Clinical Psychiatry News

LAS VEGAS – The risk-benefit ratio of new and emerging drugs for depression is going to be under the spotlight more than ever before, according to an expert in mood disorders.

Doug Brunk/MDedge News

“We’ve seen a shift in the landscape,” Alan F. Schatzberg, MD, said at an annual psychopharmacology update held by the Nevada Psychiatric Association. “We’ve gone from the development of ‘me too’ agents such as venlafaxine and fluoxetine to developing drugs that may have more dramatic effects, but they also may have more baggage in terms of risks. The risk-benefit ratio is going to become increasingly more of a focus in drug development, in terms of increasing risk.”

The development of the SSRIs, he continued, led to the introduction of agents that were generally effective and well tolerated.

“Particularly, they were well tolerated in terms of their wide safety margin,” said Dr. Schatzberg, who directs the Mood Disorders Center at Stanford (Calif.) University. “It’s difficult to [die by suicide] on an SSRI. Because of that, we had greater numbers of individuals treated than we did with the tricyclics.” The introduction of SSRIs led to “widening of the net in terms of the numbers of depressed patients,” he said. “Combine that with DSM-III, and DSM-IV having relatively easy criteria to obtain a diagnosis [of depression, and] you see a large group of subjects who are exposed to treatment. But a lot of those people may not respond to a particular agent. As new treatments are promulgated and tried, what you find is [that] a reasonable number of subjects are, in fact, truly resistant. That becomes a tough nut to crack for all of us who treat depression.”

Drugs for depression that clinicians commonly have in their armamentarium commonly revolve around monoaminergic function. New and emerging agents include hallucinatory serotonin 2a agonists, glutamatergic drugs such as ketamine, GABAergic neurosteroids, opioid modulators, and onabotulinumtoxinA.

In 2013, the Food and Drug Administration approved the multimodal agent vortioxetine for the treatment of major depressive disorder. The recommended dosing is 20 mg/day, and the drug appears to have a positive effect on cognition. A meta-analysis of vortioxetine, duloxetine, and placebo comparison trials evaluated the effect of each treatment on the Digit Symbol Substitution Test (Int J Neuropsychopharmacol. 2016 Jun 15;19[10]). Vortioxetine was superior to placebo in all three trials and was superior to duloxetine in two trials. Duloxetine was not superior to placebo in two trials.

In two double-blind studies, researchers evaluated the effects of psilocybin in cancer patients with comorbid depression and anxiety. Full doses of psilocybin were 0.3 mg/kg or 22-30 mg per 70 kg. Both studies demonstrated sustained responses at full doses (J Psychopharmacol. 2016 Nov 30;30[12]:1181-97 and J Psychopharmacol. 2016 Nov 30;30[12]:1165-80).

In an open label study of psilocybin in refractory major depression, 12 patients received 10 mg on day 1 and 25 mg on day 8. Eight of 12 patients responded at 1 week, and 7 of the 12 maintained response at 3 months (Lancet Psychiatry. 2016 Jul;3[7]:619-27). According to Dr. Schatzberg, this trial became the basis for a blinded study that Compass is conducting in the United States and in the United Kingdom in which refractory patients are going to be randomized to 1 mg, 10 mg, or 25 mg psilocybin using an independent rater. “These patients are accompanied during this experience by two therapists for up to 8 hours. It’s not quite a guided therapy; it’s kind of a safety net therapy if the patient needs [help]. We’ll see what happens.”

Another agent being studied is ketamine, which works on the glutamate system, an excitatory neurotransmitter. “Glutamate is the juice that keeps us going,” said Dr. Schatzberg, who is also the Kenneth T. Norris Jr. professor of psychiatry and behavioral sciences at the university. “We can’t live without glutamate.” An anesthetic agent that has been used for 50 years, ketamine is a N-methyl-d-aspartate antagonist that has mu opioid agonist effects and stimulant properties. “It causes a psychotomimetic dissociative kind of reaction,” he said. “The doses used in depression are subanesthetic. They may put the patient asleep, but they usually don’t. The problem with the antidepressant effect is that 70% of people who initially respond don’t continue to respond beyond 1 week.”

In a randomized, placebo-controlled, double-blind crossover study of patients with treatment-resistant depression, subjects who received ketamine showed significant improvement in depression, compared with subjects who received placebo within 110 minutes after injection, which remained significant throughout the following week (Arch Gen Psychiatry. 2006;63:856-64). Specifically, of the 17 subjects treated with ketamine, 71% met response and 29% met remission criteria the day following ketamine infusion. Thirty-five percent of subjects maintained response for at least 1 week.

In an effort to further evaluate the antidepressive effects of ketamine, researchers conducted a two-site, parallel-arm, randomized, controlled trial of a single infusion of ketamine, compared with an active placebo control condition, the anesthetic midazolam (Am J Psychiatry. 2013 Oct;170[10]:1134-42). After adjustment for baseline scores and site, the Montgomery-Åsberg Depression Rating Scale score was lower in the ketamine group than in the midazolam group by 7.95 points. The likelihood of response at 24 hours was greater with ketamine than with midazolam (odds ratio, 2.18), with response rates of 64% and 28%, respectively.

In 2019, the FDA approved esketamine nasal spray, in conjunction with an oral antidepressant, for the treatment of depression in adults with treatment-resistant depression. In three phase 3 double-blind studies of esketamine in treatment-refractory depression, one was positive, two were nearly positive, and the effect sizes were mild. One maintenance discontinuation trial was positive. Based on this data, Dr. Schatzberg said, there “is not much evidence” that patients get further gain beyond an antidepressant alone in the first 24-48 hours following ketamine administration. “It makes me wonder: Should we be continuing to give this drug intranasally beyond 48 hours?” he asked. “The reason I have concern is that in certain cultures, ketamine is a highly abusable drug.”

He and Gerard Sanacora, PhD, MD, addressed the topic in a 2015 opinion piece entitled, “Ketamine: Promising path or false prophecy in the development of novel therapeutics for mood disorders?”

“If we step back for a moment and look at where we are – an intravenously administered agent that is a street drug of abuse, works rapidly, and whose enantiomers are being studied by industry for intranasal use – we should be anxious, “ they wrote (Neuropsychopharmacology. 2015 Jan;40[2]:259-67). “We need to be as careful and conservative as possible and understand how it is acting and rule out the possibility of whether it acts as an opioid.”

Dr. Schatzberg disclosed that he has served a consultant to Alkermes, Avanir, Bracket, Compass, Delpor, Epiodyne, Janssen, Jazz, Lundbeck, McKinsey, Merck, Myriad Genetics, Owl, Neuronetics, Pfizer, Sage, and Sunovion. He has received research funding from Janssen and also holds an ownership interest in Corcept, Dermira, Delpor, Epiodyne, Incyte Genetics, Madrigal, Maerck, Owl Analytics, Seattle Genetics, Titan, and Xhale.

[email protected]

LAS VEGAS – The risk-benefit ratio of new and emerging drugs for depression is going to be under the spotlight more than ever before, according to an expert in mood disorders.

Doug Brunk/MDedge News

“We’ve seen a shift in the landscape,” Alan F. Schatzberg, MD, said at an annual psychopharmacology update held by the Nevada Psychiatric Association. “We’ve gone from the development of ‘me too’ agents such as venlafaxine and fluoxetine to developing drugs that may have more dramatic effects, but they also may have more baggage in terms of risks. The risk-benefit ratio is going to become increasingly more of a focus in drug development, in terms of increasing risk.”

The development of the SSRIs, he continued, led to the introduction of agents that were generally effective and well tolerated.

“Particularly, they were well tolerated in terms of their wide safety margin,” said Dr. Schatzberg, who directs the Mood Disorders Center at Stanford (Calif.) University. “It’s difficult to [die by suicide] on an SSRI. Because of that, we had greater numbers of individuals treated than we did with the tricyclics.” The introduction of SSRIs led to “widening of the net in terms of the numbers of depressed patients,” he said. “Combine that with DSM-III, and DSM-IV having relatively easy criteria to obtain a diagnosis [of depression, and] you see a large group of subjects who are exposed to treatment. But a lot of those people may not respond to a particular agent. As new treatments are promulgated and tried, what you find is [that] a reasonable number of subjects are, in fact, truly resistant. That becomes a tough nut to crack for all of us who treat depression.”

Drugs for depression that clinicians commonly have in their armamentarium commonly revolve around monoaminergic function. New and emerging agents include hallucinatory serotonin 2a agonists, glutamatergic drugs such as ketamine, GABAergic neurosteroids, opioid modulators, and onabotulinumtoxinA.

In 2013, the Food and Drug Administration approved the multimodal agent vortioxetine for the treatment of major depressive disorder. The recommended dosing is 20 mg/day, and the drug appears to have a positive effect on cognition. A meta-analysis of vortioxetine, duloxetine, and placebo comparison trials evaluated the effect of each treatment on the Digit Symbol Substitution Test (Int J Neuropsychopharmacol. 2016 Jun 15;19[10]). Vortioxetine was superior to placebo in all three trials and was superior to duloxetine in two trials. Duloxetine was not superior to placebo in two trials.

In two double-blind studies, researchers evaluated the effects of psilocybin in cancer patients with comorbid depression and anxiety. Full doses of psilocybin were 0.3 mg/kg or 22-30 mg per 70 kg. Both studies demonstrated sustained responses at full doses (J Psychopharmacol. 2016 Nov 30;30[12]:1181-97 and J Psychopharmacol. 2016 Nov 30;30[12]:1165-80).

In an open label study of psilocybin in refractory major depression, 12 patients received 10 mg on day 1 and 25 mg on day 8. Eight of 12 patients responded at 1 week, and 7 of the 12 maintained response at 3 months (Lancet Psychiatry. 2016 Jul;3[7]:619-27). According to Dr. Schatzberg, this trial became the basis for a blinded study that Compass is conducting in the United States and in the United Kingdom in which refractory patients are going to be randomized to 1 mg, 10 mg, or 25 mg psilocybin using an independent rater. “These patients are accompanied during this experience by two therapists for up to 8 hours. It’s not quite a guided therapy; it’s kind of a safety net therapy if the patient needs [help]. We’ll see what happens.”

Another agent being studied is ketamine, which works on the glutamate system, an excitatory neurotransmitter. “Glutamate is the juice that keeps us going,” said Dr. Schatzberg, who is also the Kenneth T. Norris Jr. professor of psychiatry and behavioral sciences at the university. “We can’t live without glutamate.” An anesthetic agent that has been used for 50 years, ketamine is a N-methyl-d-aspartate antagonist that has mu opioid agonist effects and stimulant properties. “It causes a psychotomimetic dissociative kind of reaction,” he said. “The doses used in depression are subanesthetic. They may put the patient asleep, but they usually don’t. The problem with the antidepressant effect is that 70% of people who initially respond don’t continue to respond beyond 1 week.”

In a randomized, placebo-controlled, double-blind crossover study of patients with treatment-resistant depression, subjects who received ketamine showed significant improvement in depression, compared with subjects who received placebo within 110 minutes after injection, which remained significant throughout the following week (Arch Gen Psychiatry. 2006;63:856-64). Specifically, of the 17 subjects treated with ketamine, 71% met response and 29% met remission criteria the day following ketamine infusion. Thirty-five percent of subjects maintained response for at least 1 week.

In an effort to further evaluate the antidepressive effects of ketamine, researchers conducted a two-site, parallel-arm, randomized, controlled trial of a single infusion of ketamine, compared with an active placebo control condition, the anesthetic midazolam (Am J Psychiatry. 2013 Oct;170[10]:1134-42). After adjustment for baseline scores and site, the Montgomery-Åsberg Depression Rating Scale score was lower in the ketamine group than in the midazolam group by 7.95 points. The likelihood of response at 24 hours was greater with ketamine than with midazolam (odds ratio, 2.18), with response rates of 64% and 28%, respectively.

In 2019, the FDA approved esketamine nasal spray, in conjunction with an oral antidepressant, for the treatment of depression in adults with treatment-resistant depression. In three phase 3 double-blind studies of esketamine in treatment-refractory depression, one was positive, two were nearly positive, and the effect sizes were mild. One maintenance discontinuation trial was positive. Based on this data, Dr. Schatzberg said, there “is not much evidence” that patients get further gain beyond an antidepressant alone in the first 24-48 hours following ketamine administration. “It makes me wonder: Should we be continuing to give this drug intranasally beyond 48 hours?” he asked. “The reason I have concern is that in certain cultures, ketamine is a highly abusable drug.”

He and Gerard Sanacora, PhD, MD, addressed the topic in a 2015 opinion piece entitled, “Ketamine: Promising path or false prophecy in the development of novel therapeutics for mood disorders?”

“If we step back for a moment and look at where we are – an intravenously administered agent that is a street drug of abuse, works rapidly, and whose enantiomers are being studied by industry for intranasal use – we should be anxious, “ they wrote (Neuropsychopharmacology. 2015 Jan;40[2]:259-67). “We need to be as careful and conservative as possible and understand how it is acting and rule out the possibility of whether it acts as an opioid.”

Dr. Schatzberg disclosed that he has served a consultant to Alkermes, Avanir, Bracket, Compass, Delpor, Epiodyne, Janssen, Jazz, Lundbeck, McKinsey, Merck, Myriad Genetics, Owl, Neuronetics, Pfizer, Sage, and Sunovion. He has received research funding from Janssen and also holds an ownership interest in Corcept, Dermira, Delpor, Epiodyne, Incyte Genetics, Madrigal, Maerck, Owl Analytics, Seattle Genetics, Titan, and Xhale.

[email protected]

LAS VEGAS – The risk-benefit ratio of new and emerging drugs for depression is going to be under the spotlight more than ever before, according to an expert in mood disorders.

Doug Brunk/MDedge News

“We’ve seen a shift in the landscape,” Alan F. Schatzberg, MD, said at an annual psychopharmacology update held by the Nevada Psychiatric Association. “We’ve gone from the development of ‘me too’ agents such as venlafaxine and fluoxetine to developing drugs that may have more dramatic effects, but they also may have more baggage in terms of risks. The risk-benefit ratio is going to become increasingly more of a focus in drug development, in terms of increasing risk.”

The development of the SSRIs, he continued, led to the introduction of agents that were generally effective and well tolerated.

“Particularly, they were well tolerated in terms of their wide safety margin,” said Dr. Schatzberg, who directs the Mood Disorders Center at Stanford (Calif.) University. “It’s difficult to [die by suicide] on an SSRI. Because of that, we had greater numbers of individuals treated than we did with the tricyclics.” The introduction of SSRIs led to “widening of the net in terms of the numbers of depressed patients,” he said. “Combine that with DSM-III, and DSM-IV having relatively easy criteria to obtain a diagnosis [of depression, and] you see a large group of subjects who are exposed to treatment. But a lot of those people may not respond to a particular agent. As new treatments are promulgated and tried, what you find is [that] a reasonable number of subjects are, in fact, truly resistant. That becomes a tough nut to crack for all of us who treat depression.”

Drugs for depression that clinicians commonly have in their armamentarium commonly revolve around monoaminergic function. New and emerging agents include hallucinatory serotonin 2a agonists, glutamatergic drugs such as ketamine, GABAergic neurosteroids, opioid modulators, and onabotulinumtoxinA.

In 2013, the Food and Drug Administration approved the multimodal agent vortioxetine for the treatment of major depressive disorder. The recommended dosing is 20 mg/day, and the drug appears to have a positive effect on cognition. A meta-analysis of vortioxetine, duloxetine, and placebo comparison trials evaluated the effect of each treatment on the Digit Symbol Substitution Test (Int J Neuropsychopharmacol. 2016 Jun 15;19[10]). Vortioxetine was superior to placebo in all three trials and was superior to duloxetine in two trials. Duloxetine was not superior to placebo in two trials.

In two double-blind studies, researchers evaluated the effects of psilocybin in cancer patients with comorbid depression and anxiety. Full doses of psilocybin were 0.3 mg/kg or 22-30 mg per 70 kg. Both studies demonstrated sustained responses at full doses (J Psychopharmacol. 2016 Nov 30;30[12]:1181-97 and J Psychopharmacol. 2016 Nov 30;30[12]:1165-80).

In an open label study of psilocybin in refractory major depression, 12 patients received 10 mg on day 1 and 25 mg on day 8. Eight of 12 patients responded at 1 week, and 7 of the 12 maintained response at 3 months (Lancet Psychiatry. 2016 Jul;3[7]:619-27). According to Dr. Schatzberg, this trial became the basis for a blinded study that Compass is conducting in the United States and in the United Kingdom in which refractory patients are going to be randomized to 1 mg, 10 mg, or 25 mg psilocybin using an independent rater. “These patients are accompanied during this experience by two therapists for up to 8 hours. It’s not quite a guided therapy; it’s kind of a safety net therapy if the patient needs [help]. We’ll see what happens.”

Another agent being studied is ketamine, which works on the glutamate system, an excitatory neurotransmitter. “Glutamate is the juice that keeps us going,” said Dr. Schatzberg, who is also the Kenneth T. Norris Jr. professor of psychiatry and behavioral sciences at the university. “We can’t live without glutamate.” An anesthetic agent that has been used for 50 years, ketamine is a N-methyl-d-aspartate antagonist that has mu opioid agonist effects and stimulant properties. “It causes a psychotomimetic dissociative kind of reaction,” he said. “The doses used in depression are subanesthetic. They may put the patient asleep, but they usually don’t. The problem with the antidepressant effect is that 70% of people who initially respond don’t continue to respond beyond 1 week.”

In a randomized, placebo-controlled, double-blind crossover study of patients with treatment-resistant depression, subjects who received ketamine showed significant improvement in depression, compared with subjects who received placebo within 110 minutes after injection, which remained significant throughout the following week (Arch Gen Psychiatry. 2006;63:856-64). Specifically, of the 17 subjects treated with ketamine, 71% met response and 29% met remission criteria the day following ketamine infusion. Thirty-five percent of subjects maintained response for at least 1 week.

In an effort to further evaluate the antidepressive effects of ketamine, researchers conducted a two-site, parallel-arm, randomized, controlled trial of a single infusion of ketamine, compared with an active placebo control condition, the anesthetic midazolam (Am J Psychiatry. 2013 Oct;170[10]:1134-42). After adjustment for baseline scores and site, the Montgomery-Åsberg Depression Rating Scale score was lower in the ketamine group than in the midazolam group by 7.95 points. The likelihood of response at 24 hours was greater with ketamine than with midazolam (odds ratio, 2.18), with response rates of 64% and 28%, respectively.

In 2019, the FDA approved esketamine nasal spray, in conjunction with an oral antidepressant, for the treatment of depression in adults with treatment-resistant depression. In three phase 3 double-blind studies of esketamine in treatment-refractory depression, one was positive, two were nearly positive, and the effect sizes were mild. One maintenance discontinuation trial was positive. Based on this data, Dr. Schatzberg said, there “is not much evidence” that patients get further gain beyond an antidepressant alone in the first 24-48 hours following ketamine administration. “It makes me wonder: Should we be continuing to give this drug intranasally beyond 48 hours?” he asked. “The reason I have concern is that in certain cultures, ketamine is a highly abusable drug.”

