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AUSTIN, TEXAS – A simple process change helped increase the number of genetic screenings for hereditary cancer risk performed in community ob.gyn. practices, according to Mark S. DeFrancesco, MD, and his associates.

Few community-based ob.gyns. routinely screen their patients for hereditary cancer risks, Dr. DeFrancesco said at the annual meeting of the American College of Obstetricians and Gynecologists, despite ACOG’s position that they are fully trained and qualified to do so. He and his colleagues studied an intervention aimed at streamlining and standardizing genetic assessment in their practice.

A team of physicians, staff, genetic counselors, and process engineers analyzed how hereditary cancer risk assessment was being done at five clinical sites of two community ob.gyn. practices – Dr. DeFrancesco’s practice in Waterbury, Conn., and that of Richard Waldman, MD, in Syracuse, N.Y. – then refined workflows and added tools to create a turnkey process for assessment and screening, Dr. DeFrancesco said.

Under the new process, patients completed a family cancer history in the exam room prior to seeing their physician. Genetic testing was offered to patients who met National Comprehensive Cancer Network (NCCN) guidelines for hereditary/familial high-risk assessment for breast and ovarian cancer (J Natl Compr Canc Netw. 2017 Jan;15[1]:9-20). Those who chose to be tested were able to provide a saliva sample in the office. Counseling was provided to appropriate patients.

The number of patients tested for hereditary risk of breast and ovarian cancer increased dramatically with the new process. During the 8-week period after the intervention, 4% (165) were tested out of 4,107 total patients seen; during the 8 weeks preceding, 1% (43) of 3,882 patients were tested.

Overall, 92.8% (3,811) of patients seen after the intervention provided a family cancer history. Almost a quarter – 23.5% (906) – met NCCN criteria for genetic testing.

A total of 318 patients agreed to undergo genetic testing and 165 (51.9%) completed the process. Nine patients (5.5%) were found to carry a pathogenic gene variant associated with hereditary breast and/or ovarian cancer or Lynch syndrome, Dr. DeFrancesco and colleagues reported.

Patients and providers also were surveyed regarding their experience with the new process. Patients overwhelming noted that they understood the information provided (98.8%), and that they were satisfied with the overall process (97.6%). All 15 providers said that they would continue to use the new process in their practice and most – 13 of 15 – said they found the process thorough and felt comfortable recommending genetic counseling without referral to a genetic counselor (2 were undecided).

“I think that this study really proves the concept that in a community-based practice, we can test our patients,” Dr. DeFrancesco said in an interview.

Myriad Genetics sponsored the study. Dr. DeFrancesco reported no financial conflicts of interest. His coauthors include employees of Myriad Genetics, some with ownership interests.

[email protected]

SOURCE: DeFrancesco, MS et al. ACOG 2018 3K.

AUSTIN, TEXAS – A simple process change helped increase the number of genetic screenings for hereditary cancer risk performed in community ob.gyn. practices, according to Mark S. DeFrancesco, MD, and his associates.

Few community-based ob.gyns. routinely screen their patients for hereditary cancer risks, Dr. DeFrancesco said at the annual meeting of the American College of Obstetricians and Gynecologists, despite ACOG’s position that they are fully trained and qualified to do so. He and his colleagues studied an intervention aimed at streamlining and standardizing genetic assessment in their practice.

A team of physicians, staff, genetic counselors, and process engineers analyzed how hereditary cancer risk assessment was being done at five clinical sites of two community ob.gyn. practices – Dr. DeFrancesco’s practice in Waterbury, Conn., and that of Richard Waldman, MD, in Syracuse, N.Y. – then refined workflows and added tools to create a turnkey process for assessment and screening, Dr. DeFrancesco said.

Under the new process, patients completed a family cancer history in the exam room prior to seeing their physician. Genetic testing was offered to patients who met National Comprehensive Cancer Network (NCCN) guidelines for hereditary/familial high-risk assessment for breast and ovarian cancer (J Natl Compr Canc Netw. 2017 Jan;15[1]:9-20). Those who chose to be tested were able to provide a saliva sample in the office. Counseling was provided to appropriate patients.

The number of patients tested for hereditary risk of breast and ovarian cancer increased dramatically with the new process. During the 8-week period after the intervention, 4% (165) were tested out of 4,107 total patients seen; during the 8 weeks preceding, 1% (43) of 3,882 patients were tested.

Overall, 92.8% (3,811) of patients seen after the intervention provided a family cancer history. Almost a quarter – 23.5% (906) – met NCCN criteria for genetic testing.

A total of 318 patients agreed to undergo genetic testing and 165 (51.9%) completed the process. Nine patients (5.5%) were found to carry a pathogenic gene variant associated with hereditary breast and/or ovarian cancer or Lynch syndrome, Dr. DeFrancesco and colleagues reported.

Patients and providers also were surveyed regarding their experience with the new process. Patients overwhelming noted that they understood the information provided (98.8%), and that they were satisfied with the overall process (97.6%). All 15 providers said that they would continue to use the new process in their practice and most – 13 of 15 – said they found the process thorough and felt comfortable recommending genetic counseling without referral to a genetic counselor (2 were undecided).

“I think that this study really proves the concept that in a community-based practice, we can test our patients,” Dr. DeFrancesco said in an interview.

Myriad Genetics sponsored the study. Dr. DeFrancesco reported no financial conflicts of interest. His coauthors include employees of Myriad Genetics, some with ownership interests.

[email protected]

SOURCE: DeFrancesco, MS et al. ACOG 2018 3K.

AUSTIN, TEXAS – A simple process change helped increase the number of genetic screenings for hereditary cancer risk performed in community ob.gyn. practices, according to Mark S. DeFrancesco, MD, and his associates.

Few community-based ob.gyns. routinely screen their patients for hereditary cancer risks, Dr. DeFrancesco said at the annual meeting of the American College of Obstetricians and Gynecologists, despite ACOG’s position that they are fully trained and qualified to do so. He and his colleagues studied an intervention aimed at streamlining and standardizing genetic assessment in their practice.

A team of physicians, staff, genetic counselors, and process engineers analyzed how hereditary cancer risk assessment was being done at five clinical sites of two community ob.gyn. practices – Dr. DeFrancesco’s practice in Waterbury, Conn., and that of Richard Waldman, MD, in Syracuse, N.Y. – then refined workflows and added tools to create a turnkey process for assessment and screening, Dr. DeFrancesco said.

Under the new process, patients completed a family cancer history in the exam room prior to seeing their physician. Genetic testing was offered to patients who met National Comprehensive Cancer Network (NCCN) guidelines for hereditary/familial high-risk assessment for breast and ovarian cancer (J Natl Compr Canc Netw. 2017 Jan;15[1]:9-20). Those who chose to be tested were able to provide a saliva sample in the office. Counseling was provided to appropriate patients.

The number of patients tested for hereditary risk of breast and ovarian cancer increased dramatically with the new process. During the 8-week period after the intervention, 4% (165) were tested out of 4,107 total patients seen; during the 8 weeks preceding, 1% (43) of 3,882 patients were tested.

Overall, 92.8% (3,811) of patients seen after the intervention provided a family cancer history. Almost a quarter – 23.5% (906) – met NCCN criteria for genetic testing.

A total of 318 patients agreed to undergo genetic testing and 165 (51.9%) completed the process. Nine patients (5.5%) were found to carry a pathogenic gene variant associated with hereditary breast and/or ovarian cancer or Lynch syndrome, Dr. DeFrancesco and colleagues reported.

