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. 2020 Oct-Dec;56(4):437-443.
doi: 10.4415/ANN_20_04_05.

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

Francesca Abbonizio  1 , Hamisa J Hassan  1 , Elena Santagostino  2 , Romano Arcieri  3 , Mauro Biffoni  1 , Adele Giampaolo  1 ; Italian Association of Haemophilia Centres; Italian Association of Haemophilia Centres
Collaborators, Affiliations
Free article

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

Francesca Abbonizio et al. Ann Ist Super Sanita. 2020 Oct-Dec.
Free article

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.

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