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Assessing the effectiveness and cost‐effectiveness of prophylaxis against bleeding in patients with severe haemophilia and severe von Willebrand's disease
Author(s) -
Miners A. H.,
Sabin C. A.,
Tolley K. H.,
Lee C. A.
Publication year - 1998
Publication title -
journal of internal medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.625
H-Index - 160
eISSN - 1365-2796
pISSN - 0954-6820
DOI - 10.1111/j.1365-2796.1998.00396.x
Subject(s) - medicine , haemophilia , haemophilia a , von willebrand disease , pediatrics , cost effectiveness , surgery , clotting factor , prophylactic treatment , von willebrand factor , platelet , risk analysis (engineering)
. Miners AH, Sabin CA, Tolley KH, Lee CA (Royal Free Hospital School of Medicine, London; University Hospital Queen's Medical Centre, Nottingham; and Royal Free Hampstead NHS Trust, London, UK). Assessing the effectiveness and cost‐effectiveness of prophylaxis against bleeding in patients with severe haemophilia and severe von Willebrand's disease. J Intern Med 1998; 244 : 515–22. 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 £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.