戈利木单抗改善难治性幼年型皮肌炎患儿肌力的疗效分析及文献复习

目的:探讨难治性幼年型皮肌炎(JDM)患儿应用戈利木单抗(GLM)的临床治疗效果。方法:收集2019年2月广州市妇女儿童医疗中心过敏免疫风湿科确诊的1例JDM患儿的临床资料，分析总结治疗效果并进行文献复习。结果:患儿，男，7岁，亚急性起病，病情迁延，以皮疹、肢体乏力和吞咽功能障碍为主要表现，查体见向阳疹及Gottron...

Curative effect analysis and literature review on the efficacy of Golimumab in improving the muscle strength of children with refractory juvenile dermatomyositis

Objective To investigate the therapeutic efficacy of Golimumab in the treatment of children with refractory juvenile dermatomyositis(JDM).Methods The clinical data of a child diagnosed with JDM in the Department of Allergy,Immunology and Rheumatology of Guangzhou Women and Children′s Medical Center in February 2019 were collected.The treatment effect was studied and literature review was conducted.Results The patient was a 7-year-old boy with subacute onset of the disease.The illness protracted,and main manifestations included skin rashes,limb weakness,and swallowing dysfunction.Physical examination showed heliotropic rashes,Gottron papules,positive Gower,proximal limb muscle strength gradeⅢ-Ⅳ,distal limb muscle strength gradeⅣ,and a choking cough when swallowing fluid food.Laboratory tests revealed alanine aminotransferase(ALT)of 36 U/L,aspartate aminotransferase(AST)of 115 U/L,alkaline phosphatase of 69 U/L,lactate dehydrogenase of 941 U/L,creatine kinase of 974 U/L,hypersensitive C-reactive protein of 26 mg/L and an erythrocyte sedimentation rate(ESR)of 52 mm/1 h.Antinuclear antibody spectra were negative.Electromyography suggested myogenic damage.Thigh magnetic resonance imaging indicated diffuse abnormal signal shadows in the subcutaneous fat,muscles and muscle spaces of both hips,thighs and knee joints.The child was diagnosed with JDM,and given standardized treatment of Methylprednisolone,intravenous immunoglobulin,Methotrexate and Hydroxychloroquine sulfate.However,after the treatment,the facial rashes were still red,proximal limb muscle strength and swallowing dysfunction did not improve,the choking cough symptom still existed,and a Cushing face appeared.Recheck results showed ALT of 24 U/L,AST of 32 U/L,alkaline phosphatase of 56 U/L,lactate dehydrogenase of 216 U/L,creatine kinase of 527 U/L,hypersensitive C-reactive protein of 8 mg/L and an ESR of 15 mm/1 h.Refractory JDM was considered.After negotiating with the patient′s family members,they agreed to treat the patient with Golimumab 50 mg by subcutaneous injection once a month.Then tapered prednisone gradually,stopped Hydroxychloroquine sulfate tablets and continued to give the patient oral Methotrexate.After two doses of Golimumab 50 mg,proximal limb muscle strength and swallowing function improved markedly.After the third subcutaneous injection of Golimumab,proximal limb muscle strength improved to gradeⅣ-Ⅴ,and he was able to go up and down stairs,squat and stand up after squatting.Besides,dysphagia and the choking cough disappeared,and skin rashes improved.Recheck results suggested a normal ESR and creatine kinase levels.Magnetic resonance imaging of thighs indicated no muscle inflammation.Conclusions Golimumab works well in the treatment of refractory JDM and can effectively improve muscle strength.Therefore,it can be used as a treatment option for refractory JDM.