Emicizumab in pediatric hemophilia: Bleeding and surgical outcomes from a single-center retrospective study

Pediatr Blood Cancer. 2021 Nov;68(11):e29325. doi: 10.1002/pbc.29325. Epub 2021 Sep 7.

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.

MeSH terms

  • Antibodies, Bispecific / therapeutic use*
  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Child
  • Hemophilia A* / complications
  • Hemophilia A* / drug therapy
  • Hemorrhage* / prevention & control
  • Humans
  • Retrospective Studies
  • Treatment Outcome

Substances

  • Antibodies, Bispecific
  • Antibodies, Monoclonal, Humanized
  • emicizumab