AB0984 Development of a Juvenile Systemic Sclerosis Response Index (JSSRI)

BackgroundJuvenile systemic sclerosis (jSSc) is an orphan disease. We have currently new promising effective medication to treat systemic sclerosis, but no valid outcome measures to assess the activity of the disease.ObjectivesIn the frame of the juvenile scleroderma working group of Paediatric Rheumatology European Society (PRES JSWG), we developed a provisional response index for jSSc.MethodsThe facilitating committee designed a 3-round Delphi-exercise to obtain a core set of disease activity measures in jSSc. In Round 1, they were asked to add unique items in 21 preleminary domains. Round 2 judged the items' applicability and importance using a 1-10 scale. Previous respondents were asked to re-rate uncertain (score 4-7) items in Round 3. The aforementioned were followed by a face-to-face Nominal Group discussion to decide final items and domain structure.Results52 participants of Round 1 suggested a total of 131 items for the 21 domains. In round 2, 56 responders rated the significance of the items. The Nominal Group accepted 11 main domains for further validation containing 41 items (see table 1).Table 1Global disease activity GDA according to physician and according to parent by VASBiomarkers ESR, CRP, HemoglobinRaynaud phenomenon and Digital ulcers Raynaud activity score, Raynaud severity, Number of ulcers, New ulcers, Digital ulcer severitySkin mRSS, Newly occurred calcinosis, Newly occurred teleangiectasia, Presence of scleroderma-related edemaPulmonary FVC, DLCO, 6 minute walk test, Borg indexCardiac Left Ventricular and Right Ventricular ejection fraction, Newly occurred carditis, Newly occurred arrythmia, Pro-BNP level New onset or worsening of PAHGastrointestinal New onset of swallowing difficulties, Change in stool frequency, New sign of refluxRenal Newly occurred hypertension, Newly occurred renal crisis, Proteinuria, Creatinine clearence, Change in GFRHealth related quality of life and function PEDSQL, CHAQ Pain VAS, Fatigue VASEssential paediatric data to collect: Weight, Height, Growth velocity, Tanner stage.ConclusionsWe reached consensus and developed a Response Index, which should help to conduct clinical trials in jSSc. The validation of the JSSRI is planned in the frame of the Juvenile Scleroderma Inception Cohort.Disclosure of InterestNone declared