SALT LAKE CITY – A recently approved adjuvanted herpes zoster vaccine)(Shingrix) effectively and safely prevented herpes zoster in a population of patients with multiple myeloma and other hematologic malignancies who received autologous hematopoietic stem cell transplantation.

The use of recombinant varicella zoster virus glycoprotein E in combination with an adjuvant system gives immunosuppressed individuals who have received hematopoietic stem cell transplantation (HSCT) a safe option for prevention of herpes zoster (HZ), said Javier de la Serna, MD, PhD, speaking at the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation.

Kari Oakes/Frontline Medical News

koakes@frontlinemedcom.com

SOURCE: de la Serna J et al. 2018 BMT Tandem Meetings, Abstract LBA2.

SALT LAKE CITY – A recently approved adjuvanted herpes zoster vaccine)(Shingrix) effectively and safely prevented herpes zoster in a population of patients with multiple myeloma and other hematologic malignancies who received autologous hematopoietic stem cell transplantation.

The use of recombinant varicella zoster virus glycoprotein E in combination with an adjuvant system gives immunosuppressed individuals who have received hematopoietic stem cell transplantation (HSCT) a safe option for prevention of herpes zoster (HZ), said Javier de la Serna, MD, PhD, speaking at the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation.

Kari Oakes/Frontline Medical News

koakes@frontlinemedcom.com

SOURCE: de la Serna J et al. 2018 BMT Tandem Meetings, Abstract LBA2.

SALT LAKE CITY – A recently approved adjuvanted herpes zoster vaccine)(Shingrix) effectively and safely prevented herpes zoster in a population of patients with multiple myeloma and other hematologic malignancies who received autologous hematopoietic stem cell transplantation.

The use of recombinant varicella zoster virus glycoprotein E in combination with an adjuvant system gives immunosuppressed individuals who have received hematopoietic stem cell transplantation (HSCT) a safe option for prevention of herpes zoster (HZ), said Javier de la Serna, MD, PhD, speaking at the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation.

Kari Oakes/Frontline Medical News

koakes@frontlinemedcom.com

SOURCE: de la Serna J et al. 2018 BMT Tandem Meetings, Abstract LBA2.

Obstructive sleep apnea (OSA) contributes a major health burden to society due to its high prevalence and substantial neurocognitive and cardiovascular consequences. Estimates suggest that at least 10% of adults in North America are afflicted with OSA, making it probably the most common respiratory disease in the developed world (Peppard et al. Am J Epidemiol. 2013;177[9]:1006). Nasal CPAP is a highly efficacious therapy that has been shown to improve neurocognitive and cardiovascular outcomes. However, CPAP is not always well tolerated. Alternative therapies, such as oral appliances and upper airway surgery, have highly variable efficacy, and evidence of important clinical benefits are uncertain. Therefore, efforts are ongoing to determine optimal alternative strategies for therapy.

In order to treat any condition optimally, one needs to be able to predict who is at highest risk of developing the condition, then to assess the consequences if left untreated, and finally to be able to predict response to various treatment options. Currently, the OSA field is still in its early stages of our understanding. Clinically, we are often faced with patients who have varying presentations and manifestations, but, for reasons that are unclear. For instance, two individuals with the same body mass index may have very different clinical manifestations, one with severe OSA and one without any OSA. Similarly, two individuals with an apnea hypopnea index of 40 events per hour (ie, severe OSA) may have very different symptoms attributable to OSA, eg, one could be asymptomatic and the other could be debilitated from sleepiness. We and others have been making efforts to determine why these phenomenon occur. At present, the techniques to define mechanisms underlying OSA are labor-intensive, requiring one or two overnight experiments to gather meaningful data. Although we are gathering new insights based on these techniques, efforts are ongoing to simplify these approaches and to make assessment of pathophysiologic characteristics more accessible to the clinician (Orr et al. Am J Respir Crit Care Med. 2017 Nov 30. doi: 10.1164/rccm.201707-1357LE. [Epub ahead of print]).

We ultimately believe that a thorough analysis of a sleep recording combined with demographic data and other readily available clinical data (perhaps plasma biomarkers) may yield sufficient information for us to know why OSA is occurring and what interventions might be helpful for an individual patient. Currently, our use of the polysomnogram to derive only an apnea hypopnea index does not take full advantage of the available data. An apnea hypopnea index can be readily obtained from home sleep testing and does not truly provide much insight into why a given individual has OSA, what symptoms are attributable to OSA, and what interventions might be considered for the afflicted individual. By analogy, if the only useful data derived from an ECG were a heart rate, the test would rapidly become obsolete. Along these lines, if the only role for the sleep clinician was to prescribe CPAP to everyone with an AHI greater than 5/h, there would be little need or interest in specialized training. In contrast, we suggest that rich insights regarding pathophysiology and mechanisms should be gathered and may influence clinical management of patients afflicted with OSA. Thus, we encourage more thorough analyses of available data to maximize information gleaned and, ultimately, to optimize clinical outcomes.

Recent studies suggest that sleep apnea occurs for varying reasons, a concept that is now thought to be clinically important (Jordan et al. Lancet. 2014;383[9918]:736). We draw a crucial distinction between endotypes (mechanisms underlying disease) and phenotypes (clinical expression of disease). Important endotypes include compromised upper airway anatomy, dysfunction in pharyngeal dilator muscles, unstable ventilatory control (high loop gain), and low arousal threshold (wake up easily), among others. Important phenotypes of sleep apnea are emerging and still evolving to include minimally symptomatic OSA, OSA with daytime sleepiness, and OSA with major cardiometabolic risk, among others. Several important concepts have emerged regarding different OSA endotypes and phenotypes:

1 The mechanism underlying OSA may predict potential response to therapeutic interventions. For instance, the endotype of OSA with unstable ventilatory control (high loop gain) may respond to agents such as oxygen and acetazolamide, which serve to stabilize control of breathing. In patients with anatomical compromise at the level of the velopharynx, uvulopalatopharyngoplasty may be an effective intervention. For patients with multiple pathophysiologic abnormalities, combination therapy may be required to alleviate OSA (Edwards et al. Sleep. 2016;9[11]:1973).

2 Given that OSA has many underlying etiologies, efforts are underway to determine whether individuals with different risk factors for OSA develop their disease based on varying mechanisms. As an example, people with posttraumatic stress disorder (PTSD) may be at increased risk of OSA perhaps on the basis of a low threshold for arousal (Orr et al. JCSM. 2017, 13[1]: 57-63). Another example would be patients with neuromuscular disease who may be at risk of OSA primarily based on impaired pharyngeal dilator muscle function.

3 A new concept is emerging whereby endotypes of OSA may actually predict differing OSA phenotypes. In theory, loop gain-driven OSA may have different consequences from OSA driven by compromise of pharyngeal anatomy. To this point, data suggest that OSA in the elderly may not have as many consequences as OSA in younger people matched on severity of illness. OSA in the elderly has lower loop gain than OSA in younger people and is associated with less negative intrathoracic pressure at the time of arousal as compared with younger individuals with OSA (Kobayashi et al. Chest. 2010; 137[6]:1310). As such, the endotype of OSA in the elderly may explain why the clinical consequences are fewer than in the younger OSA counterparts.

4 The mechanism underlying OSA may be important in determining response to clinical interventions, such as nasal CPAP. Patients with a low arousal threshold may be prone to insomnia when placed on CPAP and could theoretically be poorly tolerant of therapy based on disrupted sleep architecture. Such patients may benefit from non-myorelaxant hypnotic therapy to consolidate sleep and improve CPAP adherence. In addition, patients with high loop gain (unstable ventilatory control) may be prone to develop central apneas when placed on CPAP therapy (Stanchina et al. Ann Am Thorac Soc. 2015;12[9]:1351). These patients may benefit from newer technologies, eg, auto or adaptive servo ventilation - ASV. High loop gain has also been shown to predict failure of upper airway surgery as a treatment for OSA by several groups (Li et al. JCSM. 2017;13[9]:1029). Such patients should, perhaps, undergo nonsurgical therapies for OSA.

We emphasize that some of the points being made are somewhat speculative and, thus, encourage further basic and clinical research to test our assumptions. Robust, multicenter clinical trials assessing hard outcomes will ultimately be required to change the current standard of care. Nonetheless, we believe that a more thorough understanding of OSA pathogenesis can help guide clinical care today and will be critical to the optimal treatment of afflicted individuals tomorrow.

Dr. Owens is Assistant Clinical Professor of Medicine; Dr. Deacon is a Post-Doctoral Research Scholar; and Dr. Malhotra is Kenneth M. Moser Professor of Medicine and Chief, Division of Pulmonary, Critical Care and Sleep Medicine, University of California San Diego.

Obstructive sleep apnea (OSA) contributes a major health burden to society due to its high prevalence and substantial neurocognitive and cardiovascular consequences. Estimates suggest that at least 10% of adults in North America are afflicted with OSA, making it probably the most common respiratory disease in the developed world (Peppard et al. Am J Epidemiol. 2013;177[9]:1006). Nasal CPAP is a highly efficacious therapy that has been shown to improve neurocognitive and cardiovascular outcomes. However, CPAP is not always well tolerated. Alternative therapies, such as oral appliances and upper airway surgery, have highly variable efficacy, and evidence of important clinical benefits are uncertain. Therefore, efforts are ongoing to determine optimal alternative strategies for therapy.

