Acne vulgaris is significantly more common in people with hidradenitis suppurativa (HS) than in the general population, according to Sara Wertenteil and her colleagues in the department of dermatology at Hofstra University in Hempstead, N.Y.

Using data collected by IBM Watson Health, the study authors examined a total of 48,050 adults with HS and 16.9 million adults in the general U.S. population. In this study population, 15.2% of adults with HS had acne, compared with only 2.9% of adults in the general population (P less than .0001), the investigators wrote in the Journal of the American Academy of Dermatology.

After adjusting for age, sex, obesity, smoking status, and polycystic ovarian syndrome (PCOS) status, the odds ratio of adults with HS having acne was 4.51 over those without HS (95% confidence interval, 4.40-4.63). In all subgroups measured (male; female; adults aged 18-44 years, 45-64 years, and 65 years and older; white; nonwhite; obese; nonobese; smoker; nonsmoker; positive for PCOS; non-PCOS) adults with HS were significantly more likely to have acne. The strongest association was in patients who were aged 65 years and older (odds ratio, 10.14; 95% CI, 8.97-11.46).

“Patients with HS have an increased prevalence of [acne vulgaris]. Clinicians treating HS patients should be aware of this burden and its potential implications including a further impact on quality of life. Management strategies should include consideration of both conditions, either with treatments that have overlapping efficacy, or with concomitant therapies,” the authors concluded.

The study was sponsored in part by AbbVie. One coauthor reported having served as an advisor for AbbVie, Pfizer, Janssen, and Asana Biosciences.

[email protected]

SOURCE: Wertentiel S et al. J Am Acad Dermatol. 2018 Oct 1. doi: 10.1016/j.jaad.2018.09.040.

Acne vulgaris is significantly more common in people with hidradenitis suppurativa (HS) than in the general population, according to Sara Wertenteil and her colleagues in the department of dermatology at Hofstra University in Hempstead, N.Y.

Using data collected by IBM Watson Health, the study authors examined a total of 48,050 adults with HS and 16.9 million adults in the general U.S. population. In this study population, 15.2% of adults with HS had acne, compared with only 2.9% of adults in the general population (P less than .0001), the investigators wrote in the Journal of the American Academy of Dermatology.

After adjusting for age, sex, obesity, smoking status, and polycystic ovarian syndrome (PCOS) status, the odds ratio of adults with HS having acne was 4.51 over those without HS (95% confidence interval, 4.40-4.63). In all subgroups measured (male; female; adults aged 18-44 years, 45-64 years, and 65 years and older; white; nonwhite; obese; nonobese; smoker; nonsmoker; positive for PCOS; non-PCOS) adults with HS were significantly more likely to have acne. The strongest association was in patients who were aged 65 years and older (odds ratio, 10.14; 95% CI, 8.97-11.46).

“Patients with HS have an increased prevalence of [acne vulgaris]. Clinicians treating HS patients should be aware of this burden and its potential implications including a further impact on quality of life. Management strategies should include consideration of both conditions, either with treatments that have overlapping efficacy, or with concomitant therapies,” the authors concluded.

The study was sponsored in part by AbbVie. One coauthor reported having served as an advisor for AbbVie, Pfizer, Janssen, and Asana Biosciences.

[email protected]

SOURCE: Wertentiel S et al. J Am Acad Dermatol. 2018 Oct 1. doi: 10.1016/j.jaad.2018.09.040.

Acne vulgaris is significantly more common in people with hidradenitis suppurativa (HS) than in the general population, according to Sara Wertenteil and her colleagues in the department of dermatology at Hofstra University in Hempstead, N.Y.

Using data collected by IBM Watson Health, the study authors examined a total of 48,050 adults with HS and 16.9 million adults in the general U.S. population. In this study population, 15.2% of adults with HS had acne, compared with only 2.9% of adults in the general population (P less than .0001), the investigators wrote in the Journal of the American Academy of Dermatology.

After adjusting for age, sex, obesity, smoking status, and polycystic ovarian syndrome (PCOS) status, the odds ratio of adults with HS having acne was 4.51 over those without HS (95% confidence interval, 4.40-4.63). In all subgroups measured (male; female; adults aged 18-44 years, 45-64 years, and 65 years and older; white; nonwhite; obese; nonobese; smoker; nonsmoker; positive for PCOS; non-PCOS) adults with HS were significantly more likely to have acne. The strongest association was in patients who were aged 65 years and older (odds ratio, 10.14; 95% CI, 8.97-11.46).

“Patients with HS have an increased prevalence of [acne vulgaris]. Clinicians treating HS patients should be aware of this burden and its potential implications including a further impact on quality of life. Management strategies should include consideration of both conditions, either with treatments that have overlapping efficacy, or with concomitant therapies,” the authors concluded.

The study was sponsored in part by AbbVie. One coauthor reported having served as an advisor for AbbVie, Pfizer, Janssen, and Asana Biosciences.

[email protected]

SOURCE: Wertentiel S et al. J Am Acad Dermatol. 2018 Oct 1. doi: 10.1016/j.jaad.2018.09.040.

SAN FRANCISCO – The new herpes zoster subunit vaccine (Shingrix) is on the short list of essential vaccines for immunocompromised adults, including those on biologics.

Ongoing research is demonstrating efficacy and safety in renal transplants patients, as well as those with hematologic cancer and stem cell transplants, according to Lorry Rubin, MD, director of pediatric infectious diseases at Cohen Children’s Medical Center, Queens, and professor of pediatrics at Hofstra University, Hempstead, N.Y.

Immunocompromised people, including those on biologics, should be immunized against a variety of diseases just like everyone else, but it’s tricky. There’s considerable variability in how biologics affect the immune system and subsequent vaccine potency. Timing is important, and although live vaccines are generally a no-go, there’s one class of biologics with which they’re safe, he said.

In a wide-ranging interview at IDWeek 2018, an annual scientific meeting on infectious diseases, Dr. Rubin shared his advice on immunizing the immunocompromised, including the other vaccines on the short list. He also tackled the common concern that vaccinations might trigger rejection in transplant patients.

He’s well qualified to address the issues: Dr. Rubin was lead author on the 2013 Infectious Diseases Society of America guidelines on vaccinating immunocompromised patients.

[email protected]

SAN FRANCISCO – The new herpes zoster subunit vaccine (Shingrix) is on the short list of essential vaccines for immunocompromised adults, including those on biologics.

Ongoing research is demonstrating efficacy and safety in renal transplants patients, as well as those with hematologic cancer and stem cell transplants, according to Lorry Rubin, MD, director of pediatric infectious diseases at Cohen Children’s Medical Center, Queens, and professor of pediatrics at Hofstra University, Hempstead, N.Y.

Immunocompromised people, including those on biologics, should be immunized against a variety of diseases just like everyone else, but it’s tricky. There’s considerable variability in how biologics affect the immune system and subsequent vaccine potency. Timing is important, and although live vaccines are generally a no-go, there’s one class of biologics with which they’re safe, he said.

In a wide-ranging interview at IDWeek 2018, an annual scientific meeting on infectious diseases, Dr. Rubin shared his advice on immunizing the immunocompromised, including the other vaccines on the short list. He also tackled the common concern that vaccinations might trigger rejection in transplant patients.

He’s well qualified to address the issues: Dr. Rubin was lead author on the 2013 Infectious Diseases Society of America guidelines on vaccinating immunocompromised patients.

[email protected]

SAN FRANCISCO – The new herpes zoster subunit vaccine (Shingrix) is on the short list of essential vaccines for immunocompromised adults, including those on biologics.

Ongoing research is demonstrating efficacy and safety in renal transplants patients, as well as those with hematologic cancer and stem cell transplants, according to Lorry Rubin, MD, director of pediatric infectious diseases at Cohen Children’s Medical Center, Queens, and professor of pediatrics at Hofstra University, Hempstead, N.Y.

Immunocompromised people, including those on biologics, should be immunized against a variety of diseases just like everyone else, but it’s tricky. There’s considerable variability in how biologics affect the immune system and subsequent vaccine potency. Timing is important, and although live vaccines are generally a no-go, there’s one class of biologics with which they’re safe, he said.

In a wide-ranging interview at IDWeek 2018, an annual scientific meeting on infectious diseases, Dr. Rubin shared his advice on immunizing the immunocompromised, including the other vaccines on the short list. He also tackled the common concern that vaccinations might trigger rejection in transplant patients.

He’s well qualified to address the issues: Dr. Rubin was lead author on the 2013 Infectious Diseases Society of America guidelines on vaccinating immunocompromised patients.

[email protected]

SAN FRANCISCO – Direct-acting antiretrovirals are more effective than pegylated interferon/ribavirin in reducing cardiovascular risk in people with hepatitis C virus, according to a review of over 30,000 patients in the ERCHIVES (Electronically Retrieved Cohort of HCV Infected Veterans) database of the Veterans Health Administration system.

In the study, 12,667 patients were treated with direct-acting antiretrovirals (DAAs) and 4,436 with pegylated interferon/ribavirin (PEG/RBV). Each subject was matched to an untreated control based on alcohol use, diabetes, and other confounders. Patients with HIV, hepatitis B, or previously diagnosed cardiovascular disease were excluded.

