Prospective cost-effectiveness study of regular continuous prophylactic replacement therapy in adults with severe hemophilia A

Background: Prophylaxis is effective in children with haemophilia. Adults with advanced haemophilic arthropathy can probably benefit from a continuous prophylactic replacement therapy (PRT), but no data are available on cost end efficacy. Methods: A prospective study was designed to evaluate cost-effectiveness of PRT with a B-domain-deleted recombinant FVIII in adults with severe haemophilia, aged 18–60 years, with ‡18 bleeds in the previous year. PRT: 25 IU kg-1 of Refacto® 3 times a week for 6 months. Bleeding rate and FVIII consumption was evaluated over the previous 6-month on-demand treatment (ODT) period and the PRT period. Medical costs were quantified adopting the perspective of the National Health Service. The incremental cost-effectiveness ratio (ICER) was calculated as ratio of cost and bleed difference of ODT and PRT. Confidence intervals (CIs) were generated by non-parametric bootstrap procedure. A cost-effectiveness plan and acceptability curve were created. Results: Nineteen patients, aged 23–58 years (mean = 33.2) with a mean of 2.97 events/patient/ month (median = 1.67, range: 0.5–15) were enrolled. Bleeding frequency decreased to 0.48 events/ patient/ month. The incremental cost of PRT vs. ODT was estimated to be € 11,619/patient/month (95% CI € 7,649–15,589) with an additional effect of 2.49 bleeds avoided/patient/month (1.06–3.93). The ICER was estimated to be € 5184 per bleed avoided (€1,071–9,297). The likelihood of PRT being cost-effective was 95% with a ceiling ratio of € 9000 per bleed avoided. Conclusions: These findings showed PRT with Refacto® in adults with haemophilia is cost-effective, but it requires more resources than ODT. Our cost-effectiveness results can represent the reference for other similar evaluations.