ArticleHaemophilia : the official journal of the World Federation of Hemophilia
Safety, Efficacy and Treatment Patterns of rIX-FP in Previously Untreated Paediatric Haemophilia B Patients: A Retrospective Chart Review in Japan.
Article in Haemophilia : the official journal of the World Federation of Hemophilia. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
backgroundLimited real-world data exist on recombinant fusion protein-linking coagulation factor IX (FIX) with albumin (rIX-FP) in paediatric previously untreated patients (PUPs) with haemophilia B, particularly in infants.
aimsTo evaluate the treatment patterns, safety and efficacy of rIX-FP in paediatric PUPs in Japan.
methodsRetrospective chart review of 21 PUPs who initiated rIX-FP between May 2017 and September 2023, with follow-up to June 2024.
resultsAt first administration, 95% (19/20) of patients were <2 years old, and 84% (16/19) weighed <10 kg. The mean treatment duration was 27 months. Prophylaxis was received by 90% (18/20), with 56% (10/18) on every 2 weeks and 44% (8/18) on a weekly regimen. The proportion of patients with ≥25 exposure days was 100% in the weekly and 90% in every 2 weeks groups. No patients developed inhibitors or discontinued treatment. Among those on prophylaxis, no bleeding events occurred in patients ≥2 years, while 88% (15/17) of those <2 years experienced superficial perioral, or subcutaneous bleeding, with 2/15 requiring treatment. Every 2 weeks regimen had lower FIX trough levels than the weekly regimen (median: 4% vs. 10%) and a higher annualized bleeding rate (ABR: 8.2 vs. 4.9). The most common bleeding type was traumatic (ABR: 4.8), followed by spontaneous (1.6) and undetermined (0.7). All treated bleeds resolved successfully. In vivo recovery (IVR) positively correlated with body weight in patients <10 kg.
conclusionThis study provides real-world insights into rIX-FP use in paediatric PUPs, demonstrating its favourable safety, effective prophylaxis, and lower IVR in infants.
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