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Real-world experience on the tolerability and safety of emicizumab prophylaxis in paediatric patients with severe haemophilia A with and without FVIII inhibitors.

Eman HassanLancashire JonathanMotwani Jayashree
Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2021)
The real-world experience of emicizumab prophylaxis in our cohort showed that emicizumab was safe and well tolerated in paediatric PwHA with and without inhibitors. Long-term assessment is crucial to monitor major adverse events, recurrence of inhibitors, and development of ADAs.
Keyphrases
  • intensive care unit
  • emergency department
  • open label
  • randomized controlled trial
  • clinical trial
  • study protocol
  • free survival
  • clinical evaluation