Department of Melanoma Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
J Immunother Cancer. 2019 Nov 21;7(1):319. doi: 10.1186/s40425-019-0774-y.
Myasthenia gravis (MG) is a rare but life-threatening adverse event of immune checkpoint inhibitors (ICI). Given the limited evidence, data from a large cohort of patients is needed to aid in recognition and management of this fatal complication.
We reviewed our institutional databases to identify patients who had cancer and MG in the setting of ICI. We systematically reviewed the literature through August 2018 to identify all similar reported patients. We collected data on clinical and diagnostic features, management, and outcomes of these cases.
Sixty-five patients were identified. Median age was 73 years; 42 (65%) were males, 31 (48%) had metastatic melanoma, and 13 (20%) had a preexisting MG before ICI initiation. Most patients received anti-PD-1 (82%). Sixty-three patients (97%) developed ICI-related MG (new onset or disease flare) after a median of 4 weeks (1 to 16 weeks) of ICI initiation. Twenty-four patients (37%) experienced concurrent myositis, and respiratory failure occurred in 29 (45%). ICI was discontinued in 61 patients (97%). Death was reported in 24 patients (38%); 15 (23%) due to MG complication. A better outcome was observed in patients who received intravenous immunoglobulin (IVIG) or plasmapheresis (PLEX) as first-line therapy than in those who received steroids alone (95% vs 63% improvement of MG symptoms, p = 0.011).
MG is a life-threatening adverse event of acute onset and rapid progression after ICI initiation. Early use of IVIG or PLEX, regardless of initial symptoms severity, may lead to better outcomes than steroids alone. Our data suggest the need to reassess the current recommendations for management of ICI-related MG until prospective longitudinal studies are conducted to establish the ideal management approach for these patients.
重症肌无力（MG）是免疫检查点抑制剂（ICI）的一种罕见但危及生命的不良反应。鉴于证据有限，需要来自大量患者队列的数据来帮助识别和管理这种致命并发症。
我们回顾了我们的机构数据库，以确定在 ICI 背景下患有癌症和 MG 的患者。我们通过 2018 年 8 月系统地审查了文献，以确定所有类似报道的患者。我们收集了这些病例的临床和诊断特征、管理和结局的数据。
共确定了 65 例患者。中位年龄为 73 岁；42 例（65%）为男性，31 例（48%）患有转移性黑色素瘤，13 例（20%）在开始 ICI 前存在 MG。大多数患者接受了抗 PD-1（82%）治疗。63 例（97%）患者在 ICI 开始后中位时间为 4 周（1 至 16 周）出现 ICI 相关 MG（新发或疾病加重）。24 例（37%）患者同时出现肌炎，29 例（45%）患者出现呼吸衰竭。61 例患者（97%）停用 ICI。24 例患者（38%）报告死亡；其中 15 例（23%）死于 MG 并发症。与单独使用类固醇相比，首先使用静脉注射免疫球蛋白（IVIG）或血浆置换（PLEX）治疗的患者的 MG 症状改善更好（95% vs 63%，p=0.011）。
MG 是 ICI 后急性发作和快速进展的危及生命的不良反应。早期使用 IVIG 或 PLEX，无论初始症状严重程度如何，可能比单独使用类固醇的效果更好。我们的数据表明，需要重新评估目前关于 ICI 相关 MG 管理的建议，直到进行前瞻性纵向研究确定这些患者的理想管理方法。
