State Key Laboratory of Experimental Hematology, Tianjin Laboratory of Blood Disease Gene Therapy, CAMS Key Laboratory of Gene Therapy for Blood Diseases, National Clinical Research Center for Hematological Disorders, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
Analysis Group, Inc., Beijing, China.
Haemophilia. 2020 Jul;26(4):584-590. doi: 10.1111/hae.14029. Epub 2020 May 20.
The management of haemophilia is critical to minimize the risk of disability and reduce the burden on China's healthcare system.
This study was based on a single centre in China and was conducted to understand the evolution of real-world haemophilia care over the past 15 years.
We retrospectively analysed clinical characteristics, diagnosis, treatment and medical expenditures of 428 patients with haemophilia from January 2004 to December 2018 from the Institute of Hematology & Blood Diseases Hospital in Tianjin, China.
The delayed diagnosis time significantly decreased from 13.3 ± 5.1 years before 2004 to 0.4 ± 0.4 year in 2014-2018 (P < .05). Among children and adults receiving prophylactic treatment, the annual factor consumption increased from 2004-2008 (168.8 IU/kg in children and 120.7 IU/kg in adults) to 2009-2013 (389.2 IU/kg in children and 316.2 IU/kg in adults) and 2014-2018 (1328.0 IU/kg in children and 878.8 IU/kg in adults, P < .001). The annual medical insurance expenditure for haemophilia had increased steadily over the past 10 years. The number of patients tested regularly for inhibitors increased from 2004 (1.9% [2/105]) to 2018 (21.5% [59/275]). The seroprevalence of hepatitis C virus (HCV) was 33.8% during the years examined, while the incidence rates of HCV among patients significantly decreased (7.3% in 2008 to 0.4% in 2018).
Significant improvements in the management of haemophilia were observed from 2004 to 2018. These results highlight the joint effort of the reimbursement policy and drug regulatory management paving the way for a better future for patients with haemophilia in China.
为了最大程度地降低残疾风险并减轻中国医疗体系的负担，对血友病的管理至关重要。
本研究基于中国的一个单中心，旨在了解过去 15 年真实世界中血友病治疗的演变。
我们回顾性分析了 2004 年 1 月至 2018 年 12 月期间来自中国天津血液病研究所的 428 例血友病患者的临床特征、诊断、治疗和医疗支出。
诊断延迟时间从 2004 年之前的 13.3±5.1 年显著缩短至 2014-2018 年的 0.4±0.4 年（P<.05）。在接受预防性治疗的儿童和成人中，年因子消耗量从 2004-2008 年（儿童 168.8IU/kg，成人 120.7IU/kg）增加到 2009-2013 年（儿童 389.2IU/kg，成人 316.2IU/kg）和 2014-2018 年（儿童 1328.0IU/kg，成人 878.8IU/kg，P<.001）。过去 10 年来，血友病的年度医疗保险支出稳步增长。定期检测抑制剂的患者数量从 2004 年的 1.9%（105 例中的 2 例）增加到 2018 年的 21.5%（275 例中的 59 例）。丙型肝炎病毒（HCV）的血清流行率在研究期间为 33.8%，而 HCV 在患者中的发生率显著降低（2008 年为 7.3%，2018 年为 0.4%）。
从 2004 年到 2018 年，血友病的管理得到了显著改善。这些结果突显了报销政策和药物监管管理的共同努力，为中国血友病患者的未来铺平了道路。