Over a follow-up of about 10 years, there were 2,361 strokes, heart attacks, or other cardiovascular (CV) events among untreated patients, which translated to an incidence of 30.9 events per 1,000 patient years. In the PEG/RBV group, there were 804 events, yielding an incidence of 23.5 per 1,000 patient years. The DAA group fared better, with 435 events and an incident rate of 16.3.

Sustained virologic response also was associated with lower CV risk, and the odds of attaining it were about 25% greater with DAAs vs. PEG/RBV. That might have played a role in the findings, since hepatitis C is known to be associated with CV disease and the virus has been found in atherosclerotic plaques.

Past investigations have been mixed on whether or not hepatitis C virus treatment reduces CV risk, but lead investigator Adeel Ajwad Butt, MD, professor of medicine at Cornell University, New York, said that his study was stronger than what has come before. He explained why, and also why the findings matter, in an interview at ID Week 2018, an annual scientific meeting on infectious diseases.

Most of the subjects were men, about a quarter were black, and the median age at baseline was 58 years.

Dr. Butt disclosed institutional research grants from Merck and Gilead.

[email protected]

SOURCE: Butt AA et al. ID Week 2018 abstract 930.

SAN FRANCISCO – Direct-acting antiretrovirals are more effective than pegylated interferon/ribavirin in reducing cardiovascular risk in people with hepatitis C virus, according to a review of over 30,000 patients in the ERCHIVES (Electronically Retrieved Cohort of HCV Infected Veterans) database of the Veterans Health Administration system.

In the study, 12,667 patients were treated with direct-acting antiretrovirals (DAAs) and 4,436 with pegylated interferon/ribavirin (PEG/RBV). Each subject was matched to an untreated control based on alcohol use, diabetes, and other confounders. Patients with HIV, hepatitis B, or previously diagnosed cardiovascular disease were excluded.

Over a follow-up of about 10 years, there were 2,361 strokes, heart attacks, or other cardiovascular (CV) events among untreated patients, which translated to an incidence of 30.9 events per 1,000 patient years. In the PEG/RBV group, there were 804 events, yielding an incidence of 23.5 per 1,000 patient years. The DAA group fared better, with 435 events and an incident rate of 16.3.

Sustained virologic response also was associated with lower CV risk, and the odds of attaining it were about 25% greater with DAAs vs. PEG/RBV. That might have played a role in the findings, since hepatitis C is known to be associated with CV disease and the virus has been found in atherosclerotic plaques.

Past investigations have been mixed on whether or not hepatitis C virus treatment reduces CV risk, but lead investigator Adeel Ajwad Butt, MD, professor of medicine at Cornell University, New York, said that his study was stronger than what has come before. He explained why, and also why the findings matter, in an interview at ID Week 2018, an annual scientific meeting on infectious diseases.

Most of the subjects were men, about a quarter were black, and the median age at baseline was 58 years.

Dr. Butt disclosed institutional research grants from Merck and Gilead.

[email protected]

SOURCE: Butt AA et al. ID Week 2018 abstract 930.

SAN FRANCISCO – Direct-acting antiretrovirals are more effective than pegylated interferon/ribavirin in reducing cardiovascular risk in people with hepatitis C virus, according to a review of over 30,000 patients in the ERCHIVES (Electronically Retrieved Cohort of HCV Infected Veterans) database of the Veterans Health Administration system.

In the study, 12,667 patients were treated with direct-acting antiretrovirals (DAAs) and 4,436 with pegylated interferon/ribavirin (PEG/RBV). Each subject was matched to an untreated control based on alcohol use, diabetes, and other confounders. Patients with HIV, hepatitis B, or previously diagnosed cardiovascular disease were excluded.

Over a follow-up of about 10 years, there were 2,361 strokes, heart attacks, or other cardiovascular (CV) events among untreated patients, which translated to an incidence of 30.9 events per 1,000 patient years. In the PEG/RBV group, there were 804 events, yielding an incidence of 23.5 per 1,000 patient years. The DAA group fared better, with 435 events and an incident rate of 16.3.

Sustained virologic response also was associated with lower CV risk, and the odds of attaining it were about 25% greater with DAAs vs. PEG/RBV. That might have played a role in the findings, since hepatitis C is known to be associated with CV disease and the virus has been found in atherosclerotic plaques.

Past investigations have been mixed on whether or not hepatitis C virus treatment reduces CV risk, but lead investigator Adeel Ajwad Butt, MD, professor of medicine at Cornell University, New York, said that his study was stronger than what has come before. He explained why, and also why the findings matter, in an interview at ID Week 2018, an annual scientific meeting on infectious diseases.

Most of the subjects were men, about a quarter were black, and the median age at baseline was 58 years.

Dr. Butt disclosed institutional research grants from Merck and Gilead.

[email protected]

SOURCE: Butt AA et al. ID Week 2018 abstract 930.

SAN FRANCISCO – In early October, the Food and Drug Administration approved omadacycline (Nuzyra) for the treatment of community-acquired bacterial pneumonia and acute bacterial skin and skin structure infections in adults.

The drug is a synthetic tetracycline designed to overcome some of the resistance mechanisms that can undermine traditional tetracycline drugs. It gained approval on the strength of the Oasis-1 (NCT03482011) and Oasis-2 (NCT03535194) trials, which demonstrated the drug’s noninferiority to linezolid.

At IDWeek 2018, researchers combined the data from the two pivotal trials to gain more power in some of the secondary endpoints, such as adverse events.

Paul McGovern, MD, vice president of clinical and medical affairs at Paratek, which markets omadacycline, discussed the results of the analysis at an annual scientific meeting on infectious diseases.

Combined, the two studies included 691 patients who received omadacycline and 689 who received linezolid. The two drugs achieved similar results for early clinical response, defined as at least a 20% reduction in lesion size 48-72 hours after the first dose. The mean reduction in baseline lesion area at day 3 was 53.4% in the omadacycline group and 53.0% in the linezolid group. At the end of the treatment period, those values were 93.9% and 93.7%, respectively, Dr. McGovern reported.

A total of 28.5% of patients receiving omadacycline reported drug-related treatment-emergent adverse events, compared with 16.1% of the linezolid group. The omadacycline group experienced higher frequency of nausea (21.9% vs. 8.7%) and vomiting (11.4% vs. 3.9%), he said.

The Oasis 1 and Oasis 2 studies were funded by Paratek. Dr. McGovern is an employee of Paratek.

SOURCE: McGovern P et al. IDWeek 2018, poster abstract 1347.

SAN FRANCISCO – In early October, the Food and Drug Administration approved omadacycline (Nuzyra) for the treatment of community-acquired bacterial pneumonia and acute bacterial skin and skin structure infections in adults.

The drug is a synthetic tetracycline designed to overcome some of the resistance mechanisms that can undermine traditional tetracycline drugs. It gained approval on the strength of the Oasis-1 (NCT03482011) and Oasis-2 (NCT03535194) trials, which demonstrated the drug’s noninferiority to linezolid.

At IDWeek 2018, researchers combined the data from the two pivotal trials to gain more power in some of the secondary endpoints, such as adverse events.

Paul McGovern, MD, vice president of clinical and medical affairs at Paratek, which markets omadacycline, discussed the results of the analysis at an annual scientific meeting on infectious diseases.

Combined, the two studies included 691 patients who received omadacycline and 689 who received linezolid. The two drugs achieved similar results for early clinical response, defined as at least a 20% reduction in lesion size 48-72 hours after the first dose. The mean reduction in baseline lesion area at day 3 was 53.4% in the omadacycline group and 53.0% in the linezolid group. At the end of the treatment period, those values were 93.9% and 93.7%, respectively, Dr. McGovern reported.

A total of 28.5% of patients receiving omadacycline reported drug-related treatment-emergent adverse events, compared with 16.1% of the linezolid group. The omadacycline group experienced higher frequency of nausea (21.9% vs. 8.7%) and vomiting (11.4% vs. 3.9%), he said.

The Oasis 1 and Oasis 2 studies were funded by Paratek. Dr. McGovern is an employee of Paratek.

SOURCE: McGovern P et al. IDWeek 2018, poster abstract 1347.

SAN FRANCISCO – In early October, the Food and Drug Administration approved omadacycline (Nuzyra) for the treatment of community-acquired bacterial pneumonia and acute bacterial skin and skin structure infections in adults.

The drug is a synthetic tetracycline designed to overcome some of the resistance mechanisms that can undermine traditional tetracycline drugs. It gained approval on the strength of the Oasis-1 (NCT03482011) and Oasis-2 (NCT03535194) trials, which demonstrated the drug’s noninferiority to linezolid.

At IDWeek 2018, researchers combined the data from the two pivotal trials to gain more power in some of the secondary endpoints, such as adverse events.

Paul McGovern, MD, vice president of clinical and medical affairs at Paratek, which markets omadacycline, discussed the results of the analysis at an annual scientific meeting on infectious diseases.

