Emicizumab in hemophilia A with inhibitors: clinical and economic impact of its use in a Cuban patient

Abstract

Introduction: The management of a patient with severe hemophilia A with inhibitors is a challenge for any healthcare professional. The present analysis shows the clinical and economic impact of the therapeutic approach in the most critical patient of the Cuban Hemophilia Cohort. Objective: To evaluate the economic and clinical impact of the implementation of Emicizumab therapy in the first Cuban patient. Case presentation: Adult patient who started to use recombinant activated factor VII episodically during adolescence when he was diagnosed with the presence of high-responding antibodies against factor VIII. During the years that he used this medication, he had recorded between 95 and 105 bleeds annually. In 2018, he presented with severe hemoperitoneum and was admitted to the intensive care unit with high doses of recombinant factor VII activated, multiple transfusions of packed red blood cells, and other care typical of a critically ill patient; his evolution was satisfactory. He started emicizumab prophylaxis 7 months after this event and is currently on Week 133 of treatment. He has not presented with further bleeding, nor has he reported adverse reactions to this treatment. The biannual cost savings for on-demand treatment and prophylaxis have been US $792,509.24. Conclusions: Emicizumab prophylaxis improved the patient’s quality of life and that of his family. From the perspective of the Cuban health system, the use of emicizumab in this clinical case was satisfactory and its experience will be analyzed in new patients.