Clinical Evidence and Safety Profile of Emicizumab for the Management of Children with Hemophilia A

Emicizumab is a bispecific, humanized, monoclonal antibody mimicking the factor (F) VIII cofactor activity in mediating the generation of FXa by FIXa in patients with hemophilia A (HA). This subcutaneous non-factor agent has been recently extensively approved for the prophylaxis of patients of HA pa...

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Bibliographic Details
Published in:	Drug design, development and therapy development and therapy, 2020-02, Vol.14, p.469-481
Main Author:	Le Quellec, Sandra
Format:	Article
Language:	eng
Subjects:	Adolescents Adults Antibodies Antibodies, Bispecific - therapeutic use Antibodies, Monoclonal, Humanized - therapeutic use Bleeding Blood coagulation factor VIII Blood coagulation factors Caregivers Child Children Clinical trials Comparative analysis Diseases emicizumab Factor VII Factor VIII Factor VIII - therapeutic use Health aspects Hemophilia hemophilia a Hemophilia A - drug therapy Humans Inhibitors Mimicry Monoclonal antibodies Patients Pediatrics Prophylaxis Quality of life Review Safety Safety and security measures Safety regulations subcutaneous
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Summary:	Emicizumab is a bispecific, humanized, monoclonal antibody mimicking the factor (F) VIII cofactor activity in mediating the generation of FXa by FIXa in patients with hemophilia A (HA). This subcutaneous non-factor agent has been recently extensively approved for the prophylaxis of patients of HA patients with and without FVIII-inhibitors of all ages, although few data are currently available in children. In Phase 3 clinical trials and case series, emicizumab prophylaxis significantly reduced bleeding rates compared to previous treatment in HA adolescents and children with or without FVIII-inhibitors and was generally well tolerated. In addition, subcutaneous administration of emicizumab provided beneficial effects on health-related quality of life, and lessened the burden of the disease in HA patients as well as in their caregivers. However, additional prospective studies are required to evaluate the long-term safety of emicizumab prophylaxis in very young patients, including previously untreated patients. The aim of this paper was to review the limited data available on the use of emicizumab prophylaxis in children and to highlight the need for further studies to address remaining concerns.
ISSN:	1177-8881 1177-8881