Proposed Response Parameters for Twelve-Month Drug Trial in Juvenile Systemic Sclerosis: Results of the Hamburg International Consensus Meetings.

Autor: Foeldvari I; Schön Klinik Hamburg Eilbek, Hamburg, Germany., Torok KS; University of Pittsburgh and Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania., Anton J; Hospital Sant Joan de Déu and Universitat de Barcelona, Barcelona, Spain., Blakley M; Indiana University School of Medicine and Riley Hospital for Children at IU Health, Indianapolis., Constantin T; Semmelweis University, Budapest, Hungary., Curran M; University of Colorado School of Medicine and Children's Hospital Colorado, Aurora., Cutolo M; University of Genoa and IRCCS San Martino Polyclinic Hospital, Genoa, Italy., Denton C; Royal Free London NHS Foundation Trust, London, UK., Fligelstone K; Scleroderma & Raynaud's United Kingdom, London, UK., Ingegnoli F; University of Milan, ASST G. Pini, Milan, Italy., Li SC; Hackensack University Medical Center, Hackensack, New Jersey., Němcová D; Charles University, Prague, Czech Republic., Orteu C; Royal Free London, London, UK., Pilkington C; Great Ormond Street Hospital, London, UK., Smith V; Ghent University, Ghent University Hospital, VIB Inflammation Research Center, and ERN ReCONNET, Ghent, Belgium., Stevens A; Children's Hospital Research Institute and University of Washington, Seattle, and Janssen Pharmaceutical Companies of Johnson & Johnson, Spring House, Pennsylvania., Klotsche J; German Rheumatism Research Center, Berlin, Germany., Khanna D; University of Michigan, Ann Arbor., Costa-Reis P; Hospital de Santa Maria, Faculdade de Medicina, and Universidade de Lisboa, Lisbon, Portugal., Del Galdo F; University of Leeds and Leeds Teaching Hospital Trust, Leeds, UK., Hinrichs B; Asklepios Klinik Nord-Heidberg, Hamburg, Germany., Kasapcopur O; Cerrahpasa Medical School and Istanbul University-Cerrahpasa, Istanbul, Turkey., Pain C; Alder Hey Children's Foundation NHS Trust, Liverpool, UK., Ruperto N; IRCCSG Istituto G. Gaslini, Genoa, Italy., Zheng A; Chinese Organization for Scleroderma, Chengdu City, Sichuan Province, China., Furst DE; University of California, Los Angeles, University of Washington, Seattle, and University of Florence, Florence, Italy.
Jazyk: angličtina
Zdroj: Arthritis care & research [Arthritis Care Res (Hoboken)] 2023 Dec; Vol. 75 (12), pp. 2453-2462. Date of Electronic Publication: 2023 Sep 12.
DOI: 10.1002/acr.25171
Abstrakt: 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.
(© 2023 American College of Rheumatology.)
Databáze: MEDLINE
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