Combined, the two studies included 691 patients who received omadacycline and 689 who received linezolid. The two drugs achieved similar results for early clinical response, defined as at least a 20% reduction in lesion size 48-72 hours after the first dose. The mean reduction in baseline lesion area at day 3 was 53.4% in the omadacycline group and 53.0% in the linezolid group. At the end of the treatment period, those values were 93.9% and 93.7%, respectively, Dr. McGovern reported.

A total of 28.5% of patients receiving omadacycline reported drug-related treatment-emergent adverse events, compared with 16.1% of the linezolid group. The omadacycline group experienced higher frequency of nausea (21.9% vs. 8.7%) and vomiting (11.4% vs. 3.9%), he said.

The Oasis 1 and Oasis 2 studies were funded by Paratek. Dr. McGovern is an employee of Paratek.

SOURCE: McGovern P et al. IDWeek 2018, poster abstract 1347.

Photo from Novartis

The cost-effectiveness of tisagenlecleucel (Kymriah) depends on long-term clinical outcomes, which are presently unknown, according to investigators.

If the long-term outcomes are more modest than clinical trials suggest, then payers may be unwilling to cover the costly therapy, reported John K. Lin, MD, of Stanford University, and his colleagues.

Lowering the price or setting up an outcomes-based pricing structure may be necessary to get insurers to cover the therapy.

Tisagenlecleucel is an anti-CD19 chimeric antigen receptor (CAR) T-cell therapy that was approved by the U.S. Food and Drug Administration in August 2017 for relapsed or refractory pediatric B-cell acute lymphoblastic leukemia (ALL).

In 2018, the FDA expanded the indication for tisagenlecleucel to include adults with relapsed or refractory large B-cell lymphoma, though outcomes from lymphoma trials are not analyzed in the current study.

At a wholesale acquisition cost of $475,000 per infusion, it is the most expensive existing oncology therapy to date, and can be accompanied by expensive, potentially fatal adverse effects.

However, clinical trials suggest that tisagenlecleucel can offer years of relapse-free remission, thereby allowing patients to forgo other expensive therapies such as hematopoietic stem cell transplantation (HSCT).

“Although tisagenlecleucel-induced remission rates are promising, compared with those of established therapies (greater than 80% vs. less than 50%), only short-term follow-up data currently exist,” the investigators wrote in the Journal of Clinical Oncology.

“Given the high cost and broad applicability in other malignancies of tisagenlecleucel, a pressing question for policy makers, payers, patients, and clinicians is whether the cost of therapy represents reasonable value.”

The study used a Markov model to assess various long-term clinical outcome rates and cost thresholds of tisagenlecleucel. The lifetime cost of therapy was assessed and compared with costs of existing therapies.

The results showed that a 5-year relapse free survival rate of 40% would make the present cost ($475,000) of tisagenlecleucel economically reasonable. In this scenario, the increased life expectancy would be 12.1 years and would result in an additional 5.07 quality-adjusted life years (QALY) gained at a cost of $61,000 per QALY, compared with blinatumomab.

But if long-term outcomes are less favorable, tisagenlecleucel becomes much less cost effective. A 5-year relapse-free survival rate of 20% would drop increased life expectancy to 3.8 years, resulting in 1.80 QALYs gained and raising the cost to $151,000 per QALY.

“Our results suggest that at tisagenlecleucel’s current price and payment structure, its economic value is uncertain,” the investigators wrote.

They suggested a price drop to $200,000 or $350,000, which would allow the drug to remain cost effective even in a worse-case scenario, in which patients relapse and tisagenlecleucel is a bridge to transplant.

Another option is to move to outcomes-based pricing. Making payment conditional on 7 months of remission would make the treatment cost effective, according to the analysis.

“Price reductions of tisagenlecleucel or payment only for longer-term remissions would favorably influence cost-effectiveness, even if long-term clinical outcomes are modest,” the investigators wrote.

The study was funded by a Veterans Affairs Office of Academic Affiliations advanced fellowship in health service and research development, and a National Center for Advancing Translational Science Clinical and Translational Science Award.

One of the study coauthors reported consulting and research funding from Novartis. 

Photo from Novartis

The cost-effectiveness of tisagenlecleucel (Kymriah) depends on long-term clinical outcomes, which are presently unknown, according to investigators.

If the long-term outcomes are more modest than clinical trials suggest, then payers may be unwilling to cover the costly therapy, reported John K. Lin, MD, of Stanford University, and his colleagues.

Lowering the price or setting up an outcomes-based pricing structure may be necessary to get insurers to cover the therapy.

Tisagenlecleucel is an anti-CD19 chimeric antigen receptor (CAR) T-cell therapy that was approved by the U.S. Food and Drug Administration in August 2017 for relapsed or refractory pediatric B-cell acute lymphoblastic leukemia (ALL).

In 2018, the FDA expanded the indication for tisagenlecleucel to include adults with relapsed or refractory large B-cell lymphoma, though outcomes from lymphoma trials are not analyzed in the current study.

At a wholesale acquisition cost of $475,000 per infusion, it is the most expensive existing oncology therapy to date, and can be accompanied by expensive, potentially fatal adverse effects.

However, clinical trials suggest that tisagenlecleucel can offer years of relapse-free remission, thereby allowing patients to forgo other expensive therapies such as hematopoietic stem cell transplantation (HSCT).

“Although tisagenlecleucel-induced remission rates are promising, compared with those of established therapies (greater than 80% vs. less than 50%), only short-term follow-up data currently exist,” the investigators wrote in the Journal of Clinical Oncology.

“Given the high cost and broad applicability in other malignancies of tisagenlecleucel, a pressing question for policy makers, payers, patients, and clinicians is whether the cost of therapy represents reasonable value.”

The study used a Markov model to assess various long-term clinical outcome rates and cost thresholds of tisagenlecleucel. The lifetime cost of therapy was assessed and compared with costs of existing therapies.

The results showed that a 5-year relapse free survival rate of 40% would make the present cost ($475,000) of tisagenlecleucel economically reasonable. In this scenario, the increased life expectancy would be 12.1 years and would result in an additional 5.07 quality-adjusted life years (QALY) gained at a cost of $61,000 per QALY, compared with blinatumomab.

But if long-term outcomes are less favorable, tisagenlecleucel becomes much less cost effective. A 5-year relapse-free survival rate of 20% would drop increased life expectancy to 3.8 years, resulting in 1.80 QALYs gained and raising the cost to $151,000 per QALY.

“Our results suggest that at tisagenlecleucel’s current price and payment structure, its economic value is uncertain,” the investigators wrote.

They suggested a price drop to $200,000 or $350,000, which would allow the drug to remain cost effective even in a worse-case scenario, in which patients relapse and tisagenlecleucel is a bridge to transplant.

Another option is to move to outcomes-based pricing. Making payment conditional on 7 months of remission would make the treatment cost effective, according to the analysis.

“Price reductions of tisagenlecleucel or payment only for longer-term remissions would favorably influence cost-effectiveness, even if long-term clinical outcomes are modest,” the investigators wrote.

The study was funded by a Veterans Affairs Office of Academic Affiliations advanced fellowship in health service and research development, and a National Center for Advancing Translational Science Clinical and Translational Science Award.

One of the study coauthors reported consulting and research funding from Novartis. 

Photo from Novartis

The cost-effectiveness of tisagenlecleucel (Kymriah) depends on long-term clinical outcomes, which are presently unknown, according to investigators.

If the long-term outcomes are more modest than clinical trials suggest, then payers may be unwilling to cover the costly therapy, reported John K. Lin, MD, of Stanford University, and his colleagues.

Lowering the price or setting up an outcomes-based pricing structure may be necessary to get insurers to cover the therapy.

Tisagenlecleucel is an anti-CD19 chimeric antigen receptor (CAR) T-cell therapy that was approved by the U.S. Food and Drug Administration in August 2017 for relapsed or refractory pediatric B-cell acute lymphoblastic leukemia (ALL).

In 2018, the FDA expanded the indication for tisagenlecleucel to include adults with relapsed or refractory large B-cell lymphoma, though outcomes from lymphoma trials are not analyzed in the current study.

At a wholesale acquisition cost of $475,000 per infusion, it is the most expensive existing oncology therapy to date, and can be accompanied by expensive, potentially fatal adverse effects.

However, clinical trials suggest that tisagenlecleucel can offer years of relapse-free remission, thereby allowing patients to forgo other expensive therapies such as hematopoietic stem cell transplantation (HSCT).

“Although tisagenlecleucel-induced remission rates are promising, compared with those of established therapies (greater than 80% vs. less than 50%), only short-term follow-up data currently exist,” the investigators wrote in the Journal of Clinical Oncology.

“Given the high cost and broad applicability in other malignancies of tisagenlecleucel, a pressing question for policy makers, payers, patients, and clinicians is whether the cost of therapy represents reasonable value.”

The study used a Markov model to assess various long-term clinical outcome rates and cost thresholds of tisagenlecleucel. The lifetime cost of therapy was assessed and compared with costs of existing therapies.

