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Serious bleeds in pediatric persons with hemophilia A on emicizumab prophylaxis

Jessica Garcia1,2, Matthew R Hammer3,2, Ayesha Zia1,2

  • 1Division of Hematology/Oncology, Department of Pediatrics, UT Southwestern Medical Center, Dallas, Texas, USA.

Research and Practice in Thrombosis and Haemostasis
|December 6, 2023
PubMed

Insights

Serious bleeds can still occur in pediatric patients with hemophilia A despite using emicizumab. This real-world data suggests careful monitoring and treatment adjustments are crucial for managing bleeding events in this population.

Area of Science:

  • Pediatric Hematology
  • Hemophilia A Treatment
  • Pharmacovigilance

Background:

  • Emicizumab shows promise in clinical trials for preventing bleeds in hemophilia A.
  • Real-world data on emicizumab's effectiveness in preventing serious bleeds in pediatric hemophilia A patients is limited.

Purpose of the Study:

  • To assess the real-world effectiveness of emicizumab in preventing serious bleeds in pediatric patients with hemophilia A.

Main Methods:

  • Retrospective chart review of 37 pediatric males (≤18 years) on emicizumab prophylaxis.
  • Median follow-up duration of 30.5 months.
  • Data collected from Children's Medical Center, Dallas, Texas.

Main Results:

  • Four pediatric patients with severe hemophilia A experienced serious bleeds (provoked or unprovoked) requiring hospitalization.
  • Patients had hemophilia A with or without inhibitors.

Conclusions:

  • Serious bleeds can occur in pediatric severe hemophilia A patients on emicizumab.
  • Clinicians should counsel patients on risks and consider treatment adjustments (e.g., emicizumab titration, Factor VIII) for suboptimal bleeding control or high-risk activities.
Abstract

Keywords:
bleedemicizumabhemophilia Apediatricprophylaxis

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