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FDA issues guidance for hemophilia gene therapy development
The Food and Drug Administration issued its first-ever guidance on the development of human gene therapy for hemophilia.
The draft document provides a glimpse at the agency’s current thinking on clinical trial design and preclinical considerations in developing gene therapies. Specifically, it provides information to help companies set surrogate endpoints when seeking accelerated approval for hemophilia gene therapy products.
The hemophilia guidance is one of three disease-specific guidance documents. The other two clinical areas are retinal disorders and rare diseases.
FDA officials also issued updates to three more technical documents that deal with manufacturing of gene therapies.
The overall framework is similar to what FDA officials issued in 2017 for regenerative medicine. In a statement, FDA Commissioner Scott Gottlieb, MD, said the agency was responding to an increasing number of gene therapy approvals and anticipated development.
“Once just a theory, gene therapies are now a therapeutic reality for some patients,” Dr. Gottlieb said. “These platforms may have the potential to treat and cure some of our most intractable and vexing diseases. The policy framework we construct for how these products should be developed, reviewed by regulators, and reimbursed, will help set the stage for the continued advancement of this new market.”
The Food and Drug Administration issued its first-ever guidance on the development of human gene therapy for hemophilia.
The draft document provides a glimpse at the agency’s current thinking on clinical trial design and preclinical considerations in developing gene therapies. Specifically, it provides information to help companies set surrogate endpoints when seeking accelerated approval for hemophilia gene therapy products.
The hemophilia guidance is one of three disease-specific guidance documents. The other two clinical areas are retinal disorders and rare diseases.
FDA officials also issued updates to three more technical documents that deal with manufacturing of gene therapies.
The overall framework is similar to what FDA officials issued in 2017 for regenerative medicine. In a statement, FDA Commissioner Scott Gottlieb, MD, said the agency was responding to an increasing number of gene therapy approvals and anticipated development.
“Once just a theory, gene therapies are now a therapeutic reality for some patients,” Dr. Gottlieb said. “These platforms may have the potential to treat and cure some of our most intractable and vexing diseases. The policy framework we construct for how these products should be developed, reviewed by regulators, and reimbursed, will help set the stage for the continued advancement of this new market.”
The Food and Drug Administration issued its first-ever guidance on the development of human gene therapy for hemophilia.
The draft document provides a glimpse at the agency’s current thinking on clinical trial design and preclinical considerations in developing gene therapies. Specifically, it provides information to help companies set surrogate endpoints when seeking accelerated approval for hemophilia gene therapy products.
The hemophilia guidance is one of three disease-specific guidance documents. The other two clinical areas are retinal disorders and rare diseases.
FDA officials also issued updates to three more technical documents that deal with manufacturing of gene therapies.
The overall framework is similar to what FDA officials issued in 2017 for regenerative medicine. In a statement, FDA Commissioner Scott Gottlieb, MD, said the agency was responding to an increasing number of gene therapy approvals and anticipated development.
“Once just a theory, gene therapies are now a therapeutic reality for some patients,” Dr. Gottlieb said. “These platforms may have the potential to treat and cure some of our most intractable and vexing diseases. The policy framework we construct for how these products should be developed, reviewed by regulators, and reimbursed, will help set the stage for the continued advancement of this new market.”
Fish pedicures
A letter published in JAMA Dermatology describes an otherwise healthy woman in her 20s who experienced nail abnormalities some months after having a fish pedicure. Onychomadesis, or transverse splitting of the nail plate, occurs when the nail matrix has arrested in producing the nail plate. It can be thought of as more severe form of Beau’s lines, in which the nail itself actually breaks and separates from the proximal nail plate and eventually sheds.
Fish pedicures have a long-standing history in Mediterranean and Middle Eastern cultures for aiding such skin conditions as psoriasis and helping to remove scaly skin. The Garra rufa fish are nonmigratory freshwater fish native to the Persian Gulf and Eastern Mediterranean. Suction allows them to attach to rocks and eat plankton. These “doctor fish,” as they are nicknamed, when placed in a warm bath of 25°C to 30°C, will also eat human skin when starved of their natural food source. As the JAMA Dermatology letter mentions, this was demonstrated in a study in Kangal, Turkey, where Garra rufa fish were used to improve psoriasis by feeding on psoriasis plaques but not normal skin. After 3 weeks of therapy with Garra rufa in 67 patients, there was a 72% reduction in the Psoriasis Area and Severity Index (PASI) score from baseline (Evid Based Complement Alternat Med. 2006 Dec;3[4]:483-8).
Popular in the United States and Europe about a decade ago, fish pedicures have now been banned in 10 U.S. states and in some parts of Europe. While the trend in the United States has waned, fish pedicures have recently become more popular in vacation destinations, such as the Caribbean. The inherent concern of fish pedicures is risk of infection as the same fish are used successively and cannot be adequately sanitized between people.
Two cases of staphylococcus infections and one of Mycobacterium marinum have been reported after fish pedicures. Whether these infections were caused by the fish or the water source, however, remains to be determined. If the fish were transmitting infections, it seems that more infections would likely have been reported, considering the widespread popularity in the past. I, like Antonella Tosti, MD, who commented in a CNN report on the JAMA Dermatology case, also doubt that the fish pedicure alone caused onychomadesis in this woman. In order for onychomadesis to occur, there would have had to have been significant trauma to all 10 nails at the matrix. Would the fish been able to have caused the same amount of trauma to all 10 nails in one setting? While it is possible, I believe a more likely explanation would be an alternate endogenous or exogenous source.
Traditional medicine has been used to enhance beauty and cure ailments for thousands of years before the advent of modern medicine as demonstrated by the Kangal study. Before discounting fish pedicures completely, perhaps some thought should also be given to how this practice affects wildlife and the fish. The CNN report refers to a 2011 investigation by the U.K.’s Fish Health Inspectorate, which “found a bacterial outbreak among thousands of these fish, which had been transported from Indonesia to the United Kingdom pedicure spas. Fish were found with bulging eyes, many hemorrhaging around the gills and mouth. The culprit was found to be a streptococcal bacteria, a strain that is associated with fish like tilapia, according to David Verner-Jeffreys, a senior microbiologist at the Centre for Environment, Fisheries and Aquaculture Science in the U.K.”
Whether or not these fish would pose any risk to humans is unknown, but certainly, this practice adversely affects the welfare of the fish and their environment. The overharvesting of these fish has led the Turkish government to introduce legal protections for the country’s Garra rufa in an attempt to combat overfishing and exploitation.
Perhaps fish pedicures solely for aesthetic reasons should not be practiced because of the potential infection risk – as well as the harm (to both humans and fish) and overharvesting of the fish. If used properly, these fish, however, could be an aid in treating certain skin pathologies.
Dr. Wesley and Dr. Talakoub are co-contributors to this column. Dr. Wesley practices dermatology in Beverly Hills, Calif. Dr. Talakoub is in private practice in McLean, Va. This month’s column is by Dr. Wesley. Write to them at [email protected]. They had no relevant disclosures.
A letter published in JAMA Dermatology describes an otherwise healthy woman in her 20s who experienced nail abnormalities some months after having a fish pedicure. Onychomadesis, or transverse splitting of the nail plate, occurs when the nail matrix has arrested in producing the nail plate. It can be thought of as more severe form of Beau’s lines, in which the nail itself actually breaks and separates from the proximal nail plate and eventually sheds.
Fish pedicures have a long-standing history in Mediterranean and Middle Eastern cultures for aiding such skin conditions as psoriasis and helping to remove scaly skin. The Garra rufa fish are nonmigratory freshwater fish native to the Persian Gulf and Eastern Mediterranean. Suction allows them to attach to rocks and eat plankton. These “doctor fish,” as they are nicknamed, when placed in a warm bath of 25°C to 30°C, will also eat human skin when starved of their natural food source. As the JAMA Dermatology letter mentions, this was demonstrated in a study in Kangal, Turkey, where Garra rufa fish were used to improve psoriasis by feeding on psoriasis plaques but not normal skin. After 3 weeks of therapy with Garra rufa in 67 patients, there was a 72% reduction in the Psoriasis Area and Severity Index (PASI) score from baseline (Evid Based Complement Alternat Med. 2006 Dec;3[4]:483-8).
Popular in the United States and Europe about a decade ago, fish pedicures have now been banned in 10 U.S. states and in some parts of Europe. While the trend in the United States has waned, fish pedicures have recently become more popular in vacation destinations, such as the Caribbean. The inherent concern of fish pedicures is risk of infection as the same fish are used successively and cannot be adequately sanitized between people.
Two cases of staphylococcus infections and one of Mycobacterium marinum have been reported after fish pedicures. Whether these infections were caused by the fish or the water source, however, remains to be determined. If the fish were transmitting infections, it seems that more infections would likely have been reported, considering the widespread popularity in the past. I, like Antonella Tosti, MD, who commented in a CNN report on the JAMA Dermatology case, also doubt that the fish pedicure alone caused onychomadesis in this woman. In order for onychomadesis to occur, there would have had to have been significant trauma to all 10 nails at the matrix. Would the fish been able to have caused the same amount of trauma to all 10 nails in one setting? While it is possible, I believe a more likely explanation would be an alternate endogenous or exogenous source.
Traditional medicine has been used to enhance beauty and cure ailments for thousands of years before the advent of modern medicine as demonstrated by the Kangal study. Before discounting fish pedicures completely, perhaps some thought should also be given to how this practice affects wildlife and the fish. The CNN report refers to a 2011 investigation by the U.K.’s Fish Health Inspectorate, which “found a bacterial outbreak among thousands of these fish, which had been transported from Indonesia to the United Kingdom pedicure spas. Fish were found with bulging eyes, many hemorrhaging around the gills and mouth. The culprit was found to be a streptococcal bacteria, a strain that is associated with fish like tilapia, according to David Verner-Jeffreys, a senior microbiologist at the Centre for Environment, Fisheries and Aquaculture Science in the U.K.”
Whether or not these fish would pose any risk to humans is unknown, but certainly, this practice adversely affects the welfare of the fish and their environment. The overharvesting of these fish has led the Turkish government to introduce legal protections for the country’s Garra rufa in an attempt to combat overfishing and exploitation.
Perhaps fish pedicures solely for aesthetic reasons should not be practiced because of the potential infection risk – as well as the harm (to both humans and fish) and overharvesting of the fish. If used properly, these fish, however, could be an aid in treating certain skin pathologies.
