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

Abstract 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 (...

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Main Authors: Alan Kivitz, Jacques Eric Gottenberg, Martin Bergman, Chunfu Qiu, Hubert van Hoogstraten, Ron de Nijs, Louis Bessette
Format: Article
Language:English
Published: Adis, Springer Healthcare 2024-09-01
Series:Rheumatology and Therapy
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Online Access:https://doi.org/10.1007/s40744-024-00715-9
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author Alan Kivitz
Jacques Eric Gottenberg
Martin Bergman
Chunfu Qiu
Hubert van Hoogstraten
Ron de Nijs
Louis Bessette
author_facet Alan Kivitz
Jacques Eric Gottenberg
Martin Bergman
Chunfu Qiu
Hubert van Hoogstraten
Ron de Nijs
Louis Bessette
author_sort Alan Kivitz
collection DOAJ
description Abstract 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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spelling doaj-art-f98da2876c6e42148dfe1f0f8228c4792024-11-17T12:48:20ZengAdis, Springer HealthcareRheumatology and Therapy2198-65762198-65842024-09-011161533155010.1007/s40744-024-00715-9Real-world Effectiveness of Sarilumab in RA: Results from the Open-label, Prospective, Single-arm Observational PROFILE StudyAlan Kivitz0Jacques Eric Gottenberg1Martin Bergman2Chunfu Qiu3Hubert van Hoogstraten4Ron de Nijs5Louis Bessette6Altoona Centre for Clinical ResearchDepartment of Rheumatology, Hôpitaux de HautepierreDepartment of Medicine, Drexel University College of MedicineSanofiSanofiDepartment of Rheumatology, Elkerliek HospitalDepartment of Medicine, Université LavalAbstract 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.https://doi.org/10.1007/s40744-024-00715-9Antirheumatic agentsPatient-reported outcome measuresQuality of lifeReal-worldRheumatoid arthritisSarilumab
spellingShingle Alan Kivitz
Jacques Eric Gottenberg
Martin Bergman
Chunfu Qiu
Hubert van Hoogstraten
Ron de Nijs
Louis Bessette
Real-world Effectiveness of Sarilumab in RA: Results from the Open-label, Prospective, Single-arm Observational PROFILE Study
Rheumatology and Therapy
Antirheumatic agents
Patient-reported outcome measures
Quality of life
Real-world
Rheumatoid arthritis
Sarilumab
title Real-world Effectiveness of Sarilumab in RA: Results from the Open-label, Prospective, Single-arm Observational PROFILE Study
title_full Real-world Effectiveness of Sarilumab in RA: Results from the Open-label, Prospective, Single-arm Observational PROFILE Study
title_fullStr Real-world Effectiveness of Sarilumab in RA: Results from the Open-label, Prospective, Single-arm Observational PROFILE Study
title_full_unstemmed Real-world Effectiveness of Sarilumab in RA: Results from the Open-label, Prospective, Single-arm Observational PROFILE Study
title_short Real-world Effectiveness of Sarilumab in RA: Results from the Open-label, Prospective, Single-arm Observational PROFILE Study
title_sort real world effectiveness of sarilumab in ra results from the open label prospective single arm observational profile study
topic Antirheumatic agents
Patient-reported outcome measures
Quality of life
Real-world
Rheumatoid arthritis
Sarilumab
url https://doi.org/10.1007/s40744-024-00715-9
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