College of Medicine, King Saud Bin Abdulaziz University for Health Sciences, Jeddah, Saudi Arabia; King Abdullah International Medical Research Center, Jeddah, Saudi Arabia; Department of Neurosciences, Ministry of The National Guard-Health Affairs, Jeddah, Saudi Arabia.
College of Medicine, King Saud Bin Abdulaziz University for Health Sciences, Jeddah, Saudi Arabia; King Abdullah International Medical Research Center, Jeddah, Saudi Arabia.
Mult Scler Relat Disord. 2024 May;85:105524. doi: 10.1016/j.msard.2024.105524. Epub 2024 Mar 1.
Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune disorder characterized by relapses of inflammation and demyelination primarily affecting the optic nerve and the spinal cord. C5 complement inhibition is an effective therapeutic approach in the treatment of NMOSD. In this systematic review and meta-analysis, we aimed to determine the role of C5 inhibitors in the treatment of patients with seropositive anti-aquaporin-4 antibody (AQP4+IgG) NMOSD.
This systematic review follows the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guideline. Relevant articles were systematically searched through Medline, Embase, Cochrane Central Register of Controlled Trials (CENTRAL), and Web of Science databases until October 6th, 2023. We included randomized clinical trials (RCTs) that investigated the treatment with C5 inhibitors compared to placebo in patients with seropositive NMOSD. The primary endpoint was the rates of first adjudicated relapse. Secondary endpoints included different disability and quality of life measures. The random-effects model was used for all statistical analyses.
Two RCTs with a total of 201 patients were included. C5 inhibitors demonstrated significant reduction of first adjudicated relapse (risk ratio (RR) = 0.05, 95 % CI 0.01-0.15) and Hauser Ambulation Index (HAI) (mean difference (MD): -0.79, 95 % CI -1.27 to -0.31). There was no significant difference between the two groups in Expanded Disability Status Scale (EDSS) (MD -0.23, 95 % CI -0.54-0.08). C5 inhibitors significantly improved the mean change in EQ-5D index (MD 0.08, 95 % CI 0.01-0.14; P = 0.02); however, no significant difference was shown in the mean change in EQ-5D VAS (MD 3.79, 95 % CI -1.61 to 9.19; P = 0.17). Safety measures were comparable between C5 inhibitors and placebo.
NMOSD Patients with AQP4+IgG receiving C5 inhibitors have lower rate of relapses and improved levels of disability and quality of life. Real-world studies are warranted to establish the long-term safety of C5 inhibitors.
视神经脊髓炎谱系疾病（NMOSD）是一种自身免疫性疾病，其特征是炎症和脱髓鞘的复发，主要影响视神经和脊髓。C5 补体抑制是治疗 NMOSD 的有效治疗方法。在这项系统评价和荟萃分析中，我们旨在确定 C5 抑制剂在治疗血清阳性抗水通道蛋白 4 抗体（AQP4+IgG）NMOSD 患者中的作用。
本系统评价遵循《系统评价和荟萃分析的首选报告项目》（PRISMA）指南。通过 Medline、Embase、Cochrane 中央对照试验注册中心（CENTRAL）和 Web of Science 数据库系统地搜索相关文章，截至 2023 年 10 月 6 日。我们纳入了比较 C5 抑制剂与安慰剂治疗血清阳性 NMOSD 患者的随机临床试验（RCT）。主要终点是首次判定的复发率。次要终点包括不同的残疾和生活质量指标。所有统计分析均采用随机效应模型。
共有 2 项 RCT 纳入了 201 名患者。C5 抑制剂显著降低了首次判定的复发率（风险比（RR）=0.05，95%CI 0.01-0.15）和 Hauser 步行指数（HAI）（平均差异（MD）：-0.79，95%CI-1.27 至-0.31）。两组之间扩展残疾状况量表（EDSS）（MD-0.23，95%CI-0.54-0.08）无显著差异。C5 抑制剂显著改善了 EQ-5D 指数的平均变化（MD 0.08，95%CI 0.01-0.14；P=0.02）；然而，EQ-5D VAS 的平均变化（MD 3.79，95%CI-1.61 至 9.19；P=0.17）无显著差异。C5 抑制剂和安慰剂之间的安全性措施相当。
接受 C5 抑制剂治疗的血清阳性 AQP4+IgG NMOSD 患者复发率较低，残疾和生活质量水平得到改善。需要进行真实世界研究以确定 C5 抑制剂的长期安全性。
