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.

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-fitz/Stockphoto

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-fitz/Stockphoto

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-fitz/Stockphoto

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].

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.

[email protected]

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.

[email protected]

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.

[email protected]

SOURCE: Goodman S et al. Contraception. 2018 Apr 18. doi: 10.1016/j.contraception.2018.04.009.

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.

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.

STOCKHOLM –Adding the monoclonal antibody elotuzumab to pomalidomide and dexamethasone nearly doubled the overall response rate and median progression-free survival in patients with relapsed/refractory multiple myeloma compared with pomalidomide and dexamethasone alone, results of the phase 2 ELOQUENT-3 trial showed.

Neil Osterweil/MDedge News

After a minimum follow-up of 9.1 months, median progression-free survival (PFS) for 60 patients assigned to receive elotuzumab (Empliciti), pomalidomide (Pomalyst), and dexamethasone (EPd) was 10.3 months, compared with 4.7 months for 60 patients assigned to pomalidomide and dexamethasone (Pd). This difference translated into a hazard ratio (HR) of 0.54 (P = .0078) favoring EPd, reported Meletios A. Dimopoulos, MD, of the National and Kapodistrian University of Athens (Greece).

“The study met its primary endpoint, which was specifically designed to detect a large treatment effect in a relatively small sample of patients. Elotuzumab with pomalidomide and dexamethasone showed a significant and clinically meaningful 46% reduction in the risk of progression or death,” he said at the annual congress of the European Hematology Association.

Elotuzumab is an immunoglobulin G (IgG) monoclonal antibody that targets signaling lymphocytic activation molecule F7 (SLAMF7) expressed on multiple myeloma cells. Pomalidomide, an immunomodulator, may act synergistically with elotuzumab through several different mechanisms to increase killing of multiple myeloma cells, Dr. Dimopoulos said.

In ELOQUENT-3, patients with relapsed or refractory multiple myeloma after 2 or more prior lines of therapy, including lenalidomide (Revlimid) and a proteasome inhibitor and no prior pomalidomide were enrolled and randomly assigned to receive either pomalidomide 4 mg orally on days 1-21 of each 28-day cycle plus oral dexamethasone 40 mg equivalent weekly, or to the same regimen plus intravenous elotuzumab 10 mg/kg weekly for cycles 1 and 2, and 20 mg/kg every 4 weeks for cycle 3 and subsequent cycles.

The trial met its primary endpoint of investigator-assessed PFS, with a 46% reduction in the risk of progression or death with EPd compared with Pd.

An analysis of PFS by subgroups showed that EPd was significantly superior to Pd for patients younger than 65 years, those with International Staging System stage I-II at study entry, patients with lactate dehydrogenase levels below 300 IU/L at baseline, patients who had two or three prior lines of therapy vs. four or more, and those who had disease that was refractory to both lenalidomide and a proteasome inhibitor.

EPd was also associated with a trend toward better PFS in an analysis combining patients with high-risk cytogenetics (deletion 17p or translocation 14;16) or high LDH levels, with a median of 7.7 months compared with 3.6 months for Pd. However, the HR, 0.55, was not statistically significant, likely because of the small sample size.

Similarly, the elotuzumab-containing combination showed a nonsignificant trend toward better PFS among patients without high risk disease, with a median PFS not reached, vs. not reached, vs. 4.7 months for patients treated with Pd.

The overall response rate with EPd was 53%, compared with 26% for Pd (odds ratio 3.5, P = .0029). The responses in the elotuzumab arm consisted of 8% complete response, 12% very good partial responses, and 33% partial responses. The respective rates in the Pd group were 2%, 7%, and 18%.

The median duration of response with EPd was not reached at the time of the database lock, compared with 8.3 months with Pd.

A preliminary analysis of overall survival showed a trend favoring EPd (13 deaths out to 22 months of follow-up, compared with 18 deaths out to 20 months in the Pd arm; HR 0.62, nonsignificant).

There were five treatment-related deaths in the EPd arm, and eight in the Pd arm. Grade 1 or 2 infusion reactions occurred in three patients in the EPd arm.

Other adverse events were comparable between the arms, with 57% of patients in the EPd arm and 60% in the Pd arm having at least one grade 3 or 4 adverse event.

“The hematologic toxicity was driven by pomalidomide and low-dose dexamethasone. For unclear reasons, there was less grade 3 or 4 neutropenia with the addition of elotuzumab to pomalidomide/dexamethasone, and also the infection rate was lower in the EPd arm,” Dr. Dimopoulos said.
 

SOURCE: Dimopoulos MA et al. EHA Congress, Abstract LB2606.

STOCKHOLM –Adding the monoclonal antibody elotuzumab to pomalidomide and dexamethasone nearly doubled the overall response rate and median progression-free survival in patients with relapsed/refractory multiple myeloma compared with pomalidomide and dexamethasone alone, results of the phase 2 ELOQUENT-3 trial showed.

Neil Osterweil/MDedge News

After a minimum follow-up of 9.1 months, median progression-free survival (PFS) for 60 patients assigned to receive elotuzumab (Empliciti), pomalidomide (Pomalyst), and dexamethasone (EPd) was 10.3 months, compared with 4.7 months for 60 patients assigned to pomalidomide and dexamethasone (Pd). This difference translated into a hazard ratio (HR) of 0.54 (P = .0078) favoring EPd, reported Meletios A. Dimopoulos, MD, of the National and Kapodistrian University of Athens (Greece).

“The study met its primary endpoint, which was specifically designed to detect a large treatment effect in a relatively small sample of patients. Elotuzumab with pomalidomide and dexamethasone showed a significant and clinically meaningful 46% reduction in the risk of progression or death,” he said at the annual congress of the European Hematology Association.

Elotuzumab is an immunoglobulin G (IgG) monoclonal antibody that targets signaling lymphocytic activation molecule F7 (SLAMF7) expressed on multiple myeloma cells. Pomalidomide, an immunomodulator, may act synergistically with elotuzumab through several different mechanisms to increase killing of multiple myeloma cells, Dr. Dimopoulos said.

In ELOQUENT-3, patients with relapsed or refractory multiple myeloma after 2 or more prior lines of therapy, including lenalidomide (Revlimid) and a proteasome inhibitor and no prior pomalidomide were enrolled and randomly assigned to receive either pomalidomide 4 mg orally on days 1-21 of each 28-day cycle plus oral dexamethasone 40 mg equivalent weekly, or to the same regimen plus intravenous elotuzumab 10 mg/kg weekly for cycles 1 and 2, and 20 mg/kg every 4 weeks for cycle 3 and subsequent cycles.

The trial met its primary endpoint of investigator-assessed PFS, with a 46% reduction in the risk of progression or death with EPd compared with Pd.

An analysis of PFS by subgroups showed that EPd was significantly superior to Pd for patients younger than 65 years, those with International Staging System stage I-II at study entry, patients with lactate dehydrogenase levels below 300 IU/L at baseline, patients who had two or three prior lines of therapy vs. four or more, and those who had disease that was refractory to both lenalidomide and a proteasome inhibitor.

EPd was also associated with a trend toward better PFS in an analysis combining patients with high-risk cytogenetics (deletion 17p or translocation 14;16) or high LDH levels, with a median of 7.7 months compared with 3.6 months for Pd. However, the HR, 0.55, was not statistically significant, likely because of the small sample size.

Similarly, the elotuzumab-containing combination showed a nonsignificant trend toward better PFS among patients without high risk disease, with a median PFS not reached, vs. not reached, vs. 4.7 months for patients treated with Pd.

The overall response rate with EPd was 53%, compared with 26% for Pd (odds ratio 3.5, P = .0029). The responses in the elotuzumab arm consisted of 8% complete response, 12% very good partial responses, and 33% partial responses. The respective rates in the Pd group were 2%, 7%, and 18%.

The median duration of response with EPd was not reached at the time of the database lock, compared with 8.3 months with Pd.

A preliminary analysis of overall survival showed a trend favoring EPd (13 deaths out to 22 months of follow-up, compared with 18 deaths out to 20 months in the Pd arm; HR 0.62, nonsignificant).

There were five treatment-related deaths in the EPd arm, and eight in the Pd arm. Grade 1 or 2 infusion reactions occurred in three patients in the EPd arm.

Other adverse events were comparable between the arms, with 57% of patients in the EPd arm and 60% in the Pd arm having at least one grade 3 or 4 adverse event.

“The hematologic toxicity was driven by pomalidomide and low-dose dexamethasone. For unclear reasons, there was less grade 3 or 4 neutropenia with the addition of elotuzumab to pomalidomide/dexamethasone, and also the infection rate was lower in the EPd arm,” Dr. Dimopoulos said.
 

SOURCE: Dimopoulos MA et al. EHA Congress, Abstract LB2606.

STOCKHOLM –Adding the monoclonal antibody elotuzumab to pomalidomide and dexamethasone nearly doubled the overall response rate and median progression-free survival in patients with relapsed/refractory multiple myeloma compared with pomalidomide and dexamethasone alone, results of the phase 2 ELOQUENT-3 trial showed.

Neil Osterweil/MDedge News

After a minimum follow-up of 9.1 months, median progression-free survival (PFS) for 60 patients assigned to receive elotuzumab (Empliciti), pomalidomide (Pomalyst), and dexamethasone (EPd) was 10.3 months, compared with 4.7 months for 60 patients assigned to pomalidomide and dexamethasone (Pd). This difference translated into a hazard ratio (HR) of 0.54 (P = .0078) favoring EPd, reported Meletios A. Dimopoulos, MD, of the National and Kapodistrian University of Athens (Greece).