Dr. Wesley and Dr. Talakoub are co-contributors to this column. Dr. Wesley practices dermatology in Beverly Hills, Calif. Dr. Talakoub is in private practice in McLean, Va. This month’s column is by Dr. Wesley. Write to them at [email protected]. They had no relevant disclosures.
A letter published in JAMA Dermatology describes an otherwise healthy woman in her 20s who experienced nail abnormalities some months after having a fish pedicure. Onychomadesis, or transverse splitting of the nail plate, occurs when the nail matrix has arrested in producing the nail plate. It can be thought of as more severe form of Beau’s lines, in which the nail itself actually breaks and separates from the proximal nail plate and eventually sheds.
Fish pedicures have a long-standing history in Mediterranean and Middle Eastern cultures for aiding such skin conditions as psoriasis and helping to remove scaly skin. The Garra rufa fish are nonmigratory freshwater fish native to the Persian Gulf and Eastern Mediterranean. Suction allows them to attach to rocks and eat plankton. These “doctor fish,” as they are nicknamed, when placed in a warm bath of 25°C to 30°C, will also eat human skin when starved of their natural food source. As the JAMA Dermatology letter mentions, this was demonstrated in a study in Kangal, Turkey, where Garra rufa fish were used to improve psoriasis by feeding on psoriasis plaques but not normal skin. After 3 weeks of therapy with Garra rufa in 67 patients, there was a 72% reduction in the Psoriasis Area and Severity Index (PASI) score from baseline (Evid Based Complement Alternat Med. 2006 Dec;3[4]:483-8).
Popular in the United States and Europe about a decade ago, fish pedicures have now been banned in 10 U.S. states and in some parts of Europe. While the trend in the United States has waned, fish pedicures have recently become more popular in vacation destinations, such as the Caribbean. The inherent concern of fish pedicures is risk of infection as the same fish are used successively and cannot be adequately sanitized between people.
Two cases of staphylococcus infections and one of Mycobacterium marinum have been reported after fish pedicures. Whether these infections were caused by the fish or the water source, however, remains to be determined. If the fish were transmitting infections, it seems that more infections would likely have been reported, considering the widespread popularity in the past. I, like Antonella Tosti, MD, who commented in a CNN report on the JAMA Dermatology case, also doubt that the fish pedicure alone caused onychomadesis in this woman. In order for onychomadesis to occur, there would have had to have been significant trauma to all 10 nails at the matrix. Would the fish been able to have caused the same amount of trauma to all 10 nails in one setting? While it is possible, I believe a more likely explanation would be an alternate endogenous or exogenous source.
Traditional medicine has been used to enhance beauty and cure ailments for thousands of years before the advent of modern medicine as demonstrated by the Kangal study. Before discounting fish pedicures completely, perhaps some thought should also be given to how this practice affects wildlife and the fish. The CNN report refers to a 2011 investigation by the U.K.’s Fish Health Inspectorate, which “found a bacterial outbreak among thousands of these fish, which had been transported from Indonesia to the United Kingdom pedicure spas. Fish were found with bulging eyes, many hemorrhaging around the gills and mouth. The culprit was found to be a streptococcal bacteria, a strain that is associated with fish like tilapia, according to David Verner-Jeffreys, a senior microbiologist at the Centre for Environment, Fisheries and Aquaculture Science in the U.K.”
Whether or not these fish would pose any risk to humans is unknown, but certainly, this practice adversely affects the welfare of the fish and their environment. The overharvesting of these fish has led the Turkish government to introduce legal protections for the country’s Garra rufa in an attempt to combat overfishing and exploitation.
Perhaps fish pedicures solely for aesthetic reasons should not be practiced because of the potential infection risk – as well as the harm (to both humans and fish) and overharvesting of the fish. If used properly, these fish, however, could be an aid in treating certain skin pathologies.
Dr. Wesley and Dr. Talakoub are co-contributors to this column. Dr. Wesley practices dermatology in Beverly Hills, Calif. Dr. Talakoub is in private practice in McLean, Va. This month’s column is by Dr. Wesley. Write to them at [email protected]. They had no relevant disclosures.
Medicare’s bundled pay plan didn’t deliver big cost savings
Participation in Medicare’s bundled payments initiative didn’t significantly change payments per episode or care outcomes for the top five medical conditions selected under the program, a new analysis shows.
Payments for the common conditions remained around $24,000 per episode before and during participation in the Bundled Payments for Care Improvement (BPCI) initiative for the 125 participating hospitals evaluated in this study, conducted by Karen E. Joynt Maddox, MD, of Washington University, St. Louis, and her coauthors.
The finding contrasts with a previous study showing that hospitals in BPCI successfully lowered overall Medicare payments for patients who underwent joint replacement.
“Bundling of services to encourage more efficient care has great face validity and enjoys bipartisan support,” Dr. Joynt Maddox and her colleagues wrote. “For such bundling to work for medical conditions, however, more time, new care strategies and partnerships, or additional incentives may be required.”
The Center for Medicare & Medicaid Innovation initiated the voluntary BPCI demonstration project in 2013. The program targets 48 conditions that account for about 70% of Medicare spending. Hospitals that achieve cost targets for a specific condition get to keep a portion of the savings, and they reimburse Medicare for part of the difference when costs are exceeded.
The present study focused on 2013-2015 Medicare claims for the five medical conditions that account for two-thirds of patients enrolled in medical bundles: congestive heart failure, pneumonia, chronic obstructive pulmonary disease, sepsis, and acute myocardial infarction.
Mean baseline payments per episode for those conditions were $24,280 before participation in the BPCI. After hospitals joined, their average payments per episode were $23,993 (P = .41). For a set of matched control hospitals, payments were a mean of $23,901 at baseline and $23,503 in the corresponding follow-up period (P = .08).
That amounted to a $286 payment reduction for BPCI hospitals and a $398 reduction for controls, a difference of $112 (P = .79), the study investigators reported.
Changes in length of stay, readmissions, emergency department use, and clinical complexity of cases from baseline to follow-up periods was not significantly different between BPCI and control hospitals. For example, 90-day mortality increases were seen in both groups, and the degree of increase was not statistically different between the groups.
Those data help fill a gap in research on the BPCI program and BPCI Advanced, a related version of the demonstration project that will have its first cohort of participants starting Oct. 1, 2018.
“Despite the importance of episode-based payment, there has been little research examining its efficacy or determining whether it has unintended consequences, such as hospitals’ selecting patients with relatively less complex conditions to reduce costs and improve outcomes,” Dr. Joynt Maddox and her colleagues cautioned.
It’s unclear why the previous joint replacement study showed a successful reduction in costs under BPCI, while the new study did not. However, patients in the new analysis of the most common bundled conditions were older and had higher rates of poverty and disability.
“As a result of these complexities, patients admitted for medical conditions may have had post-acute care needs that were less amenable to intervention,” Dr. Joynt Maddox said.
The investigators added that hospitals’ lack of effective influence on post–acute-care services may blunt their ability to achieve greater savings under BPCI. Better relationships with skilled nursing facilities, long-term care hospitals, home health agencies, and inpatient rehabilitation facilities could make a difference.
The Commonwealth Fund supported the study. One study author reported personal fees from HHS outside the submitted work, and another reported that he is an associate editor for the New England Journal of Medicine. No other disclosures were reported.
SOURCE: Joynt Maddox KE et al. N Engl J Med. 2018 Jul 19;379(3):260-9.
Participation in Medicare’s bundled payments initiative didn’t significantly change payments per episode or care outcomes for the top five medical conditions selected under the program, a new analysis shows.
Payments for the common conditions remained around $24,000 per episode before and during participation in the Bundled Payments for Care Improvement (BPCI) initiative for the 125 participating hospitals evaluated in this study, conducted by Karen E. Joynt Maddox, MD, of Washington University, St. Louis, and her coauthors.
The finding contrasts with a previous study showing that hospitals in BPCI successfully lowered overall Medicare payments for patients who underwent joint replacement.
“Bundling of services to encourage more efficient care has great face validity and enjoys bipartisan support,” Dr. Joynt Maddox and her colleagues wrote. “For such bundling to work for medical conditions, however, more time, new care strategies and partnerships, or additional incentives may be required.”
The Center for Medicare & Medicaid Innovation initiated the voluntary BPCI demonstration project in 2013. The program targets 48 conditions that account for about 70% of Medicare spending. Hospitals that achieve cost targets for a specific condition get to keep a portion of the savings, and they reimburse Medicare for part of the difference when costs are exceeded.
The present study focused on 2013-2015 Medicare claims for the five medical conditions that account for two-thirds of patients enrolled in medical bundles: congestive heart failure, pneumonia, chronic obstructive pulmonary disease, sepsis, and acute myocardial infarction.
Mean baseline payments per episode for those conditions were $24,280 before participation in the BPCI. After hospitals joined, their average payments per episode were $23,993 (P = .41). For a set of matched control hospitals, payments were a mean of $23,901 at baseline and $23,503 in the corresponding follow-up period (P = .08).
That amounted to a $286 payment reduction for BPCI hospitals and a $398 reduction for controls, a difference of $112 (P = .79), the study investigators reported.
Changes in length of stay, readmissions, emergency department use, and clinical complexity of cases from baseline to follow-up periods was not significantly different between BPCI and control hospitals. For example, 90-day mortality increases were seen in both groups, and the degree of increase was not statistically different between the groups.
Those data help fill a gap in research on the BPCI program and BPCI Advanced, a related version of the demonstration project that will have its first cohort of participants starting Oct. 1, 2018.
“Despite the importance of episode-based payment, there has been little research examining its efficacy or determining whether it has unintended consequences, such as hospitals’ selecting patients with relatively less complex conditions to reduce costs and improve outcomes,” Dr. Joynt Maddox and her colleagues cautioned.
It’s unclear why the previous joint replacement study showed a successful reduction in costs under BPCI, while the new study did not. However, patients in the new analysis of the most common bundled conditions were older and had higher rates of poverty and disability.
“As a result of these complexities, patients admitted for medical conditions may have had post-acute care needs that were less amenable to intervention,” Dr. Joynt Maddox said.
The investigators added that hospitals’ lack of effective influence on post–acute-care services may blunt their ability to achieve greater savings under BPCI. Better relationships with skilled nursing facilities, long-term care hospitals, home health agencies, and inpatient rehabilitation facilities could make a difference.
The Commonwealth Fund supported the study. One study author reported personal fees from HHS outside the submitted work, and another reported that he is an associate editor for the New England Journal of Medicine. No other disclosures were reported.
