Ellen and Martin Prosserman Centre for Neuromuscular Diseases, Toronto General Hospital, University Health Network, University of Toronto, Toronto, ON, Canada.
Eur J Neurol. 2021 Feb;28(2):639-646. doi: 10.1111/ene.14547. Epub 2020 Oct 15.
Long-term treatment of myasthenia gravis (MG) includes symptomatic and course-modifying therapies that target the immune system. Recently, both intravenous immunoglobulin (IVIG) and subcutaneous immunoglobulin (SCIG) have emerged as viable options for chronic therapy, considering the favourable safety-efficacy profile and possible immunosuppressant sparing properties. The aim was to investigate the outcomes of the long-term care of generalized MG with immunoglobulin (Ig).
This is a retrospective, repeated-measures design study. Charts of generalized MG patients, treated with IVIG/SCIG for at least 6 months, from January 2015 to January 2020, were analysed. The primary outcome was the mean change in Myasthenia Gravis Impairment Index (MGII) after treatment with Ig, comparing baseline to IVIG and SCIG treatment periods. Secondary outcomes included the changes in pyridostigmine, immunosuppressive medications and patient-reported outcome 'percentage of normal' (0%-100%).
Thirty-four patients were treated with chronic Ig therapy (30 IVIG/SCIG, three SCIG, one IVIG). The mean durations of IVIG and SCIG periods were 21.8 ± 19.4 (range 3-64) months and 19.5 ± 11.3 (range 5-45) months respectively. There was a significant reduction in MGII scores (27.7 ± 15.7 baseline; 22.0 ± 17.4 IVIG period; 19.5 ± 18.1 SCIG period; F = 17.9; d.f. = 1.7; P < 0.01), pyridostigmine and immunosuppressant use (P = 0.00). The outcome 'percentage of normal' had a significant positive association with both treatments (P = 0.00).
Our study results suggest that patients can be successfully transitioned to IVIG and from IVIG to SCIG in the chronic treatment of generalized MG with reductions in impairments and use of other medications and improvement in overall status with Ig therapy. Prospective, randomized studies are needed to clarify costs and comparative effectiveness.
重症肌无力(MG)的长期治疗包括针对免疫系统的对症和改善病程的治疗。静脉注射免疫球蛋白(IVIG)和皮下免疫球蛋白(SCIG)最近已成为慢性治疗的可行选择,因为它们具有良好的安全性-疗效特征和可能的免疫抑制剂节省作用。本研究旨在调查免疫球蛋白(Ig)治疗对全身性 MG 长期治疗的效果。
这是一项回顾性、重复测量设计的研究。分析了 2015 年 1 月至 2020 年 1 月期间,接受至少 6 个月 IVIG/SCIG 治疗的全身性 MG 患者的图表。主要结局是比较 Ig 治疗前后,使用 Myasthenia Gravis Impairment Index(MGII)的平均值变化,比较基线、IVIG 和 SCIG 治疗期。次要结局包括吡啶斯的明、免疫抑制剂药物和患者报告的“正常百分比”(0%-100%)的变化。
34 例患者接受慢性 Ig 治疗(30 例 IVIG/SCIG,3 例 SCIG,1 例 IVIG)。IVIG 和 SCIG 治疗期的平均持续时间分别为 21.8±19.4(范围 3-64)个月和 19.5±11.3(范围 5-45)个月。MGII 评分显著降低(基线 27.7±15.7;IVIG 期 22.0±17.4;SCIG 期 19.5±18.1;F=17.9;d.f.=1.7;P<0.01),吡啶斯的明和免疫抑制剂的使用减少(P=0.00)。“正常百分比”的结果与两种治疗方法均有显著正相关(P=0.00)。
我们的研究结果表明,在全身性 MG 的慢性治疗中,患者可以成功地从 IVIG 过渡到 IVIG 和 SCIG,从而减少损伤和其他药物的使用,改善整体状态。需要前瞻性、随机研究来澄清成本和比较效果。