Pos0988-hpr sensitivity to change of the scleroid compared to the ucla git score in a cohort of ssc patients with lower gi symptoms

Background Systemic sclerosis (SSc) is a multi-organ disease and patient-reported outcome measures (PROMs) are important to better understand the complexity and impact of the disease. This heterogeneous disease has a wide specter of symptoms, and there are several different PROMs in use for this disease-group to assess all possible symptoms. Recently, a SSc-specific patient-derived questionnaire The Systemic Sclerosis Impact of Disease (ScleroID) has been developed and validated. Longitudinally, the questionnaire performed better than other comparators with regard to change over time. The ScleroID has so far not been used in a clinical study, nor been compared with the Scleroderma Clinical Trials Consortium Gastrointestinal Scale (UCLA GIT score) 2.0. Objectives: i: To estimate the sensitivity to change in ScleroID with the UCLA GIT score as comparator. ii: To assess association between upper and lower GI in ScleroID and the UCLA GIT score. Methods We assessed the ScleroID and the UCLA GIT score in a 20 weeks randomized clinical trial, including patients with predominantly lower GI symptoms and without severe cardiopulmonary complications. The ScleroID includes questions about the severity of Raynaud`s phenomenon, hand function, upper and lower GI symptoms, life choice and mobility. It is scored using a 10 points Likert scale, with scores ranging from 0 (no impairment) to 10 (extreme impairment). The UCLA GIT score is a seven –item scale asking for frequency of experienced reflux, distention/bloating, diarrhea, fecal soilage, constipation, emotional well-being and social functioning, where all scales are scored from 0 (none of the days) to 3 (5-7 days a week). Changes in the scores from baseline to week 20 were analyzed by Paired- Samples T test. The Standardized response mean (SRM) was computed and interpreted according to Cohen’s effect size index, in which 0.2 refers to a small difference, 0.5 to a moderate difference, and 0.8 or more to a large difference. Correlation were determined by the Spearman correlation coefficient at baseline. Results The study cohort included 67 SSc patients with mean age of 61 years and mean disease duration of 10 years (table 1). After week 20, the ScleroID and UCLA GIT total score showed low sensitivity to change, with SRM 0,42 (95% CI 0,10-0,68) and 0,45 (95%CI 0,18-0,77), respectively (Figure 1). Upper GI score of the ScleroID showed a strong correlation with UCLA reflux (r=0.799, p< 0.001). Lower GI of the ScleroID correlated moderately with UCLA reflux (r=0.399, p=0.008), constipation (r=0.306, p=0.031) and distention/bloating (r=0.444, p<0.001). In total, 54% of the patients reported an improvement in upper GI symptoms and 51% in lower GI symptoms of ScleroID. For the UCLA GIT score 56% reported improvement in reflux, 59% in bloating/distention and 33% in diarrhea. Conclusion The ScleroID and UCLA GIT score showed a low sensitivity to change after 20 week in a lower GI predominant SSc-cohort without severe organ involvement. The majority of the patients reported improvement in upper and lower GI symptoms in both questionnaires. Table 1. Demographics SSc patients (n = 67) Age at assessment, years, mean, (SD ) 61 (11.5) Disease duration at assessment, years, mean (SD ) 10 (7.2) Female sex, n (% ) 62 (93) Limited cutaneous SSc, n (% ) 60 (89) Anti-centromere AB, n (% ) 51 (76) Organ involvement Interstitial lung disease, n (% ) 11 (16) Pulmonary arterial hypertension, n (% ) 1 (1) Gastrointestinal involvement, n (% ) 67 (100) Bloating 44 (66) Diarrhea 23 (34) Treatment Immunosuppresives, n (% ) 13 (19) Figure 1. Disclosure of Interests: Maylen N Carstens: None declared, Torhild Garen: None declared, Henriette Didriksen: None declared, Håvard Fretheim Speakers bureau: Boehringer Ingelheim, Consultant of: Bayer, Grant/research support from: GSK/Actelion, Imon Barua: None declared, Inger-Lise Knutsen: None declared, Tone Karaaslan: None declared, Tove Hatletveit: None declared, Maiju E Pesonen: None declared, Øyvind Midtvedt: None declared, Øyvind Molberg: None declared, Anna-Maria Hoffmann-Vold Speakers bureau: Boehringer Ingelheim, Jannsen, Medscape, Merck Sharp & Dohme and Roche, Consultant of: ARXX, Boehringer Ingelheim, Genentech, Jannsen, Medscape, Merck Sharp & Dohme and Roche, Grant/research support from:? Boehringer Ingelheim, Jannsen.