Evaluation of Children with Psoriasis from the BiPe Cohort: Are Patients Using Biotherapies in Real Life Eligible for Phase III Clinical Studies?
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Phase III clinical trials of biotherapies for childhood psoriasis are designed for a selected population, which can differ from real-life patients.
Our objective was to assess the proportion of children with psoriasis that received biotherapy in the biological treatments for pediatric psoriasis (BiPe) cohort that would be excluded from phase III clinical trials of these treatments.
Data concerning initiation of the first biotherapy from all patients included in the BiPe cohort were analyzed. Ineligibility was assessed after applying the exclusion criteria used in the principal phase III trials of etanercept, adalimumab, and ustekinumab for childhood psoriasis.
Of the 134 patients included, 73 (54.5%) were ineligible for at least one randomized controlled trial based on one or more exclusion criteria. Amongst the 63 children treated with etanercept, 35 (55.5%) were ineligible: 22 because of the type of psoriasis, 12 because of concomitant treatment, and six because of psoriasis severity based on psoriasis assessment severity index (PASI) and physician global assessment (PGA) scores (PASI < 12 and PGA < 3). Amongst the 44 children treated with adalimumab, 32 (72.7%) were ineligible: 17 because of the clinical type of psoriasis, 12 because of psoriasis severity (PASI < 20 and PGA < 4), and seven because of concomitant treatment. Amongst the 27 children patients treated with ustekinumab, 12 (44.4%) were ineligible: eight because of psoriasis severity (PASI < 12 and PGA < 3), five because of the clinical type of psoriasis, and one because of concomitant treatment. Drug survival and the frequency of serious adverse events did not differ between eligible and ineligible patients.
The majority of children treated with biotherapies in real-life practice differ from those in phase III trials, most commonly because of the clinical type of their psoriasis, the disease severity being lower than required and the use of prior or concomitant psoriasis treatment. Efficacy and safety results from phase III clinical trials in selected populations may not sufficiently reflect what is seen in real life, thus results from real-life cohort studies are necessary.
The authors thank G. Chaby, R. Deborde, P. Plantin, A. Maruani, M. Piram, F. Maccari, A.-C. Fougerousse, I. Kupfer-Bessaguet, X. Balguérie, H. Barthelemy, L. Martin, N. Quiles-Tsimaratos, L. Mery-Brossard, V. Pallure, D. Lons-Danic, D. Bouilly-Auvray, M. Beylot-Barry, and E. Puzenat for their kind collaboration. Language editing services were provided by Dr. E. Pilling and editing services by Dr. M. Rommet (Santé Active Edition).
Compliance with ethical standards
This study did not receive any specific funding.
Conflict of interest
ZR has undertaken paid activities as a consultant, advisor, or speaker for Janssen Cilag, Pfizer, Abbvie, and Novartis. MP and JPL have undertaken paid activities as a consultant, advisor, or speaker for Abbvie. FA has undertaken activities as a consultant, advisor, or speaker for Abbvie, Janssen, Celgene, Leo Pharma, Amgen, Abbvie, Novartis, and Pfizer. EM has undertaken paid activities as a consultant, advisor, or speaker for Abbvie, Boehringer-Ingelheim, Janssen, Celgene, Leo Pharma, Amgen, AstraZeneca, Abbvie, Novartis, and Pfizer. CP, AB, ACB, MSF, JMH, SB, CG, AL, CA, BS, CD, SHR, SM, AP, EB have no conflicts of interest that are directly relevant to the content of this article.
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