Identifying Current Assessment and Treatment Strategies in Juvenile Systemic Sclerosis: An International Effort to Improve Patient Outcomes

Project Period:

Grant Category:
Small Grant

Disease Area:
Scleroderma, SVARD

Juvenile Systemic Sclerosis (jSSc) is an inflammatory and scaring disease that affects the skin and multiple organs. More than 1/3 of children develop disability and have a substantial mortality risk. Studies are needed to identify the most effective therapies, but the rarity of jSSc makes conducting such studies in a single country difficult. Standard tools and definitions are needed to evaluate treatment response and define if patients have improved on or failed a given therapy. This study aims to develop tools and standards needed for trials by surveying pediatric rheumatologists in multiple countries to identify currently used assessments, treatments, and definitions. A group of jSSc experts will review and discuss this information in comparison to best available evidence from the literature to come to agreement on what standards are feasible and effective to use in future international treatment trials, to be able to work towards identifying the most effective treatments.


Natalia Vasquez-Canizares, MD

Montefiore Medical Center