The Food and Drug Administration has issued a safety alert approving new boxed warnings about increased blood clot and mortality risk associated with the 10-mg, twice-daily dose of tofacitinib (Xeljanz), as well as a new limitation for patients with ulcerative colitis receiving the medication.

Tofacitinib, a Janus kinase inhibitor, was first approved by the FDA in 2012 for the treatment of rheumatoid arthritis (RA). An indication for psoriatic arthritis was added in 2017, and one for ulcerative colitis was added in 2018.

After the 2012 approval, the FDA commissioned a postmarketing trial in patients with RA on background methotrexate to evaluate safety and the risk of cancer, heart-related events, and infection. The 5- and 10-mg tofacitinib twice daily doses are being analyzed in an ongoing study in comparison with a tumor necrosis factor (TNF) inhibitor.

An interim analysis of the trial’s data, as of January 2019, found an increased risk of blood clots and death in patients receiving 10-mg tofacitinib twice daily, compared with the TNF inhibitor and the twice-daily, 5-mg dose. Overall, there were 19 cases of blood clots in the lung out of 3,884 patient-years of follow-up in patients who received tofacitinib 10 mg twice daily, compared with 3 cases out of 3,982 patient-years in patients who received TNF inhibitors. There were also 45 cases of death from all causes during follow-up for tofacitinib 10 mg twice daily, compared with 25 cases in patients who received TNF inhibitors.

Patients with symptoms of thrombosis also receiving tofacitinib should immediately discontinue the medication. Tofacitinib should not be given to patients with ulcerative colitis unless they are not treated effectively with a TNF inhibitor or do not tolerate TNF inhibitors; ulcerative colitis patients should receive the lowest effective dosage, and if the higher dosage is necessary, it should be limited to the shortest amount of time possible, the FDA noted.

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The Food and Drug Administration has issued a safety alert approving new boxed warnings about increased blood clot and mortality risk associated with the 10-mg, twice-daily dose of tofacitinib (Xeljanz), as well as a new limitation for patients with ulcerative colitis receiving the medication.

Tofacitinib, a Janus kinase inhibitor, was first approved by the FDA in 2012 for the treatment of rheumatoid arthritis (RA). An indication for psoriatic arthritis was added in 2017, and one for ulcerative colitis was added in 2018.

After the 2012 approval, the FDA commissioned a postmarketing trial in patients with RA on background methotrexate to evaluate safety and the risk of cancer, heart-related events, and infection. The 5- and 10-mg tofacitinib twice daily doses are being analyzed in an ongoing study in comparison with a tumor necrosis factor (TNF) inhibitor.

An interim analysis of the trial’s data, as of January 2019, found an increased risk of blood clots and death in patients receiving 10-mg tofacitinib twice daily, compared with the TNF inhibitor and the twice-daily, 5-mg dose. Overall, there were 19 cases of blood clots in the lung out of 3,884 patient-years of follow-up in patients who received tofacitinib 10 mg twice daily, compared with 3 cases out of 3,982 patient-years in patients who received TNF inhibitors. There were also 45 cases of death from all causes during follow-up for tofacitinib 10 mg twice daily, compared with 25 cases in patients who received TNF inhibitors.

Patients with symptoms of thrombosis also receiving tofacitinib should immediately discontinue the medication. Tofacitinib should not be given to patients with ulcerative colitis unless they are not treated effectively with a TNF inhibitor or do not tolerate TNF inhibitors; ulcerative colitis patients should receive the lowest effective dosage, and if the higher dosage is necessary, it should be limited to the shortest amount of time possible, the FDA noted.

[email protected]

The Food and Drug Administration has issued a safety alert approving new boxed warnings about increased blood clot and mortality risk associated with the 10-mg, twice-daily dose of tofacitinib (Xeljanz), as well as a new limitation for patients with ulcerative colitis receiving the medication.

Tofacitinib, a Janus kinase inhibitor, was first approved by the FDA in 2012 for the treatment of rheumatoid arthritis (RA). An indication for psoriatic arthritis was added in 2017, and one for ulcerative colitis was added in 2018.

After the 2012 approval, the FDA commissioned a postmarketing trial in patients with RA on background methotrexate to evaluate safety and the risk of cancer, heart-related events, and infection. The 5- and 10-mg tofacitinib twice daily doses are being analyzed in an ongoing study in comparison with a tumor necrosis factor (TNF) inhibitor.

An interim analysis of the trial’s data, as of January 2019, found an increased risk of blood clots and death in patients receiving 10-mg tofacitinib twice daily, compared with the TNF inhibitor and the twice-daily, 5-mg dose. Overall, there were 19 cases of blood clots in the lung out of 3,884 patient-years of follow-up in patients who received tofacitinib 10 mg twice daily, compared with 3 cases out of 3,982 patient-years in patients who received TNF inhibitors. There were also 45 cases of death from all causes during follow-up for tofacitinib 10 mg twice daily, compared with 25 cases in patients who received TNF inhibitors.

Patients with symptoms of thrombosis also receiving tofacitinib should immediately discontinue the medication. Tofacitinib should not be given to patients with ulcerative colitis unless they are not treated effectively with a TNF inhibitor or do not tolerate TNF inhibitors; ulcerative colitis patients should receive the lowest effective dosage, and if the higher dosage is necessary, it should be limited to the shortest amount of time possible, the FDA noted.

[email protected]

In 2016, two researchers published a meta-analysis on gray matter abnormalities in youth who had conduct problems.

The study by Jack C. Rogers, PhD, and Stephane A. De Brito, PhD, found 13 well-done studies that included 394 youth with conduct problems and 390 typically developing youth. Compared with the typically developing youth, the conduct-disordered youth had decreased gray matter volume (JAMA Psychiatry. 2016 Jan;73[1]:64-72).

As I knew one of the researchers in one of the studies that made the cut, I called him up and asked whether their research had controlled for fetal alcohol exposure. They had not. I found this very curious because my experience is that youth who have been labeled with conduct disorder often have histories of prenatal fetal alcohol exposure. In addition, my understanding is that youth who have been exposed to prenatal alcohol often have symptoms of conduct disorder. Furthermore, research has shown that such youth have smaller brains (Dev Med Child Neurol. 2001 Mar;43[3]:148-54). I wondered whether the youth studied in that trial had been exposed to alcohol prenatally.

More recently, this problem has resurfaced. An article by Antonia N. Kaczkurkin, PhD, and associates about a large sample of youth was nicely done. But again, the variable of fetal alcohol exposure was not considered. The study was an elegant one that provides a strong rationale for consideration of a “psychopathology factor” in human life (Am J Psychiatry. 2019 Jun 24. doi: 10.1176/appi.ajp.2019.1807035). It shored up that argument by doing neuroimaging studies on a reasonably large sample of youth and showed that reduced cortical thickness (gray matter volume) was associated with overall psychopathology. With the exception of failing to consider the variable of fetal alcohol exposure, the study is a valuable addition to our understanding of what might be going on with psychiatric disorders.

Unfortunately – while hating to sound like a broken record – I noticed that there was no consideration of fetal alcohol exposure as a cause for the findings of the study. It does not seem like a large leap to hypothesize some of these brain imaging studies that find smaller brain components associated with psychopathology and conduct disorder to be a dynamic of fetal alcohol exposure.

It seems to me that we made a huge mistake in public health in asking women only whether they were drinking while they were pregnant because it was the wrong question. The right question is – “When did you realize you were pregnant, and were you doing any social drinking before you knew you were pregnant?”

Without understanding the etiology of the smaller brains in patients with conduct disorder or psychopathology, we are missing a golden opportunity to prevent such problems. The former editor of the American Journal of Psychiatry – Robert A. Freedman, MD – suggests that by giving phosphatidyl choline to pregnant women, such problems could be prevented.

Dr. Bell is a staff psychiatrist at Jackson Park Hospital’s Medical/Surgical-Psychiatry Inpatient Unit in Chicago, clinical psychiatrist emeritus in the department of psychiatry at the University of Illinois at Chicago, former president/CEO of Community Mental Health Council, and former director of the Institute for Juvenile Research (birthplace of child psychiatry), also in Chicago. In 2019, he was awarded the Adolph Meyer Award by the American Psychiatric Association for lifetime achievement in psychiatric research.

In 2016, two researchers published a meta-analysis on gray matter abnormalities in youth who had conduct problems.

The study by Jack C. Rogers, PhD, and Stephane A. De Brito, PhD, found 13 well-done studies that included 394 youth with conduct problems and 390 typically developing youth. Compared with the typically developing youth, the conduct-disordered youth had decreased gray matter volume (JAMA Psychiatry. 2016 Jan;73[1]:64-72).

As I knew one of the researchers in one of the studies that made the cut, I called him up and asked whether their research had controlled for fetal alcohol exposure. They had not. I found this very curious because my experience is that youth who have been labeled with conduct disorder often have histories of prenatal fetal alcohol exposure. In addition, my understanding is that youth who have been exposed to prenatal alcohol often have symptoms of conduct disorder. Furthermore, research has shown that such youth have smaller brains (Dev Med Child Neurol. 2001 Mar;43[3]:148-54). I wondered whether the youth studied in that trial had been exposed to alcohol prenatally.

More recently, this problem has resurfaced. An article by Antonia N. Kaczkurkin, PhD, and associates about a large sample of youth was nicely done. But again, the variable of fetal alcohol exposure was not considered. The study was an elegant one that provides a strong rationale for consideration of a “psychopathology factor” in human life (Am J Psychiatry. 2019 Jun 24. doi: 10.1176/appi.ajp.2019.1807035). It shored up that argument by doing neuroimaging studies on a reasonably large sample of youth and showed that reduced cortical thickness (gray matter volume) was associated with overall psychopathology. With the exception of failing to consider the variable of fetal alcohol exposure, the study is a valuable addition to our understanding of what might be going on with psychiatric disorders.

Unfortunately – while hating to sound like a broken record – I noticed that there was no consideration of fetal alcohol exposure as a cause for the findings of the study. It does not seem like a large leap to hypothesize some of these brain imaging studies that find smaller brain components associated with psychopathology and conduct disorder to be a dynamic of fetal alcohol exposure.

It seems to me that we made a huge mistake in public health in asking women only whether they were drinking while they were pregnant because it was the wrong question. The right question is – “When did you realize you were pregnant, and were you doing any social drinking before you knew you were pregnant?”

Without understanding the etiology of the smaller brains in patients with conduct disorder or psychopathology, we are missing a golden opportunity to prevent such problems. The former editor of the American Journal of Psychiatry – Robert A. Freedman, MD – suggests that by giving phosphatidyl choline to pregnant women, such problems could be prevented.

Dr. Bell is a staff psychiatrist at Jackson Park Hospital’s Medical/Surgical-Psychiatry Inpatient Unit in Chicago, clinical psychiatrist emeritus in the department of psychiatry at the University of Illinois at Chicago, former president/CEO of Community Mental Health Council, and former director of the Institute for Juvenile Research (birthplace of child psychiatry), also in Chicago. In 2019, he was awarded the Adolph Meyer Award by the American Psychiatric Association for lifetime achievement in psychiatric research.

In 2016, two researchers published a meta-analysis on gray matter abnormalities in youth who had conduct problems.

The study by Jack C. Rogers, PhD, and Stephane A. De Brito, PhD, found 13 well-done studies that included 394 youth with conduct problems and 390 typically developing youth. Compared with the typically developing youth, the conduct-disordered youth had decreased gray matter volume (JAMA Psychiatry. 2016 Jan;73[1]:64-72).

As I knew one of the researchers in one of the studies that made the cut, I called him up and asked whether their research had controlled for fetal alcohol exposure. They had not. I found this very curious because my experience is that youth who have been labeled with conduct disorder often have histories of prenatal fetal alcohol exposure. In addition, my understanding is that youth who have been exposed to prenatal alcohol often have symptoms of conduct disorder. Furthermore, research has shown that such youth have smaller brains (Dev Med Child Neurol. 2001 Mar;43[3]:148-54). I wondered whether the youth studied in that trial had been exposed to alcohol prenatally.

