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Efficacy and safety of full-length pegylated recombinant factor VIII with extended half-life in previously treated patients with hemophilia A: comparison of data between the general and Japanese study populations
- Source :
- International journal of hematology. 106(5)
- Publication Year :
- 2017
-
Abstract
- Rurioctocog alfa pegol (BAX 855) is a novel third-generation recombinant factor VIII whose active ingredient is chemically modified with polyethylene glycol. A global multicenter phase 2/3 study of the product in 137 patients (including 11 patients from Japan) with severe hemophilia A aged 12–65 years, reported an extended half-life and a good tolerability profile, as well as a significantly lower annualized bleeding rate in the prophylactic treatment arm than in the on-demand treatment arm. Using descriptive statistics, a post hoc analysis was performed to compare the pharmacokinetics, safety, and efficacy profiles of the product in the Japanese subpopulation and the overall population. Extended half-life was demonstrated in the Japanese subpopulation. The mean [standard deviation (SD)] annualized bleeding rates in the prophylactic treatment arm were 3.7 (4.7) for the overall population (n = 120) and 4.0 (3.4) for the Japanese subpopulation (n = 11). The proportion of bleeds reported as excellent or good was 94.9% (149/157) in the overall population, whereas that in the Japanese subpopulation was 92.3% (12/13). No FVIII inhibition or anaphylactic reaction was reported in the Japanese subpopulation. The post hoc comparisons demonstrated similar pharmacokinetic, safety, and efficacy profiles between the overall population and the Japanese subpopulation.
- Subjects :
- Adult
Male
medicine.medical_specialty
Adolescent
Population
Hemorrhage
030204 cardiovascular system & hematology
Hemophilia A
Recombinant factor viii
Gastroenterology
03 medical and health sciences
0302 clinical medicine
Pharmacokinetics
Japan
Internal medicine
Post-hoc analysis
medicine
Humans
education
Child
Aged
education.field_of_study
Hematology
Factor VIII
business.industry
Half-life
Middle Aged
Surgery
Tolerability
030220 oncology & carcinogenesis
Female
Previously treated
business
Subjects
Details
- ISSN :
- 18653774
- Volume :
- 106
- Issue :
- 5
- Database :
- OpenAIRE
- Journal :
- International journal of hematology
- Accession number :
- edsair.doi.dedup.....61879b01d63ee79f851e57a6d0022dfc