沙利鲁单抗对 RA 的实际疗效：开放标签、前瞻性、单臂观察 PROFILE 研究的结果

IF 2.9 3区医学 Q2 RHEUMATOLOGY Rheumatology and Therapy Pub Date : 2024-09-16 DOI:10.1007/s40744-024-00715-9

Alan Kivitz, Jacques Eric Gottenberg, Martin Bergman, Chunfu Qiu, Hubert van Hoogstraten, Ron de Nijs, Louis Bessette

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引用次数: 0

摘要

导言：为期1年的PROspective sarilumab（preFILled注射器/笔）跨国观察（PROFILE）研究评估了sarilumab在中重度类风湿关节炎（RA）患者中的实际有效性和安全性。方法安全性终点包括不良事件（AE）和实验室异常。安全性终点包括不良事件（AEs）和实验室异常，有效性终点包括 ACR 核心指标。主要终点是临床疾病活动指数（CDAI）与基线相比的变化。结果共有595名患者接受了治疗，其中223人（37.5%）接受了sarilumab单药治疗，372人（62.5%）接受了联合治疗。开始使用沙利单抗后，CDAI平均（标清）评分在第24周[11.4 (10.3)]出现改善，并维持到第52周[10.0 (10.5)]，平均（标清）降幅分别为-14.9 (12.7)和-14.4 (12.9)。接受单一疗法和联合疗法的患者的疾病活动性均有持续改善，且改善程度相似。据报道，达到缓解和低疾病活动度的患者比例有所增加。到第52周时，两组患者的身体功能和生活质量均有所改善。没有出现新的安全性信号。报告治疗突发不良事件（TEAE）或严重治疗突发不良事件（SAE）的患者比例分别为66.2%和5.9%，两个治疗组之间的比例相似。总体而言，15.6%的患者因TEAE而中断了沙利单抗治疗。结论在这项为期1年的观察性真实世界研究中，sarilumab治疗改善了临床疗效。该研究于2018年1月11日登录德国网站（保罗-埃利希研究所），NIS编号：423。

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Real-world Effectiveness of Sarilumab in RA: Results from the Open-label, Prospective, Single-arm Observational PROFILE Study

Introduction

The 1-year PROspective sarilumab (preFILled syringe/pen) multinational, obsErvational (PROFILE) study evaluated the real-world effectiveness and safety of sarilumab in patients with moderate-to-severe rheumatoid arthritis (RA).

Methods

Safety endpoints included adverse events (AEs) and lab abnormalities. Effectiveness endpoints included the ACR core set. The primary endpoint was the change from baseline in Clinical Disease Activity Index (CDAI). All statistics are descriptive and p values were nominal.

Results

In total, 595 patients were treated, of whom 223 (37.5%) received sarilumab monotherapy and 372 (62.5%) received combination therapy. Upon initiation of sarilumab, an improvement in the mean (SD) CDAI score was observed at week 24 [11.4 (10.3)] and was maintained through week 52 [10.0 (10.5)], resulting in a mean [SD] reduction of −14.9 (12.7) and −14.4 (12.9), respectively. There were consistent improvements in disease activity that were similar for patients on monotherapy vs. combination therapy. An increase in the proportion of patients achieving remission and low disease activity was reported. By week 52, both groups had improved physical function and quality of life. There were no new safety signals. The proportions of any patients reporting a treatment-emergent adverse event (TEAE) or serious treatment-emergent AE (SAE) was 66.2% and 5.9%, respectively, and were similar between both treatment groups. Overall, 15.6% of patients discontinued sarilumab treatment due to TEAEs. The most commonly reported TEAE of interest was neutropenia (14.1%).

Conclusions

In this 1-year, observational real-world study, sarilumab therapy resulted in improved clinical outcomes. The safety profile was consistent with that observed in sarilumab randomized clinical trials.

This study was entered on the German website (Paul Ehrlich Institute) on January 11, 2018, with NIS No.: 423.

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来源期刊

Rheumatology and Therapy RHEUMATOLOGY-

CiteScore

6.00

自引率

5.30%

发文量

审稿时长

6 weeks

期刊介绍： Aims and Scope Rheumatology and Therapy is an international, open access, peer reviewed, rapid publication journal dedicated to the publication of high-quality clinical (all phases), observational, real-world and health outcomes research around the discovery, development, and use of rheumatologic therapies. Studies relating to diagnosis, pharmacoeconomics, public health, quality of life, and patient care, management, and education are also welcomed. Areas of focus include, but are not limited to, rheumatoid arthritis, gout, gouty arthritis, psoriatic arthritis, osteoarthritis, juvenile idiopathic/rheumatoid arthritis, systemic lupus erythematosus, axial spondyloarthritis, Pompe’s disease, inflammatory joint conditions, musculoskeletal conditions, systemic sclerosis, and fibromyalgia. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, case reports, trial protocols, communications and letters. 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