Department of Paediatrics, MDUK Neuromuscular Centre, University of Oxford, Oxford, UK.
Department of Paediatric Neurology, John Radcliffe Hospital, Oxford, UK.
Paediatr Drugs. 2024 Nov;26(6):719-740. doi: 10.1007/s40272-024-00649-3. Epub 2024 Aug 28.
Myasthenia gravis (MG) is an antibody-mediated disorder of the neuromuscular junction affecting children and adults. MG is a treatable condition with most patients requiring immunosuppression for disease control and/or remission. Juvenile myasthenia gravis (JMG) is rare in comparison with adult-onset MG but given the same underlying pathophysiology, treatment strategies are similar to those in adults. Until recently, there were only a few randomised controlled trials (RCTs) for MG treatments in adults and none in children, and management strategies were primarily based on expert consensus. In addition, treatment options for refractory MG cases have been severely limited, resulting in poor long-term quality of life in such patients due to the significant disease burden. Recently, there have been several RCTs focussing on novel therapeutic strategies with potentially promising outcomes, suggesting a change in MG management over the coming years and access to more effective and faster-acting drugs for MG patients. This paper will review current and new MG treatments including efgartigimod, eculizumab, rozanolixizumab, ravulizumab, and zilucoplan, with a focus on juvenile myasthenia gravis.
重症肌无力（MG）是一种影响儿童和成人的神经肌肉接头的抗体介导疾病。MG 是一种可治疗的疾病，大多数患者需要免疫抑制来控制和/或缓解疾病。与成人发病的 MG 相比，青少年型 MG 较为罕见，但由于具有相同的潜在病理生理学，因此治疗策略与成人相似。直到最近，成人 MG 的治疗方法仅有少数随机对照试验（RCT），而儿童则没有，管理策略主要基于专家共识。此外，难治性 MG 病例的治疗选择受到严重限制，由于疾病负担沉重，导致此类患者的长期生活质量较差。最近，有几项 RCT 专注于具有潜在前景的新治疗策略，这表明未来几年 MG 的管理方式将会发生变化，MG 患者将获得更有效和起效更快的药物。本文将综述目前和新的 MG 治疗方法，包括依氟鸟氨酸、依库珠单抗、罗唑尼莫司单抗、瑞昔利珠单抗和zilucoplan，重点关注青少年型 MG。
