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

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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… Click to show full abstract

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.

Keywords: late prophylaxis; effectiveness analysis; cost effectiveness; prophylaxis demand; severe haemophilia

Journal Title: Haemophilia
Year Published: 2017

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