“The study met its primary endpoint, which was specifically designed to detect a large treatment effect in a relatively small sample of patients. Elotuzumab with pomalidomide and dexamethasone showed a significant and clinically meaningful 46% reduction in the risk of progression or death,” he said at the annual congress of the European Hematology Association.

Elotuzumab is an immunoglobulin G (IgG) monoclonal antibody that targets signaling lymphocytic activation molecule F7 (SLAMF7) expressed on multiple myeloma cells. Pomalidomide, an immunomodulator, may act synergistically with elotuzumab through several different mechanisms to increase killing of multiple myeloma cells, Dr. Dimopoulos said.

In ELOQUENT-3, patients with relapsed or refractory multiple myeloma after 2 or more prior lines of therapy, including lenalidomide (Revlimid) and a proteasome inhibitor and no prior pomalidomide were enrolled and randomly assigned to receive either pomalidomide 4 mg orally on days 1-21 of each 28-day cycle plus oral dexamethasone 40 mg equivalent weekly, or to the same regimen plus intravenous elotuzumab 10 mg/kg weekly for cycles 1 and 2, and 20 mg/kg every 4 weeks for cycle 3 and subsequent cycles.

The trial met its primary endpoint of investigator-assessed PFS, with a 46% reduction in the risk of progression or death with EPd compared with Pd.

An analysis of PFS by subgroups showed that EPd was significantly superior to Pd for patients younger than 65 years, those with International Staging System stage I-II at study entry, patients with lactate dehydrogenase levels below 300 IU/L at baseline, patients who had two or three prior lines of therapy vs. four or more, and those who had disease that was refractory to both lenalidomide and a proteasome inhibitor.

EPd was also associated with a trend toward better PFS in an analysis combining patients with high-risk cytogenetics (deletion 17p or translocation 14;16) or high LDH levels, with a median of 7.7 months compared with 3.6 months for Pd. However, the HR, 0.55, was not statistically significant, likely because of the small sample size.

Similarly, the elotuzumab-containing combination showed a nonsignificant trend toward better PFS among patients without high risk disease, with a median PFS not reached, vs. not reached, vs. 4.7 months for patients treated with Pd.

The overall response rate with EPd was 53%, compared with 26% for Pd (odds ratio 3.5, P = .0029). The responses in the elotuzumab arm consisted of 8% complete response, 12% very good partial responses, and 33% partial responses. The respective rates in the Pd group were 2%, 7%, and 18%.

The median duration of response with EPd was not reached at the time of the database lock, compared with 8.3 months with Pd.

A preliminary analysis of overall survival showed a trend favoring EPd (13 deaths out to 22 months of follow-up, compared with 18 deaths out to 20 months in the Pd arm; HR 0.62, nonsignificant).

There were five treatment-related deaths in the EPd arm, and eight in the Pd arm. Grade 1 or 2 infusion reactions occurred in three patients in the EPd arm.

Other adverse events were comparable between the arms, with 57% of patients in the EPd arm and 60% in the Pd arm having at least one grade 3 or 4 adverse event.

“The hematologic toxicity was driven by pomalidomide and low-dose dexamethasone. For unclear reasons, there was less grade 3 or 4 neutropenia with the addition of elotuzumab to pomalidomide/dexamethasone, and also the infection rate was lower in the EPd arm,” Dr. Dimopoulos said.
 

SOURCE: Dimopoulos MA et al. EHA Congress, Abstract LB2606.

Transcatheter aortic valve replacement has entered a new stage of development, and so needed a tweaked set of standards for how existing programs operate and what new program need to open, said a panel of experts formed by the four U.S. societies with the closest links to this procedure.

U.S. transcatheter aortic valve replacement (TAVR) programs have “matured as a therapeutic option” since its commercial U.S. introduction in 2012, said a revised statement of operator and institutional recommendations and requirements issued on July 18 by the American Association for Thoracic Surgery, the American College of Cardiology, the Society for Cardiovascular Angiography and Interventions, and the Society of Thoracic Surgeons. A writing panel formed by these four groups prepared the revision, published online in the Journal of the American College of Cardiology on July 18, to replace the first set of recommendations for running U.S. TAVR programs that came out in 2012 (J Am Coll Cardiol. 2012 May 29;59[22]:2028-42).

“The main thrust is to ensure and allow for the metrics of quality TAVR,” said Joseph E. Bavaria, MD, cochair of the writing panel and professor of surgery and codirector of the transcatheter valve program at the University of Pennsylvania in Philadelphia. “We’re trying to force continuous quality improvement across U.S. TAVR teams,” Dr. Bavaria explained in an interview.

The key to this change will be the data collected on every U.S. TAVR patient in the Transcatheter Valve Therapy registry maintained by the American College of Cardiology and the Society of Thoracic Surgeons, which now has data on more than 120,000 patients who have undergone TAVR at what are now 582 active U.S. TAVR programs, noted Carl L. Tommaso, MD, an interventional cardiologist with NorthShore Medical Group in Bannockburn, Ill., and cochair of the writing panel. “You need to do risk adjustment to measure quality of care,” and the robust database that now exists has begun to make this possible, said Dr. Tommaso, who neither performs TAVR procedures nor participates on a TAVR team. Statistical analyses based on this substantial and always-expanding database of TAVR patients now allows for risk-adjusted assessment of in hospital and 30-day mortality, and risk-adjusted evaluation of 1-year mortality and quality-of-life outcomes are expected within the next couple of years.

“We’re still not yet at the point of having good, risk-adjusted models” for all these measures, but our hope is that in the next 2-7 years we can move completely to quality measures, as has already been done for percutaneous coronary interventions” and away from procedure volume, which currently serves as a surrogate marker for a TAVR program’s competence.

The new document continues to call for TAVR programs to average at least 50 TAVR procedures a year or at least 100 every 2 years, but primarily to insure that each TAVR program can generate enough data about its performance to produce statistically reliable numbers.

“The volume floors are only there because you can’t measure quality without volume,” said Dr. Bavaria. “It’s impossible to measure quality without a certain procedure volume.”

Dr. Bavaria stressed, however, that the goal of the new document is not to limit TAVR programs based on their procedure volume, especially because another goal of the document is to ensure reasonable geographic access to TAVR for U.S. patients. “This document does not advocate for any program to shut down,” he declared. On top of that, “we have no problem with new programs,” although the document noted that “the major threat to low volume sites growing and achieving higher levels of experience is the opening of additional sites in the same geographic region.”

In 2017, 204 of the 525 sites (39%) performing TAVR at that time were performing fewer than 50 procedures annually, the document said, but added that many TAVR centers now operate in what are predominantly rural regions “and it is important that they remain active if they can document acceptable quality even if they should fall below volume thresholds to maintain patient access to care.”

Dr. Tommaso said that perhaps a TAVR center in Alaska, for example, might not meet the 50 cases/year standard, “but I don’t think anyone would worry if the volume was low because we’re serving patients in Alaska.” Currently, 84% of TAVR centers that have been operating for more than 2 years meet the 50 procedures/year threshold, he added. And TAVR centers now operate in 49 of the 50 states, with only Wyoming lacking a TAVR facility within its borders. Despite this, use of TAVR among Wyoming residents is comparable to the rate in Illinois, Dr. Bavaria said.

Both cochairs also highlighted that, with TAVR now approved for patients at moderate risk for aortic-valve surgery, the number of patients who are TAVR candidates has grown, and it’s possible that pending trial results will soon broaden TAVR’s availability to low-risk patients, a step that would greatly further expand the potential patient pool for the procedure.

The revised recommendations and requirements will make it “a little easier to start a new program, except now, for the first time, you need to start with an operator who is already experienced with TAVR,” noted Dr. Bavaria. “The TAVR technology is now mature enough that it’s inappropriate to have learning-curve mortality.” But aside from this the new standards lower the bar a bit for a center’s volume of percutaneous coronary interventions and surgical aortic valve replacements. The revision also maintains that an examination by and consultation with a single cardiac surgeon by a prospective TAVR patient is adequate, similar to the 2012 document, although the Center for Medicare & Medicaid Services mandated in its coverage decision that prospective TAVR patients consult with two cardiac surgeons, the so-called “two-surgeon rule.” If CMS eliminated the two-surgeon rule it would “streamline” the process that patients go through when being assessed for TAVR, Dr. Tommaso observed, and both he and Dr. Bavaria expressed hope that the new document might prompt CMS to reconsider this guidance.

“We felt that two surgeons weren’t needed,” but the document specifies that both the surgeon and the cardiologist whom a prospective patient consults before finalizing plans for the intervention should both be members of the multidisciplinary team that performs the procedure. Until now, these clinicians weren’t specified as necessarily members of the TAVR team, Dr. Bavaria said.

One additional new element in the revised document is specification of shared decision making as the mechanism patients should go through when considering TAVR relative to their other management options, Dr. Tommaso said.Dr. Bavaria and Dr. Tommaso had no disclosures.

[email protected]
 

SOURCE: Bavaria JE et al. JACC. 2018 Jul18. doi:10.1016/j.jacc.2018.07.002.
 

Transcatheter aortic valve replacement has entered a new stage of development, and so needed a tweaked set of standards for how existing programs operate and what new program need to open, said a panel of experts formed by the four U.S. societies with the closest links to this procedure.

