Background/Purpose: A national registry was set up, enrolling new onset JDM cases in 18 hospitals, during 3-years (2015-2018) with 2-years follow up, in a low resource country aiming at first evaluating clinical presentation and disease activity scores by DAS-20 Disease Activity Scoring tool over 2-years-follow up.

Methods: Questionnaires inquiring cases, within 6 months of the first symptoms, were filled out by participating physicians with clinical and functional assessments at baseline , 6, 12, 18 and 24 months. Data capture, storage and analysis were carried in one of centres. Protocol ethics approval and informed consent was obtained for all. Disease Activity Score (DAS20) tool training was provided (Bode RK et al. Arthritis Rheum 2003, 49:7-15). Investigations included: muscle enzymes, full blood count, ESR, renal function, MRI, EMG, and muscle biopsy, calcinosis image, overlap features and auto-antibodies.

Results: Ninety six cases were enrolled from 18 referral hospitals, 60 towns (zip code) in the five geographic regions of a continental country (207.75 million population). Of those cases, 61 (64%) were female with mean (SD) age 10.7(4.2) years. Presenting signs and symptoms frequency was: face and extremities rash (97%), generalized muscle weakness (95%), fatigue (88%), myalgia (87%), arthralgia (61%), fever (51%), irritability (41%), facial oedema (47%), body oedema (27%) , weight loss (37%), joint oedema (37%), stiffness (37%) , dysphagia (40%), dysphonia (27%), dyspnoea (20%), abdominal pain (24%), chest pain (6%). Onset calcinosis was described in 12 of 91 reports (13%). Only 42 cases performed EMG and 32 had a muscle biopsy and MRI was performed in 22 and US in 7. The muscle enzymes values mean (SD) IU was: CK 1835 (3822), LDH 972 (801), AST 204 (622), ALT 118 (208), Aldolase 20 (25,9). Treatment received, included: 81/90 (90%) high dose oral prednisone, 46/90 (51%) IV methylprednisolone, 72/90 (80%), methotrexate, 40/91 (44%) Hydroxycloroquine, 15/91 (16%) IVIG, 3/91 (3%) cyclosporine, 3/90 (3%) azathioprine, 4/90 (4%) IV cyclophosphamide and 1/90 (1%) received rituximab. Disease activity status scored by DAS20 at baseline and follow up indicated wide variation and significant improvement over 2-years. The mean± SD scores of DAS20 were: baseline n=96 (8±5), n=96 (6m) (2.7±4.4), n=96 (12m) (1.5±3.2), n=73 (18m) (1±3) and n=96 (24m) (0.5±2.3). Values compared by Poisson model (p 0.0001) and Wald post-test indicated significant difference between each of the visits, from baseline to 24 months.

Conclusion: This preliminary analysis of a registry included case ascertainment and feasibility of standardized outcome measures for JDM (DAS20), under mainstay treatment with prednisone and methotrexate. It indicated a good performance of clinical assessment by a quantitative tool, performed by physicians in low resource settings, in spite of limited access to all diagnostic pathway tools, imaging and biomarkers.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/the-brazilian-registry-of-juvenile-dermatomyositis-jdm-i-onset-clinical-features-and-disease-activity-scores-by-das-20-over-2-years-follow-up/