More recently, this problem has resurfaced. An article by Antonia N. Kaczkurkin, PhD, and associates about a large sample of youth was nicely done. But again, the variable of fetal alcohol exposure was not considered. The study was an elegant one that provides a strong rationale for consideration of a “psychopathology factor” in human life (Am J Psychiatry. 2019 Jun 24. doi: 10.1176/appi.ajp.2019.1807035). It shored up that argument by doing neuroimaging studies on a reasonably large sample of youth and showed that reduced cortical thickness (gray matter volume) was associated with overall psychopathology. With the exception of failing to consider the variable of fetal alcohol exposure, the study is a valuable addition to our understanding of what might be going on with psychiatric disorders.

Unfortunately – while hating to sound like a broken record – I noticed that there was no consideration of fetal alcohol exposure as a cause for the findings of the study. It does not seem like a large leap to hypothesize some of these brain imaging studies that find smaller brain components associated with psychopathology and conduct disorder to be a dynamic of fetal alcohol exposure.

It seems to me that we made a huge mistake in public health in asking women only whether they were drinking while they were pregnant because it was the wrong question. The right question is – “When did you realize you were pregnant, and were you doing any social drinking before you knew you were pregnant?”

Without understanding the etiology of the smaller brains in patients with conduct disorder or psychopathology, we are missing a golden opportunity to prevent such problems. The former editor of the American Journal of Psychiatry – Robert A. Freedman, MD – suggests that by giving phosphatidyl choline to pregnant women, such problems could be prevented.

Dr. Bell is a staff psychiatrist at Jackson Park Hospital’s Medical/Surgical-Psychiatry Inpatient Unit in Chicago, clinical psychiatrist emeritus in the department of psychiatry at the University of Illinois at Chicago, former president/CEO of Community Mental Health Council, and former director of the Institute for Juvenile Research (birthplace of child psychiatry), also in Chicago. In 2019, he was awarded the Adolph Meyer Award by the American Psychiatric Association for lifetime achievement in psychiatric research.

The general fertility rate in the United States decreased 2% between 2017 and 2018, according to a report from the Centers for Disease Control and Prevention.

©fotolia

Fertility rates, defined as births per 1,000 women aged 15-44 years, declined for all racial/ethnic groups studied.

Teen birth rates, or births among girls aged 15-19 years, declined from 2017 to 2018 as well.

These data come from the National Vital Statistics System’s Natality Data File, which includes information from birth certificates for all births in the United States.

The data show a decline in the general fertility rate from 60.3 per 1,000 women in 2017 to 59.1 per 1,000 women in 2018, a significant decrease (P less than .05).

Fertility rates declined across the three largest racial/ethnic groups studied, decreasing:

• 3% in Hispanic women, from 67.6 to 65.9 per 1,000.
• 2% in non-Hispanic black women, from 63.1 to 62.0 per 1,000.
• 2% in non-Hispanic white women, from 57.2 to 56.3 per 1,000.

Similarly, teen birth rates declined 7% from 2017 to 2018, decreasing from 18.8 to 17.4 births per 1,000 girls aged 15-19 years (P less than .05). Rates decreased:

• 8% in Hispanic teens, from 28.9 to 26.7 per 1,000.
• 4% in non-Hispanic black teens, from 27.5 to 26.3 per 1,000.
• 8% in non-Hispanic white teens, from 13.2 to 12.1 per 1,000.

The data also show an increase in the rate of vaginal births after previous cesarean (VBAC) delivery. The percentage of VBAC deliveries increased from 12.8% in 2017 to 13.3% in 2018 (P less than .05).

VBAC delivery rates increased across all racial/ethnic groups studied, although the increase among non-Hispanic back women was not significant.

Finally, the report shows an increase in preterm and early term births from 2017 to 2018. Preterm deliveries (less than 37 weeks of gestation) increased from 9.93% to 10.02%, and early term deliveries (37-38 weeks) increased from 26.00% to 26.53% (P less than .05).

At the same time, full-term births (39-40 weeks) decreased from 57.49% to 57.24%, and late- and post-term births (41 weeks or more) decreased from 6.58 % to 6.20% (P less than .05). These findings were consistent across the racial/ethnic groups studied.

[email protected]

SOURCE: Martin JA et al. NCHS Data Brief. 2019 July; no 346.

The general fertility rate in the United States decreased 2% between 2017 and 2018, according to a report from the Centers for Disease Control and Prevention.

©fotolia

Fertility rates, defined as births per 1,000 women aged 15-44 years, declined for all racial/ethnic groups studied.

Teen birth rates, or births among girls aged 15-19 years, declined from 2017 to 2018 as well.

These data come from the National Vital Statistics System’s Natality Data File, which includes information from birth certificates for all births in the United States.

The data show a decline in the general fertility rate from 60.3 per 1,000 women in 2017 to 59.1 per 1,000 women in 2018, a significant decrease (P less than .05).

Fertility rates declined across the three largest racial/ethnic groups studied, decreasing:

• 3% in Hispanic women, from 67.6 to 65.9 per 1,000.
• 2% in non-Hispanic black women, from 63.1 to 62.0 per 1,000.
• 2% in non-Hispanic white women, from 57.2 to 56.3 per 1,000.

Similarly, teen birth rates declined 7% from 2017 to 2018, decreasing from 18.8 to 17.4 births per 1,000 girls aged 15-19 years (P less than .05). Rates decreased:

• 8% in Hispanic teens, from 28.9 to 26.7 per 1,000.
• 4% in non-Hispanic black teens, from 27.5 to 26.3 per 1,000.
• 8% in non-Hispanic white teens, from 13.2 to 12.1 per 1,000.

The data also show an increase in the rate of vaginal births after previous cesarean (VBAC) delivery. The percentage of VBAC deliveries increased from 12.8% in 2017 to 13.3% in 2018 (P less than .05).

VBAC delivery rates increased across all racial/ethnic groups studied, although the increase among non-Hispanic back women was not significant.

Finally, the report shows an increase in preterm and early term births from 2017 to 2018. Preterm deliveries (less than 37 weeks of gestation) increased from 9.93% to 10.02%, and early term deliveries (37-38 weeks) increased from 26.00% to 26.53% (P less than .05).

At the same time, full-term births (39-40 weeks) decreased from 57.49% to 57.24%, and late- and post-term births (41 weeks or more) decreased from 6.58 % to 6.20% (P less than .05). These findings were consistent across the racial/ethnic groups studied.

[email protected]

SOURCE: Martin JA et al. NCHS Data Brief. 2019 July; no 346.

The general fertility rate in the United States decreased 2% between 2017 and 2018, according to a report from the Centers for Disease Control and Prevention.

©fotolia

Fertility rates, defined as births per 1,000 women aged 15-44 years, declined for all racial/ethnic groups studied.

Teen birth rates, or births among girls aged 15-19 years, declined from 2017 to 2018 as well.

These data come from the National Vital Statistics System’s Natality Data File, which includes information from birth certificates for all births in the United States.

The data show a decline in the general fertility rate from 60.3 per 1,000 women in 2017 to 59.1 per 1,000 women in 2018, a significant decrease (P less than .05).

Fertility rates declined across the three largest racial/ethnic groups studied, decreasing:

• 3% in Hispanic women, from 67.6 to 65.9 per 1,000.
• 2% in non-Hispanic black women, from 63.1 to 62.0 per 1,000.
• 2% in non-Hispanic white women, from 57.2 to 56.3 per 1,000.

Similarly, teen birth rates declined 7% from 2017 to 2018, decreasing from 18.8 to 17.4 births per 1,000 girls aged 15-19 years (P less than .05). Rates decreased:

• 8% in Hispanic teens, from 28.9 to 26.7 per 1,000.
• 4% in non-Hispanic black teens, from 27.5 to 26.3 per 1,000.
• 8% in non-Hispanic white teens, from 13.2 to 12.1 per 1,000.

The data also show an increase in the rate of vaginal births after previous cesarean (VBAC) delivery. The percentage of VBAC deliveries increased from 12.8% in 2017 to 13.3% in 2018 (P less than .05).

VBAC delivery rates increased across all racial/ethnic groups studied, although the increase among non-Hispanic back women was not significant.

Finally, the report shows an increase in preterm and early term births from 2017 to 2018. Preterm deliveries (less than 37 weeks of gestation) increased from 9.93% to 10.02%, and early term deliveries (37-38 weeks) increased from 26.00% to 26.53% (P less than .05).

At the same time, full-term births (39-40 weeks) decreased from 57.49% to 57.24%, and late- and post-term births (41 weeks or more) decreased from 6.58 % to 6.20% (P less than .05). These findings were consistent across the racial/ethnic groups studied.

[email protected]

SOURCE: Martin JA et al. NCHS Data Brief. 2019 July; no 346.

The DTaP, hepatitis B virus, and Haemophilus influenzae type b (Hib) vaccine was found to be noninferior to a similar, commercially available vaccine in infants, according to a study in Vaccine.

MarianVejcik/Getty Images

In this phase 3, randomized, single-blind, multicenter, noninferiority study, Sai Krishna Susarla of Human Biologicals Institute, which developed the test vaccine, and colleagues randomized 405 infants aged 6-8 weeks 2:1 to three doses of either the test vaccine or the comparator, Pentavac SD (Serum Institute of India). The percentages of seroconversion for diphtheria, pertussis, hepatitis B, and Hib were 98.44%, 92.61%, 99.22%, and 95.72% for the test vaccine, respectively, and 90.0%, 89.23%, 100%, and 90.77% for the comparator. In keeping with some previous studies, the percentages for tetanus were low at 50.97% with the test vaccine and 30.23% with the comparator. Despite the low seroconversion for tetanus, the test vaccine was determined to be noninferior to the comparator for it and the other four diseases it targets. The safety profile was also found to be comparable.

Although the study’s major limitation is that it was conducted in only one country, “the strength of the study is considered to be good” because “compliance to protocol was good, deviations were minimal, and ... very few subjects were withdrawn,” the researchers wrote.

Some of the researchers were employees of the sponsor, Human Biologicals Institute, which developed the test vaccine. Other researchers had no financial interest in the test vaccine and were unrelated to the sponsor, but did receive research grants for conducting the study at their respective sites.

[email protected]

SOURCE: Susarla SK et al. Vaccine. 2019 Jul 19. doi: 10.1016/j.vaccine.2019.06.067.

The DTaP, hepatitis B virus, and Haemophilus influenzae type b (Hib) vaccine was found to be noninferior to a similar, commercially available vaccine in infants, according to a study in Vaccine.

MarianVejcik/Getty Images

In this phase 3, randomized, single-blind, multicenter, noninferiority study, Sai Krishna Susarla of Human Biologicals Institute, which developed the test vaccine, and colleagues randomized 405 infants aged 6-8 weeks 2:1 to three doses of either the test vaccine or the comparator, Pentavac SD (Serum Institute of India). The percentages of seroconversion for diphtheria, pertussis, hepatitis B, and Hib were 98.44%, 92.61%, 99.22%, and 95.72% for the test vaccine, respectively, and 90.0%, 89.23%, 100%, and 90.77% for the comparator. In keeping with some previous studies, the percentages for tetanus were low at 50.97% with the test vaccine and 30.23% with the comparator. Despite the low seroconversion for tetanus, the test vaccine was determined to be noninferior to the comparator for it and the other four diseases it targets. The safety profile was also found to be comparable.

Although the study’s major limitation is that it was conducted in only one country, “the strength of the study is considered to be good” because “compliance to protocol was good, deviations were minimal, and ... very few subjects were withdrawn,” the researchers wrote.

Some of the researchers were employees of the sponsor, Human Biologicals Institute, which developed the test vaccine. Other researchers had no financial interest in the test vaccine and were unrelated to the sponsor, but did receive research grants for conducting the study at their respective sites.

