Analysis of the German compassionate use program on spesolimab in patients with generalized pustular psoriasis: evidence outside of clinical trials

  • Background Generalized pustular psoriasis (GPP) is a potentially life-threatening orphan disease. Interleukin (IL)-36 is a known pathogenetic key driver of GPP. The IL-36 receptor inhibitor spesolimab has shown efficacy and safety in clinical trials. However, evidence for spesolimab outside of clinical trials is limited. Objectives To provide additional evidence for the use of spesolimab beyond clinical trials, we evaluated individual patient data as part of the spesolimab Compassionate Use Program (CUP) for GPP patients in Germany. Materials & Methods Adult patients with an acute GPP flare received 900 mg spesolimab intravenously at baseline and received a second dose on day 8. Data on demographics, efficacy and adverse events were collected from participating sites at baseline, on day 8 and at four weeks. Results The database included datasets from 12 GPP patients. At baseline, 72% of patients with complete data regarding efficacy (n=7) had a GPPGA (GeneralizedBackground Generalized pustular psoriasis (GPP) is a potentially life-threatening orphan disease. Interleukin (IL)-36 is a known pathogenetic key driver of GPP. The IL-36 receptor inhibitor spesolimab has shown efficacy and safety in clinical trials. However, evidence for spesolimab outside of clinical trials is limited. Objectives To provide additional evidence for the use of spesolimab beyond clinical trials, we evaluated individual patient data as part of the spesolimab Compassionate Use Program (CUP) for GPP patients in Germany. Materials & Methods Adult patients with an acute GPP flare received 900 mg spesolimab intravenously at baseline and received a second dose on day 8. Data on demographics, efficacy and adverse events were collected from participating sites at baseline, on day 8 and at four weeks. Results The database included datasets from 12 GPP patients. At baseline, 72% of patients with complete data regarding efficacy (n=7) had a GPPGA (Generalized Pustular Psoriasis Physician Global Assessment) of ≥3, and all patients a PS (pustulation subscore) of ≥3. On day 8, 43% of patients had a GPPGA ≤1 and 72% a PS ≤1. After four weeks, all patients had a GPPGA ≤1 and 86% a PS ≤1. No drug-related adverse events were reported. Conclusion These findings confirm the results of international, randomized clinical trials in a real-world setting. As spesolimab is no longer available in Germany, this study provides important information that cannot be replicated in this country.show moreshow less

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Author:Frederik Krefting, Julia WelzelORCiDGND, Janis ThammORCiD, Karisa ThölkenGND, Johannes Wohlrab, Valentina Laura Müller, Alexander Kreuter, Franziska Rueff, Michael Sticherling, Stefanie Hölsken, Jan-Malte Placke, Wiebke Sondermann
URN:urn:nbn:de:bvb:384-opus4-1191529
Frontdoor URLhttps://opus.bibliothek.uni-augsburg.de/opus4/119152
ISSN:1167-1122OPAC
ISSN:1952-4013OPAC
Parent Title (English):European Journal of Dermatology
Publisher:JLE
Place of publication:Arcueil
Type:Article
Language:English
Year of first Publication:2024
Publishing Institution:Universität Augsburg
Release Date:2025/02/17
Volume:34
Issue:6
First Page:643
Last Page:650
DOI:https://doi.org/10.1684/ejd.2024.4785
Institutes:Medizinische Fakultät
Medizinische Fakultät / Universitätsklinikum
Medizinische Fakultät / Lehrstuhl für Dermatologie
Dewey Decimal Classification:6 Technik, Medizin, angewandte Wissenschaften / 61 Medizin und Gesundheit / 610 Medizin und Gesundheit
Licence (German):CC-BY-NC-ND 4.0: Creative Commons: Namensnennung - Nicht kommerziell - Keine Bearbeitung (mit Print on Demand)