U.S. transcatheter aortic valve replacement (TAVR) programs have “matured as a therapeutic option” since its commercial U.S. introduction in 2012, said a revised statement of operator and institutional recommendations and requirements issued on July 18 by the American Association for Thoracic Surgery, the American College of Cardiology, the Society for Cardiovascular Angiography and Interventions, and the Society of Thoracic Surgeons. A writing panel formed by these four groups prepared the revision, published online in the Journal of the American College of Cardiology on July 18, to replace the first set of recommendations for running U.S. TAVR programs that came out in 2012 (J Am Coll Cardiol. 2012 May 29;59[22]:2028-42).

“The main thrust is to ensure and allow for the metrics of quality TAVR,” said Joseph E. Bavaria, MD, cochair of the writing panel and professor of surgery and codirector of the transcatheter valve program at the University of Pennsylvania in Philadelphia. “We’re trying to force continuous quality improvement across U.S. TAVR teams,” Dr. Bavaria explained in an interview.

The key to this change will be the data collected on every U.S. TAVR patient in the Transcatheter Valve Therapy registry maintained by the American College of Cardiology and the Society of Thoracic Surgeons, which now has data on more than 120,000 patients who have undergone TAVR at what are now 582 active U.S. TAVR programs, noted Carl L. Tommaso, MD, an interventional cardiologist with NorthShore Medical Group in Bannockburn, Ill., and cochair of the writing panel. “You need to do risk adjustment to measure quality of care,” and the robust database that now exists has begun to make this possible, said Dr. Tommaso, who neither performs TAVR procedures nor participates on a TAVR team. Statistical analyses based on this substantial and always-expanding database of TAVR patients now allows for risk-adjusted assessment of in hospital and 30-day mortality, and risk-adjusted evaluation of 1-year mortality and quality-of-life outcomes are expected within the next couple of years.

“We’re still not yet at the point of having good, risk-adjusted models” for all these measures, but our hope is that in the next 2-7 years we can move completely to quality measures, as has already been done for percutaneous coronary interventions” and away from procedure volume, which currently serves as a surrogate marker for a TAVR program’s competence.

The new document continues to call for TAVR programs to average at least 50 TAVR procedures a year or at least 100 every 2 years, but primarily to insure that each TAVR program can generate enough data about its performance to produce statistically reliable numbers.

“The volume floors are only there because you can’t measure quality without volume,” said Dr. Bavaria. “It’s impossible to measure quality without a certain procedure volume.”

Dr. Bavaria stressed, however, that the goal of the new document is not to limit TAVR programs based on their procedure volume, especially because another goal of the document is to ensure reasonable geographic access to TAVR for U.S. patients. “This document does not advocate for any program to shut down,” he declared. On top of that, “we have no problem with new programs,” although the document noted that “the major threat to low volume sites growing and achieving higher levels of experience is the opening of additional sites in the same geographic region.”

In 2017, 204 of the 525 sites (39%) performing TAVR at that time were performing fewer than 50 procedures annually, the document said, but added that many TAVR centers now operate in what are predominantly rural regions “and it is important that they remain active if they can document acceptable quality even if they should fall below volume thresholds to maintain patient access to care.”

Dr. Tommaso said that perhaps a TAVR center in Alaska, for example, might not meet the 50 cases/year standard, “but I don’t think anyone would worry if the volume was low because we’re serving patients in Alaska.” Currently, 84% of TAVR centers that have been operating for more than 2 years meet the 50 procedures/year threshold, he added. And TAVR centers now operate in 49 of the 50 states, with only Wyoming lacking a TAVR facility within its borders. Despite this, use of TAVR among Wyoming residents is comparable to the rate in Illinois, Dr. Bavaria said.

Both cochairs also highlighted that, with TAVR now approved for patients at moderate risk for aortic-valve surgery, the number of patients who are TAVR candidates has grown, and it’s possible that pending trial results will soon broaden TAVR’s availability to low-risk patients, a step that would greatly further expand the potential patient pool for the procedure.

The revised recommendations and requirements will make it “a little easier to start a new program, except now, for the first time, you need to start with an operator who is already experienced with TAVR,” noted Dr. Bavaria. “The TAVR technology is now mature enough that it’s inappropriate to have learning-curve mortality.” But aside from this the new standards lower the bar a bit for a center’s volume of percutaneous coronary interventions and surgical aortic valve replacements. The revision also maintains that an examination by and consultation with a single cardiac surgeon by a prospective TAVR patient is adequate, similar to the 2012 document, although the Center for Medicare & Medicaid Services mandated in its coverage decision that prospective TAVR patients consult with two cardiac surgeons, the so-called “two-surgeon rule.” If CMS eliminated the two-surgeon rule it would “streamline” the process that patients go through when being assessed for TAVR, Dr. Tommaso observed, and both he and Dr. Bavaria expressed hope that the new document might prompt CMS to reconsider this guidance.

“We felt that two surgeons weren’t needed,” but the document specifies that both the surgeon and the cardiologist whom a prospective patient consults before finalizing plans for the intervention should both be members of the multidisciplinary team that performs the procedure. Until now, these clinicians weren’t specified as necessarily members of the TAVR team, Dr. Bavaria said.

One additional new element in the revised document is specification of shared decision making as the mechanism patients should go through when considering TAVR relative to their other management options, Dr. Tommaso said.Dr. Bavaria and Dr. Tommaso had no disclosures.

[email protected]
 

SOURCE: Bavaria JE et al. JACC. 2018 Jul18. doi:10.1016/j.jacc.2018.07.002.
 

Transcatheter aortic valve replacement has entered a new stage of development, and so needed a tweaked set of standards for how existing programs operate and what new program need to open, said a panel of experts formed by the four U.S. societies with the closest links to this procedure.

U.S. transcatheter aortic valve replacement (TAVR) programs have “matured as a therapeutic option” since its commercial U.S. introduction in 2012, said a revised statement of operator and institutional recommendations and requirements issued on July 18 by the American Association for Thoracic Surgery, the American College of Cardiology, the Society for Cardiovascular Angiography and Interventions, and the Society of Thoracic Surgeons. A writing panel formed by these four groups prepared the revision, published online in the Journal of the American College of Cardiology on July 18, to replace the first set of recommendations for running U.S. TAVR programs that came out in 2012 (J Am Coll Cardiol. 2012 May 29;59[22]:2028-42).

“The main thrust is to ensure and allow for the metrics of quality TAVR,” said Joseph E. Bavaria, MD, cochair of the writing panel and professor of surgery and codirector of the transcatheter valve program at the University of Pennsylvania in Philadelphia. “We’re trying to force continuous quality improvement across U.S. TAVR teams,” Dr. Bavaria explained in an interview.

The key to this change will be the data collected on every U.S. TAVR patient in the Transcatheter Valve Therapy registry maintained by the American College of Cardiology and the Society of Thoracic Surgeons, which now has data on more than 120,000 patients who have undergone TAVR at what are now 582 active U.S. TAVR programs, noted Carl L. Tommaso, MD, an interventional cardiologist with NorthShore Medical Group in Bannockburn, Ill., and cochair of the writing panel. “You need to do risk adjustment to measure quality of care,” and the robust database that now exists has begun to make this possible, said Dr. Tommaso, who neither performs TAVR procedures nor participates on a TAVR team. Statistical analyses based on this substantial and always-expanding database of TAVR patients now allows for risk-adjusted assessment of in hospital and 30-day mortality, and risk-adjusted evaluation of 1-year mortality and quality-of-life outcomes are expected within the next couple of years.

“We’re still not yet at the point of having good, risk-adjusted models” for all these measures, but our hope is that in the next 2-7 years we can move completely to quality measures, as has already been done for percutaneous coronary interventions” and away from procedure volume, which currently serves as a surrogate marker for a TAVR program’s competence.

The new document continues to call for TAVR programs to average at least 50 TAVR procedures a year or at least 100 every 2 years, but primarily to insure that each TAVR program can generate enough data about its performance to produce statistically reliable numbers.

“The volume floors are only there because you can’t measure quality without volume,” said Dr. Bavaria. “It’s impossible to measure quality without a certain procedure volume.”

Dr. Bavaria stressed, however, that the goal of the new document is not to limit TAVR programs based on their procedure volume, especially because another goal of the document is to ensure reasonable geographic access to TAVR for U.S. patients. “This document does not advocate for any program to shut down,” he declared. On top of that, “we have no problem with new programs,” although the document noted that “the major threat to low volume sites growing and achieving higher levels of experience is the opening of additional sites in the same geographic region.”

In 2017, 204 of the 525 sites (39%) performing TAVR at that time were performing fewer than 50 procedures annually, the document said, but added that many TAVR centers now operate in what are predominantly rural regions “and it is important that they remain active if they can document acceptable quality even if they should fall below volume thresholds to maintain patient access to care.”

Dr. Tommaso said that perhaps a TAVR center in Alaska, for example, might not meet the 50 cases/year standard, “but I don’t think anyone would worry if the volume was low because we’re serving patients in Alaska.” Currently, 84% of TAVR centers that have been operating for more than 2 years meet the 50 procedures/year threshold, he added. And TAVR centers now operate in 49 of the 50 states, with only Wyoming lacking a TAVR facility within its borders. Despite this, use of TAVR among Wyoming residents is comparable to the rate in Illinois, Dr. Bavaria said.

