Children with chronic headache and migraine who are severely functionally impaired demonstrated linear improvement in pain‐specific patient‐reported outcomes over time, according to a recent study that aimed to evaluate the trajectory of recovery for children undergoing intensive interdisciplinary pain rehabilitation treatment (IIPT). A retrospective analysis was conducted of patient‐reported outcomes in a clinical database of 135 children (mean age 15.2 [SD=2.2] and 74% female) admitted to an IIPT program between the years 2008 and 2014. Available data across 5 separate time points (up to 1‐year post‐discharge) were reviewed. Researchers found:

• A statistically significant improvement was noted in pain‐specific measures of functioning, including daily functioning, emotional functioning, family functioning, and school absences over a 12‐month period.
• A more general measure of quality of life improved during the program, based upon child and parent reports, although these gains did not continue to improve post‐discharge.
• As expected, although children did not report a reduction in pain during rehabilitation, they did report a significant drop in perceived pain in the 12 months following discharge from the program.

Benore E, Webster EE, Wang L, Banez G. Longitudinal analysis of patient‐reported outcomes from an interdisciplinary pediatric pain rehabilitation program for children with chronic migraine and headache. [Published online ahead of print August 23, 2018]. Headache. doi:10.1111/head.13389.

Children with chronic headache and migraine who are severely functionally impaired demonstrated linear improvement in pain‐specific patient‐reported outcomes over time, according to a recent study that aimed to evaluate the trajectory of recovery for children undergoing intensive interdisciplinary pain rehabilitation treatment (IIPT). A retrospective analysis was conducted of patient‐reported outcomes in a clinical database of 135 children (mean age 15.2 [SD=2.2] and 74% female) admitted to an IIPT program between the years 2008 and 2014. Available data across 5 separate time points (up to 1‐year post‐discharge) were reviewed. Researchers found:

• A statistically significant improvement was noted in pain‐specific measures of functioning, including daily functioning, emotional functioning, family functioning, and school absences over a 12‐month period.
• A more general measure of quality of life improved during the program, based upon child and parent reports, although these gains did not continue to improve post‐discharge.
• As expected, although children did not report a reduction in pain during rehabilitation, they did report a significant drop in perceived pain in the 12 months following discharge from the program.

Benore E, Webster EE, Wang L, Banez G. Longitudinal analysis of patient‐reported outcomes from an interdisciplinary pediatric pain rehabilitation program for children with chronic migraine and headache. [Published online ahead of print August 23, 2018]. Headache. doi:10.1111/head.13389.

Children with chronic headache and migraine who are severely functionally impaired demonstrated linear improvement in pain‐specific patient‐reported outcomes over time, according to a recent study that aimed to evaluate the trajectory of recovery for children undergoing intensive interdisciplinary pain rehabilitation treatment (IIPT). A retrospective analysis was conducted of patient‐reported outcomes in a clinical database of 135 children (mean age 15.2 [SD=2.2] and 74% female) admitted to an IIPT program between the years 2008 and 2014. Available data across 5 separate time points (up to 1‐year post‐discharge) were reviewed. Researchers found:

• A statistically significant improvement was noted in pain‐specific measures of functioning, including daily functioning, emotional functioning, family functioning, and school absences over a 12‐month period.
• A more general measure of quality of life improved during the program, based upon child and parent reports, although these gains did not continue to improve post‐discharge.
• As expected, although children did not report a reduction in pain during rehabilitation, they did report a significant drop in perceived pain in the 12 months following discharge from the program.

Benore E, Webster EE, Wang L, Banez G. Longitudinal analysis of patient‐reported outcomes from an interdisciplinary pediatric pain rehabilitation program for children with chronic migraine and headache. [Published online ahead of print August 23, 2018]. Headache. doi:10.1111/head.13389.

Preliminary data indicate that hybrid cognitive‐behavioral therapy is feasible and acceptable for youth with co‐occurring chronic migraine and insomnia, according to a recent study. Researchers conducted a single‐arm pilot trial to evaluate the feasibility and acceptability of delivering cognitive‐behavioral therapy for insomnia to 21 youth (mean age 15.5 years) with co‐occurring chronic migraine and insomnia. Adolescents completed up to 6 individual treatment sessions over 6 to 12 weeks, and 1 booster session 1 month later. Assessments included a prospective 7‐day headache and sleep diary, and self‐report measures of insomnia, sleep quality, sleep habits, and activity limitations at pre‐treatment, immediate post‐treatment, and 3‐month follow‐up. Researchers found:

• Adolescents demonstrated good treatment adherence and families rated the intervention as highly acceptable.
• Preliminary analyses indicated improvements from pre‐treatment to post‐treatment in primary outcomes of headache days (M=4.7, SD=2.1 vs M=2.8, SD=2.7) and insomnia symptoms (M=16.9, SD=5.2 vs M=9.5, SD=6.2), which were maintained at 3‐month follow‐up (M=2.7, SD=2.8; M=9.3, SD=5.0, respectively).
• Improvements were also found in secondary outcomes of pain‐related activity limitations as well as sleep quality, sleep hygiene, and sleep patterns.

Law EF, Tham SW, Aaron RV, Dudeney J, Palermo TM. Hybrid cognitive‐behavioral therapy intervention for adolescents with co‐occurring migraine and insomnia: A single‐arm pilot trial. [Published online ahead of print August 27, 2018]. Headache. doi:10.1111/head.13355.