He and Gerard Sanacora, PhD, MD, addressed the topic in a 2015 opinion piece entitled, “Ketamine: Promising path or false prophecy in the development of novel therapeutics for mood disorders?”

“If we step back for a moment and look at where we are – an intravenously administered agent that is a street drug of abuse, works rapidly, and whose enantiomers are being studied by industry for intranasal use – we should be anxious, “ they wrote (Neuropsychopharmacology. 2015 Jan;40[2]:259-67). “We need to be as careful and conservative as possible and understand how it is acting and rule out the possibility of whether it acts as an opioid.”

Dr. Schatzberg disclosed that he has served a consultant to Alkermes, Avanir, Bracket, Compass, Delpor, Epiodyne, Janssen, Jazz, Lundbeck, McKinsey, Merck, Myriad Genetics, Owl, Neuronetics, Pfizer, Sage, and Sunovion. He has received research funding from Janssen and also holds an ownership interest in Corcept, Dermira, Delpor, Epiodyne, Incyte Genetics, Madrigal, Maerck, Owl Analytics, Seattle Genetics, Titan, and Xhale.

[email protected]

LAS VEGAS – No evidence-based medications are approved for the treatment of avoidant/restrictive food intake disorder, but olanzapine is a sensible option to consider, according to Timothy D. Brewerton, MD.

Doug Brunk/MDedge News

A newly classified disorder in the DSM-5, avoidant/restrictive food intake disorder (ARFID) is an eating/feeding disturbance manifested by the persistent failure to meet appropriate nutritional energy needs associated with one or more of the following factors: significant weight loss or failure to achieve expected growth, significant nutritional deficiency, dependence on enteral feeding or oral nutritional supplements to survive, and marked interference with psychosocial functioning. ARFID “is not better explained by lack of available food or by culturally sanctioned practices,” Dr. Brewerton, an affiliate professor of psychiatry and behavioral sciences at the Medical University of South Carolina, Charleston, said at an annual psychopharmacology update held by the Nevada Psychiatric Association. “It does not occur during the course of anorexia nervosa or bulimia nervosa, and there is no evidence of a disturbance in the way in which one’s body weight or shape is experienced. That’s what distinguishes it from anorexia nervosa.”

He added that, although many cases of ARFID have autism spectrum disorder traits, ARFID “is not attributable to a concurrent medical condition or to mental disorder.” When it does occur, the severity of the feeding disorder far outweighs that of the medical condition.”

Several ARFID case reports have been published in the medical literature, yet no randomized controlled trials exist to date. “Treatment is multidisciplinary and involves cognitive-behavioral therapy, sometimes occupational therapy,” said Dr. Brewerton, who is a founding fellow and former board member of the Academy for Eating Disorders.

He and his colleagues published a report on nine ARFID cases treated in an eating disorders program (J Child Adolesc Psychopharmacol. 2017;27[10]:920-2). The patients were treated with adjunctive olanzapine at a starting dose of 0.9 mg/day and discharge dose 2.8 mg/day. For the starting dose, “you have to break the [olanzapine pills] in pieces,” he said. “You have to have a pill cutter. What you don’t want to do is overwhelm the patient or their family members with oversedation. Too often, people start them with 2.5 mg or 5 mg [of olanzapine] right off the bat.”

The researchers found a significant increase in weight gain pre- versus post-olanzapine treatment (a mean of 3.3 pounds vs. 13.1 pounds, respectively; P less than .04) and BMI-for-weight percentiles (P less than .009). “Patients also had improvements in their Clinical Global Impressions Scale scores and a reduction in their associated anxious, depressive, and cognitive symptoms,” Dr. Brewerton said.

Similar findings were observed in a subsequent Canadian study of six ARFID cases (J Eat Disord. 2018;6:20). Five females and one male were treated with a combination of medical monitoring, family therapy, medication (including olanzapine, fluoxetine and in two cases, cyproheptadine), and CBT. At treatment termination, all six patients had achieved their goal weight.

At the University of California, San Diego, researchers led by Emily Gray, MD, conducted a retrospective chart review of six females and eight males with ARFID who ranged in age from 7 to 23 years. All but one of the 14 patients had a co-occurring diagnosis, including general anxiety disorder, social anxiety, unspecified anxiety, ADHD, major depressive disorder, and autism spectrum disorder (J Am Acad Child Adoles Psychiatry. 2018;57[4]:288-9). They were treated with mirtazapine at a dosing range of 7.5-45 mg per day. The average change in BMI per week rose from 0.10 BMI points per week pretreatment to 0.23 BMI points per week post-treatment (P less than .05).

“These cases illustrate that the judicious use of low-dose olanzapine, when used as an adjunct to other treatment modalities, may facilitate eating, weight gain, and the reduction of anxious, depressive, and cognitive symptoms,” said Dr. Brewerton, who also has a private practice in Charleston.

Dr. Brewerton disclosed that he is a consultant to Monte Nido & Affiliates and Sunovion. He receives royalties from Taylor & Francis and Springer-Verlag, and holds ownership interest in Monte Nido & Affiliates.

[email protected]

LAS VEGAS – No evidence-based medications are approved for the treatment of avoidant/restrictive food intake disorder, but olanzapine is a sensible option to consider, according to Timothy D. Brewerton, MD.

Doug Brunk/MDedge News

A newly classified disorder in the DSM-5, avoidant/restrictive food intake disorder (ARFID) is an eating/feeding disturbance manifested by the persistent failure to meet appropriate nutritional energy needs associated with one or more of the following factors: significant weight loss or failure to achieve expected growth, significant nutritional deficiency, dependence on enteral feeding or oral nutritional supplements to survive, and marked interference with psychosocial functioning. ARFID “is not better explained by lack of available food or by culturally sanctioned practices,” Dr. Brewerton, an affiliate professor of psychiatry and behavioral sciences at the Medical University of South Carolina, Charleston, said at an annual psychopharmacology update held by the Nevada Psychiatric Association. “It does not occur during the course of anorexia nervosa or bulimia nervosa, and there is no evidence of a disturbance in the way in which one’s body weight or shape is experienced. That’s what distinguishes it from anorexia nervosa.”

He added that, although many cases of ARFID have autism spectrum disorder traits, ARFID “is not attributable to a concurrent medical condition or to mental disorder.” When it does occur, the severity of the feeding disorder far outweighs that of the medical condition.”

Several ARFID case reports have been published in the medical literature, yet no randomized controlled trials exist to date. “Treatment is multidisciplinary and involves cognitive-behavioral therapy, sometimes occupational therapy,” said Dr. Brewerton, who is a founding fellow and former board member of the Academy for Eating Disorders.

He and his colleagues published a report on nine ARFID cases treated in an eating disorders program (J Child Adolesc Psychopharmacol. 2017;27[10]:920-2). The patients were treated with adjunctive olanzapine at a starting dose of 0.9 mg/day and discharge dose 2.8 mg/day. For the starting dose, “you have to break the [olanzapine pills] in pieces,” he said. “You have to have a pill cutter. What you don’t want to do is overwhelm the patient or their family members with oversedation. Too often, people start them with 2.5 mg or 5 mg [of olanzapine] right off the bat.”

The researchers found a significant increase in weight gain pre- versus post-olanzapine treatment (a mean of 3.3 pounds vs. 13.1 pounds, respectively; P less than .04) and BMI-for-weight percentiles (P less than .009). “Patients also had improvements in their Clinical Global Impressions Scale scores and a reduction in their associated anxious, depressive, and cognitive symptoms,” Dr. Brewerton said.

Similar findings were observed in a subsequent Canadian study of six ARFID cases (J Eat Disord. 2018;6:20). Five females and one male were treated with a combination of medical monitoring, family therapy, medication (including olanzapine, fluoxetine and in two cases, cyproheptadine), and CBT. At treatment termination, all six patients had achieved their goal weight.

At the University of California, San Diego, researchers led by Emily Gray, MD, conducted a retrospective chart review of six females and eight males with ARFID who ranged in age from 7 to 23 years. All but one of the 14 patients had a co-occurring diagnosis, including general anxiety disorder, social anxiety, unspecified anxiety, ADHD, major depressive disorder, and autism spectrum disorder (J Am Acad Child Adoles Psychiatry. 2018;57[4]:288-9). They were treated with mirtazapine at a dosing range of 7.5-45 mg per day. The average change in BMI per week rose from 0.10 BMI points per week pretreatment to 0.23 BMI points per week post-treatment (P less than .05).

“These cases illustrate that the judicious use of low-dose olanzapine, when used as an adjunct to other treatment modalities, may facilitate eating, weight gain, and the reduction of anxious, depressive, and cognitive symptoms,” said Dr. Brewerton, who also has a private practice in Charleston.

Dr. Brewerton disclosed that he is a consultant to Monte Nido & Affiliates and Sunovion. He receives royalties from Taylor & Francis and Springer-Verlag, and holds ownership interest in Monte Nido & Affiliates.

[email protected]

LAS VEGAS – No evidence-based medications are approved for the treatment of avoidant/restrictive food intake disorder, but olanzapine is a sensible option to consider, according to Timothy D. Brewerton, MD.

Doug Brunk/MDedge News

A newly classified disorder in the DSM-5, avoidant/restrictive food intake disorder (ARFID) is an eating/feeding disturbance manifested by the persistent failure to meet appropriate nutritional energy needs associated with one or more of the following factors: significant weight loss or failure to achieve expected growth, significant nutritional deficiency, dependence on enteral feeding or oral nutritional supplements to survive, and marked interference with psychosocial functioning. ARFID “is not better explained by lack of available food or by culturally sanctioned practices,” Dr. Brewerton, an affiliate professor of psychiatry and behavioral sciences at the Medical University of South Carolina, Charleston, said at an annual psychopharmacology update held by the Nevada Psychiatric Association. “It does not occur during the course of anorexia nervosa or bulimia nervosa, and there is no evidence of a disturbance in the way in which one’s body weight or shape is experienced. That’s what distinguishes it from anorexia nervosa.”

He added that, although many cases of ARFID have autism spectrum disorder traits, ARFID “is not attributable to a concurrent medical condition or to mental disorder.” When it does occur, the severity of the feeding disorder far outweighs that of the medical condition.”

Several ARFID case reports have been published in the medical literature, yet no randomized controlled trials exist to date. “Treatment is multidisciplinary and involves cognitive-behavioral therapy, sometimes occupational therapy,” said Dr. Brewerton, who is a founding fellow and former board member of the Academy for Eating Disorders.

He and his colleagues published a report on nine ARFID cases treated in an eating disorders program (J Child Adolesc Psychopharmacol. 2017;27[10]:920-2). The patients were treated with adjunctive olanzapine at a starting dose of 0.9 mg/day and discharge dose 2.8 mg/day. For the starting dose, “you have to break the [olanzapine pills] in pieces,” he said. “You have to have a pill cutter. What you don’t want to do is overwhelm the patient or their family members with oversedation. Too often, people start them with 2.5 mg or 5 mg [of olanzapine] right off the bat.”

The researchers found a significant increase in weight gain pre- versus post-olanzapine treatment (a mean of 3.3 pounds vs. 13.1 pounds, respectively; P less than .04) and BMI-for-weight percentiles (P less than .009). “Patients also had improvements in their Clinical Global Impressions Scale scores and a reduction in their associated anxious, depressive, and cognitive symptoms,” Dr. Brewerton said.

Similar findings were observed in a subsequent Canadian study of six ARFID cases (J Eat Disord. 2018;6:20). Five females and one male were treated with a combination of medical monitoring, family therapy, medication (including olanzapine, fluoxetine and in two cases, cyproheptadine), and CBT. At treatment termination, all six patients had achieved their goal weight.

At the University of California, San Diego, researchers led by Emily Gray, MD, conducted a retrospective chart review of six females and eight males with ARFID who ranged in age from 7 to 23 years. All but one of the 14 patients had a co-occurring diagnosis, including general anxiety disorder, social anxiety, unspecified anxiety, ADHD, major depressive disorder, and autism spectrum disorder (J Am Acad Child Adoles Psychiatry. 2018;57[4]:288-9). They were treated with mirtazapine at a dosing range of 7.5-45 mg per day. The average change in BMI per week rose from 0.10 BMI points per week pretreatment to 0.23 BMI points per week post-treatment (P less than .05).

“These cases illustrate that the judicious use of low-dose olanzapine, when used as an adjunct to other treatment modalities, may facilitate eating, weight gain, and the reduction of anxious, depressive, and cognitive symptoms,” said Dr. Brewerton, who also has a private practice in Charleston.

Dr. Brewerton disclosed that he is a consultant to Monte Nido & Affiliates and Sunovion. He receives royalties from Taylor & Francis and Springer-Verlag, and holds ownership interest in Monte Nido & Affiliates.

[email protected]

More research suggests that a single infusion of ketamine combined with counseling may help alcohol-dependent patients curb their drinking.
 

In a pilot study of 40 participants, those who were randomly assigned to receive intravenous ketamine plus outpatient motivational enhancement therapy (MET) showed greater abstinence rates, longer time to relapse, and fewer heavy drinking days than did those who received MET plus midazolam.

The findings support a U.K. study published late last year showing that a single dose of intravenous ketamine plus therapy that focused on reactivating drinking-related “maladaptive reward memories” reduced drinking urges and alcohol intake more than just ketamine or a placebo infusion alone (Nat Commun. 2019 Nov 26;10[1]:5187).

“I think the take-home message is that behavioral treatment can be helpful, but there are vulnerabilities that can get in the way,” current study investigator Elias Dakwar, MD, of the New York State Psychiatric Institute, Columbia University, New York, said in an interview.

“It’s an important area of research to understand in order to make behavioral treatments more effective, and ketamine appears to have the properties to address those vulnerabilities,” Dr. Dakwar said.

The study was published in the American Journal of Psychiatry (2019 Dec 2. doi: 10.1176/appi.ajp.2019.19070684).
 

Real-world approach

Pathologic alcohol use is responsible for an estimated 3.8% of all deaths globally, yet current interventions for alcohol use disorder have limited efficacy, the researchers noted.

New treatments with innovative mechanisms would be valuable, they added.

Ketamine is a high-affinity N-methyl-d-aspartate receptor (NMDAR) antagonist.

Previously, research offered “promising results” with the use of ketamine for cocaine use disorder, including increased motivation to quit and decreased craving, Dr. Dakwar noted.

“Those results led us to think about how ketamine might be helpful for other substance use disorders, especially given the overlap in clinical vulnerabilities and epidemiology,” he said.

The study from the U.K. researchers was conducted in 90 patients with harmful drinking behavior but who had not been diagnosed with alcohol use disorder.

Dr. Dakwar noted that this was “a nontreatment study. None of the people there had alcohol use disorder; they were heavy drinkers. Also, the effects there were fairly modest.

“My interest was how to integrate ketamine into a clinical, real-world framework that could be helpful for people,” he added.

The study included 40 participants (52.5% women; 70.3% white; mean age, 53 years) with alcohol dependence whose average consumption was five drinks per day.

All entered a 5-week outpatient program of MET, which involved engaging in strategies to promote motivation and self-directed change.

During the program’s second week, the participants were randomly assigned to received a 52-minute IV infusion of ketamine 0.71 mg/kg (n = 17) or the benzodiazepine midazolam 0.025 mg/kg (n = 23).

This ketamine dose was selected “because it was the highest dose tolerated by participants in preliminary studies,” the researchers reported.

“Midazolam was chosen as the active control because it alters consciousness without any known persistent ... effect on alcohol dependence,” they added.

The “timeline follow back method” was used to assess alcohol use after treatment. Abstinence was confirmed by measuring urine ethyl glucuronide levels with urine toxicology tests.

Other measures included use of a visual analogue scale, the Clinical Institute Withdrawal Assessment, and the modified Perceived Stress Scale.

Primary outcome met

Results showed that 47.1% of the ketamine group and 59.1% of the midazolam group used alcohol during the 21 days after treatment infusion; 17.6% and 40.9%, respectively, had a heavy drinking day.

For the primary outcome measure of alcohol abstinence, the “quadratic effect of time was significant” (P = .004), as was time-by-treatment interaction (P less than .001).

Although the model-estimated proportions of alcohol abstinence remained stable for the ketamine group for 21 days post infusion, the proportions decreased significantly for the control group.

The odds of having a heavy drinking day did not change significantly after treatment for the ketamine group (odds ratio, 0.98; P = .74) but increased significantly with each postinfusion day for the midazolam group (OR, 1.19; P less than .001).

For the ketamine group, time to relapse was also significantly longer (P = .04).

No significant differences were found between the groups in rates of withdrawal, craving, or stress sensitivity.

A new direction?

The most common adverse events after treatment were sedation, seen in 12 members of the midazolam group and in 8 members of the ketamine group, and headache, seen in four and six members, respectively.

Although two ketamine-group members experienced mild agitation for up to 1 hour post infusion, no incidents of persistent psychoactive effects were reported in either group.

No participants who received ketamine dropped out during the study period; among those who received midazolam, six dropped out.

“These preliminary data suggest new directions in integrated pharmacotherapy-behavioral treatments for alcohol use disorder,” the investigators wrote.

However, a larger patient population will be needed in future research in order to “replicate these promising results,” they added.

Dr. Dakwar noted that the time to first drink after treatment was comparable between the groups.

“But what was different in the ketamine group was that they didn’t continue drinking after that first drink. They didn’t initiate heavy drinking, they didn’t relapse, they were able to bounce back and stay with the program,” he said.

“It was surprising but still consistent with the central hypothesis that ketamine provides this opportunity for setting the foundation for the requisite commitment so that, once things become difficult, they’re still able to maintain recovery,” Dr. Dakwar said.

‘Provocative findings’

In an accompanying editorial, Sanjay J. Mathew, MD, of the department of psychiatry and behavioral sciences at Baylor College of Medicine in Houston, and Rebecca B. Price, PhD, of the department of psychiatry at the University of Pittsburgh, noted that ketamine’s effects on abstinence “were robust” in this trial.

“It is also noteworthy that, in spite of recruiting from a population of patients with active and significant substance use history (a group that has routinely been excluded from ketamine trials in depression), no participant showed evidence of new drug-seeking behaviors,” Dr. Mathew and Dr. Price wrote.

“Overall, these findings are provocative and hypothesis generating but certainly not definitive because of the small sample size,” they add.

Other limitations cited include the short follow-up period and the fact that only half of the participants were available for a 6-month follow-up telephone interview. In addition, generalizability was limited because the population did not have additional medical or psychiatric illnesses or additional substance use disorders, the editorialists wrote.

Because of the limitations, the investigators “are appropriately circumspect about the immediate clinical implications of this small pilot study.”

