Analysis on clinical features and treatment outcomes of anti-MDA5 antibody-positive juvenile dermatomyositis
ZHANG Junmei
XUE Yuan
KUANG Weiying
LI Chao
DENG Jianghong
TAN Xiaohua
LI Shipeng
LI Caifeng
Abstract：Objective To analyze the clinical features and treatment outcomes of anti-melanoma differentiation-associated gene 5(MDA5)antibody-positive juvenile dermatomyositis(JDM).Methods The clinical data of 487 pediatric patients with JDM who were admitted to the Department of Rheumatology of Beijing Children's Hospital,Capital Medical University from June 2015 to February 2022 were collected,and among the pediatric patients,41 cases(8.42％)were positive for anti-MDA5 antibody.The clinical characteristics,disease evaluation,therapeutic drugs and disease outcome of the pediatric patients were summarized and analyzed.Results In the 41 cases of JDM with positive anti-MDA5 antibody,the most common clinical manifestation was abnormal skin and mucous membrane,followed by muscle weakness,and fever and arthritis were also common,and some pediatric patients had respiratory symptoms and skin ulcers.The majority of Cutaneous Assessment Tool(CAT)scores were 3 to 5 points.The scores of Childhood Myositis Assessment Scale(CMAS)were(33.88±9.57)points.The incidence rate of interstitial lung disease(ILD)was 87.80％,and 12.20％of the pediatric patients developed rapid progressive ILD(RP-ILD).Subcutaneous calcification occurred in 5 cases(12.20％)during the course of the disease.There were 90.24％of the anti-MDA5 antibody-positive JDM patients had high-risk physical signs.Intravenous methylprednisolone(IVMP)(63.41％),intravenous immunogloblin(IVIG)(68.29％)and oral cyclosporine A(65.85％)were the main treatment methods.Nine pediatric patients(21.95％)were orally administered tofacitinib.There were 75.61％of the pediatric patients presenting with a single course of the disease,and 19.51％with recurrent courses,and 4.88％with persistent active courses.The complete clinical response rates at 1,2,3 and 4 years were 65.79％(25/38),65.38％(17/26),63.64％(14/22)and 70.59％(12/17),respectively.The five-year clinical complete remission rate was 14.29％(2/14),and the two-year disease recurrence rate was 19.23％(5/26).During the 2-year follow-up,95.00％(19/20)of the pediatric patients'lung lesions were completely absorbed.At 3,6,9 and 12 months of follow-up,the median dose of prednisone was 1.40 mg/kg,1.00 mg/kg,0.71 mg/kg and 0.60 mg/kg,respectively,and the median duration of glucocorticoid discontinuation was 69 months.Until February 2022,glucocorticoids were discontinued in 15 of 41 cases(36.59％)and all drugs were discontinued in 9 cases(21.95％).Conclusion Anti-MDA5 antibody-positive JDM is a special type of JDM,which mainly presents with characteristic rash,muscle weakness,fever and arthritis,and ILD is the main hazard.The complete clinical remission rate of the disease is still low,and the recurrence rate is relatively high.
Keywords：Anti-melanoma differentiation-associated gene 5(MDA5)antibodyJuvenile dermatomyositis(JDM)Clinical featureTreatmentFollow-up
Publication Date：2024-09-28
Online Publishing Date：2025-08-15(First online date of this platform, not the publication date of the document)
Pages：6( 961-966 )