Preliminary data indicate that hybrid cognitive‐behavioral therapy is feasible and acceptable for youth with co‐occurring chronic migraine and insomnia, according to a recent study. Researchers conducted a single‐arm pilot trial to evaluate the feasibility and acceptability of delivering cognitive‐behavioral therapy for insomnia to 21 youth (mean age 15.5 years) with co‐occurring chronic migraine and insomnia. Adolescents completed up to 6 individual treatment sessions over 6 to 12 weeks, and 1 booster session 1 month later. Assessments included a prospective 7‐day headache and sleep diary, and self‐report measures of insomnia, sleep quality, sleep habits, and activity limitations at pre‐treatment, immediate post‐treatment, and 3‐month follow‐up. Researchers found:

• Adolescents demonstrated good treatment adherence and families rated the intervention as highly acceptable.
• Preliminary analyses indicated improvements from pre‐treatment to post‐treatment in primary outcomes of headache days (M=4.7, SD=2.1 vs M=2.8, SD=2.7) and insomnia symptoms (M=16.9, SD=5.2 vs M=9.5, SD=6.2), which were maintained at 3‐month follow‐up (M=2.7, SD=2.8; M=9.3, SD=5.0, respectively).
• Improvements were also found in secondary outcomes of pain‐related activity limitations as well as sleep quality, sleep hygiene, and sleep patterns.

Law EF, Tham SW, Aaron RV, Dudeney J, Palermo TM. Hybrid cognitive‐behavioral therapy intervention for adolescents with co‐occurring migraine and insomnia: A single‐arm pilot trial. [Published online ahead of print August 27, 2018]. Headache. doi:10.1111/head.13355.

Preliminary data indicate that hybrid cognitive‐behavioral therapy is feasible and acceptable for youth with co‐occurring chronic migraine and insomnia, according to a recent study. Researchers conducted a single‐arm pilot trial to evaluate the feasibility and acceptability of delivering cognitive‐behavioral therapy for insomnia to 21 youth (mean age 15.5 years) with co‐occurring chronic migraine and insomnia. Adolescents completed up to 6 individual treatment sessions over 6 to 12 weeks, and 1 booster session 1 month later. Assessments included a prospective 7‐day headache and sleep diary, and self‐report measures of insomnia, sleep quality, sleep habits, and activity limitations at pre‐treatment, immediate post‐treatment, and 3‐month follow‐up. Researchers found:

• Adolescents demonstrated good treatment adherence and families rated the intervention as highly acceptable.
• Preliminary analyses indicated improvements from pre‐treatment to post‐treatment in primary outcomes of headache days (M=4.7, SD=2.1 vs M=2.8, SD=2.7) and insomnia symptoms (M=16.9, SD=5.2 vs M=9.5, SD=6.2), which were maintained at 3‐month follow‐up (M=2.7, SD=2.8; M=9.3, SD=5.0, respectively).
• Improvements were also found in secondary outcomes of pain‐related activity limitations as well as sleep quality, sleep hygiene, and sleep patterns.

Law EF, Tham SW, Aaron RV, Dudeney J, Palermo TM. Hybrid cognitive‐behavioral therapy intervention for adolescents with co‐occurring migraine and insomnia: A single‐arm pilot trial. [Published online ahead of print August 27, 2018]. Headache. doi:10.1111/head.13355.

The frontal lobes are pretty important. They help us plan and concentrate, and they keep us from being impulsive and distracted. They help to override those pesky emotions that can interfere with objective thought.

In Vulcans, I imagine, the frontal lobes are huge.

We put a lot of faith into them in this field. We have to stay calm and try to reason during stressful times, often with people who aren’t quite as clear headed at that moment.

Gilitukha/Thinkstock

I think we all like to believe we’re creatures of our intellects: able to think dispassionately about the current case in front of us, to make decisions based on established facts and data. And, generally, most of us do a good job.

But sometimes it doesn’t work that way.

One day in late July, I was working my way through the usual afternoon patients at the office, checking test results, making decisions – the everyday stuff. After 20 years, this has become routine.

At 1:48, while talking to a patient, an email crossed my screen. As usual, I glanced at it to make sure it wasn’t a patient emergency ... nope. It was mine.

Because of a rapidly moving forest fire in southern California, my daughter’s summer camp was being evacuated. She was safe, but they were being moved to a high school that was being used as an evacuation center in Banning, Calif. We were asked to come get her as soon as safely possible.

And, just like that, my frontal lobes got moved to the back seat.

Granted, I didn’t panic. I didn’t cancel the patients I had waiting. I completed my current appointment, then took a few extra minutes to look at the schedule with my secretary to see where we could move the next day’s patients so I could drive to California in the morning. Then I went on with my day.

I still had three more patients left. Although none of them said anything, I’m sure they noticed I wasn’t mentally all there. I probably seemed distracted, checking my screen a few more times than I should have to see whether there were further updates. I don’t think I made any bad decisions about treatment, but I certainly wasn’t at the top of my game. A few days later, after things had settled down, I reread my notes from the day to make sure I hadn’t missed anything.

It’s a surprising reminder of how powerful the older, nonrational parts of our brains are. Although they didn’t take over, they certainly affected my ability to focus on the task at hand. There’s a reason those areas exist, too, even if we keep them hidden in our daily lives.
 

Dr. Block has a solo neurology practice in Scottsdale, Ariz.

The frontal lobes are pretty important. They help us plan and concentrate, and they keep us from being impulsive and distracted. They help to override those pesky emotions that can interfere with objective thought.

In Vulcans, I imagine, the frontal lobes are huge.

We put a lot of faith into them in this field. We have to stay calm and try to reason during stressful times, often with people who aren’t quite as clear headed at that moment.

Gilitukha/Thinkstock

I think we all like to believe we’re creatures of our intellects: able to think dispassionately about the current case in front of us, to make decisions based on established facts and data. And, generally, most of us do a good job.

But sometimes it doesn’t work that way.

One day in late July, I was working my way through the usual afternoon patients at the office, checking test results, making decisions – the everyday stuff. After 20 years, this has become routine.

At 1:48, while talking to a patient, an email crossed my screen. As usual, I glanced at it to make sure it wasn’t a patient emergency ... nope. It was mine.

Because of a rapidly moving forest fire in southern California, my daughter’s summer camp was being evacuated. She was safe, but they were being moved to a high school that was being used as an evacuation center in Banning, Calif. We were asked to come get her as soon as safely possible.

