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 (Ͻ 1 u dL Ϫ1 ) 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 £547 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.

In 1997 the UK Haemophilia Centre Directors Organization published the guidelines for diagnosis and management of von Willebrand disease (vWD). The guidelines stated that desmopressin (DDAVP) should be used in type 1 and type 2N vWD, that it might be useful in other types 2 vWD and that it is ineffective in type 3 vWD. If patients are unresponsive to DDAVP or if it is contraindicated, the treatment of choice is clotting factor concentrates (CFC). In the light of these guidelines, we audited our practice for 1997. Furthermore, we undertook a retrospective review of the changing patterns of treatment of vWD between 1980 and 1997. During 1997, 10 patients with vWD received DDAVP and another 30 patients were treated with CFC (a total of 1.2 million IU): Haemate P (Centeon, Germany) and/or 8Y (BPL, Elstree, UK). Few patients had clear contraindications to DDAVP, but several patients with type 1 and 2 vWD received CFC on the basis of age or reduced levels of von Willebrand factor - where DDAVP was considered suboptimal for adequate haemostasis. However, this assumption was made without a preliminary test dose to assess the response to DDAVP. The analysis of treatment of vWD for the past 17 years showed that cryoprecipitate was discontinued from use in the early 1990s and that both DDAVP and CFC usage has been on the increase. In conclusion, the audit illustrated a general good adherence to guidelines but it highlighted the need for a DDAVP test before using CFC.

No abstract