在接受那他珠单抗治疗的早期复发缓解型多发性硬化症患者中，认知处理速度、残疾和患者报告结局的改善：一项为期 4 年的真实世界、开放标签研究结果。

Improvements in Cognitive Processing Speed, Disability, and Patient-Reported Outcomes in Patients with Early Relapsing-Remitting Multiple Sclerosis Treated with Natalizumab: Results of a 4-year, Real-World, Open-Label Study.

机构信息

Weill Cornell Medical College, Cornell University, 1305 York Ave #2F, New York, NY, 10065, USA.

Northwestern University, Chicago, IL, USA.

出版信息

CNS Drugs. 2022 Sep;36(9):977-993. doi: 10.1007/s40263-022-00950-0. Epub 2022 Sep 5.

BACKGROUND

STRIVE was a prospective, 4-year, multicenter, observational, open-label, single-arm study of natalizumab treatment in anti-JC virus antibody-negative patients with early relapsing-remitting multiple sclerosis (RRMS).

OBJECTIVE

Study objectives examined the effects of natalizumab on cognitive processing speed, confirmed disability improvement (CDI), and patient-reported outcomes (PROs).

METHODS

Clinical and PRO secondary endpoints were assessed annually over 4 years in STRIVE. The Symbol Digit Modalities Test (SDMT) was used as a measure of cognitive processing speed. PROs were assessed using the Multiple Sclerosis Impact Score (MSIS-29) and the Work Productivity and Activity Impairment Questionnaire (WPAI).

RESULTS

At all four annual assessments, the proportion of patients in the intent-to-treat (ITT) population (N = 222) who exhibited clinically meaningful improvement in their SDMT score from baseline (i.e., change ≥ 4 points) ranged from 41.9 to 54.0%. The cumulative probability of CDI at 4 years in patients in the ITT population with a baseline Expanded Disability Status Scale score ≥ 2 (N = 133) was 43.9%. Statistically significant reductions in the mean change from screening in the MSIS-29 physical and psychological scores, indicating improved quality of life, were observed over all 4 years (P ≤ 0.0012 for all). A statistically significant decrease from screening in the impact of MS on regular activities, signifying an improvement in this WPAI measure, was also observed over all 4 years of the study.

CONCLUSION

These results further extend our knowledge of the effectiveness, specifically regarding improvements in cognitive processing speed, disability and PROs, of long-term natalizumab treatment in early RRMS patients.

CLINICALTRIALS

GOV: NCT01485003 (5 December 2011).

背景

STRIVE 是一项为期 4 年、多中心、观察性、开放性、单臂研究，旨在评估那他珠单抗治疗抗-JC 病毒抗体阴性的早期复发缓解型多发性硬化（RRMS）患者的疗效。

目的

研究目的评估那他珠单抗对认知处理速度、确认残疾改善（CDI）和患者报告的结局（PROs）的影响。

方法

STRIVE 研究中每年评估一次临床和 PRO 次要终点。符号数字模态测试（SDMT）用于评估认知处理速度。使用多发性硬化影响评分（MSIS-29）和工作生产力和活动障碍问卷（WPAI）评估 PROs。

结果

在所有 4 年的评估中，意向治疗（ITT）人群（N = 222）中，有 41.9%至 54.0%的患者从基线开始，其 SDMT 评分有临床意义的改善（即变化≥4 分）。在基线扩展残疾状态量表评分≥2 的 ITT 人群中（N = 133），4 年内 CDI 的累积概率为 43.9%。在所有 4 年的研究中，MSIS-29 身体和心理评分的平均变化从筛查时开始呈显著降低，表明生活质量得到改善（所有 P ≤ 0.0012）。在所有 4 年的研究中，也观察到 WPAI 中衡量常规活动的 MS 影响指标的显著降低，表明该指标得到改善。