SOURCE: Joynt Maddox KE et al. N Engl J Med. 2018 Jul 19;379(3):260-9.
Participation in Medicare’s bundled payments initiative didn’t significantly change payments per episode or care outcomes for the top five medical conditions selected under the program, a new analysis shows.
Payments for the common conditions remained around $24,000 per episode before and during participation in the Bundled Payments for Care Improvement (BPCI) initiative for the 125 participating hospitals evaluated in this study, conducted by Karen E. Joynt Maddox, MD, of Washington University, St. Louis, and her coauthors.
The finding contrasts with a previous study showing that hospitals in BPCI successfully lowered overall Medicare payments for patients who underwent joint replacement.
“Bundling of services to encourage more efficient care has great face validity and enjoys bipartisan support,” Dr. Joynt Maddox and her colleagues wrote. “For such bundling to work for medical conditions, however, more time, new care strategies and partnerships, or additional incentives may be required.”
The Center for Medicare & Medicaid Innovation initiated the voluntary BPCI demonstration project in 2013. The program targets 48 conditions that account for about 70% of Medicare spending. Hospitals that achieve cost targets for a specific condition get to keep a portion of the savings, and they reimburse Medicare for part of the difference when costs are exceeded.
The present study focused on 2013-2015 Medicare claims for the five medical conditions that account for two-thirds of patients enrolled in medical bundles: congestive heart failure, pneumonia, chronic obstructive pulmonary disease, sepsis, and acute myocardial infarction.
Mean baseline payments per episode for those conditions were $24,280 before participation in the BPCI. After hospitals joined, their average payments per episode were $23,993 (P = .41). For a set of matched control hospitals, payments were a mean of $23,901 at baseline and $23,503 in the corresponding follow-up period (P = .08).
That amounted to a $286 payment reduction for BPCI hospitals and a $398 reduction for controls, a difference of $112 (P = .79), the study investigators reported.
Changes in length of stay, readmissions, emergency department use, and clinical complexity of cases from baseline to follow-up periods was not significantly different between BPCI and control hospitals. For example, 90-day mortality increases were seen in both groups, and the degree of increase was not statistically different between the groups.
Those data help fill a gap in research on the BPCI program and BPCI Advanced, a related version of the demonstration project that will have its first cohort of participants starting Oct. 1, 2018.
“Despite the importance of episode-based payment, there has been little research examining its efficacy or determining whether it has unintended consequences, such as hospitals’ selecting patients with relatively less complex conditions to reduce costs and improve outcomes,” Dr. Joynt Maddox and her colleagues cautioned.
It’s unclear why the previous joint replacement study showed a successful reduction in costs under BPCI, while the new study did not. However, patients in the new analysis of the most common bundled conditions were older and had higher rates of poverty and disability.
“As a result of these complexities, patients admitted for medical conditions may have had post-acute care needs that were less amenable to intervention,” Dr. Joynt Maddox said.
The investigators added that hospitals’ lack of effective influence on post–acute-care services may blunt their ability to achieve greater savings under BPCI. Better relationships with skilled nursing facilities, long-term care hospitals, home health agencies, and inpatient rehabilitation facilities could make a difference.
The Commonwealth Fund supported the study. One study author reported personal fees from HHS outside the submitted work, and another reported that he is an associate editor for the New England Journal of Medicine. No other disclosures were reported.
SOURCE: Joynt Maddox KE et al. N Engl J Med. 2018 Jul 19;379(3):260-9.
FROM NEW ENGLAND JOURNAL OF MEDICINE
Key clinical point: Participation in Medicare’s Bundled Payments for Care Improvement (BPCI) initiative didn’t significantly change payments per episode for the top five medical conditions selected under the program.
Major finding: Baseline payments per episode for those conditions were a mean of $24,280 before participation in the BPCI, and $23,993 after adoption (P = .41).
Study details: A retrospective analysis of Medicare data for 125 hospitals participating in the program and matched control hospitals.
Disclosures: The Commonwealth Fund supported the study. One study author reported personal fees from HHS outside the submitted work, and another reported that he is an associate editor for the New England Journal of Medicine.
Source: Joynt Maddox KE et al. N Engl J Med. 2018 Jul 19;379(3):260-9.
Site of morphea lesions predicts risk of extracutaneous manifestations
LAKE TAHOE, CALIF. – Morphea lesions on the extensor extremities, face, and superior head are associated with higher rates of extracutaneous involvement, results from a multicenter retrospective study showed.
“We know that risk is highest with linear morphea,” lead study author Yvonne E. Chiu, MD, said at the annual meeting of the Society for Pediatric Dermatology. “Specifically, . However, risk stratification within each of those sites has never really been studied before.”
Dr. Chiu, who is a pediatric dermatologist at the Medical College of Wisconsin and Children’s Hospital of Wisconsin in Milwaukee, and her associates carried out a 14-site retrospective study in an effort to characterize morphea lesional distribution and to determine which sites had the highest risk for extracutaneous manifestations. They limited the analysis to patients with pediatric-onset morphea before the age of 18 and adequate lesional photographs in their clinical record. Patients with extragenital lichen sclerosis and atrophoderma were included in the analysis, but those with pansclerotic morphea and eosinophilic fasciitis were excluded. The researchers used custom web-based software to map the morphea lesions, and linked those data to a REDCap database where demographic and clinical information was stored. From this, the researchers tracked neurologic symptoms such as seizures, migraine headaches, other headaches, or any other neurologic signs or symptoms; neurologic testing results from those who underwent MRI, CT, and EEG; musculoskeletal symptoms such as arthritis, arthralgias, joint contracture, leg length discrepancy, and other musculoskeletal issues, as well as ophthalmologic manifestations including uveitis and other ophthalmologic symptoms. Logistic regression was used to analyze association of body sites with extracutaneous involvement.
Dr. Chiu, who also directs the dermatology residency program at the Medical College of Wisconsin, reported findings from 826 patients with 2,467 skin lesions of morphea, or an average of about 1.92 lesions per patient. Consistent with prior reports, most patients were female (73%), and the most prevalent subtype was linear morphea (56%), followed by plaque (29%), generalized (8%), and mixed (7%).
The trunk was the single most commonly affected body site, seen in 36% of cases. “However, if you lumped all body sites together, the extremities were the most commonly affected site (44%), while 16% of lesions involved the head and 4% involved the neck,” Dr. Chiu said. Patients with linear morphea had the highest rate of extracutaneous involvement. Specifically, 34% had musculoskeletal involvement, 24% had neurologic involvement, and 10% had ophthalmologic involvement. There were small rates of extracutaneous manifestations in the other types of morphea as well.
The most common musculoskeletal complications among patients with linear morphea were arthralgias (20%) and joint contractures (17%), followed by other musculoskeletal complications (15%), leg length discrepancy (5%), and arthritis (2%). Contrary to previously published reports, nonmigraine headaches were more common than seizures among patients with linear morphea (17% vs. 4%, respectively), while 4% of subjects had migraine headaches. Of the 134 subjects who underwent neuroimaging, 19% had abnormal results. Ophthalmologic complications were rare among patients overall, with the exception of those who had linear morphea. Of these cases, 1% had uveitis, and 9% had some other ophthalmologic condition.
Among all patients, the researchers found that left-extremity and extensor-extremity lesions had a stronger association with musculoskeletal involvement (odds ratios of 1.26 and 1.94, respectively). “The reasons for this are unclear,” Dr. Chiu said. “We didn’t assess handedness in our study, but that perhaps could explain it; 90% of the general population is right-hand dominant, so perhaps there’s some sort of protective effect if you’re using an extremity more. Joint contractures showed the greatest discrepancy between left and right extremity. So perhaps if you’re using that one side more, you’re less likely to have a joint contracture.”
When the researchers limited the analysis to head lesions, they observed no significant difference in the lesions between the left and right head (OR, 0.72), but anterior head lesions had a stronger association with neurologic signs or symptoms, compared with posterior head lesions (OR, 2.56), as did superior head lesions, compared with inferior head lesions (OR, 2.23). The association between head lesion location and ophthalmologic involvement was not significant.
“The odds of extracutaneous manifestations vary by site of morphea lesions, with higher odds seen on the left extremity, extensor extremity, the anterior head, and the superior head,” Dr. Chiu concluded. “Further research can be done to perhaps help us decide whether this necessitates difference in management or screening.”
The project was funded by the Pediatric Dermatology Research Alliance and the SPD. Dr. Chiu reported having no relevant financial disclosures.
[email protected]
LAKE TAHOE, CALIF. – Morphea lesions on the extensor extremities, face, and superior head are associated with higher rates of extracutaneous involvement, results from a multicenter retrospective study showed.
“We know that risk is highest with linear morphea,” lead study author Yvonne E. Chiu, MD, said at the annual meeting of the Society for Pediatric Dermatology. “Specifically, . However, risk stratification within each of those sites has never really been studied before.”
Dr. Chiu, who is a pediatric dermatologist at the Medical College of Wisconsin and Children’s Hospital of Wisconsin in Milwaukee, and her associates carried out a 14-site retrospective study in an effort to characterize morphea lesional distribution and to determine which sites had the highest risk for extracutaneous manifestations. They limited the analysis to patients with pediatric-onset morphea before the age of 18 and adequate lesional photographs in their clinical record. Patients with extragenital lichen sclerosis and atrophoderma were included in the analysis, but those with pansclerotic morphea and eosinophilic fasciitis were excluded. The researchers used custom web-based software to map the morphea lesions, and linked those data to a REDCap database where demographic and clinical information was stored. From this, the researchers tracked neurologic symptoms such as seizures, migraine headaches, other headaches, or any other neurologic signs or symptoms; neurologic testing results from those who underwent MRI, CT, and EEG; musculoskeletal symptoms such as arthritis, arthralgias, joint contracture, leg length discrepancy, and other musculoskeletal issues, as well as ophthalmologic manifestations including uveitis and other ophthalmologic symptoms. Logistic regression was used to analyze association of body sites with extracutaneous involvement.
Dr. Chiu, who also directs the dermatology residency program at the Medical College of Wisconsin, reported findings from 826 patients with 2,467 skin lesions of morphea, or an average of about 1.92 lesions per patient. Consistent with prior reports, most patients were female (73%), and the most prevalent subtype was linear morphea (56%), followed by plaque (29%), generalized (8%), and mixed (7%).
