A Comparison of the Incidence Rate for Joint Bleeding and FVIII Consumption between On-Demand versus Prophylaxis Factor VIII Replacement Therapy and the Direct Cost of Prophylaxis Treatment in Severe Haemophilia A Patients

Malays J Med Sci. 2023 Feb;30(1):129-136. doi: 10.21315/mjms2023.30.1.11. Epub 2023 Feb 28.

Abstract

Background: Haemophilia A is a bleeding disorder caused by inadequate clotting factor VIII (FVIII). There are two main modes of treatment approach in severe haemophilia A patients either with on-demand or prophylaxis therapy with clotting factor FVIII concentrates. In this study, a comparison was made between the bleeding incidence rate of the on-demand and prophylaxis group in severe haemophilia A patients at Ampang Hospital, Malaysia.

Methods: A retrospective study involving patients with severe haemophilia was conducted. The patient's self-reported bleeding frequency was retrieved from the patient's treatment folder from January to December 2019.

Results: Fourteen patients received on-demand therapy, while the other 24 patients received prophylaxis treatment. The total number of joint bleeds in the prophylaxis group was significantly lower compared to the on-demand group (2.79 bleeds versus 21.36 bleeds [P < 0.001]). Furthermore, the total annual usage of FVIII was higher in the prophylaxis group compared to the on-demand group (1,506 IU/kg/year [± 905.98] versus 365.26 IU/kg/year [± 223.90], P = 0.001).

Conclusion: Prophylaxis FVIII therapy is an effective treatment in reducing the frequency of bleeding joints. However, this treatment approach is associated with high cost due to the high consumption of FVIII.

Keywords: bleeding rate; factor VIII; haemophilia; haemorrhage; prophylaxis.