana Mehran

While the two new risk scores looked promising, they need further, external validation with additional, diverse patient cohorts, Roxana Mehran, MD, cautioned at the American Heart Association scientific sessions.

“Don’t change anything until we externally validate this,” urged Dr. Mehran, professor and director of the Center for Interventional Cardiovascular Research and Clinical Trials at Icahn School of Medicine at Mount Sinai in New York. External validation of the two new risk scores is in progress with planned reporting of the results in 2022, she said in an interview.

One of the two new algorithms, which both predict a patient’s risk for developing acute kidney injury (AKI) as a result of receiving iodinated contrast media within 48 hours of a percutaneous coronary intervention (PCI), relies on eight easily available variables taken from a patient’s medical record just prior to undergoing PCI: age, type of coronary disease (ranging from asymptomatic or stable angina to ST-segment elevation MI), estimated glomerular filtration rate, left ventricular ejection fraction, diabetes, hemoglobin, basal glucose, and heart failure.

This risk score accounted for 72% (a C-statistic of 0.72) of the observed AKI episodes during the derivation phase, which used data from 14,616 consecutive Mount Sinai patients who underwent PCI during January 2012–December 2017.

Internal validation using data from 5,606 consecutive Mount Sinai patients who underwent PCI during January 2018–December 2020 showed that the eight-item formula accounted for 84% of all incident AKI events occurring during or within 48 hours of a PCI procedure.
 

Accounting for periprocedural variables

A second risk score included the eight preprocedural variables plus four additional periprocedural variables: complex PCI anatomy, contrast volume during the procedure, development of a periprocedural bleed, and having slow or no reflow into affected coronaries (less than TIMI grade 3 flow) immediately after the procedure. The second model produced a C-statistic of 0.74 during derivation and accounted for 86% of incident AKI events in the validation analysis.

The data Dr. Mehran reported appeared in The Lancet .

She and her coauthors designed these two new algorithms to replace a “widely used” and externally validated risk score that Dr. Mehran and associates introduced in 2004. Despite its merits, the 17-year-old scoring formula has limitations including “low discrimination” with a C-statistic of 0.67, derivation from data that’s now 20 years old, and exclusion of patients with ST-elevation MIs, the authors said in the new report.

Dr. Mehran encouraged interventional cardiologists to use both new risk scores (once externally validated) when possible.

The eight-item preprocedural model “gives clinicians an idea about a patient’s risk [for incident AKI] before they go into the catheterization laboratory,” and then they can further refine the risk assessment during the procedure based on the four periprocedural risk factors, she explained. The goal is to target “tailored preventive strategies” to patients identified by the scoring algorithms as being at high risk for AKI.
 

 

 

A role for preventive measures

Preventive strategies to consider for higher-risk patients include limiting the administered volume of iodinated contrast media, increasing hydration, and avoiding nephrotoxic agents, Dr. Mehran said. The two new risk-assessment tools will “allow for better evaluation of PCI patients” when testing “innovative strategies and treatments” designed to help avoid contrast-associated AKI.

“The focus to date has been on measures to protect renal function from contrast media, based on indirect data,” Estelle C. Nijssen, MSc, and Joachim E. Wildberger, MD, wrote in an editorial that accompanied the published report. “The effect of prophylactic measures on longer-term averse outcomes is still unclear,” they noted. “Perhaps our focus should shift from contrast and renal function to the heart, the role of which has probably been undervalued in this setting,” wrote Ms. Nijssen, a researcher at Maastricht (The Netherlands) University, and Dr. Wildberger, professor and chairman of the department of radiology at Maastricht University.

The editorial’s authors noted that the two new risk scores have the advantage of relying on variables that are “readily available in clinical practice.” But they also noted several limitations, such as the model’s development from largely low-risk patients who had a low, roughly 30% prevalence of chronic kidney disease. During 9 full years studied, 2012-2020, the annual incidence of AKI showed a downward trend, with an incidence of just over 3% in 2020.

Dr. Mehran attributed this decline in AKI to “great work identifying high-risk patients” and using the prophylactic measures she cited. But even when occurring at relatively low incidence, “AKI is still an important complication that is associated with mortality post PCI,” she stressed.

Establishing a safe contrast dose

“The study is great, and helps reinforce the risk factors that are most important to consider when risk stratifying patients prior to PCI,” said Neal Yuan, MD, a cardiologist at the University of California, San Francisco, who has studied contrast-associated AKI in patients who undergo PCI. The report from Dr. Mehran also “confirms in a large cohort the association between contrast-associated AKI and death,” and describes “an easy method for calculating risk,” he said in an interview.

Dr. Yuan agreed on the need for external validation, and once adequately validated he called for incorporation of the risk score into EHRs. Another important issue for future study is “how much [AKI] risk is too much risk,” he said.

The risk factors identified in Dr. Mehran’s report “are some of the same ones identified in previous studies. Even though this was a more contemporary dataset, there is not a ton of new [findings]; it mainly strengthens findings from prior studies.”

Results published by Dr. Yuan and his associates in 2020 used data from more than 20,000 U.S. patients who underwent PCI to try to identify a generally safe upper limit for the dose of iodinated contrast.

The main purpose for performing AKI risk stratification on PCI patients is to “identify high-risk patients and use preventive strategies when treating these patients.” Current AKI preventive strategies “mainly fall into intravascular volume expansion, and reduced contrast.” What’s less clear is “how to operationalize reduced contrast,” he said.

The report by Dr. Yuan showed that “about 10% of PCI patients were at very high risk” for contrast-associated AKI “no matter what is done.” In contrast, about two-thirds of PCI patients “could receive lots of contrast and still be very unlikely to develop AKI,” Dr. Yuan said.

He voiced some skepticism about the willingness of many clinicians to routinely use a formal risk score to assess their patients scheduled for PCI.

Most operators “approximate AKI risk based on variables such as age and creatinine level, but few take time to put the variables into a calculator to get an exact risk number.” In a “small survey” he ran, he found that these rough approximations often ignore important risk factors like hemoglobin level. This inertia by clinicians against routinely using a risk score could be addressed, at least in part, by integrating the risk score into an EHR for automatic calculation, Dr. Yuan suggested.

Dr. Mehran noted that the risk score that she introduced in 2004 is used “in many EHRs to identify high-risk patients.”

The current study received no commercial or external funding. Dr. Mehran has been a consultant to Boston Scientific, Cine-Med Research, CIRM, and Janssen, and she holds equity in Applied Therapeutics, Elixir Medical, and STEL. Dr. Wildberger had no relevant disclosures. Ms. Nijssen and Dr. Yuan had no disclosures.

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Peripheral manifestations make mark on spondyloarthritis trajectory

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Tue, 12/07/2021 - 09:28

Peripheral manifestations contribute significantly to disease activity in adults with spondyloarthritis (SpA), point toward a generally worse prognosis, and play a big role in defining the phenotypic clustering of the heterogenous disease, according to findings from what researchers called the first prospective study “to comprehensively describe the prevalence, clinical patterns, and prognostic implications of peripheral manifestations across the entire SpA spectrum.”

The stratification of patients in the study based on the presence of peripheral manifestations (arthritis, enthesitis, and/or dactylitis) led to the identification of an endotype with unfavorable outcomes, which not only has prognostic value but supports the need for an endotype-based treatment approach rather than one centered on C-reactive protein (CRP) and the Ankylosing Spondylitis Disease Activity Score (ASDAS).

The findings “advocate strongly for the presence of a distinct SpA endotype, based on potentially different immunopathological mechanisms and characterized by high disease activity at initial presentation with lack of substantial improvement upon follow-up,” first author Ann-Sophie De Craemer, MD, of Ghent, Belgium, and colleagues wrote.

Because the diagnostic and prognostic value of peripheral manifestations has not been well studied in SpA in general and in newly diagnosed patients in particular, Dr. De Craemer and associates decided to analyze their impact in 367 patients in the Be-Giant (BelGian Inflammatory Arthritis and spoNdylitis cohorT) cohort, a multicenter, prospective, observational cohort of newly diagnosed patients with SpA in Belgium. The study was published in Rheumatology.

The study population included 257 (70%) patients with axial-predominant SpA (axSpA) as classified by Assessment of Spondyloarthritis International Society criteria and 110 (30%) with peripheral-predominant SpA (pSpA) as defined by ASAS criteria. A total of 52 patients with axSpA had peripheral manifestations at baseline. The mean age of the patients was 34 years, and 52% were male.

The 162 patients with peripheral manifestations included 143 with arthritis, 52 with enthesitis, and 55 with dactylitis.

Two patient clusters emerge from data

In a cluster analysis that used baseline clinical features, the researchers divided the patients into cluster A (of which 242 of 248 were patients with axSpA) and cluster B (of which 104 of 119 were patients with pSpA). Most of the patients with peripheral manifestations were in cluster B (117 of 162 [72%]), compared with cluster A (45 of 162 [28%]).

A longitudinal analysis included 195 patients who completed a minimum 2-year follow-up. The longitudinal analysis identified high- and low-disease activity trajectories in each cluster.

In axSpA-predominant cluster A, patients with “high” trajectory had high disease activity levels at baseline (mean ASDAS-CRP, 3.2) that remained relatively stable, while those in the low-trajectory group (62%) had less disease activity at baseline (mean ASDAS-CRP, 2.0), which then further declined during follow-up.

Patients in the high trajectory in cluster A were more often affected by peripheral manifestations, “which remained a significant predictor in multivariate analysis,” with an odds ratio of 2.4, the researchers noted. In addition, patients with peripheral manifestations were significantly more likely to have persistent high disease activity despite starting biologics earlier than patients without peripheral manifestations (hazard ratio, 2.1).

Patients in pSpA-predominant cluster B showed differences that were similar to those seen in cluster A in terms of high– and low–disease activity trajectories (mean ASDAS-CRP of 3.6 and 2.8, respectively), but among these patients, a high level of disease activity was significantly associated with elevated CRP, rather than with peripheral disease, the researchers said.

The study findings were limited by several factors, including the exclusion of patients who did not complete the follow-up, which reduced the sample size for longitudinal analysis. However, the results were strengthened by the inclusion of patients from the full SpA spectrum, a geographically spread-out patient population, and a study design that mirrored clinical practice, the researchers noted.

The Be-Giant cohort was supported by an unrestricted grant from AbbVie. Several authors reported financial relationships with AbbVie and other pharmaceutical companies.

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Peripheral manifestations contribute significantly to disease activity in adults with spondyloarthritis (SpA), point toward a generally worse prognosis, and play a big role in defining the phenotypic clustering of the heterogenous disease, according to findings from what researchers called the first prospective study “to comprehensively describe the prevalence, clinical patterns, and prognostic implications of peripheral manifestations across the entire SpA spectrum.”

The stratification of patients in the study based on the presence of peripheral manifestations (arthritis, enthesitis, and/or dactylitis) led to the identification of an endotype with unfavorable outcomes, which not only has prognostic value but supports the need for an endotype-based treatment approach rather than one centered on C-reactive protein (CRP) and the Ankylosing Spondylitis Disease Activity Score (ASDAS).

The findings “advocate strongly for the presence of a distinct SpA endotype, based on potentially different immunopathological mechanisms and characterized by high disease activity at initial presentation with lack of substantial improvement upon follow-up,” first author Ann-Sophie De Craemer, MD, of Ghent, Belgium, and colleagues wrote.

