A survey of factor prophylaxis in boys with haemophilia followed in North American haemophilia treatment centres

Haemophilia. 2003 May:9 Suppl 1:19-26; discussion 26. doi: 10.1046/j.1365-2516.9.s1.12.x.

Abstract

A survey was conducted in 2002 to determine the pattern of factor prophylaxis use in boys <or=18 years of age with haemophilia followed in North American treatment centres. Responses were obtained from 4553 cases (74% haemophilia A, 26% haemophilia B). The frequency of prophylaxis, defined as factor infusion greater than or equal to once per week for >or=45 weeks per year, was significantly higher for haemophilia A vs. haemophilia B cases (51% vs. 32%, P< 0.0001), and for boys with severe haemophilia A living in Canada vs. the USA (77% vs. 47%, P< 0.0001). Use of full-dose prophylaxis, defined as the infusion of 25-40 IU kg(-1) of factor VIII on alternate days (minimum three times per week) or 25-40 IU kg(-1) of factor IX twice weekly, was similar for boys <or=5 years of age in both Canada and the USA (30% and 33% haemophilia A and 35% and 13% haemophilia B). Reasons for initiating prophylaxis included a history of joint bleeding (88%) and age <or=2 years (23%). For prophylaxis triggered by joint bleeding, 38% of haemophilia treatment centres indicated that they would initiate prophylaxis after the first joint bleed and 66% after a history of target joint bleeding, defined most frequently as 2-4 bleeds over a 3-6 consecutive month period. A central venous line was used to ensure easy venous access for full-dose prophylaxis therapy in 80% of boys <or=5 years of age. These data offer a basis for projecting long-term factor concentrate needs for persons with haemophilia living in North America.

MeSH terms

  • Adolescent
  • Canada
  • Catheterization, Central Venous
  • Child
  • Child, Preschool
  • Clinical Trials as Topic
  • Decision Making
  • Health Surveys
  • Hemophilia A / prevention & control*
  • Hemophilia B / prevention & control*
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Prospective Studies
  • United States