Both cochairs also highlighted that, with TAVR now approved for patients at moderate risk for aortic-valve surgery, the number of patients who are TAVR candidates has grown, and it’s possible that pending trial results will soon broaden TAVR’s availability to low-risk patients, a step that would greatly further expand the potential patient pool for the procedure.

The revised recommendations and requirements will make it “a little easier to start a new program, except now, for the first time, you need to start with an operator who is already experienced with TAVR,” noted Dr. Bavaria. “The TAVR technology is now mature enough that it’s inappropriate to have learning-curve mortality.” But aside from this the new standards lower the bar a bit for a center’s volume of percutaneous coronary interventions and surgical aortic valve replacements. The revision also maintains that an examination by and consultation with a single cardiac surgeon by a prospective TAVR patient is adequate, similar to the 2012 document, although the Center for Medicare & Medicaid Services mandated in its coverage decision that prospective TAVR patients consult with two cardiac surgeons, the so-called “two-surgeon rule.” If CMS eliminated the two-surgeon rule it would “streamline” the process that patients go through when being assessed for TAVR, Dr. Tommaso observed, and both he and Dr. Bavaria expressed hope that the new document might prompt CMS to reconsider this guidance.

“We felt that two surgeons weren’t needed,” but the document specifies that both the surgeon and the cardiologist whom a prospective patient consults before finalizing plans for the intervention should both be members of the multidisciplinary team that performs the procedure. Until now, these clinicians weren’t specified as necessarily members of the TAVR team, Dr. Bavaria said.

One additional new element in the revised document is specification of shared decision making as the mechanism patients should go through when considering TAVR relative to their other management options, Dr. Tommaso said.Dr. Bavaria and Dr. Tommaso had no disclosures.

[email protected]
 

SOURCE: Bavaria JE et al. JACC. 2018 Jul18. doi:10.1016/j.jacc.2018.07.002.
 

Spinal muscular atrophy (SMA) is now among the disorders officially included in the Recommended Uniform Screening Panel (RUSP), which is used by state public health departments to screen newborns for genetic disorders.

Secretary of the Department of Health and Human Services Alex M. Azar II formally added SMA to the panel July 2 on the recommendation of the Advisory Committee on Heritable Disorders in Newborns and Children.

AngelIce/Thinkstock

“Adding SMA to the list will help ensure that babies born with SMA are identified, so that they have the opportunity to benefit from early treatment and intervention,” according to a statement from the Muscular Dystrophy Association about the decision. “This testing can also provide families with a genetic diagnosis – information that often is required to determine whether their child is eligible to participate in clinical trials.”

Adding SMA to the RUSP does not mean states must screen newborns for the disorder. Each state’s public health apparatus decides independently whether to accept the recommendation and which disorders on the RUSP to screen for. Most states screen for most disorders on the RUSP. Evidence compiled by the advisory committee suggested wide variation in resources, infrastructure, funding, and time to implementation among states.

An estimated 1 in 11,000 newborns have SMA, a disorder caused by mutations in the survival motor neuron 1 (SMN1) gene. SMA affects motor neurons in the brain stem and spinal cord leading to motor weakness and atrophy. The only treatment for SMA had been palliative care until the Food and Drug Administration approved nusinersen (Spinraza) for the disorder in December 2016, although the drug’s approval has raised some ethical questions.^1-3

After reviewing the evidence at their February 8, 2018 meeting, the advisory committee recommended the addition of spinal muscular atrophy screening to the RUSP in a March 8, 2018, letter from committee chair Joseph A. Bocchini Jr., MD, who is a professor and the chairman of the department of pediatrics at Louisiana State University Health in Shreveport.

Secretary Azar accepted the recommendation based on the evidence the committee provided; he also requested a follow-up report within 2 years “describing the status of implementing newborn screening for SMA and clinical outcomes of early treatment, including any potential harms, for infants diagnosed with SMA.”

The advisory committee makes its recommendations to the HHS on which heritable disorders to include in the RUSP after they have assessed a systematic, evidence-based review assigned by the committee to an external independent group. Alex R. Kemper, MD, MPH, a professor of pediatrics at the Ohio State University and division chief of ambulatory pediatrics at Nationwide Children’s Hospital, both in Columbus, led the review group for SMA. Dr. Kemper is also deputy editor of the journal Pediatrics and a member of the U.S. Preventive Services Task Force.

Wikimedia Commons/WWsgConnect/CC-SA 4.0

According to Secretary Azar’s summary in his July 2, 2018, letter of acceptance, the evidence review suggested that “early screening and treatment can lead to decreased mortality for individuals with SMA and improved motor milestones.”

Dr. Kemper elaborated in an interview that, “SMA can be detected through newborn screening, and treatment is now available that can not only reduce the risk of death but decrease the development of neurologic impairment. As with adding any condition to newborn screening, public health laboratories will need to develop strategies to incorporate the screening test. The current FDA-approved treatment, nusinersen, is delivered by lumbar puncture into the spinal fluid. In addition, there are exciting advances in gene therapy leading to new treatment approaches.”

Approximately 95% of SMA cases result from the deletion of exon 7 from both alleles of SMN1. (Other rarer cases are caused by mutations in different genes.) Without the SMN protein produced by SMN1, a person gradually loses muscle function.

A similar gene, SMN2, also can produce the SMN protein but in much lower amounts, typically less than 10% of what a person needs. People can, however, have multiple copies of SMN2, which can produce slightly more SMN protein for a slower disease process.

The five types of spinal muscular atrophy are determined according to symptom onset, which directly correlates with disorder severity and prognosis. Just over half (54%) of SMA cases are Type I, in which progressive weakness occurs over the first 6 months of life and results in early death. Only 18% of children with Type I live past age 4 years, and 68% die by age 2 years. Type 0 is rarer but more severe, usually causing fetal loss or early infant death.

Type II represents 18% of SMA cases and causes progressive weakness by age 15 months. Most people with Type II survive to their 30s but then experience respiratory failure and rarely reach their fourth decade. Individuals with Types III and IV typically have a normal lifespan and only begin to see progressive muscle weakness after 1 year old or in adulthood.

Dr. Kemper’s group focused on the three types diagnosed in infancy: types I, II, and III.

Dr. Kemper emphasized in an interview that “it will be critical to make sure that infants diagnosed with SMA through newborn screening receive follow-up shortly afterward to determine whether they would benefit from nusinersen. More information is needed about the long-term outcomes of those infants who begin treatment following newborn screening so we not only know about outcomes in later childhood and adolescence but treatment approaches can be further refined and personalized.”

Nusinersen works by altering the splicing of precursor messenger RNA in SMN2 so that the mRNA strands are longer, which thereby increases how much SMN protein is produced. Concerns about the medication, however, have included its cost – $750,000 in the first year and $375,000 every following year for life – and potential adverse events from repeated administration. Nusinersen is injected into the spinal canal four times in the first year and then once annually, and the painful injections require patient immobilization. Potential adverse events include thrombocytopenia and nephrotoxicity, along with potential complications from repeated lumbar punctures over time.²

Other concerns about the drug include its limited evidence base, lack of long-term data, associated costs with administration (for example, travel costs), the potential for patients taking nusinersen to be excluded from future clinical trials on other treatments, and ensuring parents have enough information on the drug’s limitations and potential risks to provide adequate informed consent.²

Yet evidence to date is favorable in children with early onset. Dr. Bocchini wrote in the letter to Secretary Azar that “limited data suggest that treatment effect is greater when the treatment is initiated before symptoms develop and when the individual has more copies of SMN2.”

Dr. Kemper’s group concluded that screening can detect SMA in newborns and that treatment can modify disease course. “Grey literature suggests those with total disease duration less than or equal to 12 weeks before nusinersen treatment were more likely to have better outcomes than those with longer periods of disease duration.”

“Presymptomatic treatment alters the natural history” of the disorder, the group found, although outcome data past 1 year of age are not yet available. Based on findings from a New York pilot program, they predicted that nationwide newborn screening would avert 33 deaths and 48 cases of children who were dependent on a ventilator among an annual cohort of 4 million births.

At the time of the evidence review, Massachusetts, Minnesota, Missouri, North Carolina, New York, Utah, and Wisconsin initiated pilot programs or whole-population mandated screening for SMA. Of the three states that reported costs, all reported costs at $1 or less per screen.

The research for the evidence review was funded by a Health Resources and Services Administration grant to Duke University, Durham, N.C. No disclosures were provided for evidence review group members.
 

References

1. Gene Ther. 2017 Sep;24(9):534-8.

2. JAMA Intern Med. 2018 Jun 1;178(6):743-44.

3. JAMA Pediatr. 2018 Feb 1;172(2):188-92.

Spinal muscular atrophy (SMA) is now among the disorders officially included in the Recommended Uniform Screening Panel (RUSP), which is used by state public health departments to screen newborns for genetic disorders.

Secretary of the Department of Health and Human Services Alex M. Azar II formally added SMA to the panel July 2 on the recommendation of the Advisory Committee on Heritable Disorders in Newborns and Children.

AngelIce/Thinkstock

“Adding SMA to the list will help ensure that babies born with SMA are identified, so that they have the opportunity to benefit from early treatment and intervention,” according to a statement from the Muscular Dystrophy Association about the decision. “This testing can also provide families with a genetic diagnosis – information that often is required to determine whether their child is eligible to participate in clinical trials.”

