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[Practice changes in the management of major paediatric haemophilia]

Carine Cerato-Blanc1, Fabrice Monpoux1

  • 1Service d'hémato-oncologie infantile, hôpital Archet 2, centre hospitalier universitaire de Nice, 151 route Saint-Antoine-de-Ginestière, 06202 Nice, France.

Revue De L'Infirmiere
|September 27, 2021
PubMed

Insights

Emicizumab, a novel subcutaneous non-replacement therapy, is transforming severe hemophilia management in children. Initial results integrated into multi-professional care show promising outcomes for young patients.

Area of Science:

  • Pediatric Hematology
  • Pharmacology

Background:

  • Severe hemophilia management in children traditionally relies on replacement therapies.
  • Recent advancements aim to improve treatment efficacy and patient quality of life.

Purpose of the Study:

  • To share initial experiences with emicizumab, a novel subcutaneous non-replacement therapy.
  • To evaluate the integration of emicizumab into the multi-professional care of young children with severe hemophilia.

Main Methods:

  • A case series or experience sharing from the University Hospital of Nice.
  • Inclusion of emicizumab within a comprehensive, multi-professional pediatric care model.

Main Results:

  • Emicizumab demonstrated encouraging initial results in managing severe hemophilia.
  • The subcutaneous, non-replacement therapy offers a new therapeutic option.

Conclusions:

  • Emicizumab represents a significant therapeutic innovation for young children with severe hemophilia.
  • Integration into multi-professional care is key to successful implementation.

Keywords:
emicizumabhaemophiliahémophilieinnovation thérapeutiquemanagementprise en chargequality of lifequalité de viesafetysécuritétherapeutic innovation

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