Céline Phan1, Alain Beauchet2, Anne-Claire Burztejn3, Maëlla Severino-Freire4, Juliette Mazereeuw-Hautier4, Sébastien Barbarot5, Céline Girard6, Audrey Lasek7, Ziad Reguiai8, Claire Abasq9, Bruno Sassolas9, Catherine Droitcourt10, Marc Perrussel10, Smail Hadj-Rabia11, Stéphanie Mallet12, Alice Phan13, Jean-Philippe Lacour14, Emmanuelle Bourrat15, François Aubin16, Emmanuel Mahé17. 1. Service de Dermatologie, Hôpital Victor Dupouy, 69 rue du Lieutenant-Colonel Prud'hon, 95100, Argenteuil, France. celine.phan@ch-argenteuil.fr. 2. Département de Santé Publique, Centre Hospitalier Universitaire Ambroise Paré, Assistance Publique-Hôpitaux de Paris, Boulogne-Billancourt, France. 3. Service de Dermatologie, Hôpital Brabois, 54000, Vandoeuvre Les Nancy, France. 4. Service de Dermatologie, Centre Hospitalier Universitaire de Toulouse, Toulouse, France. 5. Service de Dermatologie, Hôtel Dieu, Nantes, France. 6. Service de Dermatologie, Centre Hospitalier Universitaire de Montpellier, Montpellier, France. 7. Service de Dermatologie, Hôpital Saint Vincent de Paul, Université Catholique de Lille, Lille, France. 8. Service de Dermatologie, Polyclinique Courlancy, Hôpital Robert Debré, Reims, France. 9. Service de Dermatologie, Centre Hospitalier Universitaire de Brest, Brest, France. 10. Service de Dermatologie, Centre Hospitalier Universitaire Pontchaillou, Université de Rennes, Rennes, France. 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. 12. Service de Dermatologie, Vénéréologie et Cancérologie Cutanée, Hôpital de la Timone, Assistance-publique-Hôpitaux de Marseille, Marseille, France. 13. Service de Pédiatrie, Hôpital Femme-Mère-Enfant, Hospices Civils de Lyon, Bron, France. 14. Service de Dermatologie, Hôpital Archet 2, ESPIC CHU-Lenval, Nice, France. 15. Service de Pédiatrie Générale, Hôpital Robert Debré, Assistance Publique-Hôpitaux de Paris, Paris, France. 16. Service de Dermatologie, Centre Hospitalier Régional Universitaire de Besançon, Université de Franche Comté, Besançon, France. 17. Service de Dermatologie, Hôpital Victor Dupouy, 69 rue du Lieutenant-Colonel Prud'hon, 95100, Argenteuil, France.
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.
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 childrenpatients 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.