Dipartimento di Oncologia e Medicina Molecolare, Istituto Superiore di Sanità, Rome, Italy.
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy.
Ann Ist Super Sanita. 2020 Oct-Dec;56(4):437-443. doi: 10.4415/ANN_20_04_05.
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.
Data presented are from the Italian National Registry of Congenital Coagulopathies (NRCC) - survey 2017.
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.
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.
在血友病治疗领域发生重大治疗变革的时期，我们提供了意大利重症血友病（0-12 岁）儿童的最新统计数据。
本研究数据来自意大利先天性凝血障碍国家登记处（NRCC）-2017 年调查。
重症甲型血友病（HA）患儿 242 例，重症乙型血友病（HB）患儿 48 例。92.1%的重症 HA 患儿和 88.6%的重症 HB 患儿采用预防治疗。39 名患儿（14.8%）正在接受抑制剂治疗。HA 重症患儿接受的 FVIII 占总用量的 11.1%，其中 4.6%为延长半衰期（EHL）制剂。HB 重症患儿接受的 FIX 占总 FIX 用量的 7.2%，其中 19.1%为 EHL-FIX。
分析的儿科人群具有较高的治疗依从性，因此这些数据可能成为未来几年使用新型替代疗法的基准。
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