And, just like that, my frontal lobes got moved to the back seat.

Granted, I didn’t panic. I didn’t cancel the patients I had waiting. I completed my current appointment, then took a few extra minutes to look at the schedule with my secretary to see where we could move the next day’s patients so I could drive to California in the morning. Then I went on with my day.

I still had three more patients left. Although none of them said anything, I’m sure they noticed I wasn’t mentally all there. I probably seemed distracted, checking my screen a few more times than I should have to see whether there were further updates. I don’t think I made any bad decisions about treatment, but I certainly wasn’t at the top of my game. A few days later, after things had settled down, I reread my notes from the day to make sure I hadn’t missed anything.

It’s a surprising reminder of how powerful the older, nonrational parts of our brains are. Although they didn’t take over, they certainly affected my ability to focus on the task at hand. There’s a reason those areas exist, too, even if we keep them hidden in our daily lives.
 

Dr. Block has a solo neurology practice in Scottsdale, Ariz.

The frontal lobes are pretty important. They help us plan and concentrate, and they keep us from being impulsive and distracted. They help to override those pesky emotions that can interfere with objective thought.

In Vulcans, I imagine, the frontal lobes are huge.

We put a lot of faith into them in this field. We have to stay calm and try to reason during stressful times, often with people who aren’t quite as clear headed at that moment.

Gilitukha/Thinkstock

I think we all like to believe we’re creatures of our intellects: able to think dispassionately about the current case in front of us, to make decisions based on established facts and data. And, generally, most of us do a good job.

But sometimes it doesn’t work that way.

One day in late July, I was working my way through the usual afternoon patients at the office, checking test results, making decisions – the everyday stuff. After 20 years, this has become routine.

At 1:48, while talking to a patient, an email crossed my screen. As usual, I glanced at it to make sure it wasn’t a patient emergency ... nope. It was mine.

Because of a rapidly moving forest fire in southern California, my daughter’s summer camp was being evacuated. She was safe, but they were being moved to a high school that was being used as an evacuation center in Banning, Calif. We were asked to come get her as soon as safely possible.

And, just like that, my frontal lobes got moved to the back seat.

Granted, I didn’t panic. I didn’t cancel the patients I had waiting. I completed my current appointment, then took a few extra minutes to look at the schedule with my secretary to see where we could move the next day’s patients so I could drive to California in the morning. Then I went on with my day.

I still had three more patients left. Although none of them said anything, I’m sure they noticed I wasn’t mentally all there. I probably seemed distracted, checking my screen a few more times than I should have to see whether there were further updates. I don’t think I made any bad decisions about treatment, but I certainly wasn’t at the top of my game. A few days later, after things had settled down, I reread my notes from the day to make sure I hadn’t missed anything.

It’s a surprising reminder of how powerful the older, nonrational parts of our brains are. Although they didn’t take over, they certainly affected my ability to focus on the task at hand. There’s a reason those areas exist, too, even if we keep them hidden in our daily lives.
 

Dr. Block has a solo neurology practice in Scottsdale, Ariz.

In a letter to Congressional leaders, President Trump has announced a pay freeze for all civilian federal employees in 2019. The decision will not impact active duty service members who are still are expected to receive a 2.6% pay raise next year.

The decision aims to circumvent a 2.1% across the board increase and additional locality pay increases that would average 25.7%. "We must maintain efforts to put our Nation on a fiscally sustainable course, and Federal agency budgets cannot sustain such increases. Accordingly, I have determined that it is appropriate to exercise my authority to set alternative across-the-board and locality pay adjustments for 2019."

In the letter, the President disputed the notion that locality pay was important for keeping and attracting quality applicants for positions. Currently, the Veterans Health Administration has more  than 40,000 job openings.

"I have determined that for 2019, both across‑the‑board pay increases and locality pay increases will be set at zero.  These alternative pay plan decisions will not materially affect our ability to attract and retain a well‑qualified Federal workforce.

President Trump had already included the pay freeze in his budget proposal. While Congress can override the President's decision, congressional action is not expected.

In a letter to Congressional leaders, President Trump has announced a pay freeze for all civilian federal employees in 2019. The decision will not impact active duty service members who are still are expected to receive a 2.6% pay raise next year.

The decision aims to circumvent a 2.1% across the board increase and additional locality pay increases that would average 25.7%. "We must maintain efforts to put our Nation on a fiscally sustainable course, and Federal agency budgets cannot sustain such increases. Accordingly, I have determined that it is appropriate to exercise my authority to set alternative across-the-board and locality pay adjustments for 2019."

In the letter, the President disputed the notion that locality pay was important for keeping and attracting quality applicants for positions. Currently, the Veterans Health Administration has more  than 40,000 job openings.

"I have determined that for 2019, both across‑the‑board pay increases and locality pay increases will be set at zero.  These alternative pay plan decisions will not materially affect our ability to attract and retain a well‑qualified Federal workforce.

President Trump had already included the pay freeze in his budget proposal. While Congress can override the President's decision, congressional action is not expected.

In a letter to Congressional leaders, President Trump has announced a pay freeze for all civilian federal employees in 2019. The decision will not impact active duty service members who are still are expected to receive a 2.6% pay raise next year.

The decision aims to circumvent a 2.1% across the board increase and additional locality pay increases that would average 25.7%. "We must maintain efforts to put our Nation on a fiscally sustainable course, and Federal agency budgets cannot sustain such increases. Accordingly, I have determined that it is appropriate to exercise my authority to set alternative across-the-board and locality pay adjustments for 2019."

In the letter, the President disputed the notion that locality pay was important for keeping and attracting quality applicants for positions. Currently, the Veterans Health Administration has more  than 40,000 job openings.

