APHM Service de Neuropédiatrie, Hôpital La Timone-Enfants, 264, Rue St Pierre Marseille, 13385, cedex 5, France.
Aix-Marseille Université, Marseille, France.
Muscle Nerve. 2018 Apr;57(4):603-609. doi: 10.1002/mus.25965. Epub 2017 Oct 11.
In this study we determined the clinical, paraclinical, and treatment-related features of juvenile myasthenia gravis (JMG) as well as the clinical course in a cohort of French children.
We conducted a retrospective study of 40 patients with JMG at 2 French pediatric neurology departments from April 2004 to April 2014.
Among the patients, 70% had generalized JMG, 52% had positive acetylcholine receptor antibodies, 8% had muscle-specific kinase antibodies, and 40% were seronegative. Treatment with acetylcholinesterase inhibitors was effective and sufficient in 47% of patients. The 6 patients with generalized JMG treated with rituximab and/or immunoadsorption showed improvement. Thirty percent of the patients required hospitalization in an intensive care unit during follow-up (mean 4.7 years). Remission without treatment occurred in 18% of patients.
As with adults, JMG has high morbidity, particularly among children with generalized symptoms, and rituximab should be considered early in the course of the disease as a second-line treatment. Muscle Nerve 57: 603-609, 2018.
本研究旨在确定法国儿童青少年重症肌无力（JMG）的临床、实验室检查和治疗相关特征及疾病过程。
我们对 2004 年 4 月至 2014 年 4 月期间在法国 2 个儿科神经病学部门的 40 例 JMG 患者进行了回顾性研究。
70%的患者为全身型 JMG，52%的患者乙酰胆碱受体抗体阳性，8%的患者肌肉特异性激酶抗体阳性，40%的患者为阴性。47%的患者对乙酰胆碱酯酶抑制剂治疗有效且足够。6 例接受利妥昔单抗和/或免疫吸附治疗的全身型 JMG 患者病情改善。30%的患者在随访期间（平均 4.7 年）需要入住重症监护病房。18%的患者未经治疗即可缓解。
与成人一样，JMG 的发病率较高，特别是全身型症状的儿童，利妥昔单抗应作为疾病早期的二线治疗药物。肌肉神经 57: 603-609, 2018.
