ABSTRACT
OBJECTIVES: To investigate the effectiveness of low dose secondary/tertiary prophylaxis in severe Hemophilia A children and determine improvements in their daily life. METHODS: Thirty Hemophilia A children (≤ 12 y) with factor VIII <2% and less than two joint bleeds without inhibitors, were given prophylaxis with recombinant Fc fusion long acting factor VIII (ELOCTATE) at 10 IU.kg-1 twice weekly for 1 y. Earlier, patients received on-demand FVIII for a minimum of six months. Outcome was measured in terms of annual bleeding rate, Hemophilia Joint Health Score (HJHS) and child activity/participation was measured in terms of school absenteeism, School Activity Participation Score and Daily Activity Score according to Beijing Children Hospital assessment scale. RESULTS: A total of 30 children were included in the study. There was reduction in annual joint bleeds by 85.76% (14.5 to 2.2) and school absenteeism (days/month) by 86% (17.38 to 2.42) before and after prophylaxis respectively. Majority (43%) showed moderate improvement in daily activity score. Mean HJHS score was 8.3. There was mild improvement in School Activity Participation Score in 57%. Mean annual hospitalization rate reduced from 8.7 to 1.1 with improvement in joint scores. Mean annual factor consumption decreased from 1944.2 IU.kg-1 to 1560.3 IU.kg-1. CONCLUSIONS: With low dose secondary/tertiary prophylaxis, there is significant reduction in the annual joint bleed rate with improvement in joint health and child activity. As factor consumption is reduced, this has a positive effect on cost benefit; and is a very feasible option in developing countries.