Because the diagnostic and prognostic value of peripheral manifestations has not been well studied in SpA in general and in newly diagnosed patients in particular, Dr. De Craemer and associates decided to analyze their impact in 367 patients in the Be-Giant (BelGian Inflammatory Arthritis and spoNdylitis cohorT) cohort, a multicenter, prospective, observational cohort of newly diagnosed patients with SpA in Belgium. The study was published in Rheumatology.

The study population included 257 (70%) patients with axial-predominant SpA (axSpA) as classified by Assessment of Spondyloarthritis International Society criteria and 110 (30%) with peripheral-predominant SpA (pSpA) as defined by ASAS criteria. A total of 52 patients with axSpA had peripheral manifestations at baseline. The mean age of the patients was 34 years, and 52% were male.

The 162 patients with peripheral manifestations included 143 with arthritis, 52 with enthesitis, and 55 with dactylitis.

Two patient clusters emerge from data

In a cluster analysis that used baseline clinical features, the researchers divided the patients into cluster A (of which 242 of 248 were patients with axSpA) and cluster B (of which 104 of 119 were patients with pSpA). Most of the patients with peripheral manifestations were in cluster B (117 of 162 [72%]), compared with cluster A (45 of 162 [28%]).

A longitudinal analysis included 195 patients who completed a minimum 2-year follow-up. The longitudinal analysis identified high- and low-disease activity trajectories in each cluster.

In axSpA-predominant cluster A, patients with “high” trajectory had high disease activity levels at baseline (mean ASDAS-CRP, 3.2) that remained relatively stable, while those in the low-trajectory group (62%) had less disease activity at baseline (mean ASDAS-CRP, 2.0), which then further declined during follow-up.

Patients in the high trajectory in cluster A were more often affected by peripheral manifestations, “which remained a significant predictor in multivariate analysis,” with an odds ratio of 2.4, the researchers noted. In addition, patients with peripheral manifestations were significantly more likely to have persistent high disease activity despite starting biologics earlier than patients without peripheral manifestations (hazard ratio, 2.1).

Patients in pSpA-predominant cluster B showed differences that were similar to those seen in cluster A in terms of high– and low–disease activity trajectories (mean ASDAS-CRP of 3.6 and 2.8, respectively), but among these patients, a high level of disease activity was significantly associated with elevated CRP, rather than with peripheral disease, the researchers said.

The study findings were limited by several factors, including the exclusion of patients who did not complete the follow-up, which reduced the sample size for longitudinal analysis. However, the results were strengthened by the inclusion of patients from the full SpA spectrum, a geographically spread-out patient population, and a study design that mirrored clinical practice, the researchers noted.

The Be-Giant cohort was supported by an unrestricted grant from AbbVie. Several authors reported financial relationships with AbbVie and other pharmaceutical companies.

Peripheral manifestations contribute significantly to disease activity in adults with spondyloarthritis (SpA), point toward a generally worse prognosis, and play a big role in defining the phenotypic clustering of the heterogenous disease, according to findings from what researchers called the first prospective study “to comprehensively describe the prevalence, clinical patterns, and prognostic implications of peripheral manifestations across the entire SpA spectrum.”

The stratification of patients in the study based on the presence of peripheral manifestations (arthritis, enthesitis, and/or dactylitis) led to the identification of an endotype with unfavorable outcomes, which not only has prognostic value but supports the need for an endotype-based treatment approach rather than one centered on C-reactive protein (CRP) and the Ankylosing Spondylitis Disease Activity Score (ASDAS).

The findings “advocate strongly for the presence of a distinct SpA endotype, based on potentially different immunopathological mechanisms and characterized by high disease activity at initial presentation with lack of substantial improvement upon follow-up,” first author Ann-Sophie De Craemer, MD, of Ghent, Belgium, and colleagues wrote.

Because the diagnostic and prognostic value of peripheral manifestations has not been well studied in SpA in general and in newly diagnosed patients in particular, Dr. De Craemer and associates decided to analyze their impact in 367 patients in the Be-Giant (BelGian Inflammatory Arthritis and spoNdylitis cohorT) cohort, a multicenter, prospective, observational cohort of newly diagnosed patients with SpA in Belgium. The study was published in Rheumatology.

The study population included 257 (70%) patients with axial-predominant SpA (axSpA) as classified by Assessment of Spondyloarthritis International Society criteria and 110 (30%) with peripheral-predominant SpA (pSpA) as defined by ASAS criteria. A total of 52 patients with axSpA had peripheral manifestations at baseline. The mean age of the patients was 34 years, and 52% were male.

The 162 patients with peripheral manifestations included 143 with arthritis, 52 with enthesitis, and 55 with dactylitis.

Two patient clusters emerge from data

In a cluster analysis that used baseline clinical features, the researchers divided the patients into cluster A (of which 242 of 248 were patients with axSpA) and cluster B (of which 104 of 119 were patients with pSpA). Most of the patients with peripheral manifestations were in cluster B (117 of 162 [72%]), compared with cluster A (45 of 162 [28%]).

A longitudinal analysis included 195 patients who completed a minimum 2-year follow-up. The longitudinal analysis identified high- and low-disease activity trajectories in each cluster.

In axSpA-predominant cluster A, patients with “high” trajectory had high disease activity levels at baseline (mean ASDAS-CRP, 3.2) that remained relatively stable, while those in the low-trajectory group (62%) had less disease activity at baseline (mean ASDAS-CRP, 2.0), which then further declined during follow-up.

Patients in the high trajectory in cluster A were more often affected by peripheral manifestations, “which remained a significant predictor in multivariate analysis,” with an odds ratio of 2.4, the researchers noted. In addition, patients with peripheral manifestations were significantly more likely to have persistent high disease activity despite starting biologics earlier than patients without peripheral manifestations (hazard ratio, 2.1).

Patients in pSpA-predominant cluster B showed differences that were similar to those seen in cluster A in terms of high– and low–disease activity trajectories (mean ASDAS-CRP of 3.6 and 2.8, respectively), but among these patients, a high level of disease activity was significantly associated with elevated CRP, rather than with peripheral disease, the researchers said.

The study findings were limited by several factors, including the exclusion of patients who did not complete the follow-up, which reduced the sample size for longitudinal analysis. However, the results were strengthened by the inclusion of patients from the full SpA spectrum, a geographically spread-out patient population, and a study design that mirrored clinical practice, the researchers noted.

The Be-Giant cohort was supported by an unrestricted grant from AbbVie. Several authors reported financial relationships with AbbVie and other pharmaceutical companies.

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‘Best Hospitals for Maternity’ offers national perspective

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Tue, 12/07/2021 - 00:15

 

In a new evaluation of hospital maternity services, 237 facilities in 38 states earned high-performing status from U.S. News & World Report.

“The Best Hospitals for Maternity” looked at data for 2019 and covered uncomplicated but not high-risk pregnancies. “All families deserve to be informed on how hospitals perform on key indicators of quality, which is why U.S. News has compiled and published a trove of maternal health data from hospitals across the country,” Ben Harder, managing editor and chief of health analysis at U.S. News, said in a written statement.

The 237 best performers were selected from an overall pool of 571 hospitals that participated in the analysis, representing every state except Alaska, Arkansas, and Vermont, U.S. News said, noting that about 2,700 hospitals in the United States offer maternity services.

California has the highest number of best performers, 33, followed by Illinois and New Jersey, both with 15. Colorado is home to 14 best-performing hospitals, Michigan has 12, and North Carolina and Wisconsin each have 12. “Hospitals that performed well had fewer newborn complications, fewer early deliveries and fewer C-sections, compared to other hospitals across the nation,” Mr. Harder said.

The composite score constructed by U.S. News involved five quality measures: nulliparous, term, singleton, and vertex cesarean delivery rates; early elective delivery rates; unexpected newborn complications rates; routine vaginal birth after cesarean (VBAC) delivery availability; and exclusive breast milk–feeding rates. The composite score averaged 80.0 for high performers and 54.9 for the other participating hospitals, U.S. News reported.

Averages for the high performers on each of the five measures looked like this:
 

  • C-section rate, 21.1%.
  • Early elective delivery rate, 1.3%.
  • Overall unexpected newborn complication rates, 2.38%
  • Exclusive breast milk–feeding rate, 58.6%.
  • VBAC availability, 92.4%.

Data for four measures were collected from the hospitals via online survey over a 4-month window that began on April 29, 2021. Rates of early elective delivery came from the Centers for Medicare & Medicaid Services Care Compare.

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In a new evaluation of hospital maternity services, 237 facilities in 38 states earned high-performing status from U.S. News & World Report.

“The Best Hospitals for Maternity” looked at data for 2019 and covered uncomplicated but not high-risk pregnancies. “All families deserve to be informed on how hospitals perform on key indicators of quality, which is why U.S. News has compiled and published a trove of maternal health data from hospitals across the country,” Ben Harder, managing editor and chief of health analysis at U.S. News, said in a written statement.

The 237 best performers were selected from an overall pool of 571 hospitals that participated in the analysis, representing every state except Alaska, Arkansas, and Vermont, U.S. News said, noting that about 2,700 hospitals in the United States offer maternity services.

California has the highest number of best performers, 33, followed by Illinois and New Jersey, both with 15. Colorado is home to 14 best-performing hospitals, Michigan has 12, and North Carolina and Wisconsin each have 12. “Hospitals that performed well had fewer newborn complications, fewer early deliveries and fewer C-sections, compared to other hospitals across the nation,” Mr. Harder said.

The composite score constructed by U.S. News involved five quality measures: nulliparous, term, singleton, and vertex cesarean delivery rates; early elective delivery rates; unexpected newborn complications rates; routine vaginal birth after cesarean (VBAC) delivery availability; and exclusive breast milk–feeding rates. The composite score averaged 80.0 for high performers and 54.9 for the other participating hospitals, U.S. News reported.

Averages for the high performers on each of the five measures looked like this:
 

  • C-section rate, 21.1%.
  • Early elective delivery rate, 1.3%.
  • Overall unexpected newborn complication rates, 2.38%
  • Exclusive breast milk–feeding rate, 58.6%.
  • VBAC availability, 92.4%.

Data for four measures were collected from the hospitals via online survey over a 4-month window that began on April 29, 2021. Rates of early elective delivery came from the Centers for Medicare & Medicaid Services Care Compare.

 

In a new evaluation of hospital maternity services, 237 facilities in 38 states earned high-performing status from U.S. News & World Report.

“The Best Hospitals for Maternity” looked at data for 2019 and covered uncomplicated but not high-risk pregnancies. “All families deserve to be informed on how hospitals perform on key indicators of quality, which is why U.S. News has compiled and published a trove of maternal health data from hospitals across the country,” Ben Harder, managing editor and chief of health analysis at U.S. News, said in a written statement.

The 237 best performers were selected from an overall pool of 571 hospitals that participated in the analysis, representing every state except Alaska, Arkansas, and Vermont, U.S. News said, noting that about 2,700 hospitals in the United States offer maternity services.

