Services in Health Economics, Brussels, Belgium.
Services in Health Economics, Brussels, Belgium.
J Neurol Sci. 2024 Nov 15;466:123264. doi: 10.1016/j.jns.2024.123264. Epub 2024 Oct 4.
This post-hoc analysis evaluates the long-term efficacy of efgartigimod versus placebo in adult patients with generalized myasthenia gravis (gMG) with acetylcholine-receptor autoantibodies (AChR-Ab+), based on data from the ADAPT RCT and its open-label extension ADAPT+.
Changes from baseline in Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores were assessed by treatment group over the ADAPT (up to 20 weeks) and ADAPT+ time horizon (extended to 64 weeks for efgartigimod group patients). Response to treatment was defined as 5-point reduction in QMG or 3-point reduction in MG-ADL vs. baseline values.
AChR-Ab+ patients treated with efgartigimod spent a substantially greater percentage of time in response in ADAPT based on at least a 5-point change in QMG compared to the placebo group (44 % versus 13 % respectively, p = 0.0034). Analyses based on a 3-point change in MG-ADL in ADAPT showed the percentage of time in response was nearly double for efgartigimod versus placebo (59 % versus 30 % respectively, p = 0.010). These trends were also maintained using different response definitions, as well as in patients with and without prior immune therapy exposure and by time from diagnosis (<7 years versus ≥7 years).
The clinical benefit of efgartigimod was sustained over repeat treatment cycles and maintained over the long term. Response to treatment was consistent regardless of response definition and was repeated in different patient subgroups. Overall, the results of this analysis indicate that efgartigimod is an effective therapeutic option, demonstrating a robust benefit among AChR-Ab+ patients with gMG.
本事后分析评估了 efgartigimod 对比安慰剂在乙酰胆碱受体抗体（AChR-Ab+）阳性的全身型重症肌无力（gMG）成年患者中的长期疗效，数据来自 ADAPT RCT 及其开放标签扩展研究 ADAPT+。
根据 ADAPT（最多 20 周）和 ADAPT+时间范围（efgartigimod 组患者延长至 64 周）内各治疗组相对于基线的定量重症肌无力（QMG）和重症肌无力日常生活活动量（MG-ADL）评分变化，评估治疗效果。治疗应答定义为 QMG 评分降低 5 分或 MG-ADL 评分降低 3 分，与基线值相比。
与安慰剂组相比，接受 efgartigimod 治疗的 AChR-Ab+患者在 ADAPT 中至少 QMG 评分改善 5 分的应答时间比例明显更高（分别为 44%和 13%，p=0.0034）。基于 ADAPT 中 MG-ADL 改善 3 分的分析显示，efgartigimod 组的应答时间比例几乎是安慰剂组的两倍（分别为 59%和 30%，p=0.010）。这些趋势在使用不同的应答定义以及既往免疫治疗暴露和诊断后时间（<7 年和≥7 年）的患者中也保持一致。
efgartigimod 的临床获益在重复治疗周期中得以维持，并在长期内得以保持。治疗应答一致，无论应答定义如何，且在不同患者亚组中重复出现。总的来说，该分析结果表明，efgartigimod 是一种有效的治疗选择，在 AChR-Ab+的 gMG 患者中显示出了强大的获益。
