Efficacy and safety of maintenance intravenous immunoglobulin in generalized myasthenia gravis patients with acetylcholine receptor antibodies: A multicenter, double-blind, placebo-controlled trial.

IF 2.8 3区 医学 Q2 CLINICAL NEUROLOGY Muscle & Nerve Pub Date : 2024-11-07 DOI:10.1002/mus.28289
Vera Bril, Tomasz Berkowicz, Andrzej Szczudlik, Michael W Nicolle, Josef Bednarik, Petr Hon, Antanas Vaitkus, Tuan Vu, Csilla Rozsa, Tim Magnus, Gyula Panczel, Toomas Toomsoo, Mamatha Pasnoor, Tahseen Mozaffar, Miriam Freimer, Ulrike Reuner, László Vécsei, Nizar Souayah, Todd Levine, Robert M Pascuzzi, Marinos C Dalakas, Michael Rivner, Rhonda Griffin, Montse Querolt Coll, Elsa Mondou
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Abstract

Introduction/aims: Prospective, randomized, controlled trials of intravenous immunoglobulin (IVIG) maintenance therapy in myasthenia gravis (MG) are lacking. In this trial, we evaluated the safety and efficacy of caprylate/chromatography-purified IVIG; (IGIV-C) in patients with generalized MG undergoing standard care.

Methods: Sixty-two patients enrolled in this phase 2, multicenter, international, randomized trial (1:1 IGIV-C [2 g/kg loading dose; 1 g/kg every 3 weeks through week 21] or placebo). Efficacy was assessed by changes in Quantitative MG (QMG) score at week 24 versus baseline (primary endpoint) and percentage of patients with clinical improvement in QMG, MG Composite (MGC), and MG-Activities of Daily Living (MG-ADL) scores (secondary endpoints). Safety assessments reported all adverse events (AEs).

Results: The change in QMG at 24 weeks was -5.1 for IGIV-C and -3.1 for placebo (p = .187). Seventy percent of patients in the IGIV-C group had improvement in MG-ADL (≥2-point decrease) versus 40.6% in the placebo group (p = .025). Patients showing clinical improvement in QMG and MGC (≥3-point decrease) were 70.0% for IGIV-C versus 59.4% for placebo (p = .442) and 60.0% for IGIV-C versus 53.1% for placebo (p = .610). IGIV-C was well tolerated; serious AEs were similar between arms. Three of four MG exacerbations requiring hospitalizations occurred in the IGIV-C arm with one death.

Discussion: Several efficacy parameters showed numerical results greater than those seen in the placebo group. This was a small study and may have been underpowered to see significant differences. Additional studies may be warranted to fully determine the efficacy of IVIG maintenance therapy in MG.

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乙酰胆碱受体抗体全身性肌无力患者静脉注射免疫球蛋白的疗效和安全性:一项多中心、双盲、安慰剂对照试验。
简介/目的:目前尚缺乏对重症肌无力(MG)患者进行静脉注射免疫球蛋白(IVIG)维持治疗的前瞻性、随机对照试验。在这项试验中,我们评估了接受标准治疗的全身性 MG 患者使用丙烯酸酯/色谱纯化 IVIG(IGIV-C)的安全性和有效性:62名患者参加了这项2期多中心国际随机试验(1:1 IGIV-C[2克/千克负荷剂量;第21周之前每3周1克/千克]或安慰剂)。疗效通过第24周时MG定量评分(QMG)相对于基线的变化(主要终点)以及QMG、MG综合评分(MGC)和MG-日常生活活动评分(MG-ADL)临床改善的患者比例(次要终点)进行评估。安全性评估报告了所有不良事件(AEs):24周时,IGIV-C的QMG变化为-5.1,安慰剂为-3.1(p = .187)。IGIV-C组70%的患者MG-ADL有所改善(降幅≥2分),而安慰剂组为40.6%(p = .025)。QMG和MGC临床改善(≥3分)的患者中,IGIV-C组为70.0%,安慰剂组为59.4%(p = .442);IGIV-C组为60.0%,安慰剂组为53.1%(p = .610)。IGIV-C 的耐受性良好;各组的严重 AE 相似。IGIV-C治疗组出现了四次需要住院治疗的MG恶化中的三次,其中一次死亡:讨论:多项疗效参数的数值结果均高于安慰剂组。这项研究规模较小,可能没有达到观察显著差异的能力。要全面确定IVIG维持治疗对MG的疗效,可能还需要进行更多的研究。
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来源期刊
Muscle & Nerve
Muscle & Nerve 医学-临床神经学
CiteScore
6.40
自引率
5.90%
发文量
287
审稿时长
3-6 weeks
期刊介绍: Muscle & Nerve is an international and interdisciplinary publication of original contributions, in both health and disease, concerning studies of the muscle, the neuromuscular junction, the peripheral motor, sensory and autonomic neurons, and the central nervous system where the behavior of the peripheral nervous system is clarified. Appearing monthly, Muscle & Nerve publishes clinical studies and clinically relevant research reports in the fields of anatomy, biochemistry, cell biology, electrophysiology and electrodiagnosis, epidemiology, genetics, immunology, pathology, pharmacology, physiology, toxicology, and virology. The Journal welcomes articles and reports on basic clinical electrophysiology and electrodiagnosis. We expedite some papers dealing with timely topics to keep up with the fast-moving pace of science, based on the referees'' recommendation.
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