In order to treat any condition optimally, one needs to be able to predict who is at highest risk of developing the condition, then to assess the consequences if left untreated, and finally to be able to predict response to various treatment options. Currently, the OSA field is still in its early stages of our understanding. Clinically, we are often faced with patients who have varying presentations and manifestations, but, for reasons that are unclear. For instance, two individuals with the same body mass index may have very different clinical manifestations, one with severe OSA and one without any OSA. Similarly, two individuals with an apnea hypopnea index of 40 events per hour (ie, severe OSA) may have very different symptoms attributable to OSA, eg, one could be asymptomatic and the other could be debilitated from sleepiness. We and others have been making efforts to determine why these phenomenon occur. At present, the techniques to define mechanisms underlying OSA are labor-intensive, requiring one or two overnight experiments to gather meaningful data. Although we are gathering new insights based on these techniques, efforts are ongoing to simplify these approaches and to make assessment of pathophysiologic characteristics more accessible to the clinician (Orr et al. Am J Respir Crit Care Med. 2017 Nov 30. doi: 10.1164/rccm.201707-1357LE. [Epub ahead of print]).

We ultimately believe that a thorough analysis of a sleep recording combined with demographic data and other readily available clinical data (perhaps plasma biomarkers) may yield sufficient information for us to know why OSA is occurring and what interventions might be helpful for an individual patient. Currently, our use of the polysomnogram to derive only an apnea hypopnea index does not take full advantage of the available data. An apnea hypopnea index can be readily obtained from home sleep testing and does not truly provide much insight into why a given individual has OSA, what symptoms are attributable to OSA, and what interventions might be considered for the afflicted individual. By analogy, if the only useful data derived from an ECG were a heart rate, the test would rapidly become obsolete. Along these lines, if the only role for the sleep clinician was to prescribe CPAP to everyone with an AHI greater than 5/h, there would be little need or interest in specialized training. In contrast, we suggest that rich insights regarding pathophysiology and mechanisms should be gathered and may influence clinical management of patients afflicted with OSA. Thus, we encourage more thorough analyses of available data to maximize information gleaned and, ultimately, to optimize clinical outcomes.

Recent studies suggest that sleep apnea occurs for varying reasons, a concept that is now thought to be clinically important (Jordan et al. Lancet. 2014;383[9918]:736). We draw a crucial distinction between endotypes (mechanisms underlying disease) and phenotypes (clinical expression of disease). Important endotypes include compromised upper airway anatomy, dysfunction in pharyngeal dilator muscles, unstable ventilatory control (high loop gain), and low arousal threshold (wake up easily), among others. Important phenotypes of sleep apnea are emerging and still evolving to include minimally symptomatic OSA, OSA with daytime sleepiness, and OSA with major cardiometabolic risk, among others. Several important concepts have emerged regarding different OSA endotypes and phenotypes:

1 The mechanism underlying OSA may predict potential response to therapeutic interventions. For instance, the endotype of OSA with unstable ventilatory control (high loop gain) may respond to agents such as oxygen and acetazolamide, which serve to stabilize control of breathing. In patients with anatomical compromise at the level of the velopharynx, uvulopalatopharyngoplasty may be an effective intervention. For patients with multiple pathophysiologic abnormalities, combination therapy may be required to alleviate OSA (Edwards et al. Sleep. 2016;9[11]:1973).

2 Given that OSA has many underlying etiologies, efforts are underway to determine whether individuals with different risk factors for OSA develop their disease based on varying mechanisms. As an example, people with posttraumatic stress disorder (PTSD) may be at increased risk of OSA perhaps on the basis of a low threshold for arousal (Orr et al. JCSM. 2017, 13[1]: 57-63). Another example would be patients with neuromuscular disease who may be at risk of OSA primarily based on impaired pharyngeal dilator muscle function.

3 A new concept is emerging whereby endotypes of OSA may actually predict differing OSA phenotypes. In theory, loop gain-driven OSA may have different consequences from OSA driven by compromise of pharyngeal anatomy. To this point, data suggest that OSA in the elderly may not have as many consequences as OSA in younger people matched on severity of illness. OSA in the elderly has lower loop gain than OSA in younger people and is associated with less negative intrathoracic pressure at the time of arousal as compared with younger individuals with OSA (Kobayashi et al. Chest. 2010; 137[6]:1310). As such, the endotype of OSA in the elderly may explain why the clinical consequences are fewer than in the younger OSA counterparts.

4 The mechanism underlying OSA may be important in determining response to clinical interventions, such as nasal CPAP. Patients with a low arousal threshold may be prone to insomnia when placed on CPAP and could theoretically be poorly tolerant of therapy based on disrupted sleep architecture. Such patients may benefit from non-myorelaxant hypnotic therapy to consolidate sleep and improve CPAP adherence. In addition, patients with high loop gain (unstable ventilatory control) may be prone to develop central apneas when placed on CPAP therapy (Stanchina et al. Ann Am Thorac Soc. 2015;12[9]:1351). These patients may benefit from newer technologies, eg, auto or adaptive servo ventilation - ASV. High loop gain has also been shown to predict failure of upper airway surgery as a treatment for OSA by several groups (Li et al. JCSM. 2017;13[9]:1029). Such patients should, perhaps, undergo nonsurgical therapies for OSA.

We emphasize that some of the points being made are somewhat speculative and, thus, encourage further basic and clinical research to test our assumptions. Robust, multicenter clinical trials assessing hard outcomes will ultimately be required to change the current standard of care. Nonetheless, we believe that a more thorough understanding of OSA pathogenesis can help guide clinical care today and will be critical to the optimal treatment of afflicted individuals tomorrow.

Dr. Owens is Assistant Clinical Professor of Medicine; Dr. Deacon is a Post-Doctoral Research Scholar; and Dr. Malhotra is Kenneth M. Moser Professor of Medicine and Chief, Division of Pulmonary, Critical Care and Sleep Medicine, University of California San Diego.

Obstructive sleep apnea (OSA) contributes a major health burden to society due to its high prevalence and substantial neurocognitive and cardiovascular consequences. Estimates suggest that at least 10% of adults in North America are afflicted with OSA, making it probably the most common respiratory disease in the developed world (Peppard et al. Am J Epidemiol. 2013;177[9]:1006). Nasal CPAP is a highly efficacious therapy that has been shown to improve neurocognitive and cardiovascular outcomes. However, CPAP is not always well tolerated. Alternative therapies, such as oral appliances and upper airway surgery, have highly variable efficacy, and evidence of important clinical benefits are uncertain. Therefore, efforts are ongoing to determine optimal alternative strategies for therapy.

In order to treat any condition optimally, one needs to be able to predict who is at highest risk of developing the condition, then to assess the consequences if left untreated, and finally to be able to predict response to various treatment options. Currently, the OSA field is still in its early stages of our understanding. Clinically, we are often faced with patients who have varying presentations and manifestations, but, for reasons that are unclear. For instance, two individuals with the same body mass index may have very different clinical manifestations, one with severe OSA and one without any OSA. Similarly, two individuals with an apnea hypopnea index of 40 events per hour (ie, severe OSA) may have very different symptoms attributable to OSA, eg, one could be asymptomatic and the other could be debilitated from sleepiness. We and others have been making efforts to determine why these phenomenon occur. At present, the techniques to define mechanisms underlying OSA are labor-intensive, requiring one or two overnight experiments to gather meaningful data. Although we are gathering new insights based on these techniques, efforts are ongoing to simplify these approaches and to make assessment of pathophysiologic characteristics more accessible to the clinician (Orr et al. Am J Respir Crit Care Med. 2017 Nov 30. doi: 10.1164/rccm.201707-1357LE. [Epub ahead of print]).

We ultimately believe that a thorough analysis of a sleep recording combined with demographic data and other readily available clinical data (perhaps plasma biomarkers) may yield sufficient information for us to know why OSA is occurring and what interventions might be helpful for an individual patient. Currently, our use of the polysomnogram to derive only an apnea hypopnea index does not take full advantage of the available data. An apnea hypopnea index can be readily obtained from home sleep testing and does not truly provide much insight into why a given individual has OSA, what symptoms are attributable to OSA, and what interventions might be considered for the afflicted individual. By analogy, if the only useful data derived from an ECG were a heart rate, the test would rapidly become obsolete. Along these lines, if the only role for the sleep clinician was to prescribe CPAP to everyone with an AHI greater than 5/h, there would be little need or interest in specialized training. In contrast, we suggest that rich insights regarding pathophysiology and mechanisms should be gathered and may influence clinical management of patients afflicted with OSA. Thus, we encourage more thorough analyses of available data to maximize information gleaned and, ultimately, to optimize clinical outcomes.

