Breakthrough Bleeding Episodes at Minimum and Improvement in Quality of Life in a Child with Severe Hemophilia A with Inhibitors Treated with Emicizumab: A Case Report from Chile.

Abstract

Patient: Male, 10-year-oldFinal Diagnosis: Hemophilia ASymptoms: HemorrhageMedication:—Clinical Procedure: —Specialty: Hematology • Pediatrics and NeonatologyObjective:Rare diseaseBackground:People with hemophilia A have shown osteomuscular complications that have a significant impact on their quality of life (QoL) and on health care costs. Patients with hemophilia A with inhibitors living in developing countries such as Chile face a high disease and treatment burden. Emicizumab, a humanized bispecific monoclonal antibody, is associated with improvements in QoL and reduction in the financial impact of the disease related to treatment. This case report describes the impact of emicizumab on a patient with severe hemophilia A with inhibitors in terms of breakthrough bleeding control, improvements in QoL, and reduced financial impact after a year of treatment, in a country where this medication is not routinely available.Case Report:A 10-year-old child with severe hemophilia A with inhibitors had several restrictions in his daily life due to multiple incidences of breakthrough bleeding. He was on episodic treatment with bypassing agents, and in the year prior to treatment with emicizumab he had 18 bleeding episodes. After 1 year on prophylaxis treatment with emicizumab, the patient had only 1 bleeding episode (94.4% of reduction), improved pain control (5-point reduction on the visual analogue scale), a decrease in the Hemophilia Joint Health Score from 39 to 19, the QoL perception increased by 86% on the standardized Haemo-QoL-kids, and a 70% reduction in treatment costs versus the costs of episodic treatment with bypassing agents.Conclusions:After 1 year of treatment with emicizumab, this patient had substantial improvements in the evaluated parameters. Further investigations with emicizumab are needed to assess its possible effects on public health policies.