The results showed that a 5-year relapse free survival rate of 40% would make the present cost ($475,000) of tisagenlecleucel economically reasonable. In this scenario, the increased life expectancy would be 12.1 years and would result in an additional 5.07 quality-adjusted life years (QALY) gained at a cost of $61,000 per QALY, compared with blinatumomab.

But if long-term outcomes are less favorable, tisagenlecleucel becomes much less cost effective. A 5-year relapse-free survival rate of 20% would drop increased life expectancy to 3.8 years, resulting in 1.80 QALYs gained and raising the cost to $151,000 per QALY.

“Our results suggest that at tisagenlecleucel’s current price and payment structure, its economic value is uncertain,” the investigators wrote.

They suggested a price drop to $200,000 or $350,000, which would allow the drug to remain cost effective even in a worse-case scenario, in which patients relapse and tisagenlecleucel is a bridge to transplant.

Another option is to move to outcomes-based pricing. Making payment conditional on 7 months of remission would make the treatment cost effective, according to the analysis.

“Price reductions of tisagenlecleucel or payment only for longer-term remissions would favorably influence cost-effectiveness, even if long-term clinical outcomes are modest,” the investigators wrote.

The study was funded by a Veterans Affairs Office of Academic Affiliations advanced fellowship in health service and research development, and a National Center for Advancing Translational Science Clinical and Translational Science Award.

One of the study coauthors reported consulting and research funding from Novartis. 

The approval of the 9-valent human papillomavirus (HPV) vaccine has been expanded to include an older age group, men and women aged 27-45 years, the Food and Drug Administration announced on Oct. 5.

Wikimedia Commons/FitzColinGerald/Creative Commons License

The vaccine (Gardasil 9) was previously approved for those aged 9-26 years.

The approval “represents an important opportunity to help prevent HPV-related diseases and cancers in a broader age range,” Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research, said in the FDA statement announcing the approval.

“The Centers for Disease Control and Prevention has stated that HPV vaccination prior to becoming infected with the HPV types covered by the vaccine has the potential to prevent more than 90 percent of these cancers, or 31,200 cases every year, from ever developing,” he added.

Gardasil 9, approved in 2014, covers the four HPV types included in the original Gardasil vaccine approved in 2006, plus five additional HPV types.

The approval is based on the results of a study and follow-up of about 3,200 women aged 27-45 years, followed for an average of 3.5 years, which found that the vaccine was 88% percent effective “in the prevention of a combined endpoint of persistent infection, genital warts, vulvar and vaginal precancerous lesions, cervical precancerous lesions, and cervical cancer related to HPV types covered by the vaccine,” according to the FDA. The vaccine’s effectiveness in men in this age group is “inferred” from these results and from data on Gardasil in men aged 16-26 years, as well as “immunogenicity data from a clinical trial in which 150 men, 27 through 45 years of age, received a 3-dose regimen of Gardasil over 6 months,” the FDA statement noted.

Based on safety data in about 13,000 men and women, injection-site pain, swelling, redness, and headaches are the most common adverse reactions associated with Gardasil 9, the statement said. Gardasil 9 is manufactured by Merck.

[email protected]

The approval of the 9-valent human papillomavirus (HPV) vaccine has been expanded to include an older age group, men and women aged 27-45 years, the Food and Drug Administration announced on Oct. 5.

Wikimedia Commons/FitzColinGerald/Creative Commons License

The vaccine (Gardasil 9) was previously approved for those aged 9-26 years.

The approval “represents an important opportunity to help prevent HPV-related diseases and cancers in a broader age range,” Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research, said in the FDA statement announcing the approval.

“The Centers for Disease Control and Prevention has stated that HPV vaccination prior to becoming infected with the HPV types covered by the vaccine has the potential to prevent more than 90 percent of these cancers, or 31,200 cases every year, from ever developing,” he added.

Gardasil 9, approved in 2014, covers the four HPV types included in the original Gardasil vaccine approved in 2006, plus five additional HPV types.

The approval is based on the results of a study and follow-up of about 3,200 women aged 27-45 years, followed for an average of 3.5 years, which found that the vaccine was 88% percent effective “in the prevention of a combined endpoint of persistent infection, genital warts, vulvar and vaginal precancerous lesions, cervical precancerous lesions, and cervical cancer related to HPV types covered by the vaccine,” according to the FDA. The vaccine’s effectiveness in men in this age group is “inferred” from these results and from data on Gardasil in men aged 16-26 years, as well as “immunogenicity data from a clinical trial in which 150 men, 27 through 45 years of age, received a 3-dose regimen of Gardasil over 6 months,” the FDA statement noted.

Based on safety data in about 13,000 men and women, injection-site pain, swelling, redness, and headaches are the most common adverse reactions associated with Gardasil 9, the statement said. Gardasil 9 is manufactured by Merck.

[email protected]

The approval of the 9-valent human papillomavirus (HPV) vaccine has been expanded to include an older age group, men and women aged 27-45 years, the Food and Drug Administration announced on Oct. 5.

Wikimedia Commons/FitzColinGerald/Creative Commons License

The vaccine (Gardasil 9) was previously approved for those aged 9-26 years.

The approval “represents an important opportunity to help prevent HPV-related diseases and cancers in a broader age range,” Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research, said in the FDA statement announcing the approval.

“The Centers for Disease Control and Prevention has stated that HPV vaccination prior to becoming infected with the HPV types covered by the vaccine has the potential to prevent more than 90 percent of these cancers, or 31,200 cases every year, from ever developing,” he added.

Gardasil 9, approved in 2014, covers the four HPV types included in the original Gardasil vaccine approved in 2006, plus five additional HPV types.

The approval is based on the results of a study and follow-up of about 3,200 women aged 27-45 years, followed for an average of 3.5 years, which found that the vaccine was 88% percent effective “in the prevention of a combined endpoint of persistent infection, genital warts, vulvar and vaginal precancerous lesions, cervical precancerous lesions, and cervical cancer related to HPV types covered by the vaccine,” according to the FDA. The vaccine’s effectiveness in men in this age group is “inferred” from these results and from data on Gardasil in men aged 16-26 years, as well as “immunogenicity data from a clinical trial in which 150 men, 27 through 45 years of age, received a 3-dose regimen of Gardasil over 6 months,” the FDA statement noted.

Based on safety data in about 13,000 men and women, injection-site pain, swelling, redness, and headaches are the most common adverse reactions associated with Gardasil 9, the statement said. Gardasil 9 is manufactured by Merck.

[email protected]

SAN FRANCISCO – For the first time, the World Health Organization’s Monitored Emergency Use of Unregistered and Investigational Interventions (MEURI) protocol is being field tested in the Democratic Republic of Congo (DRC), where four different therapeutics are being delivered to Ebola patients. MEURI was created after controversy surrounding the 2014-2016 Ebola outbreak in West Africa, which ultimately led to the decision to integrate research into outbreak responses.

gl0ck/Thinkstock

MEURI is broadly designed for pathogens that have no proven intervention, on the premise that in a disease with high mortality, it can be ethically appropriate to provide experimental therapies during a response, as long as a set of criteria are met that ensure patient autonomy and give patients a reasonable opportunity to improve their condition.

The original plan was to employ experimental drugs under the MEURI umbrella during an Ebola outbreak in the DRC that began in May, but an effective response contained it so quickly that the program was canceled. However, when another Ebola outbreak occurred on Aug. 27, the material was still in the country and ready to be deployed. “That made it very easy to begin using them,” said Elizabeth Higgs, MD, global health science advisor for the division of clinical research at the National Institute for Allergy and Infectious Disease.

Dr. Higgs spoke about the developments during a late-breakers session at IDWeek 2018, an annual scientific meeting on infectious disease.

The therapies under consideration are Zmapp (Leaf Biopharmaceutical), which includes three chimeric monoclonal antibodies that target the main surface protein of the Ebola virus; mAb114 (NIAID), which was isolated in 1995 from a human survivor of Ebola and binds to the core of the Ebola surface protein; Remdesivir (Gilead), a small molecule that is believed to interfere with viral replication; and Regeneron’s Ebola triple monoclonal antibody cocktail, which also targets the surface protein.

Through Oct. 2, 43 Ebola patients have received one of the four drugs: 19 are cured and have been discharged, 12 have died, and 12 are still in recovery. Dr. Higgs cautioned against reading anything into those numbers, however, since no randomization was involved.

Although compassionate use is the primary aim of MEURI, it may be lead to some scientific benefit. The most that can be hoped for is to glean some insight into the safety of the interventions, although even that information is limited. “A colleague and I were talking about this the other day. After the first X number of patients, they were all still alive. What can you say from that? I think what you can say is that it didn’t kill them,” Dr. Higgs said.

Still, researchers are collecting laboratory data from patients to monitor their responses. “With Remdesivir we’re closely examining their transaminase, for example, so I think it will be helpful,” said Dr. Higgs.

SOURCE: Higgs E et al. IDWeek 2018.

