Extended half-life pegylated, full-length recombinant factor VIII for prophylaxis in children with severe haemophilia A.
In: Haemophilia, Jg. 23 (2017-03-01), Heft 2, S. 238-246
Online
academicJournal
Zugriff:
Introduction Primary factor VIII ( FVIII) prophylaxis is the optimal treatment in children with severe haemophilia A. They are expected to benefit from extended half-life ( T1/2) FVIII coverage by reduced infusion frequency while maintaining haemostatic efficacy. Aims To determine immunogenicity, pharmacokinetics ( PK), efficacy, safety and quality of life of prophylaxis with a polyethylene glycol (peg)-ylated FVIII ( BAX 855) based on full-length recombinant FVIII ( ADVATE) in paediatric previously treated patients ( PTPs) with severe haemophilia A. Methods PTPs <12 years without history of FVIII inhibitors received twice-weekly infusions of 50 ± 10 IU kg−1 BAX 855 for ≥50 exposure days. Prophylactic dose increases to ≤80 IU kg−1 were allowed under predefined conditions. PK was evaluated after single infusions of 60 ± 5 IU kg−1. Results T1/2 and mean residence time were extended 1.3- to 1.5-fold compared to ADVATE ( n = 31), depending on the analysis used. The point estimate for the mean annualized bleeding rate in 66 subjects receiving a median of 1.9 weekly infusions of 51.3 IU kg−1 of BAX 855 each was 3.04 (median 2.0); 1.10 (median 0) for joint and 1.16 (median 0) for spontaneous bleeds. Overall, 38% of subjects had zero bleeds. No bleeds were severe. Haemostatic efficacy was rated excellent or good for 90% of bleeds; 91% were treated with one or two infusions. In 8/14 subjects all target joints resolved. No subject developed FVIII inhibitors or persistent binding antibodies that affected safety or efficacy. No adverse reactions occurred. Conclusion Twice-weekly prophylaxis with BAX 855 was safe and efficacious in paediatric PTPs with severe haemophilia A. [ABSTRACT FROM AUTHOR]
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Extended half-life pegylated, full-length recombinant factor VIII for prophylaxis in children with severe haemophilia A.
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Autor/in / Beteiligte Person: | Mullins, E. S. ; Stasyshyn, O. ; Alvarez‐Román, M. T. ; Osman, D. ; Liesner, R. ; Engl, W. ; Sharkhawy, M. ; Abbuehl, B. E. |
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Zeitschrift: | Haemophilia, Jg. 23 (2017-03-01), Heft 2, S. 238-246 |
Veröffentlichung: | 2017 |
Medientyp: | academicJournal |
ISSN: | 1351-8216 (print) |
DOI: | 10.1111/hae.13119 |
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