1. Decreased Bleeding Rates in Patients with Hemophilia A Switching from Standard-Half-Life FVIII to BAY 94-9027 Prophylaxis.
- Author
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Mancuso ME, Reding MT, Negrier C, Kerlin BA, Rangarajan S, and Simpson ML
- Subjects
- Adolescent, Adult, Child, Clinical Trials, Phase I as Topic, Clinical Trials, Phase II as Topic, Coagulants adverse effects, Drug Administration Schedule, Factor VIII adverse effects, Hemarthrosis blood, Hemarthrosis diagnosis, Hemarthrosis prevention & control, Hemophilia A blood, Hemophilia A diagnosis, Hemorrhage blood, Hemorrhage diagnosis, Humans, Male, Middle Aged, Polyethylene Glycols adverse effects, Randomized Controlled Trials as Topic, Retrospective Studies, Time Factors, Treatment Outcome, Young Adult, Coagulants administration & dosage, Drug Substitution, Factor VIII administration & dosage, Hemophilia A drug therapy, Hemorrhage prevention & control, Polyethylene Glycols administration & dosage
- 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., Competing Interests: M.E.M. has received consultancy fees from Bayer Healthcare, CSL Behring, Novo Nordisk, Pfizer, Roche, Sobi, Shire, Octapharma, Kedrion, and Grifols. M.T.R. has received institutional research support from Bayer and BioMarin, and received honoraria for serving on advisory boards and speakers bureaus for Bayer, Novo Nordisk, Sanofi Genzyme, and Takeda. C.N. has received grant/research support, honoraria, or consultation fees from Alnylam, Baxalta/Shire, Bayer, CSL Behring, LFB, Novo Nordisk, Octapharma, Roche, Pfizer, and Sobi. B.A.K. has received research support from CSL Behring and Novo Nordisk, and received honoraria for serving on advisory boards and speaker bureaus from Bayer, BioMarin, CSL Behring, Novo Nordisk, Octapharma, Pfizer, Roche, Sobi, and Takeda. S.R. has received institutional research grants from Sangamo, and participated in speaker bureaus for Shire. M.L.S. has received institutional research grants from Bayer, Bioverativ/Sanofi, Daichii-Sankyo, Octapharma, Novo Nordisk, Baxalta (Shire, Takeda), and Roche (Genentech), and received honoraria and consultation fees from Bayer, BioMarin, Pfizer, Roche, Spark, and Novo Nordisk., (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/).)
- Published
- 2021
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