SAN FRANCISCO – For the first time, the World Health Organization’s Monitored Emergency Use of Unregistered and Investigational Interventions (MEURI) protocol is being field tested in the Democratic Republic of Congo (DRC), where four different therapeutics are being delivered to Ebola patients. MEURI was created after controversy surrounding the 2014-2016 Ebola outbreak in West Africa, which ultimately led to the decision to integrate research into outbreak responses.

gl0ck/Thinkstock

MEURI is broadly designed for pathogens that have no proven intervention, on the premise that in a disease with high mortality, it can be ethically appropriate to provide experimental therapies during a response, as long as a set of criteria are met that ensure patient autonomy and give patients a reasonable opportunity to improve their condition.

The original plan was to employ experimental drugs under the MEURI umbrella during an Ebola outbreak in the DRC that began in May, but an effective response contained it so quickly that the program was canceled. However, when another Ebola outbreak occurred on Aug. 27, the material was still in the country and ready to be deployed. “That made it very easy to begin using them,” said Elizabeth Higgs, MD, global health science advisor for the division of clinical research at the National Institute for Allergy and Infectious Disease.

Dr. Higgs spoke about the developments during a late-breakers session at IDWeek 2018, an annual scientific meeting on infectious disease.

The therapies under consideration are Zmapp (Leaf Biopharmaceutical), which includes three chimeric monoclonal antibodies that target the main surface protein of the Ebola virus; mAb114 (NIAID), which was isolated in 1995 from a human survivor of Ebola and binds to the core of the Ebola surface protein; Remdesivir (Gilead), a small molecule that is believed to interfere with viral replication; and Regeneron’s Ebola triple monoclonal antibody cocktail, which also targets the surface protein.

Through Oct. 2, 43 Ebola patients have received one of the four drugs: 19 are cured and have been discharged, 12 have died, and 12 are still in recovery. Dr. Higgs cautioned against reading anything into those numbers, however, since no randomization was involved.

Although compassionate use is the primary aim of MEURI, it may be lead to some scientific benefit. The most that can be hoped for is to glean some insight into the safety of the interventions, although even that information is limited. “A colleague and I were talking about this the other day. After the first X number of patients, they were all still alive. What can you say from that? I think what you can say is that it didn’t kill them,” Dr. Higgs said.

Still, researchers are collecting laboratory data from patients to monitor their responses. “With Remdesivir we’re closely examining their transaminase, for example, so I think it will be helpful,” said Dr. Higgs.

SOURCE: Higgs E et al. IDWeek 2018.

SAN FRANCISCO – For the first time, the World Health Organization’s Monitored Emergency Use of Unregistered and Investigational Interventions (MEURI) protocol is being field tested in the Democratic Republic of Congo (DRC), where four different therapeutics are being delivered to Ebola patients. MEURI was created after controversy surrounding the 2014-2016 Ebola outbreak in West Africa, which ultimately led to the decision to integrate research into outbreak responses.

gl0ck/Thinkstock

MEURI is broadly designed for pathogens that have no proven intervention, on the premise that in a disease with high mortality, it can be ethically appropriate to provide experimental therapies during a response, as long as a set of criteria are met that ensure patient autonomy and give patients a reasonable opportunity to improve their condition.

The original plan was to employ experimental drugs under the MEURI umbrella during an Ebola outbreak in the DRC that began in May, but an effective response contained it so quickly that the program was canceled. However, when another Ebola outbreak occurred on Aug. 27, the material was still in the country and ready to be deployed. “That made it very easy to begin using them,” said Elizabeth Higgs, MD, global health science advisor for the division of clinical research at the National Institute for Allergy and Infectious Disease.

Dr. Higgs spoke about the developments during a late-breakers session at IDWeek 2018, an annual scientific meeting on infectious disease.

The therapies under consideration are Zmapp (Leaf Biopharmaceutical), which includes three chimeric monoclonal antibodies that target the main surface protein of the Ebola virus; mAb114 (NIAID), which was isolated in 1995 from a human survivor of Ebola and binds to the core of the Ebola surface protein; Remdesivir (Gilead), a small molecule that is believed to interfere with viral replication; and Regeneron’s Ebola triple monoclonal antibody cocktail, which also targets the surface protein.

Through Oct. 2, 43 Ebola patients have received one of the four drugs: 19 are cured and have been discharged, 12 have died, and 12 are still in recovery. Dr. Higgs cautioned against reading anything into those numbers, however, since no randomization was involved.

Although compassionate use is the primary aim of MEURI, it may be lead to some scientific benefit. The most that can be hoped for is to glean some insight into the safety of the interventions, although even that information is limited. “A colleague and I were talking about this the other day. After the first X number of patients, they were all still alive. What can you say from that? I think what you can say is that it didn’t kill them,” Dr. Higgs said.

Still, researchers are collecting laboratory data from patients to monitor their responses. “With Remdesivir we’re closely examining their transaminase, for example, so I think it will be helpful,” said Dr. Higgs.

SOURCE: Higgs E et al. IDWeek 2018.

MONTEREY, CALIF. – Looking for a better treatment for a stubborn dermatologic condition? Pick up the telephone, work closely with nondermatologists, and find a dermatology specialty pharmacy. Seek out clinical trials that fit your patient’s needs. And be aware that data may not provide the best dosage protocols.

These tips and more came from Neal Bhatia, MD, director of clinical dermatology at Therapeutics Clinical Research in San Diego, who spoke about best practices in drug therapy at the annual Coastal Dermatology Symposium. Dr. Bhatia urged colleagues not to give up on conditions such as vitiligo, alopecia areata, severe atopic dermatitis (AD), granulomatous disorders, recalcitrant urticaria, and itching, even when there is resistance from insurance companies. Instead, he said, rely on persistence and the power of a united front with other specialists.

Rheumatologists, oncologists, and allergists may be helpful allies in certain cases, he said, as can dermatologic specialty pharmacies. And if you’re dealing with a medical director of an insurance company, he said, make sure to call. Don’t write a letter or send a fax.

Because some treatments never get evaluated in clinical trials because of cost or lack of interest, he also recommended that dermatologists keep an eye on anecdotal protocols, which can provide helpful “real-world options,” he said in an interview. “Searching for conclusions from case reports and small independent studies can be just as informative and beneficial to patient care as pivotal data from large late-phase studies. Practical information, pearls on management, and other important tips can be found in anecdotes that were either too small or short in duration to be conducted as a validated trial.”

One option is to check ClinicalTrials.gov for a trial; another is to pursue an investigator-initiated study. “Some companies will offer the option to fund a small study for one or several patients to get them treatment and drugs without a high expense for the study or funding for the site’s costs,” Dr. Bhatia said.

In his presentation, Dr. Bhatia referred to a variety of dermatologic medications are showing promising results in trials, and some relatively new options.

Topical hypochlorous acid (Sebuderm gel) for dermatoses, such as seborrheic dermatitis. This prescription nonsteroidal gel is now available in the United States and was cleared by the Food and Drug Administration for seborrhea and seborrheic dermatitis in 2015. Dr. Bhatia referred to a study presented in a poster at a meeting in 2017 that showed improvement in 20 of 24 patients with mild to moderate seborrheic dermatitis treated with this product after 28 days. None of the patients worsened, and overall disease activity fell by more than half.

Loyon lotion (Cetiol oil and dimethicone), a nonmedicated descaling treatment for scaly patches in infants with cradle cap (seborrheic dermatitis). A 2014 pilot study found that it improved scaling in 80% of 20 infants and children aged 3-36 months over 8 days, and it reached “treatment success” in 50% (Dermatol Ther [Heidelb]. 2014 Dec;4[2]:221-32).

(As for cost, GoodRx.com states that various pharmacies sell one 8-ounce [227-gram] bottle of Sebuderm gel for about $300 with a coupon. Loyon is also expensive, with a GoodRx.com listing its price at about $300, with coupon, for one 50-ml spray bottle.)

Dr. Bhatia also reviewed drugs in the research pipeline. Several drugs for AD are in early phases of research, while some phase 3 trials involving Janus kinase (JAK) inhibitors for AD are starting – or are close to starting.

He pointed to other research that’s underway on treatments for acne and rosacea. Three acne drugs that he said are “almost here” are minocycline gel and cortexolone 17 alpha-propionate 1% cream. He also referred to sarecycline, a tetracycline-derived antibiotic taken orally, which was approved by the Food and Drug Administration on October 2 for moderate to severe acne vulgaris in patients aged 9 years and older.

This symposium was jointly presented by the University of Louisville (Ky.) and the Global Academy for Medical Education. This publication and the Global Academy for Medical Education are both owned by Frontline Medical Communications.

Dr. Bhatia reported affiliations with multiple drugmakers: Abbvie, Aclaris, Almirall, Bayer, Biofrontera, BioPharmX, Dermira, Encore, EPI Health, Ferndale, Foamix, Galderma, IntraDerm, ISDIN, La Roche-Posay, Leo, Mayne, Menlo, Novartis, Ortho, Pfizer, Pierre Fabre, Promius, Regeneron, Sanofi, Skinfix, Soligenix, Sun, and Vidac.
 

