Child with Hemophilia A presenting claw hand deformity treated with Emicizumabe: excellent clinical result associated to lower financial cost

Abstract

Objective: Exemplify how prophylaxis using Emicizumabe (EMZ) in children with Hemophilia A can be more effective and less costly than standard management. Patients presenting antibodies against Factor VIII are treated with prophylactic intravenous bypassing medicines, e.g., recombinant Factor VIIa (rFVIIa) and Immunotolerance (ITI), attempting the eradication of the inhibitor. Venous access can hamper the success of ITI, and central venous catheters are frequently needed, with their risks. Methodology: we report the case of an 1y2m hemophilia A child weighting 10kg, presenting right claw hand deformity caused by a hematoma. His inhibitor was 15 U.B. He was not on prophylaxis with bypass due to difficult venous access, so we decided to start EMZ, together with aggressive daily rehabilitation and hand orthosis. EMZ doses were weekly 3 mg/kg in the first 4 weeks, followed by weekly 1,5 mg/kg. We analyzed annualized bleeding rate (ABR), individual cost of medicines and medium body weight. Results: this is a highly bleeder patient, and costs with rFVIIa to treat bleeds represented 150%, compared to costs with prophylaxis (graph1). Within the same period, EMZ costs would be BR$ 286,677.996. He had an excellent recovery (figures 1 and 2). Discussion: EMZ represented 97% of saving with medicines, and allowed rehabilitation without any new bleed. Conclusions: hemophilia management should be individualized, especially in toddlers and children, when injuries are potentially severe and lifelong. We propose prophylaxis with EMZ before the attempt of insertion of central lines for ITI, which could be postponed, when better peripheric venous access develops.