"I have determined that for 2019, both across‑the‑board pay increases and locality pay increases will be set at zero.  These alternative pay plan decisions will not materially affect our ability to attract and retain a well‑qualified Federal workforce.

President Trump had already included the pay freeze in his budget proposal. While Congress can override the President's decision, congressional action is not expected.

The Food and Drug Administration (FDA) has approved Jivi (antihemophilic factor [recombinant] PEGylated-aucl) for the treatment of hemophilia A.

Jivi (formerly BAY94-9027), a DNA-derived, factor VIII concentrate, is now approved for use in previously treated adults and adolescents – aged 12 years and older – with hemophilia A.

The product is also approved for on-demand treatment and control of bleeding episodes, for perioperative management of bleeding, and as routine prophylaxis to reduce the frequency of bleeding episodes.

The initial recommended prophylactic regimen is dosing twice weekly (30-40 IU/kg) with the ability to dose every 5 days (45-60 IU/kg) and to further individually adjust to less or more frequent dosing based on bleeding episodes.

The FDA’s approval of Jivi is based on results from the phase 2/3 PROTECT VIII trial. Some results from this trial were published in the Journal of Thrombosis and Haemostasis (2017 Mar;15[3]:411-419). Additional results are available in the prescribing information for Jivi. The product is marketed by Bayer.

PROTECT VIII enrolled previously treated adults and adolescents with severe hemophilia A. In part A, researchers evaluated different dosing regimens for Jivi used as prophylaxis and on-demand treatment. An optional extension study was available to patients who completed part A. In part B, researchers evaluated Jivi for perioperative management.

In part A, there were 132 patients in the intent‐to‐treat population – 112 in the prophylaxis group and 20 in the on-demand group.

Patients received Jivi for 36 weeks. For the first 10 weeks, patients in the prophylaxis group received twice-weekly dosing at 25 IU/kg.

Patients with more than one bleed during this time went on to receive 30-40 IU/kg twice weekly. Patients with one or fewer bleeds were eligible for randomization to dosing every 5 days (45-60 IU/kg) or every 7 days (60 IU/kg).

The median annualized bleeding rate (ABR) for the patients who were treated twice weekly and were not eligible for randomization (n = 13) improved after their dose increase. Their median ABR decreased from 17.4 to 4.1.

The median ABR for patients who were randomized to treatment every 5 days (n = 43) was 1.9. The median ABR was also 1.9 for patients who were eligible for randomization but continued on twice-weekly treatment (n = 11). The median ABR for patients who completed prophylaxis with dosing every 7 days (32/43) was 0.96. The median ABR for patients treated on demand was 24.1.

There were 388 treated bleeds in the on-demand group and 317 treated bleeds in the prophylaxis group.

The safety data in the prescribing information includes 221 patients from three studies. The 17 patients who received Jivi for perioperative management were excluded from the pooled safety analysis but included in the analysis for inhibitor development.

In the 148 patients aged 12 years and older who were exposed to Jivi, adverse events included abdominal pain, nausea, vomiting, injection site reactions, pyrexia, hypersensitivity, dizziness, headache, insomnia, cough, erythema, pruritus, rash, and flushing.

A factor VIII inhibitor was reported in one adult patient, but repeat testing did not confirm the report.

One adult patient with asthma had a clinical hypersensitivity reaction and a transient increase of IgM anti-PEG antibody titer, which was negative upon retesting.

[email protected]

The Food and Drug Administration (FDA) has approved Jivi (antihemophilic factor [recombinant] PEGylated-aucl) for the treatment of hemophilia A.

Jivi (formerly BAY94-9027), a DNA-derived, factor VIII concentrate, is now approved for use in previously treated adults and adolescents – aged 12 years and older – with hemophilia A.

The product is also approved for on-demand treatment and control of bleeding episodes, for perioperative management of bleeding, and as routine prophylaxis to reduce the frequency of bleeding episodes.

The initial recommended prophylactic regimen is dosing twice weekly (30-40 IU/kg) with the ability to dose every 5 days (45-60 IU/kg) and to further individually adjust to less or more frequent dosing based on bleeding episodes.

The FDA’s approval of Jivi is based on results from the phase 2/3 PROTECT VIII trial. Some results from this trial were published in the Journal of Thrombosis and Haemostasis (2017 Mar;15[3]:411-419). Additional results are available in the prescribing information for Jivi. The product is marketed by Bayer.

PROTECT VIII enrolled previously treated adults and adolescents with severe hemophilia A. In part A, researchers evaluated different dosing regimens for Jivi used as prophylaxis and on-demand treatment. An optional extension study was available to patients who completed part A. In part B, researchers evaluated Jivi for perioperative management.

In part A, there were 132 patients in the intent‐to‐treat population – 112 in the prophylaxis group and 20 in the on-demand group.

Patients received Jivi for 36 weeks. For the first 10 weeks, patients in the prophylaxis group received twice-weekly dosing at 25 IU/kg.

Patients with more than one bleed during this time went on to receive 30-40 IU/kg twice weekly. Patients with one or fewer bleeds were eligible for randomization to dosing every 5 days (45-60 IU/kg) or every 7 days (60 IU/kg).

The median annualized bleeding rate (ABR) for the patients who were treated twice weekly and were not eligible for randomization (n = 13) improved after their dose increase. Their median ABR decreased from 17.4 to 4.1.

The median ABR for patients who were randomized to treatment every 5 days (n = 43) was 1.9. The median ABR was also 1.9 for patients who were eligible for randomization but continued on twice-weekly treatment (n = 11). The median ABR for patients who completed prophylaxis with dosing every 7 days (32/43) was 0.96. The median ABR for patients treated on demand was 24.1.

There were 388 treated bleeds in the on-demand group and 317 treated bleeds in the prophylaxis group.

The safety data in the prescribing information includes 221 patients from three studies. The 17 patients who received Jivi for perioperative management were excluded from the pooled safety analysis but included in the analysis for inhibitor development.

