Bleeding Episodes in Patients With Haemophilia B Receiving Prophylactic Factor IX Treatment: A Systematic Review and Meta‐Analysis

Abstract

ABSTRACTBackgroundProphylaxis with coagulation factor concentrates is the mainstay of treatment in severe hemophilia A and B. Data on bleeding rates in persons with congenital haemophilia B (PwcHB) receiving prophylaxis are inconsistent.AimThis systematic review and meta‐analysis were aimed at assessing bleeding outcomes, including annualised bleeding rates (ABR) and the proportion of patients with zero bleeding events, in PwcHB receiving prophylaxis with plasma‐derived or recombinant FIX products with standard (rSHL) or extended half‐life (rEHL).MethodsA systematic search was conducted using the bibliographic database Medline, Embase and Cochrane Central Register. The protocol was registered on PROSPERO (registration number: CRD42024592785).ResultsThe search yielded 2440 citations and a total of 42 studies (2 randomised and 40 nonrandomised) were included in the final analysis. The pooled estimated mean (95% confidence interval [CI]) ABR was significantly lower in PwcHB treated prophylactically with rEHL FIX than in those receiving rSHL FIX products (1.29 [95% CI: 0.91, 1.66] vs. 3.12 [95% CI: 2.63, 3.62], p < 0.01). The proportion of participants with zero bleeding events was significantly higher in PwcHB treated prophylactically with rEHL FIX than in those receiving rSHL FIX (0.53 [95% CI: 0.37, 0.69] vs. 0.24 [95% CI: 0.14, 0.39], p = 0.01). The ABR did not differ according to age groups (more or less than 12 years).ConclusionThe results of this meta‐analysis suggest that compared to standard half‐life FIX concentrates, prophylaxis with rEHL FIX products is associated with a reduction in ABR and a higher proportion of patients with no bleeding episodes.