[email protected]

SOURCE: Susarla SK et al. Vaccine. 2019 Jul 19. doi: 10.1016/j.vaccine.2019.06.067.

The DTaP, hepatitis B virus, and Haemophilus influenzae type b (Hib) vaccine was found to be noninferior to a similar, commercially available vaccine in infants, according to a study in Vaccine.

MarianVejcik/Getty Images

In this phase 3, randomized, single-blind, multicenter, noninferiority study, Sai Krishna Susarla of Human Biologicals Institute, which developed the test vaccine, and colleagues randomized 405 infants aged 6-8 weeks 2:1 to three doses of either the test vaccine or the comparator, Pentavac SD (Serum Institute of India). The percentages of seroconversion for diphtheria, pertussis, hepatitis B, and Hib were 98.44%, 92.61%, 99.22%, and 95.72% for the test vaccine, respectively, and 90.0%, 89.23%, 100%, and 90.77% for the comparator. In keeping with some previous studies, the percentages for tetanus were low at 50.97% with the test vaccine and 30.23% with the comparator. Despite the low seroconversion for tetanus, the test vaccine was determined to be noninferior to the comparator for it and the other four diseases it targets. The safety profile was also found to be comparable.

Although the study’s major limitation is that it was conducted in only one country, “the strength of the study is considered to be good” because “compliance to protocol was good, deviations were minimal, and ... very few subjects were withdrawn,” the researchers wrote.

Some of the researchers were employees of the sponsor, Human Biologicals Institute, which developed the test vaccine. Other researchers had no financial interest in the test vaccine and were unrelated to the sponsor, but did receive research grants for conducting the study at their respective sites.

[email protected]

SOURCE: Susarla SK et al. Vaccine. 2019 Jul 19. doi: 10.1016/j.vaccine.2019.06.067.

ORLANDO – Inhaler choice for patients with chronic obstructive pulmonary disease (COPD) should be chosen on the basis of access, cost, prescriber, patient ability, and patient preference.

That’s according to Gabriel Ortiz, MPAS, PA-C, DFAAPA, a speaker at the Cardiovascular & Respiratory Summit by Global Academy for Medical Education.

There are several treatment options available in inhaler form for patients with COPD, including short-acting and long-acting beta₂-agonists (SABA/LABA), short-acting and long-acting antimuscarinics (SAMA/LAMA), combination therapy, and triple therapy. But education and training on how to use an inhaler is also important, Mr. Ortiz said in his presentation.

Providers should help a patient choose an inhaler they are most likely to use. “It’s difficult to actuate and inhale, so there’s a lot of education that goes into that,” said Mr. Ortiz, of Pediatric Pulmonary Services in El Paso, Texas. “What we do for our patients is, we educate them, we tell them to demonstrate it, then bring [the] inhaler back on the next visit and demonstrate to us every time.”

“Make sure that they demonstrate because, as patients get older, they may lose their ability to actuate and inhale,” he added.

Adherence to therapy should also be considered before changing from a current therapy, he added. Mr. Ortiz described a scenario in which a prescription was filled, but because of the cost, the patient reduced the dose by half to make the therapy last longer.

“We could be the best providers in the world, prescribing the best medication in the world. If the medication doesn’t get to where we need it, it’s not going to help anybody,” he said.

Providers should also use nonpharmacological treatments to prevent or keep COPD from progressing. Smoking cessation is key to reducing the risk of developing COPD, and it is not clear whether e-cigarettes aid in smoking cessation, despite companies that market these products making that claim. “We have a huge e-cigarette epidemic here,” he said.

Instead, Mr. Ortiz recommended identifying which patients are current tobacco users, encouraging them to quit, determining whether the patient is willing to make a commitment to cut down on tobacco use or stop entirely, helping draft a quit plan and obtaining intra- and extratreatment social support for smoking cessation, and scheduling follow-up. Providers should discuss smoking cessation at each visit, and it may take multiple visits before a patient is willing to consider quitting, he said.

Another measure providers can take is making sure patients with COPD have received influenza and pneumococcal vaccinations because this can lower the risk of respiratory tract infections. Physical activity, training in exercise, nutritional support, and pulmonary rehabilitation can improve their health status, dyspnea, and exercise tolerance. While C-level evidence shows education alone is not effective according to Global Initiative for Chronic Obstructive Lung Disease (GOLD) guidelines, providers working with patients through self-management interventions have been shown to improve health status and decrease COPD-related hospitalizations.

Oxygen therapy has been effective for patients with severe resting chronic hypoxia and improves survival, said Mr. Ortiz. For patients who gradually decline in health despite treatment, palliative, hospice, and end-of-life care may be an option. “Remember that as acute exacerbations continue, that increases the risk of death,” he said. “We want to try and prevent [those] exacerbations and improve quality of life.”

During follow-up visits, providers should continue performing spirometry tests annually to measure decline in forced expiratory volume in 1 second, information on symptoms that have presented since the previous visit, details of any exacerbations that occurred, and current smoking status. When making decisions to adjust therapy, providers should examine the effectiveness of the current regimen and consider the dose of prescribed medications, whether the patient is adhering to the regimen, inhaler technique, and any side effects.

Mr. Ortiz reports also being a paid employee for Thermo Fisher, a biotechnology product development company based in Waltham, Mass. Global Academy for Medical Education and this news organization are owned by the same parent company.


 

ORLANDO – Inhaler choice for patients with chronic obstructive pulmonary disease (COPD) should be chosen on the basis of access, cost, prescriber, patient ability, and patient preference.

That’s according to Gabriel Ortiz, MPAS, PA-C, DFAAPA, a speaker at the Cardiovascular & Respiratory Summit by Global Academy for Medical Education.

There are several treatment options available in inhaler form for patients with COPD, including short-acting and long-acting beta₂-agonists (SABA/LABA), short-acting and long-acting antimuscarinics (SAMA/LAMA), combination therapy, and triple therapy. But education and training on how to use an inhaler is also important, Mr. Ortiz said in his presentation.

Providers should help a patient choose an inhaler they are most likely to use. “It’s difficult to actuate and inhale, so there’s a lot of education that goes into that,” said Mr. Ortiz, of Pediatric Pulmonary Services in El Paso, Texas. “What we do for our patients is, we educate them, we tell them to demonstrate it, then bring [the] inhaler back on the next visit and demonstrate to us every time.”

“Make sure that they demonstrate because, as patients get older, they may lose their ability to actuate and inhale,” he added.

Adherence to therapy should also be considered before changing from a current therapy, he added. Mr. Ortiz described a scenario in which a prescription was filled, but because of the cost, the patient reduced the dose by half to make the therapy last longer.

“We could be the best providers in the world, prescribing the best medication in the world. If the medication doesn’t get to where we need it, it’s not going to help anybody,” he said.

Providers should also use nonpharmacological treatments to prevent or keep COPD from progressing. Smoking cessation is key to reducing the risk of developing COPD, and it is not clear whether e-cigarettes aid in smoking cessation, despite companies that market these products making that claim. “We have a huge e-cigarette epidemic here,” he said.

Instead, Mr. Ortiz recommended identifying which patients are current tobacco users, encouraging them to quit, determining whether the patient is willing to make a commitment to cut down on tobacco use or stop entirely, helping draft a quit plan and obtaining intra- and extratreatment social support for smoking cessation, and scheduling follow-up. Providers should discuss smoking cessation at each visit, and it may take multiple visits before a patient is willing to consider quitting, he said.

Another measure providers can take is making sure patients with COPD have received influenza and pneumococcal vaccinations because this can lower the risk of respiratory tract infections. Physical activity, training in exercise, nutritional support, and pulmonary rehabilitation can improve their health status, dyspnea, and exercise tolerance. While C-level evidence shows education alone is not effective according to Global Initiative for Chronic Obstructive Lung Disease (GOLD) guidelines, providers working with patients through self-management interventions have been shown to improve health status and decrease COPD-related hospitalizations.

Oxygen therapy has been effective for patients with severe resting chronic hypoxia and improves survival, said Mr. Ortiz. For patients who gradually decline in health despite treatment, palliative, hospice, and end-of-life care may be an option. “Remember that as acute exacerbations continue, that increases the risk of death,” he said. “We want to try and prevent [those] exacerbations and improve quality of life.”

During follow-up visits, providers should continue performing spirometry tests annually to measure decline in forced expiratory volume in 1 second, information on symptoms that have presented since the previous visit, details of any exacerbations that occurred, and current smoking status. When making decisions to adjust therapy, providers should examine the effectiveness of the current regimen and consider the dose of prescribed medications, whether the patient is adhering to the regimen, inhaler technique, and any side effects.

Mr. Ortiz reports also being a paid employee for Thermo Fisher, a biotechnology product development company based in Waltham, Mass. Global Academy for Medical Education and this news organization are owned by the same parent company.


 

ORLANDO – Inhaler choice for patients with chronic obstructive pulmonary disease (COPD) should be chosen on the basis of access, cost, prescriber, patient ability, and patient preference.

That’s according to Gabriel Ortiz, MPAS, PA-C, DFAAPA, a speaker at the Cardiovascular & Respiratory Summit by Global Academy for Medical Education.

There are several treatment options available in inhaler form for patients with COPD, including short-acting and long-acting beta₂-agonists (SABA/LABA), short-acting and long-acting antimuscarinics (SAMA/LAMA), combination therapy, and triple therapy. But education and training on how to use an inhaler is also important, Mr. Ortiz said in his presentation.

Providers should help a patient choose an inhaler they are most likely to use. “It’s difficult to actuate and inhale, so there’s a lot of education that goes into that,” said Mr. Ortiz, of Pediatric Pulmonary Services in El Paso, Texas. “What we do for our patients is, we educate them, we tell them to demonstrate it, then bring [the] inhaler back on the next visit and demonstrate to us every time.”

“Make sure that they demonstrate because, as patients get older, they may lose their ability to actuate and inhale,” he added.

Adherence to therapy should also be considered before changing from a current therapy, he added. Mr. Ortiz described a scenario in which a prescription was filled, but because of the cost, the patient reduced the dose by half to make the therapy last longer.

“We could be the best providers in the world, prescribing the best medication in the world. If the medication doesn’t get to where we need it, it’s not going to help anybody,” he said.

Providers should also use nonpharmacological treatments to prevent or keep COPD from progressing. Smoking cessation is key to reducing the risk of developing COPD, and it is not clear whether e-cigarettes aid in smoking cessation, despite companies that market these products making that claim. “We have a huge e-cigarette epidemic here,” he said.

Instead, Mr. Ortiz recommended identifying which patients are current tobacco users, encouraging them to quit, determining whether the patient is willing to make a commitment to cut down on tobacco use or stop entirely, helping draft a quit plan and obtaining intra- and extratreatment social support for smoking cessation, and scheduling follow-up. Providers should discuss smoking cessation at each visit, and it may take multiple visits before a patient is willing to consider quitting, he said.

Another measure providers can take is making sure patients with COPD have received influenza and pneumococcal vaccinations because this can lower the risk of respiratory tract infections. Physical activity, training in exercise, nutritional support, and pulmonary rehabilitation can improve their health status, dyspnea, and exercise tolerance. While C-level evidence shows education alone is not effective according to Global Initiative for Chronic Obstructive Lung Disease (GOLD) guidelines, providers working with patients through self-management interventions have been shown to improve health status and decrease COPD-related hospitalizations.

Oxygen therapy has been effective for patients with severe resting chronic hypoxia and improves survival, said Mr. Ortiz. For patients who gradually decline in health despite treatment, palliative, hospice, and end-of-life care may be an option. “Remember that as acute exacerbations continue, that increases the risk of death,” he said. “We want to try and prevent [those] exacerbations and improve quality of life.”

During follow-up visits, providers should continue performing spirometry tests annually to measure decline in forced expiratory volume in 1 second, information on symptoms that have presented since the previous visit, details of any exacerbations that occurred, and current smoking status. When making decisions to adjust therapy, providers should examine the effectiveness of the current regimen and consider the dose of prescribed medications, whether the patient is adhering to the regimen, inhaler technique, and any side effects.

