Cost-Effectiveness of Prophylaxis with Anti-Inhibitor Complex Concentrate in Patients with Hemophilia A and Inhibitors: Results From the Pro-FEIBA Study

• Published in: Blood 2011-11, Vol.118 (21), p.4187-4187
• Main Authors: Gringeri, Alessandro, Leissinger, Cindy A., Cortesi, Paolo, Jo, Hyejin, Mantovani, Lorenzo
• Format: Article
• Language: English
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood.V118.21.4187.4187

Abstract 4187 Prophylaxis with anti-inhibitor complex concentrate (AICC; FEIBA NF, Baxter Healthcare Corporation, Westlake Village, CA) has been shown to safely and significantly decrease the frequency of joint and other bleeding events in patients with severe hemophilia A and inhibitors. Nonetheless, AICC prophylaxis is an expensive therapeutic intervention, and evidence of its cost-effectiveness is needed to guide healthcare decision-making in this era of global economic downturn. Hemophilia A patients >2 years of age with inhibitors and using bypassing therapy to treat bleeding were enrolled in a prospective, randomized, crossover study comparing 6 months of AICC infused prophylactically at a dose of 85U/kg ± 15% on 3 nonconsecutive days per week with 6 months of AICC used on-demand for bleeding episodes at a dose of 85 U/kg ± 15%. The 2 treatment periods were separated by a 3-month washout during which patients used on-demand therapy for bleeding. Clotting factor costs were quantitated in 2011 US dollars using the US price for US patients ($1.56 per unit) and the average price in Europe for European patients ($1.13 per unit). Twenty-six subjects (median age 28.7 years; range, 2.8–62.8) completed both treatment periods and were evaluable per protocol for the cost-effectiveness analysis. The per-patient cost of 6 months of AICC prophylaxis was $493,633 compared with $205,549 for on-demand therapy. These totals were calculated by adding the per-patient cost of all clotting factors used during the prophylaxis period (AICC – $489,473 and recombinant activated factor VII [rFVIIa] – $4,160) and the on-demand period (AICC – $198,862473, rFVIIa – $6,272, and FVIII – $415). The incremental cost of AICC per bleed avoided during the prophylaxis period was $35,565, or $585/kg body weight for our somewhat older study population (mean body weight 60.8 kg). Sixteen of 26 subjects (62%) experienced a ≥50% reduction in bleeding events (“good responders”). For these subjects, the per-patient cost of 6 months of AICC prophylaxis was $499,453 as compared with $202,757 for on-demand therapy. The incremental cost-effectiveness ratio for the prophylaxis versus the on-demand period was $27,282 per bleeding event avoided ($449/kg body weight). In subjects with a