Prophylactic treatment for severe haemophilia: comparison of an intermediate‐dose to a high‐dose regimen

Abstract

A multicentre study was performed in Sweden and the Netherlands, comparing effects of two prophylactic regimens in 128 patients with severe haemophilia, born 1970-90. 42 Swedish patients (high-dose prophylaxis), were compared with 86 Dutch patients (intermediate-dose prophylaxis). Patients were evaluated at the date of their last radiological score according to Pettersson. Annual clotting factor consumption and bleeding frequency were registered for a period of three years before evaluation. Patients in the high-dose group were younger at evaluation (median 15.2 vs. 17.9 years), started prophylaxis earlier (median 2 vs. 5 years), and used 2.19 times more clotting factor kg-1 year-1. Patients treated with high-dose prophylaxis had fewer joint bleeds (median 0.3 year-1 vs. 3.3 year-1) and the proportion of patients without arthropathy as measured by the Pettersson score was higher (69% vs. 32%), however, the age-adjusted difference in scores (median 0 points vs. 4 points) was small and at present not statistically significant. Clinical scores and quality of life were similar. These findings suggest that, compared with intermediate-dose prophylaxis, high-dose prophylaxis significantly increases treatment costs and reduces joint bleeds over a period of 3 years, but only slightly reduces arthropathy after 17 years of follow-up.

Pharmacokinetics and dose requirements of factor VIII over the age range 3–74 years

“…The principle of dosing FVIII to a target trough level of 0.01 U/mL has been questioned [4,36], however, no other treatment strategy has been shown to be superior. A correlation between time below 0.01 U/mL and risk of bleeding during prophylactic treatment has been demonstrated twice [18,37].…”

Effects of the factor V G1691A mutation and the factor II G20210A variant on the clinical expression of severe hemophilia A in children results of a multicenter study

“…In addition, the course of bleeding episodes has been controversially discussed with respect to individualized therapeutic regimens employed by the treating hemophilia center. [15][16][17][18][19] Since 1985, patients enrolled from the different pediatric hemophilia treatment centres in Germany have been treated by the same medical teams using the same treatment protocols. Similar to the Canadian hemophilic cohort recently reported, 11 these patients have been on treatment protocols that have remained unchanged with respect to treatment indications and the criteria chosen to treat a bleeding episode on demand or on prophylaxis.…”

Evaluation of prophylactic therapy in haemophilia with global coagulation tests

“…2 Although the prevalence of F5F8D in the general population is estimated to be rare (1 in 1 000 000 births), an increased frequency has been observed in regions where consanguineous marriage is practised. …”