Cost‐effectiveness analysis of late prophylaxis vs. on‐demand treatment for severe haemophilia A in Italy

Long‐term regular administrations of factor VIII (FVIII) concentrate (prophylaxis) initiated at an early age prevents bleeding in patients with severe haemophilia A (HA). The 5‐year prospective Italian POTTER study provided evidence of benefits in adolescents and adults of late prophylaxis (LP) vs. on‐demand therapy (OD) in reducing bleeding episodes and joint morbidity and improving quality of life; however, costs were increased.

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