01O 全身性肌无力患者在不同治疗周期对罗扎尼珠单抗的反应：事后分析

IF 2.7 4区医学 Q2 CLINICAL NEUROLOGY

Neuromuscular Disorders Pub Date : 2024-10-01 DOI:10.1016/j.nmd.2024.07.012

J. Vissing , J. Grosskreutz , A. Habib , Z. Mahuwala , R. Mantegazza , R. Pascuzzi , S. Sacconi , T. Vu , R. Beau Lejdstrom , B. Greve , F. Grimson , T. Tarancón , V. Bril

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引用次数: 0

摘要

在3期MycarinG（NCT03971422）研究中，与安慰剂相比，罗扎尼单抗7毫克/千克或10毫克/千克一个周期（六次，每周一次，皮下注射）可显著改善肌无力（MG）特异性结果。在MycarinG之后，患者可以参加MG0004（NCT04124965）、MG0007（NCT04650854）或直接参加MG0007的开放标签延伸项目（OLEs）。我们根据第一周期的反应评估全身型MG患者在多个治疗周期中对罗扎尼单抗的反应。MG0004是一种为期≤52周的慢性OLE，每周一次罗扎尼珠单抗7毫克/千克或10毫克/千克。在MG0007中，经过一个为期六周的周期（罗扎尼珠单抗7毫克/千克或10毫克/千克）后，在症状恶化时进行后续周期的治疗。对于≥2个症状驱动周期的患者，对MycarinG、MG0004（前6周）和MG0007（中期分析；数据截止日期：2022年7月8日）的数据进行了汇总。分析每个周期第43天的MG-日常生活活动（MG-ADL）和定量MG（QMG）反应率（分别比基线改善≥2.0分和≥3.0分）。根据第一周期的反应情况对反应率进行了事后分析。总体而言，127 名患者的症状驱动周期≥2 个。在第 1 周期，第 43 天时分别有 74.0% （94/127）和 68.5% （87/127）的患者对 MG-ADL 和 QMG 有反应。在 MG-ADL 第 1 周期应答者中，MG-ADL 应答率在随后的周期中保持较高水平（第 2 周期：78.7% [74/94]；第 3 周期：77.1% [54/70]；第 4 周期：78.0% [46/59]）。在 QMG 第一周期应答者中也观察到类似的 QMG 应答模式（第二周期：67.4% [58/86]；第三周期：76.2% [48/63]；第四周期：69.2% [36/52]）。在 33 名（26.0%）MG-ADL 在第一周期无应答者中，63.6%（21/33）在第二周期有反应。在 40 名（31.5%）第一周期无应答的 QMG 患者中，51.3%（20/39）在第二周期时有了反应。无论初始反应如何，接受罗扎尼单抗治疗的患者在多个周期内均表现出较高的反应率。初始无应答者可能会从额外的罗扎尼单抗治疗周期中获益。

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01O Response to rozanolixizumab across treatment cycles in patients with generalised myasthenia gravis: a post hoc analysis

In the Phase 3 MycarinG (NCT03971422) study, one cycle (six once-weekly subcutaneous infusions) of rozanolixizumab 7mg/kg or 10mg/kg significantly improved myasthenia Gravis (MG)-specific outcomes versus placebo. After MycarinG, patients could enrol in open-label extensions (OLEs) MG0004 (NCT04124965) then MG0007 (NCT04650854), or MG0007 directly. We evaluate response to rozanolixizumab over multiple treatment cycles in patients with generalised MG based on Cycle 1 response. MG0004 was a ≤52-week OLE of chronic, once-weekly rozanolixizumab 7mg/kg or 10mg/kg. In MG0007, after one six-week cycle (rozanolixizumab 7mg/kg or 10mg/kg), subsequent cycles were administered upon symptom worsening. Data were pooled across MycarinG, MG0004 (first 6 weeks) and MG0007 (interim analysis; data cut-off: 08 July 2022) for patients with ≥2 symptom-driven cycles. MG-Activities of Daily Living (MG-ADL) and Quantitative MG (QMG) response rate (≥2.0- and ≥3.0-point improvement from baseline, respectively) at Day 43 in each cycle was analysed. Post hoc analyses of response rates were conducted based on Cycle 1 response. Overall, 127 patients had ≥2 symptom-driven cycles. In Cycle 1, 74.0% (94/127) and 68.5% (87/127) of patients were MG-ADL and QMG responders, respectively, at Day 43. Among MG-ADL Cycle 1 responders, MG-ADL response rates remained high over subsequent cycles (Cycle 2: 78.7% [74/94]; Cycle 3: 77.1% [54/70]; Cycle 4: 78.0% [46/59]). Similar patterns were observed for QMG response among QMG Cycle 1 responders (Cycle 2: 67.4% [58/86]; Cycle 3: 76.2% [48/63]; Cycle 4: 69.2% [36/52]). Of 33 (26.0%) MG-ADL non-responders at Cycle 1, 63.6% (21/33) were responders at Cycle 2. Of 40 (31.5%) QMG non-responders at Cycle 1, 51.3% (20/39) were responders at Cycle 2. Patients receiving rozanolixizumab demonstrated a high response rate over multiple cycles irrespective of initial response. Initial non-responders may benefit from additional rozanolixizumab treatment cycles.

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来源期刊

Neuromuscular Disorders 医学-临床神经学

CiteScore

4.60

自引率

3.60%

发文量

543

审稿时长

53 days

期刊介绍： This international, multidisciplinary journal covers all aspects of neuromuscular disorders in childhood and adult life (including the muscular dystrophies, spinal muscular atrophies, hereditary neuropathies, congenital myopathies, myasthenias, myotonic syndromes, metabolic myopathies and inflammatory myopathies). The Editors welcome original articles from all areas of the field: • Clinical aspects, such as new clinical entities, case studies of interest, treatment, management and rehabilitation (including biomechanics, orthotic design and surgery). • Basic scientific studies of relevance to the clinical syndromes, including advances in the fields of molecular biology and genetics. • Studies of animal models relevant to the human diseases. The journal is aimed at a wide range of clinicians, pathologists, associated paramedical professionals and clinical and basic scientists with an interest in the study of neuromuscular disorders.