Mr. Ortiz reports also being a paid employee for Thermo Fisher, a biotechnology product development company based in Waltham, Mass. Global Academy for Medical Education and this news organization are owned by the same parent company.


 

The Florida Society of Rheumatology is an excellent state conference that is very well attended because of the comprehensive clinical topics covered by esteemed faculty. Every year, there is a balance of patient care lectures and updates in advocacy, billing, and coding. Clinicians need a combination of both arenas to be successful with the day-to-day practice of rheumatology and to render evidenced-based patient care. This year, the FSR certainly delivered on this mission as reflected by articles on these presentations published at MDedge Rheumatology. An added focus this year was how to leverage technology in a rheumatology practice to capture patient-reported outcomes (PROs) to better understand issues affecting our patient population and improve therapy plans where indicated.

In his lecture on digital PROs, Jeffrey Curtis, MD, explained the difference between active capture of data through tools such as the Routine Assessment of Patient Index Data 3 (RAPID3) or Health Assessment Questionnaire (HAQ) and passive capture through wearable devices such as a Fitbit or Apple watch. A key point for the audience was that this information improves clinical care and improves medical decision making, and thus all rheumatologists should consider using these tools in practice. Dr. Curtis, William J. Koopman Endowed Professor in Rheumatology and Immunology and director of the UAB Arthritis Clinical Intervention Program at the University of Alabama at Birmingham, is well aware of the practical concerns that face clinicians, namely that this is time consuming. He suggests to keep it short and find a tool that works for you in your practice to understand how your patients are progressing on a treatment regimen. He was clear that “data for the sake of data is not compelling for patients [or clinicians].” The ideal is not to paralyze your practice and drown in patient questionnaires but rather to empower patients to report using standardized tools so we can effect change that will help us to treat rheumatic diseases.

An important point mentioned during this lecture was to keep in mind that, if a patient appears to be a “nonresponder” on RAPID3, for example, it is important to understand whether the patient has a confounding comorbidity, such as fibromyalgia, that may account for the limited improvement.

Michelle Petri, MD, gave two excellent talks at FSR this year. Her lectures are packed with excellent pearls about treating patients with systemic lupus erythematosus. Interestingly, she said to never underestimate the prognostic factor of a low C3. This can indicate a worse clinical course is ahead. In addition, she reminds us as clinicians to protect the kidneys of our lupus patients who have renal disease by avoiding common toxins such as NSAIDs and CT contrast. Of course, she reminds us to use the lowest dose of steroids possible during flares, as prednisone is directly or indirectly responsible for 80% of organ damage over 15 years. She reminds us that lupus patients do not die of lupus. They have a 2.66-fold higher risk of cardiovascular events than the general public. In addition to maintaining lupus patients on hydroxychloroquine, Dr. Petri, professor of medicine and director of the Hopkins Lupus Center at Johns Hopkins University, Baltimore, noted that vitamin D can have cardiovascular and hematologic benefits along with reducing thrombosis in some clinical studies. Low vitamin D was significantly associated with deep venous thrombosis.

In his lecture, Leonard Calabrese, DO, made a compelling argument for the rheumatologists in the audience to call the local oncologists with whom they work. We need to discuss and collaborate on the care of patients experiencing immune-mediated adverse events from exposure to checkpoint inhibitors used to treat malignancy. There is a limited mechanistic understanding of these adverse events, but as rheumatologists we need to get involved and help these patients. We are the experts in managing these newly emerging autoimmune events. We can help to create the best possible therapeutic interventions to help our oncology colleagues with these challenging cases, Dr. Calabrese, professor of medicine and chair of clinical immunology at the Cleveland Clinic, said.

Besides paying our dues to be members of the FSR, it is important for us as rheumatologists to get involved at the state legislature and national level to bring about change for our practices and patients. Currently, the climate can be hostile for reimbursement and for our patients to get the therapies they need. In another presentation, Angus Worthing, MD, chair of the American College of Rheumatology’s Government Affairs Committee, described recent successes at the national level, and he also discussed how we can have our voices heard at the state and national level to protect our profession and the people who rely on our expertise. The FSR and other state rheumatology organizations, as well as the ACR, need our support to continue to be the collective voice for what is right for clinicians and patients alike.
 

Dr. Oberstein is a practicing rheumatologist at the University of Miami Health System and is senior medical director of musculoskeletal at Modernizing Medicine in Boca Raton, Fla. She has no relevant disclosures to report.

The Florida Society of Rheumatology is an excellent state conference that is very well attended because of the comprehensive clinical topics covered by esteemed faculty. Every year, there is a balance of patient care lectures and updates in advocacy, billing, and coding. Clinicians need a combination of both arenas to be successful with the day-to-day practice of rheumatology and to render evidenced-based patient care. This year, the FSR certainly delivered on this mission as reflected by articles on these presentations published at MDedge Rheumatology. An added focus this year was how to leverage technology in a rheumatology practice to capture patient-reported outcomes (PROs) to better understand issues affecting our patient population and improve therapy plans where indicated.

In his lecture on digital PROs, Jeffrey Curtis, MD, explained the difference between active capture of data through tools such as the Routine Assessment of Patient Index Data 3 (RAPID3) or Health Assessment Questionnaire (HAQ) and passive capture through wearable devices such as a Fitbit or Apple watch. A key point for the audience was that this information improves clinical care and improves medical decision making, and thus all rheumatologists should consider using these tools in practice. Dr. Curtis, William J. Koopman Endowed Professor in Rheumatology and Immunology and director of the UAB Arthritis Clinical Intervention Program at the University of Alabama at Birmingham, is well aware of the practical concerns that face clinicians, namely that this is time consuming. He suggests to keep it short and find a tool that works for you in your practice to understand how your patients are progressing on a treatment regimen. He was clear that “data for the sake of data is not compelling for patients [or clinicians].” The ideal is not to paralyze your practice and drown in patient questionnaires but rather to empower patients to report using standardized tools so we can effect change that will help us to treat rheumatic diseases.

An important point mentioned during this lecture was to keep in mind that, if a patient appears to be a “nonresponder” on RAPID3, for example, it is important to understand whether the patient has a confounding comorbidity, such as fibromyalgia, that may account for the limited improvement.

Michelle Petri, MD, gave two excellent talks at FSR this year. Her lectures are packed with excellent pearls about treating patients with systemic lupus erythematosus. Interestingly, she said to never underestimate the prognostic factor of a low C3. This can indicate a worse clinical course is ahead. In addition, she reminds us as clinicians to protect the kidneys of our lupus patients who have renal disease by avoiding common toxins such as NSAIDs and CT contrast. Of course, she reminds us to use the lowest dose of steroids possible during flares, as prednisone is directly or indirectly responsible for 80% of organ damage over 15 years. She reminds us that lupus patients do not die of lupus. They have a 2.66-fold higher risk of cardiovascular events than the general public. In addition to maintaining lupus patients on hydroxychloroquine, Dr. Petri, professor of medicine and director of the Hopkins Lupus Center at Johns Hopkins University, Baltimore, noted that vitamin D can have cardiovascular and hematologic benefits along with reducing thrombosis in some clinical studies. Low vitamin D was significantly associated with deep venous thrombosis.

In his lecture, Leonard Calabrese, DO, made a compelling argument for the rheumatologists in the audience to call the local oncologists with whom they work. We need to discuss and collaborate on the care of patients experiencing immune-mediated adverse events from exposure to checkpoint inhibitors used to treat malignancy. There is a limited mechanistic understanding of these adverse events, but as rheumatologists we need to get involved and help these patients. We are the experts in managing these newly emerging autoimmune events. We can help to create the best possible therapeutic interventions to help our oncology colleagues with these challenging cases, Dr. Calabrese, professor of medicine and chair of clinical immunology at the Cleveland Clinic, said.

Besides paying our dues to be members of the FSR, it is important for us as rheumatologists to get involved at the state legislature and national level to bring about change for our practices and patients. Currently, the climate can be hostile for reimbursement and for our patients to get the therapies they need. In another presentation, Angus Worthing, MD, chair of the American College of Rheumatology’s Government Affairs Committee, described recent successes at the national level, and he also discussed how we can have our voices heard at the state and national level to protect our profession and the people who rely on our expertise. The FSR and other state rheumatology organizations, as well as the ACR, need our support to continue to be the collective voice for what is right for clinicians and patients alike.
 

Dr. Oberstein is a practicing rheumatologist at the University of Miami Health System and is senior medical director of musculoskeletal at Modernizing Medicine in Boca Raton, Fla. She has no relevant disclosures to report.

The Florida Society of Rheumatology is an excellent state conference that is very well attended because of the comprehensive clinical topics covered by esteemed faculty. Every year, there is a balance of patient care lectures and updates in advocacy, billing, and coding. Clinicians need a combination of both arenas to be successful with the day-to-day practice of rheumatology and to render evidenced-based patient care. This year, the FSR certainly delivered on this mission as reflected by articles on these presentations published at MDedge Rheumatology. An added focus this year was how to leverage technology in a rheumatology practice to capture patient-reported outcomes (PROs) to better understand issues affecting our patient population and improve therapy plans where indicated.

In his lecture on digital PROs, Jeffrey Curtis, MD, explained the difference between active capture of data through tools such as the Routine Assessment of Patient Index Data 3 (RAPID3) or Health Assessment Questionnaire (HAQ) and passive capture through wearable devices such as a Fitbit or Apple watch. A key point for the audience was that this information improves clinical care and improves medical decision making, and thus all rheumatologists should consider using these tools in practice. Dr. Curtis, William J. Koopman Endowed Professor in Rheumatology and Immunology and director of the UAB Arthritis Clinical Intervention Program at the University of Alabama at Birmingham, is well aware of the practical concerns that face clinicians, namely that this is time consuming. He suggests to keep it short and find a tool that works for you in your practice to understand how your patients are progressing on a treatment regimen. He was clear that “data for the sake of data is not compelling for patients [or clinicians].” The ideal is not to paralyze your practice and drown in patient questionnaires but rather to empower patients to report using standardized tools so we can effect change that will help us to treat rheumatic diseases.

An important point mentioned during this lecture was to keep in mind that, if a patient appears to be a “nonresponder” on RAPID3, for example, it is important to understand whether the patient has a confounding comorbidity, such as fibromyalgia, that may account for the limited improvement.

Michelle Petri, MD, gave two excellent talks at FSR this year. Her lectures are packed with excellent pearls about treating patients with systemic lupus erythematosus. Interestingly, she said to never underestimate the prognostic factor of a low C3. This can indicate a worse clinical course is ahead. In addition, she reminds us as clinicians to protect the kidneys of our lupus patients who have renal disease by avoiding common toxins such as NSAIDs and CT contrast. Of course, she reminds us to use the lowest dose of steroids possible during flares, as prednisone is directly or indirectly responsible for 80% of organ damage over 15 years. She reminds us that lupus patients do not die of lupus. They have a 2.66-fold higher risk of cardiovascular events than the general public. In addition to maintaining lupus patients on hydroxychloroquine, Dr. Petri, professor of medicine and director of the Hopkins Lupus Center at Johns Hopkins University, Baltimore, noted that vitamin D can have cardiovascular and hematologic benefits along with reducing thrombosis in some clinical studies. Low vitamin D was significantly associated with deep venous thrombosis.

In his lecture, Leonard Calabrese, DO, made a compelling argument for the rheumatologists in the audience to call the local oncologists with whom they work. We need to discuss and collaborate on the care of patients experiencing immune-mediated adverse events from exposure to checkpoint inhibitors used to treat malignancy. There is a limited mechanistic understanding of these adverse events, but as rheumatologists we need to get involved and help these patients. We are the experts in managing these newly emerging autoimmune events. We can help to create the best possible therapeutic interventions to help our oncology colleagues with these challenging cases, Dr. Calabrese, professor of medicine and chair of clinical immunology at the Cleveland Clinic, said.