The trunk was the single most commonly affected body site, seen in 36% of cases. “However, if you lumped all body sites together, the extremities were the most commonly affected site (44%), while 16% of lesions involved the head and 4% involved the neck,” Dr. Chiu said. Patients with linear morphea had the highest rate of extracutaneous involvement. Specifically, 34% had musculoskeletal involvement, 24% had neurologic involvement, and 10% had ophthalmologic involvement. There were small rates of extracutaneous manifestations in the other types of morphea as well.
The most common musculoskeletal complications among patients with linear morphea were arthralgias (20%) and joint contractures (17%), followed by other musculoskeletal complications (15%), leg length discrepancy (5%), and arthritis (2%). Contrary to previously published reports, nonmigraine headaches were more common than seizures among patients with linear morphea (17% vs. 4%, respectively), while 4% of subjects had migraine headaches. Of the 134 subjects who underwent neuroimaging, 19% had abnormal results. Ophthalmologic complications were rare among patients overall, with the exception of those who had linear morphea. Of these cases, 1% had uveitis, and 9% had some other ophthalmologic condition.
Among all patients, the researchers found that left-extremity and extensor-extremity lesions had a stronger association with musculoskeletal involvement (odds ratios of 1.26 and 1.94, respectively). “The reasons for this are unclear,” Dr. Chiu said. “We didn’t assess handedness in our study, but that perhaps could explain it; 90% of the general population is right-hand dominant, so perhaps there’s some sort of protective effect if you’re using an extremity more. Joint contractures showed the greatest discrepancy between left and right extremity. So perhaps if you’re using that one side more, you’re less likely to have a joint contracture.”
When the researchers limited the analysis to head lesions, they observed no significant difference in the lesions between the left and right head (OR, 0.72), but anterior head lesions had a stronger association with neurologic signs or symptoms, compared with posterior head lesions (OR, 2.56), as did superior head lesions, compared with inferior head lesions (OR, 2.23). The association between head lesion location and ophthalmologic involvement was not significant.
“The odds of extracutaneous manifestations vary by site of morphea lesions, with higher odds seen on the left extremity, extensor extremity, the anterior head, and the superior head,” Dr. Chiu concluded. “Further research can be done to perhaps help us decide whether this necessitates difference in management or screening.”
The project was funded by the Pediatric Dermatology Research Alliance and the SPD. Dr. Chiu reported having no relevant financial disclosures.
[email protected]
LAKE TAHOE, CALIF. – Morphea lesions on the extensor extremities, face, and superior head are associated with higher rates of extracutaneous involvement, results from a multicenter retrospective study showed.
“We know that risk is highest with linear morphea,” lead study author Yvonne E. Chiu, MD, said at the annual meeting of the Society for Pediatric Dermatology. “Specifically, . However, risk stratification within each of those sites has never really been studied before.”
Dr. Chiu, who is a pediatric dermatologist at the Medical College of Wisconsin and Children’s Hospital of Wisconsin in Milwaukee, and her associates carried out a 14-site retrospective study in an effort to characterize morphea lesional distribution and to determine which sites had the highest risk for extracutaneous manifestations. They limited the analysis to patients with pediatric-onset morphea before the age of 18 and adequate lesional photographs in their clinical record. Patients with extragenital lichen sclerosis and atrophoderma were included in the analysis, but those with pansclerotic morphea and eosinophilic fasciitis were excluded. The researchers used custom web-based software to map the morphea lesions, and linked those data to a REDCap database where demographic and clinical information was stored. From this, the researchers tracked neurologic symptoms such as seizures, migraine headaches, other headaches, or any other neurologic signs or symptoms; neurologic testing results from those who underwent MRI, CT, and EEG; musculoskeletal symptoms such as arthritis, arthralgias, joint contracture, leg length discrepancy, and other musculoskeletal issues, as well as ophthalmologic manifestations including uveitis and other ophthalmologic symptoms. Logistic regression was used to analyze association of body sites with extracutaneous involvement.
Dr. Chiu, who also directs the dermatology residency program at the Medical College of Wisconsin, reported findings from 826 patients with 2,467 skin lesions of morphea, or an average of about 1.92 lesions per patient. Consistent with prior reports, most patients were female (73%), and the most prevalent subtype was linear morphea (56%), followed by plaque (29%), generalized (8%), and mixed (7%).
The trunk was the single most commonly affected body site, seen in 36% of cases. “However, if you lumped all body sites together, the extremities were the most commonly affected site (44%), while 16% of lesions involved the head and 4% involved the neck,” Dr. Chiu said. Patients with linear morphea had the highest rate of extracutaneous involvement. Specifically, 34% had musculoskeletal involvement, 24% had neurologic involvement, and 10% had ophthalmologic involvement. There were small rates of extracutaneous manifestations in the other types of morphea as well.
The most common musculoskeletal complications among patients with linear morphea were arthralgias (20%) and joint contractures (17%), followed by other musculoskeletal complications (15%), leg length discrepancy (5%), and arthritis (2%). Contrary to previously published reports, nonmigraine headaches were more common than seizures among patients with linear morphea (17% vs. 4%, respectively), while 4% of subjects had migraine headaches. Of the 134 subjects who underwent neuroimaging, 19% had abnormal results. Ophthalmologic complications were rare among patients overall, with the exception of those who had linear morphea. Of these cases, 1% had uveitis, and 9% had some other ophthalmologic condition.
Among all patients, the researchers found that left-extremity and extensor-extremity lesions had a stronger association with musculoskeletal involvement (odds ratios of 1.26 and 1.94, respectively). “The reasons for this are unclear,” Dr. Chiu said. “We didn’t assess handedness in our study, but that perhaps could explain it; 90% of the general population is right-hand dominant, so perhaps there’s some sort of protective effect if you’re using an extremity more. Joint contractures showed the greatest discrepancy between left and right extremity. So perhaps if you’re using that one side more, you’re less likely to have a joint contracture.”
When the researchers limited the analysis to head lesions, they observed no significant difference in the lesions between the left and right head (OR, 0.72), but anterior head lesions had a stronger association with neurologic signs or symptoms, compared with posterior head lesions (OR, 2.56), as did superior head lesions, compared with inferior head lesions (OR, 2.23). The association between head lesion location and ophthalmologic involvement was not significant.
“The odds of extracutaneous manifestations vary by site of morphea lesions, with higher odds seen on the left extremity, extensor extremity, the anterior head, and the superior head,” Dr. Chiu concluded. “Further research can be done to perhaps help us decide whether this necessitates difference in management or screening.”
The project was funded by the Pediatric Dermatology Research Alliance and the SPD. Dr. Chiu reported having no relevant financial disclosures.
[email protected]
REPORTING FROM SPD 2018
Key clinical point: Extracutaneous involvement is more likely when morphea lesions are present on the extensor extremities, face, and superior head.
Major finding: Patients with linear morphea had the highest rate of extracutaneous involvement. Specifically, 34% had musculoskeletal involvement, 24% had neurologic involvement, and 10% had ophthalmologic involvement.
Study details: A multicenter retrospective study of 826 patients with 2,467 skin lesions of morphea.
Disclosures: The study was funded by the Pediatric Dermatology Research Alliance and the SPD. Dr. Chiu reported having no relevant financial disclosures.
Pfizer launches phase 3 gene therapy study in hemophilia B
Pfizer has begun early work on a phase 3 study of an investigational gene therapy to treat hemophilia B.
Pfizer, along with Spark Therapeutics, launched a phase 3 lead-in study at multiple centers to evaluate the efficacy and safety of current factor IX prophylaxis replacement therapy in the usual care setting. The efficacy data from the lead-in study will become the within-subject control group for patients who enroll in the next part of the
Fidanacogene elaparvovec is a vector containing a bioengineered adeno-associated virus capsid and a high-activity human coagulation factor IX gene. In an ongoing phase 1/2 trial of fidanacogene elaparvovec, all 15 participants with hemophilia B were able to discontinue routine infusion of factor IX concentrates without serious adverse events, according to data released by Pfizer and Spark Therapeutics in May 2018.
Pfizer has begun early work on a phase 3 study of an investigational gene therapy to treat hemophilia B.
Pfizer, along with Spark Therapeutics, launched a phase 3 lead-in study at multiple centers to evaluate the efficacy and safety of current factor IX prophylaxis replacement therapy in the usual care setting. The efficacy data from the lead-in study will become the within-subject control group for patients who enroll in the next part of the
Fidanacogene elaparvovec is a vector containing a bioengineered adeno-associated virus capsid and a high-activity human coagulation factor IX gene. In an ongoing phase 1/2 trial of fidanacogene elaparvovec, all 15 participants with hemophilia B were able to discontinue routine infusion of factor IX concentrates without serious adverse events, according to data released by Pfizer and Spark Therapeutics in May 2018.
Pfizer has begun early work on a phase 3 study of an investigational gene therapy to treat hemophilia B.
Pfizer, along with Spark Therapeutics, launched a phase 3 lead-in study at multiple centers to evaluate the efficacy and safety of current factor IX prophylaxis replacement therapy in the usual care setting. The efficacy data from the lead-in study will become the within-subject control group for patients who enroll in the next part of the
Fidanacogene elaparvovec is a vector containing a bioengineered adeno-associated virus capsid and a high-activity human coagulation factor IX gene. In an ongoing phase 1/2 trial of fidanacogene elaparvovec, all 15 participants with hemophilia B were able to discontinue routine infusion of factor IX concentrates without serious adverse events, according to data released by Pfizer and Spark Therapeutics in May 2018.
Extended data support ixekizumab for plaque psoriasis
followed over a 4-year extension of a randomized trial.
Patients from a previous study of ixekizumab were treated with 120 mg at the start of the extension, and then 80 mg subcutaneously every 4 weeks, Claus Zachariae, MD, of the University Hospital of Copenhagen Gentofte, and his coauthors reported in the Journal of the American Academy of Dermatology.
At week 208, 82% of the patients achieved Psoriasis Area and Severity Index (PASI) 75, 65% achieved PASI 90, and 45% achieved PASI 100; 65% scored a 0 or 1 on the Physician’s Global Assessment Scale. In addition, 45% of patients reported a score of 0 on the Physician Global Assessment. Patients also reported a decrease in itching from baseline.
A total of 17% of patients experienced a serious adverse event and 87% of the patients experienced at least one treatment-related adverse event by the end of the 4-year extension period. Most of the reported events were mild to moderate; the most common were nasopharyngitis (23%), sinusitis (13%), upper respiratory tract infection (13%), and headache (10%).
