Office of Chief Medical Officer, Johnson & Johnson, New Brunswick, New Jersey, USA.
Department of Statistics and Decision Sciences, Janssen Research & Development, LLC, Titusville, New Jersey, USA.
J Womens Health (Larchmt). 2024 Apr;33(4):480-490. doi: 10.1089/jwh.2023.0037. Epub 2024 Feb 1.
Multiple sclerosis (MS) is threefold more prevalent in women than men. However, sex-specific efficacy analysis for MS disease-modifying therapies is not typically performed. analyses of data from female patients enrolled in the phase 3, double-blind OPTIMUM study of relapsing MS were carried out. Eligible adults were randomized to ponesimod 20 mg or teriflunomide 14 mg once daily for up to 108 weeks. The primary endpoint was annualized relapse rate (ARR); secondary endpoints included change in symptom domain of Fatigue Symptom and Impact Questionnaire-Relapsing Multiple Sclerosis (FSIQ-RMS) at week 108, number of combined unique active lesions (CUALs) per year on magnetic resonance imaging, and time to 12- and 24-week confirmed disability accumulation (CDA). A total of 735 female patients (581 of childbearing potential) were randomized to ponesimod ( = 363, 49.4%) or teriflunomide ( = 372, 50.6%). Relative risk reduction in the ARR for ponesimod versus teriflunomide was 33.1% (mean, 0.192 vs. 0.286, respectively; < 0.002). Mean difference in FSIQ-RMS for ponesimod versus teriflunomide was -4.34 (0.12 vs. 4.46; = 0.002); rate ratio in CUALs per year, 0.601 (1.45 vs. 2.41; < 0.0001), and hazard ratio for time to 12- and 24-week CDA risk estimates, 0.83 (10.7% vs. 12.9%; = 0.38) and 0.91 (8.8% vs. 9.7%; = 0.69), respectively. Incidence of treatment-emergent adverse events was similar between treatment groups (89.0% and 90.1%). Analyses demonstrate the efficacy and safety of ponesimod, versus active comparator, for women with relapsing MS, supporting data-informed decision-making for women with MS. Clinical Trial Registration Number: NCT02425644.
多发性硬化症（MS）在女性中的患病率是男性的三倍。然而，通常不会针对 MS 疾病修正治疗进行特定于性别的疗效分析。对纳入 3 期、双盲 OPTIMUM 复发型 MS 研究的女性患者进行数据分析。符合条件的成年人被随机分配接受 poniesimod 20mg 或特立氟胺 14mg 每日一次，最长 108 周。主要终点是年化复发率（ARR）；次要终点包括第 108 周疲劳症状和影响问卷-复发型多发性硬化症（FSIQ-RMS）症状域的变化、每年联合独特活跃病变（CUALs）的数量以及 12 周和 24 周时确认残疾进展（CDA）的时间。共有 735 名女性患者（581 名有生育潜力）被随机分配至 poniesimod（n=363，49.4%）或 teriflunomide（n=372，50.6%）。poniesimod 与 teriflunomide 相比，ARR 的相对风险降低了 33.1%（平均值分别为 0.192 和 0.286； < 0.002）。poniesimod 与 teriflunomide 相比，FSIQ-RMS 的平均差异为-4.34（0.12 对 4.46； = 0.002）；每年 CUALs 的比率为 0.601（1.45 对 2.41； < 0.0001），12 周和 24 周 CDA 风险估计的风险比分别为 0.83（10.7%对 12.9%； = 0.38）和 0.91（8.8%对 9.7%； = 0.69）。治疗出现的不良事件发生率在治疗组之间相似（89.0%和 90.1%）。分析表明，poniesimod 对复发型 MS 女性的疗效和安全性不劣于活性对照药物，为 MS 女性提供了基于数据的决策支持。临床试验注册号：NCT02425644。
