PB0650 - To predict the impact of low dose prophylaxis vs. on demand treatment in severe Hemophilia A patients in Pakistan

Background: A gold standard Prophylactic application of clotting factor concentrates particularly in children with hemophilia. However, prophylaxis is not practiced in Pakistan due to ill-affordability of clotting factor concentrates.

Aims: To determined the impact of low dose prophylaxis vs. on demand treatment in severe Hemophilia A (HA) patients.

Methods: A prospective observational study conducted from 2018 to 2021. Severe HA patients were sub-grouped into prophylaxis and on-demand treatment with administration of FVIII of 15 IU/kg twice weekly in the prophylaxis group. Comparison was evaluated between both the groups with (Hemophilia Joint Health Score (HJHS) and Functional Independence Score (FISH)).

Results: A total of 50 patients were recruited in the study; 25 patients in each group with median (IQR) age on demand patients were 4(6.2) while 6(4.5) years in prophylaxis group. Overall Joint assessment scoring of patients is mentioned in Table 1. Significant association of annual bleeding rate (ABR) between on demand and prophylaxis group includes bruising 21(72.4) vs. 8(27.6), joint pain 14(66.7) vs. 7(33.3), with p-value=0.001. However, comparison of outcomes of on demand vs. prophylaxis showed insignificant differences in days of hospitalization i.e. lower in the prophylaxis group than on demand group (44% vs. 55.6; p=0.769), no inhibitor was reported in the prophylaxis while 2 (100%) patients in the on-demand group (Figure 1). Estimated given dose of factor was about 477231 IU/kg/year and 593562 IU/kg/year in on demand and prophylaxis group respectively. 18(36) showed significant absenteeism from the school in which 16(88.6) were from on demand whereas 2(11.1) patients from the prophylaxis group i.e. (p-value=0.001) due to bleeding episodes.

Conclusion(s): This study showed that FISH and HJHS had positive impact on the prophylactic group than on the demand group. Overall health of the patients improved after receiving prophylaxis with less bleeding events and better social lives.