Still, the results “affirm the potential of rational combinatorial approaches for a vexing medical and public health problem,” Dr. Mathew and Dr. Price concluded.

The study was funded by grants from the National Institute on Alcohol Abuse and Alcoholism, the National Institute on Drug Abuse, and the New York State Psychiatric Institute. The study authors and Dr. Price reported no relevant financial relationships. Dr Mathew reported serving as a consultant to or having received research support from several companies, including Alkermes, Allergan, Clexio Biosciences, and Janssen. The original article includes a full list of his disclosures.
 

A version of this article first appeared on Medscape.com.

More research suggests that a single infusion of ketamine combined with counseling may help alcohol-dependent patients curb their drinking.
 

In a pilot study of 40 participants, those who were randomly assigned to receive intravenous ketamine plus outpatient motivational enhancement therapy (MET) showed greater abstinence rates, longer time to relapse, and fewer heavy drinking days than did those who received MET plus midazolam.

The findings support a U.K. study published late last year showing that a single dose of intravenous ketamine plus therapy that focused on reactivating drinking-related “maladaptive reward memories” reduced drinking urges and alcohol intake more than just ketamine or a placebo infusion alone (Nat Commun. 2019 Nov 26;10[1]:5187).

“I think the take-home message is that behavioral treatment can be helpful, but there are vulnerabilities that can get in the way,” current study investigator Elias Dakwar, MD, of the New York State Psychiatric Institute, Columbia University, New York, said in an interview.

“It’s an important area of research to understand in order to make behavioral treatments more effective, and ketamine appears to have the properties to address those vulnerabilities,” Dr. Dakwar said.

The study was published in the American Journal of Psychiatry (2019 Dec 2. doi: 10.1176/appi.ajp.2019.19070684).
 

Real-world approach

Pathologic alcohol use is responsible for an estimated 3.8% of all deaths globally, yet current interventions for alcohol use disorder have limited efficacy, the researchers noted.

New treatments with innovative mechanisms would be valuable, they added.

Ketamine is a high-affinity N-methyl-d-aspartate receptor (NMDAR) antagonist.

Previously, research offered “promising results” with the use of ketamine for cocaine use disorder, including increased motivation to quit and decreased craving, Dr. Dakwar noted.

“Those results led us to think about how ketamine might be helpful for other substance use disorders, especially given the overlap in clinical vulnerabilities and epidemiology,” he said.

The study from the U.K. researchers was conducted in 90 patients with harmful drinking behavior but who had not been diagnosed with alcohol use disorder.

Dr. Dakwar noted that this was “a nontreatment study. None of the people there had alcohol use disorder; they were heavy drinkers. Also, the effects there were fairly modest.

“My interest was how to integrate ketamine into a clinical, real-world framework that could be helpful for people,” he added.

The study included 40 participants (52.5% women; 70.3% white; mean age, 53 years) with alcohol dependence whose average consumption was five drinks per day.

All entered a 5-week outpatient program of MET, which involved engaging in strategies to promote motivation and self-directed change.

During the program’s second week, the participants were randomly assigned to received a 52-minute IV infusion of ketamine 0.71 mg/kg (n = 17) or the benzodiazepine midazolam 0.025 mg/kg (n = 23).

This ketamine dose was selected “because it was the highest dose tolerated by participants in preliminary studies,” the researchers reported.

“Midazolam was chosen as the active control because it alters consciousness without any known persistent ... effect on alcohol dependence,” they added.

The “timeline follow back method” was used to assess alcohol use after treatment. Abstinence was confirmed by measuring urine ethyl glucuronide levels with urine toxicology tests.

Other measures included use of a visual analogue scale, the Clinical Institute Withdrawal Assessment, and the modified Perceived Stress Scale.

Primary outcome met

Results showed that 47.1% of the ketamine group and 59.1% of the midazolam group used alcohol during the 21 days after treatment infusion; 17.6% and 40.9%, respectively, had a heavy drinking day.

For the primary outcome measure of alcohol abstinence, the “quadratic effect of time was significant” (P = .004), as was time-by-treatment interaction (P less than .001).

Although the model-estimated proportions of alcohol abstinence remained stable for the ketamine group for 21 days post infusion, the proportions decreased significantly for the control group.

The odds of having a heavy drinking day did not change significantly after treatment for the ketamine group (odds ratio, 0.98; P = .74) but increased significantly with each postinfusion day for the midazolam group (OR, 1.19; P less than .001).

For the ketamine group, time to relapse was also significantly longer (P = .04).

No significant differences were found between the groups in rates of withdrawal, craving, or stress sensitivity.

A new direction?

The most common adverse events after treatment were sedation, seen in 12 members of the midazolam group and in 8 members of the ketamine group, and headache, seen in four and six members, respectively.

Although two ketamine-group members experienced mild agitation for up to 1 hour post infusion, no incidents of persistent psychoactive effects were reported in either group.

No participants who received ketamine dropped out during the study period; among those who received midazolam, six dropped out.

“These preliminary data suggest new directions in integrated pharmacotherapy-behavioral treatments for alcohol use disorder,” the investigators wrote.

However, a larger patient population will be needed in future research in order to “replicate these promising results,” they added.

Dr. Dakwar noted that the time to first drink after treatment was comparable between the groups.

“But what was different in the ketamine group was that they didn’t continue drinking after that first drink. They didn’t initiate heavy drinking, they didn’t relapse, they were able to bounce back and stay with the program,” he said.

“It was surprising but still consistent with the central hypothesis that ketamine provides this opportunity for setting the foundation for the requisite commitment so that, once things become difficult, they’re still able to maintain recovery,” Dr. Dakwar said.

‘Provocative findings’

In an accompanying editorial, Sanjay J. Mathew, MD, of the department of psychiatry and behavioral sciences at Baylor College of Medicine in Houston, and Rebecca B. Price, PhD, of the department of psychiatry at the University of Pittsburgh, noted that ketamine’s effects on abstinence “were robust” in this trial.

“It is also noteworthy that, in spite of recruiting from a population of patients with active and significant substance use history (a group that has routinely been excluded from ketamine trials in depression), no participant showed evidence of new drug-seeking behaviors,” Dr. Mathew and Dr. Price wrote.

“Overall, these findings are provocative and hypothesis generating but certainly not definitive because of the small sample size,” they add.

Other limitations cited include the short follow-up period and the fact that only half of the participants were available for a 6-month follow-up telephone interview. In addition, generalizability was limited because the population did not have additional medical or psychiatric illnesses or additional substance use disorders, the editorialists wrote.

Because of the limitations, the investigators “are appropriately circumspect about the immediate clinical implications of this small pilot study.”

Still, the results “affirm the potential of rational combinatorial approaches for a vexing medical and public health problem,” Dr. Mathew and Dr. Price concluded.

The study was funded by grants from the National Institute on Alcohol Abuse and Alcoholism, the National Institute on Drug Abuse, and the New York State Psychiatric Institute. The study authors and Dr. Price reported no relevant financial relationships. Dr Mathew reported serving as a consultant to or having received research support from several companies, including Alkermes, Allergan, Clexio Biosciences, and Janssen. The original article includes a full list of his disclosures.
 

A version of this article first appeared on Medscape.com.

More research suggests that a single infusion of ketamine combined with counseling may help alcohol-dependent patients curb their drinking.
 

In a pilot study of 40 participants, those who were randomly assigned to receive intravenous ketamine plus outpatient motivational enhancement therapy (MET) showed greater abstinence rates, longer time to relapse, and fewer heavy drinking days than did those who received MET plus midazolam.

The findings support a U.K. study published late last year showing that a single dose of intravenous ketamine plus therapy that focused on reactivating drinking-related “maladaptive reward memories” reduced drinking urges and alcohol intake more than just ketamine or a placebo infusion alone (Nat Commun. 2019 Nov 26;10[1]:5187).

“I think the take-home message is that behavioral treatment can be helpful, but there are vulnerabilities that can get in the way,” current study investigator Elias Dakwar, MD, of the New York State Psychiatric Institute, Columbia University, New York, said in an interview.

“It’s an important area of research to understand in order to make behavioral treatments more effective, and ketamine appears to have the properties to address those vulnerabilities,” Dr. Dakwar said.

The study was published in the American Journal of Psychiatry (2019 Dec 2. doi: 10.1176/appi.ajp.2019.19070684).
 

Real-world approach

Pathologic alcohol use is responsible for an estimated 3.8% of all deaths globally, yet current interventions for alcohol use disorder have limited efficacy, the researchers noted.

New treatments with innovative mechanisms would be valuable, they added.

Ketamine is a high-affinity N-methyl-d-aspartate receptor (NMDAR) antagonist.

Previously, research offered “promising results” with the use of ketamine for cocaine use disorder, including increased motivation to quit and decreased craving, Dr. Dakwar noted.

“Those results led us to think about how ketamine might be helpful for other substance use disorders, especially given the overlap in clinical vulnerabilities and epidemiology,” he said.

The study from the U.K. researchers was conducted in 90 patients with harmful drinking behavior but who had not been diagnosed with alcohol use disorder.

Dr. Dakwar noted that this was “a nontreatment study. None of the people there had alcohol use disorder; they were heavy drinkers. Also, the effects there were fairly modest.

“My interest was how to integrate ketamine into a clinical, real-world framework that could be helpful for people,” he added.

The study included 40 participants (52.5% women; 70.3% white; mean age, 53 years) with alcohol dependence whose average consumption was five drinks per day.

All entered a 5-week outpatient program of MET, which involved engaging in strategies to promote motivation and self-directed change.

During the program’s second week, the participants were randomly assigned to received a 52-minute IV infusion of ketamine 0.71 mg/kg (n = 17) or the benzodiazepine midazolam 0.025 mg/kg (n = 23).

This ketamine dose was selected “because it was the highest dose tolerated by participants in preliminary studies,” the researchers reported.

“Midazolam was chosen as the active control because it alters consciousness without any known persistent ... effect on alcohol dependence,” they added.

The “timeline follow back method” was used to assess alcohol use after treatment. Abstinence was confirmed by measuring urine ethyl glucuronide levels with urine toxicology tests.

Other measures included use of a visual analogue scale, the Clinical Institute Withdrawal Assessment, and the modified Perceived Stress Scale.

Primary outcome met

Results showed that 47.1% of the ketamine group and 59.1% of the midazolam group used alcohol during the 21 days after treatment infusion; 17.6% and 40.9%, respectively, had a heavy drinking day.

For the primary outcome measure of alcohol abstinence, the “quadratic effect of time was significant” (P = .004), as was time-by-treatment interaction (P less than .001).

Although the model-estimated proportions of alcohol abstinence remained stable for the ketamine group for 21 days post infusion, the proportions decreased significantly for the control group.

The odds of having a heavy drinking day did not change significantly after treatment for the ketamine group (odds ratio, 0.98; P = .74) but increased significantly with each postinfusion day for the midazolam group (OR, 1.19; P less than .001).

For the ketamine group, time to relapse was also significantly longer (P = .04).

No significant differences were found between the groups in rates of withdrawal, craving, or stress sensitivity.

A new direction?

The most common adverse events after treatment were sedation, seen in 12 members of the midazolam group and in 8 members of the ketamine group, and headache, seen in four and six members, respectively.

Although two ketamine-group members experienced mild agitation for up to 1 hour post infusion, no incidents of persistent psychoactive effects were reported in either group.

No participants who received ketamine dropped out during the study period; among those who received midazolam, six dropped out.

“These preliminary data suggest new directions in integrated pharmacotherapy-behavioral treatments for alcohol use disorder,” the investigators wrote.

However, a larger patient population will be needed in future research in order to “replicate these promising results,” they added.

Dr. Dakwar noted that the time to first drink after treatment was comparable between the groups.

“But what was different in the ketamine group was that they didn’t continue drinking after that first drink. They didn’t initiate heavy drinking, they didn’t relapse, they were able to bounce back and stay with the program,” he said.

“It was surprising but still consistent with the central hypothesis that ketamine provides this opportunity for setting the foundation for the requisite commitment so that, once things become difficult, they’re still able to maintain recovery,” Dr. Dakwar said.

‘Provocative findings’

In an accompanying editorial, Sanjay J. Mathew, MD, of the department of psychiatry and behavioral sciences at Baylor College of Medicine in Houston, and Rebecca B. Price, PhD, of the department of psychiatry at the University of Pittsburgh, noted that ketamine’s effects on abstinence “were robust” in this trial.

“It is also noteworthy that, in spite of recruiting from a population of patients with active and significant substance use history (a group that has routinely been excluded from ketamine trials in depression), no participant showed evidence of new drug-seeking behaviors,” Dr. Mathew and Dr. Price wrote.

“Overall, these findings are provocative and hypothesis generating but certainly not definitive because of the small sample size,” they add.

Other limitations cited include the short follow-up period and the fact that only half of the participants were available for a 6-month follow-up telephone interview. In addition, generalizability was limited because the population did not have additional medical or psychiatric illnesses or additional substance use disorders, the editorialists wrote.

Because of the limitations, the investigators “are appropriately circumspect about the immediate clinical implications of this small pilot study.”

Still, the results “affirm the potential of rational combinatorial approaches for a vexing medical and public health problem,” Dr. Mathew and Dr. Price concluded.

The study was funded by grants from the National Institute on Alcohol Abuse and Alcoholism, the National Institute on Drug Abuse, and the New York State Psychiatric Institute. The study authors and Dr. Price reported no relevant financial relationships. Dr Mathew reported serving as a consultant to or having received research support from several companies, including Alkermes, Allergan, Clexio Biosciences, and Janssen. The original article includes a full list of his disclosures.
 

A version of this article first appeared on Medscape.com.

As I write this, the 2019 novel coronavirus* continues to spread, exceeding 59,000 cases and 1,300 deaths worldwide. With it spreads fear. In the modern world of social media, misinformation spreads even faster than disease.

Delpixel/Shutterstock

The news about a novel and deadly illness crowds out more substantial worries. Humans are not particularly good at assessing risk or responding rationally and consistently to it. Risk is hard to fully define. If you look up “risk” in Merriam Webster’s online dictionary, you get the simple definition of “possibility of loss or injury; peril.” If you look up risk in Wikipedia, you get 12 pages of explanation and 8 more pages of links and references.

People handle risk differently. Some people are more risk adverse than others. Some get a pleasurable thrill from risk, whether a slot machine or a parachute jump. Most people really don’t comprehend small probabilities, with tens of billions of dollars spent annually on U.S. lotteries.

Because 98% of people who get COVID-19 are recovering, this is not an extinction-level event or the zombie apocalypse. It is a major health hazard, and one where morbidity and mortality might be assuaged by an early and effective public health response, including the population’s adoption of good habits such as hand washing, cough etiquette, and staying home when ill. But fear, discrimination, and misinformation may do more damage than the virus itself.

Three key factors may help reduce the fear factor.

One key factor is accurate communication of health information to the public. This has been severely harmed in the last few years by the promotion of gossip on social media, such as Facebook, within newsfeeds without any vetting, along with a smaller component of deliberate misinformation from untraceable sources. Compare this situation with the decision in May 1988 when Surgeon General C. Everett Koop chose to snail mail a brochure on AIDS to every household in America. It was unprecedented. One element of this communication is the public’s belief that government and health care officials will responsibly and timely convey the information. There are accusations that the Chinese government initially impeded early warnings about COVID-19. Dr. Koop, to his great credit and lifesaving leadership, overcame queasiness within the Reagan administration about issues of morality and taste in discussing some of the HIV information. Alas, no similar leadership occurred in the decade of the 2010s when deaths from the opioid epidemic in the United States skyrocketed to claim more lives annually than car accidents or suicide.

A second factor is the credibility of the scientists. Antivaxxers, climate change deniers, and mercenary scientists have severely damaged that credibility of science, compared with the trust in scientists 50 years ago during the Apollo moon shot.

A third factor is perspective. Poor journalism and clickbait can focus excessively on the rare events as news. Airline crashes make the front page while fatal car accidents, claiming a hundred times more lives annually, don’t even merit a story in local media. Someone wins the lottery weekly but few pay attention to those suffering from gambling debts.

Influenza is killing many times more people than the 2019 novel coronavirus, but the news is focused on cruise ships. In the United States, influenza annually will strike tens of millions, with about 10 per 1,000 hospitalized and 0.5 per 1,000 dying. The novel coronavirus is more lethal. SARS (a coronavirus epidemic in 2003) had 8,000 cases with a mortality rate of 96 per 1,000 while the novel 2019 strain so far is killing about 20 per 1,000. That value may be an overestimate, because there may be a significant fraction of COVID-19 patients with symptoms mild enough that they do not seek medical care and do not get tested and counted.

For perspective, in 1952 the United States reported 50,000 cases of polio (meningitis or paralytic) annually with 3,000 deaths. As many as 95% of cases of poliovirus infection have no or mild symptoms and would not have been reported, so the case fatality rate estimate is skewed. In the 1950s, the United States averaged about 500,000 cases of measles per year, with about 500 deaths annually for a case fatality rate of about 1 per 1,000 in a population that was well nourished with good medical care. In malnourished children without access to modern health care, the case fatality rate can be as high as 100 per 1,000, which is why globally measles killed 142,000 people in 2018, a substantial improvement from 536,000 deaths globally in 2000, but still a leading killer of children worldwide. Vaccines had reduced the annual death toll of polio and measles in the U.S. to zero.

In comparison, in this country the annual incidences are about 70,000 overdose deaths, 50,000 suicides, and 40,000 traffic deaths.

Reassurance is the most common product sold by pediatricians. We look for low-probability, high-impact bad things. Usually we don’t find them and can reassure parents that the child will be okay. Sometimes we spot a higher-risk situation and intervene. My job is to worry professionally so that parents can worry less.

COVID-19 worries me, but irrational people worry me more. The real enemies are fear, disinformation, discrimination, and economic warfare.
 

Dr. Powell is a pediatric hospitalist and clinical ethics consultant living in St. Louis. Email him at [email protected].

*This article was updated 2/21/2020.

As I write this, the 2019 novel coronavirus* continues to spread, exceeding 59,000 cases and 1,300 deaths worldwide. With it spreads fear. In the modern world of social media, misinformation spreads even faster than disease.

Delpixel/Shutterstock

The news about a novel and deadly illness crowds out more substantial worries. Humans are not particularly good at assessing risk or responding rationally and consistently to it. Risk is hard to fully define. If you look up “risk” in Merriam Webster’s online dictionary, you get the simple definition of “possibility of loss or injury; peril.” If you look up risk in Wikipedia, you get 12 pages of explanation and 8 more pages of links and references.

People handle risk differently. Some people are more risk adverse than others. Some get a pleasurable thrill from risk, whether a slot machine or a parachute jump. Most people really don’t comprehend small probabilities, with tens of billions of dollars spent annually on U.S. lotteries.

