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

Children's Hospital Research Institute and University of Washington Seattle and Janssen Pharmaceutical Companies of Johnson and Johnson Spring House Pennsylvania

Chinese Organization for Scleroderma Chengdu City Sichuan Province China

German Rheumatism Research Center Berlin Germany

Ghent University Ghent University Hospital VIB Inflammation Research Center and ERN ReCONNET Ghent Belgium

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 London UK

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 California Los Angeles University of Washington Seattle and University of Florence Florence Italy

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

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