Assessing the effectiveness and cost-effectiveness of prophylaxis against bleeding in patients with severe haemophilia and severe von Willebrand's disease.

OBJECTIVES: To assess the effectiveness and cost-effectiveness of prophylaxis with clotting factor against bleeding in patients with severe haemophilia and von Willebrand's disease (vWD).

DESIGN: Treatment details that related to 179 patients with severe (< 1 u dL-1) haemophilia A, B and vWD were retrospectively examined for the period 1980-95. A subgroup of these patients, 25 adults and 22 children, who had previously received treatment on demand and who had switched to treating with prophylaxis, were studied in order to examine the effects of the change. The cost-effectiveness of prophylaxis was also analysed using another subgroup of 38 patients and by adjusting their treatment details by age and method of treatment.

SETTING: Data were obtained on patients who were solely registered at the Royal Free Hospital Haemophilia Centre (RFHHC), London, UK.

OUTCOME MEASURE: Bleeds.

RESULTS: The median annual number of bleeds decreased from 23.5 (range 1-107) in 1980, to 14 (range 0-45) in 1995 (P < 0.0001). Switching from treating on demand to prophylaxis reduced bleeding frequency in 41 out of 47 patients within the period of 1 year. At the base scenario, switching to prophylaxis cost an additional pound547 per averted bleed; however, this figure was highly sensitive to certain variables.

CONCLUSION: Prophylaxis can reduce bleeding frequency but requires more clotting factor than treatment on demand. More detailed proof of cost-effectiveness is likely to require the use of modelling techniques.