Haemophilia. 2025 Nov 28. doi: 10.1111/hae.70177. Online ahead of print.
ABSTRACT
BACKGROUND: Modern prophylaxis in haemophilia aims not only to prevent bleeding but also to ensure a quality of life (QoL) comparable to healthy peers. Real-world data on health-related QoL (HRQoL) in children receiving personalised pharmacokinetic (PK)-guided prophylaxis are limited.
AIM: To compare HRQoL outcomes in Polish children with haemophilia A who switched from standard plasma-derived factor VIII (pdFVIII) prophylaxis to recombinant factor VIII (rFVIII), either as standard or PK-tailored prophylaxis.
METHODS: In this nationwide study, 131 boys with severe or moderate haemophilia A were assessed before and ≥26 weeks after switching from pdFVIII to rFVIII. Patients either continued standard prophylaxis (Group 1, n = 28) or started PK-tailored prophylaxis (Group 2, n = 103). Bleeding outcomes (annualised bleeding rate [ABR], annualised joint bleeding rate [AJBR]) and QoL (EQ-5D-5L, Visual Analogue Scale [VAS]) were compared using Student’s t-test and Wilcoxon test.
RESULTS: Switching to rFVIII significantly reduced ABR (1.73→0.76; p = 0.004) and AJBR (0.65→0.33; p = 0.009). QoL improved overall, with higher VAS scores (88.6→91.2; p = 0.02) and lower EQ-5D-5L scores (5.78→5.54; p = 0.01). Improvements were most pronounced in Group 2, where both bleeding rates and QoL significantly improved, despite a higher weekly number of i.v. injections. No significant QoL changes were observed in Group 1.
CONCLUSION: Personalised PK-based rFVIII prophylaxis leads to significant improvements in bleeding control and HRQoL in Polish children with haemophilia A. These results support the integration of PK-guided prophylaxis into routine clinical practice.
PMID:41313624 | DOI:10.1111/hae.70177