Patients and providers also were surveyed regarding their experience with the new process. Patients overwhelming noted that they understood the information provided (98.8%), and that they were satisfied with the overall process (97.6%). All 15 providers said that they would continue to use the new process in their practice and most – 13 of 15 – said they found the process thorough and felt comfortable recommending genetic counseling without referral to a genetic counselor (2 were undecided).

“I think that this study really proves the concept that in a community-based practice, we can test our patients,” Dr. DeFrancesco said in an interview.

Myriad Genetics sponsored the study. Dr. DeFrancesco reported no financial conflicts of interest. His coauthors include employees of Myriad Genetics, some with ownership interests.

[email protected]

SOURCE: DeFrancesco, MS et al. ACOG 2018 3K.

AUSTIN, TEXAS – The experiences of one safety net hospital showed the feasibility of delivering prenatal care to low-risk, uninsured women in a prepaid, bundled package.

Women with low-risk pregnancies saw quality of care better than that provided with Medicaid coverage when receiving bundled care. The adjusted odds ratio for predefined adequacy of care was 3.75 for the low-risk bundled care recipients compared with those on Medicaid (P = .015), according to the experience at Grady Memorial Hospital, Atlanta, presented at the annual clinical and scientific sessions of the American College of Obstetricians and Gynecologists.

For hospitals with large numbers of undocumented patients and others who are uninsured but ineligible for Medicaid, considerable cost savings could be realized, said Erin Duncan, MD, who completed the work while in training at Emory University.

“Using data from previous studies, Grady Memorial Hospital could see a savings of over $1 million per year by providing care to its undocumented population,” she and her collaborators wrote in the poster accompanying the presentation.

Dr. Duncan said that since implementation in 2010, about 40% of deliveries at the facility have occurred under the “Grady Healthy Baby” (GHB) bundle.

The one-payment package of bundled prenatal care was developed assuming that most participants would have low-risk pregnancies, said Dr. Duncan, who is currently an ob.gyn. in private practice in the Atlanta area.

To look further into maternal and pregnancy characteristics of GHB participants and compare them with those on Medicaid, Dr. Duncan and her collaborators performed a retrospective cohort study. Examining viable singleton pregnancies delivered at Grady between 2011 and 2014, the investigators compared 100 randomly selected GHB participants with 100 randomly selected Medicaid participants.

Comparing patients receiving care under GHB and Medicaid, Dr. Duncan and her colleagues found that “GHB participants were older, more likely to be Hispanic, and less likely to be black compared to Medicaid recipients (P less than .001 for all,)” they wrote in the poster accompanying the presentation.

Hispanic patients made up 59% of the GHB group, compared with 8% of the Medicaid group, said Dr. Duncan, adding in an interview that over half of Hispanics in the state of Georgia during the study period were undocumented.

Parity was similar between the two groups, as were gestational age at delivery and mode of delivery.

In their analysis, Dr. Duncan and her collaborators looked at both complexity and adequacy of care for the 200 patients studied. They found that there was no significant difference in the number of patients in each care group who remained low risk throughout their pregnancies, transitioned from low risk to high risk, or entered prenatal care with a high risk pregnancy, a circumstance that occurred in about 1 in 10 pregnancies.

For the approximately 50% of patients who remained low risk through their pregnancies, care under the GHB model was significantly more likely to be assessed as adequate throughout pregnancy than for those patients on Medicaid (61.7% vs 35.5%, P = .001).

Patients who became high risk during prenatal care were no more likely to receive adequate care under one model than the other.

For high risk patients, delivery of adequate care happened only under the Medicaid care model. Numbers in this group were small; 7 of 100 GHB and 15 of 100 Medicaid patients entered prenatal care with high risk pregnancies. However, no high risk GHB patients received adequate care, while that standard was met for 80% of the Medicaid patients (P less than .001).

Adequacy of care was assessed using the Kotelchuck index for low-risk pregnancies; this model assumes care is “adequate” when 80% of the number of expected visits were attended by the woman receiving prenatal care. Additionally, care was deemed adequate for high-risk pregnancies if at least 80% of the number of expected ultrasound appointments were attended.

“In the current political climate, this study has implications for all pregnancies that begin as uninsured, regardless of maternal documentation status,” wrote Dr. Duncan and her colleagues.

Dr. Duncan reported no conflicts of interest.

[email protected]

SOURCE: Duncan, E et al. ACOG 2018, Abstract 28C.

AUSTIN, TEXAS – The experiences of one safety net hospital showed the feasibility of delivering prenatal care to low-risk, uninsured women in a prepaid, bundled package.

Women with low-risk pregnancies saw quality of care better than that provided with Medicaid coverage when receiving bundled care. The adjusted odds ratio for predefined adequacy of care was 3.75 for the low-risk bundled care recipients compared with those on Medicaid (P = .015), according to the experience at Grady Memorial Hospital, Atlanta, presented at the annual clinical and scientific sessions of the American College of Obstetricians and Gynecologists.

For hospitals with large numbers of undocumented patients and others who are uninsured but ineligible for Medicaid, considerable cost savings could be realized, said Erin Duncan, MD, who completed the work while in training at Emory University.

“Using data from previous studies, Grady Memorial Hospital could see a savings of over $1 million per year by providing care to its undocumented population,” she and her collaborators wrote in the poster accompanying the presentation.

Dr. Duncan said that since implementation in 2010, about 40% of deliveries at the facility have occurred under the “Grady Healthy Baby” (GHB) bundle.

The one-payment package of bundled prenatal care was developed assuming that most participants would have low-risk pregnancies, said Dr. Duncan, who is currently an ob.gyn. in private practice in the Atlanta area.

To look further into maternal and pregnancy characteristics of GHB participants and compare them with those on Medicaid, Dr. Duncan and her collaborators performed a retrospective cohort study. Examining viable singleton pregnancies delivered at Grady between 2011 and 2014, the investigators compared 100 randomly selected GHB participants with 100 randomly selected Medicaid participants.

Comparing patients receiving care under GHB and Medicaid, Dr. Duncan and her colleagues found that “GHB participants were older, more likely to be Hispanic, and less likely to be black compared to Medicaid recipients (P less than .001 for all,)” they wrote in the poster accompanying the presentation.

Hispanic patients made up 59% of the GHB group, compared with 8% of the Medicaid group, said Dr. Duncan, adding in an interview that over half of Hispanics in the state of Georgia during the study period were undocumented.

Parity was similar between the two groups, as were gestational age at delivery and mode of delivery.

In their analysis, Dr. Duncan and her collaborators looked at both complexity and adequacy of care for the 200 patients studied. They found that there was no significant difference in the number of patients in each care group who remained low risk throughout their pregnancies, transitioned from low risk to high risk, or entered prenatal care with a high risk pregnancy, a circumstance that occurred in about 1 in 10 pregnancies.

For the approximately 50% of patients who remained low risk through their pregnancies, care under the GHB model was significantly more likely to be assessed as adequate throughout pregnancy than for those patients on Medicaid (61.7% vs 35.5%, P = .001).

Patients who became high risk during prenatal care were no more likely to receive adequate care under one model than the other.

For high risk patients, delivery of adequate care happened only under the Medicaid care model. Numbers in this group were small; 7 of 100 GHB and 15 of 100 Medicaid patients entered prenatal care with high risk pregnancies. However, no high risk GHB patients received adequate care, while that standard was met for 80% of the Medicaid patients (P less than .001).

Adequacy of care was assessed using the Kotelchuck index for low-risk pregnancies; this model assumes care is “adequate” when 80% of the number of expected visits were attended by the woman receiving prenatal care. Additionally, care was deemed adequate for high-risk pregnancies if at least 80% of the number of expected ultrasound appointments were attended.

