Kivitz Alan, Gottenberg Jacques Eric, Bergman Martin, Qiu Chunfu, van Hoogstraten Hubert, de Nijs Ron, Bessette Louis
Altoona Centre for Clinical Research, 175 Meadowbrook Lane, Duncansville, PA, 16635-8445, USA.
Department of Rheumatology, Hôpitaux de Hautepierre, Strasbourg, France.
Rheumatol Ther. 2024 Dec;11(6):1533-1550. doi: 10.1007/s40744-024-00715-9. Epub 2024 Sep 16.
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).
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.
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%).
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.
为期1年的前瞻性赛立珠单抗（预填充注射器/笔）多国观察性（PROFILE）研究评估了赛立珠单抗在中度至重度类风湿性关节炎（RA）患者中的实际疗效和安全性。
安全性终点包括不良事件（AE）和实验室异常。有效性终点包括美国风湿病学会（ACR）核心指标集。主要终点是临床疾病活动指数（CDAI）相对于基线的变化。所有统计数据均为描述性数据，P值为名义值。
总共595例患者接受了治疗，其中223例（37.5%）接受了赛立珠单抗单药治疗，372例（62.5%）接受了联合治疗。开始使用赛立珠单抗后，在第24周观察到平均（标准差）CDAI评分有所改善[11.4（10.3）]，并持续至第52周[10.0（10.5）]，平均[标准差]分别降低了-14.9（12.7）和-14.4（12.9）。单药治疗与联合治疗患者的疾病活动度均持续改善且相似。报告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。未发现新的安全信号。报告出现治疗中出现的不良事件（TEAE）或严重治疗中出现的不良事件（SAE）的患者比例分别为66.2%和5.9%，两个治疗组相似。总体而言，15.6%的患者因TEAE而停用赛立珠单抗治疗。最常报告的关注TEAE是中性粒细胞减少症（14.1%）。
在这项为期1年的观察性实际研究中，赛立珠单抗治疗改善了临床结局。安全性与赛立珠单抗随机临床试验中观察到的情况一致。本研究于2018年1月11日在德国网站（保罗·埃利希研究所）登记，编号为NIS：423。
