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

Objectives. In a period of important therapeutic changes in the field of haemophilia care, we provide updated data on children with severe haemophilia (0-12 years of age) in Italy.Methods. Data presented are from the National Registry of Congenital Coagulopathies - survey 2017.Results. Children with severe HA were 242, those with severe HB were 48. Prophylaxis was adopted in 92.6% of child patients with severe HA and 88.6% with severe HB.  Thirty-nine children were under treatment for inhibitors.The FVIII prescribed to children with severe HA represented 11.1% of the total prescribed FVIII and 4.6% was extended half-life (EHL) FVIII. The FIX accounted for 7.2% of the total prescribed FIX and 19.1% was EHL-FIX.Conclusion. The child population analysed is characterized by a great adherence to therapy, so its therapeutic data may constitute a benchmark for future use of alternative therapeutic treatments in the coming years.