Adding SMA to the RUSP does not mean states must screen newborns for the disorder. Each state’s public health apparatus decides independently whether to accept the recommendation and which disorders on the RUSP to screen for. Most states screen for most disorders on the RUSP. Evidence compiled by the advisory committee suggested wide variation in resources, infrastructure, funding, and time to implementation among states.

An estimated 1 in 11,000 newborns have SMA, a disorder caused by mutations in the survival motor neuron 1 (SMN1) gene. SMA affects motor neurons in the brain stem and spinal cord leading to motor weakness and atrophy. The only treatment for SMA had been palliative care until the Food and Drug Administration approved nusinersen (Spinraza) for the disorder in December 2016, although the drug’s approval has raised some ethical questions.^1-3

After reviewing the evidence at their February 8, 2018 meeting, the advisory committee recommended the addition of spinal muscular atrophy screening to the RUSP in a March 8, 2018, letter from committee chair Joseph A. Bocchini Jr., MD, who is a professor and the chairman of the department of pediatrics at Louisiana State University Health in Shreveport.

Secretary Azar accepted the recommendation based on the evidence the committee provided; he also requested a follow-up report within 2 years “describing the status of implementing newborn screening for SMA and clinical outcomes of early treatment, including any potential harms, for infants diagnosed with SMA.”

The advisory committee makes its recommendations to the HHS on which heritable disorders to include in the RUSP after they have assessed a systematic, evidence-based review assigned by the committee to an external independent group. Alex R. Kemper, MD, MPH, a professor of pediatrics at the Ohio State University and division chief of ambulatory pediatrics at Nationwide Children’s Hospital, both in Columbus, led the review group for SMA. Dr. Kemper is also deputy editor of the journal Pediatrics and a member of the U.S. Preventive Services Task Force.

Wikimedia Commons/WWsgConnect/CC-SA 4.0

According to Secretary Azar’s summary in his July 2, 2018, letter of acceptance, the evidence review suggested that “early screening and treatment can lead to decreased mortality for individuals with SMA and improved motor milestones.”

Dr. Kemper elaborated in an interview that, “SMA can be detected through newborn screening, and treatment is now available that can not only reduce the risk of death but decrease the development of neurologic impairment. As with adding any condition to newborn screening, public health laboratories will need to develop strategies to incorporate the screening test. The current FDA-approved treatment, nusinersen, is delivered by lumbar puncture into the spinal fluid. In addition, there are exciting advances in gene therapy leading to new treatment approaches.”

Approximately 95% of SMA cases result from the deletion of exon 7 from both alleles of SMN1. (Other rarer cases are caused by mutations in different genes.) Without the SMN protein produced by SMN1, a person gradually loses muscle function.

A similar gene, SMN2, also can produce the SMN protein but in much lower amounts, typically less than 10% of what a person needs. People can, however, have multiple copies of SMN2, which can produce slightly more SMN protein for a slower disease process.

The five types of spinal muscular atrophy are determined according to symptom onset, which directly correlates with disorder severity and prognosis. Just over half (54%) of SMA cases are Type I, in which progressive weakness occurs over the first 6 months of life and results in early death. Only 18% of children with Type I live past age 4 years, and 68% die by age 2 years. Type 0 is rarer but more severe, usually causing fetal loss or early infant death.

Type II represents 18% of SMA cases and causes progressive weakness by age 15 months. Most people with Type II survive to their 30s but then experience respiratory failure and rarely reach their fourth decade. Individuals with Types III and IV typically have a normal lifespan and only begin to see progressive muscle weakness after 1 year old or in adulthood.

Dr. Kemper’s group focused on the three types diagnosed in infancy: types I, II, and III.

Dr. Kemper emphasized in an interview that “it will be critical to make sure that infants diagnosed with SMA through newborn screening receive follow-up shortly afterward to determine whether they would benefit from nusinersen. More information is needed about the long-term outcomes of those infants who begin treatment following newborn screening so we not only know about outcomes in later childhood and adolescence but treatment approaches can be further refined and personalized.”

Nusinersen works by altering the splicing of precursor messenger RNA in SMN2 so that the mRNA strands are longer, which thereby increases how much SMN protein is produced. Concerns about the medication, however, have included its cost – $750,000 in the first year and $375,000 every following year for life – and potential adverse events from repeated administration. Nusinersen is injected into the spinal canal four times in the first year and then once annually, and the painful injections require patient immobilization. Potential adverse events include thrombocytopenia and nephrotoxicity, along with potential complications from repeated lumbar punctures over time.²

Other concerns about the drug include its limited evidence base, lack of long-term data, associated costs with administration (for example, travel costs), the potential for patients taking nusinersen to be excluded from future clinical trials on other treatments, and ensuring parents have enough information on the drug’s limitations and potential risks to provide adequate informed consent.²

Yet evidence to date is favorable in children with early onset. Dr. Bocchini wrote in the letter to Secretary Azar that “limited data suggest that treatment effect is greater when the treatment is initiated before symptoms develop and when the individual has more copies of SMN2.”

Dr. Kemper’s group concluded that screening can detect SMA in newborns and that treatment can modify disease course. “Grey literature suggests those with total disease duration less than or equal to 12 weeks before nusinersen treatment were more likely to have better outcomes than those with longer periods of disease duration.”

“Presymptomatic treatment alters the natural history” of the disorder, the group found, although outcome data past 1 year of age are not yet available. Based on findings from a New York pilot program, they predicted that nationwide newborn screening would avert 33 deaths and 48 cases of children who were dependent on a ventilator among an annual cohort of 4 million births.

At the time of the evidence review, Massachusetts, Minnesota, Missouri, North Carolina, New York, Utah, and Wisconsin initiated pilot programs or whole-population mandated screening for SMA. Of the three states that reported costs, all reported costs at $1 or less per screen.

The research for the evidence review was funded by a Health Resources and Services Administration grant to Duke University, Durham, N.C. No disclosures were provided for evidence review group members.
 

References

1. Gene Ther. 2017 Sep;24(9):534-8.

2. JAMA Intern Med. 2018 Jun 1;178(6):743-44.

3. JAMA Pediatr. 2018 Feb 1;172(2):188-92.

Spinal muscular atrophy (SMA) is now among the disorders officially included in the Recommended Uniform Screening Panel (RUSP), which is used by state public health departments to screen newborns for genetic disorders.

Secretary of the Department of Health and Human Services Alex M. Azar II formally added SMA to the panel July 2 on the recommendation of the Advisory Committee on Heritable Disorders in Newborns and Children.

AngelIce/Thinkstock

“Adding SMA to the list will help ensure that babies born with SMA are identified, so that they have the opportunity to benefit from early treatment and intervention,” according to a statement from the Muscular Dystrophy Association about the decision. “This testing can also provide families with a genetic diagnosis – information that often is required to determine whether their child is eligible to participate in clinical trials.”

Adding SMA to the RUSP does not mean states must screen newborns for the disorder. Each state’s public health apparatus decides independently whether to accept the recommendation and which disorders on the RUSP to screen for. Most states screen for most disorders on the RUSP. Evidence compiled by the advisory committee suggested wide variation in resources, infrastructure, funding, and time to implementation among states.

An estimated 1 in 11,000 newborns have SMA, a disorder caused by mutations in the survival motor neuron 1 (SMN1) gene. SMA affects motor neurons in the brain stem and spinal cord leading to motor weakness and atrophy. The only treatment for SMA had been palliative care until the Food and Drug Administration approved nusinersen (Spinraza) for the disorder in December 2016, although the drug’s approval has raised some ethical questions.^1-3

After reviewing the evidence at their February 8, 2018 meeting, the advisory committee recommended the addition of spinal muscular atrophy screening to the RUSP in a March 8, 2018, letter from committee chair Joseph A. Bocchini Jr., MD, who is a professor and the chairman of the department of pediatrics at Louisiana State University Health in Shreveport.

Secretary Azar accepted the recommendation based on the evidence the committee provided; he also requested a follow-up report within 2 years “describing the status of implementing newborn screening for SMA and clinical outcomes of early treatment, including any potential harms, for infants diagnosed with SMA.”

The advisory committee makes its recommendations to the HHS on which heritable disorders to include in the RUSP after they have assessed a systematic, evidence-based review assigned by the committee to an external independent group. Alex R. Kemper, MD, MPH, a professor of pediatrics at the Ohio State University and division chief of ambulatory pediatrics at Nationwide Children’s Hospital, both in Columbus, led the review group for SMA. Dr. Kemper is also deputy editor of the journal Pediatrics and a member of the U.S. Preventive Services Task Force.

Wikimedia Commons/WWsgConnect/CC-SA 4.0

According to Secretary Azar’s summary in his July 2, 2018, letter of acceptance, the evidence review suggested that “early screening and treatment can lead to decreased mortality for individuals with SMA and improved motor milestones.”

Dr. Kemper elaborated in an interview that, “SMA can be detected through newborn screening, and treatment is now available that can not only reduce the risk of death but decrease the development of neurologic impairment. As with adding any condition to newborn screening, public health laboratories will need to develop strategies to incorporate the screening test. The current FDA-approved treatment, nusinersen, is delivered by lumbar puncture into the spinal fluid. In addition, there are exciting advances in gene therapy leading to new treatment approaches.”

Approximately 95% of SMA cases result from the deletion of exon 7 from both alleles of SMN1. (Other rarer cases are caused by mutations in different genes.) Without the SMN protein produced by SMN1, a person gradually loses muscle function.

