关键词: MG-ADL acetylcholine receptor antibody positive complement inhibition generalized myasthenia gravis ravulizumab

来  源:   DOI:10.3389/fneur.2024.1378080   PDF(Pubmed)

Abstract:
UNASSIGNED: To describe the early experience of ravulizumab use in acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR+ve gMG).
UNASSIGNED: This multicenter retrospective study included AChR+ve gMG patients who were treated with ravulizumab and had both pre- and post-ravulizumab myasthenia gravis activities of daily living (MG-ADL) scores. Clinical information regarding MG history, concomitant treatment(s), MG-ADL, other MG-specific measures, and adverse events were recorded.
UNASSIGNED: A total of 18 patients with mean age of 61.83 (±16.08, n = 18) years were included in this cohort. In 10 complement inhibitor naive patients, a clinically meaningful reduction in mean Mg-ADL (baseline: 6.6 (±3.58) vs. 4.4 (±2.28), post ravulizumab) was seen. 6 out of 10 patients (60%) had clinically meaningful reduction post ravulizumab and two achieved minimum symptom expression (MSE). In 8 patients switched from eculizumab to ravulizumab, further reduction was noted in post ravulizumab mean MG-ADL (Baseline: 3.25 (±3.34) vs. 1.5 (±2.34) post ravulizumab). None of the patients who switched from eculizumab to ravulizumab experienced worsening symptoms. Eleven out of 14 (78.5%) patients on prednisone therapy were able to reduce their prednisone dose post-ravulizumab. None of the patients experienced any major side effects.
UNASSIGNED: In our clinical practice, 60% of AChR+ve gMG complement inhibitor naive patients experienced a clinically meaningful improvement in MG-ADL scores with ravulizumab. Patients were safely switched from eculizumab to ravulizumab and had further improvement in their mean MG-ADL scores. Of those on prednisone therapy, the majority were able to reduce their prednisone dosage.
摘要:
描述ravulizumab在乙酰胆碱受体抗体阳性的全身性重症肌无力(AChRvegMG)中使用的早期经验。
这项多中心回顾性研究包括AChR+vegMG患者,这些患者接受了ravulizumab治疗,并且具有ravulizumab前后的重症肌无力日常生活活动能力(MG-ADL)评分。关于MG病史的临床信息,伴随治疗,MG-ADL,其他MG特定的措施,并记录不良事件.
共有18名平均年龄为61.83(±16.08,n=18)岁的患者纳入该队列。在10名补体抑制剂初治患者中,平均Mg-ADL的临床意义降低(基线:6.6(±3.58)与4.4(±2.28),后ravulizumab)被看到。10名患者中有6名(60%)在ravulizumab后有临床意义的减少,两名患者达到了最低症状表达(MSE)。在8例患者中,从依库珠单抗转换为ravulizumab,ravulizumab后平均MG-ADL进一步降低(基线:3.25(±3.34)vs.1.5(±2.34)后ravulizumab)。从eculizumab转为ravulizumab的患者均未出现症状恶化。接受泼尼松治疗的14名患者中有11名(78.5%)能够在ravulizumab后减少其泼尼松剂量。没有患者出现任何严重的副作用。
在我们的临床实践中,60%的AChR+vegMG补体抑制剂初治患者使用ravulizumab的MG-ADL评分有临床意义的改善。患者安全地从eculizumab切换到ravulizumab,并且其平均MG-ADL评分进一步改善。在接受泼尼松治疗的人中,大多数能够减少他们的泼尼松剂量。
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