“In the current political climate, this study has implications for all pregnancies that begin as uninsured, regardless of maternal documentation status,” wrote Dr. Duncan and her colleagues.

Dr. Duncan reported no conflicts of interest.

[email protected]

SOURCE: Duncan, E et al. ACOG 2018, Abstract 28C.

AUSTIN, TEXAS – The experiences of one safety net hospital showed the feasibility of delivering prenatal care to low-risk, uninsured women in a prepaid, bundled package.

Women with low-risk pregnancies saw quality of care better than that provided with Medicaid coverage when receiving bundled care. The adjusted odds ratio for predefined adequacy of care was 3.75 for the low-risk bundled care recipients compared with those on Medicaid (P = .015), according to the experience at Grady Memorial Hospital, Atlanta, presented at the annual clinical and scientific sessions of the American College of Obstetricians and Gynecologists.

For hospitals with large numbers of undocumented patients and others who are uninsured but ineligible for Medicaid, considerable cost savings could be realized, said Erin Duncan, MD, who completed the work while in training at Emory University.

“Using data from previous studies, Grady Memorial Hospital could see a savings of over $1 million per year by providing care to its undocumented population,” she and her collaborators wrote in the poster accompanying the presentation.

Dr. Duncan said that since implementation in 2010, about 40% of deliveries at the facility have occurred under the “Grady Healthy Baby” (GHB) bundle.

The one-payment package of bundled prenatal care was developed assuming that most participants would have low-risk pregnancies, said Dr. Duncan, who is currently an ob.gyn. in private practice in the Atlanta area.

To look further into maternal and pregnancy characteristics of GHB participants and compare them with those on Medicaid, Dr. Duncan and her collaborators performed a retrospective cohort study. Examining viable singleton pregnancies delivered at Grady between 2011 and 2014, the investigators compared 100 randomly selected GHB participants with 100 randomly selected Medicaid participants.

Comparing patients receiving care under GHB and Medicaid, Dr. Duncan and her colleagues found that “GHB participants were older, more likely to be Hispanic, and less likely to be black compared to Medicaid recipients (P less than .001 for all,)” they wrote in the poster accompanying the presentation.

Hispanic patients made up 59% of the GHB group, compared with 8% of the Medicaid group, said Dr. Duncan, adding in an interview that over half of Hispanics in the state of Georgia during the study period were undocumented.

Parity was similar between the two groups, as were gestational age at delivery and mode of delivery.

In their analysis, Dr. Duncan and her collaborators looked at both complexity and adequacy of care for the 200 patients studied. They found that there was no significant difference in the number of patients in each care group who remained low risk throughout their pregnancies, transitioned from low risk to high risk, or entered prenatal care with a high risk pregnancy, a circumstance that occurred in about 1 in 10 pregnancies.

For the approximately 50% of patients who remained low risk through their pregnancies, care under the GHB model was significantly more likely to be assessed as adequate throughout pregnancy than for those patients on Medicaid (61.7% vs 35.5%, P = .001).

Patients who became high risk during prenatal care were no more likely to receive adequate care under one model than the other.

For high risk patients, delivery of adequate care happened only under the Medicaid care model. Numbers in this group were small; 7 of 100 GHB and 15 of 100 Medicaid patients entered prenatal care with high risk pregnancies. However, no high risk GHB patients received adequate care, while that standard was met for 80% of the Medicaid patients (P less than .001).

Adequacy of care was assessed using the Kotelchuck index for low-risk pregnancies; this model assumes care is “adequate” when 80% of the number of expected visits were attended by the woman receiving prenatal care. Additionally, care was deemed adequate for high-risk pregnancies if at least 80% of the number of expected ultrasound appointments were attended.

“In the current political climate, this study has implications for all pregnancies that begin as uninsured, regardless of maternal documentation status,” wrote Dr. Duncan and her colleagues.

Dr. Duncan reported no conflicts of interest.

[email protected]

SOURCE: Duncan, E et al. ACOG 2018, Abstract 28C.

WASHINGTON – A bill working its way through the U.S. Congress that would mandate study of the feasibility of a three-digit phone number for suicide prevention and mental health crisis has the potential to establish not only a much-needed national hotline, but more broadly “reboot U.S. crisis care,” Michael F. Hogan, PhD, said at the annual conference of the American Association of Suicidology.

“If we get this legislation passed and some funding, it is a remarkable opportunity to get it right,” said Dr. Hogan, a mental health policy consultant based in Delmar, N.Y., and former commissioner of the New York State Office of Mental Health. A single, “N11,” three-digit phone number for suicide prevention, substance abuse, and mental health crises would “provide a skeleton for a whole new system of crisis centers.”

The pending legislation, the National Suicide Hotline Improvement Act of 2017, “would create parity between brain health and heart health,” Dr. Hogan noted in a video interview. The bill passed the Senate in November 2017, and in late March 2018 had a positive hearing before the Energy and Commerce Committee of the House of Representatives. “The prospects are pretty good,” he said.

Currently, U.S. services are “a mess,” bemoaned Dr. Hogan, who attributed the problem to abandonment of a national program starting in 1981 because of funding changes introduced by the Reagan administration. The centerpiece of the current U.S. system, the National Suicide Prevention Lifeline, receives limited and indirect federal support and relies on coordination among more than 160 local crisis line operations across the country that get no federal funding. Many calls into the Lifeline are, by necessity, answered outside of the locality or even the state from where the call was placed. Inadequacies in the resources available to help people who are suicidal or having other mental health crises have placed the response burden on police departments and emergency departments, “the worst place to go” for mental health care, Dr. Hogan said.

“The primary way around the country to address the problem is cops and EDs; that’s expensive and bad. When you don’t have good crisis services, people go to emergency departments where it’s ‘go upstairs or go home,’ ” he lamented. “We need EMS for the brain; we need one number to call.”

Establishment of a centralized and funded U.S. crisis call system would tie into other measures aimed at transforming national crisis services called for by the Crisis Now program of the National Association of State Mental Health Program Directors, Dr. Hogan said.

Dr. Hogan had no disclosures.

[email protected]

WASHINGTON – A bill working its way through the U.S. Congress that would mandate study of the feasibility of a three-digit phone number for suicide prevention and mental health crisis has the potential to establish not only a much-needed national hotline, but more broadly “reboot U.S. crisis care,” Michael F. Hogan, PhD, said at the annual conference of the American Association of Suicidology.

“If we get this legislation passed and some funding, it is a remarkable opportunity to get it right,” said Dr. Hogan, a mental health policy consultant based in Delmar, N.Y., and former commissioner of the New York State Office of Mental Health. A single, “N11,” three-digit phone number for suicide prevention, substance abuse, and mental health crises would “provide a skeleton for a whole new system of crisis centers.”

The pending legislation, the National Suicide Hotline Improvement Act of 2017, “would create parity between brain health and heart health,” Dr. Hogan noted in a video interview. The bill passed the Senate in November 2017, and in late March 2018 had a positive hearing before the Energy and Commerce Committee of the House of Representatives. “The prospects are pretty good,” he said.

Currently, U.S. services are “a mess,” bemoaned Dr. Hogan, who attributed the problem to abandonment of a national program starting in 1981 because of funding changes introduced by the Reagan administration. The centerpiece of the current U.S. system, the National Suicide Prevention Lifeline, receives limited and indirect federal support and relies on coordination among more than 160 local crisis line operations across the country that get no federal funding. Many calls into the Lifeline are, by necessity, answered outside of the locality or even the state from where the call was placed. Inadequacies in the resources available to help people who are suicidal or having other mental health crises have placed the response burden on police departments and emergency departments, “the worst place to go” for mental health care, Dr. Hogan said.

