Haemophilia : the official journal of the World Federation of Hemophilia · 2025
Safety, Efficacy and Treatment Patterns of rIX-FP in Previously Untreated Paediatric Haemophilia B Patients: A Retrospective Chart Review in Japan.
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Nogami Keiji, Nagae Chiai, Yono Masatoshi, Terasaka Naoki
Аннотация
BACKGROUND: Limited 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.
AIMS: To evaluate the treatment patterns, safety and efficacy of rIX-FP in paediatric PUPs in Japan.
METHODS: Retrospective chart review of 21 PUPs who initiated rIX-FP between May 2017 and September 2023, with follow-up to June 2024.
RESULTS: At 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.
CONCLUSION: This study provides real-world insights into rIX-FP use in paediatric PUPs, demonstrating its favourable safety, effective prophylaxis, and lower IVR in infants.
AIMS: To evaluate the treatment patterns, safety and efficacy of rIX-FP in paediatric PUPs in Japan.
METHODS: Retrospective chart review of 21 PUPs who initiated rIX-FP between May 2017 and September 2023, with follow-up to June 2024.
RESULTS: At 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.
CONCLUSION: This study provides real-world insights into rIX-FP use in paediatric PUPs, demonstrating its favourable safety, effective prophylaxis, and lower IVR in infants.