The Brazilian Registry of Juvenile Dermatomyositis (JDM): I- Onset Clinical Features and Disease Activity Scores by DAS-20 over 2-Years-Follow Up

Beatriz Carneiro¹, Adriana Elias¹, Teresa Robazzi², Ana Julia Moraes³, Sheila Oliveira⁴, Flavio Sztajnbok⁵, Luciana Carvalho⁶, Luciana Marques⁷, Silvana Sacchetti⁸, Maria Teresa Terreri⁹, Simone Appenzelle¹⁰, Roberto Marini¹¹, Andre Cavalcante¹², Marcia Bandeira¹³, Cristina Magalhaes¹⁴, Melissa Fraga¹⁵, Iloite Scheibel¹⁶, Isabela Daud¹, Darcisio Antonio¹⁷, Claudio Len¹⁸, Clovis Silva¹⁹, Taciana Fernandes¹⁷ and Claudia Magalhaes²⁰, ¹Instituto da Criança - Universidade de São Paulo (USP), São Paulo, Brazil, ²Universidade Federal da Bahia, Brazil, ³Universidade Federal do Para, Brazil, ⁴Universidade Federal do Rio de janeiro, Rio de Janeiro, ⁵Universidade Estadual do Rio de Janeiro, Rio de Janeiro, Brazil, ⁶Universidade de Sao Paulo- Ribeirao Preto, São Paulo, Brazil, ⁷Hospital Albert Sabin, Brazil, ⁸Santa Casa de Sao Paulo, Brazil, ⁹UNIFESP, São Paulo, Brazil, ¹⁰University of Campinas, Campinas, Sao Paulo, Brazil, ¹¹UNICAMP, São Paulo, Brazil, ¹²Hospital Materno-Infantil de Goiania, Brazil, ¹³Hospital Pequeno Principe- Curitiba, Brazil, ¹⁴Hospital Jose de Alencar - Brasilia, Brazil, ¹⁵Hospital Darcy Vargas, Brazil, ¹⁶Hospital Conceição de Porto Alegre, Brazil, ¹⁷Universidade Estadual Paulista (UNESP) Botucatu, Brazil, ¹⁸Universidade Federal de São Paulo - Unifesp, São Paulo, Brazil, ¹⁹Universidade de São Paulo, São Paulo, Brazil, ²⁰São Paulo State University, Pediatric Rheumatology Division, Botucatu, Brazil

Meeting: 2023 Pediatric Rheumatology Symposium

Keywords: dermatomyositis, Myopathies, Myositis, Outcome measures, registry

Session Information

Date: Friday, March 31, 2023

Title: Posters: Clinical and Therapeutic II

Session Type: Poster Session B

Session Time: 5:00PM-6:00PM

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.

Disclosures: B. Carneiro: None; A. Elias: None; T. Robazzi: None; A. Moraes: None; S. Oliveira: None; F. Sztajnbok: None; L. Carvalho: None; L. Marques: None; S. Sacchetti: None; M. Terreri: None; S. Appenzelle: None; R. Marini: None; A. Cavalcante: None; M. Bandeira: None; C. Magalhaes: None; M. Fraga: None; I. Scheibel: None; I. Daud: None; D. Antonio: None; C. Len: None; C. Silva: None; T. Fernandes: None; C. Magalhaes: None.

To cite this abstract in AMA style:

Carneiro B, Elias A, Robazzi T, Moraes A, Oliveira S, Sztajnbok F, Carvalho L, Marques L, Sacchetti S, Terreri M, Appenzelle S, Marini R, Cavalcante A, Bandeira M, Magalhaes C, Fraga M, Scheibel I, Daud I, Antonio D, Len C, Silva C, Fernandes T, Magalhaes C. The Brazilian Registry of Juvenile Dermatomyositis (JDM): I- Onset Clinical Features and Disease Activity Scores by DAS-20 over 2-Years-Follow Up [abstract]. Arthritis Rheumatol. 2023; 75 (suppl 4). 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/. Accessed .

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