A similar gene, SMN2, also can produce the SMN protein but in much lower amounts, typically less than 10% of what a person needs. People can, however, have multiple copies of SMN2, which can produce slightly more SMN protein for a slower disease process.

The five types of spinal muscular atrophy are determined according to symptom onset, which directly correlates with disorder severity and prognosis. Just over half (54%) of SMA cases are Type I, in which progressive weakness occurs over the first 6 months of life and results in early death. Only 18% of children with Type I live past age 4 years, and 68% die by age 2 years. Type 0 is rarer but more severe, usually causing fetal loss or early infant death.

Type II represents 18% of SMA cases and causes progressive weakness by age 15 months. Most people with Type II survive to their 30s but then experience respiratory failure and rarely reach their fourth decade. Individuals with Types III and IV typically have a normal lifespan and only begin to see progressive muscle weakness after 1 year old or in adulthood.

Dr. Kemper’s group focused on the three types diagnosed in infancy: types I, II, and III.

Dr. Kemper emphasized in an interview that “it will be critical to make sure that infants diagnosed with SMA through newborn screening receive follow-up shortly afterward to determine whether they would benefit from nusinersen. More information is needed about the long-term outcomes of those infants who begin treatment following newborn screening so we not only know about outcomes in later childhood and adolescence but treatment approaches can be further refined and personalized.”

Nusinersen works by altering the splicing of precursor messenger RNA in SMN2 so that the mRNA strands are longer, which thereby increases how much SMN protein is produced. Concerns about the medication, however, have included its cost – $750,000 in the first year and $375,000 every following year for life – and potential adverse events from repeated administration. Nusinersen is injected into the spinal canal four times in the first year and then once annually, and the painful injections require patient immobilization. Potential adverse events include thrombocytopenia and nephrotoxicity, along with potential complications from repeated lumbar punctures over time.²

Other concerns about the drug include its limited evidence base, lack of long-term data, associated costs with administration (for example, travel costs), the potential for patients taking nusinersen to be excluded from future clinical trials on other treatments, and ensuring parents have enough information on the drug’s limitations and potential risks to provide adequate informed consent.²

Yet evidence to date is favorable in children with early onset. Dr. Bocchini wrote in the letter to Secretary Azar that “limited data suggest that treatment effect is greater when the treatment is initiated before symptoms develop and when the individual has more copies of SMN2.”

Dr. Kemper’s group concluded that screening can detect SMA in newborns and that treatment can modify disease course. “Grey literature suggests those with total disease duration less than or equal to 12 weeks before nusinersen treatment were more likely to have better outcomes than those with longer periods of disease duration.”

“Presymptomatic treatment alters the natural history” of the disorder, the group found, although outcome data past 1 year of age are not yet available. Based on findings from a New York pilot program, they predicted that nationwide newborn screening would avert 33 deaths and 48 cases of children who were dependent on a ventilator among an annual cohort of 4 million births.

At the time of the evidence review, Massachusetts, Minnesota, Missouri, North Carolina, New York, Utah, and Wisconsin initiated pilot programs or whole-population mandated screening for SMA. Of the three states that reported costs, all reported costs at $1 or less per screen.

The research for the evidence review was funded by a Health Resources and Services Administration grant to Duke University, Durham, N.C. No disclosures were provided for evidence review group members.
 

References

1. Gene Ther. 2017 Sep;24(9):534-8.

2. JAMA Intern Med. 2018 Jun 1;178(6):743-44.

3. JAMA Pediatr. 2018 Feb 1;172(2):188-92.

BARCELONA – There was no overall survival advantage for patients with metastatic colorectal cancer treated with the immune checkpoint inhibitor atezolizumab (Tencentriq) either in combination with cobimetinib (Cotellic) or as monotherapy compared with regorafenib (Stivarga), results of the randomized phase 3 IMblaze370 trial showed.

Neil Osterweil/MDedge News

Median overall survival (OS, the primary endpoint) for 183 patients treated with atezolizumab and cobimetinib was 8.9 months, compared with 7.1 months for 90 patients treated with atezolizumab monotherapy, and 8.5 months for 90 patients treated with regorafenib. None of the differences were statistically significant, reported Johanna C. Bendell, MD, of the Sarah Cannon Research Institute in Nashville, Tenn.

“The lack of clinical activity may be due to the immune-excluded phenotype of metastatic colorectal cancer. The dual inhibition of the PD-L1 immune checkpoint and MAP kinase–mediated immune suppression may not be sufficient to generate that immune response that we need for antitumor activity,” she said at the European Society for Medical Oncology World Congress on Gastrointestinal Cancer.

In an interview, Dr. Bendell said that “we were all hoping that we were going to see some benefit, and we had some preliminary data suggesting that we would. What we still need to see is whether there are potentially populations of patients who might have benefited more, which hopefully will come out of the biomarker analysis.”

The results demonstrate the need for controlled comparison trials to detect true clinical benefits from a specific agent or combination, she added.

Microsatellite-stable disease, which accounts for approximately 95% of cases of metastatic colorectal cancer (MSS mCRC) is considered to be an immune-excluded or “cold” tumor type due to a lack of tumor-infiltrating lymphocytes. Single agent inhibitors of the programmed death 1 protein and its ligand (PD1/PD-L1) have only minimal activity in MSS mCRC, she noted.

In preclinical studies, there was evidence to suggest that the combination of atezolizumab and cobimetinib could augment antitumor T-cell responses. The combination also was shown to be safe and to demonstrate “promising” clinical activity in a phase 1b trial conducted by Dr. Bendell and her colleagues.

To see whether the initial promise of atezolizumab in this population held up under closer scrutiny, the investigators enrolled patients with MSS mCRC refractory to chemotherapy and randomly assigned them on a 2:1:1 basis to receive either atezolizumab plus cobimetinib, atezolizumab monotherapy, or regorafenib.

Atezolizumab was administered intravenously at 840 mg every 2 weeks in the combination arm, and at 1,200 mg every 3 weeks in the monotherapy arm. Cobimetinib was administered orally at 60 mg on a 21-days-on/7-days-off schedule. Regorafenib was administered orally at 160 mg on a 21-days-on/7-days-off schedule.

As noted, the trial did not meet its primary endpoint of an OS benefit in the intention-to-treat population. In addition, there were no significant differences in either median progression-free survival, overall response rates, or duration of response.

Response rates for both the atezolizumab/cobimetinib combination and atezolizumab monotherapy arms were consistent with published data for other PD-1/PD-L1 inhibitors, and the safety of the combination was consistent with the known safety profiles of the individual agents, Dr. Bendell said.

The investigators are conducting extensive biomarker evaluations and gene expression profiles, and hope to present the results of these studies at a future conference, she added.

SOURCE: Bendell J et al. ESMO GI 2018. Abstract LBA-004.

BARCELONA – There was no overall survival advantage for patients with metastatic colorectal cancer treated with the immune checkpoint inhibitor atezolizumab (Tencentriq) either in combination with cobimetinib (Cotellic) or as monotherapy compared with regorafenib (Stivarga), results of the randomized phase 3 IMblaze370 trial showed.

Neil Osterweil/MDedge News

Median overall survival (OS, the primary endpoint) for 183 patients treated with atezolizumab and cobimetinib was 8.9 months, compared with 7.1 months for 90 patients treated with atezolizumab monotherapy, and 8.5 months for 90 patients treated with regorafenib. None of the differences were statistically significant, reported Johanna C. Bendell, MD, of the Sarah Cannon Research Institute in Nashville, Tenn.

“The lack of clinical activity may be due to the immune-excluded phenotype of metastatic colorectal cancer. The dual inhibition of the PD-L1 immune checkpoint and MAP kinase–mediated immune suppression may not be sufficient to generate that immune response that we need for antitumor activity,” she said at the European Society for Medical Oncology World Congress on Gastrointestinal Cancer.

In an interview, Dr. Bendell said that “we were all hoping that we were going to see some benefit, and we had some preliminary data suggesting that we would. What we still need to see is whether there are potentially populations of patients who might have benefited more, which hopefully will come out of the biomarker analysis.”

The results demonstrate the need for controlled comparison trials to detect true clinical benefits from a specific agent or combination, she added.

Microsatellite-stable disease, which accounts for approximately 95% of cases of metastatic colorectal cancer (MSS mCRC) is considered to be an immune-excluded or “cold” tumor type due to a lack of tumor-infiltrating lymphocytes. Single agent inhibitors of the programmed death 1 protein and its ligand (PD1/PD-L1) have only minimal activity in MSS mCRC, she noted.

In preclinical studies, there was evidence to suggest that the combination of atezolizumab and cobimetinib could augment antitumor T-cell responses. The combination also was shown to be safe and to demonstrate “promising” clinical activity in a phase 1b trial conducted by Dr. Bendell and her colleagues.

To see whether the initial promise of atezolizumab in this population held up under closer scrutiny, the investigators enrolled patients with MSS mCRC refractory to chemotherapy and randomly assigned them on a 2:1:1 basis to receive either atezolizumab plus cobimetinib, atezolizumab monotherapy, or regorafenib.

Atezolizumab was administered intravenously at 840 mg every 2 weeks in the combination arm, and at 1,200 mg every 3 weeks in the monotherapy arm. Cobimetinib was administered orally at 60 mg on a 21-days-on/7-days-off schedule. Regorafenib was administered orally at 160 mg on a 21-days-on/7-days-off schedule.