Recent studies suggest that sleep apnea occurs for varying reasons, a concept that is now thought to be clinically important (Jordan et al. Lancet. 2014;383[9918]:736). We draw a crucial distinction between endotypes (mechanisms underlying disease) and phenotypes (clinical expression of disease). Important endotypes include compromised upper airway anatomy, dysfunction in pharyngeal dilator muscles, unstable ventilatory control (high loop gain), and low arousal threshold (wake up easily), among others. Important phenotypes of sleep apnea are emerging and still evolving to include minimally symptomatic OSA, OSA with daytime sleepiness, and OSA with major cardiometabolic risk, among others. Several important concepts have emerged regarding different OSA endotypes and phenotypes:

1 The mechanism underlying OSA may predict potential response to therapeutic interventions. For instance, the endotype of OSA with unstable ventilatory control (high loop gain) may respond to agents such as oxygen and acetazolamide, which serve to stabilize control of breathing. In patients with anatomical compromise at the level of the velopharynx, uvulopalatopharyngoplasty may be an effective intervention. For patients with multiple pathophysiologic abnormalities, combination therapy may be required to alleviate OSA (Edwards et al. Sleep. 2016;9[11]:1973).

2 Given that OSA has many underlying etiologies, efforts are underway to determine whether individuals with different risk factors for OSA develop their disease based on varying mechanisms. As an example, people with posttraumatic stress disorder (PTSD) may be at increased risk of OSA perhaps on the basis of a low threshold for arousal (Orr et al. JCSM. 2017, 13[1]: 57-63). Another example would be patients with neuromuscular disease who may be at risk of OSA primarily based on impaired pharyngeal dilator muscle function.

3 A new concept is emerging whereby endotypes of OSA may actually predict differing OSA phenotypes. In theory, loop gain-driven OSA may have different consequences from OSA driven by compromise of pharyngeal anatomy. To this point, data suggest that OSA in the elderly may not have as many consequences as OSA in younger people matched on severity of illness. OSA in the elderly has lower loop gain than OSA in younger people and is associated with less negative intrathoracic pressure at the time of arousal as compared with younger individuals with OSA (Kobayashi et al. Chest. 2010; 137[6]:1310). As such, the endotype of OSA in the elderly may explain why the clinical consequences are fewer than in the younger OSA counterparts.

4 The mechanism underlying OSA may be important in determining response to clinical interventions, such as nasal CPAP. Patients with a low arousal threshold may be prone to insomnia when placed on CPAP and could theoretically be poorly tolerant of therapy based on disrupted sleep architecture. Such patients may benefit from non-myorelaxant hypnotic therapy to consolidate sleep and improve CPAP adherence. In addition, patients with high loop gain (unstable ventilatory control) may be prone to develop central apneas when placed on CPAP therapy (Stanchina et al. Ann Am Thorac Soc. 2015;12[9]:1351). These patients may benefit from newer technologies, eg, auto or adaptive servo ventilation - ASV. High loop gain has also been shown to predict failure of upper airway surgery as a treatment for OSA by several groups (Li et al. JCSM. 2017;13[9]:1029). Such patients should, perhaps, undergo nonsurgical therapies for OSA.

We emphasize that some of the points being made are somewhat speculative and, thus, encourage further basic and clinical research to test our assumptions. Robust, multicenter clinical trials assessing hard outcomes will ultimately be required to change the current standard of care. Nonetheless, we believe that a more thorough understanding of OSA pathogenesis can help guide clinical care today and will be critical to the optimal treatment of afflicted individuals tomorrow.

Dr. Owens is Assistant Clinical Professor of Medicine; Dr. Deacon is a Post-Doctoral Research Scholar; and Dr. Malhotra is Kenneth M. Moser Professor of Medicine and Chief, Division of Pulmonary, Critical Care and Sleep Medicine, University of California San Diego.

SAN DIEGO – Whether the glut of applications contending for the roughly 420 dermatology residency positions in recent years may be corrupting the process was the topic of discussion at a session on dermatoethics at the annual meeting of the American Academy of Dermatology.

“I think it is unethical and we need to address it,” Jane M. Grant-Kels, MD, said during the session. “What we are doing through the process of physicians getting into dermatology residency programs is telling them to lie to us and to do well on a single examination,” the United States Medical Licensing Examination.

Mitchel Zoler/Frontline Medical News

“I don’t believe that dermatology match is broken, unethical, or unfair. The match is not perfect, but it’s fair,” contended Dr. Bercovitch, professor of dermatology at Brown University, Providence, R.I. “The problem [of an application glut] is real, but it’s not an ethical issue.”

Dr. Grant-Kels sees it in ethical terms because, in her view, “everyone is gaming the system. It makes applicants liars” when they profess interest in moving to a remote location or planning to practice a certain type of dermatology.

Mitchel Zoler/Frontline Medical News

As a result, residency programs feel forced to apply blind filters that generally cull out more than a third of the applications received. Dr. Grant-Kels decried the need for programs to impose arbitrary barriers to entering dermatology based on a score from a single examination or other criteria like membership in Alpha Omega Alpha or current location.

“Blanket screening methods run the risk of excluding genuinely interested and qualified candidates who do not fall above a threshold. This violates the principal of nonmaleficence,” she said. “Screens are unfair.”

[polldaddy:{"method":"iframe","type":"survey","src":"//newspolls2017.polldaddy.com/s/dermatology-residency-match?iframe=1"}]

Dr. Grant-Kels proposed a pair of potential remedies: putting a cap on the number of applications someone can make and – a more realistic approach – mentors’ giving guidance to prospective applicants.

“It’s a problem that kids are applying to dermatology programs who have no business applying, who really don’t have a chance,” she said.

Dr. Bercovitch noted that most dermatology residency programs are too small and that, while the number of residency slots has been rising, it has not kept pace with increasing demand from physicians seeking residency slots. He saw no ethical reason for physicians to feel they should rein in the number of applications they file, and he said the only obligations for residency programs are to strictly adhere to the Match rules and both federal and state civil rights and labor laws and to be nondiscriminatory and avoid nepotism and conflicts of interest. Because programs cannot seriously consider nor interview several hundred applicants each year, some type of filtering is needed, and no filter is fair or perfect, he conceded.

“Filters are inherently unfair” to certain applicants, “but how else to effectively screen” hundreds of applications, Dr. Bercovitch asked.

“We need to talk about this. It’s not a good system. If we don’t talk about it, it will never change,” Dr. Grant-Kels said.

Dr. Grant-Kels and Dr. Bercovitch had no disclosures.

mzoler@frontlinemedcom.com

SAN DIEGO – Whether the glut of applications contending for the roughly 420 dermatology residency positions in recent years may be corrupting the process was the topic of discussion at a session on dermatoethics at the annual meeting of the American Academy of Dermatology.

“I think it is unethical and we need to address it,” Jane M. Grant-Kels, MD, said during the session. “What we are doing through the process of physicians getting into dermatology residency programs is telling them to lie to us and to do well on a single examination,” the United States Medical Licensing Examination.

Mitchel Zoler/Frontline Medical News

“I don’t believe that dermatology match is broken, unethical, or unfair. The match is not perfect, but it’s fair,” contended Dr. Bercovitch, professor of dermatology at Brown University, Providence, R.I. “The problem [of an application glut] is real, but it’s not an ethical issue.”

Dr. Grant-Kels sees it in ethical terms because, in her view, “everyone is gaming the system. It makes applicants liars” when they profess interest in moving to a remote location or planning to practice a certain type of dermatology.

Mitchel Zoler/Frontline Medical News

As a result, residency programs feel forced to apply blind filters that generally cull out more than a third of the applications received. Dr. Grant-Kels decried the need for programs to impose arbitrary barriers to entering dermatology based on a score from a single examination or other criteria like membership in Alpha Omega Alpha or current location.

“Blanket screening methods run the risk of excluding genuinely interested and qualified candidates who do not fall above a threshold. This violates the principal of nonmaleficence,” she said. “Screens are unfair.”

[polldaddy:{"method":"iframe","type":"survey","src":"//newspolls2017.polldaddy.com/s/dermatology-residency-match?iframe=1"}]

Dr. Grant-Kels proposed a pair of potential remedies: putting a cap on the number of applications someone can make and – a more realistic approach – mentors’ giving guidance to prospective applicants.

“It’s a problem that kids are applying to dermatology programs who have no business applying, who really don’t have a chance,” she said.

Dr. Bercovitch noted that most dermatology residency programs are too small and that, while the number of residency slots has been rising, it has not kept pace with increasing demand from physicians seeking residency slots. He saw no ethical reason for physicians to feel they should rein in the number of applications they file, and he said the only obligations for residency programs are to strictly adhere to the Match rules and both federal and state civil rights and labor laws and to be nondiscriminatory and avoid nepotism and conflicts of interest. Because programs cannot seriously consider nor interview several hundred applicants each year, some type of filtering is needed, and no filter is fair or perfect, he conceded.

“Filters are inherently unfair” to certain applicants, “but how else to effectively screen” hundreds of applications, Dr. Bercovitch asked.

“We need to talk about this. It’s not a good system. If we don’t talk about it, it will never change,” Dr. Grant-Kels said.