In the 148 patients aged 12 years and older who were exposed to Jivi, adverse events included abdominal pain, nausea, vomiting, injection site reactions, pyrexia, hypersensitivity, dizziness, headache, insomnia, cough, erythema, pruritus, rash, and flushing.

A factor VIII inhibitor was reported in one adult patient, but repeat testing did not confirm the report.

One adult patient with asthma had a clinical hypersensitivity reaction and a transient increase of IgM anti-PEG antibody titer, which was negative upon retesting.

[email protected]

The Food and Drug Administration (FDA) has approved Jivi (antihemophilic factor [recombinant] PEGylated-aucl) for the treatment of hemophilia A.

Jivi (formerly BAY94-9027), a DNA-derived, factor VIII concentrate, is now approved for use in previously treated adults and adolescents – aged 12 years and older – with hemophilia A.

The product is also approved for on-demand treatment and control of bleeding episodes, for perioperative management of bleeding, and as routine prophylaxis to reduce the frequency of bleeding episodes.

The initial recommended prophylactic regimen is dosing twice weekly (30-40 IU/kg) with the ability to dose every 5 days (45-60 IU/kg) and to further individually adjust to less or more frequent dosing based on bleeding episodes.

The FDA’s approval of Jivi is based on results from the phase 2/3 PROTECT VIII trial. Some results from this trial were published in the Journal of Thrombosis and Haemostasis (2017 Mar;15[3]:411-419). Additional results are available in the prescribing information for Jivi. The product is marketed by Bayer.

PROTECT VIII enrolled previously treated adults and adolescents with severe hemophilia A. In part A, researchers evaluated different dosing regimens for Jivi used as prophylaxis and on-demand treatment. An optional extension study was available to patients who completed part A. In part B, researchers evaluated Jivi for perioperative management.

In part A, there were 132 patients in the intent‐to‐treat population – 112 in the prophylaxis group and 20 in the on-demand group.

Patients received Jivi for 36 weeks. For the first 10 weeks, patients in the prophylaxis group received twice-weekly dosing at 25 IU/kg.

Patients with more than one bleed during this time went on to receive 30-40 IU/kg twice weekly. Patients with one or fewer bleeds were eligible for randomization to dosing every 5 days (45-60 IU/kg) or every 7 days (60 IU/kg).

The median annualized bleeding rate (ABR) for the patients who were treated twice weekly and were not eligible for randomization (n = 13) improved after their dose increase. Their median ABR decreased from 17.4 to 4.1.

The median ABR for patients who were randomized to treatment every 5 days (n = 43) was 1.9. The median ABR was also 1.9 for patients who were eligible for randomization but continued on twice-weekly treatment (n = 11). The median ABR for patients who completed prophylaxis with dosing every 7 days (32/43) was 0.96. The median ABR for patients treated on demand was 24.1.

There were 388 treated bleeds in the on-demand group and 317 treated bleeds in the prophylaxis group.

The safety data in the prescribing information includes 221 patients from three studies. The 17 patients who received Jivi for perioperative management were excluded from the pooled safety analysis but included in the analysis for inhibitor development.

In the 148 patients aged 12 years and older who were exposed to Jivi, adverse events included abdominal pain, nausea, vomiting, injection site reactions, pyrexia, hypersensitivity, dizziness, headache, insomnia, cough, erythema, pruritus, rash, and flushing.

A factor VIII inhibitor was reported in one adult patient, but repeat testing did not confirm the report.

One adult patient with asthma had a clinical hypersensitivity reaction and a transient increase of IgM anti-PEG antibody titer, which was negative upon retesting.

[email protected]

We need your help to spread the word

Please help us spread the news about peripheral arterial disease. It’s easy – SVS has updated its online resources for physicians and resources for patients. Both web pages have videos, web links, scholarly articles and more, appropriate for each audience. Please share the links to people on your email list. Help us reach more patients and caregivers and spread awareness about PAD!

We need your help to spread the word

Please help us spread the news about peripheral arterial disease. It’s easy – SVS has updated its online resources for physicians and resources for patients. Both web pages have videos, web links, scholarly articles and more, appropriate for each audience. Please share the links to people on your email list. Help us reach more patients and caregivers and spread awareness about PAD!

We need your help to spread the word

Please help us spread the news about peripheral arterial disease. It’s easy – SVS has updated its online resources for physicians and resources for patients. Both web pages have videos, web links, scholarly articles and more, appropriate for each audience. Please share the links to people on your email list. Help us reach more patients and caregivers and spread awareness about PAD!

Drugmakers and physician groups expressed concern about connecting patient reported outcomes (PRO) to coverage decisions for chimeric antigen receptor T-cell therapy (CAR T) at a recent Medicare meeting.

copyright roobcio/Thinkstock

Despite these objections, members of the Medicare Evidence Development and Coverage Advisory Committee (MEDCAC) generally expressed confidence in the use of PROs in a series of votes about PRO instruments’ use in cancer care trials.

MEDCAC opened a national coverage analysis in May 2018 at the request of UnitedHealthcare. The insurer had asked the Centers for Medicare & Medicaid Services to clarify the circumstances for coverage of the new CAR T therapies.

In presentations at an Aug. 22 MEDCAC meeting, representatives from the two manufacturers that have approved CAR T treatments on the market cautioned against using PROs in the context of coverage decisions, even though both companies used PRO data in their clinical trials and are collecting it in the postmarketing period.

“While Kite recognizes the importance of PROs in ... clinical trials, the PRO CAR T science, where these instruments are most appropriate for CAR T, remains a still to be determined and still is evolving and still quite early in the developments,” said William Go, MD, PhD, vice president of clinical development at Kite Pharmaceuticals, a Gilead company.

Despite PROs playing an active role in the ongoing development and clinical trials that Kite is running, in the context of CAR T, “we feel PROs are not quite ready for real-world coverage decisions at this time,” Dr. Go said.