Besides paying our dues to be members of the FSR, it is important for us as rheumatologists to get involved at the state legislature and national level to bring about change for our practices and patients. Currently, the climate can be hostile for reimbursement and for our patients to get the therapies they need. In another presentation, Angus Worthing, MD, chair of the American College of Rheumatology’s Government Affairs Committee, described recent successes at the national level, and he also discussed how we can have our voices heard at the state and national level to protect our profession and the people who rely on our expertise. The FSR and other state rheumatology organizations, as well as the ACR, need our support to continue to be the collective voice for what is right for clinicians and patients alike.
 

Dr. Oberstein is a practicing rheumatologist at the University of Miami Health System and is senior medical director of musculoskeletal at Modernizing Medicine in Boca Raton, Fla. She has no relevant disclosures to report.

The recently published PIONEER-HF study attempts to move sacubitril/valsartan (Entresto) therapy to the inpatient environment to improve patient and physician acceptance of this therapy for patients with heart failure (N Engl J Med. 2019 Feb 7;380;539-48).

When given to outpatients in the PARADIGM-HF trial, the combination was superior to enalapril for reducing the risks of death and hospitalization for heart failure (N Engl J Med 2014;371:993-1004.) Specifically, sacubitril/valsartan decreased mortality by 15% and hospitalization by 21% as an outpatient therapy for patients with systolic heart failure. Nevertheless, there has not been widespread adoption of this approach. It is well known that physicians can be slow to adopt new therapies, but one overriding factor may be the cost of the drug compared to enalapril, one of the first drugs shown to be effective in heart failure therapy (Entresto costs more than $4,000 per year; enalapril costs about $120 per year).

The investigators in the PIONEER-HF study compared Entresto to enalapril over a 2-month period in patients hospitalized with systolic heart failure. To accelerate the trial, the investigators used the proportional change in patients’ N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels as the primary endpoint rather than the traditional outcome of morbidity and mortality. In the short term, no significant clinical benefits were observed, but there was a significant decrease in NT-proBNP of about 30% (P less than .001).

The investigators suggested that this finding extended the previous benefit observed with Entresto during outpatient initiation and could be used as a rationale for initiating Entresto therapy in the hospital. This earlier application of the therapy could make the drug more widely acceptable.

Considerable investigation in BNP measurement has occurred over the last few years, and although it is clear that BNP is elevated in heart failure patients, there is no evidence to confirm that the decrease in BNP is associated with improved outcome. BNP will fall with decrease in ventricular volume, which may have significant physiologic mechanisms but ventricular volume could decrease with fall in blood pressure that may have occurred in this population since hypotension tended to be more frequent with Entresto than with enalapril. The traditional measure of heart failure benefit with beta-blockers, ACE inhibitors, and aldosterone antagonists in the inpatient and early postdischarge period has depended on clinical outcomes.

Regardless of the physiologic explanation of this fall in BNP, we must pause in our assumptions when a surrogate measure is used to assess clinical benefit as inpatient therapy. The Food and Drug Administration has long given up using surrogate measures as proof of efficacy, and rightly so. Clinical medicine is replete with dubious drug benefits based on surrogate measures. Let’s not forget that only a few years ago suppression of premature ventricular contractions was considered to be a measure of the pharmacologic prevention of sudden death. We have come a long way from that and other clinical missteps to use BNP, an uncertain marker at best of clinical improvement, as a surrogate for the improvement in heart failure.

There is a substantial amount of data supporting the benefit of Entresto in the clinical management of outpatients with heart failure without using the PIONEER-HF trial results as a pretense to initiate therapy when patients are hospitalized. One might suggest that if Novartis is concerned about introducing the drug in the clinical management of heart failure, the company might consider the possibility of decreasing its price.

Dr. Goldstein is professor of medicine at Wayne State University and the division head emeritus of cardiovascular medicine at Henry Ford Hospital, both in Detroit.

The recently published PIONEER-HF study attempts to move sacubitril/valsartan (Entresto) therapy to the inpatient environment to improve patient and physician acceptance of this therapy for patients with heart failure (N Engl J Med. 2019 Feb 7;380;539-48).

When given to outpatients in the PARADIGM-HF trial, the combination was superior to enalapril for reducing the risks of death and hospitalization for heart failure (N Engl J Med 2014;371:993-1004.) Specifically, sacubitril/valsartan decreased mortality by 15% and hospitalization by 21% as an outpatient therapy for patients with systolic heart failure. Nevertheless, there has not been widespread adoption of this approach. It is well known that physicians can be slow to adopt new therapies, but one overriding factor may be the cost of the drug compared to enalapril, one of the first drugs shown to be effective in heart failure therapy (Entresto costs more than $4,000 per year; enalapril costs about $120 per year).

The investigators in the PIONEER-HF study compared Entresto to enalapril over a 2-month period in patients hospitalized with systolic heart failure. To accelerate the trial, the investigators used the proportional change in patients’ N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels as the primary endpoint rather than the traditional outcome of morbidity and mortality. In the short term, no significant clinical benefits were observed, but there was a significant decrease in NT-proBNP of about 30% (P less than .001).

The investigators suggested that this finding extended the previous benefit observed with Entresto during outpatient initiation and could be used as a rationale for initiating Entresto therapy in the hospital. This earlier application of the therapy could make the drug more widely acceptable.

Considerable investigation in BNP measurement has occurred over the last few years, and although it is clear that BNP is elevated in heart failure patients, there is no evidence to confirm that the decrease in BNP is associated with improved outcome. BNP will fall with decrease in ventricular volume, which may have significant physiologic mechanisms but ventricular volume could decrease with fall in blood pressure that may have occurred in this population since hypotension tended to be more frequent with Entresto than with enalapril. The traditional measure of heart failure benefit with beta-blockers, ACE inhibitors, and aldosterone antagonists in the inpatient and early postdischarge period has depended on clinical outcomes.

Regardless of the physiologic explanation of this fall in BNP, we must pause in our assumptions when a surrogate measure is used to assess clinical benefit as inpatient therapy. The Food and Drug Administration has long given up using surrogate measures as proof of efficacy, and rightly so. Clinical medicine is replete with dubious drug benefits based on surrogate measures. Let’s not forget that only a few years ago suppression of premature ventricular contractions was considered to be a measure of the pharmacologic prevention of sudden death. We have come a long way from that and other clinical missteps to use BNP, an uncertain marker at best of clinical improvement, as a surrogate for the improvement in heart failure.

There is a substantial amount of data supporting the benefit of Entresto in the clinical management of outpatients with heart failure without using the PIONEER-HF trial results as a pretense to initiate therapy when patients are hospitalized. One might suggest that if Novartis is concerned about introducing the drug in the clinical management of heart failure, the company might consider the possibility of decreasing its price.

Dr. Goldstein is professor of medicine at Wayne State University and the division head emeritus of cardiovascular medicine at Henry Ford Hospital, both in Detroit.

The recently published PIONEER-HF study attempts to move sacubitril/valsartan (Entresto) therapy to the inpatient environment to improve patient and physician acceptance of this therapy for patients with heart failure (N Engl J Med. 2019 Feb 7;380;539-48).

When given to outpatients in the PARADIGM-HF trial, the combination was superior to enalapril for reducing the risks of death and hospitalization for heart failure (N Engl J Med 2014;371:993-1004.) Specifically, sacubitril/valsartan decreased mortality by 15% and hospitalization by 21% as an outpatient therapy for patients with systolic heart failure. Nevertheless, there has not been widespread adoption of this approach. It is well known that physicians can be slow to adopt new therapies, but one overriding factor may be the cost of the drug compared to enalapril, one of the first drugs shown to be effective in heart failure therapy (Entresto costs more than $4,000 per year; enalapril costs about $120 per year).

The investigators in the PIONEER-HF study compared Entresto to enalapril over a 2-month period in patients hospitalized with systolic heart failure. To accelerate the trial, the investigators used the proportional change in patients’ N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels as the primary endpoint rather than the traditional outcome of morbidity and mortality. In the short term, no significant clinical benefits were observed, but there was a significant decrease in NT-proBNP of about 30% (P less than .001).

The investigators suggested that this finding extended the previous benefit observed with Entresto during outpatient initiation and could be used as a rationale for initiating Entresto therapy in the hospital. This earlier application of the therapy could make the drug more widely acceptable.

Considerable investigation in BNP measurement has occurred over the last few years, and although it is clear that BNP is elevated in heart failure patients, there is no evidence to confirm that the decrease in BNP is associated with improved outcome. BNP will fall with decrease in ventricular volume, which may have significant physiologic mechanisms but ventricular volume could decrease with fall in blood pressure that may have occurred in this population since hypotension tended to be more frequent with Entresto than with enalapril. The traditional measure of heart failure benefit with beta-blockers, ACE inhibitors, and aldosterone antagonists in the inpatient and early postdischarge period has depended on clinical outcomes.

Regardless of the physiologic explanation of this fall in BNP, we must pause in our assumptions when a surrogate measure is used to assess clinical benefit as inpatient therapy. The Food and Drug Administration has long given up using surrogate measures as proof of efficacy, and rightly so. Clinical medicine is replete with dubious drug benefits based on surrogate measures. Let’s not forget that only a few years ago suppression of premature ventricular contractions was considered to be a measure of the pharmacologic prevention of sudden death. We have come a long way from that and other clinical missteps to use BNP, an uncertain marker at best of clinical improvement, as a surrogate for the improvement in heart failure.

There is a substantial amount of data supporting the benefit of Entresto in the clinical management of outpatients with heart failure without using the PIONEER-HF trial results as a pretense to initiate therapy when patients are hospitalized. One might suggest that if Novartis is concerned about introducing the drug in the clinical management of heart failure, the company might consider the possibility of decreasing its price.

Dr. Goldstein is professor of medicine at Wayne State University and the division head emeritus of cardiovascular medicine at Henry Ford Hospital, both in Detroit.

LAKE BUENA VISTA, FLA. – Patient-reported outcomes (PROs) have the potential to transform patient care in rheumatology, according to Jeffrey Curtis, MD.

Courtesy UAB Photo

“You probably already use a lot of PROs in your data; even if you measure nothing via a questionnaire, you are still collecting it in a qualitative way – you just might not call it that,” he told attendees at the annual meeting of the Florida Society of Rheumatology.

The key to making the most of PROs is efficient collection of relevant, interpretable, actionable data for improving patient care and outcomes, said Dr. Curtis, professor of medicine in the division of clinical immunology and rheumatology at the University of Alabama at Birmingham.
 

PROs: The “what” and “why”

A wide variety of tools are available to capture PROs during daily practice, Dr. Curtis said. Active data capture tools include rheumatology- or domain-specific measures such as the Bath Ankylosing Spondylitis Disease Activity and Functional Indices (BASDAI and BASFI), the Routine Assessment of Patient Index Data 3 (RAPID3), the original and Multidimensional Health Assessment Questionnaires (HAQ and MDHAQ), as well as disease-agnostic measures like the 36-item Short Form Survey (SF-36), EuroQol-5D (EQ-5D), the Work Productivity and Activity Impairment Questionnaire (WPAI), and the National Institutes of Health Patient-Reported Outcomes Measurement Information System (PROMIS) instruments, he explained, adding that passive data also can be derived from various sources, including social media platforms, activity trackers, and reports regarding balance and falls, sleep quality and duration, heart rate and rhythm, and galvanic skin resistance.

Many of these measures represent things patients can track at home between office visits, he said.

However, such measures represent “what we could have” in terms of patient data, whereas “what we do have” falls far short of that, he noted, citing a study in which he and his colleagues found that the use of quantitative measurement for U.S. rheumatoid arthritis (RA) patients is increasing over time, but remains low with only 58% of 439 rheumatologists who responded to an email survey reporting use of such measures (J Rheumatol. 2018;45[1]:40-4).