“The primary way around the country to address the problem is cops and EDs; that’s expensive and bad. When you don’t have good crisis services, people go to emergency departments where it’s ‘go upstairs or go home,’ ” he lamented. “We need EMS for the brain; we need one number to call.”

Establishment of a centralized and funded U.S. crisis call system would tie into other measures aimed at transforming national crisis services called for by the Crisis Now program of the National Association of State Mental Health Program Directors, Dr. Hogan said.

Dr. Hogan had no disclosures.

[email protected]

WASHINGTON – A bill working its way through the U.S. Congress that would mandate study of the feasibility of a three-digit phone number for suicide prevention and mental health crisis has the potential to establish not only a much-needed national hotline, but more broadly “reboot U.S. crisis care,” Michael F. Hogan, PhD, said at the annual conference of the American Association of Suicidology.

“If we get this legislation passed and some funding, it is a remarkable opportunity to get it right,” said Dr. Hogan, a mental health policy consultant based in Delmar, N.Y., and former commissioner of the New York State Office of Mental Health. A single, “N11,” three-digit phone number for suicide prevention, substance abuse, and mental health crises would “provide a skeleton for a whole new system of crisis centers.”

The pending legislation, the National Suicide Hotline Improvement Act of 2017, “would create parity between brain health and heart health,” Dr. Hogan noted in a video interview. The bill passed the Senate in November 2017, and in late March 2018 had a positive hearing before the Energy and Commerce Committee of the House of Representatives. “The prospects are pretty good,” he said.

Currently, U.S. services are “a mess,” bemoaned Dr. Hogan, who attributed the problem to abandonment of a national program starting in 1981 because of funding changes introduced by the Reagan administration. The centerpiece of the current U.S. system, the National Suicide Prevention Lifeline, receives limited and indirect federal support and relies on coordination among more than 160 local crisis line operations across the country that get no federal funding. Many calls into the Lifeline are, by necessity, answered outside of the locality or even the state from where the call was placed. Inadequacies in the resources available to help people who are suicidal or having other mental health crises have placed the response burden on police departments and emergency departments, “the worst place to go” for mental health care, Dr. Hogan said.

“The primary way around the country to address the problem is cops and EDs; that’s expensive and bad. When you don’t have good crisis services, people go to emergency departments where it’s ‘go upstairs or go home,’ ” he lamented. “We need EMS for the brain; we need one number to call.”

Establishment of a centralized and funded U.S. crisis call system would tie into other measures aimed at transforming national crisis services called for by the Crisis Now program of the National Association of State Mental Health Program Directors, Dr. Hogan said.

Dr. Hogan had no disclosures.

[email protected]

DALLAS – Most people rely on physicians, family, and friends to obtain relevant information about cosmetic procedures, but a knowledge gap exists regarding which cosmetic providers are medical doctors.

Those are two key findings from a national survey that set out to assess how consumers research, educate themselves, and choose cosmetic procedures and providers. At the annual conference of the American Society for Laser Medicine and Surgery, study author Adam J. Wulkan, MD, discussed results from the 20-item survey, which was based on responses from 323 people who have obtained or have considered obtaining a cosmetic procedure such as laser hair removal.

Dr. Wulkan is a dermatologist at Massachusetts General Hospital, Boston. He reported having no financial disclosures. Study coauthor Mathew Avram, MD, serves on the medical advisory board of Sciton and on the scientific advisory boards of Sienna Biopharmaceuticals, Cytrellis, and Allergan. He also is consultant for Merz Aesthetics, Allergan, Soliton, Invasix, and Revance, and has intellectual property with Cytrellis. He also holds stock options with Cytrellis, Invasix, and Zalea.

[email protected]

DALLAS – Most people rely on physicians, family, and friends to obtain relevant information about cosmetic procedures, but a knowledge gap exists regarding which cosmetic providers are medical doctors.

Those are two key findings from a national survey that set out to assess how consumers research, educate themselves, and choose cosmetic procedures and providers. At the annual conference of the American Society for Laser Medicine and Surgery, study author Adam J. Wulkan, MD, discussed results from the 20-item survey, which was based on responses from 323 people who have obtained or have considered obtaining a cosmetic procedure such as laser hair removal.

Dr. Wulkan is a dermatologist at Massachusetts General Hospital, Boston. He reported having no financial disclosures. Study coauthor Mathew Avram, MD, serves on the medical advisory board of Sciton and on the scientific advisory boards of Sienna Biopharmaceuticals, Cytrellis, and Allergan. He also is consultant for Merz Aesthetics, Allergan, Soliton, Invasix, and Revance, and has intellectual property with Cytrellis. He also holds stock options with Cytrellis, Invasix, and Zalea.

[email protected]

DALLAS – Most people rely on physicians, family, and friends to obtain relevant information about cosmetic procedures, but a knowledge gap exists regarding which cosmetic providers are medical doctors.

Those are two key findings from a national survey that set out to assess how consumers research, educate themselves, and choose cosmetic procedures and providers. At the annual conference of the American Society for Laser Medicine and Surgery, study author Adam J. Wulkan, MD, discussed results from the 20-item survey, which was based on responses from 323 people who have obtained or have considered obtaining a cosmetic procedure such as laser hair removal.

Dr. Wulkan is a dermatologist at Massachusetts General Hospital, Boston. He reported having no financial disclosures. Study coauthor Mathew Avram, MD, serves on the medical advisory board of Sciton and on the scientific advisory boards of Sienna Biopharmaceuticals, Cytrellis, and Allergan. He also is consultant for Merz Aesthetics, Allergan, Soliton, Invasix, and Revance, and has intellectual property with Cytrellis. He also holds stock options with Cytrellis, Invasix, and Zalea.

[email protected]

LOS ANGELES – A reorganization of stroke systems of care is needed to meet the rising demand for screening, triaging, and treating acute ischemic stroke patients who may benefit from mechanical thrombectomy, according to Lawrence Wechsler, MD.

In a video interview at the annual meeting of the American Academy of Neurology, Dr. Wechsler described steps being taken at the University of Pittsburgh Medical Center’s comprehensive stroke center to handle the additional workload.

UPMC conducts telemedicine acute stroke evaluations of patients at community hospitals’ primary stroke centers in the greater Pittsburgh area to make sure that only the cases that require mechanical thrombectomy are transferred to them for specialized care, while also continuing to see nontransferred patients via telemedicine for follow-up, said Dr. Wechsler, chair of the department of neurology at UPMC and founder of its Stroke Institute and telestroke network.

This sort of solution may be more feasible and practical for comprehensive stroke centers to implement in order to manage the number of cases, instead of expanding neurology residencies, capping stroke services, adding a nonteaching service, adding advanced practice providers, or increasing the case loads of vascular neurology fellows and attending neurologists, he said.

[email protected]

LOS ANGELES – A reorganization of stroke systems of care is needed to meet the rising demand for screening, triaging, and treating acute ischemic stroke patients who may benefit from mechanical thrombectomy, according to Lawrence Wechsler, MD.

In a video interview at the annual meeting of the American Academy of Neurology, Dr. Wechsler described steps being taken at the University of Pittsburgh Medical Center’s comprehensive stroke center to handle the additional workload.

UPMC conducts telemedicine acute stroke evaluations of patients at community hospitals’ primary stroke centers in the greater Pittsburgh area to make sure that only the cases that require mechanical thrombectomy are transferred to them for specialized care, while also continuing to see nontransferred patients via telemedicine for follow-up, said Dr. Wechsler, chair of the department of neurology at UPMC and founder of its Stroke Institute and telestroke network.

