Emicizumab prophylaxis in infants: Single-centre experience

Sarina Levy-Mendelovich, Noa Greenberg-kushnir, Ivan Budnik, Assaf Arie Barg, Omri Cohen, Einat Avishai, Tami Barazani-Brutman, Tami Livnat, Gili Kenet

Research output: Contribution to journalArticlepeer-review

Abstract

The hallmark of haemophilia A (HA) therapy is prophylaxis, aimed at spontaneous bleeding prevention. Emicizumab provides a viable alternative to intravenous factor replacement therapy. However, data on its use in infants are limited. This single-centre open arm prospective study reports on emicizumab prophylaxis in infants. We included severe HA patients under 1 year who started emicizumab prophylaxis since 2018, with longitudinal follow-up. The study collected data on demographics, clinical and laboratory variables, the occurrence of bleeding events, surgeries and treatment outcomes. Of the 27 enrolled infants, whose median age at prophylaxis initiation was 7 months, 24 primarily choose to start emicizumab therapy (3/27 switched from FVIII prophylaxis due to development of FVIII inhibitors). The median age for prophylaxis initiation decreased to 3 months in 2023. Following emicizumab initiation, the median calculated ABR decreased, and no intracranial haemorrhages were observed. Thrombin generation showed a significant improvement in peak height and endogenous thrombin potential at steady state after a loading period. Our study highlights a shift towards early prophylaxis in the era of non-replacement therapies. It underscores the need for continuous evaluation and refinement of treatment approaches, emphasizing personalized care and diligent monitoring in the evolving field of paediatric haemophilia care.

Original languageEnglish
Pages (from-to)1375-1382
Number of pages8
JournalBritish Journal of Haematology
Volume204
Issue number4
StatePublished - Apr 2024
Externally publishedYes

Bibliographical note

Publisher Copyright:
© 2024 British Society for Haematology and John Wiley & Sons Ltd.

Keywords

  • emicizumab
  • haemophilia A
  • infants
  • paediatric
  • prophylaxis

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