Abstract Number: PB0323
Meeting: ISTH 2020 Congress
Background: Emicizumab, a bispecific antibody mimicking activated factor VIII (FVIII), has been proven to be efficacious in a Japanese multicenter, open-label study in pediatric patients with hemophilia A without inhibitors (Hemophilia. 2019; 25(6): 979-987).
Aims: To determine the efficacy and safety of emicizumab in clinical practice, we report a study using emicizumab at our institution.
Methods: We enrolled 12 pediatric patients with hemophilia A (10 severe and 2 moderate), who had started receiving emicizumab by January 2020. Baseline median age was 0.95 years, ranging from 6 days to 10 years. Before the start of the emicizumab regimen, 3 patients received FVIII prophylaxis, 5 received on-demand treatment, and 4 received no treatment. Emicizumab treatment was started to prevent severe bleeding early in life or switch from prophylaxis to high-risk intravenous injection. The median duration of emicizumab treatment was 31.9 weeks
Results: The median annualized bleeding rates before and during emicizumab treatment were, respectively, 1.0 and 1.6 for the prophylaxis group, 8.6 and 1.1 for the on-demand group, and 0.0 and 0.0 for the untreated group. Except for one case of bleeding from a cervical lymphangioma, all patients had traumatic, mild, subcutaneous bleeding. Injection site reactions were noted in 2 cases. No neutralizing anti-emicizumab antibodies had been developed.
Conclusions: Although the observation period was short, safe and efficacious treatment for pediatric patients with hemophilia A without inhibitors, including 4 previously untreated patients, was observed at our institution. Emicizumab can prevent bleeding early in life, which has been difficult to achieve with FVIII prophylaxis, and may lead to a reduction in severe bleeding, including intracranial bleeding. Laboratory monitoring of the administration of emicizumab and the effect of emicizumab on preventing arthropathy remain to be further studied.
To cite this abstract in AMA style:Mori M, Nagae C, Umezawa Y, Sudo A, Ashikaga T, Yamashita A, Taki M. Emicizumab Treatment in Pediatric Patients with Hemophilia A without Inhibitors: A Single-Institution Study [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/emicizumab-treatment-in-pediatric-patients-with-hemophilia-a-without-inhibitors-a-single-institution-study/. Accessed January 28, 2022.
« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/emicizumab-treatment-in-pediatric-patients-with-hemophilia-a-without-inhibitors-a-single-institution-study/