Those using the measures were more likely to be in group practice and to prescribe tumor necrosis factor inhibitors, and the tools they reported using most often were the HAQ (35.5%) and RAPID3 (27.1%).

Reasons given for not using quantitative measurement included time constraints and electronic availability.

Of note, simulated case scenarios included in the study demonstrated that providing more quantitative information increased the likelihood that a patient would change to a different disease-modifying antirheumatic drug or biologic.

Almost anything clinically relevant can be quantified, but it’s really hard to improve and address problems you’re not measuring, Dr. Curtis said.

“I would contend that PROs are an important part of holistic rheumatology care, and they absolutely impact real and perceived treatment responses,” he added.

In fact, in a study presented at the 2018 European League Against Rheumatism Congress, he and his colleagues found that PROMIS scores with respect to pain interference, sleep disturbance, and fatigue tracked closely with RA patients’ view of their health status and with Clinical Disease Activity Index (CDAI) scores.
 

PROs: The “how”

“Is it merely enough to collect patient data? Is that going to solve the problem? Well, probably not – it really needs to be actionable,” he said. “Outcomes don’t get better by themselves; you really need to be collecting data that you, personally, will find valuable for your patients, and ideally it needs to mean something to patients.”

Many of these suggestions are potentially actionable, he noted.

“You can download these forms on paper; this is already connected or connectable to some people’s electronic health record,” he said. “At a minimum, talk to your EHR vendor about whether this might be available, and if not, why not.”

Choose in-office tools that are quick and simple to use, he advised, noting that he finds 6-8 minutes ideal for patient completion of questionnaires and other measures.

“It’s quite reasonable to write a PRO order,” he said. “The notion would be that you decide what specific PROs you want Mrs. Smith to give you, how often you’d like for her to tell you about those things (what you want from her might be different from the next patient), and she can give you that data from a smartphone or maybe something that she wears, and only the data that you asked for comes back.”

Successful collection of such data requires patient engagement in the process, he said, noting that the Center for Education and Research and Therapeutics of Musculoskeletal Disorders at UAB was recently awarded a grant to help develop an arthritis research registry called Arthritis Power, through which patients can provide data via smartphones, track their own health outcomes, participate in studies and surveys, access educational tools, and receive reminders and feedback.

“One of the things that’s quite important to help engage patients is to encourage them. This isn’t one-way data transfer,” he said.

Keeping them engaged requires “contributive science messaging.” That is, telling them they are “part of something bigger [and that they are] helping answer research questions that patients care about.”

It also helps to “bring back value to them” by explaining that you can help them make their data useful for improving their health and that you can derive insights for or with them based on their data.

“You can ‘game-ify’ it and make it fun,” he said, adding that leveraging the social connections associated with some tools can also help.

However, the promise of better access to needed resources, physicians, and the health care system is perhaps the most compelling point for patient engagement, he said.

“[You can say] to your patient, ‘Mrs. Smith, I’d really like to have your Fitbit or Apple Watch data, and I’d like you to tell me how you’re doing, on your smartphone, once or twice a month – it will take about 10 minutes – because I, as your doctor, think I can take better care of you,’ ” he said. “If that’s the ask, I think that might be the most compelling reason for patients to say yes.”

Of note, a number of patient measures are now compensable, Dr. Curtis said, mentioning depression screening using a PROMIS instrument as one example.

Additionally, two American College of Rheumatology work groups are revising the ACR recommendations on functional status measures and will soon generate an “ACR-approved list” of measures, he said.

He stressed, however, that it in addition to understanding the value of specific tools, it is important to know their limitations.

In the PREDICT study, he and his colleagues demonstrated that patient-reported RAPID3 data, when compared with investigator-based CDAI data for assessing RA patients’ response to certolizumab at 12 weeks and predicting response at 52 weeks, resulted in 11.9% fewer patients being classified as responders (64.7% vs. 76.4%), but the actual response rates at week 52 were similar, with 31.5% and 32.3% of patients in the groups, respectively, achieving a low level of disease activity (Arthritis Rheumatol. 2015;67[12]:3104-12).

The concern regarding the finding is that an insurance company may refuse to continue paying for a drug because of the perceived lack of response and thereby unnecessarily force a switch to an alternate drug based on faulty data, he explained.

“That would be the real-world analog of what this trial evaluated,” he said, adding that this has important implications for treat-to-target, pay-for-performance, and merit-based incentive payment systems. “My point is that we need to know the limitations of our tools ... and it’s to not let insurance [companies] write rules for us ... based upon certain tools that have limitations.”

Dr. Curtis reported funding from the National Institute of Arthritis and Musculoskeletal and Skin Diseases and the Patient-Centered Outcomes Research Institute. He has also consulted for or received research grants from Amgen, AbbVie, Bristol-Myers Squibb, CORRONA, Lilly, Janssen, Myriad, Novartis, Roche, Pfizer, and Sanofi/Regeneron.

[email protected]

LAKE BUENA VISTA, FLA. – Patient-reported outcomes (PROs) have the potential to transform patient care in rheumatology, according to Jeffrey Curtis, MD.

Courtesy UAB Photo

“You probably already use a lot of PROs in your data; even if you measure nothing via a questionnaire, you are still collecting it in a qualitative way – you just might not call it that,” he told attendees at the annual meeting of the Florida Society of Rheumatology.

The key to making the most of PROs is efficient collection of relevant, interpretable, actionable data for improving patient care and outcomes, said Dr. Curtis, professor of medicine in the division of clinical immunology and rheumatology at the University of Alabama at Birmingham.
 

PROs: The “what” and “why”

A wide variety of tools are available to capture PROs during daily practice, Dr. Curtis said. Active data capture tools include rheumatology- or domain-specific measures such as the Bath Ankylosing Spondylitis Disease Activity and Functional Indices (BASDAI and BASFI), the Routine Assessment of Patient Index Data 3 (RAPID3), the original and Multidimensional Health Assessment Questionnaires (HAQ and MDHAQ), as well as disease-agnostic measures like the 36-item Short Form Survey (SF-36), EuroQol-5D (EQ-5D), the Work Productivity and Activity Impairment Questionnaire (WPAI), and the National Institutes of Health Patient-Reported Outcomes Measurement Information System (PROMIS) instruments, he explained, adding that passive data also can be derived from various sources, including social media platforms, activity trackers, and reports regarding balance and falls, sleep quality and duration, heart rate and rhythm, and galvanic skin resistance.

Many of these measures represent things patients can track at home between office visits, he said.

However, such measures represent “what we could have” in terms of patient data, whereas “what we do have” falls far short of that, he noted, citing a study in which he and his colleagues found that the use of quantitative measurement for U.S. rheumatoid arthritis (RA) patients is increasing over time, but remains low with only 58% of 439 rheumatologists who responded to an email survey reporting use of such measures (J Rheumatol. 2018;45[1]:40-4).

Those using the measures were more likely to be in group practice and to prescribe tumor necrosis factor inhibitors, and the tools they reported using most often were the HAQ (35.5%) and RAPID3 (27.1%).

Reasons given for not using quantitative measurement included time constraints and electronic availability.

Of note, simulated case scenarios included in the study demonstrated that providing more quantitative information increased the likelihood that a patient would change to a different disease-modifying antirheumatic drug or biologic.

Almost anything clinically relevant can be quantified, but it’s really hard to improve and address problems you’re not measuring, Dr. Curtis said.

“I would contend that PROs are an important part of holistic rheumatology care, and they absolutely impact real and perceived treatment responses,” he added.

In fact, in a study presented at the 2018 European League Against Rheumatism Congress, he and his colleagues found that PROMIS scores with respect to pain interference, sleep disturbance, and fatigue tracked closely with RA patients’ view of their health status and with Clinical Disease Activity Index (CDAI) scores.
 

PROs: The “how”

“Is it merely enough to collect patient data? Is that going to solve the problem? Well, probably not – it really needs to be actionable,” he said. “Outcomes don’t get better by themselves; you really need to be collecting data that you, personally, will find valuable for your patients, and ideally it needs to mean something to patients.”

Many of these suggestions are potentially actionable, he noted.

“You can download these forms on paper; this is already connected or connectable to some people’s electronic health record,” he said. “At a minimum, talk to your EHR vendor about whether this might be available, and if not, why not.”

Choose in-office tools that are quick and simple to use, he advised, noting that he finds 6-8 minutes ideal for patient completion of questionnaires and other measures.

“It’s quite reasonable to write a PRO order,” he said. “The notion would be that you decide what specific PROs you want Mrs. Smith to give you, how often you’d like for her to tell you about those things (what you want from her might be different from the next patient), and she can give you that data from a smartphone or maybe something that she wears, and only the data that you asked for comes back.”

Successful collection of such data requires patient engagement in the process, he said, noting that the Center for Education and Research and Therapeutics of Musculoskeletal Disorders at UAB was recently awarded a grant to help develop an arthritis research registry called Arthritis Power, through which patients can provide data via smartphones, track their own health outcomes, participate in studies and surveys, access educational tools, and receive reminders and feedback.

“One of the things that’s quite important to help engage patients is to encourage them. This isn’t one-way data transfer,” he said.

Keeping them engaged requires “contributive science messaging.” That is, telling them they are “part of something bigger [and that they are] helping answer research questions that patients care about.”

It also helps to “bring back value to them” by explaining that you can help them make their data useful for improving their health and that you can derive insights for or with them based on their data.

“You can ‘game-ify’ it and make it fun,” he said, adding that leveraging the social connections associated with some tools can also help.

However, the promise of better access to needed resources, physicians, and the health care system is perhaps the most compelling point for patient engagement, he said.

“[You can say] to your patient, ‘Mrs. Smith, I’d really like to have your Fitbit or Apple Watch data, and I’d like you to tell me how you’re doing, on your smartphone, once or twice a month – it will take about 10 minutes – because I, as your doctor, think I can take better care of you,’ ” he said. “If that’s the ask, I think that might be the most compelling reason for patients to say yes.”

Of note, a number of patient measures are now compensable, Dr. Curtis said, mentioning depression screening using a PROMIS instrument as one example.

Additionally, two American College of Rheumatology work groups are revising the ACR recommendations on functional status measures and will soon generate an “ACR-approved list” of measures, he said.

He stressed, however, that it in addition to understanding the value of specific tools, it is important to know their limitations.

In the PREDICT study, he and his colleagues demonstrated that patient-reported RAPID3 data, when compared with investigator-based CDAI data for assessing RA patients’ response to certolizumab at 12 weeks and predicting response at 52 weeks, resulted in 11.9% fewer patients being classified as responders (64.7% vs. 76.4%), but the actual response rates at week 52 were similar, with 31.5% and 32.3% of patients in the groups, respectively, achieving a low level of disease activity (Arthritis Rheumatol. 2015;67[12]:3104-12).

The concern regarding the finding is that an insurance company may refuse to continue paying for a drug because of the perceived lack of response and thereby unnecessarily force a switch to an alternate drug based on faulty data, he explained.

“That would be the real-world analog of what this trial evaluated,” he said, adding that this has important implications for treat-to-target, pay-for-performance, and merit-based incentive payment systems. “My point is that we need to know the limitations of our tools ... and it’s to not let insurance [companies] write rules for us ... based upon certain tools that have limitations.”

Dr. Curtis reported funding from the National Institute of Arthritis and Musculoskeletal and Skin Diseases and the Patient-Centered Outcomes Research Institute. He has also consulted for or received research grants from Amgen, AbbVie, Bristol-Myers Squibb, CORRONA, Lilly, Janssen, Myriad, Novartis, Roche, Pfizer, and Sanofi/Regeneron.

[email protected]

LAKE BUENA VISTA, FLA. – Patient-reported outcomes (PROs) have the potential to transform patient care in rheumatology, according to Jeffrey Curtis, MD.

Courtesy UAB Photo

“You probably already use a lot of PROs in your data; even if you measure nothing via a questionnaire, you are still collecting it in a qualitative way – you just might not call it that,” he told attendees at the annual meeting of the Florida Society of Rheumatology.