This sort of solution may be more feasible and practical for comprehensive stroke centers to implement in order to manage the number of cases, instead of expanding neurology residencies, capping stroke services, adding a nonteaching service, adding advanced practice providers, or increasing the case loads of vascular neurology fellows and attending neurologists, he said.

[email protected]

LOS ANGELES – A reorganization of stroke systems of care is needed to meet the rising demand for screening, triaging, and treating acute ischemic stroke patients who may benefit from mechanical thrombectomy, according to Lawrence Wechsler, MD.

In a video interview at the annual meeting of the American Academy of Neurology, Dr. Wechsler described steps being taken at the University of Pittsburgh Medical Center’s comprehensive stroke center to handle the additional workload.

UPMC conducts telemedicine acute stroke evaluations of patients at community hospitals’ primary stroke centers in the greater Pittsburgh area to make sure that only the cases that require mechanical thrombectomy are transferred to them for specialized care, while also continuing to see nontransferred patients via telemedicine for follow-up, said Dr. Wechsler, chair of the department of neurology at UPMC and founder of its Stroke Institute and telestroke network.

This sort of solution may be more feasible and practical for comprehensive stroke centers to implement in order to manage the number of cases, instead of expanding neurology residencies, capping stroke services, adding a nonteaching service, adding advanced practice providers, or increasing the case loads of vascular neurology fellows and attending neurologists, he said.

[email protected]

LOS ANGELES – New results from phase 3 randomized trials of the prophylactic migraine treatment eptinezumab show significant reductions in the number of monthly migraine headache days experienced by patients with chronic or frequent episodic migraines.

Eptinezumab, an experimental monoclonal antibody delivered by intravenous infusion, is one of several antimigraine agents in development that targets calcitonin gene-related peptide (CGRP), a key mediator of migraine.

At the annual meeting of the American Academy of Neurology, Richard Lipton, MD, of Albert Einstein College of Medicine in New York, presented results from PROMISE 2, a phase 3 randomized, placebo-controlled trial of eptinezumab in patients with chronic migraine, or 15 or more days with migraine per month.

The investigators randomized 1,072 patients to quarterly IV infusions of eptinezumab 100 or 300 mg or placebo.

The vast majority of patients in the study were women, (86%-90% across groups) with a mean age of about 40 years. Patients reported 11-12 years of chronic migraine and about 16 migraine days per month at baseline, Dr. Lipton told the conference, reflecting a high level of disability in the cohort.

The primary endpoint of the study was mean change in monthly migraine days from baseline through week 12. Dr. Lipton reported that the placebo group saw a 5.6-day reduction in migraine, while the 100-mg group saw a 7.7-day reduction, and patients receiving the 300-mg dose saw an 8.2-day reduction during the first 12 weeks after injection (P less than .0001 for both).

One-third of patients receiving the highest dose saw a 75% or greater reduction in monthly migraine days by week 12, “a relatively high bar” to meet, Dr. Lipton said. Some 61% of patients on the high dose saw a reduction of 50% or more in the same time period.

SOURCE: Saper J et al. AAN 2018, Abstract S20.001 and Lipton R et al. AAN 2018, Clinical Trials Plenary Session Abstract.

LOS ANGELES – New results from phase 3 randomized trials of the prophylactic migraine treatment eptinezumab show significant reductions in the number of monthly migraine headache days experienced by patients with chronic or frequent episodic migraines.

Eptinezumab, an experimental monoclonal antibody delivered by intravenous infusion, is one of several antimigraine agents in development that targets calcitonin gene-related peptide (CGRP), a key mediator of migraine.

At the annual meeting of the American Academy of Neurology, Richard Lipton, MD, of Albert Einstein College of Medicine in New York, presented results from PROMISE 2, a phase 3 randomized, placebo-controlled trial of eptinezumab in patients with chronic migraine, or 15 or more days with migraine per month.

The investigators randomized 1,072 patients to quarterly IV infusions of eptinezumab 100 or 300 mg or placebo.

The vast majority of patients in the study were women, (86%-90% across groups) with a mean age of about 40 years. Patients reported 11-12 years of chronic migraine and about 16 migraine days per month at baseline, Dr. Lipton told the conference, reflecting a high level of disability in the cohort.

The primary endpoint of the study was mean change in monthly migraine days from baseline through week 12. Dr. Lipton reported that the placebo group saw a 5.6-day reduction in migraine, while the 100-mg group saw a 7.7-day reduction, and patients receiving the 300-mg dose saw an 8.2-day reduction during the first 12 weeks after injection (P less than .0001 for both).

One-third of patients receiving the highest dose saw a 75% or greater reduction in monthly migraine days by week 12, “a relatively high bar” to meet, Dr. Lipton said. Some 61% of patients on the high dose saw a reduction of 50% or more in the same time period.

SOURCE: Saper J et al. AAN 2018, Abstract S20.001 and Lipton R et al. AAN 2018, Clinical Trials Plenary Session Abstract.

LOS ANGELES – New results from phase 3 randomized trials of the prophylactic migraine treatment eptinezumab show significant reductions in the number of monthly migraine headache days experienced by patients with chronic or frequent episodic migraines.

Eptinezumab, an experimental monoclonal antibody delivered by intravenous infusion, is one of several antimigraine agents in development that targets calcitonin gene-related peptide (CGRP), a key mediator of migraine.

At the annual meeting of the American Academy of Neurology, Richard Lipton, MD, of Albert Einstein College of Medicine in New York, presented results from PROMISE 2, a phase 3 randomized, placebo-controlled trial of eptinezumab in patients with chronic migraine, or 15 or more days with migraine per month.

The investigators randomized 1,072 patients to quarterly IV infusions of eptinezumab 100 or 300 mg or placebo.

The vast majority of patients in the study were women, (86%-90% across groups) with a mean age of about 40 years. Patients reported 11-12 years of chronic migraine and about 16 migraine days per month at baseline, Dr. Lipton told the conference, reflecting a high level of disability in the cohort.

The primary endpoint of the study was mean change in monthly migraine days from baseline through week 12. Dr. Lipton reported that the placebo group saw a 5.6-day reduction in migraine, while the 100-mg group saw a 7.7-day reduction, and patients receiving the 300-mg dose saw an 8.2-day reduction during the first 12 weeks after injection (P less than .0001 for both).

One-third of patients receiving the highest dose saw a 75% or greater reduction in monthly migraine days by week 12, “a relatively high bar” to meet, Dr. Lipton said. Some 61% of patients on the high dose saw a reduction of 50% or more in the same time period.

SOURCE: Saper J et al. AAN 2018, Abstract S20.001 and Lipton R et al. AAN 2018, Clinical Trials Plenary Session Abstract.

LOS ANGELES – Neurologists can play an important role in helping patients gain access to high-cost, breakthrough drugs, while at the same time guiding patients to lower-cost options whenever possible, speakers said at the annual meeting of the American Academy of Neurology.

The use of the Orphan Drug approval pathway established in 1983 has gained a great deal of steam for rare neurologic diseases in recent years with the approval of a number of drugs, such as nusinersen (Spinraza) for spinal muscular atrophy, eteplirsen (Exondys 51) for Duchenne muscular dystrophy, and edaravone (Radicava) for amyotrophic lateral sclerosis, said Nicholas Johnson, MD, a pediatric neuromuscular disease specialist at the University of Utah, Salt Lake City.