Dr. Grant-Kels and Dr. Bercovitch had no disclosures.

mzoler@frontlinemedcom.com

SAN DIEGO – Whether the glut of applications contending for the roughly 420 dermatology residency positions in recent years may be corrupting the process was the topic of discussion at a session on dermatoethics at the annual meeting of the American Academy of Dermatology.

“I think it is unethical and we need to address it,” Jane M. Grant-Kels, MD, said during the session. “What we are doing through the process of physicians getting into dermatology residency programs is telling them to lie to us and to do well on a single examination,” the United States Medical Licensing Examination.

Mitchel Zoler/Frontline Medical News

“I don’t believe that dermatology match is broken, unethical, or unfair. The match is not perfect, but it’s fair,” contended Dr. Bercovitch, professor of dermatology at Brown University, Providence, R.I. “The problem [of an application glut] is real, but it’s not an ethical issue.”

Dr. Grant-Kels sees it in ethical terms because, in her view, “everyone is gaming the system. It makes applicants liars” when they profess interest in moving to a remote location or planning to practice a certain type of dermatology.

Mitchel Zoler/Frontline Medical News

As a result, residency programs feel forced to apply blind filters that generally cull out more than a third of the applications received. Dr. Grant-Kels decried the need for programs to impose arbitrary barriers to entering dermatology based on a score from a single examination or other criteria like membership in Alpha Omega Alpha or current location.

“Blanket screening methods run the risk of excluding genuinely interested and qualified candidates who do not fall above a threshold. This violates the principal of nonmaleficence,” she said. “Screens are unfair.”

[polldaddy:{"method":"iframe","type":"survey","src":"//newspolls2017.polldaddy.com/s/dermatology-residency-match?iframe=1"}]

Dr. Grant-Kels proposed a pair of potential remedies: putting a cap on the number of applications someone can make and – a more realistic approach – mentors’ giving guidance to prospective applicants.

“It’s a problem that kids are applying to dermatology programs who have no business applying, who really don’t have a chance,” she said.

Dr. Bercovitch noted that most dermatology residency programs are too small and that, while the number of residency slots has been rising, it has not kept pace with increasing demand from physicians seeking residency slots. He saw no ethical reason for physicians to feel they should rein in the number of applications they file, and he said the only obligations for residency programs are to strictly adhere to the Match rules and both federal and state civil rights and labor laws and to be nondiscriminatory and avoid nepotism and conflicts of interest. Because programs cannot seriously consider nor interview several hundred applicants each year, some type of filtering is needed, and no filter is fair or perfect, he conceded.

“Filters are inherently unfair” to certain applicants, “but how else to effectively screen” hundreds of applications, Dr. Bercovitch asked.

“We need to talk about this. It’s not a good system. If we don’t talk about it, it will never change,” Dr. Grant-Kels said.

Dr. Grant-Kels and Dr. Bercovitch had no disclosures.

mzoler@frontlinemedcom.com

Physicians should aim for hemoglobin A_1c levels between 7% and 8% in patients with type 2 diabetes, according to recommendations by the American College of Physicians.

But the guidance drew strong condemnation from diabetes and endocrinology organizations, which warned the higher target range could do more harm than good.

Evidence presented in the ACP statement shows the intensive therapy commonly used to reach the previous target of 6.5%-7% is likely either to increase risk of adverse cardiovascular and hypoglycemic events or have the same risk as less intensive therapy.

“Treatment with drugs targeted to 7%, in contrast to 8%, does not prevent heart attacks or strokes,” said Jack Ende, MD, president of the ACP. “But it does result in substantial harm, including low blood sugar, increased medication burden, and increased cost.”

The ACP recommends clinicians should talk with patients about creating personalized treatment plans based on the risks and benefits of different drugs, what the patients prefers, life expectancy, and cost of care.

But many diabetes organizations aren't embracing the new target levels. 

The ACP's recommendations could keep many diabetes patients from achieving all the benefits from long-term intensive glucose control, warned the Endocrine Society, the American Diabetes Association, the American Association for Clinical Endocrinologists, and the American Association of Diabetes Educators in a joint statement. And the ACP's recommendations overlook the positive impact that newer diabetes drugs have shown on morbidity and mortality in high-risk type 2 diabetes patients.

Coauthor Michael J. Barry, MD, reported receiving grants from the nonprofit Healthwise, outside of this statement.

All other authors reported no relevant financial disclosures.

ezimmerman@frontlinemedcom.com

Physicians should aim for hemoglobin A_1c levels between 7% and 8% in patients with type 2 diabetes, according to recommendations by the American College of Physicians.

But the guidance drew strong condemnation from diabetes and endocrinology organizations, which warned the higher target range could do more harm than good.

Evidence presented in the ACP statement shows the intensive therapy commonly used to reach the previous target of 6.5%-7% is likely either to increase risk of adverse cardiovascular and hypoglycemic events or have the same risk as less intensive therapy.

“Treatment with drugs targeted to 7%, in contrast to 8%, does not prevent heart attacks or strokes,” said Jack Ende, MD, president of the ACP. “But it does result in substantial harm, including low blood sugar, increased medication burden, and increased cost.”

The ACP recommends clinicians should talk with patients about creating personalized treatment plans based on the risks and benefits of different drugs, what the patients prefers, life expectancy, and cost of care.

But many diabetes organizations aren't embracing the new target levels. 

The ACP's recommendations could keep many diabetes patients from achieving all the benefits from long-term intensive glucose control, warned the Endocrine Society, the American Diabetes Association, the American Association for Clinical Endocrinologists, and the American Association of Diabetes Educators in a joint statement. And the ACP's recommendations overlook the positive impact that newer diabetes drugs have shown on morbidity and mortality in high-risk type 2 diabetes patients.

Coauthor Michael J. Barry, MD, reported receiving grants from the nonprofit Healthwise, outside of this statement.

All other authors reported no relevant financial disclosures.

ezimmerman@frontlinemedcom.com

Physicians should aim for hemoglobin A_1c levels between 7% and 8% in patients with type 2 diabetes, according to recommendations by the American College of Physicians.

But the guidance drew strong condemnation from diabetes and endocrinology organizations, which warned the higher target range could do more harm than good.

Evidence presented in the ACP statement shows the intensive therapy commonly used to reach the previous target of 6.5%-7% is likely either to increase risk of adverse cardiovascular and hypoglycemic events or have the same risk as less intensive therapy.

“Treatment with drugs targeted to 7%, in contrast to 8%, does not prevent heart attacks or strokes,” said Jack Ende, MD, president of the ACP. “But it does result in substantial harm, including low blood sugar, increased medication burden, and increased cost.”

The ACP recommends clinicians should talk with patients about creating personalized treatment plans based on the risks and benefits of different drugs, what the patients prefers, life expectancy, and cost of care.

But many diabetes organizations aren't embracing the new target levels. 

The ACP's recommendations could keep many diabetes patients from achieving all the benefits from long-term intensive glucose control, warned the Endocrine Society, the American Diabetes Association, the American Association for Clinical Endocrinologists, and the American Association of Diabetes Educators in a joint statement. And the ACP's recommendations overlook the positive impact that newer diabetes drugs have shown on morbidity and mortality in high-risk type 2 diabetes patients.

Coauthor Michael J. Barry, MD, reported receiving grants from the nonprofit Healthwise, outside of this statement.

All other authors reported no relevant financial disclosures.

ezimmerman@frontlinemedcom.com

Prophylactic haloperidol did not prevent delirium or improve survival in a placebo-controlled trial of 1,789 critically ill adults at 21 ICUs in the Netherlands.

Haloperidol is used routinely in ICUs to both treat and prevent delirium, which strikes up to half of ICU patients and is associated with prolonged mechanical ventilation, longer ICU and hospital stays, and increased mortality. Results of past studies have been mixed, with some showing a benefit for haloperidol in the ICU and others not.

“These findings do not support the use of prophylactic haloperidol in critically ill adults,” said the authors of a new study, led by Mark van den Boogaard, PhD, of Radboud University Medical Center, Nijmegen, the Netherlands (JAMA. 2018 Feb 20;319[7]:680-90).

The subjects were all expected to be in the ICU for at least 2 days, and were not delirious at baseline. The patients were randomly assigned to receive one of two treatments or a placebo three times daily, with 350 receiving 1 mg of haloperidol; 732 receiving 2 mg of haloperidol; and 707 receiving a 0.9% sodium chloride placebo. The 1-mg haloperidol arm was stopped early because of futility.

XiXinXing/ThinkStock

aotto@frontlinemedcom.com

SOURCE: van den Boogaard M, et al. JAMA. 2018 Feb 20;319(7):680-90.

Prophylactic haloperidol did not prevent delirium or improve survival in a placebo-controlled trial of 1,789 critically ill adults at 21 ICUs in the Netherlands.

Haloperidol is used routinely in ICUs to both treat and prevent delirium, which strikes up to half of ICU patients and is associated with prolonged mechanical ventilation, longer ICU and hospital stays, and increased mortality. Results of past studies have been mixed, with some showing a benefit for haloperidol in the ICU and others not.