The study findings were limited by several factors including the lack of blinding and lack of a placebo, the researchers noted.
However, the results demonstrate “that efficacy can be maintained at high levels for up to 4 years of ixekizumab therapy without apparent increases in health risks or safety issues,” for psoriasis patients, Dr. Zachariae and his associates said. “Longer treatment periods in larger numbers of patients will be reported for patients enrolled in the 5-year phase 3 ixekizumab studies.”
The study was supported by Eli Lilly. Dr. Zachariae disclosed relationships with Eli Lilly and other companies including Janssen, Novartis, AbbVie, and Amgen. His coauthors had financial relationships with multiple companies.
SOURCE: Zachariae C et al. J Am Acad Dermatol. 2018 Aug;79(2):294-301.e6.
followed over a 4-year extension of a randomized trial.
Patients from a previous study of ixekizumab were treated with 120 mg at the start of the extension, and then 80 mg subcutaneously every 4 weeks, Claus Zachariae, MD, of the University Hospital of Copenhagen Gentofte, and his coauthors reported in the Journal of the American Academy of Dermatology.
At week 208, 82% of the patients achieved Psoriasis Area and Severity Index (PASI) 75, 65% achieved PASI 90, and 45% achieved PASI 100; 65% scored a 0 or 1 on the Physician’s Global Assessment Scale. In addition, 45% of patients reported a score of 0 on the Physician Global Assessment. Patients also reported a decrease in itching from baseline.
A total of 17% of patients experienced a serious adverse event and 87% of the patients experienced at least one treatment-related adverse event by the end of the 4-year extension period. Most of the reported events were mild to moderate; the most common were nasopharyngitis (23%), sinusitis (13%), upper respiratory tract infection (13%), and headache (10%).
The study findings were limited by several factors including the lack of blinding and lack of a placebo, the researchers noted.
However, the results demonstrate “that efficacy can be maintained at high levels for up to 4 years of ixekizumab therapy without apparent increases in health risks or safety issues,” for psoriasis patients, Dr. Zachariae and his associates said. “Longer treatment periods in larger numbers of patients will be reported for patients enrolled in the 5-year phase 3 ixekizumab studies.”
The study was supported by Eli Lilly. Dr. Zachariae disclosed relationships with Eli Lilly and other companies including Janssen, Novartis, AbbVie, and Amgen. His coauthors had financial relationships with multiple companies.
SOURCE: Zachariae C et al. J Am Acad Dermatol. 2018 Aug;79(2):294-301.e6.
followed over a 4-year extension of a randomized trial.
Patients from a previous study of ixekizumab were treated with 120 mg at the start of the extension, and then 80 mg subcutaneously every 4 weeks, Claus Zachariae, MD, of the University Hospital of Copenhagen Gentofte, and his coauthors reported in the Journal of the American Academy of Dermatology.
At week 208, 82% of the patients achieved Psoriasis Area and Severity Index (PASI) 75, 65% achieved PASI 90, and 45% achieved PASI 100; 65% scored a 0 or 1 on the Physician’s Global Assessment Scale. In addition, 45% of patients reported a score of 0 on the Physician Global Assessment. Patients also reported a decrease in itching from baseline.
A total of 17% of patients experienced a serious adverse event and 87% of the patients experienced at least one treatment-related adverse event by the end of the 4-year extension period. Most of the reported events were mild to moderate; the most common were nasopharyngitis (23%), sinusitis (13%), upper respiratory tract infection (13%), and headache (10%).
The study findings were limited by several factors including the lack of blinding and lack of a placebo, the researchers noted.
However, the results demonstrate “that efficacy can be maintained at high levels for up to 4 years of ixekizumab therapy without apparent increases in health risks or safety issues,” for psoriasis patients, Dr. Zachariae and his associates said. “Longer treatment periods in larger numbers of patients will be reported for patients enrolled in the 5-year phase 3 ixekizumab studies.”
The study was supported by Eli Lilly. Dr. Zachariae disclosed relationships with Eli Lilly and other companies including Janssen, Novartis, AbbVie, and Amgen. His coauthors had financial relationships with multiple companies.
SOURCE: Zachariae C et al. J Am Acad Dermatol. 2018 Aug;79(2):294-301.e6.
FROM JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY
Key clinical point: Ixekizumab appears to be an effective option for long-term treatment of patients with moderate to severe psoriasis.
Major finding: A total of 82% of patients achieved PASI 75 at week 208 of the open-label extension study.
Study details: The data come from a 4-year open-label extension of a phase 2 randomized, placebo-controlled trial including 120 adults with psoriasis.
Disclosures: The study was supported by Eli Lilly. Dr. Zachariae disclosed relationships with Eli Lilly and other companies including Janssen, Novartis, AbbVie, and Amgen. His coauthors had financial relationships with multiple companies.
Source: Zachariae C et al. J Am Acad Dermatol. 2018 Aug;79(2):294-301.e6.
Most transgender teens not willing to delay hormone therapy to preserve fertility
TORONTO – The majority of transgender youth attending a pediatric gender clinic were not willing to delay starting hormone therapy in order to pursue fertility preservation, according to a survey study presented during a poster session at the Pediatric Academic Societies annual meeting.
Five percent of 66 young people and 33% of 52 parents surveyed during a visit to a hospital-based gender clinic agreed with the statement: “I would choose to delay hormone therapy to undergo fertility preservation (for my child) if asked today.”
Further, 70% of youth agreed that discomfort with a part of the body they don’t identify with was a factor that influenced their decision or thoughts about fertility preservation. Religious, financial, ethical, and demographic factors were not associated with willingness to delay treatment for fertility concerns.
“While hormone therapy has drastically improved the lives of countless transgender and gender nonconforming youth, its impact on fertility can unfairly force individuals to decide at a very early age whether or not they should preserve the ability to be a biological parent one day,” Rebecca Persky, MD, said in a press release. Dr. Persky, a former Children’s Hospital of Philadelphia (CHOP) resident, is now is a pediatric endocrinology fellow at the Eunice Kennedy Shriver National Institute of Child Health and Human Development.
A slightly greater proportion of youth (20%) and 12% of parents agreed it was important to have biological children or grandchildren. For those youth who did want to preserve the option of having biological children, that desire was associated with perceiving it as important to their parents (odds ratio, 6.07; P less than .05).
“We didn’t ask any questions about adoption of children or grandchildren, so that might have yielded different results if we had,” Dr. Persky acknowledged in an interview.
A lack of information about whether hormone therapy definitely prevents biologic fertility was associated with parents’ willingness to delay treatment for fertility preservation (OR: 24.57, P less than .05), yet 62% of parents said they felt their children were able to “make a meaningful decision about taking steps to preserve fertility at this point in (his/her/their) life.”
“I thought delaying treatment would be one of the biggest barriers, but even when we asked them if they wanted to preserve their fertility while not delaying or changing their hormone therapy, only the minority [33%] said they would be interested in that,” Dr. Persky said in an interview. “It kind of argues that a lot of these kids just don’t want to have biological children.”
She noted, however, that one limitation of the study was that many of the children surveyed already were receiving hormone therapy such that the questions engaged more on a theoretical level than a practical one.
“Not surprisingly, the strongest factor in the parents’ decisions was whether or not it was important to their child to have biological children,” said Dr. Persky.
The researchers surveyed 66 transgender and gender nonconforming youth who presented for care at the Gender and Sexuality Development Clinic at CHOP. After the findings were released, it was noted by several concerned parties on Twitter that because of the location of the study, the sample was a decidedly selected one.
The mean age was 17 years of patients and 63% of the sample were assigned female sex at birth. The mean age in the 52 parents surveyed was 48 years. The survey included 36 items on knowledge of fertility preservation, the desire to have biological children, and other factors that may affect the decision to pursue fertility preservation.
Gender-specific and age-specific analyses have not been completed, but are in the works, said Dr. Persky, who acknowledged that the area requires more qualitative research.
The authors reported no conflicts of interest.
TORONTO – The majority of transgender youth attending a pediatric gender clinic were not willing to delay starting hormone therapy in order to pursue fertility preservation, according to a survey study presented during a poster session at the Pediatric Academic Societies annual meeting.
Five percent of 66 young people and 33% of 52 parents surveyed during a visit to a hospital-based gender clinic agreed with the statement: “I would choose to delay hormone therapy to undergo fertility preservation (for my child) if asked today.”
Further, 70% of youth agreed that discomfort with a part of the body they don’t identify with was a factor that influenced their decision or thoughts about fertility preservation. Religious, financial, ethical, and demographic factors were not associated with willingness to delay treatment for fertility concerns.
“While hormone therapy has drastically improved the lives of countless transgender and gender nonconforming youth, its impact on fertility can unfairly force individuals to decide at a very early age whether or not they should preserve the ability to be a biological parent one day,” Rebecca Persky, MD, said in a press release. Dr. Persky, a former Children’s Hospital of Philadelphia (CHOP) resident, is now is a pediatric endocrinology fellow at the Eunice Kennedy Shriver National Institute of Child Health and Human Development.
A slightly greater proportion of youth (20%) and 12% of parents agreed it was important to have biological children or grandchildren. For those youth who did want to preserve the option of having biological children, that desire was associated with perceiving it as important to their parents (odds ratio, 6.07; P less than .05).
“We didn’t ask any questions about adoption of children or grandchildren, so that might have yielded different results if we had,” Dr. Persky acknowledged in an interview.
A lack of information about whether hormone therapy definitely prevents biologic fertility was associated with parents’ willingness to delay treatment for fertility preservation (OR: 24.57, P less than .05), yet 62% of parents said they felt their children were able to “make a meaningful decision about taking steps to preserve fertility at this point in (his/her/their) life.”
“I thought delaying treatment would be one of the biggest barriers, but even when we asked them if they wanted to preserve their fertility while not delaying or changing their hormone therapy, only the minority [33%] said they would be interested in that,” Dr. Persky said in an interview. “It kind of argues that a lot of these kids just don’t want to have biological children.”
She noted, however, that one limitation of the study was that many of the children surveyed already were receiving hormone therapy such that the questions engaged more on a theoretical level than a practical one.
“Not surprisingly, the strongest factor in the parents’ decisions was whether or not it was important to their child to have biological children,” said Dr. Persky.
The researchers surveyed 66 transgender and gender nonconforming youth who presented for care at the Gender and Sexuality Development Clinic at CHOP. After the findings were released, it was noted by several concerned parties on Twitter that because of the location of the study, the sample was a decidedly selected one.
