在真实环境中聚乙二醇干扰素 β-1a 治疗复发型多发性硬化症的安全性和临床疗效：Plegridy 观察性计划的中期结果。
Safety and clinical effectiveness of peginterferon beta-1a for relapsing multiple sclerosis in the real-world setting: Interim results from the Plegridy Observational Program.
机构信息
Sapienza University, S. Andrea Hospital, Rome, Italy; IRCCS Neuromed, Pozzilli, Italy.
Hope Neurology MS Center, Knoxville, TN, United States.
出版信息
Mult Scler Relat Disord. 2022 Jan;57:103350. doi: 10.1016/j.msard.2021.103350. Epub 2021 Oct 29.
BACKGROUND
The Plegridy Observational Program (POP) is an ongoing, 5-year, phase 4 real-world study of the safety and effectiveness of subcutaneous peginterferon beta-1a in patients with relapsing multiple sclerosis (RMS).
METHODS
This interim analysis from POP assessed the safety and effectiveness of peginterferon beta-1a, including subgroup analyses of patients aged < 50 and ≥ 50 years, newly diagnosed and non-newly diagnosed patients, and new and experienced peginterferon beta-1a users.
RESULTS
A total of 1208 patients enrolled in POP. Mean (standard deviation) peginterferon treatment duration in the overall population was 757.0 (529.5) days. The overall incidence of treatment-emergent adverse events (AEs) was 65.5%, and the incidence was higher in new than experienced peginterferon beta-1a users (78.1 vs 52.4%). The overall incidence of treatment-emergent serious AEs was 7.6%, and the incidence was lower in younger than older patients (5.8 vs 11.1%). No new or unexpected safety signals were reported. Overall treatment discontinuation due to AEs occurred in 20.7% of patients, with a higher proportion of new than experienced peginterferon beta-1a users (27.0 vs 14.2%) discontinuing treatment due to AEs. Flu-like symptoms and injection site reactions were significant predictors of time to treatment discontinuation. The adjusted annualized relapse rate (ARR) was 0.12 (95% confidence interval 0.11-0.13) in the overall population and was similar across all subgroups. In the overall population at 4 years, 79.1% of patients were relapse free, the estimated cumulative proportion of patients with confirmed disability worsening was 1.8%, and > 67% of patients achieved clinical no evidence of disease activity (NEDA).
CONCLUSIONS
Safety data of patients enrolled in POP are consistent with the established clinical safety profile of peginterferon beta-1a. In addition, the low ARR and high proportion of patients achieving clinical NEDA at 4 years across all subgroups indicates the effectiveness of peginterferon beta-1a in treating RMS in real-world clinical settings.
背景
Plegridy 观察计划（POP）是一项正在进行的、为期 5 年的、4 期真实世界研究，旨在评估皮下注射聚乙二醇干扰素 β-1a 在复发型多发性硬化症（RMS）患者中的安全性和有效性。
方法
本研究为 POP 的中期分析，评估了聚乙二醇干扰素 β-1a 的安全性和有效性，包括对年龄<50 岁和≥50 岁、新诊断和非新诊断患者以及新和经验丰富的聚乙二醇干扰素 β-1a 使用者的亚组分析。
结果
共有 1208 名患者入组 POP。总体人群中聚乙二醇干扰素治疗持续时间的平均值（标准差）为 757.0（529.5）天。总体治疗中出现的不良事件（AE）发生率为 65.5%，新使用者的发生率高于经验使用者（78.1%比 52.4%）。总体治疗中出现的严重不良事件发生率为 7.6%，年轻患者的发生率低于老年患者（5.8%比 11.1%）。未报告新的或意外的安全性信号。由于 AE 导致的总体治疗中止率为 20.7%，新使用者的比例高于经验使用者（27.0%比 14.2%）。流感样症状和注射部位反应是治疗中止时间的重要预测因素。调整后的年复发率（ARR）在总体人群中为 0.12（95%置信区间 0.11-0.13），且在所有亚组中相似。在 4 年时，总体人群中有 79.1%的患者无复发，估计有残疾恶化的确诊患者累积比例为 1.8%，超过 67%的患者达到临床无疾病活动（NEDA）。
结论
POP 中入组患者的安全性数据与聚乙二醇干扰素 β-1a 的既定临床安全性特征一致。此外，所有亚组在 4 年时的低 ARR 和高比例的患者达到临床 NEDA 表明，聚乙二醇干扰素 β-1a 在真实世界临床环境中治疗 RMS 的有效性。
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