Journal
HAEMOPHILIA
Volume 28, Issue 6, Pages E237-E244Publisher
WILEY
DOI: 10.1111/hae.14649
Keywords
annualized bleed rate; extended half-life; factor FVIII deficiency; haemophilia A; prophylaxis
Categories
Funding
- Helsinki University Hospital [TYH2020318]
- SOBI
- Takeda
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This study compared the clinical and laboratory outcomes between SHL and EHL FVIII prophylaxis in haemophilia A patients. The results showed that switching to EHL led to decreased infusion frequency and factor consumption, and excellent clinical efficacy.
Introduction Majority of haemophilia A patients in our comprehensive care centre have switched from standard half-life (SHL) to extended half-life (EHL) FVIII products in a short time. Aim We compared the clinical and laboratory outcomes between SHL and EHL FVIII prophylaxis in product switchers. Methods This is a retrospective inception cohort of all adult haemophilia A patients switched to EHL (rFVIIIFc or rFVIII-PEG) prophylaxis in our centre. Dosing, product utilization, annualized bleed rates (ABR), treatment regimen and pharmacokinetics by Web Accessible Population Pharmacokinetic Service (WAPPS)-Hemo were compared between SHL and EHL. Results We included 38 patients, whose median age was 38 years (range 17-75). Median FVIII dose was 23 IU/kg for SHL versus 25 IU/kg for EHL. After switching, weekly infusions decreased by 29% from median 2.8 (every 2.5 days) to 2.0 (every 3.5 days) (P = <.001) and factor consumption for prophylaxis by 17% from 60 to 50 IU/kg/week (P = <.001). Weekly infusions decreased in 71% and FVIII utilization in 55% of patients. ABR remained low (1.0 for SHL and .5 for EHL, respectively). In pharmacokinetics, the half-life of FVIII increased from median 13 to 21 h after switching. Times above .01 and .03 IU/ml improved from 85 to 131 h and from 65 to 106 h. Half-lives of the SHL products and von Willebrand factor levels predicted half-lives with the EHL products. Conclusions Our cohort study confirms the successful experience of switching to EHL FVIII products, with decreased infusion frequency, factor consumption and excellent clinical efficacy.
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