TY - JOUR
T1 - Epidemiological data and treatment strategies in children with severe haemophilia in Italy
AU - Italian Association of Haemophilia Centres
AU - Abbonizio, Francesca
AU - Hassan, Hamisa J.
AU - Santagostino, Elena
AU - Arcieri, Romano
AU - Biffoni, Mauro
AU - Giampaolo, Adele
N1 - Copyright:
This record is sourced from MEDLINE/PubMed, a database of the U.S. National Library of Medicine
PY - 2020/10/1
Y1 - 2020/10/1
N2 - 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.
AB - 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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U2 - 10.4415/ANN_20_04_05
DO - 10.4415/ANN_20_04_05
M3 - Article
C2 - 33346169
AN - SCOPUS:85098744495
VL - 56
SP - 437
EP - 443
JO - Annali dell'Istituto Superiore di Sanita
JF - Annali dell'Istituto Superiore di Sanita
SN - 0021-2571
IS - 4
ER -