Our aim was to identify clinical outcomes, serological features and possible prognostic indicators of paediatric myasthenia gravis (MG). We collected 74 MG patients with disease onset before the age of 16 years (73% pre-pubertal onset defined as ≤10 years), seen regularly at two UK specialist centres, over a period of 11 years. The cohort was multi-ethnic, with a high number of non-Caucasians (52%). Ocular presentation was seen in 38 (51%) and only 8 (21%) of these generalised. Fifty-two (70%) patients had antibodies to the acetylcholine receptor (AChR) measured by radioimmunoprecipitation, 10 (14%) had antibodies only to clustered AChRs detected by a cell based assay, 3 (4%) had muscle-specific kinase and one (1%) low-density lipoprotein receptor-related protein 4 antibody. Only 8 (11%) had no detectable antibodies. Seventeen patients attained drug free remission (Kaplan Meyer survival curve estimates 25% by 7 years). Several factors were associated with a higher likelihood of free remission: onset age ≤10 years, Asian and Caucasian races, lack of AChR antibodies on RIA, and normal repetitive nerve stimulation at diagnosis. However, in a multifactorial regression analysis, the antibody status was the only significant predictor for drug free remission, with 60% of patients with antibodies only to clustered AChR achieving this outcome. Complete drug free remission is not uncommon in paediatric MG and several factors appear to influence this outcome with antibody status being the most important. These factors can be easily evaluated at diagnosis, and may help to determine whose patients are likely to require more intensive treatments.

中文翻译：

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小儿重症肌无力：无药物缓解的预后因素

我们的目的是确定小儿重症肌无力 (MG) 的临床结果、血清学特征和可能的预后指标。我们收集了 74 名在 16 岁之前发病的 MG 患者（73% 青春期前发病定义为≤10 岁），在 11 年的时间里在两个英国专科中心定期就诊。该队列是多民族的，非白种人（52%）的人数很多。38 例 (51%) 出现眼部表现，其中仅 8 例 (21%) 全身化。五十二名 (70%) 患者有通过放射免疫沉淀法测量的乙酰胆碱受体 (AChR) 抗体，10 名 (14%) 有仅针对基于细胞的测定检测到的成簇 AChR 的抗体，3 名 (4%) 有肌肉特异性激酶和一种 (1%) 低密度脂蛋白受体相关蛋白 4 抗体。只有 8 个 (11%) 没有可检测到的抗体。17 名患者获得无药物缓解（Kaplan Meyer 生存曲线估计 7 年为 25%）。有几个因素与更高的自由缓解可能性相关：发病年龄≤10 岁、亚洲和高加索人种、RIA 缺乏 AChR 抗体以及诊断时正常的重复性神经刺激。然而，在一项多因素回归分析中，抗体状态是无药缓解的唯一重要预测因素，60% 的患者只有聚集 AChR 的抗体才能达到这一结果。完全无药物缓解在儿科 MG 中并不少见，似乎有几个因素会影响这一结果，其中抗体状态是最重要的。这些因素可以在诊断时轻松评估，并且可能有助于确定哪些患者可能需要更强化的治疗。