The mean age was 17 years of patients and 63% of the sample were assigned female sex at birth. The mean age in the 52 parents surveyed was 48 years. The survey included 36 items on knowledge of fertility preservation, the desire to have biological children, and other factors that may affect the decision to pursue fertility preservation.
Gender-specific and age-specific analyses have not been completed, but are in the works, said Dr. Persky, who acknowledged that the area requires more qualitative research.
The authors reported no conflicts of interest.
TORONTO – The majority of transgender youth attending a pediatric gender clinic were not willing to delay starting hormone therapy in order to pursue fertility preservation, according to a survey study presented during a poster session at the Pediatric Academic Societies annual meeting.
Five percent of 66 young people and 33% of 52 parents surveyed during a visit to a hospital-based gender clinic agreed with the statement: “I would choose to delay hormone therapy to undergo fertility preservation (for my child) if asked today.”
Further, 70% of youth agreed that discomfort with a part of the body they don’t identify with was a factor that influenced their decision or thoughts about fertility preservation. Religious, financial, ethical, and demographic factors were not associated with willingness to delay treatment for fertility concerns.
“While hormone therapy has drastically improved the lives of countless transgender and gender nonconforming youth, its impact on fertility can unfairly force individuals to decide at a very early age whether or not they should preserve the ability to be a biological parent one day,” Rebecca Persky, MD, said in a press release. Dr. Persky, a former Children’s Hospital of Philadelphia (CHOP) resident, is now is a pediatric endocrinology fellow at the Eunice Kennedy Shriver National Institute of Child Health and Human Development.
A slightly greater proportion of youth (20%) and 12% of parents agreed it was important to have biological children or grandchildren. For those youth who did want to preserve the option of having biological children, that desire was associated with perceiving it as important to their parents (odds ratio, 6.07; P less than .05).
“We didn’t ask any questions about adoption of children or grandchildren, so that might have yielded different results if we had,” Dr. Persky acknowledged in an interview.
A lack of information about whether hormone therapy definitely prevents biologic fertility was associated with parents’ willingness to delay treatment for fertility preservation (OR: 24.57, P less than .05), yet 62% of parents said they felt their children were able to “make a meaningful decision about taking steps to preserve fertility at this point in (his/her/their) life.”
“I thought delaying treatment would be one of the biggest barriers, but even when we asked them if they wanted to preserve their fertility while not delaying or changing their hormone therapy, only the minority [33%] said they would be interested in that,” Dr. Persky said in an interview. “It kind of argues that a lot of these kids just don’t want to have biological children.”
She noted, however, that one limitation of the study was that many of the children surveyed already were receiving hormone therapy such that the questions engaged more on a theoretical level than a practical one.
“Not surprisingly, the strongest factor in the parents’ decisions was whether or not it was important to their child to have biological children,” said Dr. Persky.
The researchers surveyed 66 transgender and gender nonconforming youth who presented for care at the Gender and Sexuality Development Clinic at CHOP. After the findings were released, it was noted by several concerned parties on Twitter that because of the location of the study, the sample was a decidedly selected one.
The mean age was 17 years of patients and 63% of the sample were assigned female sex at birth. The mean age in the 52 parents surveyed was 48 years. The survey included 36 items on knowledge of fertility preservation, the desire to have biological children, and other factors that may affect the decision to pursue fertility preservation.
Gender-specific and age-specific analyses have not been completed, but are in the works, said Dr. Persky, who acknowledged that the area requires more qualitative research.
The authors reported no conflicts of interest.
REPORTING FROM PAS 18
Key clinical point:
Major finding: Five percent of youth and 33% of parents were willing to delay hormone therapy to undergo fertility preservation.
Study details: Survey study of 66 young people attending a pediatric gender and sexuality clinic and 52 parents of those individuals.
Disclosures: The authors reported no conflicts of interest.
Behavioral sleep interventions can work – if done
Sleep has begun to emerge from the shadows to gain its rightful place in the health pantheon. With this emergence has come a motley group of physicians and self-proclaimed experts (myself included) eager to share their anecdotal evidence and opinions on how new parents can shorten the inevitable and often painfully slow process of settling in.
A study in the May 2018 Journal of Pediatrics is an attempt to provide some data-driven guidance for sleep-deprived parents and their physician-advisors (“Real-World Implementation of Infant Behavioral Sleep Interventions: Results of a Parental Survey”). Using the responses from 652 parents who participated in a Facebook peer support group, the investigators created four categories of behavior sleep intervention for the parents of infants a mean age of 6 months: Modified extinction in which parents left the room and returned to briefly reassure the infant at intervals ranging from 5 to 25 minutes; unmodified extinction in which the parents let the infant cry it out; parental presence with support in which the parents remained in the room and interacted with the child; and parental presence without support. Extinction, both modified and unmodified (50% and 35%), was far and away the most frequently used method.
There was little difference in the effectiveness of the four strategies. By 2 weeks, 79% of the parents reported success, and the interventions were successfully discontinued in a mean and mode of 7 days. Not surprisingly, with success came reduced parental stress, as well as more consistent bedtime routines and smoother transitions to sleep.
When choosing a behavioral sleep intervention to suggest, I have tried to consider what I have learned about each family’s personality in the few months I have gotten to know them. But I have always been a big fan of extinction. And I suspect that my bias is simply a reflection of my no-nonsense, let’s-get-it-done-now personality. The results of this new study suggests that my poorly disguised pessimism about parental presence strategies may have discouraged some parents from trying an intervention that might have been successful for them.
The good news is that all four strategies can be successful. The problem is helping parents find the time and energy required to make any intervention successful. In the short term, extinction or parental presence is likely to mean less sleep for the parents. If work schedules and other family stressors already have drained a family’s energy reserves, it may not be the right time to try an intervention. When it becomes clear that there is never going to be right time, it is time for a frank discussion with the family about rearranging their priorities.
Behavioral sleep interventions require consistency and a family must be prepared to commit at least a week of uninterrupted evenings to get the job done. Business trips, visitors, and social engagements must take a back seat.
Extinction strategies are going to involve some crying, and while as pediatricians, we have developed the ability to buffer ourselves from crying (ignore really isn’t the right word), we must accept that listening to one’s child is too painful for some parents. When only one parent can’t tolerate the crying, the solution may be having that parent leave the home on the intervention evenings. When both parents share the vulnerability, the better answer is a parental presence strategy.
Although this study was small with a self-selected group of parents, the good news is that behavioral sleep intervention can work. We need to share the news with young families and encourage them to just do it.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “Is My Child Overtired?: The Sleep Solution for Raising Happier, Healthier Children.” Email him at [email protected].
Sleep has begun to emerge from the shadows to gain its rightful place in the health pantheon. With this emergence has come a motley group of physicians and self-proclaimed experts (myself included) eager to share their anecdotal evidence and opinions on how new parents can shorten the inevitable and often painfully slow process of settling in.
A study in the May 2018 Journal of Pediatrics is an attempt to provide some data-driven guidance for sleep-deprived parents and their physician-advisors (“Real-World Implementation of Infant Behavioral Sleep Interventions: Results of a Parental Survey”). Using the responses from 652 parents who participated in a Facebook peer support group, the investigators created four categories of behavior sleep intervention for the parents of infants a mean age of 6 months: Modified extinction in which parents left the room and returned to briefly reassure the infant at intervals ranging from 5 to 25 minutes; unmodified extinction in which the parents let the infant cry it out; parental presence with support in which the parents remained in the room and interacted with the child; and parental presence without support. Extinction, both modified and unmodified (50% and 35%), was far and away the most frequently used method.
There was little difference in the effectiveness of the four strategies. By 2 weeks, 79% of the parents reported success, and the interventions were successfully discontinued in a mean and mode of 7 days. Not surprisingly, with success came reduced parental stress, as well as more consistent bedtime routines and smoother transitions to sleep.
When choosing a behavioral sleep intervention to suggest, I have tried to consider what I have learned about each family’s personality in the few months I have gotten to know them. But I have always been a big fan of extinction. And I suspect that my bias is simply a reflection of my no-nonsense, let’s-get-it-done-now personality. The results of this new study suggests that my poorly disguised pessimism about parental presence strategies may have discouraged some parents from trying an intervention that might have been successful for them.
The good news is that all four strategies can be successful. The problem is helping parents find the time and energy required to make any intervention successful. In the short term, extinction or parental presence is likely to mean less sleep for the parents. If work schedules and other family stressors already have drained a family’s energy reserves, it may not be the right time to try an intervention. When it becomes clear that there is never going to be right time, it is time for a frank discussion with the family about rearranging their priorities.
Behavioral sleep interventions require consistency and a family must be prepared to commit at least a week of uninterrupted evenings to get the job done. Business trips, visitors, and social engagements must take a back seat.
Extinction strategies are going to involve some crying, and while as pediatricians, we have developed the ability to buffer ourselves from crying (ignore really isn’t the right word), we must accept that listening to one’s child is too painful for some parents. When only one parent can’t tolerate the crying, the solution may be having that parent leave the home on the intervention evenings. When both parents share the vulnerability, the better answer is a parental presence strategy.
Although this study was small with a self-selected group of parents, the good news is that behavioral sleep intervention can work. We need to share the news with young families and encourage them to just do it.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “Is My Child Overtired?: The Sleep Solution for Raising Happier, Healthier Children.” Email him at [email protected].
Sleep has begun to emerge from the shadows to gain its rightful place in the health pantheon. With this emergence has come a motley group of physicians and self-proclaimed experts (myself included) eager to share their anecdotal evidence and opinions on how new parents can shorten the inevitable and often painfully slow process of settling in.
A study in the May 2018 Journal of Pediatrics is an attempt to provide some data-driven guidance for sleep-deprived parents and their physician-advisors (“Real-World Implementation of Infant Behavioral Sleep Interventions: Results of a Parental Survey”). Using the responses from 652 parents who participated in a Facebook peer support group, the investigators created four categories of behavior sleep intervention for the parents of infants a mean age of 6 months: Modified extinction in which parents left the room and returned to briefly reassure the infant at intervals ranging from 5 to 25 minutes; unmodified extinction in which the parents let the infant cry it out; parental presence with support in which the parents remained in the room and interacted with the child; and parental presence without support. Extinction, both modified and unmodified (50% and 35%), was far and away the most frequently used method.
