The use of efmoroctocog alfa (Eloctate®), a recombinant factor VIII (FVIII) fusion protein with an extended half-life (rFVIII-Fc), reduced the annual bleeding rate (ABR) and consumption of prescription FVIII in patients with severe hemophilia A, according to a new study published in the European Journal of Clinical Investigation.
During the retrospective, noninterventional, before/after clinical study (MOTHIF-2), 156 patients with severe hemophilia A were included in a cohort where the rates of prescription and dispensation of FVIII were monitored from June 2015 to June 2016 (T1) and from July 2017 to June 2018 (T2). The second cohort of 83 patients was also observed for changes in ABR during these 2 time periods.
The majority of patients in the prescription cohort were receiving recombinant FVIII (rFVIII) from Chinese hamster ovary cells (CHO; 67%) during T1, with another 30% receiving rFVIII from baby hamster kidney cells. During T2, the predominant source was rFVIII-FC (55%), with CHO being reduced to 30%.
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The patients who switched to rFVIII-Fc after T1 showed a general decrease in injection frequency, with 53 out of 156 patients receiving 3 injections per week during T1, which reduced to only 33 patients in T2 (P =.016). The number of patients receiving injections 2 times per week climbed from 75 during T1 to 96 during T2.
In the 83 patients in whom ABR was monitored, 23 received only rFVIII-Fc during T2 and showed a significant decrease in ABR from 6.4 to 4.0 (P =.00758). Patients who maintained standard half-life (SHL) treatment had similar mean ABRs between T1 and T2 (3.0 vs 3.2, respectively). It was observed that patients who did not switch to rFVIII-Fc had a lower rate of hemarthrosis than those who did.
“Our study provides an opportunity to highlight the patients who keep the same SHL FVIII therapy and shows that they have a lower primary ABR,” the authors said. “They actually seem well balanced by their prophylaxis which is why they stayed with standard FVIII, a therapy considered to be sufficiently effective. In contrast, the patients switched to rFVIII-Fc seem to have a more ‘bleeder’ phenotype.”
Horvais V, Wargny M, Repessé Y, et al. rFVIII-Fc in severe haemophilia A: the incentive switch in case of high risk of joint bleedings. Eur J Clin Invest. Published online June 14, 2022. doi:10.1111/eci.13824