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.