rFVIII‐Fc in severe haemophilia A: The incentive switch in case of high risk of joint bleedings

Abstract

Background Efmoroctocog alfa, the first recombinant factor VIII fusion protein with extended half‐life (rFVIII‐Fc), has been hypothesized to lower FVIII consumption in patients with severe Haemophilia A (pwSHA), without reducing clinical efficacy. What about real life? Method MOTHIF‐II was a noninterventional, multicentre, before/after study, via the collection of retrospective data from July 2015 to June 2016 (called T1), and from July 2017 to June 2018 (called T2), in 7 French haemophilia treatment centres. We examined the prescriptions and dispensations of factor VIII and the Annual Bleeding Rate (ABR), in pwSHA without current inhibitors on prophylaxis, before and after the introduction of rFVIII‐Fc. The data gathered from the BERHLINGO research database and from the French Healthcare claims database with a determinist pairing process based on the national unique identification number. Results A total of 156 pwSHA were included in the prescription cohort and 83 in the ABR cohort. For switched patients, the mean amounts of prescribed FVIII were significantly higher during T1 compared to T2 (4333 (2052) vs. 3921 (2029) IU/kg/year/patient, p: 0.028); a significant decrease in their ABR was also observed between T1 and T2 (6.3 (6.0) vs. 4.4 (5.4), p: 0.047). These patients had a more severe bleeding profile centred on haemarthrosis. Conclusion The results are related to those of the pivotal clinical trials for the reduction in FVIII consumption following the switch to rFVIII‐Fc, with a significant improvement in the haemorrhagic phenotype for pwSHA.

Evaluation of treatment and outcome for patients with haemophilia A and haemophilia B on extended half‐life (EHL) factor products: A 12‐month data analysis

“…The majority are 7 although other groups have reported a reduction in FVIII usage, though not substantial. 6,9,16 The reduction in FIX usage is consistent with other studies. 13,14,17,18 Changes in treatment regimen were clinical decisions and most commonly made for pharmacokinetic data, but this was concentrated in the paediatric cohort with adult treatment adjustments mostly occurring following bleeds or due to personal choice.…”

“…in 2021 15 and Dettoraki et al. in 2022, 7 although other groups have reported a reduction in FVIII usage, though not substantial 6,9,16 . The reduction in FIX usage is consistent with other studies 13,14,17,18 …”