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Proposed Response Parameters for Twelve-Month Drug Trial in Juvenile Systemic Sclerosis: Results of the Hamburg International Consensus Meetings
I. Foeldvari, KS. Torok, J. Anton, M. Blakley, T. Constantin, M. Curran, M. Cutolo, C. Denton, K. Fligelstone, F. Ingegnoli, SC. Li, D. Němcová, C. Orteu, C. Pilkington, V. Smith, A. Stevens, J. Klotsche, D. Khanna, P. Costa-Reis, F. Del Galdo,...
Jazyk angličtina Země Spojené státy americké
Typ dokumentu časopisecké články, přehledy
PubMed
37332054
DOI
10.1002/acr.25171
Knihovny.cz E-zdroje
- MeSH
- dítě MeSH
- dospělí MeSH
- konsensus MeSH
- kvalita života MeSH
- lidé MeSH
- Raynaudova nemoc * farmakoterapie MeSH
- systémová sklerodermie * diagnóza farmakoterapie komplikace MeSH
- Check Tag
- dítě MeSH
- dospělí MeSH
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- přehledy MeSH
OBJECTIVE: Juvenile systemic sclerosis (SSc) is an orphan disease, associated with high morbidity and mortality. New treatment strategies are much needed, but clearly defining appropriate outcomes is necessary if successful therapies are to be developed. Our objective here was to propose such outcomes. METHODS: This proposal is the result of 4 face-to-face consensus meetings with a 27-member multidisciplinary team of pediatric rheumatologists, adult rheumatologists, dermatologists, pediatric cardiologists, pulmonologists, gastroenterologists, a statistician, and patients. Throughout the process, we reviewed the existing adult data in this field, the more limited pediatric literature for juvenile SSc outcomes, and data from 2 juvenile SSc patient cohorts to assist in making informed, data-driven decisions. The use of items for each domain as an outcome measure in an open label 12-month clinical trial of juvenile SSc was voted and agreed upon using a nominal group technique. RESULTS: After voting, the domains agreed on were global disease activity, skin, Raynaud's phenomenon, digital ulcers, musculoskeletal, cardiac, pulmonary, renal, and gastrointestinal involvement, and quality of life. Fourteen outcome measures had 100% agreement, 1 item had 91% agreement, and 1 item had 86% agreement. The domains of biomarkers and growth/development were moved to the research agenda. CONCLUSION: We reached consensus on multiple domains and items that should be assessed in an open label, 12-month clinical juvenile SSc trial as well as a research agenda for future development.
Alder Hey Children's Foundation NHS Trust Liverpool UK
Asklepios Klinik Nord Heidberg Hamburg Germany
Cerrahpasa Medical School and Istanbul University Cerrahpasa Istanbul Turkey
Charles University Prague Czech Republic
Chinese Organization for Scleroderma Chengdu City Sichuan Province China
German Rheumatism Research Center Berlin Germany
Great Ormond Street Hospital London UK
Hackensack University Medical Center Hackensack New Jersey
Hospital de Santa Maria Faculdade de Medicina and Universidade de Lisboa Lisbon Portugal
Hospital Sant Joan de Déu and Universitat de Barcelona Barcelona Spain
Indiana University School of Medicine and Riley Hospital for Children at IU Health Indianapolis
IRCCSG Istituto G Gaslini Genoa Italy
Royal Free London NHS Foundation Trust London UK
Schön Klinik Hamburg Eilbek Hamburg Germany
Scleroderma and Raynaud's United Kingdom London UK
Semmelweis University Budapest Hungary
University of Colorado School of Medicine and Children's Hospital Colorado Aurora
University of Genoa and IRCCS San Martino Polyclinic Hospital Genoa Italy
University of Leeds and Leeds Teaching Hospital Trust Leeds UK
University of Michigan Ann Arbor
University of Milan ASST G Pini Milan Italy
University of Pittsburgh and Children's Hospital of Pittsburgh Pittsburgh Pennsylvania
Citace poskytuje Crossref.org
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