California has the highest number of best performers, 33, followed by Illinois and New Jersey, both with 15. Colorado is home to 14 best-performing hospitals, Michigan has 12, and North Carolina and Wisconsin each have 12. “Hospitals that performed well had fewer newborn complications, fewer early deliveries and fewer C-sections, compared to other hospitals across the nation,” Mr. Harder said.

The composite score constructed by U.S. News involved five quality measures: nulliparous, term, singleton, and vertex cesarean delivery rates; early elective delivery rates; unexpected newborn complications rates; routine vaginal birth after cesarean (VBAC) delivery availability; and exclusive breast milk–feeding rates. The composite score averaged 80.0 for high performers and 54.9 for the other participating hospitals, U.S. News reported.

Averages for the high performers on each of the five measures looked like this:
 

  • C-section rate, 21.1%.
  • Early elective delivery rate, 1.3%.
  • Overall unexpected newborn complication rates, 2.38%
  • Exclusive breast milk–feeding rate, 58.6%.
  • VBAC availability, 92.4%.

Data for four measures were collected from the hospitals via online survey over a 4-month window that began on April 29, 2021. Rates of early elective delivery came from the Centers for Medicare & Medicaid Services Care Compare.

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Clinical Edge Journal Scan Commentary: AML December 2021

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Fri, 05/06/2022 - 16:53
Dr. Atallah scans the journals, so you don’t have to!

Ehab Atallah, MD

This month, the phase II randomized study of azacitidine vs. azacitidine + enasidenib was reported by Dinardo et al.1 The primary endpoint was overall response rate. The study included 101 adult patients with newly diagnosed mutant-IDH2 acute myeloid leukemia (AML) who were ineligible for intensive chemotherapy. They were randomly assigned to enasidenib and azacitidine (Ena-AZA, n = 68) or azacitidine alone (AZA, n = 33). The overall response rate was significantly higher in patients receiving Ena-AZA vs. AZA (74% vs. 36%; odds ratio 4.9; P = .0003). More importantly, the rates of complete remission/complete remission with partial hematologic recovery was higher with Ena-AZA vs. AZA (57% vs. 18% P = .0001). The duration of response was 24.1 months vs 9.9 months for the Ena-AZA vs AZA group. Despite the higher and more durable responses with Ena+AZA, there was no difference in overall survival. The authors attributed that to patients receiving other effective therapies after disease progression. Overall patients tolerated the combination therapy well. The most frequent grade 3 or 4 events in the Ena-AZA vs. AZA groups were thrombocytopenia (37% vs. 19%) and neutropenia (37% vs. 25%).  

Another study from the NCRI AML 16 trial demonstrated that reduced intensity conditioning (RIC) transplant in first remission vs. chemotherapy alone improved survival in older patients with AML who lacked favorable risk cytogenetics and were considered fit for intensive treatment. During a median follow-up of 60 months from remission, patients receiving RIC transplant vs. no transplant had superior survival (37% vs. 20%; hazard ratio [HR] 0.67; P < .001). Survival benefit with transplant in first remission vs. chemotherapy alone was observed across all Wheatley risk groups (adjusted HR 0.68; P < .001). The trial including 932 patients (age 60-70 years) with AML who entered remission and lacked favorable risk. Of these, 144 underwent RIC transplants from either matched sibling donors (n = 52) or matched unrelated donors (n = 92).

The outcome of patients with AML and central nervous system (CNS) involvement at presentation was reported by Ganzel et al. In that study, the investigators evaluated the incidence of CNS involvement in 11 consecutive Eastern Cooperative Oncology Group-American College of Radiology Imaging Network (ECOG-ACRIN) clinical trials. The incidence of CNS incolvement was 1.111 % (36 of 3240 patients). CNS involvement had no effect on remission rates or survival.

 

References

  1. Dinardo CD et al. Enasidenib plus azacitidine versus azacitidine alone in patients with newly diagnosed, mutant-IDH2 acute myeloid leukaemia (AG221-AML-005): a single-arm, phase 1b and randomised, phase 2 trial. Lancet Oncol. 2021;22(11):P1597-1608 (Nov 1).
  2. Russell NH et al. Outcomes of older patients aged 60 to 70 years undergoing reduced intensity transplant for acute myeloblastic leukemia: results of the NCRI acute myeloid leukemia 16 trial. Haematologica. 2021(Oct 14).
  3. Ganzel C et al. CNS involvement in AML at diagnosis is rare and does not affect response or survival: data from 11 ECOG-ACRIN trials. Blood Adv. 2021(Nov 12);5(22):4560-4568. doi: 10.1182/bloodadvances.2021004999.

 

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Ehab L. Atallah, MD

Medical College of Wisconsin

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Medical College of Wisconsin

Dr. Atallah scans the journals, so you don’t have to!
Dr. Atallah scans the journals, so you don’t have to!

Ehab Atallah, MD

This month, the phase II randomized study of azacitidine vs. azacitidine + enasidenib was reported by Dinardo et al.1 The primary endpoint was overall response rate. The study included 101 adult patients with newly diagnosed mutant-IDH2 acute myeloid leukemia (AML) who were ineligible for intensive chemotherapy. They were randomly assigned to enasidenib and azacitidine (Ena-AZA, n = 68) or azacitidine alone (AZA, n = 33). The overall response rate was significantly higher in patients receiving Ena-AZA vs. AZA (74% vs. 36%; odds ratio 4.9; P = .0003). More importantly, the rates of complete remission/complete remission with partial hematologic recovery was higher with Ena-AZA vs. AZA (57% vs. 18% P = .0001). The duration of response was 24.1 months vs 9.9 months for the Ena-AZA vs AZA group. Despite the higher and more durable responses with Ena+AZA, there was no difference in overall survival. The authors attributed that to patients receiving other effective therapies after disease progression. Overall patients tolerated the combination therapy well. The most frequent grade 3 or 4 events in the Ena-AZA vs. AZA groups were thrombocytopenia (37% vs. 19%) and neutropenia (37% vs. 25%).  

Another study from the NCRI AML 16 trial demonstrated that reduced intensity conditioning (RIC) transplant in first remission vs. chemotherapy alone improved survival in older patients with AML who lacked favorable risk cytogenetics and were considered fit for intensive treatment. During a median follow-up of 60 months from remission, patients receiving RIC transplant vs. no transplant had superior survival (37% vs. 20%; hazard ratio [HR] 0.67; P < .001). Survival benefit with transplant in first remission vs. chemotherapy alone was observed across all Wheatley risk groups (adjusted HR 0.68; P < .001). The trial including 932 patients (age 60-70 years) with AML who entered remission and lacked favorable risk. Of these, 144 underwent RIC transplants from either matched sibling donors (n = 52) or matched unrelated donors (n = 92).

The outcome of patients with AML and central nervous system (CNS) involvement at presentation was reported by Ganzel et al. In that study, the investigators evaluated the incidence of CNS involvement in 11 consecutive Eastern Cooperative Oncology Group-American College of Radiology Imaging Network (ECOG-ACRIN) clinical trials. The incidence of CNS incolvement was 1.111 % (36 of 3240 patients). CNS involvement had no effect on remission rates or survival.

 

References

  1. Dinardo CD et al. Enasidenib plus azacitidine versus azacitidine alone in patients with newly diagnosed, mutant-IDH2 acute myeloid leukaemia (AG221-AML-005): a single-arm, phase 1b and randomised, phase 2 trial. Lancet Oncol. 2021;22(11):P1597-1608 (Nov 1).
  2. Russell NH et al. Outcomes of older patients aged 60 to 70 years undergoing reduced intensity transplant for acute myeloblastic leukemia: results of the NCRI acute myeloid leukemia 16 trial. Haematologica. 2021(Oct 14).
  3. Ganzel C et al. CNS involvement in AML at diagnosis is rare and does not affect response or survival: data from 11 ECOG-ACRIN trials. Blood Adv. 2021(Nov 12);5(22):4560-4568. doi: 10.1182/bloodadvances.2021004999.

 

Ehab Atallah, MD

This month, the phase II randomized study of azacitidine vs. azacitidine + enasidenib was reported by Dinardo et al.1 The primary endpoint was overall response rate. The study included 101 adult patients with newly diagnosed mutant-IDH2 acute myeloid leukemia (AML) who were ineligible for intensive chemotherapy. They were randomly assigned to enasidenib and azacitidine (Ena-AZA, n = 68) or azacitidine alone (AZA, n = 33). The overall response rate was significantly higher in patients receiving Ena-AZA vs. AZA (74% vs. 36%; odds ratio 4.9; P = .0003). More importantly, the rates of complete remission/complete remission with partial hematologic recovery was higher with Ena-AZA vs. AZA (57% vs. 18% P = .0001). The duration of response was 24.1 months vs 9.9 months for the Ena-AZA vs AZA group. Despite the higher and more durable responses with Ena+AZA, there was no difference in overall survival. The authors attributed that to patients receiving other effective therapies after disease progression. Overall patients tolerated the combination therapy well. The most frequent grade 3 or 4 events in the Ena-AZA vs. AZA groups were thrombocytopenia (37% vs. 19%) and neutropenia (37% vs. 25%).  

Another study from the NCRI AML 16 trial demonstrated that reduced intensity conditioning (RIC) transplant in first remission vs. chemotherapy alone improved survival in older patients with AML who lacked favorable risk cytogenetics and were considered fit for intensive treatment. During a median follow-up of 60 months from remission, patients receiving RIC transplant vs. no transplant had superior survival (37% vs. 20%; hazard ratio [HR] 0.67; P < .001). Survival benefit with transplant in first remission vs. chemotherapy alone was observed across all Wheatley risk groups (adjusted HR 0.68; P < .001). The trial including 932 patients (age 60-70 years) with AML who entered remission and lacked favorable risk. Of these, 144 underwent RIC transplants from either matched sibling donors (n = 52) or matched unrelated donors (n = 92).

The outcome of patients with AML and central nervous system (CNS) involvement at presentation was reported by Ganzel et al. In that study, the investigators evaluated the incidence of CNS involvement in 11 consecutive Eastern Cooperative Oncology Group-American College of Radiology Imaging Network (ECOG-ACRIN) clinical trials. The incidence of CNS incolvement was 1.111 % (36 of 3240 patients). CNS involvement had no effect on remission rates or survival.

 

References

  1. Dinardo CD et al. Enasidenib plus azacitidine versus azacitidine alone in patients with newly diagnosed, mutant-IDH2 acute myeloid leukaemia (AG221-AML-005): a single-arm, phase 1b and randomised, phase 2 trial. Lancet Oncol. 2021;22(11):P1597-1608 (Nov 1).
  2. Russell NH et al. Outcomes of older patients aged 60 to 70 years undergoing reduced intensity transplant for acute myeloblastic leukemia: results of the NCRI acute myeloid leukemia 16 trial. Haematologica. 2021(Oct 14).
  3. Ganzel C et al. CNS involvement in AML at diagnosis is rare and does not affect response or survival: data from 11 ECOG-ACRIN trials. Blood Adv. 2021(Nov 12);5(22):4560-4568. doi: 10.1182/bloodadvances.2021004999.

