Rituximab in multiple sclerosis: A retrospective observational study on safety and efficacy.
Rituximab in multiple sclerosis: A retrospective observational study on safety and efficacy.
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多发性硬化症中的利妥昔单抗：一项关于安全性和功效的回顾性观察研究。
DOI：
10.1212/wnl.0000000000003331
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发表时间：
2016-11-15
期刊：
影响因子：
9.9
通讯作者：
Svenningsson A
中科院分区：
文献类型：
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作者：
Salzer J;Svenningsson R;Alping P;Novakova L;Björck A;Fink K;Islam-Jakobsson P;Malmeström C;Axelsson M;Vågberg M;Sundström P;Lycke J;Piehl F;Svenningsson A
研究利妥昔单抗治疗多发性硬化症（MS）的安全性和有效性。在这项回顾性非对照观察性多中心研究中，通过瑞典 MS 登记册确定了接受超说明书利妥昔单抗治疗的 MS 患者。结果数据是从 MS 登记册和病历中收集的。根据不良事件通用术语标准，记录了 2-5 级不良事件 (AE)。总共确定了 822 名接受利妥昔单抗治疗的 MS 患者：557 名复发缓解型 MS (RRMS)、198 名继发进展型 MS (SPMS) 和 67 名原发进展型 MS (PPMS)。在基线时，26.2% 的患者有对比增强病变 (CEL)。患者每 6-12 个月接受 500 或 1,000 mg 利妥昔单抗 IV 治疗，平均治疗时间为 21.8 (SD 14.3) 个月。治疗期间，年复发率为 0.044 (RRMS)、0.038 (SPMS) 和 0.015 (PPMS)，4.6% 的患者出现 CEL。 RRMS 中的扩展残疾状态量表中位数保持不变（p = 0.42），而 SPMS 和 PPMS 中的中位数扩展残疾状态量表分别增加了 0.5 和 1.0（p = 0.10 和 0.25）。 7.8% 的输注过程中发生了与输注相关的 AE，且大多数为轻度。 72 名患者总共记录了 89 起 ≥2 级 AE（其中 76 起感染）。未发现进行性多灶性白质脑病病例。这是迄今为止报道的最大的接受利妥昔单抗治疗的多发性硬化症患者队列。这个接受不同剂量利妥昔单抗治疗的异质现实队列中的安全性、临床和 MRI 结果与之前关于 MS 中 B 细胞耗竭疗法的随机对照试验中报告的结果相似。这项研究提供了 IV 类证据，证明利妥昔单抗对于 MS 患者是安全有效的。
To investigate the safety and efficacy of rituximab in multiple sclerosis (MS). In this retrospective uncontrolled observational multicenter study, off-label rituximab-treated patients with MS were identified through the Swedish MS register. Outcome data were collected from the MS register and medical charts. Adverse events (AEs) grades 2–5 according to the Common Terminology Criteria for Adverse Events were recorded. A total of 822 rituximab-treated patients with MS were identified: 557 relapsing-remitting MS (RRMS), 198 secondary progressive MS (SPMS), and 67 primary progressive MS (PPMS). At baseline, 26.2% had contrast-enhancing lesions (CELs). Patients were treated with 500 or 1,000 mg rituximab IV every 6–12 months, during a mean 21.8 (SD 14.3) months. During treatment, the annualized relapse rates were 0.044 (RRMS), 0.038 (SPMS), and 0.015 (PPMS), and 4.6% of patients displayed CELs. Median Expanded Disability Status Scale remained unchanged in RRMS (p = 0.42) and increased by 0.5 and 1.0 in SPMS and PPMS, respectively (p = 0.10 and 0.25). Infusion-related AEs occurred during 7.8% of infusions and most were mild. A total of 89 AEs grades ≥2 (of which 76 infections) were recorded in 72 patients. No case of progressive multifocal leukoencephalopathy was detected. This is the largest cohort of patients with MS treated with rituximab reported so far. The safety, clinical, and MRI findings in this heterogeneous real-world cohort treated with different doses of rituximab were similar to those reported in previous randomized controlled trials on B-cell depletion therapy in MS. This study provides Class IV evidence that for patients with MS, rituximab is safe and effective.