Emicizumab in pediatric hemophilia: Bleeding and surgical outcomes from a single-center retrospective study
- PMID: 34490988
- DOI: 10.1002/pbc.29325
Emicizumab in pediatric hemophilia: Bleeding and surgical outcomes from a single-center retrospective study
Abstract
Real-world data describing emicizumab prophylaxis in pediatric hemophilia A is limited with current literature providing a heterogenous approach to procedural management. We performed a retrospective review on the 28 patients who have been treated with emicizumab prophylaxis at Texas Children's Hospital from 2018 to 2021. After starting emicizumab, the mean annualized bleeding rate reduced from 3.61 to 0.44. Seven surgical procedures were performed in the cohort, and all treated with pre- and postoperative factor replacement resulting in one minor bleeding event. We demonstrate a successful experience with emicizumab prophylaxis and safe perioperative approach with a focus on minimizing postoperative bleeding.
Keywords: bleeding; emicizumab; hemophilia A.
© 2021 Wiley Periodicals LLC.
References
REFERENCES
-
- Shima M, Hanabusa H, Taki M, et al. Factor VIII-mimetic function of humanized bispecific antibody in hemophilia A. N Engl J Med. 2016; 374(21):2044-2053.
-
- Mason JA, Young G. Emicizumab prophylaxis in infants with severe haemophilia A without inhibitors: illustrative real-world cases to support shared decision-making. Haemophilia. 2021. https://doi.org/10.1111/hae.14353
-
- Young G, Liesner Ri, Chang T, et al. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019;134(24):2127-2138.
-
- Mahlangu J, Oldenburg J, Paz-Priel I, et al. Emicizumab prophylaxis in patients who have hemophilia a without inhibitors. N Engl J Med. 2018;379(9):811-822.
-
- Shima M, Nogami K, Nagami S, et al. A multicentre, open-label study of emicizumab given every 2 or 4 weeks in children with severe haemophilia A without inhibitors. Haemophilia. 2019;25(6):979-987.
MeSH terms
Substances
LinkOut - more resources
Full Text Sources
Medical