There was little difference in the effectiveness of the four strategies. By 2 weeks, 79% of the parents reported success, and the interventions were successfully discontinued in a mean and mode of 7 days. Not surprisingly, with success came reduced parental stress, as well as more consistent bedtime routines and smoother transitions to sleep.
When choosing a behavioral sleep intervention to suggest, I have tried to consider what I have learned about each family’s personality in the few months I have gotten to know them. But I have always been a big fan of extinction. And I suspect that my bias is simply a reflection of my no-nonsense, let’s-get-it-done-now personality. The results of this new study suggests that my poorly disguised pessimism about parental presence strategies may have discouraged some parents from trying an intervention that might have been successful for them.
The good news is that all four strategies can be successful. The problem is helping parents find the time and energy required to make any intervention successful. In the short term, extinction or parental presence is likely to mean less sleep for the parents. If work schedules and other family stressors already have drained a family’s energy reserves, it may not be the right time to try an intervention. When it becomes clear that there is never going to be right time, it is time for a frank discussion with the family about rearranging their priorities.
Behavioral sleep interventions require consistency and a family must be prepared to commit at least a week of uninterrupted evenings to get the job done. Business trips, visitors, and social engagements must take a back seat.
Extinction strategies are going to involve some crying, and while as pediatricians, we have developed the ability to buffer ourselves from crying (ignore really isn’t the right word), we must accept that listening to one’s child is too painful for some parents. When only one parent can’t tolerate the crying, the solution may be having that parent leave the home on the intervention evenings. When both parents share the vulnerability, the better answer is a parental presence strategy.
Although this study was small with a self-selected group of parents, the good news is that behavioral sleep intervention can work. We need to share the news with young families and encourage them to just do it.
Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “Is My Child Overtired?: The Sleep Solution for Raising Happier, Healthier Children.” Email him at [email protected].
Few women know that IUDs are effective emergency contraception
Although IUDs are effective as emergency contraception, few young women knew about this use of the device, according to a recent, multisite survey published in Contraception.
Only 7.5% of young female participants in a recent study had heard that the IUD could be used for emergency contraception (EC), according to a cluster-randomized trial of 1,500 women aged 18-25 years receiving contraception counseling at 40 U.S. Planned Parenthood centers. Race, ethnicity, and insurance status had no effect on the likelihood that a woman would know about IUDs as EC.
However, the study found, 68% of women surveyed would want to know about IUD use for EC, and those for whom a pregnancy would cause the most unhappiness were especially likely to want to know about this option (adjusted odds ratio, 1.26; 95% confidence interval, 1.01-1.58; P = .04). Again, demographic factors did not affect the likelihood that women would want to know about the IUD as an alternative to EC pills.
“In this 40-site U.S. study of sexually active young women, very few had heard of the IUD as a form of EC or knew of its significantly higher efficacy compared to EC pills, but most would desire to learn about it if they needed EC,” wrote Suzan Goodman, MD, MPH, and her coauthors.
Office visits where women request EC “provide a unique and time-sensitive opportunity to prevent an unwanted pregnancy, as well as to offer ongoing contraception,” wrote Dr. Goodman of the University of California, San Francisco, and her coauthors. But previous work showed that teens and young women are not generally well-informed about EC, including the window of effectiveness and methods that can be used for EC.
Dr. Goodman and her collaborators conducted a secondary analysis of a cluster-randomized trial that delivered a 4-hour training module to half of the Planned Parenthood centers; the evidence-based training focused on contraceptive counseling, long-acting reversible contraception, and IUD placement. Patients seen at the 40 centers were surveyed before receiving contraception counseling, and at 3 and 6 months post counseling.
Participant assignment to a center where the intervention had been administered to clinic staff had no bearing on how likely they were to have heard that IUDs could be used as EC.
Of the original survey respondents, who were young women not intending to become pregnant for at least 12 months, a total of 1,138 participants completed the relevant portions of the survey. The mean age of the respondents was 21.5 years, and were mostly (94%) never married. About one-quarter (26%) had private insurance, 31% had public insurance, and 38% were uninsured. About half were white, one-quarter were black, and 17% identified as Hispanic.
Study participants were very likely to view a doctor or nurse as their most trusted sources of information about EC, with 91% identifying this source over the Internet (6%) or friends (2%). Previous work, Dr. Goodman and her colleagues wrote, has found that women are more likely to consider an IUD if they had heard about it from a health care provider.
Although incorporating the ability to place an IUD at an EC visit may present some challenges in terms of clinic work flow and staff training, it’s an option that should be considered for many reasons, the authors noted.
IUDs are overall more effective than EC pills, with marked advantages in some instances: IUDs have higher efficacy in women with obesity, for women who have had multiple episodes of intercourse within the 5-day efficacy window, and for women at the fertile point of their menstrual cycles. Once inserted, they afford the additional advantage of ongoing protection against pregnancy.
“Contraceptive education should explicitly address IUD as EC,” wrote Dr. Goodman and her coauthors. “A coordinated effort to improve counseling, provider training, and same-day access to IUD as EC will be critical to offering women their full range of contraceptive options.”
This study was funded by the William and Flora Hewlett Foundation and the National Institutes of Health. Dr. Goodman reported no conflicts of interest. Two study coauthors are employees of Planned Parenthood, which participates in unrelated studies funded by HRA Pharma.
SOURCE: Goodman S et al. Contraception. 2018 Apr 18. doi: 10.1016/j.contraception.2018.04.009.
Although IUDs are effective as emergency contraception, few young women knew about this use of the device, according to a recent, multisite survey published in Contraception.
Only 7.5% of young female participants in a recent study had heard that the IUD could be used for emergency contraception (EC), according to a cluster-randomized trial of 1,500 women aged 18-25 years receiving contraception counseling at 40 U.S. Planned Parenthood centers. Race, ethnicity, and insurance status had no effect on the likelihood that a woman would know about IUDs as EC.
However, the study found, 68% of women surveyed would want to know about IUD use for EC, and those for whom a pregnancy would cause the most unhappiness were especially likely to want to know about this option (adjusted odds ratio, 1.26; 95% confidence interval, 1.01-1.58; P = .04). Again, demographic factors did not affect the likelihood that women would want to know about the IUD as an alternative to EC pills.
“In this 40-site U.S. study of sexually active young women, very few had heard of the IUD as a form of EC or knew of its significantly higher efficacy compared to EC pills, but most would desire to learn about it if they needed EC,” wrote Suzan Goodman, MD, MPH, and her coauthors.
Office visits where women request EC “provide a unique and time-sensitive opportunity to prevent an unwanted pregnancy, as well as to offer ongoing contraception,” wrote Dr. Goodman of the University of California, San Francisco, and her coauthors. But previous work showed that teens and young women are not generally well-informed about EC, including the window of effectiveness and methods that can be used for EC.
Dr. Goodman and her collaborators conducted a secondary analysis of a cluster-randomized trial that delivered a 4-hour training module to half of the Planned Parenthood centers; the evidence-based training focused on contraceptive counseling, long-acting reversible contraception, and IUD placement. Patients seen at the 40 centers were surveyed before receiving contraception counseling, and at 3 and 6 months post counseling.
Participant assignment to a center where the intervention had been administered to clinic staff had no bearing on how likely they were to have heard that IUDs could be used as EC.
Of the original survey respondents, who were young women not intending to become pregnant for at least 12 months, a total of 1,138 participants completed the relevant portions of the survey. The mean age of the respondents was 21.5 years, and were mostly (94%) never married. About one-quarter (26%) had private insurance, 31% had public insurance, and 38% were uninsured. About half were white, one-quarter were black, and 17% identified as Hispanic.
Study participants were very likely to view a doctor or nurse as their most trusted sources of information about EC, with 91% identifying this source over the Internet (6%) or friends (2%). Previous work, Dr. Goodman and her colleagues wrote, has found that women are more likely to consider an IUD if they had heard about it from a health care provider.
Although incorporating the ability to place an IUD at an EC visit may present some challenges in terms of clinic work flow and staff training, it’s an option that should be considered for many reasons, the authors noted.
IUDs are overall more effective than EC pills, with marked advantages in some instances: IUDs have higher efficacy in women with obesity, for women who have had multiple episodes of intercourse within the 5-day efficacy window, and for women at the fertile point of their menstrual cycles. Once inserted, they afford the additional advantage of ongoing protection against pregnancy.
“Contraceptive education should explicitly address IUD as EC,” wrote Dr. Goodman and her coauthors. “A coordinated effort to improve counseling, provider training, and same-day access to IUD as EC will be critical to offering women their full range of contraceptive options.”
This study was funded by the William and Flora Hewlett Foundation and the National Institutes of Health. Dr. Goodman reported no conflicts of interest. Two study coauthors are employees of Planned Parenthood, which participates in unrelated studies funded by HRA Pharma.
SOURCE: Goodman S et al. Contraception. 2018 Apr 18. doi: 10.1016/j.contraception.2018.04.009.
Although IUDs are effective as emergency contraception, few young women knew about this use of the device, according to a recent, multisite survey published in Contraception.
Only 7.5% of young female participants in a recent study had heard that the IUD could be used for emergency contraception (EC), according to a cluster-randomized trial of 1,500 women aged 18-25 years receiving contraception counseling at 40 U.S. Planned Parenthood centers. Race, ethnicity, and insurance status had no effect on the likelihood that a woman would know about IUDs as EC.
However, the study found, 68% of women surveyed would want to know about IUD use for EC, and those for whom a pregnancy would cause the most unhappiness were especially likely to want to know about this option (adjusted odds ratio, 1.26; 95% confidence interval, 1.01-1.58; P = .04). Again, demographic factors did not affect the likelihood that women would want to know about the IUD as an alternative to EC pills.
“In this 40-site U.S. study of sexually active young women, very few had heard of the IUD as a form of EC or knew of its significantly higher efficacy compared to EC pills, but most would desire to learn about it if they needed EC,” wrote Suzan Goodman, MD, MPH, and her coauthors.
Office visits where women request EC “provide a unique and time-sensitive opportunity to prevent an unwanted pregnancy, as well as to offer ongoing contraception,” wrote Dr. Goodman of the University of California, San Francisco, and her coauthors. But previous work showed that teens and young women are not generally well-informed about EC, including the window of effectiveness and methods that can be used for EC.