Because 98% of people who get COVID-19 are recovering, this is not an extinction-level event or the zombie apocalypse. It is a major health hazard, and one where morbidity and mortality might be assuaged by an early and effective public health response, including the population’s adoption of good habits such as hand washing, cough etiquette, and staying home when ill. But fear, discrimination, and misinformation may do more damage than the virus itself.

Three key factors may help reduce the fear factor.

One key factor is accurate communication of health information to the public. This has been severely harmed in the last few years by the promotion of gossip on social media, such as Facebook, within newsfeeds without any vetting, along with a smaller component of deliberate misinformation from untraceable sources. Compare this situation with the decision in May 1988 when Surgeon General C. Everett Koop chose to snail mail a brochure on AIDS to every household in America. It was unprecedented. One element of this communication is the public’s belief that government and health care officials will responsibly and timely convey the information. There are accusations that the Chinese government initially impeded early warnings about COVID-19. Dr. Koop, to his great credit and lifesaving leadership, overcame queasiness within the Reagan administration about issues of morality and taste in discussing some of the HIV information. Alas, no similar leadership occurred in the decade of the 2010s when deaths from the opioid epidemic in the United States skyrocketed to claim more lives annually than car accidents or suicide.

A second factor is the credibility of the scientists. Antivaxxers, climate change deniers, and mercenary scientists have severely damaged that credibility of science, compared with the trust in scientists 50 years ago during the Apollo moon shot.

A third factor is perspective. Poor journalism and clickbait can focus excessively on the rare events as news. Airline crashes make the front page while fatal car accidents, claiming a hundred times more lives annually, don’t even merit a story in local media. Someone wins the lottery weekly but few pay attention to those suffering from gambling debts.

Influenza is killing many times more people than the 2019 novel coronavirus, but the news is focused on cruise ships. In the United States, influenza annually will strike tens of millions, with about 10 per 1,000 hospitalized and 0.5 per 1,000 dying. The novel coronavirus is more lethal. SARS (a coronavirus epidemic in 2003) had 8,000 cases with a mortality rate of 96 per 1,000 while the novel 2019 strain so far is killing about 20 per 1,000. That value may be an overestimate, because there may be a significant fraction of COVID-19 patients with symptoms mild enough that they do not seek medical care and do not get tested and counted.

For perspective, in 1952 the United States reported 50,000 cases of polio (meningitis or paralytic) annually with 3,000 deaths. As many as 95% of cases of poliovirus infection have no or mild symptoms and would not have been reported, so the case fatality rate estimate is skewed. In the 1950s, the United States averaged about 500,000 cases of measles per year, with about 500 deaths annually for a case fatality rate of about 1 per 1,000 in a population that was well nourished with good medical care. In malnourished children without access to modern health care, the case fatality rate can be as high as 100 per 1,000, which is why globally measles killed 142,000 people in 2018, a substantial improvement from 536,000 deaths globally in 2000, but still a leading killer of children worldwide. Vaccines had reduced the annual death toll of polio and measles in the U.S. to zero.

In comparison, in this country the annual incidences are about 70,000 overdose deaths, 50,000 suicides, and 40,000 traffic deaths.

Reassurance is the most common product sold by pediatricians. We look for low-probability, high-impact bad things. Usually we don’t find them and can reassure parents that the child will be okay. Sometimes we spot a higher-risk situation and intervene. My job is to worry professionally so that parents can worry less.

COVID-19 worries me, but irrational people worry me more. The real enemies are fear, disinformation, discrimination, and economic warfare.
 

Dr. Powell is a pediatric hospitalist and clinical ethics consultant living in St. Louis. Email him at [email protected].

*This article was updated 2/21/2020.

As I write this, the 2019 novel coronavirus* continues to spread, exceeding 59,000 cases and 1,300 deaths worldwide. With it spreads fear. In the modern world of social media, misinformation spreads even faster than disease.

Delpixel/Shutterstock

The news about a novel and deadly illness crowds out more substantial worries. Humans are not particularly good at assessing risk or responding rationally and consistently to it. Risk is hard to fully define. If you look up “risk” in Merriam Webster’s online dictionary, you get the simple definition of “possibility of loss or injury; peril.” If you look up risk in Wikipedia, you get 12 pages of explanation and 8 more pages of links and references.

People handle risk differently. Some people are more risk adverse than others. Some get a pleasurable thrill from risk, whether a slot machine or a parachute jump. Most people really don’t comprehend small probabilities, with tens of billions of dollars spent annually on U.S. lotteries.

Because 98% of people who get COVID-19 are recovering, this is not an extinction-level event or the zombie apocalypse. It is a major health hazard, and one where morbidity and mortality might be assuaged by an early and effective public health response, including the population’s adoption of good habits such as hand washing, cough etiquette, and staying home when ill. But fear, discrimination, and misinformation may do more damage than the virus itself.

Three key factors may help reduce the fear factor.

One key factor is accurate communication of health information to the public. This has been severely harmed in the last few years by the promotion of gossip on social media, such as Facebook, within newsfeeds without any vetting, along with a smaller component of deliberate misinformation from untraceable sources. Compare this situation with the decision in May 1988 when Surgeon General C. Everett Koop chose to snail mail a brochure on AIDS to every household in America. It was unprecedented. One element of this communication is the public’s belief that government and health care officials will responsibly and timely convey the information. There are accusations that the Chinese government initially impeded early warnings about COVID-19. Dr. Koop, to his great credit and lifesaving leadership, overcame queasiness within the Reagan administration about issues of morality and taste in discussing some of the HIV information. Alas, no similar leadership occurred in the decade of the 2010s when deaths from the opioid epidemic in the United States skyrocketed to claim more lives annually than car accidents or suicide.

A second factor is the credibility of the scientists. Antivaxxers, climate change deniers, and mercenary scientists have severely damaged that credibility of science, compared with the trust in scientists 50 years ago during the Apollo moon shot.

A third factor is perspective. Poor journalism and clickbait can focus excessively on the rare events as news. Airline crashes make the front page while fatal car accidents, claiming a hundred times more lives annually, don’t even merit a story in local media. Someone wins the lottery weekly but few pay attention to those suffering from gambling debts.

Influenza is killing many times more people than the 2019 novel coronavirus, but the news is focused on cruise ships. In the United States, influenza annually will strike tens of millions, with about 10 per 1,000 hospitalized and 0.5 per 1,000 dying. The novel coronavirus is more lethal. SARS (a coronavirus epidemic in 2003) had 8,000 cases with a mortality rate of 96 per 1,000 while the novel 2019 strain so far is killing about 20 per 1,000. That value may be an overestimate, because there may be a significant fraction of COVID-19 patients with symptoms mild enough that they do not seek medical care and do not get tested and counted.

For perspective, in 1952 the United States reported 50,000 cases of polio (meningitis or paralytic) annually with 3,000 deaths. As many as 95% of cases of poliovirus infection have no or mild symptoms and would not have been reported, so the case fatality rate estimate is skewed. In the 1950s, the United States averaged about 500,000 cases of measles per year, with about 500 deaths annually for a case fatality rate of about 1 per 1,000 in a population that was well nourished with good medical care. In malnourished children without access to modern health care, the case fatality rate can be as high as 100 per 1,000, which is why globally measles killed 142,000 people in 2018, a substantial improvement from 536,000 deaths globally in 2000, but still a leading killer of children worldwide. Vaccines had reduced the annual death toll of polio and measles in the U.S. to zero.

In comparison, in this country the annual incidences are about 70,000 overdose deaths, 50,000 suicides, and 40,000 traffic deaths.

Reassurance is the most common product sold by pediatricians. We look for low-probability, high-impact bad things. Usually we don’t find them and can reassure parents that the child will be okay. Sometimes we spot a higher-risk situation and intervene. My job is to worry professionally so that parents can worry less.

COVID-19 worries me, but irrational people worry me more. The real enemies are fear, disinformation, discrimination, and economic warfare.
 

Dr. Powell is a pediatric hospitalist and clinical ethics consultant living in St. Louis. Email him at [email protected].

*This article was updated 2/21/2020.

Psychiatrists do better compared with those in most specialties in finding happiness at work, according to Medscape’s 2020 Lifestyle, Happiness, and Burnout Report.

About 32% of psychiatrists reported being happy at work, according to the Medscape survey, though they lagged well behind dermatologists, who were the most satisfied with their work lives. In terms of happiness outside the office, psychiatrists were in the middle of the pack with 51% reporting that they were happy.

Somewhat fewer psychiatrists reported being burned out, compared with physicians overall, at 37% versus 41%. The biggest contributing factors to psychiatrist burnout were an overabundance of bureaucratic tasks (63%), increased time devoted to EHRs (34%), and a lack of respect from colleagues in the workplace (32%).

Psychiatrists most commonly dealt with burnout by isolating themselves from others (57%), sleeping (43%), and talking with family/friends (42%). Just under half of psychiatrists took 3-4 weeks’ vacation, compared with 44% of all physicians, and 33% took less than 3 weeks’ vacation.

More than three-quarters of psychiatrists reported that they had never felt suicidal, but 17% said that they had contemplated suicide and 1% reported that they had attempted suicide. About 45% said that they were currently seeking professional help, planning to seek help, or had used help in the past to deal with burnout or depression; 48% said that they were not planning to seek help and had not done so in the past.

In an interview, Carol A. Bernstein, MD, said it is challenging to find the meaning in these survey results.

“The challenge with surveys that measure burnout is that the drivers may be somewhat different in different specialties. I am less interested in looking at ‘who has it worse’ than I am at trying to address those systemic factors that are important for all physicians, regardless of specialty,” said Dr. Bernstein of Montefiore Medical Center/Albert Einstein College of Medicine, New York.

“The survey noted some of these factors: the increased burden of regulation and bureaucratic tasks, an EHR that was designed for billing and scheduling – not for taking care of patients – and challenges of professionalism in the workplace. These are issues that we must address for the benefit of all health care providers and patients.”

Dr. Bernstein, a past president of the American Psychiatric Association, is vice chair for faculty development and well-being at Montefiore/Albert Einstein. She is a professor in the departments of psychiatry and behavioral sciences, and obstetrics/gynecology & women’s health.

The Medscape survey was conducted from June 25 to Sept. 19, 2019, and involved 15,181 physicians.

[email protected]

Psychiatrists do better compared with those in most specialties in finding happiness at work, according to Medscape’s 2020 Lifestyle, Happiness, and Burnout Report.

About 32% of psychiatrists reported being happy at work, according to the Medscape survey, though they lagged well behind dermatologists, who were the most satisfied with their work lives. In terms of happiness outside the office, psychiatrists were in the middle of the pack with 51% reporting that they were happy.

Somewhat fewer psychiatrists reported being burned out, compared with physicians overall, at 37% versus 41%. The biggest contributing factors to psychiatrist burnout were an overabundance of bureaucratic tasks (63%), increased time devoted to EHRs (34%), and a lack of respect from colleagues in the workplace (32%).

Psychiatrists most commonly dealt with burnout by isolating themselves from others (57%), sleeping (43%), and talking with family/friends (42%). Just under half of psychiatrists took 3-4 weeks’ vacation, compared with 44% of all physicians, and 33% took less than 3 weeks’ vacation.

More than three-quarters of psychiatrists reported that they had never felt suicidal, but 17% said that they had contemplated suicide and 1% reported that they had attempted suicide. About 45% said that they were currently seeking professional help, planning to seek help, or had used help in the past to deal with burnout or depression; 48% said that they were not planning to seek help and had not done so in the past.

In an interview, Carol A. Bernstein, MD, said it is challenging to find the meaning in these survey results.

“The challenge with surveys that measure burnout is that the drivers may be somewhat different in different specialties. I am less interested in looking at ‘who has it worse’ than I am at trying to address those systemic factors that are important for all physicians, regardless of specialty,” said Dr. Bernstein of Montefiore Medical Center/Albert Einstein College of Medicine, New York.

“The survey noted some of these factors: the increased burden of regulation and bureaucratic tasks, an EHR that was designed for billing and scheduling – not for taking care of patients – and challenges of professionalism in the workplace. These are issues that we must address for the benefit of all health care providers and patients.”

Dr. Bernstein, a past president of the American Psychiatric Association, is vice chair for faculty development and well-being at Montefiore/Albert Einstein. She is a professor in the departments of psychiatry and behavioral sciences, and obstetrics/gynecology & women’s health.

The Medscape survey was conducted from June 25 to Sept. 19, 2019, and involved 15,181 physicians.

[email protected]

Psychiatrists do better compared with those in most specialties in finding happiness at work, according to Medscape’s 2020 Lifestyle, Happiness, and Burnout Report.

About 32% of psychiatrists reported being happy at work, according to the Medscape survey, though they lagged well behind dermatologists, who were the most satisfied with their work lives. In terms of happiness outside the office, psychiatrists were in the middle of the pack with 51% reporting that they were happy.

Somewhat fewer psychiatrists reported being burned out, compared with physicians overall, at 37% versus 41%. The biggest contributing factors to psychiatrist burnout were an overabundance of bureaucratic tasks (63%), increased time devoted to EHRs (34%), and a lack of respect from colleagues in the workplace (32%).

Psychiatrists most commonly dealt with burnout by isolating themselves from others (57%), sleeping (43%), and talking with family/friends (42%). Just under half of psychiatrists took 3-4 weeks’ vacation, compared with 44% of all physicians, and 33% took less than 3 weeks’ vacation.

More than three-quarters of psychiatrists reported that they had never felt suicidal, but 17% said that they had contemplated suicide and 1% reported that they had attempted suicide. About 45% said that they were currently seeking professional help, planning to seek help, or had used help in the past to deal with burnout or depression; 48% said that they were not planning to seek help and had not done so in the past.

In an interview, Carol A. Bernstein, MD, said it is challenging to find the meaning in these survey results.

“The challenge with surveys that measure burnout is that the drivers may be somewhat different in different specialties. I am less interested in looking at ‘who has it worse’ than I am at trying to address those systemic factors that are important for all physicians, regardless of specialty,” said Dr. Bernstein of Montefiore Medical Center/Albert Einstein College of Medicine, New York.

“The survey noted some of these factors: the increased burden of regulation and bureaucratic tasks, an EHR that was designed for billing and scheduling – not for taking care of patients – and challenges of professionalism in the workplace. These are issues that we must address for the benefit of all health care providers and patients.”

Dr. Bernstein, a past president of the American Psychiatric Association, is vice chair for faculty development and well-being at Montefiore/Albert Einstein. She is a professor in the departments of psychiatry and behavioral sciences, and obstetrics/gynecology & women’s health.

The Medscape survey was conducted from June 25 to Sept. 19, 2019, and involved 15,181 physicians.

[email protected]

The Trump administration on Feb. 10 argued for cutting spending for a federal agency at the forefront of the efforts to combat the coronavirus, while also seeking to slow spending in certain parts of the Medicare and Medicaid programs.

President Donald Trump presented his fiscal 2021 request to Congress for refilling the coffers of federal agencies. In any administration, an annual budget serves only as a political blueprint, as the White House document itself makes no changes in federal spending.

In Mr. Trump’s case, several of his requests for agencies within the Department of Health & Human Services run counter to recent budget trends. Republicans and Democrats in Congress have worked together in recent years to increase budgets for major federal health agencies.

But Mr. Trump asked Congress to cut annual budget authority for the National Institute of Allergy and Infectious Diseases by $430 million to $5.446 billion for fiscal 2021. In contrast, Congress has raised the annual budget for NIAID, a key agency in combating the coronavirus, from $5.545 billion in fiscal 2019 to $5.876 billion in fiscal 2020, which began in October, according to an HHS summary of Mr. Trump’s request.

For the Centers for Disease Control and Prevention, which is central to the battle against the coronavirus, Mr. Trump proposed a drop in discretionary funding to $5.627 billion. In contrast, Congress raised the CDC budget from $6.544 billion in fiscal 2019 to $6.917 in fiscal 2020.

Mr. Trump also wants to cut $559 million from the budget of the National Cancer Institute, dropping it to $5.881 billion in fiscal 2021. In contrast, Congress raised NCI’s budget from $6.121 billion in fiscal 2019 to $6.440 billion in fiscal 2020.

Mr. Trump requested a $2.6 billion reduction in the National Institutes of Health’s total discretionary budget, seeking to drop it to $37.70 billion. In contrast, Congress raised NIH’s budget from $37.887 in fiscal 2019 to $40.304 billion in fiscal 2020.

Mr. Trump’s budget proposal also includes an estimate of $152 billion in savings over a decade for Medicaid through the implementation of what the administration calls “community engagement” requirements.

The Trump administration has been at odds with Democrats for years about whether work requirements should be attached to Medicaid. “Well-designed community engagement incentives have great potential to improve health and well-being while empowering beneficiaries to rise out of poverty,” HHS said in a budget document.

Yet researchers last year reported that Arkansas’ attempt to attach work requirements to Medicaid caused almost 17,000 adults to lose this health care coverage within the first 6 months, and there was no significant difference in employment.

The researchers say this loss of coverage was partly a result of bureaucratic obstacles and confusion about the new rules. In June 2018, Arkansas became the first state to implement work requirements for Medicaid, Benjamin D. Sommers, MD, PhD, of the Harvard T.H. Chan School of Public Health, Boston, and colleagues wrote in the New England Journal of Medicine (2019 Sep 12;381[11]:1073-82). 

Budget ‘would thwart’ progress

A few medical groups on Monday quickly criticized Mr. Trump’s proposals.

“In a time where our nation continues to face significant public health challenges — including 2019 novel coronavirus, climate change, gun violence, and costly chronic diseases such as heart disease and cancer – the administration should be investing more resources in better health, not cutting federal health budgets,” said Georges C. Benjamin, MD, executive director of the American Public Health Association, in a statement.

David J. Skorton, MD, chief executive and president of the Association of American Medical Colleges (AAMC) also urged increased investment in fighting disease.

“We must continue the bipartisan budget trajectory set forth by Congress over the last several years, not reverse course,” Dr. Skorton said in a statement.

Mr. Trump’s proposed cuts in medical research “would thwart scientific progress on strategies to prevent, diagnose, treat, and cure medical conditions that affect countless patients nationwide,” he said.

In total, the new 2021 appropriations for HHS would fall by $9.46 billion to $85.667 billion under Mr. Trump’s proposal. Appropriations, also called discretionary budget authority, represents the operating budgets for federal agencies. These are decided through annual spending bills.

Congress has separate sets of laws for handling payments the federal government makes through Medicare and Medicaid. These are known as mandatory spending.

‘Untenable cuts’

AAMC’s Dr. Skorton also objected to what he termed Mr. Trump’s bid “to reduce and consolidate Medicare, Medicaid, and children’s hospital graduate medical education into a single grant program.”

This would force teaching hospitals to absorb $52 billion in “untenable cuts,” he said.

“The proposal ignores the intent of the Medicare GME program, which is to ensure an adequate physician workforce to care for Medicare beneficiaries and support the critical patient care missions of America’s teaching hospitals,” Dr. Skorton said.