 

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Clinical Edge Journal Scan Commentary: CML December 2021

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Dr. Pinilla-Ibarz scans the journals, so you don’t have to

Javier Pinilla-Ibarz MD, PhD

Patients who adhere to their tyrosine kinase inhibitor (TKI) medication regimen will achieve CML survival goals that would be expected for their age group. Imatinib is a first generation TKI inhibitor.  In a recent post hoc analysis of the ADAGIO study patients, Obeng-Kusi M et al1 showed that compared with 90% adherence, a 100% adherence to imatinib was associated with a 2-fold increase in achieving or maintaining treatment response in patients with chronic myeloid leukemia (CML), highlighting the urgent need to assess and promote patient adherence. There is virtually no margin for nonadherence, if the objective is to optimize the likelihood of treatment response, and a minimal margin to avoid impaired treatment response.

 

On the same topic, Davis TC et al.2also recently reported a study of suboptimal adherence to TKI. From the 86 patients with CML studied, almost 17.9% of participants reported nonadherence, i.e., missing at least 1 dose of CML medication in the previous week. The main reason that patients reported nonadherence were side effects, a busy schedule, and the difficulty of complying daily with the TKI regimen.

 

Cardiovascular adverse events (AE) have been described with TKI treatment at different rates, but cardiovascular risk stratification was not included in the design of many trials. Baggio D et al3  in a retrospective study included 88 patients with CML treated with any TKI and a median follow up of 3.8 months. They described the rates of major cardiovascular AEs by combining age, history of prior cardiovascular diseases, and Framingham risk score, along with additional insights from coronary artery calcium scoring (CACS). The authors found cardiovascular AEs in 0%, 10%, and 19%, of the low-, intermediate-, and high-risk groups respectively. By using CACS score they were able to reclassify patients from intermediate to low risk and none of those patients experienced a major adverse cardiovascular event.

 

The use of kinase domain mutation analysis at diagnosis is not a recommended practice by the National Comprehensive Cancer Network (NCCN) or the European Leukemia Net (ELN), but is reserved for use in patents who failed first or subsequent lines of therapies. Furthermore, previous publications in the topic have showed discordant results. More recently, the use of ultra-deep sequencing has detected low-frequency genetic mutations with high sensitivity. Park H et al.4 recently described the most common mutations found in a population of 50 CML patients treated with nilotinib. V299 L mutation associated with dasatinib resistance and nilotinib sensitivity were observed in 98% of patients. Two uncommon mutations S417Y and the V371A were associated with reduced molecular response.

 

References

  1. Obeng-Kusi M et al. No margin for non-adherence: Probabilistic Kaplan-Meier modeling of imatinib non-adherence and treatment response in CML (ADAGIO study). Leuk Res. 2021;111:106734 (Oct 21).
  2. Davis TC et al. Assessment of oral chemotherapy nonadherence in chronic myeloid leukemia patients using brief measures in community cancer clinics: a pilot study. Int J Environ Res Public Health. 2021;18(21):11045 (Oct 21).
  3. Baggio D et al. Prediction of cardiovascular events in patients with chronic myeloid leukaemia using baseline risk factors and coronary artery calcium scoring. Intern Med J. 2021;51(10):1736-40 (Oct 18).  
  4. Park H et al. Ultra-deep sequencing mutation analysis of the BCR/ABL1 kinase domain in newly diagnosed chronic myeloid leukemia patients. Leuk Res. 2021;111:106728 (Oct 15).
Author and Disclosure Information

Javier Pinilla-Ibarz MD, PhD, Senior Member, Lymphoma Section Head and Director of Immunotherapy, Malignant Hematology Department, H.Lee Moffitt Cancer Center & Research Institute

Javier Pinilla has disclosed the following relevant financial relationships:

Serve(d) as a speaker or a member of a speakers bureau for: Abbvie; Janssen; Takeda; AstraZeneca
Received research grant from: TG therapeutics; MEI; Sunesis
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Javier Pinilla-Ibarz MD, PhD, Senior Member, Lymphoma Section Head and Director of Immunotherapy, Malignant Hematology Department, H.Lee Moffitt Cancer Center & Research Institute

Javier Pinilla has disclosed the following relevant financial relationships:

Serve(d) as a speaker or a member of a speakers bureau for: Abbvie; Janssen; Takeda; AstraZeneca
Received research grant from: TG therapeutics; MEI; Sunesis
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Javier Pinilla-Ibarz MD, PhD, Senior Member, Lymphoma Section Head and Director of Immunotherapy, Malignant Hematology Department, H.Lee Moffitt Cancer Center & Research Institute

Javier Pinilla has disclosed the following relevant financial relationships:

Serve(d) as a speaker or a member of a speakers bureau for: Abbvie; Janssen; Takeda; AstraZeneca
Received research grant from: TG therapeutics; MEI; Sunesis
Dr. Pinilla-Ibarz scans the journals, so you don’t have to
Dr. Pinilla-Ibarz scans the journals, so you don’t have to

Javier Pinilla-Ibarz MD, PhD

Patients who adhere to their tyrosine kinase inhibitor (TKI) medication regimen will achieve CML survival goals that would be expected for their age group. Imatinib is a first generation TKI inhibitor.  In a recent post hoc analysis of the ADAGIO study patients, Obeng-Kusi M et al1 showed that compared with 90% adherence, a 100% adherence to imatinib was associated with a 2-fold increase in achieving or maintaining treatment response in patients with chronic myeloid leukemia (CML), highlighting the urgent need to assess and promote patient adherence. There is virtually no margin for nonadherence, if the objective is to optimize the likelihood of treatment response, and a minimal margin to avoid impaired treatment response.

 

On the same topic, Davis TC et al.2also recently reported a study of suboptimal adherence to TKI. From the 86 patients with CML studied, almost 17.9% of participants reported nonadherence, i.e., missing at least 1 dose of CML medication in the previous week. The main reason that patients reported nonadherence were side effects, a busy schedule, and the difficulty of complying daily with the TKI regimen.

 

Cardiovascular adverse events (AE) have been described with TKI treatment at different rates, but cardiovascular risk stratification was not included in the design of many trials. Baggio D et al3  in a retrospective study included 88 patients with CML treated with any TKI and a median follow up of 3.8 months. They described the rates of major cardiovascular AEs by combining age, history of prior cardiovascular diseases, and Framingham risk score, along with additional insights from coronary artery calcium scoring (CACS). The authors found cardiovascular AEs in 0%, 10%, and 19%, of the low-, intermediate-, and high-risk groups respectively. By using CACS score they were able to reclassify patients from intermediate to low risk and none of those patients experienced a major adverse cardiovascular event.

 

The use of kinase domain mutation analysis at diagnosis is not a recommended practice by the National Comprehensive Cancer Network (NCCN) or the European Leukemia Net (ELN), but is reserved for use in patents who failed first or subsequent lines of therapies. Furthermore, previous publications in the topic have showed discordant results. More recently, the use of ultra-deep sequencing has detected low-frequency genetic mutations with high sensitivity. Park H et al.4 recently described the most common mutations found in a population of 50 CML patients treated with nilotinib. V299 L mutation associated with dasatinib resistance and nilotinib sensitivity were observed in 98% of patients. Two uncommon mutations S417Y and the V371A were associated with reduced molecular response.

 

References

  1. Obeng-Kusi M et al. No margin for non-adherence: Probabilistic Kaplan-Meier modeling of imatinib non-adherence and treatment response in CML (ADAGIO study). Leuk Res. 2021;111:106734 (Oct 21).
  2. Davis TC et al. Assessment of oral chemotherapy nonadherence in chronic myeloid leukemia patients using brief measures in community cancer clinics: a pilot study. Int J Environ Res Public Health. 2021;18(21):11045 (Oct 21).
  3. Baggio D et al. Prediction of cardiovascular events in patients with chronic myeloid leukaemia using baseline risk factors and coronary artery calcium scoring. Intern Med J. 2021;51(10):1736-40 (Oct 18).  
  4. Park H et al. Ultra-deep sequencing mutation analysis of the BCR/ABL1 kinase domain in newly diagnosed chronic myeloid leukemia patients. Leuk Res. 2021;111:106728 (Oct 15).

Javier Pinilla-Ibarz MD, PhD

Patients who adhere to their tyrosine kinase inhibitor (TKI) medication regimen will achieve CML survival goals that would be expected for their age group. Imatinib is a first generation TKI inhibitor.  In a recent post hoc analysis of the ADAGIO study patients, Obeng-Kusi M et al1 showed that compared with 90% adherence, a 100% adherence to imatinib was associated with a 2-fold increase in achieving or maintaining treatment response in patients with chronic myeloid leukemia (CML), highlighting the urgent need to assess and promote patient adherence. There is virtually no margin for nonadherence, if the objective is to optimize the likelihood of treatment response, and a minimal margin to avoid impaired treatment response.

 

On the same topic, Davis TC et al.2also recently reported a study of suboptimal adherence to TKI. From the 86 patients with CML studied, almost 17.9% of participants reported nonadherence, i.e., missing at least 1 dose of CML medication in the previous week. The main reason that patients reported nonadherence were side effects, a busy schedule, and the difficulty of complying daily with the TKI regimen.

 

Cardiovascular adverse events (AE) have been described with TKI treatment at different rates, but cardiovascular risk stratification was not included in the design of many trials. Baggio D et al3  in a retrospective study included 88 patients with CML treated with any TKI and a median follow up of 3.8 months. They described the rates of major cardiovascular AEs by combining age, history of prior cardiovascular diseases, and Framingham risk score, along with additional insights from coronary artery calcium scoring (CACS). The authors found cardiovascular AEs in 0%, 10%, and 19%, of the low-, intermediate-, and high-risk groups respectively. By using CACS score they were able to reclassify patients from intermediate to low risk and none of those patients experienced a major adverse cardiovascular event.

 

The use of kinase domain mutation analysis at diagnosis is not a recommended practice by the National Comprehensive Cancer Network (NCCN) or the European Leukemia Net (ELN), but is reserved for use in patents who failed first or subsequent lines of therapies. Furthermore, previous publications in the topic have showed discordant results. More recently, the use of ultra-deep sequencing has detected low-frequency genetic mutations with high sensitivity. Park H et al.4 recently described the most common mutations found in a population of 50 CML patients treated with nilotinib. V299 L mutation associated with dasatinib resistance and nilotinib sensitivity were observed in 98% of patients. Two uncommon mutations S417Y and the V371A were associated with reduced molecular response.

 

References

  1. Obeng-Kusi M et al. No margin for non-adherence: Probabilistic Kaplan-Meier modeling of imatinib non-adherence and treatment response in CML (ADAGIO study). Leuk Res. 2021;111:106734 (Oct 21).
  2. Davis TC et al. Assessment of oral chemotherapy nonadherence in chronic myeloid leukemia patients using brief measures in community cancer clinics: a pilot study. Int J Environ Res Public Health. 2021;18(21):11045 (Oct 21).
  3. Baggio D et al. Prediction of cardiovascular events in patients with chronic myeloid leukaemia using baseline risk factors and coronary artery calcium scoring. Intern Med J. 2021;51(10):1736-40 (Oct 18).  
  4. Park H et al. Ultra-deep sequencing mutation analysis of the BCR/ABL1 kinase domain in newly diagnosed chronic myeloid leukemia patients. Leuk Res. 2021;111:106728 (Oct 15).
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Apixaban outmatches rivaroxaban for VTE in study

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Changed
Thu, 12/09/2021 - 14:48

 

Apixaban appears to be safer and more effective than rivaroxaban for reducing risk of venous thromboembolism and bleeding, based on new research.

