Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study.
Authors
Nolan, BeatriceMahlangu, Johnny
Pabinger, Ingrid
Young, Guy
Konkle, Barbara A
Barnes, Chris
Nogami, Keiji
Santagostino, Elena
Pasi, K John
Khoo, Liane
Winding, Bent
Yuan, Huixing
Fruebis, Joachim
Rudin, Dan
Oldenburg, Johannes
Issue Date
2020-03-30Keywords
bleed rateextended half-life
individualized prophylaxis
perioperative haemostasis
rFVIIIFc
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Haemophilia : the official journal of the World Federation of HemophiliaDOI
10.1111/hae.13953PubMed ID
32227570Abstract
Introduction: The efficacy and safety of recombinant factor VIII Fc fusion protein (rFVIIIFc) as an extended half-life treatment for severe haemophilia A were demonstrated in the Phase 3 A-LONG and Kids A-LONG studies. Eligible subjects who completed A-LONG and Kids A-LONG could enrol in ASPIRE (NCT01454739), an open-label extension study. Aim: To report the long-term safety and efficacy of rFVIIIFc in subjects with severe haemophilia A who enrolled in ASPIRE. Methods: Previously treated subjects received one or more of the following regimens: individualized prophylaxis (IP), weekly prophylaxis, modified prophylaxis or episodic treatment. Subjects could switch treatment regimen at any time. The primary endpoint was inhibitor development. Results: A total of 150 subjects from A-LONG and 61 subjects from Kids A-LONG enrolled in ASPIRE. Most subjects received the IP regimen (A-LONG: n = 110; Kids A-LONG: n = 59). Median (range) treatment duration in ASPIRE for subjects from A-LONG and Kids A-LONG was 3.9 (0.1-5.3) years and 3.2 (0.3-3.9) years, respectively. No inhibitors were observed (0 per 1000 subject-years; 95% confidence interval, 0-5.2) and the overall rFVIIIFc safety profile was consistent with prior studies. For subjects on the IP regimen, annualized bleed rates (ABR) remained low (median overall ABR for adults and adolescents was <1.0) and extended-dosing intervals were maintained (median of 3.5 days) for the majority of subjects in ASPIRE. Conclusion: ASPIRE results, which include up to 5 years of follow-up data, confirm earlier reports on the consistent and well-characterized safety and efficacy of rFVIIIFc treatment for severe haemophilia A.Item Type
ArticleLanguage
enEISSN
1365-2516ae974a485f413a2113503eed53cd6c53
10.1111/hae.13953
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