Dr. Goodman and her collaborators conducted a secondary analysis of a cluster-randomized trial that delivered a 4-hour training module to half of the Planned Parenthood centers; the evidence-based training focused on contraceptive counseling, long-acting reversible contraception, and IUD placement. Patients seen at the 40 centers were surveyed before receiving contraception counseling, and at 3 and 6 months post counseling.
Participant assignment to a center where the intervention had been administered to clinic staff had no bearing on how likely they were to have heard that IUDs could be used as EC.
Of the original survey respondents, who were young women not intending to become pregnant for at least 12 months, a total of 1,138 participants completed the relevant portions of the survey. The mean age of the respondents was 21.5 years, and were mostly (94%) never married. About one-quarter (26%) had private insurance, 31% had public insurance, and 38% were uninsured. About half were white, one-quarter were black, and 17% identified as Hispanic.
Study participants were very likely to view a doctor or nurse as their most trusted sources of information about EC, with 91% identifying this source over the Internet (6%) or friends (2%). Previous work, Dr. Goodman and her colleagues wrote, has found that women are more likely to consider an IUD if they had heard about it from a health care provider.
Although incorporating the ability to place an IUD at an EC visit may present some challenges in terms of clinic work flow and staff training, it’s an option that should be considered for many reasons, the authors noted.
IUDs are overall more effective than EC pills, with marked advantages in some instances: IUDs have higher efficacy in women with obesity, for women who have had multiple episodes of intercourse within the 5-day efficacy window, and for women at the fertile point of their menstrual cycles. Once inserted, they afford the additional advantage of ongoing protection against pregnancy.
“Contraceptive education should explicitly address IUD as EC,” wrote Dr. Goodman and her coauthors. “A coordinated effort to improve counseling, provider training, and same-day access to IUD as EC will be critical to offering women their full range of contraceptive options.”
This study was funded by the William and Flora Hewlett Foundation and the National Institutes of Health. Dr. Goodman reported no conflicts of interest. Two study coauthors are employees of Planned Parenthood, which participates in unrelated studies funded by HRA Pharma.
SOURCE: Goodman S et al. Contraception. 2018 Apr 18. doi: 10.1016/j.contraception.2018.04.009.
FROM CONTRACEPTION
Key clinical point: Few young women knew that an IUD is an effective emergency contraceptive.
Major finding: IUDs were identified as emergency contraception by 7.5% of respondents.
Study details: Secondary analysis of 1,138 responses from a 40-site, cluster-controlled trial of a contraceptive training intervention.
Disclosures: The study was sponsored by the William and Flora Hewlett Foundation and the National Institutes of Health. Two coauthors are employed by Planned Parenthood, which participates in unrelated research by HRA Pharma.
Source: Goodman S et al. Contraception. 2018 Apr 18. doi: 10.1016/j.contraception.2018.04.009.
HM18: ‘Things we do for no reason’
Be aware of low value care practices
Presenter
Leonard Feldman, MD, SFHM
Session summary
In the current climate of increasing national health care expenditures, the Journal of Hospital Medicine continues to expand its series “Things We Do for No Reason” to shed light on areas for improvement in the delivery of high-value care. Physicians, however, continue to practice low value care because of practice habits and lack of cost transparency. In keeping with the spirit of the journal’s series, three low-value practices were highlighted in this HM18 session, including the benefits of high-flow nasal cannula (HFNC) oxygen therapy, the value of serum albumin and prealbumin in malnutrition, and the use of nebulized versus inhaler albuterol.
HFNC oxygen therapy has become a widespread practice internationally, with large variations in implementation across all age ranges. In bronchiolitic infants, variations in time of implementation, duration of therapy, weaning policies, and outcome measures have yielded mixed results to date. Many studies are still underway, but current literature has not shown significant benefits of starting HFNC early in the illness process, and infants receiving HFNC have not had better outcomes than those who received standard therapy (low-flow nasal cannula).
Serum albumin and prealbumin are often cited as markers of malnutrition. In patients on tube feeds, such as those with cerebral palsy and complex medical conditions, current literature shows no benefit in the use of these lab markers for screening or for following to assess improvement. In addition, patients with eating disorders do not have significant correlation of their nutritive status with these markers. Therefore, the use of these labs for screening of malnutrition is of little if any value.
Finally, when comparing albuterol delivery systems, there has been no proof that nebulized albuterol is any better than metered dose inhalers (MDI) in its effect. As long as appropriate dosing of MDIs is done, the benefits are the same. In addition, side effects are fewer, and the length of stay in the ED is shorter. The more a family uses MDI inhalers, the better their technique. Studies have shown it takes approximately three attempts at using an MDI to get the technique correct, so inpatient MDI use would also decrease user error in the outpatient setting.
Key takeaways for HM
- Practice habits, lack of transparency of costs, and regional training lead to low value care practices.
- Early introduction of high-flow nasal cannula in hospitalization of an infant with bronchiolitis has not been shown to decrease length of stay or severity outcomes, according to currently available data.
- Serum albumin and prealbumin have little to no benefit in screening of malnutrition.
- Nebulized albuterol has not been proven to be more beneficial that MDI albuterol at appropriate doses.
- Repeat MDI administration has been shown to positively affect user administration techniques.
- MDI albuterol use has fewer side effects and decreased ED length of stay, compared with nebulized albuterol.
Dr. Schwenk is an associate professor of pediatrics at the University of Louisville (Ky.) and a pediatric hospitalist at Norton Children’s Hospital, Louisville.
Be aware of low value care practices
Be aware of low value care practices
Presenter
Leonard Feldman, MD, SFHM
Session summary
In the current climate of increasing national health care expenditures, the Journal of Hospital Medicine continues to expand its series “Things We Do for No Reason” to shed light on areas for improvement in the delivery of high-value care. Physicians, however, continue to practice low value care because of practice habits and lack of cost transparency. In keeping with the spirit of the journal’s series, three low-value practices were highlighted in this HM18 session, including the benefits of high-flow nasal cannula (HFNC) oxygen therapy, the value of serum albumin and prealbumin in malnutrition, and the use of nebulized versus inhaler albuterol.
HFNC oxygen therapy has become a widespread practice internationally, with large variations in implementation across all age ranges. In bronchiolitic infants, variations in time of implementation, duration of therapy, weaning policies, and outcome measures have yielded mixed results to date. Many studies are still underway, but current literature has not shown significant benefits of starting HFNC early in the illness process, and infants receiving HFNC have not had better outcomes than those who received standard therapy (low-flow nasal cannula).
Serum albumin and prealbumin are often cited as markers of malnutrition. In patients on tube feeds, such as those with cerebral palsy and complex medical conditions, current literature shows no benefit in the use of these lab markers for screening or for following to assess improvement. In addition, patients with eating disorders do not have significant correlation of their nutritive status with these markers. Therefore, the use of these labs for screening of malnutrition is of little if any value.
Finally, when comparing albuterol delivery systems, there has been no proof that nebulized albuterol is any better than metered dose inhalers (MDI) in its effect. As long as appropriate dosing of MDIs is done, the benefits are the same. In addition, side effects are fewer, and the length of stay in the ED is shorter. The more a family uses MDI inhalers, the better their technique. Studies have shown it takes approximately three attempts at using an MDI to get the technique correct, so inpatient MDI use would also decrease user error in the outpatient setting.
Key takeaways for HM
- Practice habits, lack of transparency of costs, and regional training lead to low value care practices.
- Early introduction of high-flow nasal cannula in hospitalization of an infant with bronchiolitis has not been shown to decrease length of stay or severity outcomes, according to currently available data.
- Serum albumin and prealbumin have little to no benefit in screening of malnutrition.
- Nebulized albuterol has not been proven to be more beneficial that MDI albuterol at appropriate doses.
- Repeat MDI administration has been shown to positively affect user administration techniques.
- MDI albuterol use has fewer side effects and decreased ED length of stay, compared with nebulized albuterol.
Dr. Schwenk is an associate professor of pediatrics at the University of Louisville (Ky.) and a pediatric hospitalist at Norton Children’s Hospital, Louisville.
Presenter
Leonard Feldman, MD, SFHM
Session summary
In the current climate of increasing national health care expenditures, the Journal of Hospital Medicine continues to expand its series “Things We Do for No Reason” to shed light on areas for improvement in the delivery of high-value care. Physicians, however, continue to practice low value care because of practice habits and lack of cost transparency. In keeping with the spirit of the journal’s series, three low-value practices were highlighted in this HM18 session, including the benefits of high-flow nasal cannula (HFNC) oxygen therapy, the value of serum albumin and prealbumin in malnutrition, and the use of nebulized versus inhaler albuterol.
HFNC oxygen therapy has become a widespread practice internationally, with large variations in implementation across all age ranges. In bronchiolitic infants, variations in time of implementation, duration of therapy, weaning policies, and outcome measures have yielded mixed results to date. Many studies are still underway, but current literature has not shown significant benefits of starting HFNC early in the illness process, and infants receiving HFNC have not had better outcomes than those who received standard therapy (low-flow nasal cannula).
Serum albumin and prealbumin are often cited as markers of malnutrition. In patients on tube feeds, such as those with cerebral palsy and complex medical conditions, current literature shows no benefit in the use of these lab markers for screening or for following to assess improvement. In addition, patients with eating disorders do not have significant correlation of their nutritive status with these markers. Therefore, the use of these labs for screening of malnutrition is of little if any value.
Finally, when comparing albuterol delivery systems, there has been no proof that nebulized albuterol is any better than metered dose inhalers (MDI) in its effect. As long as appropriate dosing of MDIs is done, the benefits are the same. In addition, side effects are fewer, and the length of stay in the ED is shorter. The more a family uses MDI inhalers, the better their technique. Studies have shown it takes approximately three attempts at using an MDI to get the technique correct, so inpatient MDI use would also decrease user error in the outpatient setting.
Key takeaways for HM
- Practice habits, lack of transparency of costs, and regional training lead to low value care practices.
- Early introduction of high-flow nasal cannula in hospitalization of an infant with bronchiolitis has not been shown to decrease length of stay or severity outcomes, according to currently available data.
- Serum albumin and prealbumin have little to no benefit in screening of malnutrition.
- Nebulized albuterol has not been proven to be more beneficial that MDI albuterol at appropriate doses.
- Repeat MDI administration has been shown to positively affect user administration techniques.
- MDI albuterol use has fewer side effects and decreased ED length of stay, compared with nebulized albuterol.
Dr. Schwenk is an associate professor of pediatrics at the University of Louisville (Ky.) and a pediatric hospitalist at Norton Children’s Hospital, Louisville.