Recurrent venous thromboembolism (VTE) – a composite of pulmonary embolism and deep vein thrombosis – was the primary effectiveness outcome in the retrospective analysis of new-user data from almost 40,000 patients, which was published in Annals of Internal Medicine. Safety was evaluated through a composite of intracranial and gastrointestinal bleeding.

After a median follow-up of 102 days in the apixaban group and 105 days in the rivaroxaban group, apixaban demonstrated superiority for both primary outcomes.

These real-world findings may guide selection of initial anticoagulant therapy, reported lead author Ghadeer K. Dawwas, PhD, MSc, MBA, of the University of Pennsylvania, Philadelphia, and colleagues.

“Randomized clinical trials comparing apixaban with rivaroxaban in patients with VTE are under way (for example, COBRRA (NCT03266783),” the investigators wrote. “Until the results from these trials become available (The estimated completion date for COBRRA is December 2023.), observational studies that use existing data can provide evidence on the effectiveness and safety of these alternatives to inform clinical practice.”

In the new research, apixaban was associated with a 23% lower rate of recurrent VTE (hazard ratio, 0.77; 95% confidence interval, 0.69-0.87), including a 15% lower rate of deep vein thrombosis and a 41% lower rate of pulmonary embolism. Apixaban was associated with 40% fewer bleeding events (HR, 0.60; 95% CI, 0.53-0.69]), including a 40% lower rate of GI bleeding and a 46% lower rate of intracranial bleeding.

The study involved 37,236 patients with VTE, all of whom were diagnosed in at least one inpatient encounter and initiated direct oral anticoagulant (DOAC) therapy within 30 days, according to Optum’s deidentified Clinformatics Data Mart Database. Patients were evenly split into apixaban and rivaroxaban groups, with 18,618 individuals in each. Propensity score matching was used to minimize differences in baseline characteristics.

Apixaban was associated with an absolute reduction in recurrent VTE of 0.6% and 1.1% over 2 and 6 months, respectively, as well as reductions in bleeding of 1.1% and 1.5% over the same respective time periods.

The investigators noted that these findings were maintained in various sensitivity and subgroup analyses, including a model in which patients with VTE who had transient risk factors were compared with VTE patients exhibiting chronic risk factors.

“These findings suggest that apixaban has superior effectiveness and safety, compared with rivaroxaban and may provide guidance to clinicians and patients regarding selection of an anticoagulant for treatment of VTE,” Dr. Dawwas and colleagues concluded.

Study may have missed some nuance in possible outcomes, according to vascular surgeon

Thomas Wakefield, MD, a vascular surgeon and a professor of surgery at the University of Michigan Health Frankel Cardiovascular Center, Ann Arbor, generally agreed with the investigators’ conclusion, although he noted that DOAC selection may also be influenced by other considerations.

Dr. Damon E. Houghton

“The results of this study suggest that, when choosing an agent for an individual patient, apixaban does appear to have an advantage over rivaroxaban related to recurrent VTE and bleeding,” Dr. Wakefield said in an interview. “One must keep in mind that these are not the only factors that are considered when choosing an agent and these are not the only two DOACs available. For example, rivaroxaban is given once per day while apixaban is given twice per day, and rivaroxaban has been shown to be successful in the treatment of other thrombotic disorders.”

Dr. Wakefield also pointed out that the study may have missed some nuance in possible outcomes.

“The current study looked at severe outcomes that resulted in inpatient hospitalization, so the generalization to strictly outpatient treatment and less severe outcomes cannot be inferred,” he said.

Damon E. Houghton, MD, of the department of medicine and a consultant in the department of vascular medicine and hematology at Mayo Clinic, Rochester, Minn., called the study a “very nice analysis,” highlighting the large sample size.

“The results are not a reason to abandon rivaroxaban altogether, but do suggest that, when otherwise appropriate for a patient, apixaban should be the first choice,” Dr. Houghton said in a written comment. “Hopefully this analysis will encourage more payers to create financial incentives that facilitate the use of apixaban in more patients.”

 

 

Randomized trial needed, says hematologist

Colleen Edwards, MD, of the departments of medicine, hematology, and medical oncology, at the Icahn School of Medicine at Mount Sinai, New York, had a more guarded view of the findings than Dr. Wakefield and Dr. Houghton.

Dr. Colleen Edwards

“[The investigators] certainly seem to be doing a lot of statistical gymnastics in this paper,” Dr. Edwards said in an interview. “They used all kinds of surrogates in place of real data that you would get from a randomized trial.”

For example, Dr. Edwards noted the use of prescription refills as a surrogate for medication adherence, and emphasized that inpatient observational data may not reflect outpatient therapy.

“Inpatients are constantly missing their medicines all the time,” she said. “They’re holding it for procedures, they’re NPO, they’re off the floor, so they missed their medicine. So it’s just a very different patient population than the outpatient population, which is where venous thromboembolism is treated now, by and large.”

Although Dr. Edwards suggested that the findings might guide treatment selection “a little bit,” she noted that insurance constraints and costs play a greater role, and ultimately concluded that a randomized trial is needed to materially alter clinical decision-making.

“I think we really have to wait for randomized trial before we abandon our other choices,” she said.

The investigators disclosed relationships with Merck, Celgene, UCB, and others. Dr. Wakefield reported awaiting disclosures. Dr. Houghton and Dr. Edwards reported no relevant conflicts of interest.

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Apixaban appears to be safer and more effective than rivaroxaban for reducing risk of venous thromboembolism and bleeding, based on new research.

Recurrent venous thromboembolism (VTE) – a composite of pulmonary embolism and deep vein thrombosis – was the primary effectiveness outcome in the retrospective analysis of new-user data from almost 40,000 patients, which was published in Annals of Internal Medicine. Safety was evaluated through a composite of intracranial and gastrointestinal bleeding.

After a median follow-up of 102 days in the apixaban group and 105 days in the rivaroxaban group, apixaban demonstrated superiority for both primary outcomes.

These real-world findings may guide selection of initial anticoagulant therapy, reported lead author Ghadeer K. Dawwas, PhD, MSc, MBA, of the University of Pennsylvania, Philadelphia, and colleagues.

“Randomized clinical trials comparing apixaban with rivaroxaban in patients with VTE are under way (for example, COBRRA (NCT03266783),” the investigators wrote. “Until the results from these trials become available (The estimated completion date for COBRRA is December 2023.), observational studies that use existing data can provide evidence on the effectiveness and safety of these alternatives to inform clinical practice.”

In the new research, apixaban was associated with a 23% lower rate of recurrent VTE (hazard ratio, 0.77; 95% confidence interval, 0.69-0.87), including a 15% lower rate of deep vein thrombosis and a 41% lower rate of pulmonary embolism. Apixaban was associated with 40% fewer bleeding events (HR, 0.60; 95% CI, 0.53-0.69]), including a 40% lower rate of GI bleeding and a 46% lower rate of intracranial bleeding.

The study involved 37,236 patients with VTE, all of whom were diagnosed in at least one inpatient encounter and initiated direct oral anticoagulant (DOAC) therapy within 30 days, according to Optum’s deidentified Clinformatics Data Mart Database. Patients were evenly split into apixaban and rivaroxaban groups, with 18,618 individuals in each. Propensity score matching was used to minimize differences in baseline characteristics.

Apixaban was associated with an absolute reduction in recurrent VTE of 0.6% and 1.1% over 2 and 6 months, respectively, as well as reductions in bleeding of 1.1% and 1.5% over the same respective time periods.

The investigators noted that these findings were maintained in various sensitivity and subgroup analyses, including a model in which patients with VTE who had transient risk factors were compared with VTE patients exhibiting chronic risk factors.

“These findings suggest that apixaban has superior effectiveness and safety, compared with rivaroxaban and may provide guidance to clinicians and patients regarding selection of an anticoagulant for treatment of VTE,” Dr. Dawwas and colleagues concluded.

Study may have missed some nuance in possible outcomes, according to vascular surgeon

Thomas Wakefield, MD, a vascular surgeon and a professor of surgery at the University of Michigan Health Frankel Cardiovascular Center, Ann Arbor, generally agreed with the investigators’ conclusion, although he noted that DOAC selection may also be influenced by other considerations.

Dr. Damon E. Houghton

“The results of this study suggest that, when choosing an agent for an individual patient, apixaban does appear to have an advantage over rivaroxaban related to recurrent VTE and bleeding,” Dr. Wakefield said in an interview. “One must keep in mind that these are not the only factors that are considered when choosing an agent and these are not the only two DOACs available. For example, rivaroxaban is given once per day while apixaban is given twice per day, and rivaroxaban has been shown to be successful in the treatment of other thrombotic disorders.”

Dr. Wakefield also pointed out that the study may have missed some nuance in possible outcomes.

“The current study looked at severe outcomes that resulted in inpatient hospitalization, so the generalization to strictly outpatient treatment and less severe outcomes cannot be inferred,” he said.

Damon E. Houghton, MD, of the department of medicine and a consultant in the department of vascular medicine and hematology at Mayo Clinic, Rochester, Minn., called the study a “very nice analysis,” highlighting the large sample size.

“The results are not a reason to abandon rivaroxaban altogether, but do suggest that, when otherwise appropriate for a patient, apixaban should be the first choice,” Dr. Houghton said in a written comment. “Hopefully this analysis will encourage more payers to create financial incentives that facilitate the use of apixaban in more patients.”

 

 

Randomized trial needed, says hematologist

Colleen Edwards, MD, of the departments of medicine, hematology, and medical oncology, at the Icahn School of Medicine at Mount Sinai, New York, had a more guarded view of the findings than Dr. Wakefield and Dr. Houghton.

Dr. Colleen Edwards

“[The investigators] certainly seem to be doing a lot of statistical gymnastics in this paper,” Dr. Edwards said in an interview. “They used all kinds of surrogates in place of real data that you would get from a randomized trial.”

For example, Dr. Edwards noted the use of prescription refills as a surrogate for medication adherence, and emphasized that inpatient observational data may not reflect outpatient therapy.

“Inpatients are constantly missing their medicines all the time,” she said. “They’re holding it for procedures, they’re NPO, they’re off the floor, so they missed their medicine. So it’s just a very different patient population than the outpatient population, which is where venous thromboembolism is treated now, by and large.”

Although Dr. Edwards suggested that the findings might guide treatment selection “a little bit,” she noted that insurance constraints and costs play a greater role, and ultimately concluded that a randomized trial is needed to materially alter clinical decision-making.

“I think we really have to wait for randomized trial before we abandon our other choices,” she said.

The investigators disclosed relationships with Merck, Celgene, UCB, and others. Dr. Wakefield reported awaiting disclosures. Dr. Houghton and Dr. Edwards reported no relevant conflicts of interest.

 

Apixaban appears to be safer and more effective than rivaroxaban for reducing risk of venous thromboembolism and bleeding, based on new research.

Recurrent venous thromboembolism (VTE) – a composite of pulmonary embolism and deep vein thrombosis – was the primary effectiveness outcome in the retrospective analysis of new-user data from almost 40,000 patients, which was published in Annals of Internal Medicine. Safety was evaluated through a composite of intracranial and gastrointestinal bleeding.

