Long-term effectiveness of eculizumab: Data from the International PNH Registry

Terriou, L; Lee, JW; Forsyth, C; Griffin, M; Szer, J; Roth, A; Gustovic, P; Metzger, J; Patel, AS; Patriquin, CJ

Author(s): Terriou, L; Lee, JW; Forsyth, C; Griffin, M; Szer, J; Roth, A; Gustovic, P; Metzger, J; Patel, AS; Patriquin, CJ;
Details: Publication Year 2023-11,Volume 111,Issue #5,Page 796-804
Journal Title: European Journal of Haematology
Publication Type: Research article
Abstract: OBJECTIVES: Data from the International PNH Registry (NCT01374360) were used to estimate the overall survival and first occurrence of thromboembolic events/major adverse vascular events (TEs/MAVEs) for eculizumab-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) compared with a contemporaneous untreated cohort. METHODS: Patients enrolled in the Registry from March 16, 2007, to February 14, 2022, were included. Treated patients received eculizumab for >35 days; untreated patients did not receive eculizumab at any time. Univariable and multivariable analyses were performed using a Cox proportional hazards regression model comparing eculizumab treatment periods to untreated periods and were adjusted for baseline covariates (e.g., high disease activity [HDA], transfusion dependency, and eculizumab treatment status). RESULTS: The analysis included 4118 patients. The univariable hazard ratio (HR) (95% CI) for mortality in eculizumab-treated time versus untreated time was 0.51 (0.41-0.64; p < 0.0001). Significant baseline covariates included age, sex, history of bone marrow failure, >/=4 erythrocyte transfusions within 12 months before baseline, and an estimated glomerular filtration rate </= 60 mL/min/1.73 m(2) (all p < 0.0001). In the adjusted analysis, patients with baseline HDA had the greatest reduction in mortality risk (HR [95% CI], 0.51 [0.36-0.72]). Treated patients had approximately 60% reduction in TE/MAVE risk during treated versus untreated time (HR [95% CI]: TE: 0.40 [0.26-0.62], MAVE: 0.37 [0.26-0.54]; p < 0.0001). CONCLUSION: Using data from the largest Registry of patients with PNH, with >/=14 years of overall follow-up, we demonstrate that treatment with eculizumab conferred a 49% relative benefit in survival and an approximately 60% reduction in TE/MAVE risk.
Publisher: Wiley
Keywords: Humans; Infant; *Hemoglobinuria, Paroxysmal/diagnosis/drug therapy/epidemiology; Antibodies, Monoclonal, Humanized/adverse effects; Erythrocyte Transfusion; Registries; C5 inhibitor; PNH Registry; major adverse vascular events; real-world; survival; thromboembolic events
Department(s): Clinical Haematology
PubMed ID: 37712908
Publisher's Version: https://doi.org/10.1111/ejh.14080
Open Access at Publisher's Site: https://doi.org/10.1111/ejh.14080
Terms of Use/Rights Notice: Refer to copyright notice on published article.

Creation Date: 2023-11-21 05:54:37

Last Modified: 2023-11-21 05:55:40