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Artikel ; Online: A prospective surveillance study of inhibitor development in haemophilia A patients following a population switch to a third-generation B-domain-deleted recombinant factor VIII.

Dubé, E / Bonnefoy, A / Merlen, C / Castilloux, J-F / Cloutier, S / Demers, C / Sabapathy, C A / St-Louis, J / Vezina, C / Warner, M / Rivard, G-É

Haemophilia : the official journal of the World Federation of Hemophilia

2018  Band 24, Heft 2, Seite(n) 236–244

Abstract: Introduction: Following a provincial tender, most subjects with haemophilia A in Quebec switched their treatment to a third-generation recombinant B-domain-deleted factor VIII (FVIII).: Aim: Our objective was to evaluate the incidence of inhibitor ... ...

Abstract Introduction: Following a provincial tender, most subjects with haemophilia A in Quebec switched their treatment to a third-generation recombinant B-domain-deleted factor VIII (FVIII).
Aim: Our objective was to evaluate the incidence of inhibitor development and FVIII recovery in patients following the switch of factor replacement therapy.
Methods: One hundred and thirty-five subjects were enrolled and tested for FVIII activity and inhibitors every 6 months during 1 year. Subjects with mild haemophilia A or current inhibitors were excluded. Data on demographics, bleeds and FVIII usage were collected.
Results: A total of 125 switchers and 10 non-switchers were enrolled. Most subjects had severe haemophilia A (95.6%) and were on prophylaxis (89.6%). Mean FVIII recovery was similar at 0, 6 and 12 months postswitch. Two switchers developed de novo inhibitors in the 6 months postswitch, one of which was transient. No recurrent inhibitor was observed. A small but significant increase in FVIII usage was observed for adult switchers and the whole cohort of switchers and non-switchers. There was an increase in the annualized bleeding rate (ABR) for non-joint bleeds for the whole cohort of switchers. However, no significant differences were observed in ABR for joint bleeds.
Conclusion: Our surveillance study shows comparable inhibitor development to similar published studies. A significant increase in FVIII utilization was noted for the whole cohort, switchers and non-switchers. Lastly, no clinically significant changes were observed in ABR for joint bleeds, but a difference for non-joint bleed ABRs was observed in switchers.
Mesh-Begriff(e) Adult ; Factor VIII/therapeutic use ; Female ; Hemophilia A/drug therapy ; Humans ; Male ; Prospective Studies
Chemische Substanzen Factor VIII (9001-27-8)
Sprache Englisch
Erscheinungsdatum 2018-02-01
Erscheinungsland England
Dokumenttyp Journal Article
ZDB-ID 1229713-6
ISSN 1365-2516 ; 1351-8216 ; 1355-0691
ISSN (online) 1365-2516
ISSN 1351-8216 ; 1355-0691
DOI 10.1111/hae.13410
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