Erdal Sag1, Selcan Demir1, Yelda Bilginer1, Beril Talim2, Goknur Haliloglu3, Haluk Topaloglu3, Seza Ozen4. 1. Division of Pediatric Rheumatology, Department of Pediatrics, Hacettepe University, Ankara 06100, Turkey. 2. Pediatric Pathology Unit, Department of Pediatrics, Hacettepe University, Ankara, Turkey. 3. Division of Pediatric Neurology, Department of Pediatrics, Hacettepe University, Ankara, Turkey. 4. Division of Pediatric Rheumatology, Department of Pediatrics, Hacettepe University, Ankara 06100, Turkey. Electronic address: sezaozen@hacettepe.edu.tr.
Abstract
INTRODUCTION: Juvenile dermatomyositis (JDM) is the most common inflammatory myopathy of childhood. OBJECTIVE: To analyze clinical features, paraclinical examinations, MSAs, treatment response and long-term outcome in a JDM cohort METHODS: 58 patients (35F, 23 M) from a tertiary referral center in the last two decades are included. RESULTS: Mean age at onset was 8.1 ± 4.3, with a mean follow-up period of 5.66±3.59 years. Dermatological manifestations (91%) and muscle weakness (76%) were the key diagnostic elements. Elevated serum creatine kinase levels (86%), electromyography (23/25), muscle MRI (12/15), and muscle biopsy (n = 35) were compatible with the diagnosis. Out of 46 patients tested, 34 (76%) had autoantibodies, with NXP2 (21.7%), followed by TIF1g (17.4%), MDA5 (8.7%), and Mi-2 (8.7%). Presence of TIF1g and NXP2 indicated a severe course; and Ku a much severe course compared to previous studies. Corticosteroids (100%) combined with methotrexate (93%) was the initial treatment. Biological disease modifying anti-rheumatic drugs (DMARDs) were used in 22% of the cohort. Calcinosis (36%) was the most common long-term complication, associated with disease onset ≤6 years, higher muscle biopsy scores and MDA5 positivity. Complete remission was achieved in 65.5% of the patients in a median 24 (IQR 11.8-42.5) months with a relapse rate of 26.3%. 43.9% of NXP2 and 33.3% of TIF-1 g positive patients had a relapse. Course was monophasic (31%), polyphasic (17.2%), chronic (51.8%) without mortality. CONCLUSION: Integration of clinical features with laboratory and biopsy findings may help to predict prognosis and guide treatment in JDM. In our cohort calcinosis was associated with age, MDA5 autoantibodies, and muscle biopsy scores.
INTRODUCTION:Juvenile dermatomyositis (JDM) is the most common inflammatory myopathy of childhood. OBJECTIVE: To analyze clinical features, paraclinical examinations, MSAs, treatment response and long-term outcome in a JDM cohort METHODS: 58 patients (35F, 23 M) from a tertiary referral center in the last two decades are included. RESULTS: Mean age at onset was 8.1 ± 4.3, with a mean follow-up period of 5.66±3.59 years. Dermatological manifestations (91%) and muscle weakness (76%) were the key diagnostic elements. Elevated serum creatine kinase levels (86%), electromyography (23/25), muscle MRI (12/15), and muscle biopsy (n = 35) were compatible with the diagnosis. Out of 46 patients tested, 34 (76%) had autoantibodies, with NXP2 (21.7%), followed by TIF1g (17.4%), MDA5 (8.7%), and Mi-2 (8.7%). Presence of TIF1g and NXP2 indicated a severe course; and Ku a much severe course compared to previous studies. Corticosteroids (100%) combined with methotrexate (93%) was the initial treatment. Biological disease modifying anti-rheumatic drugs (DMARDs) were used in 22% of the cohort. Calcinosis (36%) was the most common long-term complication, associated with disease onset ≤6 years, higher muscle biopsy scores and MDA5 positivity. Complete remission was achieved in 65.5% of the patients in a median 24 (IQR 11.8-42.5) months with a relapse rate of 26.3%. 43.9% of NXP2 and 33.3% of TIF-1 g positive patients had a relapse. Course was monophasic (31%), polyphasic (17.2%), chronic (51.8%) without mortality. CONCLUSION: Integration of clinical features with laboratory and biopsy findings may help to predict prognosis and guide treatment in JDM. In our cohort calcinosis was associated with age, MDA5 autoantibodies, and muscle biopsy scores.