Optimizing factor prophylaxis for the haemophilia population: Where do we stand?

Victor S. Blanchette, M. Manco-Johnson, E. Santagostino, R. Ljung

Research output: Contribution to journalArticle

Abstract

The hallmark of severe haemophilia, defined as a circulating level of factor (F) VIII (haemophilia A cases) or FIX (haemophilia B cases) of -1 of FIX twice weekly for haemophilia B cases. This protocol is, however, demanding on peripheral veins and very expensive. Modifications of the parent protocol such as starting primary prophylaxis with once-weekly infusions via peripheral veins with rapid escalation to full-dose prophylaxis or dose escalation based on frequency of bleeding are increasingly implemented in haemophilia treatment centres in countries that can afford the high cost of such programmes. These modified programmes can be achieved in the majority of young children with severe haemophilia without the need for central venous access devices (e.g. Port-a-Caths) and with avoidance of device-associated complications such as infection and thrombosis. In at least one centre, experience with arteriovenous fistulae as a strategy to ensure reliable venous access is being accumulated. The issues of compliance (adherence) to recommended prophylaxis protocols and when, if ever, to stop a programme of primary prophylaxis once started are real and require ongoing prospective studies. Such studies should incorporate outcome measures such as health-related quality-of-life and economic analyses.

Original languageEnglish
Pages (from-to)97-104
Number of pages8
JournalHaemophilia, Supplement
Volume10
Issue number4
Publication statusPublished - Oct 2004

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Keywords

  • Haemophilia
  • Prophylaxis

ASJC Scopus subject areas

  • Hematology

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