MONTEREY, CALIF. – Looking for a better treatment for a stubborn dermatologic condition? Pick up the telephone, work closely with nondermatologists, and find a dermatology specialty pharmacy. Seek out clinical trials that fit your patient’s needs. And be aware that data may not provide the best dosage protocols.

These tips and more came from Neal Bhatia, MD, director of clinical dermatology at Therapeutics Clinical Research in San Diego, who spoke about best practices in drug therapy at the annual Coastal Dermatology Symposium. Dr. Bhatia urged colleagues not to give up on conditions such as vitiligo, alopecia areata, severe atopic dermatitis (AD), granulomatous disorders, recalcitrant urticaria, and itching, even when there is resistance from insurance companies. Instead, he said, rely on persistence and the power of a united front with other specialists.

Rheumatologists, oncologists, and allergists may be helpful allies in certain cases, he said, as can dermatologic specialty pharmacies. And if you’re dealing with a medical director of an insurance company, he said, make sure to call. Don’t write a letter or send a fax.

Because some treatments never get evaluated in clinical trials because of cost or lack of interest, he also recommended that dermatologists keep an eye on anecdotal protocols, which can provide helpful “real-world options,” he said in an interview. “Searching for conclusions from case reports and small independent studies can be just as informative and beneficial to patient care as pivotal data from large late-phase studies. Practical information, pearls on management, and other important tips can be found in anecdotes that were either too small or short in duration to be conducted as a validated trial.”

One option is to check ClinicalTrials.gov for a trial; another is to pursue an investigator-initiated study. “Some companies will offer the option to fund a small study for one or several patients to get them treatment and drugs without a high expense for the study or funding for the site’s costs,” Dr. Bhatia said.

In his presentation, Dr. Bhatia referred to a variety of dermatologic medications are showing promising results in trials, and some relatively new options.

Topical hypochlorous acid (Sebuderm gel) for dermatoses, such as seborrheic dermatitis. This prescription nonsteroidal gel is now available in the United States and was cleared by the Food and Drug Administration for seborrhea and seborrheic dermatitis in 2015. Dr. Bhatia referred to a study presented in a poster at a meeting in 2017 that showed improvement in 20 of 24 patients with mild to moderate seborrheic dermatitis treated with this product after 28 days. None of the patients worsened, and overall disease activity fell by more than half.

Loyon lotion (Cetiol oil and dimethicone), a nonmedicated descaling treatment for scaly patches in infants with cradle cap (seborrheic dermatitis). A 2014 pilot study found that it improved scaling in 80% of 20 infants and children aged 3-36 months over 8 days, and it reached “treatment success” in 50% (Dermatol Ther [Heidelb]. 2014 Dec;4[2]:221-32).

(As for cost, GoodRx.com states that various pharmacies sell one 8-ounce [227-gram] bottle of Sebuderm gel for about $300 with a coupon. Loyon is also expensive, with a GoodRx.com listing its price at about $300, with coupon, for one 50-ml spray bottle.)

Dr. Bhatia also reviewed drugs in the research pipeline. Several drugs for AD are in early phases of research, while some phase 3 trials involving Janus kinase (JAK) inhibitors for AD are starting – or are close to starting.

He pointed to other research that’s underway on treatments for acne and rosacea. Three acne drugs that he said are “almost here” are minocycline gel and cortexolone 17 alpha-propionate 1% cream. He also referred to sarecycline, a tetracycline-derived antibiotic taken orally, which was approved by the Food and Drug Administration on October 2 for moderate to severe acne vulgaris in patients aged 9 years and older.

This symposium was jointly presented by the University of Louisville (Ky.) and the Global Academy for Medical Education. This publication and the Global Academy for Medical Education are both owned by Frontline Medical Communications.

Dr. Bhatia reported affiliations with multiple drugmakers: Abbvie, Aclaris, Almirall, Bayer, Biofrontera, BioPharmX, Dermira, Encore, EPI Health, Ferndale, Foamix, Galderma, IntraDerm, ISDIN, La Roche-Posay, Leo, Mayne, Menlo, Novartis, Ortho, Pfizer, Pierre Fabre, Promius, Regeneron, Sanofi, Skinfix, Soligenix, Sun, and Vidac.
 

MONTEREY, CALIF. – Looking for a better treatment for a stubborn dermatologic condition? Pick up the telephone, work closely with nondermatologists, and find a dermatology specialty pharmacy. Seek out clinical trials that fit your patient’s needs. And be aware that data may not provide the best dosage protocols.

These tips and more came from Neal Bhatia, MD, director of clinical dermatology at Therapeutics Clinical Research in San Diego, who spoke about best practices in drug therapy at the annual Coastal Dermatology Symposium. Dr. Bhatia urged colleagues not to give up on conditions such as vitiligo, alopecia areata, severe atopic dermatitis (AD), granulomatous disorders, recalcitrant urticaria, and itching, even when there is resistance from insurance companies. Instead, he said, rely on persistence and the power of a united front with other specialists.

Rheumatologists, oncologists, and allergists may be helpful allies in certain cases, he said, as can dermatologic specialty pharmacies. And if you’re dealing with a medical director of an insurance company, he said, make sure to call. Don’t write a letter or send a fax.

Because some treatments never get evaluated in clinical trials because of cost or lack of interest, he also recommended that dermatologists keep an eye on anecdotal protocols, which can provide helpful “real-world options,” he said in an interview. “Searching for conclusions from case reports and small independent studies can be just as informative and beneficial to patient care as pivotal data from large late-phase studies. Practical information, pearls on management, and other important tips can be found in anecdotes that were either too small or short in duration to be conducted as a validated trial.”

One option is to check ClinicalTrials.gov for a trial; another is to pursue an investigator-initiated study. “Some companies will offer the option to fund a small study for one or several patients to get them treatment and drugs without a high expense for the study or funding for the site’s costs,” Dr. Bhatia said.

In his presentation, Dr. Bhatia referred to a variety of dermatologic medications are showing promising results in trials, and some relatively new options.

Topical hypochlorous acid (Sebuderm gel) for dermatoses, such as seborrheic dermatitis. This prescription nonsteroidal gel is now available in the United States and was cleared by the Food and Drug Administration for seborrhea and seborrheic dermatitis in 2015. Dr. Bhatia referred to a study presented in a poster at a meeting in 2017 that showed improvement in 20 of 24 patients with mild to moderate seborrheic dermatitis treated with this product after 28 days. None of the patients worsened, and overall disease activity fell by more than half.

Loyon lotion (Cetiol oil and dimethicone), a nonmedicated descaling treatment for scaly patches in infants with cradle cap (seborrheic dermatitis). A 2014 pilot study found that it improved scaling in 80% of 20 infants and children aged 3-36 months over 8 days, and it reached “treatment success” in 50% (Dermatol Ther [Heidelb]. 2014 Dec;4[2]:221-32).

(As for cost, GoodRx.com states that various pharmacies sell one 8-ounce [227-gram] bottle of Sebuderm gel for about $300 with a coupon. Loyon is also expensive, with a GoodRx.com listing its price at about $300, with coupon, for one 50-ml spray bottle.)

Dr. Bhatia also reviewed drugs in the research pipeline. Several drugs for AD are in early phases of research, while some phase 3 trials involving Janus kinase (JAK) inhibitors for AD are starting – or are close to starting.

He pointed to other research that’s underway on treatments for acne and rosacea. Three acne drugs that he said are “almost here” are minocycline gel and cortexolone 17 alpha-propionate 1% cream. He also referred to sarecycline, a tetracycline-derived antibiotic taken orally, which was approved by the Food and Drug Administration on October 2 for moderate to severe acne vulgaris in patients aged 9 years and older.

This symposium was jointly presented by the University of Louisville (Ky.) and the Global Academy for Medical Education. This publication and the Global Academy for Medical Education are both owned by Frontline Medical Communications.

Dr. Bhatia reported affiliations with multiple drugmakers: Abbvie, Aclaris, Almirall, Bayer, Biofrontera, BioPharmX, Dermira, Encore, EPI Health, Ferndale, Foamix, Galderma, IntraDerm, ISDIN, La Roche-Posay, Leo, Mayne, Menlo, Novartis, Ortho, Pfizer, Pierre Fabre, Promius, Regeneron, Sanofi, Skinfix, Soligenix, Sun, and Vidac.
 

MONTEREY, CALIF. – Rosacea may be one disease, but it often requires more than one treatment, a dermatologist said at the annual Coastal Dermatology Symposium. Don’t assume you can just prescribe one drug like you might with acne, she advised.

“Treat everything that you see,” said dermatologist Julie C. Harper, MD, of Birmingham, Ala. “That may mean a laser or something you’re using off-label. Different lesions and signs of rosacea will require multiple modes of treatment.”

Dr. Harper offered these other pearls to consider when treating rosacea:

• Don’t get hung up on subtypes.

The four subtypes of rosacea should be used to classify lesions, not people, she said. That’s because patients can fall into more than one of the four categories – erythematotelangiectatic rosacea, papulopustular rosacea, phymatous rosacea, and ocular rosacea, she noted.