But given that only 2% of U.S. physicians are neurologists, yet 18% of rare diseases are neurologic and 11% of drugs in development overall are for neurologic diseases, there are a great deal of challenges arising for neurologists in getting access to these new high-priced drugs for their patients, said Dr. Johnson, who leads the AAN’s Neurology Drug Pricing Task Force and is also chair of the AAN Government Relations Committee.

These challenges range from increased administrative burden on staff, getting insurance approval, finding administration sites, and the ability to perform special patient assessments, he said in an interview.

Dr. Nicholas Johnson’s interview:
 

Dr. Brian Callaghan’s interview:
 

Many high-priced drugs commonly prescribed for chronic neurologic conditions and diagnostic tests also have high out-of-pocket costs for patients, but it is remarkably hard even for well-informed experts to find the actual costs that patients will pay out of pocket for such drugs and tests, according to Brian Callaghan, MD, a neuromuscular disease specialist at the University of Michigan, Ann Arbor.

[email protected]

LOS ANGELES – Neurologists can play an important role in helping patients gain access to high-cost, breakthrough drugs, while at the same time guiding patients to lower-cost options whenever possible, speakers said at the annual meeting of the American Academy of Neurology.

The use of the Orphan Drug approval pathway established in 1983 has gained a great deal of steam for rare neurologic diseases in recent years with the approval of a number of drugs, such as nusinersen (Spinraza) for spinal muscular atrophy, eteplirsen (Exondys 51) for Duchenne muscular dystrophy, and edaravone (Radicava) for amyotrophic lateral sclerosis, said Nicholas Johnson, MD, a pediatric neuromuscular disease specialist at the University of Utah, Salt Lake City.

But given that only 2% of U.S. physicians are neurologists, yet 18% of rare diseases are neurologic and 11% of drugs in development overall are for neurologic diseases, there are a great deal of challenges arising for neurologists in getting access to these new high-priced drugs for their patients, said Dr. Johnson, who leads the AAN’s Neurology Drug Pricing Task Force and is also chair of the AAN Government Relations Committee.

These challenges range from increased administrative burden on staff, getting insurance approval, finding administration sites, and the ability to perform special patient assessments, he said in an interview.

Dr. Nicholas Johnson’s interview:
 

Dr. Brian Callaghan’s interview:
 

Many high-priced drugs commonly prescribed for chronic neurologic conditions and diagnostic tests also have high out-of-pocket costs for patients, but it is remarkably hard even for well-informed experts to find the actual costs that patients will pay out of pocket for such drugs and tests, according to Brian Callaghan, MD, a neuromuscular disease specialist at the University of Michigan, Ann Arbor.

[email protected]

LOS ANGELES – Neurologists can play an important role in helping patients gain access to high-cost, breakthrough drugs, while at the same time guiding patients to lower-cost options whenever possible, speakers said at the annual meeting of the American Academy of Neurology.

The use of the Orphan Drug approval pathway established in 1983 has gained a great deal of steam for rare neurologic diseases in recent years with the approval of a number of drugs, such as nusinersen (Spinraza) for spinal muscular atrophy, eteplirsen (Exondys 51) for Duchenne muscular dystrophy, and edaravone (Radicava) for amyotrophic lateral sclerosis, said Nicholas Johnson, MD, a pediatric neuromuscular disease specialist at the University of Utah, Salt Lake City.

But given that only 2% of U.S. physicians are neurologists, yet 18% of rare diseases are neurologic and 11% of drugs in development overall are for neurologic diseases, there are a great deal of challenges arising for neurologists in getting access to these new high-priced drugs for their patients, said Dr. Johnson, who leads the AAN’s Neurology Drug Pricing Task Force and is also chair of the AAN Government Relations Committee.

These challenges range from increased administrative burden on staff, getting insurance approval, finding administration sites, and the ability to perform special patient assessments, he said in an interview.

Dr. Nicholas Johnson’s interview:
 

Dr. Brian Callaghan’s interview:
 

Many high-priced drugs commonly prescribed for chronic neurologic conditions and diagnostic tests also have high out-of-pocket costs for patients, but it is remarkably hard even for well-informed experts to find the actual costs that patients will pay out of pocket for such drugs and tests, according to Brian Callaghan, MD, a neuromuscular disease specialist at the University of Michigan, Ann Arbor.

[email protected]

LOS ANGELES – A new clinical guideline for adults with multiple sclerosis is designed to help clinicians navigate an increasingly complex landscape of treatment options, its authors say.

The new American Academy of Neurology (AAN) guideline includes some 30 recommendations related to starting a disease-modifying therapy (DMT), switching therapies, or stopping treatment. The guideline, presented April 23 at the AAN annual meeting and published online simultaneously in Neurology, is the first full MS practice guideline issued by AAN since 2002, when only a handful of medications were licensed for use in MS.

The new guideline does not present a hierarchy of DMTs to start but instead weighs evidence for 21 medications or formulations, including 8 used off label, with the idea that clinicians will tailor their choices by considering patient needs, preferences, potential adverse affects, cost of medicines, comorbidities (including depression), and likelihood of adherence, among other factors addressed.

“It’s a dramatically different landscape of the choices clinicians have,” guideline lead author Alexander Rae-Grant, MD, of the Cleveland Clinic, said at a press conference announcing the guideline.

The guideline encourages clinicians and patients “to have a detailed discussion of the risks and benefits of different therapies,” guideline coauthor Ruth Ann Marrie, MD, PhD, of the University of Manitoba, Winnipeg, said at the press conference. “You need to have that informed ... discussion to share decision making – the guideline provides information for each provider-patient dyad to make decisions specific to that patient.”

The guideline incorporates findings from nearly 50 randomized trials, although few of these were head-to-head comparisons of therapies. The authors graded evidence for each DMT as compared with placebo or other DMTs in lowering relapse rate, taking into consideration study design and size. The guideline reflects changes to diagnostic criteria made in 2010, and a classification scheme issued in 2014 for MS subtypes, both of which have complicated the extension of clinical trial findings to some patient groups. It stresses early treatment, recommending that clinicians start DMTs in people with a single clinical demyelinating event and two or more brain lesions characteristic of MS.

Several drugs used off label in MS, including azathioprine and cladribine, were included in the evidence review, with cladribine described as a cost-effective option for patients without the resources to obtain approved agents.

SOURCE: Rae-Grant A et al. Neurology. 2018;90:777-88.

LOS ANGELES – A new clinical guideline for adults with multiple sclerosis is designed to help clinicians navigate an increasingly complex landscape of treatment options, its authors say.

The new American Academy of Neurology (AAN) guideline includes some 30 recommendations related to starting a disease-modifying therapy (DMT), switching therapies, or stopping treatment. The guideline, presented April 23 at the AAN annual meeting and published online simultaneously in Neurology, is the first full MS practice guideline issued by AAN since 2002, when only a handful of medications were licensed for use in MS.

The new guideline does not present a hierarchy of DMTs to start but instead weighs evidence for 21 medications or formulations, including 8 used off label, with the idea that clinicians will tailor their choices by considering patient needs, preferences, potential adverse affects, cost of medicines, comorbidities (including depression), and likelihood of adherence, among other factors addressed.

“It’s a dramatically different landscape of the choices clinicians have,” guideline lead author Alexander Rae-Grant, MD, of the Cleveland Clinic, said at a press conference announcing the guideline.

The guideline encourages clinicians and patients “to have a detailed discussion of the risks and benefits of different therapies,” guideline coauthor Ruth Ann Marrie, MD, PhD, of the University of Manitoba, Winnipeg, said at the press conference. “You need to have that informed ... discussion to share decision making – the guideline provides information for each provider-patient dyad to make decisions specific to that patient.”