As noted, the trial did not meet its primary endpoint of an OS benefit in the intention-to-treat population. In addition, there were no significant differences in either median progression-free survival, overall response rates, or duration of response.

Response rates for both the atezolizumab/cobimetinib combination and atezolizumab monotherapy arms were consistent with published data for other PD-1/PD-L1 inhibitors, and the safety of the combination was consistent with the known safety profiles of the individual agents, Dr. Bendell said.

The investigators are conducting extensive biomarker evaluations and gene expression profiles, and hope to present the results of these studies at a future conference, she added.

SOURCE: Bendell J et al. ESMO GI 2018. Abstract LBA-004.

BARCELONA – There was no overall survival advantage for patients with metastatic colorectal cancer treated with the immune checkpoint inhibitor atezolizumab (Tencentriq) either in combination with cobimetinib (Cotellic) or as monotherapy compared with regorafenib (Stivarga), results of the randomized phase 3 IMblaze370 trial showed.

Neil Osterweil/MDedge News

Median overall survival (OS, the primary endpoint) for 183 patients treated with atezolizumab and cobimetinib was 8.9 months, compared with 7.1 months for 90 patients treated with atezolizumab monotherapy, and 8.5 months for 90 patients treated with regorafenib. None of the differences were statistically significant, reported Johanna C. Bendell, MD, of the Sarah Cannon Research Institute in Nashville, Tenn.

“The lack of clinical activity may be due to the immune-excluded phenotype of metastatic colorectal cancer. The dual inhibition of the PD-L1 immune checkpoint and MAP kinase–mediated immune suppression may not be sufficient to generate that immune response that we need for antitumor activity,” she said at the European Society for Medical Oncology World Congress on Gastrointestinal Cancer.

In an interview, Dr. Bendell said that “we were all hoping that we were going to see some benefit, and we had some preliminary data suggesting that we would. What we still need to see is whether there are potentially populations of patients who might have benefited more, which hopefully will come out of the biomarker analysis.”

The results demonstrate the need for controlled comparison trials to detect true clinical benefits from a specific agent or combination, she added.

Microsatellite-stable disease, which accounts for approximately 95% of cases of metastatic colorectal cancer (MSS mCRC) is considered to be an immune-excluded or “cold” tumor type due to a lack of tumor-infiltrating lymphocytes. Single agent inhibitors of the programmed death 1 protein and its ligand (PD1/PD-L1) have only minimal activity in MSS mCRC, she noted.

In preclinical studies, there was evidence to suggest that the combination of atezolizumab and cobimetinib could augment antitumor T-cell responses. The combination also was shown to be safe and to demonstrate “promising” clinical activity in a phase 1b trial conducted by Dr. Bendell and her colleagues.

To see whether the initial promise of atezolizumab in this population held up under closer scrutiny, the investigators enrolled patients with MSS mCRC refractory to chemotherapy and randomly assigned them on a 2:1:1 basis to receive either atezolizumab plus cobimetinib, atezolizumab monotherapy, or regorafenib.

Atezolizumab was administered intravenously at 840 mg every 2 weeks in the combination arm, and at 1,200 mg every 3 weeks in the monotherapy arm. Cobimetinib was administered orally at 60 mg on a 21-days-on/7-days-off schedule. Regorafenib was administered orally at 160 mg on a 21-days-on/7-days-off schedule.

As noted, the trial did not meet its primary endpoint of an OS benefit in the intention-to-treat population. In addition, there were no significant differences in either median progression-free survival, overall response rates, or duration of response.

Response rates for both the atezolizumab/cobimetinib combination and atezolizumab monotherapy arms were consistent with published data for other PD-1/PD-L1 inhibitors, and the safety of the combination was consistent with the known safety profiles of the individual agents, Dr. Bendell said.

The investigators are conducting extensive biomarker evaluations and gene expression profiles, and hope to present the results of these studies at a future conference, she added.

SOURCE: Bendell J et al. ESMO GI 2018. Abstract LBA-004.

Stress balls and hand-holding failed to lower patient anxiety during head and neck skin cancer removals under local anesthesia, according to a randomized trial of 135 patients at Northwestern University, Chicago.

TommL/E+/Getty Images

[email protected]

SOURCE: Yanes AF et al. JAMA Dermatol. 2018 Jul 18. doi:10.1001/jamadermatol.2018.1783.

Stress balls and hand-holding failed to lower patient anxiety during head and neck skin cancer removals under local anesthesia, according to a randomized trial of 135 patients at Northwestern University, Chicago.

TommL/E+/Getty Images

[email protected]

SOURCE: Yanes AF et al. JAMA Dermatol. 2018 Jul 18. doi:10.1001/jamadermatol.2018.1783.

Stress balls and hand-holding failed to lower patient anxiety during head and neck skin cancer removals under local anesthesia, according to a randomized trial of 135 patients at Northwestern University, Chicago.

TommL/E+/Getty Images

[email protected]

SOURCE: Yanes AF et al. JAMA Dermatol. 2018 Jul 18. doi:10.1001/jamadermatol.2018.1783.

Mentorship is a hot topic. Mentors are generally perceived as knowledgeable, kind, generous souls who will guide mentees through tough challenges and be your pal. I suggest to you that while such encounters are marvelous, and to be sought out, some of the most important lessons are taught by members of the opposite cast.

erhui1979/Getty Images

The presiding secretary of the Ohio state medical board was a brigadier general, still active reserve, tall with a bristling countenance. I was president of the Ohio Dermatological Association and was accompanied by Mark Bechtel, MD, who was our chair of state legislation. We had been invited to discuss possible office surgery regulations with the medical board. We were secure in our personal knowledge that local anesthesia was very safe, and that there had not been any deaths related to local anesthesia administered by dermatologists in Ohio (or anywhere else). We expected to tell the medical board there was nothing to worry about, and we could all go home. This was 17 years ago.

It quickly became apparent that there had been an extensive prior dialogue between the medical board and representatives of the American College of Surgeons and the American Society of Anesthesiologists. They sat at the head table with the secretary of the medical board.

“In our experience, there are really some dangerous procedures going on in the office setting under local anesthesia, and this area desperately needs regulation,” the anesthesiologist’s representative said. The surgeon’s representative chimed in: “From what I’ve heard, office surgery is a ticking time bomb and needs regulation, and as soon as possible.” This prattle went on and on, with the medical board secretary sympathetically nodding his head. I raised my hand and was ignored – and ignored. It became apparent that this was a show trial, and our opinion was not really wanted, just our attendance noted in the minutes. Finally, I stood up and protested, and pointed out that all of this “testimony” was conjecture and personal opinion, and that there was no factual basis for their claims. The president stiffened, stood up, and started barking orders.He told me to “sit down and not speak unless I was called on.” I sat down and shut up. And I was never called on. Mark Bechtel put a calming hand on my arm. Goodness, I had not been treated like that since junior high.

I soon realized that dermatologists were not at all important to the medical board, and that the medical board had no idea about our safety and efficiency and really did not care.

Following the meeting, I was told by Larry Lanier (the American Academy of Dermatology state legislative liaison at the time) that Ohio was to be the test state to restrict local anesthesia and tumescent anesthesia nationwide. He explained that some widely reported liposuction-related deaths in Florida had given the medical board the “justification” to act. He went on to explain that yes, there were no data either way, but we had better hire a lobbyist and hope for the best.

I was stunned by what I now call (in this case, rough) “mentorship” by the medical board secretary. I understood I could meekly go along – or get angry. I chose the latter, and it has greatly changed my career.

Now, this was not a hot, red, foam-at-the-mouth mad, but a slow burn, the kind that sustains, not consumes.

I went home and did a literature review and was disheartened to find absolutely nothing in the literature on the safety record of dermatologists in the office setting or on the safety of office surgery in general under local anesthesia. We had nothing to back us up.

I looked up the liposuction deaths in Florida and discovered the procedures were all done under general anesthesia or deep sedation by surgeons of one type or another. I also discovered that Florida had enacted mandatory reporting, and the reports could be had for copying costs. I ordered all available, 9 months of data.

We dermatologists passed a special assessment and hired a lobbyist who told us we were too late to have any impact on the impending restrictions. We took a resolution opposing the medical board rules – which would have eliminated using any sedation in the office, even haloperidol and tumescent anesthesia – to the state medical society and got it passed despite the medical board secretary (who was a former president of the society) testifying against us. The Florida data showed no deaths or injuries from using local anesthesia in the office by anyone, and I succeeded in getting a letter addressing that data published expeditiously in the Journal of the American Medical Association (JAMA 2001;285[20]:2582).

The president of the American Society for Dermatologic Surgery at that time, Stephen Mandy, MD, came to town and testified against the proposed restrictions along with about 60 physicians, including dermatologists, plastic surgeons, and other physicians who perform office-based surgery who we had rallied to join us from across the state. So many colleagues joined us, in fact, that some of us had to sit on the floor during the proceedings.

The proposed restrictions evaporated. I and many others have since devoted our research efforts to solidifying dermatology’s safety and quality record. Dr. Bechtel, professor of dermatology at Ohio State University, Columbus, is now secretary of the state medical board. At the last annual state meeting, I thanked the brigadier general, the former secretary of the medical board, for his unlikely mentorship. He smiled and we got our picture taken together.

Dr. Coldiron is in private practice but maintains a clinical assistant professorship at the University of Cincinnati. He cares for patients, teaches medical students and residents, and has several active clinical research projects. Dr. Coldiron is the author of more than 80 scientific letters, papers, and several book chapters, and he speaks frequently on a variety of topics. He is a past president of the American Academy of Dermatology. Write to him at [email protected].