“These findings do not support the use of prophylactic haloperidol in critically ill adults,” said the authors of a new study, led by Mark van den Boogaard, PhD, of Radboud University Medical Center, Nijmegen, the Netherlands (JAMA. 2018 Feb 20;319[7]:680-90).

The subjects were all expected to be in the ICU for at least 2 days, and were not delirious at baseline. The patients were randomly assigned to receive one of two treatments or a placebo three times daily, with 350 receiving 1 mg of haloperidol; 732 receiving 2 mg of haloperidol; and 707 receiving a 0.9% sodium chloride placebo. The 1-mg haloperidol arm was stopped early because of futility.

XiXinXing/ThinkStock

aotto@frontlinemedcom.com

SOURCE: van den Boogaard M, et al. JAMA. 2018 Feb 20;319(7):680-90.

Prophylactic haloperidol did not prevent delirium or improve survival in a placebo-controlled trial of 1,789 critically ill adults at 21 ICUs in the Netherlands.

Haloperidol is used routinely in ICUs to both treat and prevent delirium, which strikes up to half of ICU patients and is associated with prolonged mechanical ventilation, longer ICU and hospital stays, and increased mortality. Results of past studies have been mixed, with some showing a benefit for haloperidol in the ICU and others not.

“These findings do not support the use of prophylactic haloperidol in critically ill adults,” said the authors of a new study, led by Mark van den Boogaard, PhD, of Radboud University Medical Center, Nijmegen, the Netherlands (JAMA. 2018 Feb 20;319[7]:680-90).

The subjects were all expected to be in the ICU for at least 2 days, and were not delirious at baseline. The patients were randomly assigned to receive one of two treatments or a placebo three times daily, with 350 receiving 1 mg of haloperidol; 732 receiving 2 mg of haloperidol; and 707 receiving a 0.9% sodium chloride placebo. The 1-mg haloperidol arm was stopped early because of futility.

XiXinXing/ThinkStock

aotto@frontlinemedcom.com

SOURCE: van den Boogaard M, et al. JAMA. 2018 Feb 20;319(7):680-90.

Alterations in DNA damage response and repair genes may be useful biomarkers for response to immune checkpoint inhibitors in patients with urothelial carcinoma, investigators contend.

Among 60 patients with urothelial carcinoma in prospective trials of antibodies directed against the immune checkpoint inhibitor programmed cell death protein-1 and its ligand (PD-1/PD-L1), 28 (47%) had a damage response and repair (DDR) gene of any kind, and 15 (25%) had DDR mutations that are thought to be deleterious. Patients with any DDR mutations were more likely to have a clinical response to anti–PD-1/PD-L1 therapy, especially patients with likely deleterious mutations, reported Jonathan E. Rosenberg, MD and colleagues from Memorial Sloan Kettering Cancer Center, New York.

op@frontlinemedcom.com

SOURCE: Rosenberg JE et al. J Clin Oncol. 2018 Feb 28. doi: 10.1200/JCO.2017.75.7740.

Alterations in DNA damage response and repair genes may be useful biomarkers for response to immune checkpoint inhibitors in patients with urothelial carcinoma, investigators contend.

Among 60 patients with urothelial carcinoma in prospective trials of antibodies directed against the immune checkpoint inhibitor programmed cell death protein-1 and its ligand (PD-1/PD-L1), 28 (47%) had a damage response and repair (DDR) gene of any kind, and 15 (25%) had DDR mutations that are thought to be deleterious. Patients with any DDR mutations were more likely to have a clinical response to anti–PD-1/PD-L1 therapy, especially patients with likely deleterious mutations, reported Jonathan E. Rosenberg, MD and colleagues from Memorial Sloan Kettering Cancer Center, New York.

op@frontlinemedcom.com

SOURCE: Rosenberg JE et al. J Clin Oncol. 2018 Feb 28. doi: 10.1200/JCO.2017.75.7740.

Alterations in DNA damage response and repair genes may be useful biomarkers for response to immune checkpoint inhibitors in patients with urothelial carcinoma, investigators contend.

Among 60 patients with urothelial carcinoma in prospective trials of antibodies directed against the immune checkpoint inhibitor programmed cell death protein-1 and its ligand (PD-1/PD-L1), 28 (47%) had a damage response and repair (DDR) gene of any kind, and 15 (25%) had DDR mutations that are thought to be deleterious. Patients with any DDR mutations were more likely to have a clinical response to anti–PD-1/PD-L1 therapy, especially patients with likely deleterious mutations, reported Jonathan E. Rosenberg, MD and colleagues from Memorial Sloan Kettering Cancer Center, New York.

op@frontlinemedcom.com

SOURCE: Rosenberg JE et al. J Clin Oncol. 2018 Feb 28. doi: 10.1200/JCO.2017.75.7740.

MAUI, HAWAII – Two recent Scandinavian studies highlight the considerable room for improvement that exists in psoriatic arthritis outcomes, according to speakers at the 2018 Rheumatology Winter Clinical Symposium.

“We’re not doing so well. I think that in general, if you think of the treatments that we have and the treat-to-target approach, psoriatic arthritis lags behind rheumatoid arthritis, maybe by a decade,” declared Arthur Kavanaugh, MD, program director for the symposium and professor of medicine at the University of California, San Diego.

Bruce Jancin/Frontline Medical News

Another recent Scandinavian psoriatic arthritis study that particularly impressed Dr. Kavanaugh was a Danish nationwide cohort registry study that shed new light upon the burdens imposed by the disease in terms of societal costs, comorbid conditions, and disability. The subjects were 10,525 Danes with psoriatic arthritis and nearly 21,000 matched general population controls evaluated during the period from 5 years prior to diagnosis of psoriatic arthritis through 10 years after.

The costs were impressive: an average of 10,641 euros more per year per psoriatic arthritis patient, compared with controls, during the 10-year period following diagnosis, with most of the difference attributed to higher health care costs and forgone earnings due to unemployment, early retirement, or receipt of a disability pension.

At the time of their diagnosis, psoriatic arthritis patients had a significantly higher burden of comorbid conditions than did controls, including a 1.7-fold higher likelihood of cardiovascular disease, a 1.73-fold increase in risk for pulmonary disease, a 2.03-fold greater likelihood of infectious diseases, and a 1.94-fold increased risk of hematologic disorders.

Five years prior to diagnosis of psoriatic arthritis, affected individuals were 1.36-fold more likely than were controls to be on a disability pension. At diagnosis, they were 1.6-fold more likely. Ten years after diagnosis of psoriatic arthritis, they were 2.69-fold more likely to be on a disability pension. Indeed, by that point, 21.8% of Danish psoriatic arthritis patients were on disability (Ann Rheum Dis. 2017 Sep;76([9]:1495-1501).

bjancin@frontlinemedcom.com

MAUI, HAWAII – Two recent Scandinavian studies highlight the considerable room for improvement that exists in psoriatic arthritis outcomes, according to speakers at the 2018 Rheumatology Winter Clinical Symposium.

“We’re not doing so well. I think that in general, if you think of the treatments that we have and the treat-to-target approach, psoriatic arthritis lags behind rheumatoid arthritis, maybe by a decade,” declared Arthur Kavanaugh, MD, program director for the symposium and professor of medicine at the University of California, San Diego.

Bruce Jancin/Frontline Medical News

Another recent Scandinavian psoriatic arthritis study that particularly impressed Dr. Kavanaugh was a Danish nationwide cohort registry study that shed new light upon the burdens imposed by the disease in terms of societal costs, comorbid conditions, and disability. The subjects were 10,525 Danes with psoriatic arthritis and nearly 21,000 matched general population controls evaluated during the period from 5 years prior to diagnosis of psoriatic arthritis through 10 years after.

The costs were impressive: an average of 10,641 euros more per year per psoriatic arthritis patient, compared with controls, during the 10-year period following diagnosis, with most of the difference attributed to higher health care costs and forgone earnings due to unemployment, early retirement, or receipt of a disability pension.

At the time of their diagnosis, psoriatic arthritis patients had a significantly higher burden of comorbid conditions than did controls, including a 1.7-fold higher likelihood of cardiovascular disease, a 1.73-fold increase in risk for pulmonary disease, a 2.03-fold greater likelihood of infectious diseases, and a 1.94-fold increased risk of hematologic disorders.

Five years prior to diagnosis of psoriatic arthritis, affected individuals were 1.36-fold more likely than were controls to be on a disability pension. At diagnosis, they were 1.6-fold more likely. Ten years after diagnosis of psoriatic arthritis, they were 2.69-fold more likely to be on a disability pension. Indeed, by that point, 21.8% of Danish psoriatic arthritis patients were on disability (Ann Rheum Dis. 2017 Sep;76([9]:1495-1501).

bjancin@frontlinemedcom.com

MAUI, HAWAII – Two recent Scandinavian studies highlight the considerable room for improvement that exists in psoriatic arthritis outcomes, according to speakers at the 2018 Rheumatology Winter Clinical Symposium.

“We’re not doing so well. I think that in general, if you think of the treatments that we have and the treat-to-target approach, psoriatic arthritis lags behind rheumatoid arthritis, maybe by a decade,” declared Arthur Kavanaugh, MD, program director for the symposium and professor of medicine at the University of California, San Diego.