After a median follow-up of 102 days in the apixaban group and 105 days in the rivaroxaban group, apixaban demonstrated superiority for both primary outcomes.

These real-world findings may guide selection of initial anticoagulant therapy, reported lead author Ghadeer K. Dawwas, PhD, MSc, MBA, of the University of Pennsylvania, Philadelphia, and colleagues.

“Randomized clinical trials comparing apixaban with rivaroxaban in patients with VTE are under way (for example, COBRRA (NCT03266783),” the investigators wrote. “Until the results from these trials become available (The estimated completion date for COBRRA is December 2023.), observational studies that use existing data can provide evidence on the effectiveness and safety of these alternatives to inform clinical practice.”

In the new research, apixaban was associated with a 23% lower rate of recurrent VTE (hazard ratio, 0.77; 95% confidence interval, 0.69-0.87), including a 15% lower rate of deep vein thrombosis and a 41% lower rate of pulmonary embolism. Apixaban was associated with 40% fewer bleeding events (HR, 0.60; 95% CI, 0.53-0.69]), including a 40% lower rate of GI bleeding and a 46% lower rate of intracranial bleeding.

The study involved 37,236 patients with VTE, all of whom were diagnosed in at least one inpatient encounter and initiated direct oral anticoagulant (DOAC) therapy within 30 days, according to Optum’s deidentified Clinformatics Data Mart Database. Patients were evenly split into apixaban and rivaroxaban groups, with 18,618 individuals in each. Propensity score matching was used to minimize differences in baseline characteristics.

Apixaban was associated with an absolute reduction in recurrent VTE of 0.6% and 1.1% over 2 and 6 months, respectively, as well as reductions in bleeding of 1.1% and 1.5% over the same respective time periods.

The investigators noted that these findings were maintained in various sensitivity and subgroup analyses, including a model in which patients with VTE who had transient risk factors were compared with VTE patients exhibiting chronic risk factors.

“These findings suggest that apixaban has superior effectiveness and safety, compared with rivaroxaban and may provide guidance to clinicians and patients regarding selection of an anticoagulant for treatment of VTE,” Dr. Dawwas and colleagues concluded.

Study may have missed some nuance in possible outcomes, according to vascular surgeon

Thomas Wakefield, MD, a vascular surgeon and a professor of surgery at the University of Michigan Health Frankel Cardiovascular Center, Ann Arbor, generally agreed with the investigators’ conclusion, although he noted that DOAC selection may also be influenced by other considerations.

Dr. Damon E. Houghton

“The results of this study suggest that, when choosing an agent for an individual patient, apixaban does appear to have an advantage over rivaroxaban related to recurrent VTE and bleeding,” Dr. Wakefield said in an interview. “One must keep in mind that these are not the only factors that are considered when choosing an agent and these are not the only two DOACs available. For example, rivaroxaban is given once per day while apixaban is given twice per day, and rivaroxaban has been shown to be successful in the treatment of other thrombotic disorders.”

Dr. Wakefield also pointed out that the study may have missed some nuance in possible outcomes.

“The current study looked at severe outcomes that resulted in inpatient hospitalization, so the generalization to strictly outpatient treatment and less severe outcomes cannot be inferred,” he said.

Damon E. Houghton, MD, of the department of medicine and a consultant in the department of vascular medicine and hematology at Mayo Clinic, Rochester, Minn., called the study a “very nice analysis,” highlighting the large sample size.

“The results are not a reason to abandon rivaroxaban altogether, but do suggest that, when otherwise appropriate for a patient, apixaban should be the first choice,” Dr. Houghton said in a written comment. “Hopefully this analysis will encourage more payers to create financial incentives that facilitate the use of apixaban in more patients.”

 

 

Randomized trial needed, says hematologist

Colleen Edwards, MD, of the departments of medicine, hematology, and medical oncology, at the Icahn School of Medicine at Mount Sinai, New York, had a more guarded view of the findings than Dr. Wakefield and Dr. Houghton.

Dr. Colleen Edwards

“[The investigators] certainly seem to be doing a lot of statistical gymnastics in this paper,” Dr. Edwards said in an interview. “They used all kinds of surrogates in place of real data that you would get from a randomized trial.”

For example, Dr. Edwards noted the use of prescription refills as a surrogate for medication adherence, and emphasized that inpatient observational data may not reflect outpatient therapy.

“Inpatients are constantly missing their medicines all the time,” she said. “They’re holding it for procedures, they’re NPO, they’re off the floor, so they missed their medicine. So it’s just a very different patient population than the outpatient population, which is where venous thromboembolism is treated now, by and large.”

Although Dr. Edwards suggested that the findings might guide treatment selection “a little bit,” she noted that insurance constraints and costs play a greater role, and ultimately concluded that a randomized trial is needed to materially alter clinical decision-making.

“I think we really have to wait for randomized trial before we abandon our other choices,” she said.

The investigators disclosed relationships with Merck, Celgene, UCB, and others. Dr. Wakefield reported awaiting disclosures. Dr. Houghton and Dr. Edwards reported no relevant conflicts of interest.

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Tender Subcutaneous Nodule in a Prepubescent Boy

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Display Headline
Tender Subcutaneous Nodule in a Prepubescent Boy

The Diagnosis: Dermatomyofibroma

Dermatomyofibroma is an uncommon, benign, cutaneous mesenchymal neoplasm composed of fibroblasts and myofibroblasts.1-3 This skin tumor was first described in 1991 by Hugel4 in the German literature as plaquelike fibromatosis. Pediatric dermatomyofibromas are exceedingly rare, with pediatric patients ranging in age from infants to teenagers.1

Clinically, dermatomyofibromas appear as long-standing, isolated, ill-demarcated, flesh-colored, slightly hyperpigmented or erythematous nodules or plaques that may be raised or indurated.1 Dermatomyofibromas may present with constant mild pain or pruritus, though in most cases the lesions are asymptomatic.1,3 The clinical presentation of dermatomyofibroma has a few distinct differences in children compared to adults. In adulthood, dermatomyofibroma has a strong female predominance and most commonly is located on the shoulder and adjacent upper body regions, including the axilla, neck, upper arm, and upper trunk.1-3 In childhood, the majority of dermatomyofibromas occur in young boys and usually are located on the neck with other upper body regions occurring less frequently.1,2 A shared characteristic includes the tendency for dermatomyofibromas to have an initial period of enlargement followed by stabilization or slow growth.1 Reported pediatric lesions have ranged in size from 4 to 60 mm with an average size of 14.9 mm (median, 12 mm).2

The diagnosis of dermatomyofibroma is based on histopathologic features in addition to clinical presentation. Histology from punch biopsy usually reveals a noninvasive dermal proliferation of bland, uniform, slender spindle cells oriented parallel to the overlying epidermis with increased and fragmented elastic fibers.1,3 Infiltration into the mid or deep dermis is common. The adnexal structures usually are spared; the stroma contains collagen and increased small blood vessels; and there typically is no inflammatory infiltrate, except for occasional scattered mast cells.2 Cytologically, the monomorphic spindleshaped tumor cells have an ill-defined, pale, eosinophilic cytoplasm and nuclei that are elongated with tapered edges.3 Dermatomyofibroma has a variable immunohistochemical profile, as it may stain focally positive for CD34 or smooth muscle actin, with occasional staining of factor XIIIa, desmin, calponin, or vimentin.1-3 Normal to increased levels of often fragmented elastic fibers is a helpful clue in distinguishing dermatomyofibroma from dermatofibroma, hypertrophic scar, dermatofibrosarcoma protuberans, and pilar leiomyoma, in which elastic fibers typically are reduced.3 Differential diagnoses of mesenchymal tumors in children include desmoid fibromatosis, connective tissue nevus, myofibromatosis, and smooth muscle hamartoma.1

A punch biopsy with clinical observation and followup is recommended for the management of lesions in cosmetically sensitive areas or in very young children who may not tolerate surgery. In symptomatic or cosmetically unappealing cases of dermatomyofibroma, simple surgical excision remains a viable treatment option. Recurrence is uncommon, even if only partially excised, and no instances of metastasis have been reported.1-5

Dermatomyofibromas may be mistaken for several other entities both benign and malignant. For example, the benign dermatofibroma is the second most common fibrohistiocytic tumor of the skin and presents as a firm, nontender, minimally elevated to dome-shaped papule that usually measures less than or equal to 1 cm in diameter with or without overlying skin changes.5,6 It primarily is seen in adults with a slight female predominance and favors the lower extremities.5 Patients usually are asymptomatic but often report a history of local trauma at the lesion site.6 Histologically, dermatofibroma is characterized by a nodular dermal proliferation of spindleshaped fibrous cells and histiocytes in a storiform pattern (Figure 1).6 Epidermal induction with acanthosis overlying the tumor often is found with occasional basilar hyperpigmentation.5 Dermatofibroma also characteristically has trapped collagen (“collagen balls”) seen at the periphery.5,6

FIGURE 1. Dermatofibroma. Fibrohistiocytic proliferation in a storiform pattern with overlying epidermal induction and peripheral collagen trapping (H&E, original magnification ×100).

Piloleiomyomas are benign smooth muscle tumors arising from arrector pili muscles that may be solitary or multiple.5 Clinically, they typically present as firm, reddish-brown to flesh-colored papules or nodules that develop more commonly in adulthood.5,7 Piloleiomyomas favor the extremities and trunk, particularly the shoulder, and can be associated with spontaneous or induced pain. Histologically, piloleiomyomas are well circumscribed and centered within the reticular dermis situated closely to hair follicles (Figure 2).5 They are composed of numerous interlacing fascicles or whorls of smooth muscle cells with abundant eosinophilic cytoplasm and blunt-ended, cigar-shaped nuclei.5,7

FIGURE 2. Piloleiomyoma. Proliferation of smooth muscle arranged in longitudinal fascicles with blunt, cigar-shaped nuclei and eosinophilic cytoplasm (H&E, original magnification ×200).

Solitary cutaneous myofibroma is a benign fibrous tumor found in adolescents and adults and is the counterpart to infantile myofibromatosis.8 Clinically, myofibromas typically present as painless, slow-growing, firm nodules with an occasional bluish hue. Histologically, solitary cutaneous myofibromas appear in a biphasic pattern, with hemangiopericytomatous components as well as spindle cells arranged in short bundles and fascicles resembling leiomyoma (Figure 3). The spindle cells also have abundant eosinophilic cytoplasm with short plump nuclei; the random, irregularly intersecting angles can be used to help differentiate myofibromas from smooth muscle lesions.8 Solitary cutaneous myofibroma is in the differential diagnosis for dermatomyofibroma because of their shared myofibroblastic nature.9

FIGURE 3. Solitary cutaneous myofibroma. Biphasic tumor nodule resembling myofibroblasts (fusiform cells with short plump nuclei and abundant eosinophilic cytoplasm) centrally and pericytes peripherally (H&E, original magnification ×200).

