Abstract Number: PB0953
Meeting: ISTH 2020 Congress
Background: Hemophilia A (HA) is a congenital Factor VIII (FVIII) deficiency and replacement treatment using recombinant coagulation FVIII is the gold-standard therapeutical approach. Over the last few years, many patients have been switched from standard recombinant products (SHL) to extended half-life recombinant factors (EHL). However, it remains unclear whether this switching approach actually reduces the number of infusions per year without increasing annual bleeding events and subsequent need for supplementary factor infusions.
Aims: to compare the number of infusions, bleeding episodes and annual FVIII IU consumption of HA patients on prophylaxis before and after switching from SHL to EHL.
Methods: we included patients on prophylaxis referring to our hemophilia center with severe/moderate HA who had been switched from SHL to EHL (FVIII:Fc). We excluded all “on demand” patients and previously untreated patients (PUPs) from the analysis. Variables are expressed as median and interquartile range (IQR). Wilcoxon test for paired groups was used (p< 0.05 statistically significant).
Results: twenty-nine patients with HA, median age 25 years [IQR 19-37, min-max 6-71], 93% with a severe disease, fulfilled the inclusion criteria and were included in the analysis. The median number of annual infusions was 156 per patient [IQR 156-182] before switching and decreased to 122 infusions per patient [IQR 91-122, p=0.0001] after switching to EHL, with a reduction of 22% of total treatments. Median annual unit consumption before switching was 312.000 IU per patient [IQR 208.000-461.500] and decreased to 244.000 IU per patient
[IQR 169.000-339.000, p< 0.002] after switch to EHL. The mean annual bleeding rate was reduced from 0.65 to 0.13 after the switch to EHL.
Conclusions: the use of the only available EHL product (FVIII:Fc) led to a reduced number of annual FVIII infusions and unit consumption per patient together with an improvement of the clinical outcomes.
To cite this abstract in AMA style:Braham S, Gualtierotti R, Biguzzi E, Siboni SM, Bregani E, Boscarino M, Mancuso ME, Peyvandi F. A Single Center Data on Extended Half-Life Recombinant Factor VIII (FVIII:Fc) in Patients with Hemophilia A [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/a-single-center-data-on-extended-half-life-recombinant-factor-viii-fviiifc-in-patients-with-hemophilia-a/. Accessed May 6, 2021.
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