Bruce Jancin/Frontline Medical News

Another recent Scandinavian psoriatic arthritis study that particularly impressed Dr. Kavanaugh was a Danish nationwide cohort registry study that shed new light upon the burdens imposed by the disease in terms of societal costs, comorbid conditions, and disability. The subjects were 10,525 Danes with psoriatic arthritis and nearly 21,000 matched general population controls evaluated during the period from 5 years prior to diagnosis of psoriatic arthritis through 10 years after.

The costs were impressive: an average of 10,641 euros more per year per psoriatic arthritis patient, compared with controls, during the 10-year period following diagnosis, with most of the difference attributed to higher health care costs and forgone earnings due to unemployment, early retirement, or receipt of a disability pension.

At the time of their diagnosis, psoriatic arthritis patients had a significantly higher burden of comorbid conditions than did controls, including a 1.7-fold higher likelihood of cardiovascular disease, a 1.73-fold increase in risk for pulmonary disease, a 2.03-fold greater likelihood of infectious diseases, and a 1.94-fold increased risk of hematologic disorders.

Five years prior to diagnosis of psoriatic arthritis, affected individuals were 1.36-fold more likely than were controls to be on a disability pension. At diagnosis, they were 1.6-fold more likely. Ten years after diagnosis of psoriatic arthritis, they were 2.69-fold more likely to be on a disability pension. Indeed, by that point, 21.8% of Danish psoriatic arthritis patients were on disability (Ann Rheum Dis. 2017 Sep;76([9]:1495-1501).

bjancin@frontlinemedcom.com

The child is well-known in the halls in which state bureaucrats oversee health care for millions of Californians – not by name, but by a number: $21 million.

His medications alone cost state taxpayers that much in a single year, not including other health care. The boy, whose identity has not been released, was California’s most expensive Medicaid patient in recent years. His case was singled out in a tweet last year by the state’s top health care official to highlight the public insurance program’s extraordinary obligations as a backstop for low-income patients.

How on earth can a single child’s treatment cost that much? The answer: He has hemophilia and needs large quantities of a pricey drug – known as clotting factor – that makes blood coagulate.

Hemophilia drugs are among the most costly drugs in the nation, and taxpayers are footing the bill for many patients on Medicaid who could never afford them on their own. Officials in California and other states are doing what they can to manage the costs, but it’s a daunting task that highlights the complexity and secrecy of prescription drug pricing.

Tab for 145 kids: $195 million

The California boy whose drugs cost $21 million in a single year was an extreme case, and the circumstances of his care have not been disclosed because of confidentiality protections. Still, medications to treat hemophilia on average annual cost more than $270,000 per patient, according to a 2015 Express Scripts report, and they can easily soar past $1 million annually.

In contrast to more common diseases like hepatitis C, hemophilia treatment is not a state “budget buster” per se: about 4,000 patients live in California. About 1,100 of them are covered by Medi-Cal or two other government-funded programs for chronically ill children in California, according to Jennifer Kent, director of the state Department of Health Care Services and author of last year’s tweet. But the amount of money spent per person dwarfs that spent on people with other serious diseases.

One Stanford University study of 34,000 California kids with severe chronic diseases found that the tiny portion of children who needed blood factor accounted for 41% of the state’s outpatient drug spending on this entire patient population. About $195 million was spent on just 145 kids over a 3-year period, although some of that money came back to the state in rebates from drug companies – a portion of the cost that Medicaid can recoup after purchase.

Caitlin Carroll, director of public affairs for PhRMA, the pharmaceutical industry lobbying group, said high development costs and the complicated and lengthy manufacturing process play a role in how hemophilia drugs are priced. She added that federally mandated rebates significantly reduce the cost of blood factor. They amount to 17% of the average manufacturer price per unit.

Manufacturers also note that some newer and more expensive hemophilia drugs last longer and do not need to be administered as frequently, so they can prove less costly to payers overall.

Even so, some patients require a monumental investment to survive.
 

‘Extremely fortunate’

Colleen Tuite’s son Kevin, a 7-year-old, has severe hemophilia with a complication known as an inhibitor – an antibody that makes his regular blood-factor infusions less effective. Inhibitors can dramatically increase the cost of care, because massive doses of blood factor or expensive, specialized blood products known as bypassing agents may be needed.

Ms. Tuite and her husband initially were Kevin’s foster parents, then adopted the boy as a toddler. Because he has been a foster child, Kevin qualifies for Medi-Cal until he is 26.

The Monrovia, Calif., family also has private health insurance, which pays for about half of Kevin’s medical bills. These can run upward of $200,000 per month, Ms. Tuite said.

“We definitely would not have been able to adopt him without the help of Medi-Cal,” Ms. Tuite said. “We’ve been extremely fortunate.”

With the support of drug manufacturers and hemophilia advocacy groups, patients and their families have significant political clout. Some experts say they also have a moral claim on public resources: In the early days of the AIDS epidemic, thousands of the nation’s hemophilia patients died after they contracted HIV through transfusions before the virus was eliminated from the blood supply.

State health officials say the costs of hemophilia are hard to anticipate and control, even with rebates.

“We do a really aggressive job of collecting rebates on our pharmacy costs,” said Ms. Kent, California’s top Medicaid official. “But there’s just not any way around blood factor. It is just a very, very expensive product. It’s nonnegotiable for people that require it.”

In 2016, California’s Medicaid program paid at least $205 million for medications used to treat hemophilia, according to a Kaiser Health News analysis of federal Medicaid data. That figure doesn’t account for the federal rebates.

States can negotiate “supplemental” rebates with drugmakers for individual medications – but those must be kept secret under federal and some state laws. Such secrecy is becoming increasingly controversial as states continue to confront spiraling drug prices.

Limited options for states

In 2016, Pfizer sued Texas’ state health agency for giving data on the drug company’s supplemental Medicaid rebates to state lawmakers who requested it. The drugmaker alleged that releasing the confidential information would undermine the company’s competitiveness and give away trade secrets, and warned that the discounts it gave Texas could disappear.

In early October, a judge ruled that lawmakers should be able to obtain some of that data, noting dryly that “in Pfizer’s view, legislators are not necessary to carry out the state’s Medicaid program.”

Instead of seeking additional rebates from manufacturers for blood factor, some states, including Washington and Oregon, have chosen to require patients to get their blood factor from federally designated Hemophilia Treatment Centers only. That allows state Medicaid programs to take advantage of a federal drug-discount program known as “340B.”

However, officials in California said they studied that option and determined it wouldn’t save them any more money than the rebates they negotiate with drugmakers.

Whatever their approach, state health officials say they are struggling against forces they are nearly powerless to change.

“There aren’t a lot of options available to Medicaid programs in terms of controlling costs, because we don’t set the initial costs,” said Deborah Weston, pharmacy program manager for Oregon’s Medicaid program.
 

Kaiser Health News data correspondent Sydney Lupkin contributed to this report. KHN’s coverage of these topics is supported by Laura and John Arnold Foundation and Heising-Simons Foundation. This story was produced by Kaiser Health News, which publishes California Healthline, a service of the California Health Care Foundation. Kaiser Health News is a nonprofit news service covering health issues. It is an editorially independent program of the Kaiser Family Foundation that is not affiliated with Kaiser Permanente.

The child is well-known in the halls in which state bureaucrats oversee health care for millions of Californians – not by name, but by a number: $21 million.

His medications alone cost state taxpayers that much in a single year, not including other health care. The boy, whose identity has not been released, was California’s most expensive Medicaid patient in recent years. His case was singled out in a tweet last year by the state’s top health care official to highlight the public insurance program’s extraordinary obligations as a backstop for low-income patients.

How on earth can a single child’s treatment cost that much? The answer: He has hemophilia and needs large quantities of a pricey drug – known as clotting factor – that makes blood coagulate.

Hemophilia drugs are among the most costly drugs in the nation, and taxpayers are footing the bill for many patients on Medicaid who could never afford them on their own. Officials in California and other states are doing what they can to manage the costs, but it’s a daunting task that highlights the complexity and secrecy of prescription drug pricing.

Tab for 145 kids: $195 million

The California boy whose drugs cost $21 million in a single year was an extreme case, and the circumstances of his care have not been disclosed because of confidentiality protections. Still, medications to treat hemophilia on average annual cost more than $270,000 per patient, according to a 2015 Express Scripts report, and they can easily soar past $1 million annually.

In contrast to more common diseases like hepatitis C, hemophilia treatment is not a state “budget buster” per se: about 4,000 patients live in California. About 1,100 of them are covered by Medi-Cal or two other government-funded programs for chronically ill children in California, according to Jennifer Kent, director of the state Department of Health Care Services and author of last year’s tweet. But the amount of money spent per person dwarfs that spent on people with other serious diseases.

One Stanford University study of 34,000 California kids with severe chronic diseases found that the tiny portion of children who needed blood factor accounted for 41% of the state’s outpatient drug spending on this entire patient population. About $195 million was spent on just 145 kids over a 3-year period, although some of that money came back to the state in rebates from drug companies – a portion of the cost that Medicaid can recoup after purchase.