Dermatofibrosarcoma protuberans (DFSP) is an uncommon, locally invasive sarcoma with a high recurrence rate that favors young to middle-aged adults, with rare childhood onset reported.5,10,11 Clinically, DFSP typically presents as an asymptomatic, slow-growing, firm, flesh-colored, indurated plaque that develops into a violaceous to reddish-brown nodule.5 The atrophic variant of DFSP is characterized by a nonprotuberant lesion and can be especially difficult to distinguish from other entities such as dermatomyofibroma.11 The majority of DFSP lesions occur on the trunk, particularly in the shoulder or pelvic region.5 Histologically, early plaque lesions are comprised of monomorphic spindle cells arranged in long fascicles (parallel to the skin surface), infiltrating adnexal structures, and subcutaneous adipocytes in a multilayered honeycomb pattern; the spindle cells of late nodular lesions are arranged in short fascicles in a matted or storiform pattern (Figure 4).5,10 Early stages of DFSP as well as variations in childhood-onset DFSP can easily be misdiagnosed and incompletely excised.5

FIGURE 4. Atrophic dermatofibrosarcoma protuberans. Monomorphic spindle cells infiltrating adipocytes in a honeycomb pattern (H&E, original magnification ×200).

References
  1. Ma JE, Wieland CN, Tollefson MM. Dermatomyofibromas arising in children: report of two new cases and review of the literature. Pediatr Dermatol. 2017;34:347-351.
  2. Tardio JC, Azorin D, Hernandez-Nunez A, et al. Dermatomyofibromas presenting in pediatric patients: clinicopathologic characteristics and differential diagnosis. J Cutan Pathol. 2011;38:967-972.
  3. Mentzel T, Kutzner H. Dermatomyofibroma: clinicopathologic and immunohistochemical analysis of 56 cases and reappraisal of a rare and distinct cutaneous neoplasm. Am J Dermatopathol. 2009;31:44-49.
  4. Hugel H. Plaque-like dermal fibromatosis. Hautarzt. 1991;42:223-226.
  5. Bolognia JL, Jorizzo JL, Schaffer JV, eds. Dermatology. WB Saunders Co; 2012.
  6. Myers DJ, Fillman EP. Dermatofibroma. StatPearls [Internet]. StatPearls Publishing; 2020.
  7. Dilek N, Yuksel D, Sehitoglu I, et al. Cutaneous leiomyoma in a child: a case report. Oncol Lett. 2013;5:1163-1164.
  8. Roh HS, Paek JO, Yu HJ, et al. Solitary cutaneous myofibroma on the sole: an unusual localization. Ann Dermatol. 2012;24:220-222.
  9. Weedon D, Strutton G, Rubin AI, et al. Weedon’s Skin Pathology. Churchill Livingstone/Elsevier; 2010.
  10. Mendenhall WM, Zlotecki RA, Scarborough MT. Dermatofibrosarcoma protuberans. Cancer. 2004;101:2503-2508.
  11. Akay BN, Unlu E, Erdem C, et al. Dermatoscopic findings of atrophic dermatofibrosarcoma protuberans. Dermatol Pract Concept. 2015;5:71-73.
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From the San Antonio Uniformed Services Health Education Consortium, Texas. Dr. Hornberger is from the Transitional Year Program, and Drs. Jones, Wohltmann, and Lenz are from the Department of Dermatology.

The authors report no conflict of interest.

The views presented do not represent the official views of the Department of Defense or its components.

Correspondence: Maria M. Hornberger, MD, 3551 Roger Brooke Dr, JBSA Ft Sam Houston, TX 78234 (maria.m.hornberger.mil@mail.mil).

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The authors report no conflict of interest.

The views presented do not represent the official views of the Department of Defense or its components.

Correspondence: Maria M. Hornberger, MD, 3551 Roger Brooke Dr, JBSA Ft Sam Houston, TX 78234 (maria.m.hornberger.mil@mail.mil).

Author and Disclosure Information

From the San Antonio Uniformed Services Health Education Consortium, Texas. Dr. Hornberger is from the Transitional Year Program, and Drs. Jones, Wohltmann, and Lenz are from the Department of Dermatology.

The authors report no conflict of interest.

The views presented do not represent the official views of the Department of Defense or its components.

Correspondence: Maria M. Hornberger, MD, 3551 Roger Brooke Dr, JBSA Ft Sam Houston, TX 78234 (maria.m.hornberger.mil@mail.mil).

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The Diagnosis: Dermatomyofibroma

Dermatomyofibroma is an uncommon, benign, cutaneous mesenchymal neoplasm composed of fibroblasts and myofibroblasts.1-3 This skin tumor was first described in 1991 by Hugel4 in the German literature as plaquelike fibromatosis. Pediatric dermatomyofibromas are exceedingly rare, with pediatric patients ranging in age from infants to teenagers.1

Clinically, dermatomyofibromas appear as long-standing, isolated, ill-demarcated, flesh-colored, slightly hyperpigmented or erythematous nodules or plaques that may be raised or indurated.1 Dermatomyofibromas may present with constant mild pain or pruritus, though in most cases the lesions are asymptomatic.1,3 The clinical presentation of dermatomyofibroma has a few distinct differences in children compared to adults. In adulthood, dermatomyofibroma has a strong female predominance and most commonly is located on the shoulder and adjacent upper body regions, including the axilla, neck, upper arm, and upper trunk.1-3 In childhood, the majority of dermatomyofibromas occur in young boys and usually are located on the neck with other upper body regions occurring less frequently.1,2 A shared characteristic includes the tendency for dermatomyofibromas to have an initial period of enlargement followed by stabilization or slow growth.1 Reported pediatric lesions have ranged in size from 4 to 60 mm with an average size of 14.9 mm (median, 12 mm).2

The diagnosis of dermatomyofibroma is based on histopathologic features in addition to clinical presentation. Histology from punch biopsy usually reveals a noninvasive dermal proliferation of bland, uniform, slender spindle cells oriented parallel to the overlying epidermis with increased and fragmented elastic fibers.1,3 Infiltration into the mid or deep dermis is common. The adnexal structures usually are spared; the stroma contains collagen and increased small blood vessels; and there typically is no inflammatory infiltrate, except for occasional scattered mast cells.2 Cytologically, the monomorphic spindleshaped tumor cells have an ill-defined, pale, eosinophilic cytoplasm and nuclei that are elongated with tapered edges.3 Dermatomyofibroma has a variable immunohistochemical profile, as it may stain focally positive for CD34 or smooth muscle actin, with occasional staining of factor XIIIa, desmin, calponin, or vimentin.1-3 Normal to increased levels of often fragmented elastic fibers is a helpful clue in distinguishing dermatomyofibroma from dermatofibroma, hypertrophic scar, dermatofibrosarcoma protuberans, and pilar leiomyoma, in which elastic fibers typically are reduced.3 Differential diagnoses of mesenchymal tumors in children include desmoid fibromatosis, connective tissue nevus, myofibromatosis, and smooth muscle hamartoma.1

A punch biopsy with clinical observation and followup is recommended for the management of lesions in cosmetically sensitive areas or in very young children who may not tolerate surgery. In symptomatic or cosmetically unappealing cases of dermatomyofibroma, simple surgical excision remains a viable treatment option. Recurrence is uncommon, even if only partially excised, and no instances of metastasis have been reported.1-5

Dermatomyofibromas may be mistaken for several other entities both benign and malignant. For example, the benign dermatofibroma is the second most common fibrohistiocytic tumor of the skin and presents as a firm, nontender, minimally elevated to dome-shaped papule that usually measures less than or equal to 1 cm in diameter with or without overlying skin changes.5,6 It primarily is seen in adults with a slight female predominance and favors the lower extremities.5 Patients usually are asymptomatic but often report a history of local trauma at the lesion site.6 Histologically, dermatofibroma is characterized by a nodular dermal proliferation of spindleshaped fibrous cells and histiocytes in a storiform pattern (Figure 1).6 Epidermal induction with acanthosis overlying the tumor often is found with occasional basilar hyperpigmentation.5 Dermatofibroma also characteristically has trapped collagen (“collagen balls”) seen at the periphery.5,6

FIGURE 1. Dermatofibroma. Fibrohistiocytic proliferation in a storiform pattern with overlying epidermal induction and peripheral collagen trapping (H&E, original magnification ×100).

Piloleiomyomas are benign smooth muscle tumors arising from arrector pili muscles that may be solitary or multiple.5 Clinically, they typically present as firm, reddish-brown to flesh-colored papules or nodules that develop more commonly in adulthood.5,7 Piloleiomyomas favor the extremities and trunk, particularly the shoulder, and can be associated with spontaneous or induced pain. Histologically, piloleiomyomas are well circumscribed and centered within the reticular dermis situated closely to hair follicles (Figure 2).5 They are composed of numerous interlacing fascicles or whorls of smooth muscle cells with abundant eosinophilic cytoplasm and blunt-ended, cigar-shaped nuclei.5,7

FIGURE 2. Piloleiomyoma. Proliferation of smooth muscle arranged in longitudinal fascicles with blunt, cigar-shaped nuclei and eosinophilic cytoplasm (H&E, original magnification ×200).

Solitary cutaneous myofibroma is a benign fibrous tumor found in adolescents and adults and is the counterpart to infantile myofibromatosis.8 Clinically, myofibromas typically present as painless, slow-growing, firm nodules with an occasional bluish hue. Histologically, solitary cutaneous myofibromas appear in a biphasic pattern, with hemangiopericytomatous components as well as spindle cells arranged in short bundles and fascicles resembling leiomyoma (Figure 3). The spindle cells also have abundant eosinophilic cytoplasm with short plump nuclei; the random, irregularly intersecting angles can be used to help differentiate myofibromas from smooth muscle lesions.8 Solitary cutaneous myofibroma is in the differential diagnosis for dermatomyofibroma because of their shared myofibroblastic nature.9

FIGURE 3. Solitary cutaneous myofibroma. Biphasic tumor nodule resembling myofibroblasts (fusiform cells with short plump nuclei and abundant eosinophilic cytoplasm) centrally and pericytes peripherally (H&E, original magnification ×200).

Dermatofibrosarcoma protuberans (DFSP) is an uncommon, locally invasive sarcoma with a high recurrence rate that favors young to middle-aged adults, with rare childhood onset reported.5,10,11 Clinically, DFSP typically presents as an asymptomatic, slow-growing, firm, flesh-colored, indurated plaque that develops into a violaceous to reddish-brown nodule.5 The atrophic variant of DFSP is characterized by a nonprotuberant lesion and can be especially difficult to distinguish from other entities such as dermatomyofibroma.11 The majority of DFSP lesions occur on the trunk, particularly in the shoulder or pelvic region.5 Histologically, early plaque lesions are comprised of monomorphic spindle cells arranged in long fascicles (parallel to the skin surface), infiltrating adnexal structures, and subcutaneous adipocytes in a multilayered honeycomb pattern; the spindle cells of late nodular lesions are arranged in short fascicles in a matted or storiform pattern (Figure 4).5,10 Early stages of DFSP as well as variations in childhood-onset DFSP can easily be misdiagnosed and incompletely excised.5

FIGURE 4. Atrophic dermatofibrosarcoma protuberans. Monomorphic spindle cells infiltrating adipocytes in a honeycomb pattern (H&E, original magnification ×200).