A representative from Novartis agreed. “It is important to clearly define research objectives when considering whether to collect patient reported outcomes data, particularly given the burden associated with advanced disease experienced by the patients that are likely to receive this therapy,” Ilia Ferrusi, PhD, associate director, HEOR (CAR T Therapy), U.S. Oncology, Novartis, told the panel.

She noted that Novartis experienced challenges collecting PRO data, which resulted in risks to data quality and interpretation. “This could very well be amplified in larger trials or in real-world practice.”

The presenters’ comments were echoed by written comments submitted to the CMS prior to the MEDCAC meeting.

The American Society for Blood and Marrow Transplantation (ASBMT), despite supporting the collection of patient-reported outcomes, particularly in oncology, “strongly objects to any mechanism that would tie patient access or provider reimbursement to the reporting of PROs, especially in the case of CAR T therapy,” the organization said in a letter to the CMS.

Optimal PRO instruments and time points “are currently unknown” as the PROs used today were developed around chemotherapy use.

“Mandating the use of a current instrument or set of outcomes through NCD [national coverage determination] or CED [coverage with evidence development] will set a course of collecting data that is very likely to be inaccurate and inadequate,” they said.

The ASBMT also noted there is significant heterogeneity in the CAR T constructs and associated disease indications. “Products will utilize different scientific constructs and the time frames for clinical response and/or potential onset of toxicities may differ for each construct, which will challenge the establishment of set time points for data collection.”

And while presentations and comments focused on PROs in the context of CAR T, conversation and voting on the use of PROs was in the broader context of oncology clinical trials, not specific to CAR T, changing the nature of the debate and calling into question the point of the meeting and how it will inform the national coverage determination on CAR T specifically.

The June 15 meeting announcement published in the Federal Register stated that the advisory committee “will specifically focus on appraisal of evidence-based PRO assessments to provide information that impacts patients, their providers, and caregivers after a CAR T-cell therapy intervention for the patient’s cancer.”

However, the committee was asked to evaluate evidence on a number of potential PRO instruments in the broader context of oncology clinical trials without a cancer-specific or treatment-specific context, leading to a debate over how different types of cancer will have different kinds of PRO assessments related to them.

The CMS is expected to make a national coverage decision on CAR T-cell therapy by Feb. 16, 2019.

[email protected]

Drugmakers and physician groups expressed concern about connecting patient reported outcomes (PRO) to coverage decisions for chimeric antigen receptor T-cell therapy (CAR T) at a recent Medicare meeting.

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Despite these objections, members of the Medicare Evidence Development and Coverage Advisory Committee (MEDCAC) generally expressed confidence in the use of PROs in a series of votes about PRO instruments’ use in cancer care trials.

MEDCAC opened a national coverage analysis in May 2018 at the request of UnitedHealthcare. The insurer had asked the Centers for Medicare & Medicaid Services to clarify the circumstances for coverage of the new CAR T therapies.

In presentations at an Aug. 22 MEDCAC meeting, representatives from the two manufacturers that have approved CAR T treatments on the market cautioned against using PROs in the context of coverage decisions, even though both companies used PRO data in their clinical trials and are collecting it in the postmarketing period.

“While Kite recognizes the importance of PROs in ... clinical trials, the PRO CAR T science, where these instruments are most appropriate for CAR T, remains a still to be determined and still is evolving and still quite early in the developments,” said William Go, MD, PhD, vice president of clinical development at Kite Pharmaceuticals, a Gilead company.

Despite PROs playing an active role in the ongoing development and clinical trials that Kite is running, in the context of CAR T, “we feel PROs are not quite ready for real-world coverage decisions at this time,” Dr. Go said.

A representative from Novartis agreed. “It is important to clearly define research objectives when considering whether to collect patient reported outcomes data, particularly given the burden associated with advanced disease experienced by the patients that are likely to receive this therapy,” Ilia Ferrusi, PhD, associate director, HEOR (CAR T Therapy), U.S. Oncology, Novartis, told the panel.

She noted that Novartis experienced challenges collecting PRO data, which resulted in risks to data quality and interpretation. “This could very well be amplified in larger trials or in real-world practice.”

The presenters’ comments were echoed by written comments submitted to the CMS prior to the MEDCAC meeting.

The American Society for Blood and Marrow Transplantation (ASBMT), despite supporting the collection of patient-reported outcomes, particularly in oncology, “strongly objects to any mechanism that would tie patient access or provider reimbursement to the reporting of PROs, especially in the case of CAR T therapy,” the organization said in a letter to the CMS.

Optimal PRO instruments and time points “are currently unknown” as the PROs used today were developed around chemotherapy use.

“Mandating the use of a current instrument or set of outcomes through NCD [national coverage determination] or CED [coverage with evidence development] will set a course of collecting data that is very likely to be inaccurate and inadequate,” they said.

The ASBMT also noted there is significant heterogeneity in the CAR T constructs and associated disease indications. “Products will utilize different scientific constructs and the time frames for clinical response and/or potential onset of toxicities may differ for each construct, which will challenge the establishment of set time points for data collection.”

And while presentations and comments focused on PROs in the context of CAR T, conversation and voting on the use of PROs was in the broader context of oncology clinical trials, not specific to CAR T, changing the nature of the debate and calling into question the point of the meeting and how it will inform the national coverage determination on CAR T specifically.

The June 15 meeting announcement published in the Federal Register stated that the advisory committee “will specifically focus on appraisal of evidence-based PRO assessments to provide information that impacts patients, their providers, and caregivers after a CAR T-cell therapy intervention for the patient’s cancer.”

However, the committee was asked to evaluate evidence on a number of potential PRO instruments in the broader context of oncology clinical trials without a cancer-specific or treatment-specific context, leading to a debate over how different types of cancer will have different kinds of PRO assessments related to them.

The CMS is expected to make a national coverage decision on CAR T-cell therapy by Feb. 16, 2019.

