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CC BY-NC-ND 4.0 · Thromb Haemost 2021; 121(08): 1079-1086
DOI: 10.1055/a-1333-5536
Coagulation and Fibrinolysis

Decreased Bleeding Rates in Patients with Hemophilia A Switching from Standard-Half-Life FVIII to BAY 94-9027 Prophylaxis

Maria Elisa Mancuso 
1   Center for Thrombosis and Hemorrhagic Diseases, Humanitas Clinical and Research Center–IRCCS, Rozzano, Milan, Italy
,
Mark T. Reding
2   University of Minnesota Medical Center, Minneapolis, Minnesota, United States
,
Claude Negrier
3   Louis Pradel University Hospital, University Claude Bernard Lyon 1, Lyon, France
,
Bryce A. Kerlin
4   Nationwide Children's Hospital and The Ohio State University College of Medicine, Columbus, Ohio, United States
,
Savita Rangarajan
5   University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom
,
Mindy L. Simpson
6   Rush University Medical Center, Chicago, Illinois, United States
› Author Affiliations Funding This study was funded by Bayer.
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Abstract

BAY 94-9027 (damoctocog alfa pegol, Jivi) is an extended-half-life recombinant factor VIII (rFVIII) shown to be well-tolerated and efficacious in bleeding prevention in previously treated patients with severe hemophilia A. During the PROTECT VIII study, prophylaxis patients received BAY 94-9027 at intervals determined based on their bleeding phenotype, observed during a 10-week run-in treatment period with twice-weekly dosing. Those with ≤ 1 spontaneous joint or muscle bleed were randomized to either 45 to 60 IU/kg every 5 days or 60 IU/kg every 7 days; patients could increase dosing frequency to every 5 days or twice weekly in the case of bleeds. Those enrolled after the randomization arms were full, and those with ≥ 2 bleeds in the run-in period, received 30 to 40 IU/kg twice weekly. Patients completing the main study could receive open-label BAY 94-9027 in the extension phase. Dosing regimen, total, and joint annualized bleeding rates were analyzed over three periods: prestudy, main study, and extension. A total of 80 patients who were on prophylaxis treatment prior to and during the study and had prior bleed data available were evaluated in this post hoc analysis of PROTECT VIII. Most patients (> 80%) required fewer infusions with BAY 94-9027 prophylaxis versus their previous standard-half-life (SHL) rFVIII product. Lower bleeding and joint bleeding rates were observed over time from the prestudy to the extension study period in all treatment regimens. Compared with SHL FVIII, BAY 94-9027 prophylaxis allows patients to reduce infusion frequency with maintained or improved protection from bleeds.

Keywords

annualized bleeding rate - hemophilia A - prophylaxis - recombinant factor VIII - target joint


Publication History

Received: 07 May 2020

Accepted: 08 December 2020

Accepted Manuscript online:
09 December 2020

Article published online:
28 February 2021

© 2020. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution-NonDerivative-NonCommercial License, permitting copying and reproduction so long as the original work is given appropriate credit. Contents may not be used for commercial purposes, or adapted, remixed, transformed or built upon. (https://creativecommons.org/licenses/by-nc-nd/4.0/)

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