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

Annali dell'Istituto superiore di sanita

article

Francesca Abbonizio¹  Hamisa J. Hassan¹  Elena Santagostino²  on behalf of the Italian Association of Haemophilia Centres³  Romano Arcieri⁴  Mauro Biffoni¹  Adele Giampaolo¹ 

[1] Dipartimento di Oncologia e Medicina Molecolare, Istituto Superiore di Sanità;Ospedale Maggiore Policlinico;The members of the Italian Association of Haemophilia Centres (collaborators) are reported before the References;Grant Office e Trasferimento Tecnologico, Istituto Superiore di Sanità

关键词: paediatric individuals;    haemophilia A;    haemophilia B;    treatment strategies;   

DOI  :  10.4415/ANN_20_04_05

学科分类：社会科学、人文和艺术（综合）

来源: Istituto Superiore di Sanita

【 摘 要 】

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.

【 授权许可】

CC BY-NC-ND