Caitlin Carroll, director of public affairs for PhRMA, the pharmaceutical industry lobbying group, said high development costs and the complicated and lengthy manufacturing process play a role in how hemophilia drugs are priced. She added that federally mandated rebates significantly reduce the cost of blood factor. They amount to 17% of the average manufacturer price per unit.

Manufacturers also note that some newer and more expensive hemophilia drugs last longer and do not need to be administered as frequently, so they can prove less costly to payers overall.

Even so, some patients require a monumental investment to survive.
 

‘Extremely fortunate’

Colleen Tuite’s son Kevin, a 7-year-old, has severe hemophilia with a complication known as an inhibitor – an antibody that makes his regular blood-factor infusions less effective. Inhibitors can dramatically increase the cost of care, because massive doses of blood factor or expensive, specialized blood products known as bypassing agents may be needed.

Ms. Tuite and her husband initially were Kevin’s foster parents, then adopted the boy as a toddler. Because he has been a foster child, Kevin qualifies for Medi-Cal until he is 26.

The Monrovia, Calif., family also has private health insurance, which pays for about half of Kevin’s medical bills. These can run upward of $200,000 per month, Ms. Tuite said.

“We definitely would not have been able to adopt him without the help of Medi-Cal,” Ms. Tuite said. “We’ve been extremely fortunate.”

With the support of drug manufacturers and hemophilia advocacy groups, patients and their families have significant political clout. Some experts say they also have a moral claim on public resources: In the early days of the AIDS epidemic, thousands of the nation’s hemophilia patients died after they contracted HIV through transfusions before the virus was eliminated from the blood supply.

State health officials say the costs of hemophilia are hard to anticipate and control, even with rebates.

“We do a really aggressive job of collecting rebates on our pharmacy costs,” said Ms. Kent, California’s top Medicaid official. “But there’s just not any way around blood factor. It is just a very, very expensive product. It’s nonnegotiable for people that require it.”

In 2016, California’s Medicaid program paid at least $205 million for medications used to treat hemophilia, according to a Kaiser Health News analysis of federal Medicaid data. That figure doesn’t account for the federal rebates.

States can negotiate “supplemental” rebates with drugmakers for individual medications – but those must be kept secret under federal and some state laws. Such secrecy is becoming increasingly controversial as states continue to confront spiraling drug prices.

Limited options for states

In 2016, Pfizer sued Texas’ state health agency for giving data on the drug company’s supplemental Medicaid rebates to state lawmakers who requested it. The drugmaker alleged that releasing the confidential information would undermine the company’s competitiveness and give away trade secrets, and warned that the discounts it gave Texas could disappear.

In early October, a judge ruled that lawmakers should be able to obtain some of that data, noting dryly that “in Pfizer’s view, legislators are not necessary to carry out the state’s Medicaid program.”

Instead of seeking additional rebates from manufacturers for blood factor, some states, including Washington and Oregon, have chosen to require patients to get their blood factor from federally designated Hemophilia Treatment Centers only. That allows state Medicaid programs to take advantage of a federal drug-discount program known as “340B.”

However, officials in California said they studied that option and determined it wouldn’t save them any more money than the rebates they negotiate with drugmakers.

Whatever their approach, state health officials say they are struggling against forces they are nearly powerless to change.

“There aren’t a lot of options available to Medicaid programs in terms of controlling costs, because we don’t set the initial costs,” said Deborah Weston, pharmacy program manager for Oregon’s Medicaid program.
 

Kaiser Health News data correspondent Sydney Lupkin contributed to this report. KHN’s coverage of these topics is supported by Laura and John Arnold Foundation and Heising-Simons Foundation. This story was produced by Kaiser Health News, which publishes California Healthline, a service of the California Health Care Foundation. Kaiser Health News is a nonprofit news service covering health issues. It is an editorially independent program of the Kaiser Family Foundation that is not affiliated with Kaiser Permanente.

The child is well-known in the halls in which state bureaucrats oversee health care for millions of Californians – not by name, but by a number: $21 million.

His medications alone cost state taxpayers that much in a single year, not including other health care. The boy, whose identity has not been released, was California’s most expensive Medicaid patient in recent years. His case was singled out in a tweet last year by the state’s top health care official to highlight the public insurance program’s extraordinary obligations as a backstop for low-income patients.

How on earth can a single child’s treatment cost that much? The answer: He has hemophilia and needs large quantities of a pricey drug – known as clotting factor – that makes blood coagulate.

Hemophilia drugs are among the most costly drugs in the nation, and taxpayers are footing the bill for many patients on Medicaid who could never afford them on their own. Officials in California and other states are doing what they can to manage the costs, but it’s a daunting task that highlights the complexity and secrecy of prescription drug pricing.

Tab for 145 kids: $195 million

The California boy whose drugs cost $21 million in a single year was an extreme case, and the circumstances of his care have not been disclosed because of confidentiality protections. Still, medications to treat hemophilia on average annual cost more than $270,000 per patient, according to a 2015 Express Scripts report, and they can easily soar past $1 million annually.

In contrast to more common diseases like hepatitis C, hemophilia treatment is not a state “budget buster” per se: about 4,000 patients live in California. About 1,100 of them are covered by Medi-Cal or two other government-funded programs for chronically ill children in California, according to Jennifer Kent, director of the state Department of Health Care Services and author of last year’s tweet. But the amount of money spent per person dwarfs that spent on people with other serious diseases.

One Stanford University study of 34,000 California kids with severe chronic diseases found that the tiny portion of children who needed blood factor accounted for 41% of the state’s outpatient drug spending on this entire patient population. About $195 million was spent on just 145 kids over a 3-year period, although some of that money came back to the state in rebates from drug companies – a portion of the cost that Medicaid can recoup after purchase.

Caitlin Carroll, director of public affairs for PhRMA, the pharmaceutical industry lobbying group, said high development costs and the complicated and lengthy manufacturing process play a role in how hemophilia drugs are priced. She added that federally mandated rebates significantly reduce the cost of blood factor. They amount to 17% of the average manufacturer price per unit.

Manufacturers also note that some newer and more expensive hemophilia drugs last longer and do not need to be administered as frequently, so they can prove less costly to payers overall.

Even so, some patients require a monumental investment to survive.
 

‘Extremely fortunate’

Colleen Tuite’s son Kevin, a 7-year-old, has severe hemophilia with a complication known as an inhibitor – an antibody that makes his regular blood-factor infusions less effective. Inhibitors can dramatically increase the cost of care, because massive doses of blood factor or expensive, specialized blood products known as bypassing agents may be needed.

Ms. Tuite and her husband initially were Kevin’s foster parents, then adopted the boy as a toddler. Because he has been a foster child, Kevin qualifies for Medi-Cal until he is 26.

The Monrovia, Calif., family also has private health insurance, which pays for about half of Kevin’s medical bills. These can run upward of $200,000 per month, Ms. Tuite said.

“We definitely would not have been able to adopt him without the help of Medi-Cal,” Ms. Tuite said. “We’ve been extremely fortunate.”

With the support of drug manufacturers and hemophilia advocacy groups, patients and their families have significant political clout. Some experts say they also have a moral claim on public resources: In the early days of the AIDS epidemic, thousands of the nation’s hemophilia patients died after they contracted HIV through transfusions before the virus was eliminated from the blood supply.

State health officials say the costs of hemophilia are hard to anticipate and control, even with rebates.

“We do a really aggressive job of collecting rebates on our pharmacy costs,” said Ms. Kent, California’s top Medicaid official. “But there’s just not any way around blood factor. It is just a very, very expensive product. It’s nonnegotiable for people that require it.”

In 2016, California’s Medicaid program paid at least $205 million for medications used to treat hemophilia, according to a Kaiser Health News analysis of federal Medicaid data. That figure doesn’t account for the federal rebates.

States can negotiate “supplemental” rebates with drugmakers for individual medications – but those must be kept secret under federal and some state laws. Such secrecy is becoming increasingly controversial as states continue to confront spiraling drug prices.

Limited options for states

In 2016, Pfizer sued Texas’ state health agency for giving data on the drug company’s supplemental Medicaid rebates to state lawmakers who requested it. The drugmaker alleged that releasing the confidential information would undermine the company’s competitiveness and give away trade secrets, and warned that the discounts it gave Texas could disappear.

In early October, a judge ruled that lawmakers should be able to obtain some of that data, noting dryly that “in Pfizer’s view, legislators are not necessary to carry out the state’s Medicaid program.”

Instead of seeking additional rebates from manufacturers for blood factor, some states, including Washington and Oregon, have chosen to require patients to get their blood factor from federally designated Hemophilia Treatment Centers only. That allows state Medicaid programs to take advantage of a federal drug-discount program known as “340B.”

However, officials in California said they studied that option and determined it wouldn’t save them any more money than the rebates they negotiate with drugmakers.

Whatever their approach, state health officials say they are struggling against forces they are nearly powerless to change.

“There aren’t a lot of options available to Medicaid programs in terms of controlling costs, because we don’t set the initial costs,” said Deborah Weston, pharmacy program manager for Oregon’s Medicaid program.
 