The key to making the most of PROs is efficient collection of relevant, interpretable, actionable data for improving patient care and outcomes, said Dr. Curtis, professor of medicine in the division of clinical immunology and rheumatology at the University of Alabama at Birmingham.
 

PROs: The “what” and “why”

A wide variety of tools are available to capture PROs during daily practice, Dr. Curtis said. Active data capture tools include rheumatology- or domain-specific measures such as the Bath Ankylosing Spondylitis Disease Activity and Functional Indices (BASDAI and BASFI), the Routine Assessment of Patient Index Data 3 (RAPID3), the original and Multidimensional Health Assessment Questionnaires (HAQ and MDHAQ), as well as disease-agnostic measures like the 36-item Short Form Survey (SF-36), EuroQol-5D (EQ-5D), the Work Productivity and Activity Impairment Questionnaire (WPAI), and the National Institutes of Health Patient-Reported Outcomes Measurement Information System (PROMIS) instruments, he explained, adding that passive data also can be derived from various sources, including social media platforms, activity trackers, and reports regarding balance and falls, sleep quality and duration, heart rate and rhythm, and galvanic skin resistance.

Many of these measures represent things patients can track at home between office visits, he said.

However, such measures represent “what we could have” in terms of patient data, whereas “what we do have” falls far short of that, he noted, citing a study in which he and his colleagues found that the use of quantitative measurement for U.S. rheumatoid arthritis (RA) patients is increasing over time, but remains low with only 58% of 439 rheumatologists who responded to an email survey reporting use of such measures (J Rheumatol. 2018;45[1]:40-4).

Those using the measures were more likely to be in group practice and to prescribe tumor necrosis factor inhibitors, and the tools they reported using most often were the HAQ (35.5%) and RAPID3 (27.1%).

Reasons given for not using quantitative measurement included time constraints and electronic availability.

Of note, simulated case scenarios included in the study demonstrated that providing more quantitative information increased the likelihood that a patient would change to a different disease-modifying antirheumatic drug or biologic.

Almost anything clinically relevant can be quantified, but it’s really hard to improve and address problems you’re not measuring, Dr. Curtis said.

“I would contend that PROs are an important part of holistic rheumatology care, and they absolutely impact real and perceived treatment responses,” he added.

In fact, in a study presented at the 2018 European League Against Rheumatism Congress, he and his colleagues found that PROMIS scores with respect to pain interference, sleep disturbance, and fatigue tracked closely with RA patients’ view of their health status and with Clinical Disease Activity Index (CDAI) scores.
 

PROs: The “how”

“Is it merely enough to collect patient data? Is that going to solve the problem? Well, probably not – it really needs to be actionable,” he said. “Outcomes don’t get better by themselves; you really need to be collecting data that you, personally, will find valuable for your patients, and ideally it needs to mean something to patients.”

Many of these suggestions are potentially actionable, he noted.

“You can download these forms on paper; this is already connected or connectable to some people’s electronic health record,” he said. “At a minimum, talk to your EHR vendor about whether this might be available, and if not, why not.”

Choose in-office tools that are quick and simple to use, he advised, noting that he finds 6-8 minutes ideal for patient completion of questionnaires and other measures.

“It’s quite reasonable to write a PRO order,” he said. “The notion would be that you decide what specific PROs you want Mrs. Smith to give you, how often you’d like for her to tell you about those things (what you want from her might be different from the next patient), and she can give you that data from a smartphone or maybe something that she wears, and only the data that you asked for comes back.”

Successful collection of such data requires patient engagement in the process, he said, noting that the Center for Education and Research and Therapeutics of Musculoskeletal Disorders at UAB was recently awarded a grant to help develop an arthritis research registry called Arthritis Power, through which patients can provide data via smartphones, track their own health outcomes, participate in studies and surveys, access educational tools, and receive reminders and feedback.

“One of the things that’s quite important to help engage patients is to encourage them. This isn’t one-way data transfer,” he said.

Keeping them engaged requires “contributive science messaging.” That is, telling them they are “part of something bigger [and that they are] helping answer research questions that patients care about.”

It also helps to “bring back value to them” by explaining that you can help them make their data useful for improving their health and that you can derive insights for or with them based on their data.

“You can ‘game-ify’ it and make it fun,” he said, adding that leveraging the social connections associated with some tools can also help.

However, the promise of better access to needed resources, physicians, and the health care system is perhaps the most compelling point for patient engagement, he said.

“[You can say] to your patient, ‘Mrs. Smith, I’d really like to have your Fitbit or Apple Watch data, and I’d like you to tell me how you’re doing, on your smartphone, once or twice a month – it will take about 10 minutes – because I, as your doctor, think I can take better care of you,’ ” he said. “If that’s the ask, I think that might be the most compelling reason for patients to say yes.”

Of note, a number of patient measures are now compensable, Dr. Curtis said, mentioning depression screening using a PROMIS instrument as one example.

Additionally, two American College of Rheumatology work groups are revising the ACR recommendations on functional status measures and will soon generate an “ACR-approved list” of measures, he said.

He stressed, however, that it in addition to understanding the value of specific tools, it is important to know their limitations.

In the PREDICT study, he and his colleagues demonstrated that patient-reported RAPID3 data, when compared with investigator-based CDAI data for assessing RA patients’ response to certolizumab at 12 weeks and predicting response at 52 weeks, resulted in 11.9% fewer patients being classified as responders (64.7% vs. 76.4%), but the actual response rates at week 52 were similar, with 31.5% and 32.3% of patients in the groups, respectively, achieving a low level of disease activity (Arthritis Rheumatol. 2015;67[12]:3104-12).

The concern regarding the finding is that an insurance company may refuse to continue paying for a drug because of the perceived lack of response and thereby unnecessarily force a switch to an alternate drug based on faulty data, he explained.

“That would be the real-world analog of what this trial evaluated,” he said, adding that this has important implications for treat-to-target, pay-for-performance, and merit-based incentive payment systems. “My point is that we need to know the limitations of our tools ... and it’s to not let insurance [companies] write rules for us ... based upon certain tools that have limitations.”

Dr. Curtis reported funding from the National Institute of Arthritis and Musculoskeletal and Skin Diseases and the Patient-Centered Outcomes Research Institute. He has also consulted for or received research grants from Amgen, AbbVie, Bristol-Myers Squibb, CORRONA, Lilly, Janssen, Myriad, Novartis, Roche, Pfizer, and Sanofi/Regeneron.

[email protected]

LAKE BUENA VISTA, FLA. – Rheumatologists comprise a “tiny sliver” of the U.S. physician pie chart, but their voices are nevertheless being heard and making a difference regarding policies that affect the specialty, according to Angus B. Worthing, MD.

However, given the myriad policy issues on the table, more and louder voices are needed, he said at the annual meeting of the Florida Society of Rheumatology, where, as chair of the American College of Rheumatology’s Government Affairs Committee, he provided an “advocacy update” on the committee’s activities.

Recent ACR “wins” as outlined by Dr. Worthing include:

• The postponement and modification of proposed cuts to Evaluation & Management (E/M) current procedural terminology (CPT) codes.

“Last summer, Medicare proposed that instead of having low-complexity to high-complexity E/M codes, they would condense all of the doctor visits into one code, which would obviously enhance the reimbursement for low complexity, but ding and penalize the reimbursement for high-complexity visits like ours,” said Dr. Worthing, who also is a partner in a private rheumatology practice in the Washington area.

An ACR press release on the proposal led to pivotal coverage of the issue in the New York Times. “It looks like not only did they postpone those cuts, they modified [the proposal], and we’ll find out any day now in the proposal for the next year’s physician fee schedule whether they’ll scrap it entirely and instead try to plus-up E/M code reimbursement for complex patients.”

• The elimination of proposed Merit-based Incentive Payment System (MIPS) adjustments on Medicare drug reimbursement.

A cut to fee-for-service reimbursement for drug costs as a MIPS penalty could have quickly bankrupted rheumatology practices, Dr. Worthing said.

“[The success] was largely out of rheumatologists and others saying that this could quickly stop access to these treatments, because we wouldn’t be able to provide them,” he noted.

• The dampening of proposed musculoskeletal ultrasound reimbursement cuts in Medicare.

Ultrasound is a safe, effective, dynamic, and relatively low-cost diagnostic tool, but Medicare has been considering “absurd” cuts to reimbursement, Dr. Worthing said.

“We’ve been able to have good conversations with Medicare ... to bring that argument to Medicare, and we’ll find out – again, when the physician fee schedule comes out – whether they’ll continue the plan to cut diagnostic ultrasound reimbursement or whether they’ll stop cutting it.”

• The inclusion of more favorable Medicare Advantage regulations for step therapy “grandfathering.”

Medicare Advantage plans were given the chance last summer to use step therapy in Part B medicines for the first time.

“The ACR quickly told the executive branch and officials at [the Department of Health & Human Services (HHS)], that this would not be good for our patients getting medications,” Dr. Worthing said.

Going forward with that plan, and looking back just 108 days to allow people to stay on their medications – as was proposed – wouldn’t work, as some drugs are dosed every 4-6 months, or every year or every 2 years, he said.

“The look of astonishment on the [HHS] deputy secretary’s face when I told him that there was a drug in the U.S. that you give every 2 years was helpful for me to know that these people really need to hear from us before they issue these kinds of regulations,” he said.

The administration listened to the rheumatologists and is going to look back 365 days to keep people on their drugs, he said.

The ACR took the lead on these recent successes, but was also involved in a number of other wins achieved through multisociety efforts, he said.

Examples include securing a $2 billion increase in National Institutes of Health funding, eliminating “gag clauses” that prevent pharmacists from informing patients when it’s cheaper to just buy a drug rather than using their insurance; getting rid of annual caps on physical and other rehabilitation therapy for patients meeting their targets; repealing (before it could take effect) of the Independent Payment Advisory Board established by the Affordable Care Act; and – at least for now – continuing Deferred Action for Childhood Arrivals (DACA) protections that could allow recipients to stay in the country, study, and become doctors, and potentially provide care for up to 100,000 Americans, according to an estimate by the American Medical Association.

Dr. Worthing also noted that the ACR has an Insurance Subcommittee that has been instrumental in many of these and other policy wins, and he encouraged rheumatologists to contact the committee at [email protected] to report any sort of “canary-in-the-coal-mine” issues, such as refusal of coverage for a new step therapy, service, or item in your clinic that seems “absurd; doesn’t have merit.”

Send a copy of the policy behind the denial (with private health information redacted), or complete a Health Plan Complaint Form, which can be accessed at the website, he advised.

Rheumatologists can make their voices heard in other ways, he said. The ACR has a number of ongoing advocacy efforts addressing things like drug-pricing models, biosimilar agent interchangeability pathways, step therapy reform, workforce issues, and the need for higher dual X-ray absorptiometry (DXA) reimbursement.

The ACR’s Legislative Action Center offers prewritten letters on a number of timely topics, as well as information about legislators and legislation. An app is available that provides alerts about important legislation.

A timely example is DXA-related, bipartisan, bicameral legislation currently on the table that would more than double reimbursement and “improve access to this critical screening tool,” he said.

“Right now is an excellent time to tell your legislators – and there’s a prewritten letter at the [site] – that this is an important topic,” he said. “Right now, as we get into bills that might come out, spending bills or health-related bills coming up to 2020, it would be wonderful to get a lot of support behind this so that it might be added into some kind of package.”

A similar prewritten letter regarding an active bill that addresses paying off student loans for pediatricians going into subspecialties like pediatric rheumatology also is on the table and could help address workforce shortages, he noted.

“The First Amendment protects your right to petition the government for redress of grievances. I don’t have to tell you this is an era of huge tumult ... protecting [democracy and institutions], protecting your organizations, raising your voice is really important right now,” he said, referencing his new Twitter hashtag that encourages doing one #ThingADay. “You could think of advocacy as an extension of the Hippocratic oath to do no harm on a government and social level.”