The Diagnosis: Dermatomyofibroma

Dermatomyofibroma is an uncommon, benign, cutaneous mesenchymal neoplasm composed of fibroblasts and myofibroblasts.1-3 This skin tumor was first described in 1991 by Hugel4 in the German literature as plaquelike fibromatosis. Pediatric dermatomyofibromas are exceedingly rare, with pediatric patients ranging in age from infants to teenagers.1

Clinically, dermatomyofibromas appear as long-standing, isolated, ill-demarcated, flesh-colored, slightly hyperpigmented or erythematous nodules or plaques that may be raised or indurated.1 Dermatomyofibromas may present with constant mild pain or pruritus, though in most cases the lesions are asymptomatic.1,3 The clinical presentation of dermatomyofibroma has a few distinct differences in children compared to adults. In adulthood, dermatomyofibroma has a strong female predominance and most commonly is located on the shoulder and adjacent upper body regions, including the axilla, neck, upper arm, and upper trunk.1-3 In childhood, the majority of dermatomyofibromas occur in young boys and usually are located on the neck with other upper body regions occurring less frequently.1,2 A shared characteristic includes the tendency for dermatomyofibromas to have an initial period of enlargement followed by stabilization or slow growth.1 Reported pediatric lesions have ranged in size from 4 to 60 mm with an average size of 14.9 mm (median, 12 mm).2

The diagnosis of dermatomyofibroma is based on histopathologic features in addition to clinical presentation. Histology from punch biopsy usually reveals a noninvasive dermal proliferation of bland, uniform, slender spindle cells oriented parallel to the overlying epidermis with increased and fragmented elastic fibers.1,3 Infiltration into the mid or deep dermis is common. The adnexal structures usually are spared; the stroma contains collagen and increased small blood vessels; and there typically is no inflammatory infiltrate, except for occasional scattered mast cells.2 Cytologically, the monomorphic spindleshaped tumor cells have an ill-defined, pale, eosinophilic cytoplasm and nuclei that are elongated with tapered edges.3 Dermatomyofibroma has a variable immunohistochemical profile, as it may stain focally positive for CD34 or smooth muscle actin, with occasional staining of factor XIIIa, desmin, calponin, or vimentin.1-3 Normal to increased levels of often fragmented elastic fibers is a helpful clue in distinguishing dermatomyofibroma from dermatofibroma, hypertrophic scar, dermatofibrosarcoma protuberans, and pilar leiomyoma, in which elastic fibers typically are reduced.3 Differential diagnoses of mesenchymal tumors in children include desmoid fibromatosis, connective tissue nevus, myofibromatosis, and smooth muscle hamartoma.1

A punch biopsy with clinical observation and followup is recommended for the management of lesions in cosmetically sensitive areas or in very young children who may not tolerate surgery. In symptomatic or cosmetically unappealing cases of dermatomyofibroma, simple surgical excision remains a viable treatment option. Recurrence is uncommon, even if only partially excised, and no instances of metastasis have been reported.1-5

Dermatomyofibromas may be mistaken for several other entities both benign and malignant. For example, the benign dermatofibroma is the second most common fibrohistiocytic tumor of the skin and presents as a firm, nontender, minimally elevated to dome-shaped papule that usually measures less than or equal to 1 cm in diameter with or without overlying skin changes.5,6 It primarily is seen in adults with a slight female predominance and favors the lower extremities.5 Patients usually are asymptomatic but often report a history of local trauma at the lesion site.6 Histologically, dermatofibroma is characterized by a nodular dermal proliferation of spindleshaped fibrous cells and histiocytes in a storiform pattern (Figure 1).6 Epidermal induction with acanthosis overlying the tumor often is found with occasional basilar hyperpigmentation.5 Dermatofibroma also characteristically has trapped collagen (“collagen balls”) seen at the periphery.5,6

FIGURE 1. Dermatofibroma. Fibrohistiocytic proliferation in a storiform pattern with overlying epidermal induction and peripheral collagen trapping (H&E, original magnification ×100).

Piloleiomyomas are benign smooth muscle tumors arising from arrector pili muscles that may be solitary or multiple.5 Clinically, they typically present as firm, reddish-brown to flesh-colored papules or nodules that develop more commonly in adulthood.5,7 Piloleiomyomas favor the extremities and trunk, particularly the shoulder, and can be associated with spontaneous or induced pain. Histologically, piloleiomyomas are well circumscribed and centered within the reticular dermis situated closely to hair follicles (Figure 2).5 They are composed of numerous interlacing fascicles or whorls of smooth muscle cells with abundant eosinophilic cytoplasm and blunt-ended, cigar-shaped nuclei.5,7

FIGURE 2. Piloleiomyoma. Proliferation of smooth muscle arranged in longitudinal fascicles with blunt, cigar-shaped nuclei and eosinophilic cytoplasm (H&E, original magnification ×200).

Solitary cutaneous myofibroma is a benign fibrous tumor found in adolescents and adults and is the counterpart to infantile myofibromatosis.8 Clinically, myofibromas typically present as painless, slow-growing, firm nodules with an occasional bluish hue. Histologically, solitary cutaneous myofibromas appear in a biphasic pattern, with hemangiopericytomatous components as well as spindle cells arranged in short bundles and fascicles resembling leiomyoma (Figure 3). The spindle cells also have abundant eosinophilic cytoplasm with short plump nuclei; the random, irregularly intersecting angles can be used to help differentiate myofibromas from smooth muscle lesions.8 Solitary cutaneous myofibroma is in the differential diagnosis for dermatomyofibroma because of their shared myofibroblastic nature.9

FIGURE 3. Solitary cutaneous myofibroma. Biphasic tumor nodule resembling myofibroblasts (fusiform cells with short plump nuclei and abundant eosinophilic cytoplasm) centrally and pericytes peripherally (H&E, original magnification ×200).

Dermatofibrosarcoma protuberans (DFSP) is an uncommon, locally invasive sarcoma with a high recurrence rate that favors young to middle-aged adults, with rare childhood onset reported.5,10,11 Clinically, DFSP typically presents as an asymptomatic, slow-growing, firm, flesh-colored, indurated plaque that develops into a violaceous to reddish-brown nodule.5 The atrophic variant of DFSP is characterized by a nonprotuberant lesion and can be especially difficult to distinguish from other entities such as dermatomyofibroma.11 The majority of DFSP lesions occur on the trunk, particularly in the shoulder or pelvic region.5 Histologically, early plaque lesions are comprised of monomorphic spindle cells arranged in long fascicles (parallel to the skin surface), infiltrating adnexal structures, and subcutaneous adipocytes in a multilayered honeycomb pattern; the spindle cells of late nodular lesions are arranged in short fascicles in a matted or storiform pattern (Figure 4).5,10 Early stages of DFSP as well as variations in childhood-onset DFSP can easily be misdiagnosed and incompletely excised.5

FIGURE 4. Atrophic dermatofibrosarcoma protuberans. Monomorphic spindle cells infiltrating adipocytes in a honeycomb pattern (H&E, original magnification ×200).

References
  1. Ma JE, Wieland CN, Tollefson MM. Dermatomyofibromas arising in children: report of two new cases and review of the literature. Pediatr Dermatol. 2017;34:347-351.
  2. Tardio JC, Azorin D, Hernandez-Nunez A, et al. Dermatomyofibromas presenting in pediatric patients: clinicopathologic characteristics and differential diagnosis. J Cutan Pathol. 2011;38:967-972.
  3. Mentzel T, Kutzner H. Dermatomyofibroma: clinicopathologic and immunohistochemical analysis of 56 cases and reappraisal of a rare and distinct cutaneous neoplasm. Am J Dermatopathol. 2009;31:44-49.
  4. Hugel H. Plaque-like dermal fibromatosis. Hautarzt. 1991;42:223-226.
  5. Bolognia JL, Jorizzo JL, Schaffer JV, eds. Dermatology. WB Saunders Co; 2012.
  6. Myers DJ, Fillman EP. Dermatofibroma. StatPearls [Internet]. StatPearls Publishing; 2020.
  7. Dilek N, Yuksel D, Sehitoglu I, et al. Cutaneous leiomyoma in a child: a case report. Oncol Lett. 2013;5:1163-1164.
  8. Roh HS, Paek JO, Yu HJ, et al. Solitary cutaneous myofibroma on the sole: an unusual localization. Ann Dermatol. 2012;24:220-222.
  9. Weedon D, Strutton G, Rubin AI, et al. Weedon’s Skin Pathology. Churchill Livingstone/Elsevier; 2010.
  10. Mendenhall WM, Zlotecki RA, Scarborough MT. Dermatofibrosarcoma protuberans. Cancer. 2004;101:2503-2508.
  11. Akay BN, Unlu E, Erdem C, et al. Dermatoscopic findings of atrophic dermatofibrosarcoma protuberans. Dermatol Pract Concept. 2015;5:71-73.
References
  1. Ma JE, Wieland CN, Tollefson MM. Dermatomyofibromas arising in children: report of two new cases and review of the literature. Pediatr Dermatol. 2017;34:347-351.
  2. Tardio JC, Azorin D, Hernandez-Nunez A, et al. Dermatomyofibromas presenting in pediatric patients: clinicopathologic characteristics and differential diagnosis. J Cutan Pathol. 2011;38:967-972.
  3. Mentzel T, Kutzner H. Dermatomyofibroma: clinicopathologic and immunohistochemical analysis of 56 cases and reappraisal of a rare and distinct cutaneous neoplasm. Am J Dermatopathol. 2009;31:44-49.
  4. Hugel H. Plaque-like dermal fibromatosis. Hautarzt. 1991;42:223-226.
  5. Bolognia JL, Jorizzo JL, Schaffer JV, eds. Dermatology. WB Saunders Co; 2012.
  6. Myers DJ, Fillman EP. Dermatofibroma. StatPearls [Internet]. StatPearls Publishing; 2020.
  7. Dilek N, Yuksel D, Sehitoglu I, et al. Cutaneous leiomyoma in a child: a case report. Oncol Lett. 2013;5:1163-1164.
  8. Roh HS, Paek JO, Yu HJ, et al. Solitary cutaneous myofibroma on the sole: an unusual localization. Ann Dermatol. 2012;24:220-222.
  9. Weedon D, Strutton G, Rubin AI, et al. Weedon’s Skin Pathology. Churchill Livingstone/Elsevier; 2010.
  10. Mendenhall WM, Zlotecki RA, Scarborough MT. Dermatofibrosarcoma protuberans. Cancer. 2004;101:2503-2508.
  11. Akay BN, Unlu E, Erdem C, et al. Dermatoscopic findings of atrophic dermatofibrosarcoma protuberans. Dermatol Pract Concept. 2015;5:71-73.
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H&E, original magnification ×40.

H&E, original magnification ×200 (inset, original magnification ×400).

A 12-year-old boy with olive skin presented with a tender subcutaneous nodule on the back of 6 months’ duration. He reported the lesion initially grew rapidly with increasing pain for approximately 3 months with subsequent stabilization in size and modest resolution of his symptoms. Physical examination revealed a solitary, 15-mm, ill-defined, indurated, tender, subcutaneous nodule with subtle overlying hyperpigmentation on the left side of the upper back. Hematoxylin and eosin staining of a 4-mm punch biopsy revealed a nonencapsulated mass of monomorphic eosinophilic spindle cells organized into fascicles arranged predominantly parallel to the skin surface. The mass extended from the mid reticular dermis to the upper subcutis, sparing adnexal structures.

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