Kaiser Health News data correspondent Sydney Lupkin contributed to this report. KHN’s coverage of these topics is supported by Laura and John Arnold Foundation and Heising-Simons Foundation. This story was produced by Kaiser Health News, which publishes California Healthline, a service of the California Health Care Foundation. Kaiser Health News is a nonprofit news service covering health issues. It is an editorially independent program of the Kaiser Family Foundation that is not affiliated with Kaiser Permanente.

SALT LAKE CITY – Scores of 3 or higher on the Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) are associated with an increased risk of posttransplant mortality in certain patients undergoing allogeneic HCT for nonmalignant disease, according to findings from a review of more than 4,000 patients.

The exception was in patients undergoing HCT for hemoglobinopathies, Larisa Broglie, MD, reported at the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation.

The findings of the study, which is the largest to date to validate the usefulness of the HCT-CI for risk assessment in HCT patients with nonmalignant disease, have important implications for patient counseling and decision making, said Dr. Broglie of the Medical College of Wisconsin, Milwaukee.

Of 4,083 children and adults who underwent a first allogeneic HCT for a nonmalignant disease between 2007 and 2014 and who had sufficient follow-up (median, 39 months), 61% had an HCT-CI score of 0, 20% had a score of 1-2, and 19% had a score of 3 or higher.

sworcester@frontlinemedcom.com

SOURCE: Broglie L et al. Abstract 16.

SALT LAKE CITY – Scores of 3 or higher on the Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) are associated with an increased risk of posttransplant mortality in certain patients undergoing allogeneic HCT for nonmalignant disease, according to findings from a review of more than 4,000 patients.

The exception was in patients undergoing HCT for hemoglobinopathies, Larisa Broglie, MD, reported at the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation.

The findings of the study, which is the largest to date to validate the usefulness of the HCT-CI for risk assessment in HCT patients with nonmalignant disease, have important implications for patient counseling and decision making, said Dr. Broglie of the Medical College of Wisconsin, Milwaukee.

Of 4,083 children and adults who underwent a first allogeneic HCT for a nonmalignant disease between 2007 and 2014 and who had sufficient follow-up (median, 39 months), 61% had an HCT-CI score of 0, 20% had a score of 1-2, and 19% had a score of 3 or higher.

sworcester@frontlinemedcom.com

SOURCE: Broglie L et al. Abstract 16.

SALT LAKE CITY – Scores of 3 or higher on the Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) are associated with an increased risk of posttransplant mortality in certain patients undergoing allogeneic HCT for nonmalignant disease, according to findings from a review of more than 4,000 patients.

The exception was in patients undergoing HCT for hemoglobinopathies, Larisa Broglie, MD, reported at the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation.

The findings of the study, which is the largest to date to validate the usefulness of the HCT-CI for risk assessment in HCT patients with nonmalignant disease, have important implications for patient counseling and decision making, said Dr. Broglie of the Medical College of Wisconsin, Milwaukee.

Of 4,083 children and adults who underwent a first allogeneic HCT for a nonmalignant disease between 2007 and 2014 and who had sufficient follow-up (median, 39 months), 61% had an HCT-CI score of 0, 20% had a score of 1-2, and 19% had a score of 3 or higher.

sworcester@frontlinemedcom.com

SOURCE: Broglie L et al. Abstract 16.

SAN DIEGO – A simple scoring system surpassed thermal imaging for diagnosing lower extremity cellulitis in a head-to-head, single-center comparison in 67 patients.

The ALT-70 score – which tallies points for asymmetry, leukocytosis, tachycardia, and age of at least 70 years – produced a positive predictive value for lower-extremity cellulitis (LEC) of 80.4% and a negative predictive value of 90.9%, compared with values of 75.5% and 57.1%, respectively, for thermal imaging when researchers applied both methods to 67 patients, said David G. Li, a clinical research fellow in the department of dermatology at Brigham and Women’s Hospital, Boston, where the study was conducted.

Mitchel Zoler

The four-item survey can generate a score of 0-7, with a score of 0-2 suggesting need for additional monitoring, a score of 3-4 initiating a dermatology consult, and a score of 5-7 triggering immediate treatment for cellulitis, Mr. Li said. The 2017 review of ALT-70 showed that among 259 patients, those with a score of 0-2 had an 83% likelihood of having pseudocellulitis, while patients with a score of 5-7 had an 82% likelihood of having true cellulitis.

Brigham and Women's Hospital

He also presented an analysis of the results when he combined both methods, with a positive on both assessments required to produce a positive LEC diagnosis. This resulted in a positive predictive value of 86.7%, slightly higher than the 80.4% from ALT-70 alone, but the combination produced a negative predictive value of 68.2%, substantially less than the 90.9% rate with ALT-70 alone. This demonstrated the “marginal benefit” from combining the two methods, he said.

In a receiver operating characteristic curve analysis, in which the area under the curve (c-statistic) reflects a diagnostic test’s validity, ALT-70 produced a c-statistic of 0.85, thermal imaging had a c-statistic of 0.63, and when combined, the c-statistic was 0.88.

Mr. Li called for validation of the findings using larger and different patient populations.

He had no reported disclosures.

mzoler@frontlinemedcom.com

SOURCE: Li DG et al. AAD 18, Abstract 6744.

SAN DIEGO – A simple scoring system surpassed thermal imaging for diagnosing lower extremity cellulitis in a head-to-head, single-center comparison in 67 patients.

The ALT-70 score – which tallies points for asymmetry, leukocytosis, tachycardia, and age of at least 70 years – produced a positive predictive value for lower-extremity cellulitis (LEC) of 80.4% and a negative predictive value of 90.9%, compared with values of 75.5% and 57.1%, respectively, for thermal imaging when researchers applied both methods to 67 patients, said David G. Li, a clinical research fellow in the department of dermatology at Brigham and Women’s Hospital, Boston, where the study was conducted.

Mitchel Zoler

The four-item survey can generate a score of 0-7, with a score of 0-2 suggesting need for additional monitoring, a score of 3-4 initiating a dermatology consult, and a score of 5-7 triggering immediate treatment for cellulitis, Mr. Li said. The 2017 review of ALT-70 showed that among 259 patients, those with a score of 0-2 had an 83% likelihood of having pseudocellulitis, while patients with a score of 5-7 had an 82% likelihood of having true cellulitis.

Brigham and Women's Hospital

He also presented an analysis of the results when he combined both methods, with a positive on both assessments required to produce a positive LEC diagnosis. This resulted in a positive predictive value of 86.7%, slightly higher than the 80.4% from ALT-70 alone, but the combination produced a negative predictive value of 68.2%, substantially less than the 90.9% rate with ALT-70 alone. This demonstrated the “marginal benefit” from combining the two methods, he said.

In a receiver operating characteristic curve analysis, in which the area under the curve (c-statistic) reflects a diagnostic test’s validity, ALT-70 produced a c-statistic of 0.85, thermal imaging had a c-statistic of 0.63, and when combined, the c-statistic was 0.88.

Mr. Li called for validation of the findings using larger and different patient populations.

He had no reported disclosures.

mzoler@frontlinemedcom.com

SOURCE: Li DG et al. AAD 18, Abstract 6744.

SAN DIEGO – A simple scoring system surpassed thermal imaging for diagnosing lower extremity cellulitis in a head-to-head, single-center comparison in 67 patients.

The ALT-70 score – which tallies points for asymmetry, leukocytosis, tachycardia, and age of at least 70 years – produced a positive predictive value for lower-extremity cellulitis (LEC) of 80.4% and a negative predictive value of 90.9%, compared with values of 75.5% and 57.1%, respectively, for thermal imaging when researchers applied both methods to 67 patients, said David G. Li, a clinical research fellow in the department of dermatology at Brigham and Women’s Hospital, Boston, where the study was conducted.

Mitchel Zoler

The four-item survey can generate a score of 0-7, with a score of 0-2 suggesting need for additional monitoring, a score of 3-4 initiating a dermatology consult, and a score of 5-7 triggering immediate treatment for cellulitis, Mr. Li said. The 2017 review of ALT-70 showed that among 259 patients, those with a score of 0-2 had an 83% likelihood of having pseudocellulitis, while patients with a score of 5-7 had an 82% likelihood of having true cellulitis.

Brigham and Women's Hospital

He also presented an analysis of the results when he combined both methods, with a positive on both assessments required to produce a positive LEC diagnosis. This resulted in a positive predictive value of 86.7%, slightly higher than the 80.4% from ALT-70 alone, but the combination produced a negative predictive value of 68.2%, substantially less than the 90.9% rate with ALT-70 alone. This demonstrated the “marginal benefit” from combining the two methods, he said.

In a receiver operating characteristic curve analysis, in which the area under the curve (c-statistic) reflects a diagnostic test’s validity, ALT-70 produced a c-statistic of 0.85, thermal imaging had a c-statistic of 0.63, and when combined, the c-statistic was 0.88.

Mr. Li called for validation of the findings using larger and different patient populations.

He had no reported disclosures.

mzoler@frontlinemedcom.com

SOURCE: Li DG et al. AAD 18, Abstract 6744.