The guideline incorporates findings from nearly 50 randomized trials, although few of these were head-to-head comparisons of therapies. The authors graded evidence for each DMT as compared with placebo or other DMTs in lowering relapse rate, taking into consideration study design and size. The guideline reflects changes to diagnostic criteria made in 2010, and a classification scheme issued in 2014 for MS subtypes, both of which have complicated the extension of clinical trial findings to some patient groups. It stresses early treatment, recommending that clinicians start DMTs in people with a single clinical demyelinating event and two or more brain lesions characteristic of MS.

Several drugs used off label in MS, including azathioprine and cladribine, were included in the evidence review, with cladribine described as a cost-effective option for patients without the resources to obtain approved agents.

SOURCE: Rae-Grant A et al. Neurology. 2018;90:777-88.

LOS ANGELES – A new clinical guideline for adults with multiple sclerosis is designed to help clinicians navigate an increasingly complex landscape of treatment options, its authors say.

The new American Academy of Neurology (AAN) guideline includes some 30 recommendations related to starting a disease-modifying therapy (DMT), switching therapies, or stopping treatment. The guideline, presented April 23 at the AAN annual meeting and published online simultaneously in Neurology, is the first full MS practice guideline issued by AAN since 2002, when only a handful of medications were licensed for use in MS.

The new guideline does not present a hierarchy of DMTs to start but instead weighs evidence for 21 medications or formulations, including 8 used off label, with the idea that clinicians will tailor their choices by considering patient needs, preferences, potential adverse affects, cost of medicines, comorbidities (including depression), and likelihood of adherence, among other factors addressed.

“It’s a dramatically different landscape of the choices clinicians have,” guideline lead author Alexander Rae-Grant, MD, of the Cleveland Clinic, said at a press conference announcing the guideline.

The guideline encourages clinicians and patients “to have a detailed discussion of the risks and benefits of different therapies,” guideline coauthor Ruth Ann Marrie, MD, PhD, of the University of Manitoba, Winnipeg, said at the press conference. “You need to have that informed ... discussion to share decision making – the guideline provides information for each provider-patient dyad to make decisions specific to that patient.”

The guideline incorporates findings from nearly 50 randomized trials, although few of these were head-to-head comparisons of therapies. The authors graded evidence for each DMT as compared with placebo or other DMTs in lowering relapse rate, taking into consideration study design and size. The guideline reflects changes to diagnostic criteria made in 2010, and a classification scheme issued in 2014 for MS subtypes, both of which have complicated the extension of clinical trial findings to some patient groups. It stresses early treatment, recommending that clinicians start DMTs in people with a single clinical demyelinating event and two or more brain lesions characteristic of MS.

Several drugs used off label in MS, including azathioprine and cladribine, were included in the evidence review, with cladribine described as a cost-effective option for patients without the resources to obtain approved agents.

SOURCE: Rae-Grant A et al. Neurology. 2018;90:777-88.

LOS ANGELES – Results from the first-in-human trial of the investigational antisense oligonucleotide therapy IONIS-HTTRx showed strong dose-dependent reductions in the toxic huntingtin protein in Huntington’s disease patients’ cerebrospinal fluid when compared with placebo.

An exploratory analysis also showed signals of clinical improvement among patients receiving the therapy, the study’s lead author, Sarah Tabrizi, MD, PhD, of University College London, reported at the annual meeting of the American Academy of Neurology.

The findings represent a potential breakthrough in the way Huntington’s – and possibly other neurodegenerative diseases – are treated, said Dr. Tabrizi of University College London. The intrathecally-delivered drug binds to mutant huntingtin protein mRNA to reduce the level of the toxic protein being made.

In the phase 1/2a trial, Dr. Tabrizi and her colleagues enrolled 46 patients with early-stage Huntington’s disease and randomized them to four doses of IONIS-HTTRx or placebo. Patients received four monthly injections of the study drug into the cerebrospinal fluid followed by a 4-month untreated follow-up period. IONIS-HTTRx was delivered in five ascending-dose cohorts.

IONIS-HTTRx was safe and well tolerated and all patients completed the study, Dr. Tabrizi reported. “Our results show that we had significant lowering of the toxic mutant huntingtin protein in the spinal fluid of the patients,” she said, noting that patients receiving the highest doses saw 40%-60% lowering of the protein, which preclinical work suggests correlates to reductions of the protein in the brain.

Dr. Tabrizi stressed that the main outcome measures in the study were reduction of the mutant protein in the CSF and safety and tolerance of the study drug. The investigators did not expect to see clinical measures to change in such a short, small study, she said.

“But then when we looked more carefully at the data with exploratory analysis we found a link between lowering of CSF mutant huntingtin and improvement in total motor score, which is a measure of neurological function, and also, improvement in the Symbol Digit Modalities Test.”

The clinical results are exploratory and require confirmation in larger studies, Dr. Tabrizi stressed.

The study was supported by Ionis Pharmaceuticals. Roche will develop the drug in further clinical trials.

SOURCE: Tabrizi S et al. AAN 2018 abstract CT.002.

LOS ANGELES – Results from the first-in-human trial of the investigational antisense oligonucleotide therapy IONIS-HTTRx showed strong dose-dependent reductions in the toxic huntingtin protein in Huntington’s disease patients’ cerebrospinal fluid when compared with placebo.

An exploratory analysis also showed signals of clinical improvement among patients receiving the therapy, the study’s lead author, Sarah Tabrizi, MD, PhD, of University College London, reported at the annual meeting of the American Academy of Neurology.

The findings represent a potential breakthrough in the way Huntington’s – and possibly other neurodegenerative diseases – are treated, said Dr. Tabrizi of University College London. The intrathecally-delivered drug binds to mutant huntingtin protein mRNA to reduce the level of the toxic protein being made.

In the phase 1/2a trial, Dr. Tabrizi and her colleagues enrolled 46 patients with early-stage Huntington’s disease and randomized them to four doses of IONIS-HTTRx or placebo. Patients received four monthly injections of the study drug into the cerebrospinal fluid followed by a 4-month untreated follow-up period. IONIS-HTTRx was delivered in five ascending-dose cohorts.

IONIS-HTTRx was safe and well tolerated and all patients completed the study, Dr. Tabrizi reported. “Our results show that we had significant lowering of the toxic mutant huntingtin protein in the spinal fluid of the patients,” she said, noting that patients receiving the highest doses saw 40%-60% lowering of the protein, which preclinical work suggests correlates to reductions of the protein in the brain.

Dr. Tabrizi stressed that the main outcome measures in the study were reduction of the mutant protein in the CSF and safety and tolerance of the study drug. The investigators did not expect to see clinical measures to change in such a short, small study, she said.

“But then when we looked more carefully at the data with exploratory analysis we found a link between lowering of CSF mutant huntingtin and improvement in total motor score, which is a measure of neurological function, and also, improvement in the Symbol Digit Modalities Test.”

The clinical results are exploratory and require confirmation in larger studies, Dr. Tabrizi stressed.

The study was supported by Ionis Pharmaceuticals. Roche will develop the drug in further clinical trials.

SOURCE: Tabrizi S et al. AAN 2018 abstract CT.002.

LOS ANGELES – Results from the first-in-human trial of the investigational antisense oligonucleotide therapy IONIS-HTTRx showed strong dose-dependent reductions in the toxic huntingtin protein in Huntington’s disease patients’ cerebrospinal fluid when compared with placebo.

An exploratory analysis also showed signals of clinical improvement among patients receiving the therapy, the study’s lead author, Sarah Tabrizi, MD, PhD, of University College London, reported at the annual meeting of the American Academy of Neurology.