“Document the redness you see and ask them what’s bothering them the most,” she said. And ask yourself, she added, “Do I have them on everything that I should have them on?”

• Talk to patients about triggers.

For the first visit, “we have to talk to patients about skin care and triggers,” Dr. Harper noted. According to the American Academy of Dermatology, common rosacea triggers include sunlight, hairspray, heat, stress, alcohol, and spicy foods.

• Consider an ivermectin-brimonidine combination.

“Targeting inflammation in papules and pustules doesn’t necessarily translate to less background erythema,” Dr. Harper said. What to do? She pointed to a 2017 study that examined a combination treatment of ivermectin 1% topical cream (Soolantra) and brimonidine 0.33% topical gel (Mirvaso) for patients with rosacea with moderate to severe persistent erythema and inflammatory lesions. Ivermectin is indicated for inflammatory lesions, while brimonidine treats persistent erythema.

Rosacea.org

At week 12, the proportion of patients who achieved investigator global assessment of clear or almost clear was 55.8% in the combination group, versus 36.8% of those in the vehicle group (P = .007), according to the study (J Drugs Dermatol. 2017 Sep 1;16[9]:909-16). Dr. Harper highlighted the effect of brimonidine when added to ivermectin. “In a period of 3 hours,” she said, “we had twice as many people fall into clear or almost clear.”

• Consider adding botulinum toxin to your toolbox.

This “really does work,” Dr. Harper said. She pointed to a 2015 report of botulinum toxin use in two cases of refractory flushing and erythema and a 2012 report of 13 cases in patients with the same symptoms (Dermatology. 2015;230:299-301; J Drugs Dermatol. 2012 Dec;11[12]:e76-9). Dr. Harper said that she usually uses the full 50-unit dose of Botox.

• Consider a beta-blocker.

According to a 2018 report, the beta-blocker carvedilol (Coreg) showed benefit when added to other treatments in five patients with facial flushing and persistent erythema.

• Keep isotretinoin in mind.

A 2016 report suggested low-dose isotretinoin had value for difficult-to-treat papulopustular rosacea. As Dr. Harper noted, 57% of those who took isotretinoin reached the primary endpoint, versus 10% of those taking the placebo. However, relapses over 4 months were common, which is a sign that it may be wise to prescribe low doses over the long term, but not in females of child-bearing potential, she said.

The Coastal Dermatology Symposium is jointly presented by the University of Louisville and Global Academy for Medical Education. This publication and Global Academy for Medical Education are both owned by Frontline Medical Communications.

Dr. Harper disclosed speaker/advisor relationships with Allergan, Bayer, BioPharmX, Galderma, LaRoche Posay, and Ortho and has served as investigator for Bayer.

MONTEREY, CALIF. – Rosacea may be one disease, but it often requires more than one treatment, a dermatologist said at the annual Coastal Dermatology Symposium. Don’t assume you can just prescribe one drug like you might with acne, she advised.

“Treat everything that you see,” said dermatologist Julie C. Harper, MD, of Birmingham, Ala. “That may mean a laser or something you’re using off-label. Different lesions and signs of rosacea will require multiple modes of treatment.”

Dr. Harper offered these other pearls to consider when treating rosacea:

• Don’t get hung up on subtypes.

The four subtypes of rosacea should be used to classify lesions, not people, she said. That’s because patients can fall into more than one of the four categories – erythematotelangiectatic rosacea, papulopustular rosacea, phymatous rosacea, and ocular rosacea, she noted.

“Document the redness you see and ask them what’s bothering them the most,” she said. And ask yourself, she added, “Do I have them on everything that I should have them on?”

• Talk to patients about triggers.

For the first visit, “we have to talk to patients about skin care and triggers,” Dr. Harper noted. According to the American Academy of Dermatology, common rosacea triggers include sunlight, hairspray, heat, stress, alcohol, and spicy foods.

• Consider an ivermectin-brimonidine combination.

“Targeting inflammation in papules and pustules doesn’t necessarily translate to less background erythema,” Dr. Harper said. What to do? She pointed to a 2017 study that examined a combination treatment of ivermectin 1% topical cream (Soolantra) and brimonidine 0.33% topical gel (Mirvaso) for patients with rosacea with moderate to severe persistent erythema and inflammatory lesions. Ivermectin is indicated for inflammatory lesions, while brimonidine treats persistent erythema.

Rosacea.org

At week 12, the proportion of patients who achieved investigator global assessment of clear or almost clear was 55.8% in the combination group, versus 36.8% of those in the vehicle group (P = .007), according to the study (J Drugs Dermatol. 2017 Sep 1;16[9]:909-16). Dr. Harper highlighted the effect of brimonidine when added to ivermectin. “In a period of 3 hours,” she said, “we had twice as many people fall into clear or almost clear.”

• Consider adding botulinum toxin to your toolbox.

This “really does work,” Dr. Harper said. She pointed to a 2015 report of botulinum toxin use in two cases of refractory flushing and erythema and a 2012 report of 13 cases in patients with the same symptoms (Dermatology. 2015;230:299-301; J Drugs Dermatol. 2012 Dec;11[12]:e76-9). Dr. Harper said that she usually uses the full 50-unit dose of Botox.

• Consider a beta-blocker.

According to a 2018 report, the beta-blocker carvedilol (Coreg) showed benefit when added to other treatments in five patients with facial flushing and persistent erythema.

• Keep isotretinoin in mind.

A 2016 report suggested low-dose isotretinoin had value for difficult-to-treat papulopustular rosacea. As Dr. Harper noted, 57% of those who took isotretinoin reached the primary endpoint, versus 10% of those taking the placebo. However, relapses over 4 months were common, which is a sign that it may be wise to prescribe low doses over the long term, but not in females of child-bearing potential, she said.

The Coastal Dermatology Symposium is jointly presented by the University of Louisville and Global Academy for Medical Education. This publication and Global Academy for Medical Education are both owned by Frontline Medical Communications.

Dr. Harper disclosed speaker/advisor relationships with Allergan, Bayer, BioPharmX, Galderma, LaRoche Posay, and Ortho and has served as investigator for Bayer.

MONTEREY, CALIF. – Rosacea may be one disease, but it often requires more than one treatment, a dermatologist said at the annual Coastal Dermatology Symposium. Don’t assume you can just prescribe one drug like you might with acne, she advised.

“Treat everything that you see,” said dermatologist Julie C. Harper, MD, of Birmingham, Ala. “That may mean a laser or something you’re using off-label. Different lesions and signs of rosacea will require multiple modes of treatment.”

Dr. Harper offered these other pearls to consider when treating rosacea:

• Don’t get hung up on subtypes.

The four subtypes of rosacea should be used to classify lesions, not people, she said. That’s because patients can fall into more than one of the four categories – erythematotelangiectatic rosacea, papulopustular rosacea, phymatous rosacea, and ocular rosacea, she noted.

“Document the redness you see and ask them what’s bothering them the most,” she said. And ask yourself, she added, “Do I have them on everything that I should have them on?”

• Talk to patients about triggers.

For the first visit, “we have to talk to patients about skin care and triggers,” Dr. Harper noted. According to the American Academy of Dermatology, common rosacea triggers include sunlight, hairspray, heat, stress, alcohol, and spicy foods.

• Consider an ivermectin-brimonidine combination.

“Targeting inflammation in papules and pustules doesn’t necessarily translate to less background erythema,” Dr. Harper said. What to do? She pointed to a 2017 study that examined a combination treatment of ivermectin 1% topical cream (Soolantra) and brimonidine 0.33% topical gel (Mirvaso) for patients with rosacea with moderate to severe persistent erythema and inflammatory lesions. Ivermectin is indicated for inflammatory lesions, while brimonidine treats persistent erythema.

Rosacea.org

At week 12, the proportion of patients who achieved investigator global assessment of clear or almost clear was 55.8% in the combination group, versus 36.8% of those in the vehicle group (P = .007), according to the study (J Drugs Dermatol. 2017 Sep 1;16[9]:909-16). Dr. Harper highlighted the effect of brimonidine when added to ivermectin. “In a period of 3 hours,” she said, “we had twice as many people fall into clear or almost clear.”

• Consider adding botulinum toxin to your toolbox.

This “really does work,” Dr. Harper said. She pointed to a 2015 report of botulinum toxin use in two cases of refractory flushing and erythema and a 2012 report of 13 cases in patients with the same symptoms (Dermatology. 2015;230:299-301; J Drugs Dermatol. 2012 Dec;11[12]:e76-9). Dr. Harper said that she usually uses the full 50-unit dose of Botox.

• Consider a beta-blocker.

According to a 2018 report, the beta-blocker carvedilol (Coreg) showed benefit when added to other treatments in five patients with facial flushing and persistent erythema.

• Keep isotretinoin in mind.

A 2016 report suggested low-dose isotretinoin had value for difficult-to-treat papulopustular rosacea. As Dr. Harper noted, 57% of those who took isotretinoin reached the primary endpoint, versus 10% of those taking the placebo. However, relapses over 4 months were common, which is a sign that it may be wise to prescribe low doses over the long term, but not in females of child-bearing potential, she said.