[email protected]

Drugmakers and physician groups expressed concern about connecting patient reported outcomes (PRO) to coverage decisions for chimeric antigen receptor T-cell therapy (CAR T) at a recent Medicare meeting.

copyright roobcio/Thinkstock

Despite these objections, members of the Medicare Evidence Development and Coverage Advisory Committee (MEDCAC) generally expressed confidence in the use of PROs in a series of votes about PRO instruments’ use in cancer care trials.

MEDCAC opened a national coverage analysis in May 2018 at the request of UnitedHealthcare. The insurer had asked the Centers for Medicare & Medicaid Services to clarify the circumstances for coverage of the new CAR T therapies.

In presentations at an Aug. 22 MEDCAC meeting, representatives from the two manufacturers that have approved CAR T treatments on the market cautioned against using PROs in the context of coverage decisions, even though both companies used PRO data in their clinical trials and are collecting it in the postmarketing period.

“While Kite recognizes the importance of PROs in ... clinical trials, the PRO CAR T science, where these instruments are most appropriate for CAR T, remains a still to be determined and still is evolving and still quite early in the developments,” said William Go, MD, PhD, vice president of clinical development at Kite Pharmaceuticals, a Gilead company.

Despite PROs playing an active role in the ongoing development and clinical trials that Kite is running, in the context of CAR T, “we feel PROs are not quite ready for real-world coverage decisions at this time,” Dr. Go said.

A representative from Novartis agreed. “It is important to clearly define research objectives when considering whether to collect patient reported outcomes data, particularly given the burden associated with advanced disease experienced by the patients that are likely to receive this therapy,” Ilia Ferrusi, PhD, associate director, HEOR (CAR T Therapy), U.S. Oncology, Novartis, told the panel.

She noted that Novartis experienced challenges collecting PRO data, which resulted in risks to data quality and interpretation. “This could very well be amplified in larger trials or in real-world practice.”

The presenters’ comments were echoed by written comments submitted to the CMS prior to the MEDCAC meeting.

The American Society for Blood and Marrow Transplantation (ASBMT), despite supporting the collection of patient-reported outcomes, particularly in oncology, “strongly objects to any mechanism that would tie patient access or provider reimbursement to the reporting of PROs, especially in the case of CAR T therapy,” the organization said in a letter to the CMS.

Optimal PRO instruments and time points “are currently unknown” as the PROs used today were developed around chemotherapy use.

“Mandating the use of a current instrument or set of outcomes through NCD [national coverage determination] or CED [coverage with evidence development] will set a course of collecting data that is very likely to be inaccurate and inadequate,” they said.

The ASBMT also noted there is significant heterogeneity in the CAR T constructs and associated disease indications. “Products will utilize different scientific constructs and the time frames for clinical response and/or potential onset of toxicities may differ for each construct, which will challenge the establishment of set time points for data collection.”

And while presentations and comments focused on PROs in the context of CAR T, conversation and voting on the use of PROs was in the broader context of oncology clinical trials, not specific to CAR T, changing the nature of the debate and calling into question the point of the meeting and how it will inform the national coverage determination on CAR T specifically.

The June 15 meeting announcement published in the Federal Register stated that the advisory committee “will specifically focus on appraisal of evidence-based PRO assessments to provide information that impacts patients, their providers, and caregivers after a CAR T-cell therapy intervention for the patient’s cancer.”

However, the committee was asked to evaluate evidence on a number of potential PRO instruments in the broader context of oncology clinical trials without a cancer-specific or treatment-specific context, leading to a debate over how different types of cancer will have different kinds of PRO assessments related to them.

The CMS is expected to make a national coverage decision on CAR T-cell therapy by Feb. 16, 2019.

[email protected]

Citation: Stuart JJ, Tanz LJ, Missmer SA, et al. Hypertensive disorders of pregnancy and maternal cardiovascular disease risk factor development [published online July 3, 2018]. Ann Intern Med. doi:10.7326/M17-2740.

Citation: Stuart JJ, Tanz LJ, Missmer SA, et al. Hypertensive disorders of pregnancy and maternal cardiovascular disease risk factor development [published online July 3, 2018]. Ann Intern Med. doi:10.7326/M17-2740.

Citation: Stuart JJ, Tanz LJ, Missmer SA, et al. Hypertensive disorders of pregnancy and maternal cardiovascular disease risk factor development [published online July 3, 2018]. Ann Intern Med. doi:10.7326/M17-2740.

Self-management of epilepsy using a group-format, remote intervention improved mood, quality of life, and health functioning in high-risk individuals in a randomized, controlled trial.

In the 6-month trial, 120 individuals with epilepsy who had experienced at least one epilepsy-related health event in the previous 6 months were randomized either to a wait-list control group or a novel self‐management intervention.

The eight-session intervention, known as SMART, focused on modifiable factors that can be addressed with self-management, such as stress, substance abuse, routine, nutrition, and social support. It was delivered remotely over 8-10 weeks, either by telephone or online, after an initial in-person session.

“SMART combines the portability and low cost of a Web‐based intervention with the personally salient components of behavior modeling obtained by interacting with individuals who have ‘walked the walk’ in living with epilepsy,” Martha Sajatovic, MD, of Case Western Reserve University, Cleveland, and her coauthors wrote about their study published in Epilepsia.

Over the 6-month follow-up period, researchers found that individuals randomized to the intervention had a mean of 10.16 fewer negative health events, compared with a mean of 1.93 fewer events in the control group (P = .04).

When the authors looked at subcategories of negative health event counts – such as past 3-day seizure count or past 6‐month ED and hospitalization count – the differences were not significant. There was also no difference in seizure severity.

However, the study also showed significant improvements in participants’ self-rated depressive symptom severity, observer-rated depressive symptom severity, quality of life, and health functioning – both physical and mental – compared with controls. Intervention participants also reported significant improvements on the Epilepsy Self-Efficacy and Epilepsy Self-Management scales.

