Real-world analysis of extended half-life product posology in hemophilia A: results from a retrospective analysis of medical chart and claims data.

BACKGROUND

Extended half-life (EHL) products have changed the treatment landscape of patients with hemophilia as patients maintain protective FVIII levels with minimal occurrence of spontaneous bleeding.

RESEARCH DESIGN AND

METHODS

Two independent datasets from the multi-country patient record form (PRF)-based study and PicnicHealth/Komodo Health study were analyzed to understand the real-world use of EHL products for patients with hemophilia A (HA).

RESULTS

Most patients receiving EHL prophylaxis were on per-label dosing. Those receiving individualized dosing had moderate to severe disease. EHL dose adjustment occurred in 20% of patients of which 14.3% experienced a second dose adjustment. Treatment efficacy was mainly monitored via annualized bleeding rate (ABR) with quality of life, tolerability, and treatment efficacy being the primary considerations for Healthcare Personnel when selecting treatment. Switching to a different EHL or standard half-life (SHL) product did not significantly reduce median ABR while switching to non-factor therapy significantly reduced median ABR from 5.2 to 0.94, p < 0.0001. Patients on individualized dosing had higher ABRs than those on per-label dosing both before index EHL treatment and while on EHL treatment, whereas individualization via dose adjustment was associated with significant median ABR reduction (p < 0.0001).

CONCLUSIONS

Individualization may support improved outcomes in patients unable to achieve satisfactory outcomes on a per-label dosing regimen.