Epidemiological data and treatment strategies in children with severe haemophilia in Italy
Authors
Francesca Abbonizio, Hamisa J. Hassan, Elena Santagostino, on behalf of the Italian Association of Haemophilia Centres, Romano Arcieri, Mauro Biffoni, Adele Giampaolo
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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