Assessing prophylactic use and clinical outcomes in hemophilia A patients treated with rVIII-SingleChain and other common rFVIII products in Germany

Authors: Olivieri, M., Sommerer, P., Maro, G., and Yan, S.

Published: Eur.J Haematol.; January 2020

Affiliations: Paediatric Haemophilia Centre, Department of Paediatrics, Dr. von Hauner Children’s Hospital, Ludwig-Maximilians-Universitat Munchen, Munich, Germany ; CSL Behring, Hattersheim, Germany ; Adivo Associates, San Francisco, CA ; CSL Behring, King of Prussia, PA, USA.

Abstract: OBJECTIVE: To evaluate real-world outcomes with rVIII-SingleChain and other commonly used recombinant FVIII (rFVIII) products. METHODS: Hemophilia treatment centers in Germany (n=21) contributed patient chart data. Inclusion criteria were prophylactic treatment with one of five rFVIII products for >/=8 weeks. RESULTS: Male patients (n=225) were included: rVIII-SingleChain (n=40), rFVIIIFc (n=47), octocog alfa (rFVIII) (n=58), octocog alfa (BAY 81-8973) (n=40), or moroctocog alfa (n=40). In patients with severe disease (n=76), 66.7%, 70.0%, 20.0%, 7.7% and 27.3% were dosed </=2x/week, respectively. Irrespective of dosing frequency, mean annualized bleed rates (ABRs)/annualized spontaneous bleed rates (AsBRs) were 0.3/0.1, 0.8/0.4, 1.1/0.5, 1.5/0.8 and 1.4/0.6, and mean FVIII consumption (IU/kg/week) was 83.2, 97.2, 92.5, 104.0 and 102.1, respectively. Results for all patients were similar. Of the patients on prophylaxis with prior therapy and after switching to rVIII-SingleChain (n=21), mean ABR/AsBRs were 0.7/0.3 and 0.2/0.0, respectively. After switching to rVIII-SingleChain, mean FVIII consumption reduced (109.4 vs. 74.5 IU/kg/week), and percentage of patients dosed </=2x/week increased (0% to 71.4%). CONCLUSIONS: rVIII-SingleChain prophylaxis provides excellent bleeding protection, with potentially lowest factor consumption among the products assessed. Patients who switched to rVIII-SingleChain prophylaxis reduced dosing frequency and consumption compared to prior treatment, with similar or potentially lower bleeding rates.