Congratulations to Kathryn S. Torok, MD, on her June publication, “Proposed response parameters for 12 months drug trial in Juvenile Systemic Sclerosis. Results of the Hamburg International Consensus Meetings,” in Arthritis Care and Research. Torok is an Associate Professor of Pediatrics and Clinical and Translational Science in the Division of Rheumatology. 
Co-written with Ivan Foeldvari, MD, of the Hamburg Centre for Pediatric and Adolescent Rheumatology, among other contributors, this research focuses on proposing clearly defined standards for appropriate outcomes of new treatment strategies for juvenile systemic sclerosis (jSSc). This need for clearly defined outcomes of treatment for jSSc coincides with an increased need for new treatments of the disease, as it is often associated with high morbidity and mortality.
In their article, Torok, et al. propose the agreed upon results of four face-to-face meetings of a 27-member multidisciplinary team of pediatric rheumatologists, adult rheumatologists, dermatologists, pediatric cardiologists, pulmonologists, gastroenterologists, statisticians and patients. Throughout the process, the group reviewed existing adult data, the limited pediatric research on jSSc outcomes, and current data from two jSSc cohorts.
The proposed and agreed upon results of the group, noted in the publication, designated a series of domains with which to evaluate jSSc treatment outcomes: global disease activity, skin, Raynaud's phenomenon, digital ulcers, musculoskeletal, cardiac, pulmonary, renal, gastrointestinal, and quality of life.
The article then concludes that the domains should be subsequently assessed in an 12-month clinical jSSc trial and ongoing research agenda item.
The ultimate goal is to set up a standardized system to evaluate the success of much-needed new treatments for jSSc as they are developed. This proposal and efforts will ensure greater consistency and evaluation in care for pediatric patients across international boundaries and care systems.
Follow PItt Pediatrics on Twitter for further updates on Torok’s clinical and translational research and forthcoming publications over the coming weeks.