The budget also seeks cuts to Medicaid, which come in addition to the administration’s “recent proposals to scale back Medicaid coverage,” Dr. Skorton said.

“More than 26% of all Medicaid hospitalizations occur at AAMC-member teaching hospitals, even though these institutions represent only 5% of all hospitals,” Dr. Skorton said. “Each of the administration’s proposals on their own would be devastating for patients – and combined, they would be disastrous.”

Rick Pollack, the chief executive and president of the American Hospital Association, described Mr. Trump’s fiscal 2021 proposal as another bid to undermine medical care in the United States.

“Every year, we adapt to a constantly changing environment, but every year, the administration aims to gut our nation’s health care infrastructure,” Mr. Pollack said in a statement.

In it, he noted that about one in five people in America depend on Medicaid, with children accounting for a large proportion of those covered by the state-federal program.

“The budget’s proposal on Medicaid financing and service delivery would cut hundreds of billions of dollars from the Medicaid program annually,” Mr. Pollack said.

He also objected to “hundreds of billions of proposed reductions to Medicare” endorsed by Mr. Trump.

Medical malpractice overhaul

The Trump administration also offered many suggestions for changing federal laws to reduce health care spending. Among these was a proposed overhaul of the approach to medical malpractice cases.

The president’s budget proposal estimates $40 billion in savings over a decade from steps to limit medical liability, according to a report from the Office of Management and Budget (OMB).

“The current medical liability system does not work for patients or providers, nor does it promote high-quality, evidence-based care,” OMB said. “Providers practice with a threat of potentially frivolous lawsuits, and injured patients often do not receive just compensation for their injuries.”

Mr. Trump’s fiscal 2021 budget calls for a cap on noneconomic damage awards of $250,000, which would increase with inflation over time, and a 3-year statute of limitations. Under this plan, courts could also modify attorney’s fee arrangements. HHS could provide guidance to states on how to create expert panels and administrative health care tribunals to review medical liability.

These steps would lead to lower health care spending, with clinicians dropping “defensive medicine practices,” OMB said. That would benefit the Medicare and Medicaid programs as well as lowering costs of health insurance in general.

Mr. Trump’s fiscal 2021 budget also includes a series of proposals for Medicare that it estimates would, in aggregate, save $755.5 billion over a decade.

Site-neutral policy

A large chunk of the estimated Medicare savings in Mr. Trump’s fiscal 2021 health budget would come from lowering payments to hospitals for services provided in their outpatient and physician offices.

In the fiscal 2021 proposal, HHS noted that “Medicare generally pays on-campus hospital outpatient departments substantially more than physician offices for the same services.”

Mr. Trump’s budget proposal seeks a more expansive shift to what’s called a “site-neutral” payment for services delivered in hospital outpatient programs or physician offices. This would bring these payments more in line with those made to independent physician practices.

“This proposal would eliminate the often significant disparity between what Medicare pays in these different settings for the same services,” HHS said in the budget summary.

HHS estimated this change in policy would generate $117.2 billion in savings over a decade. Combined with saving from medical malpractice reforms, the Trump administration estimates these two moves combined could save about $164 billion over a decade.

The site-neutral policy has been a legal battleground, with hospital and physician groups winning a round last year. 

Another Medicare proposal included in Mr. Trump’s fiscal 2021 budget homes in on this issue for cases where a hospital owns a physician office. Medicare now pays most off-campus hospital outpatient departments higher rates than the program’s physician fee schedule dictates for the same services.

Switching to a site-neutral policy for these hospital-owned physician offices would result in $47.2 billion in savings over a decade, HHS said in the budget document.
 

This article first appeared on Medscape.com.

The Trump administration on Feb. 10 argued for cutting spending for a federal agency at the forefront of the efforts to combat the coronavirus, while also seeking to slow spending in certain parts of the Medicare and Medicaid programs.

President Donald Trump presented his fiscal 2021 request to Congress for refilling the coffers of federal agencies. In any administration, an annual budget serves only as a political blueprint, as the White House document itself makes no changes in federal spending.

In Mr. Trump’s case, several of his requests for agencies within the Department of Health & Human Services run counter to recent budget trends. Republicans and Democrats in Congress have worked together in recent years to increase budgets for major federal health agencies.

But Mr. Trump asked Congress to cut annual budget authority for the National Institute of Allergy and Infectious Diseases by $430 million to $5.446 billion for fiscal 2021. In contrast, Congress has raised the annual budget for NIAID, a key agency in combating the coronavirus, from $5.545 billion in fiscal 2019 to $5.876 billion in fiscal 2020, which began in October, according to an HHS summary of Mr. Trump’s request.

For the Centers for Disease Control and Prevention, which is central to the battle against the coronavirus, Mr. Trump proposed a drop in discretionary funding to $5.627 billion. In contrast, Congress raised the CDC budget from $6.544 billion in fiscal 2019 to $6.917 in fiscal 2020.

Mr. Trump also wants to cut $559 million from the budget of the National Cancer Institute, dropping it to $5.881 billion in fiscal 2021. In contrast, Congress raised NCI’s budget from $6.121 billion in fiscal 2019 to $6.440 billion in fiscal 2020.

Mr. Trump requested a $2.6 billion reduction in the National Institutes of Health’s total discretionary budget, seeking to drop it to $37.70 billion. In contrast, Congress raised NIH’s budget from $37.887 in fiscal 2019 to $40.304 billion in fiscal 2020.

Mr. Trump’s budget proposal also includes an estimate of $152 billion in savings over a decade for Medicaid through the implementation of what the administration calls “community engagement” requirements.

The Trump administration has been at odds with Democrats for years about whether work requirements should be attached to Medicaid. “Well-designed community engagement incentives have great potential to improve health and well-being while empowering beneficiaries to rise out of poverty,” HHS said in a budget document.

Yet researchers last year reported that Arkansas’ attempt to attach work requirements to Medicaid caused almost 17,000 adults to lose this health care coverage within the first 6 months, and there was no significant difference in employment.

The researchers say this loss of coverage was partly a result of bureaucratic obstacles and confusion about the new rules. In June 2018, Arkansas became the first state to implement work requirements for Medicaid, Benjamin D. Sommers, MD, PhD, of the Harvard T.H. Chan School of Public Health, Boston, and colleagues wrote in the New England Journal of Medicine (2019 Sep 12;381[11]:1073-82). 

Budget ‘would thwart’ progress

A few medical groups on Monday quickly criticized Mr. Trump’s proposals.

“In a time where our nation continues to face significant public health challenges — including 2019 novel coronavirus, climate change, gun violence, and costly chronic diseases such as heart disease and cancer – the administration should be investing more resources in better health, not cutting federal health budgets,” said Georges C. Benjamin, MD, executive director of the American Public Health Association, in a statement.

David J. Skorton, MD, chief executive and president of the Association of American Medical Colleges (AAMC) also urged increased investment in fighting disease.

“We must continue the bipartisan budget trajectory set forth by Congress over the last several years, not reverse course,” Dr. Skorton said in a statement.

Mr. Trump’s proposed cuts in medical research “would thwart scientific progress on strategies to prevent, diagnose, treat, and cure medical conditions that affect countless patients nationwide,” he said.

In total, the new 2021 appropriations for HHS would fall by $9.46 billion to $85.667 billion under Mr. Trump’s proposal. Appropriations, also called discretionary budget authority, represents the operating budgets for federal agencies. These are decided through annual spending bills.

Congress has separate sets of laws for handling payments the federal government makes through Medicare and Medicaid. These are known as mandatory spending.

‘Untenable cuts’

AAMC’s Dr. Skorton also objected to what he termed Mr. Trump’s bid “to reduce and consolidate Medicare, Medicaid, and children’s hospital graduate medical education into a single grant program.”

This would force teaching hospitals to absorb $52 billion in “untenable cuts,” he said.

“The proposal ignores the intent of the Medicare GME program, which is to ensure an adequate physician workforce to care for Medicare beneficiaries and support the critical patient care missions of America’s teaching hospitals,” Dr. Skorton said.

The budget also seeks cuts to Medicaid, which come in addition to the administration’s “recent proposals to scale back Medicaid coverage,” Dr. Skorton said.

“More than 26% of all Medicaid hospitalizations occur at AAMC-member teaching hospitals, even though these institutions represent only 5% of all hospitals,” Dr. Skorton said. “Each of the administration’s proposals on their own would be devastating for patients – and combined, they would be disastrous.”

Rick Pollack, the chief executive and president of the American Hospital Association, described Mr. Trump’s fiscal 2021 proposal as another bid to undermine medical care in the United States.

“Every year, we adapt to a constantly changing environment, but every year, the administration aims to gut our nation’s health care infrastructure,” Mr. Pollack said in a statement.

In it, he noted that about one in five people in America depend on Medicaid, with children accounting for a large proportion of those covered by the state-federal program.

“The budget’s proposal on Medicaid financing and service delivery would cut hundreds of billions of dollars from the Medicaid program annually,” Mr. Pollack said.

He also objected to “hundreds of billions of proposed reductions to Medicare” endorsed by Mr. Trump.

Medical malpractice overhaul

The Trump administration also offered many suggestions for changing federal laws to reduce health care spending. Among these was a proposed overhaul of the approach to medical malpractice cases.

The president’s budget proposal estimates $40 billion in savings over a decade from steps to limit medical liability, according to a report from the Office of Management and Budget (OMB).

“The current medical liability system does not work for patients or providers, nor does it promote high-quality, evidence-based care,” OMB said. “Providers practice with a threat of potentially frivolous lawsuits, and injured patients often do not receive just compensation for their injuries.”

Mr. Trump’s fiscal 2021 budget calls for a cap on noneconomic damage awards of $250,000, which would increase with inflation over time, and a 3-year statute of limitations. Under this plan, courts could also modify attorney’s fee arrangements. HHS could provide guidance to states on how to create expert panels and administrative health care tribunals to review medical liability.

These steps would lead to lower health care spending, with clinicians dropping “defensive medicine practices,” OMB said. That would benefit the Medicare and Medicaid programs as well as lowering costs of health insurance in general.

Mr. Trump’s fiscal 2021 budget also includes a series of proposals for Medicare that it estimates would, in aggregate, save $755.5 billion over a decade.

Site-neutral policy

A large chunk of the estimated Medicare savings in Mr. Trump’s fiscal 2021 health budget would come from lowering payments to hospitals for services provided in their outpatient and physician offices.

In the fiscal 2021 proposal, HHS noted that “Medicare generally pays on-campus hospital outpatient departments substantially more than physician offices for the same services.”

Mr. Trump’s budget proposal seeks a more expansive shift to what’s called a “site-neutral” payment for services delivered in hospital outpatient programs or physician offices. This would bring these payments more in line with those made to independent physician practices.

“This proposal would eliminate the often significant disparity between what Medicare pays in these different settings for the same services,” HHS said in the budget summary.

HHS estimated this change in policy would generate $117.2 billion in savings over a decade. Combined with saving from medical malpractice reforms, the Trump administration estimates these two moves combined could save about $164 billion over a decade.

The site-neutral policy has been a legal battleground, with hospital and physician groups winning a round last year. 

Another Medicare proposal included in Mr. Trump’s fiscal 2021 budget homes in on this issue for cases where a hospital owns a physician office. Medicare now pays most off-campus hospital outpatient departments higher rates than the program’s physician fee schedule dictates for the same services.

Switching to a site-neutral policy for these hospital-owned physician offices would result in $47.2 billion in savings over a decade, HHS said in the budget document.
 

This article first appeared on Medscape.com.

The Trump administration on Feb. 10 argued for cutting spending for a federal agency at the forefront of the efforts to combat the coronavirus, while also seeking to slow spending in certain parts of the Medicare and Medicaid programs.

President Donald Trump presented his fiscal 2021 request to Congress for refilling the coffers of federal agencies. In any administration, an annual budget serves only as a political blueprint, as the White House document itself makes no changes in federal spending.

In Mr. Trump’s case, several of his requests for agencies within the Department of Health & Human Services run counter to recent budget trends. Republicans and Democrats in Congress have worked together in recent years to increase budgets for major federal health agencies.

But Mr. Trump asked Congress to cut annual budget authority for the National Institute of Allergy and Infectious Diseases by $430 million to $5.446 billion for fiscal 2021. In contrast, Congress has raised the annual budget for NIAID, a key agency in combating the coronavirus, from $5.545 billion in fiscal 2019 to $5.876 billion in fiscal 2020, which began in October, according to an HHS summary of Mr. Trump’s request.

For the Centers for Disease Control and Prevention, which is central to the battle against the coronavirus, Mr. Trump proposed a drop in discretionary funding to $5.627 billion. In contrast, Congress raised the CDC budget from $6.544 billion in fiscal 2019 to $6.917 in fiscal 2020.

Mr. Trump also wants to cut $559 million from the budget of the National Cancer Institute, dropping it to $5.881 billion in fiscal 2021. In contrast, Congress raised NCI’s budget from $6.121 billion in fiscal 2019 to $6.440 billion in fiscal 2020.

Mr. Trump requested a $2.6 billion reduction in the National Institutes of Health’s total discretionary budget, seeking to drop it to $37.70 billion. In contrast, Congress raised NIH’s budget from $37.887 in fiscal 2019 to $40.304 billion in fiscal 2020.

Mr. Trump’s budget proposal also includes an estimate of $152 billion in savings over a decade for Medicaid through the implementation of what the administration calls “community engagement” requirements.

The Trump administration has been at odds with Democrats for years about whether work requirements should be attached to Medicaid. “Well-designed community engagement incentives have great potential to improve health and well-being while empowering beneficiaries to rise out of poverty,” HHS said in a budget document.

Yet researchers last year reported that Arkansas’ attempt to attach work requirements to Medicaid caused almost 17,000 adults to lose this health care coverage within the first 6 months, and there was no significant difference in employment.

The researchers say this loss of coverage was partly a result of bureaucratic obstacles and confusion about the new rules. In June 2018, Arkansas became the first state to implement work requirements for Medicaid, Benjamin D. Sommers, MD, PhD, of the Harvard T.H. Chan School of Public Health, Boston, and colleagues wrote in the New England Journal of Medicine (2019 Sep 12;381[11]:1073-82). 

Budget ‘would thwart’ progress

A few medical groups on Monday quickly criticized Mr. Trump’s proposals.

“In a time where our nation continues to face significant public health challenges — including 2019 novel coronavirus, climate change, gun violence, and costly chronic diseases such as heart disease and cancer – the administration should be investing more resources in better health, not cutting federal health budgets,” said Georges C. Benjamin, MD, executive director of the American Public Health Association, in a statement.

David J. Skorton, MD, chief executive and president of the Association of American Medical Colleges (AAMC) also urged increased investment in fighting disease.

“We must continue the bipartisan budget trajectory set forth by Congress over the last several years, not reverse course,” Dr. Skorton said in a statement.

Mr. Trump’s proposed cuts in medical research “would thwart scientific progress on strategies to prevent, diagnose, treat, and cure medical conditions that affect countless patients nationwide,” he said.

In total, the new 2021 appropriations for HHS would fall by $9.46 billion to $85.667 billion under Mr. Trump’s proposal. Appropriations, also called discretionary budget authority, represents the operating budgets for federal agencies. These are decided through annual spending bills.

Congress has separate sets of laws for handling payments the federal government makes through Medicare and Medicaid. These are known as mandatory spending.

‘Untenable cuts’

AAMC’s Dr. Skorton also objected to what he termed Mr. Trump’s bid “to reduce and consolidate Medicare, Medicaid, and children’s hospital graduate medical education into a single grant program.”

This would force teaching hospitals to absorb $52 billion in “untenable cuts,” he said.

“The proposal ignores the intent of the Medicare GME program, which is to ensure an adequate physician workforce to care for Medicare beneficiaries and support the critical patient care missions of America’s teaching hospitals,” Dr. Skorton said.

The budget also seeks cuts to Medicaid, which come in addition to the administration’s “recent proposals to scale back Medicaid coverage,” Dr. Skorton said.

“More than 26% of all Medicaid hospitalizations occur at AAMC-member teaching hospitals, even though these institutions represent only 5% of all hospitals,” Dr. Skorton said. “Each of the administration’s proposals on their own would be devastating for patients – and combined, they would be disastrous.”

Rick Pollack, the chief executive and president of the American Hospital Association, described Mr. Trump’s fiscal 2021 proposal as another bid to undermine medical care in the United States.

“Every year, we adapt to a constantly changing environment, but every year, the administration aims to gut our nation’s health care infrastructure,” Mr. Pollack said in a statement.

In it, he noted that about one in five people in America depend on Medicaid, with children accounting for a large proportion of those covered by the state-federal program.

“The budget’s proposal on Medicaid financing and service delivery would cut hundreds of billions of dollars from the Medicaid program annually,” Mr. Pollack said.

He also objected to “hundreds of billions of proposed reductions to Medicare” endorsed by Mr. Trump.

Medical malpractice overhaul

The Trump administration also offered many suggestions for changing federal laws to reduce health care spending. Among these was a proposed overhaul of the approach to medical malpractice cases.

The president’s budget proposal estimates $40 billion in savings over a decade from steps to limit medical liability, according to a report from the Office of Management and Budget (OMB).

“The current medical liability system does not work for patients or providers, nor does it promote high-quality, evidence-based care,” OMB said. “Providers practice with a threat of potentially frivolous lawsuits, and injured patients often do not receive just compensation for their injuries.”

Mr. Trump’s fiscal 2021 budget calls for a cap on noneconomic damage awards of $250,000, which would increase with inflation over time, and a 3-year statute of limitations. Under this plan, courts could also modify attorney’s fee arrangements. HHS could provide guidance to states on how to create expert panels and administrative health care tribunals to review medical liability.

These steps would lead to lower health care spending, with clinicians dropping “defensive medicine practices,” OMB said. That would benefit the Medicare and Medicaid programs as well as lowering costs of health insurance in general.

Mr. Trump’s fiscal 2021 budget also includes a series of proposals for Medicare that it estimates would, in aggregate, save $755.5 billion over a decade.

Site-neutral policy

A large chunk of the estimated Medicare savings in Mr. Trump’s fiscal 2021 health budget would come from lowering payments to hospitals for services provided in their outpatient and physician offices.

In the fiscal 2021 proposal, HHS noted that “Medicare generally pays on-campus hospital outpatient departments substantially more than physician offices for the same services.”

Mr. Trump’s budget proposal seeks a more expansive shift to what’s called a “site-neutral” payment for services delivered in hospital outpatient programs or physician offices. This would bring these payments more in line with those made to independent physician practices.

“This proposal would eliminate the often significant disparity between what Medicare pays in these different settings for the same services,” HHS said in the budget summary.

HHS estimated this change in policy would generate $117.2 billion in savings over a decade. Combined with saving from medical malpractice reforms, the Trump administration estimates these two moves combined could save about $164 billion over a decade.

