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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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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.

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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).

Author information

Authors and Affiliations

  1. Service de Dermatologie, Hôpital Victor Dupouy, 69 rue du Lieutenant-Colonel Prud’hon, 95100, Argenteuil, France

    Céline Phan & Emmanuel Mahé

  2. Département de Santé Publique, Centre Hospitalier Universitaire Ambroise Paré, Assistance Publique-Hôpitaux de Paris, Boulogne-Billancourt, France

    Alain Beauchet

  3. Service de Dermatologie, Hôpital Brabois, 54000, Vandoeuvre Les Nancy, France

    Anne-Claire Burztejn

  4. Service de Dermatologie, Centre Hospitalier Universitaire de Toulouse, Toulouse, France

    Maëlla Severino-Freire & Juliette Mazereeuw-Hautier

  5. Service de Dermatologie, Hôtel Dieu, Nantes, France

    Sébastien Barbarot

  6. Service de Dermatologie, Centre Hospitalier Universitaire de Montpellier, Montpellier, France

    Céline Girard

  7. Service de Dermatologie, Hôpital Saint Vincent de Paul, Université Catholique de Lille, Lille, France

    Audrey Lasek

  8. Service de Dermatologie, Polyclinique Courlancy, Hôpital Robert Debré, Reims, France

    Ziad Reguiai

  9. Service de Dermatologie, Centre Hospitalier Universitaire de Brest, Brest, France

    Claire Abasq & Bruno Sassolas

  10. Service de Dermatologie, Centre Hospitalier Universitaire Pontchaillou, Université de Rennes, Rennes, France

    Catherine Droitcourt & Marc Perrussel

  11. Service de Dermatologie, INSERM U1163 & Institut Imagine, Centre Hospitalier Universitaire Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Université Paris Descartes-Sorbonne, Paris Cité, Paris, France

    Smail Hadj-Rabia

  12. Service de Dermatologie, Vénéréologie et Cancérologie Cutanée, Hôpital de la Timone, Assistance-publique-Hôpitaux de Marseille, Marseille, France

    Stéphanie Mallet

  13. Service de Pédiatrie, Hôpital Femme-Mère-Enfant, Hospices Civils de Lyon, Bron, France

    Alice Phan

  14. Service de Dermatologie, Hôpital Archet 2, ESPIC CHU-Lenval, Nice, France

    Jean-Philippe Lacour

  15. Service de Pédiatrie Générale, Hôpital Robert Debré, Assistance Publique-Hôpitaux de Paris, Paris, France

    Emmanuelle Bourrat

  16. Service de Dermatologie, Centre Hospitalier Régional Universitaire de Besançon, Université de Franche Comté, Besançon, France

    François Aubin

Authors
  1. Céline Phan
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  2. Alain Beauchet
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  3. Anne-Claire Burztejn
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  4. Maëlla Severino-Freire
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  5. Juliette Mazereeuw-Hautier
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  6. Sébastien Barbarot
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  7. Céline Girard
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  8. Audrey Lasek
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  9. Ziad Reguiai
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  10. Claire Abasq
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  11. Bruno Sassolas
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  12. Catherine Droitcourt
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  13. Marc Perrussel
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  14. Smail Hadj-Rabia
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  15. Stéphanie Mallet
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  16. Alice Phan
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  17. Jean-Philippe Lacour
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  18. Emmanuelle Bourrat
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  19. François Aubin
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  20. Emmanuel Mahé
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Consortia

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

Corresponding author

Correspondence to Céline Phan.

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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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Phan, C., Beauchet, A., Burztejn, AC. et al. Evaluation of Children with Psoriasis from the BiPe Cohort: Are Patients Using Biotherapies in Real Life Eligible for Phase III Clinical Studies?. Pediatr Drugs 21, 169–175 (2019). https://doi.org/10.1007/s40272-019-00335-9

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  • DOI: https://doi.org/10.1007/s40272-019-00335-9

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