Prophylaxis with recombinant factor IX Fc fusion protein reduces the risk of bleeding and delays time to first spontaneous bleed event in previously untreated patients with haemophilia B: A post hoc analysis of the PUPs B-LONG study.

Autor: Nolan B; Haematology Department, Children's Health Ireland at Crumlin, Dublin, Republic of Ireland., Recht M; National Bleeding Disorders Foundation, New York City, New York, USA.; Yale University School of Medicine, New Haven, Connecticut, USA., Rendo P; Sanofi, Waltham, Massachusetts, USA., Falk A; Sobi, Stockholm, Sweden., Foster M; Sanofi, Cambridge, Massachusetts, USA., Casiano S; Sanofi, Cambridge, Massachusetts, USA., Rauch A; Department of Hematology and Transfusion, CHU Lille, Lille, France., Shapiro A; Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, USA.
Jazyk: angličtina
Zdroj: European journal of haematology [Eur J Haematol] 2024 Oct; Vol. 113 (4), pp. 485-492. Date of Electronic Publication: 2024 Jun 25.
DOI: 10.1111/ejh.14252
Abstrakt: Objectives: Haemophilia B (HB), characterised by deficient factor IX (FIX), leads to spontaneous bleeds. Severe cases require prophylactic FIX replacement. This post hoc analysis assessed the first spontaneous bleeds among previously untreated patients (PUPs) with HB treated with recombinant FIX Fc fusion protein (rFIXFc) (NCT02234310) to identify factors influencing bleeds.
Methods: Subjects included paediatric PUPs with HB (≤2 IU/dL endogenous FIX). Analyses described treatment patterns (on demand [OD] vs. prophylaxis) and prophylaxis type (started on vs. switched to prophylaxis). Kaplan-Meier analyses assessed the time to first spontaneous bleed, including median time to event and fitting models with predictors for treatment regimen and/or baseline age.
Results: PUPs B-LONG enrolled 33 subjects. Baseline age did not influence the time to first spontaneous bleed for any rFIXFc regimen. Those who started on prophylaxis with rFIXFc (n = 11), compared with those treated OD (n = 22), had an extended time to first spontaneous bleed. Starting prophylaxis afforded a 93% reduced risk of first spontaneous bleed versus starting OD (hazard ratio [95% confidence interval]: 0.071 [0.009-0.592]) (p = .015).
Conclusion: rFIXFc prophylaxis, particularly starting early, reduced the risk of bleeding and delayed time to first spontaneous bleed compared with rFIXFc OD. Hence, initial treatment regimens impact bleed patterns in paediatric PUPs.
(© 2024 Sanofi and The Author(s). European Journal of Haematology published by John Wiley & Sons Ltd.)
Databáze: MEDLINE
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