Mentorship is a hot topic. Mentors are generally perceived as knowledgeable, kind, generous souls who will guide mentees through tough challenges and be your pal. I suggest to you that while such encounters are marvelous, and to be sought out, some of the most important lessons are taught by members of the opposite cast.

erhui1979/Getty Images

The presiding secretary of the Ohio state medical board was a brigadier general, still active reserve, tall with a bristling countenance. I was president of the Ohio Dermatological Association and was accompanied by Mark Bechtel, MD, who was our chair of state legislation. We had been invited to discuss possible office surgery regulations with the medical board. We were secure in our personal knowledge that local anesthesia was very safe, and that there had not been any deaths related to local anesthesia administered by dermatologists in Ohio (or anywhere else). We expected to tell the medical board there was nothing to worry about, and we could all go home. This was 17 years ago.

It quickly became apparent that there had been an extensive prior dialogue between the medical board and representatives of the American College of Surgeons and the American Society of Anesthesiologists. They sat at the head table with the secretary of the medical board.

“In our experience, there are really some dangerous procedures going on in the office setting under local anesthesia, and this area desperately needs regulation,” the anesthesiologist’s representative said. The surgeon’s representative chimed in: “From what I’ve heard, office surgery is a ticking time bomb and needs regulation, and as soon as possible.” This prattle went on and on, with the medical board secretary sympathetically nodding his head. I raised my hand and was ignored – and ignored. It became apparent that this was a show trial, and our opinion was not really wanted, just our attendance noted in the minutes. Finally, I stood up and protested, and pointed out that all of this “testimony” was conjecture and personal opinion, and that there was no factual basis for their claims. The president stiffened, stood up, and started barking orders.He told me to “sit down and not speak unless I was called on.” I sat down and shut up. And I was never called on. Mark Bechtel put a calming hand on my arm. Goodness, I had not been treated like that since junior high.

I soon realized that dermatologists were not at all important to the medical board, and that the medical board had no idea about our safety and efficiency and really did not care.

Following the meeting, I was told by Larry Lanier (the American Academy of Dermatology state legislative liaison at the time) that Ohio was to be the test state to restrict local anesthesia and tumescent anesthesia nationwide. He explained that some widely reported liposuction-related deaths in Florida had given the medical board the “justification” to act. He went on to explain that yes, there were no data either way, but we had better hire a lobbyist and hope for the best.

I was stunned by what I now call (in this case, rough) “mentorship” by the medical board secretary. I understood I could meekly go along – or get angry. I chose the latter, and it has greatly changed my career.

Now, this was not a hot, red, foam-at-the-mouth mad, but a slow burn, the kind that sustains, not consumes.

I went home and did a literature review and was disheartened to find absolutely nothing in the literature on the safety record of dermatologists in the office setting or on the safety of office surgery in general under local anesthesia. We had nothing to back us up.

I looked up the liposuction deaths in Florida and discovered the procedures were all done under general anesthesia or deep sedation by surgeons of one type or another. I also discovered that Florida had enacted mandatory reporting, and the reports could be had for copying costs. I ordered all available, 9 months of data.

We dermatologists passed a special assessment and hired a lobbyist who told us we were too late to have any impact on the impending restrictions. We took a resolution opposing the medical board rules – which would have eliminated using any sedation in the office, even haloperidol and tumescent anesthesia – to the state medical society and got it passed despite the medical board secretary (who was a former president of the society) testifying against us. The Florida data showed no deaths or injuries from using local anesthesia in the office by anyone, and I succeeded in getting a letter addressing that data published expeditiously in the Journal of the American Medical Association (JAMA 2001;285[20]:2582).

The president of the American Society for Dermatologic Surgery at that time, Stephen Mandy, MD, came to town and testified against the proposed restrictions along with about 60 physicians, including dermatologists, plastic surgeons, and other physicians who perform office-based surgery who we had rallied to join us from across the state. So many colleagues joined us, in fact, that some of us had to sit on the floor during the proceedings.

The proposed restrictions evaporated. I and many others have since devoted our research efforts to solidifying dermatology’s safety and quality record. Dr. Bechtel, professor of dermatology at Ohio State University, Columbus, is now secretary of the state medical board. At the last annual state meeting, I thanked the brigadier general, the former secretary of the medical board, for his unlikely mentorship. He smiled and we got our picture taken together.

Dr. Coldiron is in private practice but maintains a clinical assistant professorship at the University of Cincinnati. He cares for patients, teaches medical students and residents, and has several active clinical research projects. Dr. Coldiron is the author of more than 80 scientific letters, papers, and several book chapters, and he speaks frequently on a variety of topics. He is a past president of the American Academy of Dermatology. Write to him at [email protected].

Mentorship is a hot topic. Mentors are generally perceived as knowledgeable, kind, generous souls who will guide mentees through tough challenges and be your pal. I suggest to you that while such encounters are marvelous, and to be sought out, some of the most important lessons are taught by members of the opposite cast.

erhui1979/Getty Images

The presiding secretary of the Ohio state medical board was a brigadier general, still active reserve, tall with a bristling countenance. I was president of the Ohio Dermatological Association and was accompanied by Mark Bechtel, MD, who was our chair of state legislation. We had been invited to discuss possible office surgery regulations with the medical board. We were secure in our personal knowledge that local anesthesia was very safe, and that there had not been any deaths related to local anesthesia administered by dermatologists in Ohio (or anywhere else). We expected to tell the medical board there was nothing to worry about, and we could all go home. This was 17 years ago.

It quickly became apparent that there had been an extensive prior dialogue between the medical board and representatives of the American College of Surgeons and the American Society of Anesthesiologists. They sat at the head table with the secretary of the medical board.

“In our experience, there are really some dangerous procedures going on in the office setting under local anesthesia, and this area desperately needs regulation,” the anesthesiologist’s representative said. The surgeon’s representative chimed in: “From what I’ve heard, office surgery is a ticking time bomb and needs regulation, and as soon as possible.” This prattle went on and on, with the medical board secretary sympathetically nodding his head. I raised my hand and was ignored – and ignored. It became apparent that this was a show trial, and our opinion was not really wanted, just our attendance noted in the minutes. Finally, I stood up and protested, and pointed out that all of this “testimony” was conjecture and personal opinion, and that there was no factual basis for their claims. The president stiffened, stood up, and started barking orders.He told me to “sit down and not speak unless I was called on.” I sat down and shut up. And I was never called on. Mark Bechtel put a calming hand on my arm. Goodness, I had not been treated like that since junior high.

I soon realized that dermatologists were not at all important to the medical board, and that the medical board had no idea about our safety and efficiency and really did not care.

Following the meeting, I was told by Larry Lanier (the American Academy of Dermatology state legislative liaison at the time) that Ohio was to be the test state to restrict local anesthesia and tumescent anesthesia nationwide. He explained that some widely reported liposuction-related deaths in Florida had given the medical board the “justification” to act. He went on to explain that yes, there were no data either way, but we had better hire a lobbyist and hope for the best.

I was stunned by what I now call (in this case, rough) “mentorship” by the medical board secretary. I understood I could meekly go along – or get angry. I chose the latter, and it has greatly changed my career.

Now, this was not a hot, red, foam-at-the-mouth mad, but a slow burn, the kind that sustains, not consumes.

I went home and did a literature review and was disheartened to find absolutely nothing in the literature on the safety record of dermatologists in the office setting or on the safety of office surgery in general under local anesthesia. We had nothing to back us up.

I looked up the liposuction deaths in Florida and discovered the procedures were all done under general anesthesia or deep sedation by surgeons of one type or another. I also discovered that Florida had enacted mandatory reporting, and the reports could be had for copying costs. I ordered all available, 9 months of data.

We dermatologists passed a special assessment and hired a lobbyist who told us we were too late to have any impact on the impending restrictions. We took a resolution opposing the medical board rules – which would have eliminated using any sedation in the office, even haloperidol and tumescent anesthesia – to the state medical society and got it passed despite the medical board secretary (who was a former president of the society) testifying against us. The Florida data showed no deaths or injuries from using local anesthesia in the office by anyone, and I succeeded in getting a letter addressing that data published expeditiously in the Journal of the American Medical Association (JAMA 2001;285[20]:2582).

The president of the American Society for Dermatologic Surgery at that time, Stephen Mandy, MD, came to town and testified against the proposed restrictions along with about 60 physicians, including dermatologists, plastic surgeons, and other physicians who perform office-based surgery who we had rallied to join us from across the state. So many colleagues joined us, in fact, that some of us had to sit on the floor during the proceedings.

The proposed restrictions evaporated. I and many others have since devoted our research efforts to solidifying dermatology’s safety and quality record. Dr. Bechtel, professor of dermatology at Ohio State University, Columbus, is now secretary of the state medical board. At the last annual state meeting, I thanked the brigadier general, the former secretary of the medical board, for his unlikely mentorship. He smiled and we got our picture taken together.

Dr. Coldiron is in private practice but maintains a clinical assistant professorship at the University of Cincinnati. He cares for patients, teaches medical students and residents, and has several active clinical research projects. Dr. Coldiron is the author of more than 80 scientific letters, papers, and several book chapters, and he speaks frequently on a variety of topics. He is a past president of the American Academy of Dermatology. Write to him at [email protected].