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Pediatric Drugs

, Volume 21, Issue 3, pp 169–175 | Cite as

Evaluation of Children with Psoriasis from the BiPe Cohort: Are Patients Using Biotherapies in Real Life Eligible for Phase III Clinical Studies?

  • Céline PhanEmail author
  • Alain Beauchet
  • Anne-Claire Burztejn
  • Maëlla Severino-Freire
  • Juliette Mazereeuw-Hautier
  • Sébastien Barbarot
  • Céline Girard
  • Audrey Lasek
  • Ziad Reguiai
  • Claire Abasq
  • Bruno Sassolas
  • Catherine Droitcourt
  • Marc Perrussel
  • Smail Hadj-Rabia
  • Stéphanie Mallet
  • Alice Phan
  • Jean-Philippe Lacour
  • Emmanuelle Bourrat
  • François Aubin
  • Emmanuel Mahé
  • for the Groupe de Recherche de la Société Française de Dermatologie Pédiatrique, and the Groupe de Recherche sur le Psoriasis de la Société Française de Dermatologie
Original Research Article
  • 45 Downloads

Abstract

Background

Phase III clinical trials of biotherapies for childhood psoriasis are designed for a selected population, which can differ from real-life patients.

Objective

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.

Methods

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.

Results

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.

Conclusion

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.

Notes

Acknowledgements

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

Funding

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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Copyright information

© Springer Nature Switzerland AG 2019

Authors and Affiliations

  • Céline Phan
    • 1
    Email author
  • Alain Beauchet
    • 2
  • Anne-Claire Burztejn
    • 3
  • Maëlla Severino-Freire
    • 4
  • Juliette Mazereeuw-Hautier
    • 4
  • Sébastien Barbarot
    • 5
  • Céline Girard
    • 6
  • Audrey Lasek
    • 7
  • Ziad Reguiai
    • 8
  • Claire Abasq
    • 9
  • Bruno Sassolas
    • 9
  • Catherine Droitcourt
    • 10
  • Marc Perrussel
    • 10
  • Smail Hadj-Rabia
    • 11
  • Stéphanie Mallet
    • 12
  • Alice Phan
    • 13
  • Jean-Philippe Lacour
    • 14
  • Emmanuelle Bourrat
    • 15
  • François Aubin
    • 16
  • Emmanuel Mahé
    • 1
  • for the Groupe de Recherche de la Société Française de Dermatologie Pédiatrique, and the Groupe de Recherche sur le Psoriasis de la Société Française de Dermatologie
  1. 1.Service de DermatologieHôpital Victor DupouyArgenteuilFrance
  2. 2.Département de Santé Publique, Centre Hospitalier Universitaire Ambroise ParéAssistance Publique-Hôpitaux de ParisBoulogne-BillancourtFrance
  3. 3.Service de DermatologieHôpital BraboisVandoeuvre Les NancyFrance
  4. 4.Service de DermatologieCentre Hospitalier Universitaire de ToulouseToulouseFrance
  5. 5.Service de DermatologieHôtel DieuNantesFrance
  6. 6.Service de DermatologieCentre Hospitalier Universitaire de MontpellierMontpellierFrance
  7. 7.Service de Dermatologie, Hôpital Saint Vincent de PaulUniversité Catholique de LilleLilleFrance
  8. 8.Service de DermatologiePolyclinique Courlancy, Hôpital Robert DebréReimsFrance
  9. 9.Service de DermatologieCentre Hospitalier Universitaire de BrestBrestFrance
  10. 10.Service de Dermatologie, Centre Hospitalier Universitaire PontchaillouUniversité de RennesRennesFrance
  11. 11.Service de Dermatologie, INSERM U1163 & Institut Imagine, Centre Hospitalier Universitaire Necker-Enfants MaladesAssistance Publique-Hôpitaux de Paris, Université Paris Descartes-Sorbonne, Paris CitéParisFrance
  12. 12.Service de Dermatologie, Vénéréologie et Cancérologie Cutanée, Hôpital de la TimoneAssistance-publique-Hôpitaux de MarseilleMarseilleFrance
  13. 13.Service de Pédiatrie, Hôpital Femme-Mère-EnfantHospices Civils de LyonBronFrance
  14. 14.Service de Dermatologie, Hôpital Archet 2ESPIC CHU-LenvalNiceFrance
  15. 15.Service de Pédiatrie Générale, Hôpital Robert DebréAssistance Publique-Hôpitaux de ParisParisFrance
  16. 16.Service de Dermatologie, Centre Hospitalier Régional Universitaire de BesançonUniversité de Franche ComtéBesançonFrance

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