Quoting Margaret Mead, Dr. Worthing reminded rheumatologists that their voices matter despite (and in fact, because of) their small numbers: “Never doubt that a small group of thoughtful, committed citizens can change the world; indeed, it’s the only thing that ever has.”

Dr. Worthing reported having no disclosures.

[email protected]

LAKE BUENA VISTA, FLA. – Rheumatologists comprise a “tiny sliver” of the U.S. physician pie chart, but their voices are nevertheless being heard and making a difference regarding policies that affect the specialty, according to Angus B. Worthing, MD.

However, given the myriad policy issues on the table, more and louder voices are needed, he said at the annual meeting of the Florida Society of Rheumatology, where, as chair of the American College of Rheumatology’s Government Affairs Committee, he provided an “advocacy update” on the committee’s activities.

Recent ACR “wins” as outlined by Dr. Worthing include:

• The postponement and modification of proposed cuts to Evaluation & Management (E/M) current procedural terminology (CPT) codes.

“Last summer, Medicare proposed that instead of having low-complexity to high-complexity E/M codes, they would condense all of the doctor visits into one code, which would obviously enhance the reimbursement for low complexity, but ding and penalize the reimbursement for high-complexity visits like ours,” said Dr. Worthing, who also is a partner in a private rheumatology practice in the Washington area.

An ACR press release on the proposal led to pivotal coverage of the issue in the New York Times. “It looks like not only did they postpone those cuts, they modified [the proposal], and we’ll find out any day now in the proposal for the next year’s physician fee schedule whether they’ll scrap it entirely and instead try to plus-up E/M code reimbursement for complex patients.”

• The elimination of proposed Merit-based Incentive Payment System (MIPS) adjustments on Medicare drug reimbursement.

A cut to fee-for-service reimbursement for drug costs as a MIPS penalty could have quickly bankrupted rheumatology practices, Dr. Worthing said.

“[The success] was largely out of rheumatologists and others saying that this could quickly stop access to these treatments, because we wouldn’t be able to provide them,” he noted.

• The dampening of proposed musculoskeletal ultrasound reimbursement cuts in Medicare.

Ultrasound is a safe, effective, dynamic, and relatively low-cost diagnostic tool, but Medicare has been considering “absurd” cuts to reimbursement, Dr. Worthing said.

“We’ve been able to have good conversations with Medicare ... to bring that argument to Medicare, and we’ll find out – again, when the physician fee schedule comes out – whether they’ll continue the plan to cut diagnostic ultrasound reimbursement or whether they’ll stop cutting it.”

• The inclusion of more favorable Medicare Advantage regulations for step therapy “grandfathering.”

Medicare Advantage plans were given the chance last summer to use step therapy in Part B medicines for the first time.

“The ACR quickly told the executive branch and officials at [the Department of Health & Human Services (HHS)], that this would not be good for our patients getting medications,” Dr. Worthing said.

Going forward with that plan, and looking back just 108 days to allow people to stay on their medications – as was proposed – wouldn’t work, as some drugs are dosed every 4-6 months, or every year or every 2 years, he said.

“The look of astonishment on the [HHS] deputy secretary’s face when I told him that there was a drug in the U.S. that you give every 2 years was helpful for me to know that these people really need to hear from us before they issue these kinds of regulations,” he said.

The administration listened to the rheumatologists and is going to look back 365 days to keep people on their drugs, he said.

The ACR took the lead on these recent successes, but was also involved in a number of other wins achieved through multisociety efforts, he said.

Examples include securing a $2 billion increase in National Institutes of Health funding, eliminating “gag clauses” that prevent pharmacists from informing patients when it’s cheaper to just buy a drug rather than using their insurance; getting rid of annual caps on physical and other rehabilitation therapy for patients meeting their targets; repealing (before it could take effect) of the Independent Payment Advisory Board established by the Affordable Care Act; and – at least for now – continuing Deferred Action for Childhood Arrivals (DACA) protections that could allow recipients to stay in the country, study, and become doctors, and potentially provide care for up to 100,000 Americans, according to an estimate by the American Medical Association.

Dr. Worthing also noted that the ACR has an Insurance Subcommittee that has been instrumental in many of these and other policy wins, and he encouraged rheumatologists to contact the committee at [email protected] to report any sort of “canary-in-the-coal-mine” issues, such as refusal of coverage for a new step therapy, service, or item in your clinic that seems “absurd; doesn’t have merit.”

Send a copy of the policy behind the denial (with private health information redacted), or complete a Health Plan Complaint Form, which can be accessed at the website, he advised.

Rheumatologists can make their voices heard in other ways, he said. The ACR has a number of ongoing advocacy efforts addressing things like drug-pricing models, biosimilar agent interchangeability pathways, step therapy reform, workforce issues, and the need for higher dual X-ray absorptiometry (DXA) reimbursement.

The ACR’s Legislative Action Center offers prewritten letters on a number of timely topics, as well as information about legislators and legislation. An app is available that provides alerts about important legislation.

A timely example is DXA-related, bipartisan, bicameral legislation currently on the table that would more than double reimbursement and “improve access to this critical screening tool,” he said.

“Right now is an excellent time to tell your legislators – and there’s a prewritten letter at the [site] – that this is an important topic,” he said. “Right now, as we get into bills that might come out, spending bills or health-related bills coming up to 2020, it would be wonderful to get a lot of support behind this so that it might be added into some kind of package.”

A similar prewritten letter regarding an active bill that addresses paying off student loans for pediatricians going into subspecialties like pediatric rheumatology also is on the table and could help address workforce shortages, he noted.

“The First Amendment protects your right to petition the government for redress of grievances. I don’t have to tell you this is an era of huge tumult ... protecting [democracy and institutions], protecting your organizations, raising your voice is really important right now,” he said, referencing his new Twitter hashtag that encourages doing one #ThingADay. “You could think of advocacy as an extension of the Hippocratic oath to do no harm on a government and social level.”

Quoting Margaret Mead, Dr. Worthing reminded rheumatologists that their voices matter despite (and in fact, because of) their small numbers: “Never doubt that a small group of thoughtful, committed citizens can change the world; indeed, it’s the only thing that ever has.”

Dr. Worthing reported having no disclosures.

[email protected]

LAKE BUENA VISTA, FLA. – Rheumatologists comprise a “tiny sliver” of the U.S. physician pie chart, but their voices are nevertheless being heard and making a difference regarding policies that affect the specialty, according to Angus B. Worthing, MD.

However, given the myriad policy issues on the table, more and louder voices are needed, he said at the annual meeting of the Florida Society of Rheumatology, where, as chair of the American College of Rheumatology’s Government Affairs Committee, he provided an “advocacy update” on the committee’s activities.

Recent ACR “wins” as outlined by Dr. Worthing include:

• The postponement and modification of proposed cuts to Evaluation & Management (E/M) current procedural terminology (CPT) codes.

“Last summer, Medicare proposed that instead of having low-complexity to high-complexity E/M codes, they would condense all of the doctor visits into one code, which would obviously enhance the reimbursement for low complexity, but ding and penalize the reimbursement for high-complexity visits like ours,” said Dr. Worthing, who also is a partner in a private rheumatology practice in the Washington area.

An ACR press release on the proposal led to pivotal coverage of the issue in the New York Times. “It looks like not only did they postpone those cuts, they modified [the proposal], and we’ll find out any day now in the proposal for the next year’s physician fee schedule whether they’ll scrap it entirely and instead try to plus-up E/M code reimbursement for complex patients.”

• The elimination of proposed Merit-based Incentive Payment System (MIPS) adjustments on Medicare drug reimbursement.

A cut to fee-for-service reimbursement for drug costs as a MIPS penalty could have quickly bankrupted rheumatology practices, Dr. Worthing said.

“[The success] was largely out of rheumatologists and others saying that this could quickly stop access to these treatments, because we wouldn’t be able to provide them,” he noted.

• The dampening of proposed musculoskeletal ultrasound reimbursement cuts in Medicare.

Ultrasound is a safe, effective, dynamic, and relatively low-cost diagnostic tool, but Medicare has been considering “absurd” cuts to reimbursement, Dr. Worthing said.

“We’ve been able to have good conversations with Medicare ... to bring that argument to Medicare, and we’ll find out – again, when the physician fee schedule comes out – whether they’ll continue the plan to cut diagnostic ultrasound reimbursement or whether they’ll stop cutting it.”

• The inclusion of more favorable Medicare Advantage regulations for step therapy “grandfathering.”

Medicare Advantage plans were given the chance last summer to use step therapy in Part B medicines for the first time.

“The ACR quickly told the executive branch and officials at [the Department of Health & Human Services (HHS)], that this would not be good for our patients getting medications,” Dr. Worthing said.

Going forward with that plan, and looking back just 108 days to allow people to stay on their medications – as was proposed – wouldn’t work, as some drugs are dosed every 4-6 months, or every year or every 2 years, he said.

“The look of astonishment on the [HHS] deputy secretary’s face when I told him that there was a drug in the U.S. that you give every 2 years was helpful for me to know that these people really need to hear from us before they issue these kinds of regulations,” he said.

The administration listened to the rheumatologists and is going to look back 365 days to keep people on their drugs, he said.

The ACR took the lead on these recent successes, but was also involved in a number of other wins achieved through multisociety efforts, he said.

Examples include securing a $2 billion increase in National Institutes of Health funding, eliminating “gag clauses” that prevent pharmacists from informing patients when it’s cheaper to just buy a drug rather than using their insurance; getting rid of annual caps on physical and other rehabilitation therapy for patients meeting their targets; repealing (before it could take effect) of the Independent Payment Advisory Board established by the Affordable Care Act; and – at least for now – continuing Deferred Action for Childhood Arrivals (DACA) protections that could allow recipients to stay in the country, study, and become doctors, and potentially provide care for up to 100,000 Americans, according to an estimate by the American Medical Association.

Dr. Worthing also noted that the ACR has an Insurance Subcommittee that has been instrumental in many of these and other policy wins, and he encouraged rheumatologists to contact the committee at [email protected] to report any sort of “canary-in-the-coal-mine” issues, such as refusal of coverage for a new step therapy, service, or item in your clinic that seems “absurd; doesn’t have merit.”

Send a copy of the policy behind the denial (with private health information redacted), or complete a Health Plan Complaint Form, which can be accessed at the website, he advised.

Rheumatologists can make their voices heard in other ways, he said. The ACR has a number of ongoing advocacy efforts addressing things like drug-pricing models, biosimilar agent interchangeability pathways, step therapy reform, workforce issues, and the need for higher dual X-ray absorptiometry (DXA) reimbursement.

The ACR’s Legislative Action Center offers prewritten letters on a number of timely topics, as well as information about legislators and legislation. An app is available that provides alerts about important legislation.

A timely example is DXA-related, bipartisan, bicameral legislation currently on the table that would more than double reimbursement and “improve access to this critical screening tool,” he said.

“Right now is an excellent time to tell your legislators – and there’s a prewritten letter at the [site] – that this is an important topic,” he said. “Right now, as we get into bills that might come out, spending bills or health-related bills coming up to 2020, it would be wonderful to get a lot of support behind this so that it might be added into some kind of package.”

A similar prewritten letter regarding an active bill that addresses paying off student loans for pediatricians going into subspecialties like pediatric rheumatology also is on the table and could help address workforce shortages, he noted.

“The First Amendment protects your right to petition the government for redress of grievances. I don’t have to tell you this is an era of huge tumult ... protecting [democracy and institutions], protecting your organizations, raising your voice is really important right now,” he said, referencing his new Twitter hashtag that encourages doing one #ThingADay. “You could think of advocacy as an extension of the Hippocratic oath to do no harm on a government and social level.”

Quoting Margaret Mead, Dr. Worthing reminded rheumatologists that their voices matter despite (and in fact, because of) their small numbers: “Never doubt that a small group of thoughtful, committed citizens can change the world; indeed, it’s the only thing that ever has.”

Dr. Worthing reported having no disclosures.

[email protected]