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  

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

作　　者：陈香元 李丰[1] 唐盈 黄怡玲 冯桂萍 曾华松[1] Chen Xiangyuan;Li Feng;Tang Ying;Huang Yiling;Feng Guiping;Zeng Huasong(Department of Allergy,Immunology and Rheumatology,Guangzhou Women and Children′s Medical Center,Affiliated to Guangzhou Medical University,Guangzhou 510623,China)

机构地区：[1]广州医科大学附属广州市妇女儿童医疗中心过敏免疫风湿科,广州510623

出　　处：《中华实用儿科临床杂志》2021年第5期376-379,共4页Chinese Journal of Applied Clinical Pediatrics

基　　金：广州市妇女儿童医疗中心内科部基金项目(NKE-PRE-2019-014)。

摘　　要：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丘疹,Gower征阳性,四肢近端肌力为Ⅲ~Ⅳ级,远端肌力Ⅳ级,吞咽流质饮食时出现呛咳,实验室检查丙氨酸转氨酶(ALT)36 U/L,天冬氨酸转氨酶(AST)115 U/L,碱性磷酸酶69 U/L,乳酸脱氢酶941 U/L,肌酸激酶974 U/L,超敏C反应蛋白26 mg/L,红细胞沉降率(ESR)52 mm/1 h,抗核抗体谱未见异常,肌电图提示肌源性损害,大腿磁共振成像示双侧臀部、大腿及膝关节皮下脂肪、肌肉及肌肉间隙内弥散性片状异常信号影。患儿被诊断为JDM,予甲泼尼龙琥珀酸钠、静脉注射免疫球蛋白、甲氨蝶呤、硫酸羟氯喹片规范治疗后,皮疹仍呈红色,四肢近端肌力无好转,仍有呛咳表现,并出现库欣面容,复查ALT 24 U/L,AST 32 U/L,碱性磷酸酶56 U/L,乳酸脱氢酶216 U/L,肌酸激酶527 U/L,超敏C反应蛋白8 mg/L,ESR 15 mm/1 h,病情考虑为难治性JDM,与家属沟通并同意后,予GLM 50 mg治疗,每月1次皮下注射,激素逐渐减量使用,停用硫酸羟氯喹片,继续口服甲氨蝶呤,经2次GLM治疗后,患儿四肢近端肌力及吞咽肌功能较前有明显好转。在第3次皮下注射后,四肢近端肌力为Ⅳ~Ⅴ级,可上下楼梯、下蹲及下蹲后站起,无吞咽异常,进食流质顺利无呛咳,颜面皮疹有好转,复查ESR、肌酸激酶等均恢复正常,大腿磁共振成像提示未见明显肌肉炎症。结论GLM对难治性JDM患儿有效,能有效改善患儿肌力,可考虑作为难治性JDM的临床治疗药物选择。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 b

关 键 词：戈利木单抗 儿童 皮肌炎 疗效 

分 类 号：R725.9[医药卫生—儿科]