UCMS and GTB Hospital, India
Title: Optimal prophylactic schedule for FVIII therapy in children with hemophilia A
Sunil Gomber is the Director & Professor in pediatrics and incharge of pediatric hematology/ oncology in a Government hospital in Delhi, India. He teaches postgraduates in pediatrics besides looking after the patient care.
Background: Prophylaxis in hemophilia is the standard of care. Optimal strategies to conduct prophylaxis in an economically prudent manner have not yet been well-defined in developing countries.
Objective: To compare the efficacy of twice weekly versus thrice weekly Factor VIII prophylactic therapy in prevention of bleeding in children with Hemophilia A & in preserving index joint structures.
Design: Open label randomized clinical trial.
Setting: Tertiary care hospital.
Study duration: April 1, 2017 to March 31, 2018.
Participants: A sample size of 40 children aged less than 18 years with moderate to severe Hemophilia A and not having any measurable inhibitor.
Methods: All children were randomized into two groups A and B followed by detailed history, clinical (HJHS 2.1 score) and radiological examination (Pettersson and ultrasound scores). Prophylaxis started with Group A receiving FVIII at a dose of 10 IU/kg thrice weekly and Group B receiving FVIII at a dose of 10 IU/kg twice weekly. Data regarding bleeding episodes during the study period was collected. All evaluation was repeated at end of study. Main Outcome variable: Annual Bleeding Rate .
Results: Out of 40 children 7 children were excluded since they were having inhibitors against FVIII. 3 chidren were lost to follow up. We analyzed 14 children in twice weekly prophylaxis group and 16 children in thrice weekly prophylaxis group. Statistically insignificant difference was found in median bleeding rate between the thrice weekly prophylaxis group [0.17(0.00- 0.19)] and twice weekly prophylaxis group [0.17(0.00- 0.25)]. The differences between the initial and final HJHS score and ultrasonography score in both the groups improved significantly. The difference in initial and final Pettersson score in both the groups was not significant.
Conclusion: Twice weekly FVIII therapy is an effective, easily administrable and economically prudent prophylactic schedule to prevent long term complications of Hemophilia A.