The Coastal Dermatology Symposium is jointly presented by the University of Louisville and Global Academy for Medical Education. This publication and Global Academy for Medical Education are both owned by Frontline Medical Communications.

Dr. Harper disclosed speaker/advisor relationships with Allergan, Bayer, BioPharmX, Galderma, LaRoche Posay, and Ortho and has served as investigator for Bayer.

Photo courtesy of NCCN

New York—Although the data set is small and not yet mature, chimeric antigen receptor (CAR) T-cell therapy appears to be a promising approach for Hodgkin lymphoma, according to Philippe Armand, MD, PhD, of Dana-Farber/Brigham and Women’s Cancer Center and the Massachusetts General Hospital Cancer Center.

While based on a handful of patients, the data do suggest this approach may play a role either by targeting CD30 or Epstein Barr virus (EBV), Dr. Armand said in a presentation at the NCCN 13^th Annual Congress: Hematologic Malignancies.

“Most importantly perhaps, like it's experience outside of Hodgkin lymphoma, it may really have curative potential, based on the long CR rates that have been already exhibited,” he told attendees at the NCCN conference.

Much of the published clinical experience to date is with CD30-directed CAR Ts, Dr. Armand said, noting that in Hodgkin lymphoma, results so far show promise for this particular approach.

In a recent phase 1 dose escalation study, 9 patients with relapsed/refractory Hodgkin lymphoma or anaplastic large-cell lymphoma (ALCL) received infusions of autologous T cells modified to express CD30-specific CAR T cells encoding the CD28 costimulatory domain, with no conditioning regimen.

Out of 7 relapsed Hodgkin patients, one had a complete response (CR) lasting beyond 2.5 years following a second infusion. Another had a CR persisting almost 2 years and 3 had transient stable disease.

One of the 2 ALCL patients had a CR lasting 9 months after a fourth infusion. No toxicities attributable to the therapy were seen, according to investigators.

The CD30 CAR T cells are being evaluated with a conditioning regimen in the phase 1 RELY-30 trial. According to Dr. Armand, preliminary results presented at the EBMT 2018 meeting showed better expansion of CAR T cells and responses in 3 out of 5 patients, including 2 CRs.

A CD30-directed CAR T-cell therapy with a 4-1bb costimulatory domain has also been tested in a small group of Hodgkin patients with a response rate of 35%, including some CRs. Response rates were lower in patients with extranodal involvement, although that needs to be validated with further study, according to Dr. Armand.

A considerable amount of active research is ongoing in China, Dr. Armand said, while a phase 1 study of T cells expressing a fully human anti-CD30 CAR is being evaluated in the United States in CD30-expressing lymphomas, he added.

Among non-CD30-targeted products, a CD19 CAR-T approach has been tried in Hodgkin lymphoma, though preliminary results suggest only transient activity.

An interesting approach has been the targeting of EBV, Dr. Armand noted. Recently reported results showed that two doses of T cells with specificity for EBV-derived tumor antigens induced clinical responses in patients with EBV-positive Hodgkin lymphoma.

The cells were engineered to express dominant-negative TGF-β receptor type 2 (DNRII).

“We know that TGF-β provides a strong immunosuppressant signal in the tumor microenvironment,” Dr. Armand said, noting that some of the responses in the 7 evaluable patients lasted 4 years or more. 

Photo courtesy of NCCN

New York—Although the data set is small and not yet mature, chimeric antigen receptor (CAR) T-cell therapy appears to be a promising approach for Hodgkin lymphoma, according to Philippe Armand, MD, PhD, of Dana-Farber/Brigham and Women’s Cancer Center and the Massachusetts General Hospital Cancer Center.

While based on a handful of patients, the data do suggest this approach may play a role either by targeting CD30 or Epstein Barr virus (EBV), Dr. Armand said in a presentation at the NCCN 13^th Annual Congress: Hematologic Malignancies.

“Most importantly perhaps, like it's experience outside of Hodgkin lymphoma, it may really have curative potential, based on the long CR rates that have been already exhibited,” he told attendees at the NCCN conference.

Much of the published clinical experience to date is with CD30-directed CAR Ts, Dr. Armand said, noting that in Hodgkin lymphoma, results so far show promise for this particular approach.

In a recent phase 1 dose escalation study, 9 patients with relapsed/refractory Hodgkin lymphoma or anaplastic large-cell lymphoma (ALCL) received infusions of autologous T cells modified to express CD30-specific CAR T cells encoding the CD28 costimulatory domain, with no conditioning regimen.

Out of 7 relapsed Hodgkin patients, one had a complete response (CR) lasting beyond 2.5 years following a second infusion. Another had a CR persisting almost 2 years and 3 had transient stable disease.

One of the 2 ALCL patients had a CR lasting 9 months after a fourth infusion. No toxicities attributable to the therapy were seen, according to investigators.

The CD30 CAR T cells are being evaluated with a conditioning regimen in the phase 1 RELY-30 trial. According to Dr. Armand, preliminary results presented at the EBMT 2018 meeting showed better expansion of CAR T cells and responses in 3 out of 5 patients, including 2 CRs.

A CD30-directed CAR T-cell therapy with a 4-1bb costimulatory domain has also been tested in a small group of Hodgkin patients with a response rate of 35%, including some CRs. Response rates were lower in patients with extranodal involvement, although that needs to be validated with further study, according to Dr. Armand.

A considerable amount of active research is ongoing in China, Dr. Armand said, while a phase 1 study of T cells expressing a fully human anti-CD30 CAR is being evaluated in the United States in CD30-expressing lymphomas, he added.

Among non-CD30-targeted products, a CD19 CAR-T approach has been tried in Hodgkin lymphoma, though preliminary results suggest only transient activity.

An interesting approach has been the targeting of EBV, Dr. Armand noted. Recently reported results showed that two doses of T cells with specificity for EBV-derived tumor antigens induced clinical responses in patients with EBV-positive Hodgkin lymphoma.

The cells were engineered to express dominant-negative TGF-β receptor type 2 (DNRII).

“We know that TGF-β provides a strong immunosuppressant signal in the tumor microenvironment,” Dr. Armand said, noting that some of the responses in the 7 evaluable patients lasted 4 years or more. 

Photo courtesy of NCCN

New York—Although the data set is small and not yet mature, chimeric antigen receptor (CAR) T-cell therapy appears to be a promising approach for Hodgkin lymphoma, according to Philippe Armand, MD, PhD, of Dana-Farber/Brigham and Women’s Cancer Center and the Massachusetts General Hospital Cancer Center.

While based on a handful of patients, the data do suggest this approach may play a role either by targeting CD30 or Epstein Barr virus (EBV), Dr. Armand said in a presentation at the NCCN 13^th Annual Congress: Hematologic Malignancies.

“Most importantly perhaps, like it's experience outside of Hodgkin lymphoma, it may really have curative potential, based on the long CR rates that have been already exhibited,” he told attendees at the NCCN conference.

Much of the published clinical experience to date is with CD30-directed CAR Ts, Dr. Armand said, noting that in Hodgkin lymphoma, results so far show promise for this particular approach.

In a recent phase 1 dose escalation study, 9 patients with relapsed/refractory Hodgkin lymphoma or anaplastic large-cell lymphoma (ALCL) received infusions of autologous T cells modified to express CD30-specific CAR T cells encoding the CD28 costimulatory domain, with no conditioning regimen.

Out of 7 relapsed Hodgkin patients, one had a complete response (CR) lasting beyond 2.5 years following a second infusion. Another had a CR persisting almost 2 years and 3 had transient stable disease.

One of the 2 ALCL patients had a CR lasting 9 months after a fourth infusion. No toxicities attributable to the therapy were seen, according to investigators.

The CD30 CAR T cells are being evaluated with a conditioning regimen in the phase 1 RELY-30 trial. According to Dr. Armand, preliminary results presented at the EBMT 2018 meeting showed better expansion of CAR T cells and responses in 3 out of 5 patients, including 2 CRs.

A CD30-directed CAR T-cell therapy with a 4-1bb costimulatory domain has also been tested in a small group of Hodgkin patients with a response rate of 35%, including some CRs. Response rates were lower in patients with extranodal involvement, although that needs to be validated with further study, according to Dr. Armand.

A considerable amount of active research is ongoing in China, Dr. Armand said, while a phase 1 study of T cells expressing a fully human anti-CD30 CAR is being evaluated in the United States in CD30-expressing lymphomas, he added.

Among non-CD30-targeted products, a CD19 CAR-T approach has been tried in Hodgkin lymphoma, though preliminary results suggest only transient activity.

An interesting approach has been the targeting of EBV, Dr. Armand noted. Recently reported results showed that two doses of T cells with specificity for EBV-derived tumor antigens induced clinical responses in patients with EBV-positive Hodgkin lymphoma.

The cells were engineered to express dominant-negative TGF-β receptor type 2 (DNRII).

“We know that TGF-β provides a strong immunosuppressant signal in the tumor microenvironment,” Dr. Armand said, noting that some of the responses in the 7 evaluable patients lasted 4 years or more.