Apremilast (Otezla), an oral drug approved for adult psoriasis, appears to reduce psoriasis severity in some children with moderate to severe psoriasis not controlled by topical therapy, according to the results of a phase 3 trial.
“Unfortunately, there are limited treatment options for pediatric patients with moderate to severe plaque psoriasis” who do not respond or cannot use topical therapy, said study investigator Anna Belloni Fortina, MD, speaking at the 31st Congress of the European Academy of Dermatology and Venereology.
“In this randomized, placebo-controlled trial, oral apremilast demonstrated effectiveness and was well tolerated,” added Belloni Fortina. “I underline oral because for children, oral administration is better than the injection treatment,” added Belloni Fortina, of Azienda Ospedale Università Padova in Italy.
Dubbed the SPROUT study, the trial set a primary endpoint of the percentage of children with a Physician’s Global Assessment (sPGA) response after 16 weeks of treatment or placebo. sPGA is a 5-point scale ranging from 0 (clear) to 4 (severe). The study enrolled children with an sPGA ≥ 3. Response was defined as a sPGA score of 0 or 1, indicating clear or almost clear skin, with at least a 2-point reduction from baseline values.
At week 16, the primary endpoint was met by 33% of 163 children treated with apremilast vs 11% of 82 children who had been given a placebo, a treatment difference of 21.7% (95% CI, 11.2%-32.1%).
A greater proportion of children treated with apremilast also achieved a major secondary endpoint, a 75% or greater reduction in the Psoriasis Area and Severity Index (PASI-75) (45.4% vs 16.1%), a treatment difference of 29.4% (95% CI, 17.8%-40.9%).
Results Unaffected by Weight and Age
Regarding apremilast, “it’s important to underline that patients were dosed according to their weight,” Belloni Fortina said.
A dose of 20 mg twice daily was given to children who weighed between 20 kg and < 50 kg, and a 30-mg twice-daily dose was given to those who weighed ≥ 50 kg.
When the data were analyzed according to weight, proportionately more children on apremilast saw a sPGA response: 47.4% vs 21.8% in the lower weight and dose range and 19.2% vs 1.6% in the higher weight and dose range.
As for PASI-75, a greater proportion of children on apremilast also responded in both the lower and upper weight ranges, a respective 52.4% and 38.7% of patients compared with 21.4% and 11% of those treated with placebo.
Data were also evaluated according to age, with a younger (aged 6-11 years) and older (age 12-17 years) group. The mean age of children was 12 years overall. Results showed a similar pattern for weight: The psoriasis of more children treated with apremilast was reduced by both measures, sPGA response, and PASI-75.
Safety of Apremilast in Children
“The overall the safety profile, during the placebo-controlled phase was comparable with the known safety profile of apremilast,” Belloni Fontina reported. “No new safety signals were identified.”
The rate of any adverse event was substantially higher in children given the active treatment, however, at 65% vs 41.3% for placebo.
Rates of severe and serious adverse events were low, at around 1.3%, and similar between the groups.
There was also a low rate of withdrawal because of side effects, although this was higher in the apremilast group (3.1% vs 1.3%).
The primary reason for withdrawal of apremilast treatment were the most commonly reported adverse events: gastrointestinal disorders, including diarrhea, nausea, upper and lower abdominal pain, and vomiting. Headache, pyrexia, and nasopharyngitis were also reported.
Despite being common, most treatment-related adverse effects resolved within 3 days, Belloni Fontina said.
Expect Further Data
Further data from the trial are to be expected because only the 16-week primary endpoint results have been released so far. The trial also included a 36-week extension phase during which all children who had originally been randomly assigned to placebo were now eligible to be treated with apremilast, and all those who were originally given the active treatment were able to continue. This extension treatment period means that data will be available for a full year of treatment, and there will also be a further 2-week observational follow-up at the end of the trial.
The study was funded by Amgen. Belloni Fontina reported acting as an investigator and advisory board member for and receiving honoraria from Amgen, Galderma, Leo Pharma, and Pfizer. She also reported speaking on behalf of Pierre-Fabre and Galderma.
Congress of the European Academy of Dermatology and Venereology: Late-breaking Oral Presentation LB-3549. Presented September 8, 2022. News release
Sara Freeman is a freelance journalist based in London, England.
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