Title :

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

Author(s) :

Terriou, Louis [Auteur]
Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286
Lee, Jong Wook [Auteur]
Catholic University of Korea
Forsyth, Cecily [Auteur]
Griffin, Morag [Auteur]
Leeds Teaching Hospitals NHS Trust
Szer, Jeff [Auteur]
Peter MacCallum Cancer Centre
Röth, Alexander [Auteur]
University Hospital Essen [AöR]
Gustovic, Philippe [Auteur]
AstraZeneca
Metzger, Jesse [Auteur]
Patel, Ami S. [Auteur]
AstraZeneca
Patriquin, Christopher J. [Auteur]
Toronto General Hospital Research Institute [Canada] [TGHRI]

Journal title :

European Journal of Haematology

Abbreviated title :

Eur J Haematol

Publication date :

2023-09-16

ISSN :

1600-0609

English keyword(s) :

C5 inhibitor
major adverse vascular events
PNH Registry
real-world
survival
thromboembolic events

HAL domain(s) :

Sciences du Vivant [q-bio]

English abstract : [en]

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 ...
Show more >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 m2 (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.Show less >

Language :

Anglais

Audience :

Internationale

Popular science :

Non

Administrative institution(s) :

Université de Lille
Inserm
CHU Lille

Collections :

• Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286

Submission date :

2024-02-09T22:21:10Z
2024-02-28T15:21:36Z