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

Abstract

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.

The effect of secondary prophylaxis versus episodic treatment on the range of motion of target joints in patients with haemophilia

“…Studies in adults on secondary prophylaxis have demonstrated a decrease in joint haemorrhages, better functional outcomes and overall improvement in quality of life, although there was progression of haemophilic arthropathy, and an increase in factor usage and cost (Smith et al , 1996; Miners et al , 1998; Saba et al , 2000; Tagliaferri et al , 2008). In a survey of haemophilia patients in Western Pennsylvania, continuation of episodic treatment was chosen because of cost (35%), absence of orthopaedic problems (30%), concerns about HIV and hepatitis (25%), fear of inhibitors (22%), need for venous access (17%), time commitment (15%), psychosocial effects (15%) and young age (7%) (Cowden et al , 1997).…”

Treatment of hemophilia: a review of current advances and ongoing issues

“…There are a number of studies in the literature on the role of secondary prophylaxis in children,13,42–45 and more recently also for adult patients 46–48. On the whole, these studies show that even delayed prophylaxis is able to reduce the frequency of bleeding as well as patients’ physical and psychologic restrictions.…”

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

“…Since its introduction in 1958 by Nilsson in Sweden [1], prophylactic treatment has been adopted as the primary treatment strategy for patients with severe haemophilia in several countries in northern Europe [2][3][4][5][6]. Since its introduction in 1958 by Nilsson in Sweden [1], prophylactic treatment has been adopted as the primary treatment strategy for patients with severe haemophilia in several countries in northern Europe [2][3][4][5][6].…”