Proposed response parameters for 12 months drug trial in Juvenile Systemic Sclerosis. Results of the Hamburg International Consensus Meetings

Objectives:Juvenile systemic sclerosis (jSSc) is an orphan disease, associated with high morbidity and mortality. New treatment strategies are much needed, but it is necessary to clearly define appropriate outcomes if successful therapies are to be developed. Here, such outcomes are proposed.Methods:This proposal is the result of four face to face consensus meetings with a 27-member multidisciplinary teamof pediatric rheumatologists, adult rheumatologists, dermatologists, pediatric cardiologists, pulmonologists, gastroenterologists, statistician and patients. Throughout the process, we reviewed the existing adult data in this field, the more limited pediatric literature for jSSc outcomes and data from two jSSc patient cohorts to assist in making informed, data-driven decisions. The use of items for each domain as an outcome measure in an open 12-month clinical trial of jSSc was voted and agreed upon using a nominal group technique. Results:After voting, the agreed domains were: global disease activity, skin, Raynaud’s phenomenon, digital ulcers, musculoskeletal, cardiac, pulmonary, renal, gastrointestinal, and quality of life. Fourteen outcome measures had 100% agreement, one item had 91% agreement and one item had 86% agreement. The domains of biomarker and growth/development were moved to the research agenda.Conclusion:We reached consensus on multiple domains and items which should be assessed in an open label 12-monthclinical jSSc trial as well as a research agenda for future development.