The site-neutral policy has been a legal battleground, with hospital and physician groups winning a round last year. 

Another Medicare proposal included in Mr. Trump’s fiscal 2021 budget homes in on this issue for cases where a hospital owns a physician office. Medicare now pays most off-campus hospital outpatient departments higher rates than the program’s physician fee schedule dictates for the same services.

Switching to a site-neutral policy for these hospital-owned physician offices would result in $47.2 billion in savings over a decade, HHS said in the budget document.
 

This article first appeared on Medscape.com.

Top-line results from the seminal phase 2/3 Dominantly Inherited Alzheimer’s Network–Trials Unit (DIAN-TU) study show that the novel drugs gantenerumab (Roche) and solanezumab (Lilly) did not meet the primary endpoint in patients with early-stage, dominantly inherited Alzheimer’s disease (AD), investigators have announced.

In the international trial, which included almost 200 participants, the two experimental agents were evaluated separately. However, initial analyses showed that neither significantly slowed cognitive decline, the primary outcome measure, nor memory loss.

Still, the researchers noted that they will continue exploring data from DIAN-TU’s cognitive and clinical outcomes and are awaiting analyses of various biomarkers.

“Although the drugs we evaluated were not successful, the trial will move us forward in understanding Alzheimer’s,” principal investigator Randall J. Bateman, MD, of Washington University, St. Louis, said in a news release.

Funders for the trial included the National Institute on Aging and the Alzheimer’s Association.

“While the top-line data fell short, the Alzheimer’s Association looks forward to a more complete report at upcoming scientific conferences. We learn from every trial,” Maria Carrillo, PhD, chief scientific officer at the Alzheimer’s Association, noted in the organization’s own release.

Rebecca M. Edelmayer, PhD, director of scientific engagement for the Alzheimer’s Association, agreed with Dr. Carrillo that, although the results were disappointing, the data will be beneficial for the field.

“It’s always a difficult day when we get news like this,” Dr. Edelmayer said in an interview. However, “this research is going to absolutely provide valuable information once we can really pick through all of the data.”

Rare condition

Dominantly inherited AD, also known as familial AD or autosomal dominant AD, is rare but can affect memory and cognitive skills in individuals as young as age 30. It is caused by mutations on chromosomes 21, 14, and/or 1 that play a part in the breakdown of amyloid proteins and formation of amyloid plaques.

Both gantenerumab and solanezumab were created to target and neutralize amyloid-beta, albeit through different mechanisms. Both are also being assessed in other trials as treatment for more common forms of AD.

As reported by Medscape Medical News, results of the phase 3 EXPEDITION3 trial of solanezumab in patients with mild AD were negative, as were two other phase 3 trials. The drug is now being evaluated in the ongoing solanezumab Anti-Amyloid Treatment in Asymptomatic Alzheimer’s (A4) study.

Although the phase 3 SCARLET ROAD trial of gantenerumab for mild AD was stopped early for futility in 2014, it was continued as an open-label extension at the high dose for 2 years. During that period, follow-up analyses showed a dramatic decline in amyloid-beta deposition in the participants – leading to the launch of the phase 3 GRADUATE 1 and GRADUATE 2 trials.

Starting in 2012, DIAN-TU was conducted at 24 sites in the United States, the United Kingdom, Canada, Europe, and Australia. It followed 194 adult patients for up to 7 years (average duration, 5 years). Its original estimated completion date was December 2020, as stated on clinicaltrials.gov.

All participants had family members with a genetic mutation that causes early-onset Alzheimer’s disease. They already had very mild symptoms of cognitive decline and memory loss at the start of the trial or were expected to develop symptoms within 15 years of enrollment.

“People who inherit the mutation are all but guaranteed to develop symptoms at about the same age their parents did,” the release noted.

“While devastating for families, such mutations allow researchers to identify people in the early stages of the disease before their behavior and memory begin to change,” it added.

The Alzheimer’s Association noted in its release that a child of a parent with the mutation has a 50/50 chance of inheriting the disease. “This form affects less than 1% of the individuals living with Alzheimer’s disease today,” Dr. Edelmayer noted.

Detailed data coming soon

Trial participants were randomly assigned to receive either solanezumab, gantenerumab, or matching placebo. To act as a comparator group, family members without the AD mutation were also included.

The primary measure was change from baseline in the DIAN-TU cognitive composite score. Secondary measures included changes on the Mini-Mental State Examination, the Functional Assessment Scale, the Neuropsychiatric Inventory Questionnaire, the 12-item International Shopping List Test, the Memory Complaint Questionnaire, and the Wechsler Memory Scale Logical Memory/Paragraph Memory test.

The researchers also conducted imaging scans and collected samples of blood and cerebrospinal fluid.

Along with announcing the negative top-line results for the trial, the investigators noted that “a more detailed analysis of the trial’s data” will be presented at the Advances in Alzheimer’s and Parkinson’s Therapies in Vienna on April 2, 2020, and at the Alzheimer’s Association International Conference in Amsterdam in July.

The researchers will continue to explore all data gathered – but already new insights have been discovered into the development and progression of AD, Dr. Bateman noted.

Included among these discoveries is that brain changes that occur as the disease progresses are similar among those with the inherited, early-onset form of AD and the late-onset form.

“The trial’s innovative design ... will make advances for future Alzheimer’s trials. Ongoing and continued research and trials will bring us closer to our goal to stop Alzheimer’s,” Dr. Bateman said. “We will continue until we are successful.”

“These results reflect the difficult nature of treating [AD] and the great need for continued research,” said Daniel Skovronsky, MD, PhD, chief scientific officer and president of Lilly Research Labs.

“If we have learned one thing after more than 30 years of Alzheimer’s research, it is that even negative results propel the science forward,” he added.

Lilly noted in a statement that the DIAN-TU top-line results will not affect its ongoing A4 study of solanezumab. Roche noted in its own statement that the findings also will not affect the company’s ongoing GRADUATE studies of gantenerumab.

“The work doesn’t stop here”

Richard J. Hodes, MD, director of the National Institute on Aging, said that DIAN-TU will advance the field’s knowledge about a complex disease.

“We look forward to learning more through the published, peer-reviewed data, which will provide a broad range of scientists with crucial information and guidance for future research,” he said.

Howard Fillit, MD, founding executive director and chief science officer at the Alzheimer’s Drug Discovery Foundation (ADDF), agreed.

“While we are disappointed that patients in this study did not see a benefit, we need to keep in mind that Alzheimer’s is a complicated disease due to complex, multifactorial causes,” he said in a statement.

“ADDF has long supported a broader approach that moves past targeting beta-amyloid and advances a diverse pipeline of drugs addressing multiple targets” in AD, Dr. Fillit added. “We need multiple ‘shots on goals’ to discover effective drugs.”

Dr. Edelmayer said the results emphasize that “this story isn’t yet completely told” and that there is still a lot to learn from the data, especially regarding the biomarkers that were tested.

“With that information, we will gain valuable insight into the outcomes that have been released but will also probably better understand where we should be putting our energies and focus moving forward,” she said.

Going forward, “we will continue this fight until we have an effective treatment for all individuals living with Alzheimer’s, whether it’s dominantly inherited [AD] or the more common version, which is the late-onset or sporadic form of the disease,” said Dr. Edelmayer.

“We have to stay optimistic. The work doesn’t stop here.”

The trial was funded by Eli Lilly, Roche, the Alzheimer’s Association, the National Institute on Aging, the GHR Foundation, and FBRI.

This article first appeared on Medscape.com.

Top-line results from the seminal phase 2/3 Dominantly Inherited Alzheimer’s Network–Trials Unit (DIAN-TU) study show that the novel drugs gantenerumab (Roche) and solanezumab (Lilly) did not meet the primary endpoint in patients with early-stage, dominantly inherited Alzheimer’s disease (AD), investigators have announced.

In the international trial, which included almost 200 participants, the two experimental agents were evaluated separately. However, initial analyses showed that neither significantly slowed cognitive decline, the primary outcome measure, nor memory loss.

Still, the researchers noted that they will continue exploring data from DIAN-TU’s cognitive and clinical outcomes and are awaiting analyses of various biomarkers.

“Although the drugs we evaluated were not successful, the trial will move us forward in understanding Alzheimer’s,” principal investigator Randall J. Bateman, MD, of Washington University, St. Louis, said in a news release.

Funders for the trial included the National Institute on Aging and the Alzheimer’s Association.

“While the top-line data fell short, the Alzheimer’s Association looks forward to a more complete report at upcoming scientific conferences. We learn from every trial,” Maria Carrillo, PhD, chief scientific officer at the Alzheimer’s Association, noted in the organization’s own release.

Rebecca M. Edelmayer, PhD, director of scientific engagement for the Alzheimer’s Association, agreed with Dr. Carrillo that, although the results were disappointing, the data will be beneficial for the field.

“It’s always a difficult day when we get news like this,” Dr. Edelmayer said in an interview. However, “this research is going to absolutely provide valuable information once we can really pick through all of the data.”

Rare condition

Dominantly inherited AD, also known as familial AD or autosomal dominant AD, is rare but can affect memory and cognitive skills in individuals as young as age 30. It is caused by mutations on chromosomes 21, 14, and/or 1 that play a part in the breakdown of amyloid proteins and formation of amyloid plaques.

Both gantenerumab and solanezumab were created to target and neutralize amyloid-beta, albeit through different mechanisms. Both are also being assessed in other trials as treatment for more common forms of AD.

As reported by Medscape Medical News, results of the phase 3 EXPEDITION3 trial of solanezumab in patients with mild AD were negative, as were two other phase 3 trials. The drug is now being evaluated in the ongoing solanezumab Anti-Amyloid Treatment in Asymptomatic Alzheimer’s (A4) study.

Although the phase 3 SCARLET ROAD trial of gantenerumab for mild AD was stopped early for futility in 2014, it was continued as an open-label extension at the high dose for 2 years. During that period, follow-up analyses showed a dramatic decline in amyloid-beta deposition in the participants – leading to the launch of the phase 3 GRADUATE 1 and GRADUATE 2 trials.

Starting in 2012, DIAN-TU was conducted at 24 sites in the United States, the United Kingdom, Canada, Europe, and Australia. It followed 194 adult patients for up to 7 years (average duration, 5 years). Its original estimated completion date was December 2020, as stated on clinicaltrials.gov.

All participants had family members with a genetic mutation that causes early-onset Alzheimer’s disease. They already had very mild symptoms of cognitive decline and memory loss at the start of the trial or were expected to develop symptoms within 15 years of enrollment.

“People who inherit the mutation are all but guaranteed to develop symptoms at about the same age their parents did,” the release noted.

“While devastating for families, such mutations allow researchers to identify people in the early stages of the disease before their behavior and memory begin to change,” it added.

The Alzheimer’s Association noted in its release that a child of a parent with the mutation has a 50/50 chance of inheriting the disease. “This form affects less than 1% of the individuals living with Alzheimer’s disease today,” Dr. Edelmayer noted.

Detailed data coming soon

Trial participants were randomly assigned to receive either solanezumab, gantenerumab, or matching placebo. To act as a comparator group, family members without the AD mutation were also included.

The primary measure was change from baseline in the DIAN-TU cognitive composite score. Secondary measures included changes on the Mini-Mental State Examination, the Functional Assessment Scale, the Neuropsychiatric Inventory Questionnaire, the 12-item International Shopping List Test, the Memory Complaint Questionnaire, and the Wechsler Memory Scale Logical Memory/Paragraph Memory test.

The researchers also conducted imaging scans and collected samples of blood and cerebrospinal fluid.

Along with announcing the negative top-line results for the trial, the investigators noted that “a more detailed analysis of the trial’s data” will be presented at the Advances in Alzheimer’s and Parkinson’s Therapies in Vienna on April 2, 2020, and at the Alzheimer’s Association International Conference in Amsterdam in July.

The researchers will continue to explore all data gathered – but already new insights have been discovered into the development and progression of AD, Dr. Bateman noted.

Included among these discoveries is that brain changes that occur as the disease progresses are similar among those with the inherited, early-onset form of AD and the late-onset form.

“The trial’s innovative design ... will make advances for future Alzheimer’s trials. Ongoing and continued research and trials will bring us closer to our goal to stop Alzheimer’s,” Dr. Bateman said. “We will continue until we are successful.”

“These results reflect the difficult nature of treating [AD] and the great need for continued research,” said Daniel Skovronsky, MD, PhD, chief scientific officer and president of Lilly Research Labs.

“If we have learned one thing after more than 30 years of Alzheimer’s research, it is that even negative results propel the science forward,” he added.

Lilly noted in a statement that the DIAN-TU top-line results will not affect its ongoing A4 study of solanezumab. Roche noted in its own statement that the findings also will not affect the company’s ongoing GRADUATE studies of gantenerumab.

“The work doesn’t stop here”

Richard J. Hodes, MD, director of the National Institute on Aging, said that DIAN-TU will advance the field’s knowledge about a complex disease.

“We look forward to learning more through the published, peer-reviewed data, which will provide a broad range of scientists with crucial information and guidance for future research,” he said.

Howard Fillit, MD, founding executive director and chief science officer at the Alzheimer’s Drug Discovery Foundation (ADDF), agreed.

“While we are disappointed that patients in this study did not see a benefit, we need to keep in mind that Alzheimer’s is a complicated disease due to complex, multifactorial causes,” he said in a statement.

“ADDF has long supported a broader approach that moves past targeting beta-amyloid and advances a diverse pipeline of drugs addressing multiple targets” in AD, Dr. Fillit added. “We need multiple ‘shots on goals’ to discover effective drugs.”

Dr. Edelmayer said the results emphasize that “this story isn’t yet completely told” and that there is still a lot to learn from the data, especially regarding the biomarkers that were tested.

“With that information, we will gain valuable insight into the outcomes that have been released but will also probably better understand where we should be putting our energies and focus moving forward,” she said.

Going forward, “we will continue this fight until we have an effective treatment for all individuals living with Alzheimer’s, whether it’s dominantly inherited [AD] or the more common version, which is the late-onset or sporadic form of the disease,” said Dr. Edelmayer.

“We have to stay optimistic. The work doesn’t stop here.”

The trial was funded by Eli Lilly, Roche, the Alzheimer’s Association, the National Institute on Aging, the GHR Foundation, and FBRI.

This article first appeared on Medscape.com.

Top-line results from the seminal phase 2/3 Dominantly Inherited Alzheimer’s Network–Trials Unit (DIAN-TU) study show that the novel drugs gantenerumab (Roche) and solanezumab (Lilly) did not meet the primary endpoint in patients with early-stage, dominantly inherited Alzheimer’s disease (AD), investigators have announced.

In the international trial, which included almost 200 participants, the two experimental agents were evaluated separately. However, initial analyses showed that neither significantly slowed cognitive decline, the primary outcome measure, nor memory loss.

Still, the researchers noted that they will continue exploring data from DIAN-TU’s cognitive and clinical outcomes and are awaiting analyses of various biomarkers.

“Although the drugs we evaluated were not successful, the trial will move us forward in understanding Alzheimer’s,” principal investigator Randall J. Bateman, MD, of Washington University, St. Louis, said in a news release.

Funders for the trial included the National Institute on Aging and the Alzheimer’s Association.

“While the top-line data fell short, the Alzheimer’s Association looks forward to a more complete report at upcoming scientific conferences. We learn from every trial,” Maria Carrillo, PhD, chief scientific officer at the Alzheimer’s Association, noted in the organization’s own release.

Rebecca M. Edelmayer, PhD, director of scientific engagement for the Alzheimer’s Association, agreed with Dr. Carrillo that, although the results were disappointing, the data will be beneficial for the field.

“It’s always a difficult day when we get news like this,” Dr. Edelmayer said in an interview. However, “this research is going to absolutely provide valuable information once we can really pick through all of the data.”

Rare condition

Dominantly inherited AD, also known as familial AD or autosomal dominant AD, is rare but can affect memory and cognitive skills in individuals as young as age 30. It is caused by mutations on chromosomes 21, 14, and/or 1 that play a part in the breakdown of amyloid proteins and formation of amyloid plaques.

Both gantenerumab and solanezumab were created to target and neutralize amyloid-beta, albeit through different mechanisms. Both are also being assessed in other trials as treatment for more common forms of AD.

As reported by Medscape Medical News, results of the phase 3 EXPEDITION3 trial of solanezumab in patients with mild AD were negative, as were two other phase 3 trials. The drug is now being evaluated in the ongoing solanezumab Anti-Amyloid Treatment in Asymptomatic Alzheimer’s (A4) study.

Although the phase 3 SCARLET ROAD trial of gantenerumab for mild AD was stopped early for futility in 2014, it was continued as an open-label extension at the high dose for 2 years. During that period, follow-up analyses showed a dramatic decline in amyloid-beta deposition in the participants – leading to the launch of the phase 3 GRADUATE 1 and GRADUATE 2 trials.

Starting in 2012, DIAN-TU was conducted at 24 sites in the United States, the United Kingdom, Canada, Europe, and Australia. It followed 194 adult patients for up to 7 years (average duration, 5 years). Its original estimated completion date was December 2020, as stated on clinicaltrials.gov.

All participants had family members with a genetic mutation that causes early-onset Alzheimer’s disease. They already had very mild symptoms of cognitive decline and memory loss at the start of the trial or were expected to develop symptoms within 15 years of enrollment.

“People who inherit the mutation are all but guaranteed to develop symptoms at about the same age their parents did,” the release noted.

“While devastating for families, such mutations allow researchers to identify people in the early stages of the disease before their behavior and memory begin to change,” it added.

The Alzheimer’s Association noted in its release that a child of a parent with the mutation has a 50/50 chance of inheriting the disease. “This form affects less than 1% of the individuals living with Alzheimer’s disease today,” Dr. Edelmayer noted.

Detailed data coming soon

Trial participants were randomly assigned to receive either solanezumab, gantenerumab, or matching placebo. To act as a comparator group, family members without the AD mutation were also included.

The primary measure was change from baseline in the DIAN-TU cognitive composite score. Secondary measures included changes on the Mini-Mental State Examination, the Functional Assessment Scale, the Neuropsychiatric Inventory Questionnaire, the 12-item International Shopping List Test, the Memory Complaint Questionnaire, and the Wechsler Memory Scale Logical Memory/Paragraph Memory test.

The researchers also conducted imaging scans and collected samples of blood and cerebrospinal fluid.

Along with announcing the negative top-line results for the trial, the investigators noted that “a more detailed analysis of the trial’s data” will be presented at the Advances in Alzheimer’s and Parkinson’s Therapies in Vienna on April 2, 2020, and at the Alzheimer’s Association International Conference in Amsterdam in July.

The researchers will continue to explore all data gathered – but already new insights have been discovered into the development and progression of AD, Dr. Bateman noted.

