重症肌无力
医学
耐受性
不利影响
临床终点
前瞻性队列研究
内科学
弱点
临床试验
神经肌肉传递
外科
作者
Guntis Karelis,Rodica Bălaşa,Jan De Bleecker,T. R. Stuchevskaya,Andrés Villa,Philip Van Damme,É. Lagrange,Jeannine M. Heckmann,Michael W. Nicolle,Crisandra Vilciu,Vera Bril,Elsa Mondou,Rhonda Griffin,Junliang Chen,Waleska Henriquez,Beatriz García,Sandra Camprubí,Jaume Ayguasanosa
出处
期刊:European Neurology
[S. Karger AG]
日期:2019-01-01
卷期号:81 (5-6): 223-230
被引量:23
摘要
Myasthenia gravis (MG) is an autoimmune disorder affecting neuromuscular transmission. Exacerbations may involve increasing bulbar weakness and/or sudden respiratory failure, both of which can be critically disabling. Management of MG exacerbations includes plasma exchange and intravenous immunoglobulin (IVIG); they are equally effective, but patients experience fewer side effects with IVIG. The objective of this study was to assess the efficacy and safety of immune globulin caprylate/chromatography purified (IGIV-C) in subjects with MG exacerbations.This prospective, open-label, non-controlled 28-day clinical trial was conducted in adults with MG Foundation of America class IVb or V status. Subjects received IGIV-C 2 g/kg over 2 consecutive days (1 g/kg/day) and were assessed for efficacy/safety on Days 7, 14, 21, and 28. The primary efficacy endpoint was the change from Baseline in quantitative MG (QMG) score to Day 14. Secondary endpoints of clinical response, Baseline to Day 14, included at least a 3-point decrease in QMG and MG Composite and a 2-point decrease in MG-activities of daily living (MG-ADL).Forty-nine subjects enrolled. The change in QMG score at Day 14 was significant (p < 0.001) in the Evaluable (-6.4, n = 43) and Safety (-6.7, n = 49) populations. Among evaluable subjects, Day 14 response rates were 77, 86, and 88% for QMG, MG Composite, and MG-ADL, respectively. IGIV-C showed good tolerability with no serious adverse events.The results of this study show that IGIV-C was effective, safe, and well tolerated in the treatment of MG exacerbations.
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