Presentation (CTI)

OBJECTIVES: To assess effectiveness, safety and budget impact of prophylactic emicizumab to prevent bleeding episodes in children with hemophilia A with inhibitors at a pediatric referral hospital.
METHODS: Systematic review of the evidence, budget impact analysis and real world data analysis on hospital costs and effects.
RESULTS: Four randomized controlled trials, 3 observational studies, 9 clinical practice guidelines and 13 economic evaluations were included in the systematic review. The evidence showed that prophylaxis with emicizumab in children with severe hemophilia A with inhibitors is safe and reduces annual bleeding rate in more than 90%, (60-90% of patients free of all bleeding), resolves 100% of target joints, enables to remove 50% of venous accesses and improves quality of life and school attendance. Economic evaluations from different contexts found it cost-effective and dominant compared to alternative treatment options, and it is considered standard of care. Real world data analysis of our hospital patients who were receiving emicizumab showed reduction of general and joint bleeding episodes, with consequent less hospitalizations and use of hospital resources. Budget impact analysis estimated that introduction of emicizumab prophylaxis in children with severe hemophilia A with inhibitors would generate hospital savings of $23.5 million pesos mostly due to a 38% decrease of annual expenses in hemophilia-specific medication.
CONCLUSIONS: The hospital Health Technology Assessment Unit strongly recommended the incorporation of emicizumab prophylaxis in children with severe hemophilia A with inhibitors to hospital management. The proposal was accepted and this strategy is now standard of care for this type of patients.