Included among these discoveries is that brain changes that occur as the disease progresses are similar among those with the inherited, early-onset form of AD and the late-onset form.

“The trial’s innovative design ... will make advances for future Alzheimer’s trials. Ongoing and continued research and trials will bring us closer to our goal to stop Alzheimer’s,” Dr. Bateman said. “We will continue until we are successful.”

“These results reflect the difficult nature of treating [AD] and the great need for continued research,” said Daniel Skovronsky, MD, PhD, chief scientific officer and president of Lilly Research Labs.

“If we have learned one thing after more than 30 years of Alzheimer’s research, it is that even negative results propel the science forward,” he added.

Lilly noted in a statement that the DIAN-TU top-line results will not affect its ongoing A4 study of solanezumab. Roche noted in its own statement that the findings also will not affect the company’s ongoing GRADUATE studies of gantenerumab.

“The work doesn’t stop here”

Richard J. Hodes, MD, director of the National Institute on Aging, said that DIAN-TU will advance the field’s knowledge about a complex disease.

“We look forward to learning more through the published, peer-reviewed data, which will provide a broad range of scientists with crucial information and guidance for future research,” he said.

Howard Fillit, MD, founding executive director and chief science officer at the Alzheimer’s Drug Discovery Foundation (ADDF), agreed.

“While we are disappointed that patients in this study did not see a benefit, we need to keep in mind that Alzheimer’s is a complicated disease due to complex, multifactorial causes,” he said in a statement.

“ADDF has long supported a broader approach that moves past targeting beta-amyloid and advances a diverse pipeline of drugs addressing multiple targets” in AD, Dr. Fillit added. “We need multiple ‘shots on goals’ to discover effective drugs.”

Dr. Edelmayer said the results emphasize that “this story isn’t yet completely told” and that there is still a lot to learn from the data, especially regarding the biomarkers that were tested.

“With that information, we will gain valuable insight into the outcomes that have been released but will also probably better understand where we should be putting our energies and focus moving forward,” she said.

Going forward, “we will continue this fight until we have an effective treatment for all individuals living with Alzheimer’s, whether it’s dominantly inherited [AD] or the more common version, which is the late-onset or sporadic form of the disease,” said Dr. Edelmayer.

“We have to stay optimistic. The work doesn’t stop here.”

The trial was funded by Eli Lilly, Roche, the Alzheimer’s Association, the National Institute on Aging, the GHR Foundation, and FBRI.

This article first appeared on Medscape.com.

U.S. rates of heroin use, heroin use disorder, and heroin injections all increased overall among adults during a recent 17-year period, but rates have plateaued, new research shows.
 

Although on the face of it this may seem like good news, investigators at the Substance Abuse and Mental Health Services Administration (SAMHSA) note that the plateau in heroin use may simply reflect a switch to fentanyl.

“The recent leveling off of heroin use might reflect shifts from heroin to illicit fentanyl-related compounds,” wrote the investigators, led by Beth Han, MD, PhD, MPH.

The study was published online Feb. 11 as a research letter in JAMA (2020;323[6]:568-71).

National data

For the study, researchers collected data from a nationally representative group of adults aged 18 years or older who participated in the 2002-2018 National Survey on Drug Use and Health (NSDUH).

The analysis included 800,500 respondents during the study period. The mean age of respondents was 34.5 years, and 53.2% were women.

Results showed that the reported past-year prevalence of heroin use increased from 0.17% in 2002 to 0.32% in 2018 (average annual percentage change [AAPC], 5.6; 95% confidence interval [CI], 1.0-10.5; P = .02). During 2002-2016, the APC was 7.6 (95% CI, 6.3-9.0; P less than .001) but then plateaued during 2016-2018 (APC, –7.1; 95% CI, –36.9 to 36.7; P = .69).

The prevalence of heroin use disorder increased from 0.10% in 2002 to 0.21% in 2018 (AAPC, 6.0; 95% CI, 3.2-8.8; P less than .001). The rate remained stable during 2002-2008, increased during 2008-2015, then plateaued during 2015-2018.

The prevalence of heroin injections increased from 0.09% in 2002 to 0.17% in 2018 (AAPC, 6.9; 95% CI, 5.7-8.0; P less than .001), although there was a dip from the previous year. This rate increased during the study period among both men and women, those aged 35-49 years, non-Hispanic whites, and those residing in the Northeast or West regions.

For individuals up to age 25 years and those living in the Midwest, the heroin injection rate stopped increasing and plateaued, but there was an overall increase during the study period.

In 2018, the rate of past-year heroin injection was highest in those in the Northeast, those up to age 49 years, men, and non-Hispanic whites.

More infectious disease testing

Prevalence of heroin injection did not increase among adults who used heroin or who had heroin use disorder. This, the researchers note, “suggests that increases in heroin injection are related to overall increases in heroin use rather than increases in the propensity to inject.”

Future research should examine differences in heroin injection trends across subgroups, the authors wrote.

The researchers advocate for expanding HIV and hepatitis testing and treatment, the provision of sterile syringes, and use of Food and Drug Administration–approved medications for opioid use disorders, particularly among populations at greatest risk – adults in the Northeast, those aged 18-49 years, men, and non-Hispanic whites.

“In parallel, interventions to prevent opioid misuse and opioid use disorder are needed to avert further increases in injection drug use,” they noted.

A limitation of the study was that the NSDUH excludes jail and prison populations and homeless people not in living shelters. In addition, the NSDUH is subject to recall bias.

The study was jointly sponsored by SAMHSA and the National Institute on Drug Abuse of the National Institutes of Health. One author reports owning stock in General Electric Co, 3M Co, and Pfizer Inc.

A version of this article first appeared on Medscape.com.

U.S. rates of heroin use, heroin use disorder, and heroin injections all increased overall among adults during a recent 17-year period, but rates have plateaued, new research shows.
 

Although on the face of it this may seem like good news, investigators at the Substance Abuse and Mental Health Services Administration (SAMHSA) note that the plateau in heroin use may simply reflect a switch to fentanyl.

“The recent leveling off of heroin use might reflect shifts from heroin to illicit fentanyl-related compounds,” wrote the investigators, led by Beth Han, MD, PhD, MPH.

The study was published online Feb. 11 as a research letter in JAMA (2020;323[6]:568-71).

National data

For the study, researchers collected data from a nationally representative group of adults aged 18 years or older who participated in the 2002-2018 National Survey on Drug Use and Health (NSDUH).

The analysis included 800,500 respondents during the study period. The mean age of respondents was 34.5 years, and 53.2% were women.

Results showed that the reported past-year prevalence of heroin use increased from 0.17% in 2002 to 0.32% in 2018 (average annual percentage change [AAPC], 5.6; 95% confidence interval [CI], 1.0-10.5; P = .02). During 2002-2016, the APC was 7.6 (95% CI, 6.3-9.0; P less than .001) but then plateaued during 2016-2018 (APC, –7.1; 95% CI, –36.9 to 36.7; P = .69).

The prevalence of heroin use disorder increased from 0.10% in 2002 to 0.21% in 2018 (AAPC, 6.0; 95% CI, 3.2-8.8; P less than .001). The rate remained stable during 2002-2008, increased during 2008-2015, then plateaued during 2015-2018.

The prevalence of heroin injections increased from 0.09% in 2002 to 0.17% in 2018 (AAPC, 6.9; 95% CI, 5.7-8.0; P less than .001), although there was a dip from the previous year. This rate increased during the study period among both men and women, those aged 35-49 years, non-Hispanic whites, and those residing in the Northeast or West regions.

For individuals up to age 25 years and those living in the Midwest, the heroin injection rate stopped increasing and plateaued, but there was an overall increase during the study period.

In 2018, the rate of past-year heroin injection was highest in those in the Northeast, those up to age 49 years, men, and non-Hispanic whites.

More infectious disease testing

Prevalence of heroin injection did not increase among adults who used heroin or who had heroin use disorder. This, the researchers note, “suggests that increases in heroin injection are related to overall increases in heroin use rather than increases in the propensity to inject.”

Future research should examine differences in heroin injection trends across subgroups, the authors wrote.

The researchers advocate for expanding HIV and hepatitis testing and treatment, the provision of sterile syringes, and use of Food and Drug Administration–approved medications for opioid use disorders, particularly among populations at greatest risk – adults in the Northeast, those aged 18-49 years, men, and non-Hispanic whites.

“In parallel, interventions to prevent opioid misuse and opioid use disorder are needed to avert further increases in injection drug use,” they noted.

A limitation of the study was that the NSDUH excludes jail and prison populations and homeless people not in living shelters. In addition, the NSDUH is subject to recall bias.

The study was jointly sponsored by SAMHSA and the National Institute on Drug Abuse of the National Institutes of Health. One author reports owning stock in General Electric Co, 3M Co, and Pfizer Inc.

A version of this article first appeared on Medscape.com.

U.S. rates of heroin use, heroin use disorder, and heroin injections all increased overall among adults during a recent 17-year period, but rates have plateaued, new research shows.
 

Although on the face of it this may seem like good news, investigators at the Substance Abuse and Mental Health Services Administration (SAMHSA) note that the plateau in heroin use may simply reflect a switch to fentanyl.

“The recent leveling off of heroin use might reflect shifts from heroin to illicit fentanyl-related compounds,” wrote the investigators, led by Beth Han, MD, PhD, MPH.

The study was published online Feb. 11 as a research letter in JAMA (2020;323[6]:568-71).

National data

For the study, researchers collected data from a nationally representative group of adults aged 18 years or older who participated in the 2002-2018 National Survey on Drug Use and Health (NSDUH).

The analysis included 800,500 respondents during the study period. The mean age of respondents was 34.5 years, and 53.2% were women.

Results showed that the reported past-year prevalence of heroin use increased from 0.17% in 2002 to 0.32% in 2018 (average annual percentage change [AAPC], 5.6; 95% confidence interval [CI], 1.0-10.5; P = .02). During 2002-2016, the APC was 7.6 (95% CI, 6.3-9.0; P less than .001) but then plateaued during 2016-2018 (APC, –7.1; 95% CI, –36.9 to 36.7; P = .69).

The prevalence of heroin use disorder increased from 0.10% in 2002 to 0.21% in 2018 (AAPC, 6.0; 95% CI, 3.2-8.8; P less than .001). The rate remained stable during 2002-2008, increased during 2008-2015, then plateaued during 2015-2018.

The prevalence of heroin injections increased from 0.09% in 2002 to 0.17% in 2018 (AAPC, 6.9; 95% CI, 5.7-8.0; P less than .001), although there was a dip from the previous year. This rate increased during the study period among both men and women, those aged 35-49 years, non-Hispanic whites, and those residing in the Northeast or West regions.

For individuals up to age 25 years and those living in the Midwest, the heroin injection rate stopped increasing and plateaued, but there was an overall increase during the study period.

In 2018, the rate of past-year heroin injection was highest in those in the Northeast, those up to age 49 years, men, and non-Hispanic whites.

More infectious disease testing

Prevalence of heroin injection did not increase among adults who used heroin or who had heroin use disorder. This, the researchers note, “suggests that increases in heroin injection are related to overall increases in heroin use rather than increases in the propensity to inject.”

Future research should examine differences in heroin injection trends across subgroups, the authors wrote.

The researchers advocate for expanding HIV and hepatitis testing and treatment, the provision of sterile syringes, and use of Food and Drug Administration–approved medications for opioid use disorders, particularly among populations at greatest risk – adults in the Northeast, those aged 18-49 years, men, and non-Hispanic whites.

“In parallel, interventions to prevent opioid misuse and opioid use disorder are needed to avert further increases in injection drug use,” they noted.

A limitation of the study was that the NSDUH excludes jail and prison populations and homeless people not in living shelters. In addition, the NSDUH is subject to recall bias.

The study was jointly sponsored by SAMHSA and the National Institute on Drug Abuse of the National Institutes of Health. One author reports owning stock in General Electric Co, 3M Co, and Pfizer Inc.

A version of this article first appeared on Medscape.com.

With myriad complex, high-tech problems facing private practice in this modern era, I am periodically reminded by long-time readers to revisit some of the low-tech issues that will always require our attention.

Few are lower tech (in most cases) and more easily overlooked than theft from within. Embezzlement remains far more common in medical offices than generally assumed – and it often occurs in full view of physicians who think everything is fine. Most embezzlers are not skillful or discreet; their transgressions may go undetected for years, simply because no one suspects it is happening.

Detecting fraud is an inexact science. There is no textbook approach that one can follow, but a few simple measures will prevent or expose the most common forms:

• Make it more difficult. Theft and embezzlement are usually products of opportunity, so minimize those opportunities. No one person should be in charge of the entire bookkeeping process: The person who enters charges should be different from the one who enters payments. The one who writes checks or makes electronic fund transfers should not balance the books, and so on. Internal audits should be done on a regular basis, and all employees should know that. Your accountant can help.
• Reconcile cash receipts daily. Embezzlement does not require sophisticated technology; the most common form is simply taking cash out of the till. In a typical scenario, a patient pays a copay of $15 in cash; the receptionist records the payment as $5, and pockets the rest. Make sure a receipt is generated for every cash transaction, and that someone other than the person accepting cash reconciles the charges, receipts, and cash totals daily.
• Inventory your stock. Cash isn’t the only susceptible commodity. If you sell cosmetics or other products, inventory your stock frequently. And office personnel are not the only potential thieves: Last year, a locum tenens physician down the street conspired with a receptionist to take cash transactions for cosmetic neurotoxins and fillers “off the books” and split the spoils. That office was being ripped off twice; first for the neurotoxin and filler materials themselves, and then for the cash proceeds.
• Separate all accounting duties. Another popular ploy is false invoicing for imaginary supplies. A friend’s experience provides a good example (retold with his permission): His bookkeeper wrote sizable checks to herself, disguising them in the ledger as payments to vendors commonly used by his practice. Since the same employee also balanced the checkbook, she got away with it for years. “It wasn’t at all clever,” he told me, “and I’m embarrassed to admit that it happened to me.” Once again, separation of duties is the key to prevention. One employee should enter invoices into the data system, another should issue the check or make the electronic transfer, and a third should match invoices to goods and services received.
• Verify expense reports. False expense reporting is a subset of the fake invoice scam. When an employee asks for reimbursement of expenses, make sure those expenses are real.
• Consider computer safeguards. Computers facilitate a lot of financial chores, but they also consolidate financial data in one place, where it is potentially accessible to anybody, anywhere. Your computer vendor should be aware of this, and there should be safeguards built into your system. Ask about them. If they aren’t there, ask why.
• Hire honest employees. All applicants look great on paper, so check their references; and with their permission, you can run background checks for a few dollars on any of several public information web sites. My columns on hiring are available on the MDedge Dermatology website.
• Look for “red flags.” Examples include employees who refuse to take vacations, because someone else will have do their work or who insist on posting expenses that are a coworker’s responsibility, “just to be nice.” Anyone obviously living beyond his or her means merits suspicion as well.
• Consider bonding your employees. Dishonesty bonds are relatively inexpensive, and provide assurance of some measure of recovery if your safeguards fail. Also, just knowing that your staff is bonded will scare off most dishonest applicants. One effective screen is a question on your employment application: “Would you object to being bonded?”

Dr. Eastern practices dermatology and dermatologic surgery in Belleville, N.J. He is the author of numerous articles and textbook chapters, and is a longtime monthly columnist for Dermatology News. Write to him at [email protected].

With myriad complex, high-tech problems facing private practice in this modern era, I am periodically reminded by long-time readers to revisit some of the low-tech issues that will always require our attention.

Few are lower tech (in most cases) and more easily overlooked than theft from within. Embezzlement remains far more common in medical offices than generally assumed – and it often occurs in full view of physicians who think everything is fine. Most embezzlers are not skillful or discreet; their transgressions may go undetected for years, simply because no one suspects it is happening.

Detecting fraud is an inexact science. There is no textbook approach that one can follow, but a few simple measures will prevent or expose the most common forms:

• Make it more difficult. Theft and embezzlement are usually products of opportunity, so minimize those opportunities. No one person should be in charge of the entire bookkeeping process: The person who enters charges should be different from the one who enters payments. The one who writes checks or makes electronic fund transfers should not balance the books, and so on. Internal audits should be done on a regular basis, and all employees should know that. Your accountant can help.
• Reconcile cash receipts daily. Embezzlement does not require sophisticated technology; the most common form is simply taking cash out of the till. In a typical scenario, a patient pays a copay of $15 in cash; the receptionist records the payment as $5, and pockets the rest. Make sure a receipt is generated for every cash transaction, and that someone other than the person accepting cash reconciles the charges, receipts, and cash totals daily.
• Inventory your stock. Cash isn’t the only susceptible commodity. If you sell cosmetics or other products, inventory your stock frequently. And office personnel are not the only potential thieves: Last year, a locum tenens physician down the street conspired with a receptionist to take cash transactions for cosmetic neurotoxins and fillers “off the books” and split the spoils. That office was being ripped off twice; first for the neurotoxin and filler materials themselves, and then for the cash proceeds.
• Separate all accounting duties. Another popular ploy is false invoicing for imaginary supplies. A friend’s experience provides a good example (retold with his permission): His bookkeeper wrote sizable checks to herself, disguising them in the ledger as payments to vendors commonly used by his practice. Since the same employee also balanced the checkbook, she got away with it for years. “It wasn’t at all clever,” he told me, “and I’m embarrassed to admit that it happened to me.” Once again, separation of duties is the key to prevention. One employee should enter invoices into the data system, another should issue the check or make the electronic transfer, and a third should match invoices to goods and services received.
• Verify expense reports. False expense reporting is a subset of the fake invoice scam. When an employee asks for reimbursement of expenses, make sure those expenses are real.
• Consider computer safeguards. Computers facilitate a lot of financial chores, but they also consolidate financial data in one place, where it is potentially accessible to anybody, anywhere. Your computer vendor should be aware of this, and there should be safeguards built into your system. Ask about them. If they aren’t there, ask why.
• Hire honest employees. All applicants look great on paper, so check their references; and with their permission, you can run background checks for a few dollars on any of several public information web sites. My columns on hiring are available on the MDedge Dermatology website.
• Look for “red flags.” Examples include employees who refuse to take vacations, because someone else will have do their work or who insist on posting expenses that are a coworker’s responsibility, “just to be nice.” Anyone obviously living beyond his or her means merits suspicion as well.
• Consider bonding your employees. Dishonesty bonds are relatively inexpensive, and provide assurance of some measure of recovery if your safeguards fail. Also, just knowing that your staff is bonded will scare off most dishonest applicants. One effective screen is a question on your employment application: “Would you object to being bonded?”

Dr. Eastern practices dermatology and dermatologic surgery in Belleville, N.J. He is the author of numerous articles and textbook chapters, and is a longtime monthly columnist for Dermatology News. Write to him at [email protected].

With myriad complex, high-tech problems facing private practice in this modern era, I am periodically reminded by long-time readers to revisit some of the low-tech issues that will always require our attention.