The majority of participants (94.2%) said the intervention was useful and that it covered the most important issues for them. A total of 92.3% believed the benefits of the SMART intervention were worth the hassle of taking part.

“SMART’s strengths are its foundation based on participatory research methods and an evidence‐based intervention, its use of peer educators facilitating empowerment and training, multimode delivery using traditional group format and telehealth approaches to eliminate barriers to care, and efficacy even in people who have long‐standing epilepsy,” the authors wrote.

“It is possible that SMART, which uses people with epilepsy as guides to help others learn to cope with the challenges of living with this common chronic neurologic condition, may help to alleviate some of the factors that prevent people with epilepsy from optimizing their quality of life.”

The study was supported by the Centers for Disease Control and Prevention. Three authors declared research grants, consultancies, royalties, or speaking positions with the private sector.

SOURCE: Sajatovic M et al. Epilepsia. 2018 Aug 10. doi: 10.1111/epi.14527.
 

Self-management of epilepsy using a group-format, remote intervention improved mood, quality of life, and health functioning in high-risk individuals in a randomized, controlled trial.

In the 6-month trial, 120 individuals with epilepsy who had experienced at least one epilepsy-related health event in the previous 6 months were randomized either to a wait-list control group or a novel self‐management intervention.

The eight-session intervention, known as SMART, focused on modifiable factors that can be addressed with self-management, such as stress, substance abuse, routine, nutrition, and social support. It was delivered remotely over 8-10 weeks, either by telephone or online, after an initial in-person session.

“SMART combines the portability and low cost of a Web‐based intervention with the personally salient components of behavior modeling obtained by interacting with individuals who have ‘walked the walk’ in living with epilepsy,” Martha Sajatovic, MD, of Case Western Reserve University, Cleveland, and her coauthors wrote about their study published in Epilepsia.

Over the 6-month follow-up period, researchers found that individuals randomized to the intervention had a mean of 10.16 fewer negative health events, compared with a mean of 1.93 fewer events in the control group (P = .04).

When the authors looked at subcategories of negative health event counts – such as past 3-day seizure count or past 6‐month ED and hospitalization count – the differences were not significant. There was also no difference in seizure severity.

However, the study also showed significant improvements in participants’ self-rated depressive symptom severity, observer-rated depressive symptom severity, quality of life, and health functioning – both physical and mental – compared with controls. Intervention participants also reported significant improvements on the Epilepsy Self-Efficacy and Epilepsy Self-Management scales.

The majority of participants (94.2%) said the intervention was useful and that it covered the most important issues for them. A total of 92.3% believed the benefits of the SMART intervention were worth the hassle of taking part.

“SMART’s strengths are its foundation based on participatory research methods and an evidence‐based intervention, its use of peer educators facilitating empowerment and training, multimode delivery using traditional group format and telehealth approaches to eliminate barriers to care, and efficacy even in people who have long‐standing epilepsy,” the authors wrote.

“It is possible that SMART, which uses people with epilepsy as guides to help others learn to cope with the challenges of living with this common chronic neurologic condition, may help to alleviate some of the factors that prevent people with epilepsy from optimizing their quality of life.”

The study was supported by the Centers for Disease Control and Prevention. Three authors declared research grants, consultancies, royalties, or speaking positions with the private sector.

SOURCE: Sajatovic M et al. Epilepsia. 2018 Aug 10. doi: 10.1111/epi.14527.
 

Self-management of epilepsy using a group-format, remote intervention improved mood, quality of life, and health functioning in high-risk individuals in a randomized, controlled trial.

In the 6-month trial, 120 individuals with epilepsy who had experienced at least one epilepsy-related health event in the previous 6 months were randomized either to a wait-list control group or a novel self‐management intervention.

The eight-session intervention, known as SMART, focused on modifiable factors that can be addressed with self-management, such as stress, substance abuse, routine, nutrition, and social support. It was delivered remotely over 8-10 weeks, either by telephone or online, after an initial in-person session.

“SMART combines the portability and low cost of a Web‐based intervention with the personally salient components of behavior modeling obtained by interacting with individuals who have ‘walked the walk’ in living with epilepsy,” Martha Sajatovic, MD, of Case Western Reserve University, Cleveland, and her coauthors wrote about their study published in Epilepsia.

Over the 6-month follow-up period, researchers found that individuals randomized to the intervention had a mean of 10.16 fewer negative health events, compared with a mean of 1.93 fewer events in the control group (P = .04).

When the authors looked at subcategories of negative health event counts – such as past 3-day seizure count or past 6‐month ED and hospitalization count – the differences were not significant. There was also no difference in seizure severity.

However, the study also showed significant improvements in participants’ self-rated depressive symptom severity, observer-rated depressive symptom severity, quality of life, and health functioning – both physical and mental – compared with controls. Intervention participants also reported significant improvements on the Epilepsy Self-Efficacy and Epilepsy Self-Management scales.

The majority of participants (94.2%) said the intervention was useful and that it covered the most important issues for them. A total of 92.3% believed the benefits of the SMART intervention were worth the hassle of taking part.

“SMART’s strengths are its foundation based on participatory research methods and an evidence‐based intervention, its use of peer educators facilitating empowerment and training, multimode delivery using traditional group format and telehealth approaches to eliminate barriers to care, and efficacy even in people who have long‐standing epilepsy,” the authors wrote.

“It is possible that SMART, which uses people with epilepsy as guides to help others learn to cope with the challenges of living with this common chronic neurologic condition, may help to alleviate some of the factors that prevent people with epilepsy from optimizing their quality of life.”

The study was supported by the Centers for Disease Control and Prevention. Three authors declared research grants, consultancies, royalties, or speaking positions with the private sector.

SOURCE: Sajatovic M et al. Epilepsia. 2018 Aug 10. doi: 10.1111/epi.14527.