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

Autor: Kivitz A; Altoona Centre for Clinical Research, 175 Meadowbrook Lane, Duncansville, PA, 16635-8445, USA. ajkivitz@yahoo.com., Gottenberg JE; Department of Rheumatology, Hôpitaux de Hautepierre, Strasbourg, France., Bergman M; Department of Medicine, Drexel University College of Medicine, Philadelphia, PA, USA., Qiu C; Sanofi, Bridgewater, NJ, USA., van Hoogstraten H; Sanofi, Bridgewater, NJ, USA., de Nijs R; Department of Rheumatology, Elkerliek Hospital, Helmond, The Netherlands., Bessette L; Department of Medicine, Université Laval, Québec, Québec, Canada.
Jazyk: angličtina
Zdroj: Rheumatology and therapy [Rheumatol Ther] 2024 Dec; Vol. 11 (6), pp. 1533-1550. Date of Electronic Publication: 2024 Sep 16.
DOI: 10.1007/s40744-024-00715-9
Abstrakt: 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.
Competing Interests: Declarations Conflict of Interest Alan Kivitz: Shareholder of Pfizer, Sanofi, GlaxoSmithKline, Gilead Sciences, Inc., and Novartis; paid consultant for AbbVie, Boehringer Ingelheim, Flexion, Janssen, Pfizer, Sanofi, Regeneron, Sun Pharma Advanced Research, and Gilead Sciences, Inc.; speaker and/or a member of speakers’ bureau for Celgene, Merck, Lilly, Novartis, Pfizer, Sanofi, Flexion, and AbbVie. Jacques Eric Gottenberg: Received research grants from Bristol-Myers Squibb, Pfizer, and Roche and speaking/consulting fees from AbbVie, Bristol-Myers Squibb, Chugai, Galapagos, Gilead, Janssen, Lilly, MSD, Pfizer, Sanofi, Roche, and UCB. Martin Bergman: Consultant, advisor, and member of speakers’ bureaus for AbbVie, Amgen, BMS, Genentech/Roche, Gilead, GSK, Janssen, Merck, Novartis, Pfizer, Sandoz, and Sanofi/Regeneron; and a shareholder of Johnson and Johnson (parent company of Janssen). Chunfu Qiu and Hubert van Hoogstraten: Employees of Sanofi and may hold stock and/or stock options in the company. Ron de Nijs has nothing to disclose. Louis Bessette: Received research grants and speaker and consulting fees from AbbVie, Amgen, Bristol-Myers Squibb, Celgene, Eli Lilly, Janssen, Merck, Novartis, Pfizer, Roche, Sanofi, and UCB. Ethical Approval The study protocol was approved by the institutional review boards/ethics committees (IRBs/ECs), and each enrolled patient provided written informed consent. The study was conducted in accordance with the principles laid by the 18th World Medical Assembly (Helsinki, 1964) and all subsequent amendments.
(© 2024. The Author(s).)
Databáze: MEDLINE
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