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Published on: September 30, 2021
Emicizumab in pediatric hemophilia: Bleeding and surgical outcomes from a single-center retrospective study
1Department of Pediatrics, Section of Hematology-Oncology, Texas Children's Cancer and Hematology Centers, Baylor College of Medicine, Houston, Texas, USA.
Insights
Emicizumab prophylaxis significantly reduced bleeding in pediatric hemophilia A patients. A safe perioperative approach was demonstrated, minimizing bleeding during surgical procedures.
Area of Science:
- Hematology
- Pediatric medicine
- Pharmacology
Background:
- Limited real-world data exists on emicizumab prophylaxis in pediatric hemophilia A.
- Current literature shows heterogeneous approaches to procedural management in this population.
Purpose of the Study:
- To evaluate the effectiveness and safety of emicizumab prophylaxis in pediatric hemophilia A patients.
- To assess the perioperative management and outcomes in patients receiving emicizumab.
Main Methods:
- Retrospective review of 28 pediatric hemophilia A patients treated with emicizumab from 2018-2021.
- Analysis of annualized bleeding rates before and after emicizumab initiation.
- Review of surgical procedures and perioperative management strategies.
Main Results:
- Mean annualized bleeding rate decreased from 3.61 to 0.44 after starting emicizumab.
- Seven surgical procedures were performed with pre- and postoperative factor replacement.
- Only one minor bleeding event occurred postoperatively across all surgical procedures.
Conclusions:
- Emicizumab prophylaxis is effective in reducing bleeding rates in pediatric hemophilia A.
- A safe and successful perioperative approach can be achieved with emicizumab, minimizing bleeding complications.
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.
