Background

Juvenile myasthenia gravis is a pediatric autoimmune disorder of the neuromuscular junction associated with significant morbidity, for which standard therapies are not always efficacious. The objective of our study was to assess the tolerability and efficacy of rituximab use in children with refractory juvenile myasthenia gravis.

Methods

We conduced a retrospective cohort study at a single tertiary care referral center to evaluate children with juvenile myasthenia gravis who were treated with rituximab. The clinical status of these participants before and after initiation of rituximab therapy was measured, focusing on numbers of hospital admissions, numbers of immunomodulatory or immunosuppressive medications needed, and Myasthenia Gravis Foundation of America severity class.

Results

Five children with juvenile myasthenia gravis were ascertained who received rituximab as part of their regimen, four of whom had elevated acetylcholine receptor antibodies and one of whom had elevated muscle-specific kinase antibodies. After initiation of rituximab therapy, all participants experienced reduced numbers of immunomodulatory medications during the follow-up period (mean 11.6 months). Four of the five subjects experienced fewer juvenile myasthenia gravis-related hospital admissions and reduced (improved) Myasthenia Gravis Foundation of America classes, with no subjects having moderate or severe symptoms following treatment with rituximab. No significant adverse events were recorded for any of the participants.

Conclusion

Rituximab was well-tolerated and efficacious in this juvenile myasthenia gravis cohort. The beneficial effect of rituximab was most pronounced in the one participant with muscle-specific kinase antibodies.

中文翻译：

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利妥昔单抗作为难治性重症肌无力的辅助维持疗法。

背景

幼年型重症肌无力是一种伴有明显发病率的神经肌肉接头的小儿自身免疫性疾病，对此标准疗法并不总是有效的。我们研究的目的是评估利妥昔单抗在难治性重症肌无力儿童中的耐受性和疗效。

方法

我们在一个三级转诊中心进行了一项回顾性队列研究，以评估接受利妥昔单抗治疗的重症肌无力少年儿童。在开始接受利妥昔单抗治疗之前和之后，对这些参与者的临床状况进行了测量，重点是住院人数，所需的免疫调节或免疫抑制药物的数量以及美国重症肌无力基金会的严重程度等级。

结果

确定了五名患有重症肌无力的儿童，他们在方案中接受了利妥昔单抗治疗，其中四名儿童的乙酰胆碱受体抗体升高，其中一人的肌肉特异性激酶抗体升高。开始利妥昔单抗治疗后，所有参与者在随访期间（平均11.6个月）经历了数量减少的免疫调节药物治疗。五名受试者中的四名经历了与重症肌无力相关的少年住院患者减少，美国重症肌无力基金会班级减少（改善），没有受试者在接受利妥昔单抗治疗后出现中度或严重症状。没有记录到任何参与者的重大不良事件。

结论

在这个青少年重症肌无力队列中，利妥昔单抗耐受性好且有效。利妥昔单抗的有益作用在肌肉特异性激酶抗体的一位参与者中最为明显。