Few are lower tech (in most cases) and more easily overlooked than theft from within. Embezzlement remains far more common in medical offices than generally assumed – and it often occurs in full view of physicians who think everything is fine. Most embezzlers are not skillful or discreet; their transgressions may go undetected for years, simply because no one suspects it is happening.

Detecting fraud is an inexact science. There is no textbook approach that one can follow, but a few simple measures will prevent or expose the most common forms:

• Make it more difficult. Theft and embezzlement are usually products of opportunity, so minimize those opportunities. No one person should be in charge of the entire bookkeeping process: The person who enters charges should be different from the one who enters payments. The one who writes checks or makes electronic fund transfers should not balance the books, and so on. Internal audits should be done on a regular basis, and all employees should know that. Your accountant can help.
• Reconcile cash receipts daily. Embezzlement does not require sophisticated technology; the most common form is simply taking cash out of the till. In a typical scenario, a patient pays a copay of $15 in cash; the receptionist records the payment as $5, and pockets the rest. Make sure a receipt is generated for every cash transaction, and that someone other than the person accepting cash reconciles the charges, receipts, and cash totals daily.
• Inventory your stock. Cash isn’t the only susceptible commodity. If you sell cosmetics or other products, inventory your stock frequently. And office personnel are not the only potential thieves: Last year, a locum tenens physician down the street conspired with a receptionist to take cash transactions for cosmetic neurotoxins and fillers “off the books” and split the spoils. That office was being ripped off twice; first for the neurotoxin and filler materials themselves, and then for the cash proceeds.
• Separate all accounting duties. Another popular ploy is false invoicing for imaginary supplies. A friend’s experience provides a good example (retold with his permission): His bookkeeper wrote sizable checks to herself, disguising them in the ledger as payments to vendors commonly used by his practice. Since the same employee also balanced the checkbook, she got away with it for years. “It wasn’t at all clever,” he told me, “and I’m embarrassed to admit that it happened to me.” Once again, separation of duties is the key to prevention. One employee should enter invoices into the data system, another should issue the check or make the electronic transfer, and a third should match invoices to goods and services received.
• Verify expense reports. False expense reporting is a subset of the fake invoice scam. When an employee asks for reimbursement of expenses, make sure those expenses are real.
• Consider computer safeguards. Computers facilitate a lot of financial chores, but they also consolidate financial data in one place, where it is potentially accessible to anybody, anywhere. Your computer vendor should be aware of this, and there should be safeguards built into your system. Ask about them. If they aren’t there, ask why.
• Hire honest employees. All applicants look great on paper, so check their references; and with their permission, you can run background checks for a few dollars on any of several public information web sites. My columns on hiring are available on the MDedge Dermatology website.
• Look for “red flags.” Examples include employees who refuse to take vacations, because someone else will have do their work or who insist on posting expenses that are a coworker’s responsibility, “just to be nice.” Anyone obviously living beyond his or her means merits suspicion as well.
• Consider bonding your employees. Dishonesty bonds are relatively inexpensive, and provide assurance of some measure of recovery if your safeguards fail. Also, just knowing that your staff is bonded will scare off most dishonest applicants. One effective screen is a question on your employment application: “Would you object to being bonded?”

Dr. Eastern practices dermatology and dermatologic surgery in Belleville, N.J. He is the author of numerous articles and textbook chapters, and is a longtime monthly columnist for Dermatology News. Write to him at [email protected].

Fear of negative events can drive changes in activity levels among patients with hemophilia A, results of the HemACTIVE study suggest.

Patients were more likely to adjust their level of physical activity due to fear of bleeding and joint damage rather than previously experienced bleeding or joint damage.

However, past experience was more likely than fear to make patients stop physical activities altogether.

Mark Skinner, of the Institute for Policy Advancement in Washington, D.C., and colleagues presented these findings in a poster from the annual congress of the European Association for Haemophilia and Allied Disorders.

Mr. Skinner, who is a hemophilia patient himself, said the goal of the HemACTIVE study is to better understand how hemophilia affects patients’ lives.

“We wanted to understand the limitations, challenges, and compromises of individuals living with hemophilia,” Mr. Skinner said. “What has motivated them or prevented them from living more full, active lives doing the kind of work, leisure, and social activities that those without hemophilia do? Is it treatment choice, is it satisfaction with treatment, is it fear?

“We wanted to do a comprehensive study that really looked at the intersection of treatment adherence and satisfaction, the emotional components that relate to those decisions, and the challenges and compromises so that we could better identify what we need to consider as patients think about either changing their therapy or changing their treatment regimen on existing therapy.”

Previous results from the HemACTIVE study showed that, although activity levels differed among hemophilia patients, all patients surveyed wanted greater activity levels, better protection from bleeding, better pain relief, and less-frequent infusions (EAHAD 2019, Abstract P084). In addition, patients who used factor VIII products with an extended half-life were more active and more likely to adhere to their prescribed treatment (ISTH 2019, Abstract PB0210).

The results reported at EAHAD 2020 focus on patients’ reasons for modifying physical activity. Patients and caregivers completed a screening phone interview, followed by a 25-minute, web-based questionnaire on patient activity.

There were 275 respondents – 194 patients with hemophilia A and 81 caregivers – from Canada, France, Germany, Italy, and the United States. Patients had severe (61%) or moderate (39%) hemophilia A, and most (67%) were receiving prophylaxis.

Most patients (70%) were “active” or “extremely/very active,” 77% of patients adjusted their activities because of their hemophilia, and nearly half of patients stopped activities because of their disease.

Fear drives adjustments in activity

Patients were sometimes more likely to adjust their activities based on fear of experiencing an event, as opposed to previously experiencing that event.

Specifically, 44% of patients adjusted their activities due to fear of joint damage, compared with 36% of patients who made adjustments because of past significant joint damage.

Similarly, 41% of patients adjusted activities due to fear of breakthrough bleeds, compared with 36% of patients who made adjustments because of past experience with bleeds and 25% who made adjustments because of significant past bleeds.

On the other hand, a similar percentage of patients adjusted activities because of past experience with pain (43%) and fear of pain (41%). And a similar percentage of patients adjusted activities because of existing joint damage restrictions (35%) and fear of joint deterioration (32%).

Past experience prompts discontinuation of activity

Overall, 47% of patients said anxiety was the most common emotional reason for stopping physical activities. However, patients were consistently more likely to stop activities because of past experience rather than fear or anxiety.

Specifically, 50% of patients stopped activities because of significant past joint damage, 46% stopped because of developing joint problems, and 38% stopped due to fear of joint damage.

More patients stopped activities because of significant past bleeds (41%) rather than fear of breakthrough bleeds (26%). More patients stopped activities because they developed chronic pain (38%) rather than fear of pain (less than 15%). And more patients stopped activities because of existing joint damage restrictions (62%) rather than fear of joint damage (34%).
 

Applying results to practice: Changing the conversation

Ideally, these findings would be used to promote individualized treatment of hemophilia driven by patients’ goals, Mr. Skinner said. By better understanding patients’ feelings and motivations, clinicians may devise more personalized treatment regimens that align with patients’ goals and improve their quality of life.

Rather than adjusting treatment based only on “hard metrics” such as bleeding events, “we need to take a more holistic approach to looking at outcomes that are more important to patients,” Mr. Skinner said. This type of approach is particularly important to Mr. Skinner as someone who has severe hemophilia A.

“Because hemophilia is a life-long disease, and you’re born with it, you make conscious or unconscious adaptations throughout your life,” he explained. “Your expectations or aspirations adjust to what you’ve been told you can or cannot do because of your hemophilia. The choices I made for my career, where I live, the type of vacations I go on, the type of sports I participate in have all been limited over the course of time, which has meant that I’ve made compromises. There are a lot of individuals with hemophilia who are making decisions that are not what their life goals are.

“What this research helps me understand is that we can change the conversation and build it around an individual patient and understand what their aspirations are. If a clinician understands what I’m wanting to achieve in life … we can build a treatment regime around helping me achieve those goals. That is known to improve adherence.

“The goal, really, is to have hemophilia as a secondary consideration. Instead of saying: ‘You have hemophilia, so these are the options available to you,’ you can say, ‘what is it that you would like to achieve, and then we’ll figure out how your treatment for hemophilia can be adjusted to help you achieve those goals.’ It may sound like a nuance, but it really is reversing the conversation. The goal setting first versus your disease comes first.”

The HemACTIVE study was supported by Bayer. Mr. Skinner disclosed relationships with Bayer and other pharmaceutical companies.

[email protected]

SOURCE: Skinner M et al. EAHAD 2020, Abstract P304.

Fear of negative events can drive changes in activity levels among patients with hemophilia A, results of the HemACTIVE study suggest.

Patients were more likely to adjust their level of physical activity due to fear of bleeding and joint damage rather than previously experienced bleeding or joint damage.

However, past experience was more likely than fear to make patients stop physical activities altogether.

Mark Skinner, of the Institute for Policy Advancement in Washington, D.C., and colleagues presented these findings in a poster from the annual congress of the European Association for Haemophilia and Allied Disorders.

Mr. Skinner, who is a hemophilia patient himself, said the goal of the HemACTIVE study is to better understand how hemophilia affects patients’ lives.

“We wanted to understand the limitations, challenges, and compromises of individuals living with hemophilia,” Mr. Skinner said. “What has motivated them or prevented them from living more full, active lives doing the kind of work, leisure, and social activities that those without hemophilia do? Is it treatment choice, is it satisfaction with treatment, is it fear?

“We wanted to do a comprehensive study that really looked at the intersection of treatment adherence and satisfaction, the emotional components that relate to those decisions, and the challenges and compromises so that we could better identify what we need to consider as patients think about either changing their therapy or changing their treatment regimen on existing therapy.”

Previous results from the HemACTIVE study showed that, although activity levels differed among hemophilia patients, all patients surveyed wanted greater activity levels, better protection from bleeding, better pain relief, and less-frequent infusions (EAHAD 2019, Abstract P084). In addition, patients who used factor VIII products with an extended half-life were more active and more likely to adhere to their prescribed treatment (ISTH 2019, Abstract PB0210).

The results reported at EAHAD 2020 focus on patients’ reasons for modifying physical activity. Patients and caregivers completed a screening phone interview, followed by a 25-minute, web-based questionnaire on patient activity.

There were 275 respondents – 194 patients with hemophilia A and 81 caregivers – from Canada, France, Germany, Italy, and the United States. Patients had severe (61%) or moderate (39%) hemophilia A, and most (67%) were receiving prophylaxis.

Most patients (70%) were “active” or “extremely/very active,” 77% of patients adjusted their activities because of their hemophilia, and nearly half of patients stopped activities because of their disease.

Fear drives adjustments in activity

Patients were sometimes more likely to adjust their activities based on fear of experiencing an event, as opposed to previously experiencing that event.

Specifically, 44% of patients adjusted their activities due to fear of joint damage, compared with 36% of patients who made adjustments because of past significant joint damage.

Similarly, 41% of patients adjusted activities due to fear of breakthrough bleeds, compared with 36% of patients who made adjustments because of past experience with bleeds and 25% who made adjustments because of significant past bleeds.

On the other hand, a similar percentage of patients adjusted activities because of past experience with pain (43%) and fear of pain (41%). And a similar percentage of patients adjusted activities because of existing joint damage restrictions (35%) and fear of joint deterioration (32%).

Past experience prompts discontinuation of activity

Overall, 47% of patients said anxiety was the most common emotional reason for stopping physical activities. However, patients were consistently more likely to stop activities because of past experience rather than fear or anxiety.

Specifically, 50% of patients stopped activities because of significant past joint damage, 46% stopped because of developing joint problems, and 38% stopped due to fear of joint damage.

More patients stopped activities because of significant past bleeds (41%) rather than fear of breakthrough bleeds (26%). More patients stopped activities because they developed chronic pain (38%) rather than fear of pain (less than 15%). And more patients stopped activities because of existing joint damage restrictions (62%) rather than fear of joint damage (34%).
 

Applying results to practice: Changing the conversation

Ideally, these findings would be used to promote individualized treatment of hemophilia driven by patients’ goals, Mr. Skinner said. By better understanding patients’ feelings and motivations, clinicians may devise more personalized treatment regimens that align with patients’ goals and improve their quality of life.

Rather than adjusting treatment based only on “hard metrics” such as bleeding events, “we need to take a more holistic approach to looking at outcomes that are more important to patients,” Mr. Skinner said. This type of approach is particularly important to Mr. Skinner as someone who has severe hemophilia A.

“Because hemophilia is a life-long disease, and you’re born with it, you make conscious or unconscious adaptations throughout your life,” he explained. “Your expectations or aspirations adjust to what you’ve been told you can or cannot do because of your hemophilia. The choices I made for my career, where I live, the type of vacations I go on, the type of sports I participate in have all been limited over the course of time, which has meant that I’ve made compromises. There are a lot of individuals with hemophilia who are making decisions that are not what their life goals are.

“What this research helps me understand is that we can change the conversation and build it around an individual patient and understand what their aspirations are. If a clinician understands what I’m wanting to achieve in life … we can build a treatment regime around helping me achieve those goals. That is known to improve adherence.

“The goal, really, is to have hemophilia as a secondary consideration. Instead of saying: ‘You have hemophilia, so these are the options available to you,’ you can say, ‘what is it that you would like to achieve, and then we’ll figure out how your treatment for hemophilia can be adjusted to help you achieve those goals.’ It may sound like a nuance, but it really is reversing the conversation. The goal setting first versus your disease comes first.”

The HemACTIVE study was supported by Bayer. Mr. Skinner disclosed relationships with Bayer and other pharmaceutical companies.

[email protected]

SOURCE: Skinner M et al. EAHAD 2020, Abstract P304.

Fear of negative events can drive changes in activity levels among patients with hemophilia A, results of the HemACTIVE study suggest.

Patients were more likely to adjust their level of physical activity due to fear of bleeding and joint damage rather than previously experienced bleeding or joint damage.

However, past experience was more likely than fear to make patients stop physical activities altogether.

Mark Skinner, of the Institute for Policy Advancement in Washington, D.C., and colleagues presented these findings in a poster from the annual congress of the European Association for Haemophilia and Allied Disorders.

Mr. Skinner, who is a hemophilia patient himself, said the goal of the HemACTIVE study is to better understand how hemophilia affects patients’ lives.

“We wanted to understand the limitations, challenges, and compromises of individuals living with hemophilia,” Mr. Skinner said. “What has motivated them or prevented them from living more full, active lives doing the kind of work, leisure, and social activities that those without hemophilia do? Is it treatment choice, is it satisfaction with treatment, is it fear?

“We wanted to do a comprehensive study that really looked at the intersection of treatment adherence and satisfaction, the emotional components that relate to those decisions, and the challenges and compromises so that we could better identify what we need to consider as patients think about either changing their therapy or changing their treatment regimen on existing therapy.”

Previous results from the HemACTIVE study showed that, although activity levels differed among hemophilia patients, all patients surveyed wanted greater activity levels, better protection from bleeding, better pain relief, and less-frequent infusions (EAHAD 2019, Abstract P084). In addition, patients who used factor VIII products with an extended half-life were more active and more likely to adhere to their prescribed treatment (ISTH 2019, Abstract PB0210).

The results reported at EAHAD 2020 focus on patients’ reasons for modifying physical activity. Patients and caregivers completed a screening phone interview, followed by a 25-minute, web-based questionnaire on patient activity.

There were 275 respondents – 194 patients with hemophilia A and 81 caregivers – from Canada, France, Germany, Italy, and the United States. Patients had severe (61%) or moderate (39%) hemophilia A, and most (67%) were receiving prophylaxis.

Most patients (70%) were “active” or “extremely/very active,” 77% of patients adjusted their activities because of their hemophilia, and nearly half of patients stopped activities because of their disease.

Fear drives adjustments in activity

Patients were sometimes more likely to adjust their activities based on fear of experiencing an event, as opposed to previously experiencing that event.

Specifically, 44% of patients adjusted their activities due to fear of joint damage, compared with 36% of patients who made adjustments because of past significant joint damage.

Similarly, 41% of patients adjusted activities due to fear of breakthrough bleeds, compared with 36% of patients who made adjustments because of past experience with bleeds and 25% who made adjustments because of significant past bleeds.

On the other hand, a similar percentage of patients adjusted activities because of past experience with pain (43%) and fear of pain (41%). And a similar percentage of patients adjusted activities because of existing joint damage restrictions (35%) and fear of joint deterioration (32%).

Past experience prompts discontinuation of activity

Overall, 47% of patients said anxiety was the most common emotional reason for stopping physical activities. However, patients were consistently more likely to stop activities because of past experience rather than fear or anxiety.

Specifically, 50% of patients stopped activities because of significant past joint damage, 46% stopped because of developing joint problems, and 38% stopped due to fear of joint damage.

More patients stopped activities because of significant past bleeds (41%) rather than fear of breakthrough bleeds (26%). More patients stopped activities because they developed chronic pain (38%) rather than fear of pain (less than 15%). And more patients stopped activities because of existing joint damage restrictions (62%) rather than fear of joint damage (34%).
 

Applying results to practice: Changing the conversation

Ideally, these findings would be used to promote individualized treatment of hemophilia driven by patients’ goals, Mr. Skinner said. By better understanding patients’ feelings and motivations, clinicians may devise more personalized treatment regimens that align with patients’ goals and improve their quality of life.

Rather than adjusting treatment based only on “hard metrics” such as bleeding events, “we need to take a more holistic approach to looking at outcomes that are more important to patients,” Mr. Skinner said. This type of approach is particularly important to Mr. Skinner as someone who has severe hemophilia A.

“Because hemophilia is a life-long disease, and you’re born with it, you make conscious or unconscious adaptations throughout your life,” he explained. “Your expectations or aspirations adjust to what you’ve been told you can or cannot do because of your hemophilia. The choices I made for my career, where I live, the type of vacations I go on, the type of sports I participate in have all been limited over the course of time, which has meant that I’ve made compromises. There are a lot of individuals with hemophilia who are making decisions that are not what their life goals are.

“What this research helps me understand is that we can change the conversation and build it around an individual patient and understand what their aspirations are. If a clinician understands what I’m wanting to achieve in life … we can build a treatment regime around helping me achieve those goals. That is known to improve adherence.

“The goal, really, is to have hemophilia as a secondary consideration. Instead of saying: ‘You have hemophilia, so these are the options available to you,’ you can say, ‘what is it that you would like to achieve, and then we’ll figure out how your treatment for hemophilia can be adjusted to help you achieve those goals.’ It may sound like a nuance, but it really is reversing the conversation. The goal setting first versus your disease comes first.”

The HemACTIVE study was supported by Bayer. Mr. Skinner disclosed relationships with Bayer and other pharmaceutical companies.

[email protected]

SOURCE: Skinner M et al. EAHAD 2020, Abstract P304.