دورية أكاديمية
أعرض في EDS

Pozelimab for CHAPLE disease: results from in-trial interviews and clinical outcome assessments.

التفاصيل البيبلوغرافية
العنوان: Pozelimab for CHAPLE disease: results from in-trial interviews and clinical outcome assessments.
المؤلفون: Litcher-Kelly L; Adelphi Values, Boston, MA, USA., Ozen A; Marmara University, Istanbul, Türkiye., Ollis S; Adelphi Values, Boston, MA, USA., Feldman HB; Tel Aviv Sourasky Medical Center and Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel., Yaworsky A; Adelphi Values, Boston, MA, USA., Medrano P; Adelphi Values, Boston, MA, USA., Chongsrisawa V; Department of Pediatrics, Faculty of Medicine, Chulalongkorn University, King Chulalongkorn Memorial Hospital, Thai Red Cross Society, Patumwan, Bangkok, Thailand., Brackin T; Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY, 10591, USA., Perlee L; Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY, 10591, USA., Walker M; Adelphi Values, Boston, MA, USA., Pradeep S; Adelphi Values, Boston, MA, USA., Lenardo MJ; Molecular Development of the Immune System Section, Laboratory of Immune System Biology, Laboratory of Clinical Immunology and Microbiology, and Clinical Genomics Program, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA., Harari OA; Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY, 10591, USA., Jalbert JJ; Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY, 10591, USA. jessica.jalbert@regeneron.com.
المصدر: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2024 Aug 08; Vol. 19 (1), pp. 290. Date of Electronic Publication: 2024 Aug 08.
نوع المنشور: Journal Article
اللغة: English
بيانات الدورية: Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE
أسماء مطبوعة: Original Publication: [London] : BioMed Central, 2006-
مواضيع طبية MeSH: Antibodies, Monoclonal, Humanized*/therapeutic use, Humans ; Female ; Male ; Adolescent ; Child, Preschool ; Child ; Young Adult ; Adult ; Outcome Assessment, Health Care
مستخلص: Background: CD55 deficiency with hyper-activation of complement, angiopathic thrombosis, and protein-losing enteropathy (CHAPLE) disease is ultra-rare (< 100 children or young adults worldwide) and potentially fatal. The study used mixed-methods approaches to assess how pozelimab impacts the signs and symptoms of CHAPLE disease from the patient perspective by combining within-trial interviews and clinical outcome assessments (COAs) (ClinicalTrials.gov, NCT04209634).
Methods: Interviews conducted with patients/caregivers at screening and week 24 assessed the signs and symptoms of CHAPLE disease, including those which were most bothersome, and evaluated the change. Patients/caregivers and clinicians completed the COAs; interview data contextualized the meaningfulness of change.
Results: Ten patients (aged 3-19 years) were enrolled; caregivers contributed to nine interviews. Abdominal pain, diarrhea, facial and peripheral edema, nausea, and vomiting are the core signs and symptoms of CHAPLE disease (≥ 90% patients experienced pre-treatment); the most bothersome signs and symptoms were abdominal pain (n = 9) and facial edema (n = 1). All core signs and symptoms were reported as resolved at week 24 interviews. Severity on global assessments changed from "mild" to "very severe" at baseline to "no signs or symptoms" at week 24. Interview results were generally consistent with sign- or symptom-specific COA scores.
Conclusions: Patients with CHAPLE disease treated with pozelimab for 24 weeks experienced complete resolution of core signs and symptoms. Mixed-methods approaches can contextualize the patient experience (how patients feel and function) in rare disease trials.
Trial Registration: Clinicaltrials.gov, NCT04209634, registered December 24, 2019, https://classic.
Clinicaltrials: gov/ct2/show/NCT04209634 .
(© 2024. The Author(s).)
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معلومات مُعتمدة: N/A Regeneron Pharmaceuticals; N/A Division of Intramural Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health
فهرسة مساهمة: Keywords: (3–10): CHAPLE disease; COA; Open-label trial; Pozelimab; Within-trial interviews
سلسلة جزيئية: ClinicalTrials.gov NCT04209634
المشرفين على المادة: 0 (Antibodies, Monoclonal, Humanized)
تواريخ الأحداث: Date Created: 20240808 Date Completed: 20240808 Latest Revision: 20240810
رمز التحديث: 20240812
مُعرف محوري في PubMed: PMC11308501
DOI: 10.1186/s13023-024-03277-9
PMID: 39118150
قاعدة البيانات: MEDLINE
الوصف
تدمد:1750-1172
DOI:10.1186/s13023-024-03277-9