Epidemiological data and treatment strategies in children with severe haemophilia in Italy

Italian Association of Haemophilia Centres

Research output: Contribution to journalArticlepeer-review

Abstract

OBJECTIVES: In a period of important therapeutic changes in the field of haemophilia care, we provide updated statistics on children with severe haemophilia (0-12 years of age) in Italy. METHODS: Data presented are from the Italian National Registry of Congenital Coagulopathies (NRCC) - survey 2017. RESULTS: Children with severe Haemophilia A (HA) were 242, those with severe haemophilia B (HB) 48. Prophylaxis was adopted in 92.1% of individuals with severe HA and 88.6% with severe HB. Thirty-nine children (14.8%) were on treatment for inhibitors. FVIII prescribed to children with severe HA represented 11.1% of the total consumption, of which 4.6% was extended half-life (EHL). FIX given to children with HB accounted for 7.2% of the total FIX, of which 19.1% was EHL-FIX. CONCLUSION: The paediatric population analysed is characterized by a great adherence to therapy, so this data may constitute a benchmark for use of new, alternative therapies in the coming years.

Original languageEnglish
Pages (from-to)437-443
Number of pages7
JournalAnnali dell'Istituto Superiore di Sanita
Volume56
Issue number4
DOIs
Publication statusPublished - Oct 1 2020

ASJC Scopus subject areas

  • Public Health, Environmental and Occupational Health

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