The findings represent a potential breakthrough in the way Huntington’s – and possibly other neurodegenerative diseases – are treated, said Dr. Tabrizi of University College London. The intrathecally-delivered drug binds to mutant huntingtin protein mRNA to reduce the level of the toxic protein being made.

In the phase 1/2a trial, Dr. Tabrizi and her colleagues enrolled 46 patients with early-stage Huntington’s disease and randomized them to four doses of IONIS-HTTRx or placebo. Patients received four monthly injections of the study drug into the cerebrospinal fluid followed by a 4-month untreated follow-up period. IONIS-HTTRx was delivered in five ascending-dose cohorts.

IONIS-HTTRx was safe and well tolerated and all patients completed the study, Dr. Tabrizi reported. “Our results show that we had significant lowering of the toxic mutant huntingtin protein in the spinal fluid of the patients,” she said, noting that patients receiving the highest doses saw 40%-60% lowering of the protein, which preclinical work suggests correlates to reductions of the protein in the brain.

Dr. Tabrizi stressed that the main outcome measures in the study were reduction of the mutant protein in the CSF and safety and tolerance of the study drug. The investigators did not expect to see clinical measures to change in such a short, small study, she said.

“But then when we looked more carefully at the data with exploratory analysis we found a link between lowering of CSF mutant huntingtin and improvement in total motor score, which is a measure of neurological function, and also, improvement in the Symbol Digit Modalities Test.”

The clinical results are exploratory and require confirmation in larger studies, Dr. Tabrizi stressed.

The study was supported by Ionis Pharmaceuticals. Roche will develop the drug in further clinical trials.

SOURCE: Tabrizi S et al. AAN 2018 abstract CT.002.

LOS ANGELES – The investigational oral calcitonin gene-related peptide (CGRP) blocker ubrogepant proved superior to placebo in treating a single, acute, moderate to severe migraine attack in the phase 3 ACHIEVE I trial.

Blocking the function of CGRP when it is pathologically released during a migraine attack is the mechanism of a bevy of antagonists under development for treating migraine, and the ACHIEVE I trial results are the first to be reported from a phase 3 trial of an oral small molecule to treat single migraine attacks rather than prevent attacks with monoclonal antibodies targeting CGRP or its receptor, according to the lead author of ACHIEVE I, Joel M. Trugman, MD, director of clinical development for Allergan.

Dr. Trugman and his colleagues recruited 1,672 adult patients (88% female, mean age 41) with a history of migraine with or without aura, and randomized them to placebo, ubrogepant 50 mg, or ubrogepant 100 mg. Patients were instructed to treat a single migraine attack of moderate to severe pain intensity and record symptoms, such as pain, light sensitivity, and sound sensitivity, before and after taking the medication.

“The paradigm for this type of trial is to treat a single, well characterized migraine attack in a large population of patients,” Dr. Trugman said in a press conference at the annual meeting of the American Academy of Neurology.

At 2 hours after dosing, the percentage of ubrogepant-treated patients achieving freedom from pain was significantly greater than the percentage of those treated with placebo (50 mg: 19.2%, P = .0023; 100 mg: 21.2%, P = .0003; placebo: 11.8%).

The percentage of ubrogepant-treated patients achieving absence of their most bothersome symptom was also significantly greater than that of placebo (50 mg: 38.6%, P = .0023; 100 mg: 37.7%, P = .0023; placebo: 27.8%).

Adverse events in the ubrogepant groups were similar to those of placebo, the most reported to be nausea, somnolence, and dry mouth.

Several authors are employees of Allergan, which sponsored the study.

SOURCE: Trugman J et al. AAN 2018 emerging science abstract 008.

LOS ANGELES – The investigational oral calcitonin gene-related peptide (CGRP) blocker ubrogepant proved superior to placebo in treating a single, acute, moderate to severe migraine attack in the phase 3 ACHIEVE I trial.

Blocking the function of CGRP when it is pathologically released during a migraine attack is the mechanism of a bevy of antagonists under development for treating migraine, and the ACHIEVE I trial results are the first to be reported from a phase 3 trial of an oral small molecule to treat single migraine attacks rather than prevent attacks with monoclonal antibodies targeting CGRP or its receptor, according to the lead author of ACHIEVE I, Joel M. Trugman, MD, director of clinical development for Allergan.

Dr. Trugman and his colleagues recruited 1,672 adult patients (88% female, mean age 41) with a history of migraine with or without aura, and randomized them to placebo, ubrogepant 50 mg, or ubrogepant 100 mg. Patients were instructed to treat a single migraine attack of moderate to severe pain intensity and record symptoms, such as pain, light sensitivity, and sound sensitivity, before and after taking the medication.

“The paradigm for this type of trial is to treat a single, well characterized migraine attack in a large population of patients,” Dr. Trugman said in a press conference at the annual meeting of the American Academy of Neurology.

At 2 hours after dosing, the percentage of ubrogepant-treated patients achieving freedom from pain was significantly greater than the percentage of those treated with placebo (50 mg: 19.2%, P = .0023; 100 mg: 21.2%, P = .0003; placebo: 11.8%).

The percentage of ubrogepant-treated patients achieving absence of their most bothersome symptom was also significantly greater than that of placebo (50 mg: 38.6%, P = .0023; 100 mg: 37.7%, P = .0023; placebo: 27.8%).

Adverse events in the ubrogepant groups were similar to those of placebo, the most reported to be nausea, somnolence, and dry mouth.

Several authors are employees of Allergan, which sponsored the study.

SOURCE: Trugman J et al. AAN 2018 emerging science abstract 008.

LOS ANGELES – The investigational oral calcitonin gene-related peptide (CGRP) blocker ubrogepant proved superior to placebo in treating a single, acute, moderate to severe migraine attack in the phase 3 ACHIEVE I trial.

Blocking the function of CGRP when it is pathologically released during a migraine attack is the mechanism of a bevy of antagonists under development for treating migraine, and the ACHIEVE I trial results are the first to be reported from a phase 3 trial of an oral small molecule to treat single migraine attacks rather than prevent attacks with monoclonal antibodies targeting CGRP or its receptor, according to the lead author of ACHIEVE I, Joel M. Trugman, MD, director of clinical development for Allergan.

Dr. Trugman and his colleagues recruited 1,672 adult patients (88% female, mean age 41) with a history of migraine with or without aura, and randomized them to placebo, ubrogepant 50 mg, or ubrogepant 100 mg. Patients were instructed to treat a single migraine attack of moderate to severe pain intensity and record symptoms, such as pain, light sensitivity, and sound sensitivity, before and after taking the medication.

“The paradigm for this type of trial is to treat a single, well characterized migraine attack in a large population of patients,” Dr. Trugman said in a press conference at the annual meeting of the American Academy of Neurology.

At 2 hours after dosing, the percentage of ubrogepant-treated patients achieving freedom from pain was significantly greater than the percentage of those treated with placebo (50 mg: 19.2%, P = .0023; 100 mg: 21.2%, P = .0003; placebo: 11.8%).

The percentage of ubrogepant-treated patients achieving absence of their most bothersome symptom was also significantly greater than that of placebo (50 mg: 38.6%, P = .0023; 100 mg: 37.7%, P = .0023; placebo: 27.8%).

Adverse events in the ubrogepant groups were similar to those of placebo, the most reported to be nausea, somnolence, and dry mouth.

Several authors are employees of Allergan, which sponsored the study.

SOURCE: Trugman J et al. AAN 2018 emerging science abstract 008.