Gulf States Hemophilia & Thrombophilia Center, University of Texas Health Science Center at Houston, Houston, TX, USA.
Division of Human Development and Disability, National Center on Birth Defects and Developmental Disabilities, CDC, Atlanta, GA, USA.
Haemophilia. 2017 Nov;23(6):910-917. doi: 10.1111/hae.13317. Epub 2017 Aug 6.
Prophylaxis is considered the optimal treatment for persons with moderate to severe haemophilia (factor activity between 1-5% of normal and <1% of normal respectively) in countries where safe factor concentrates are available and economically feasible. Historically, prophylactic treatment has not been well studied in the haemophilia B (HB) population due to difficulties in obtaining a sufficiently large sample.
This study examines the prevalence of prophylaxis use among a robust sample of persons with HB in the United States and its association with specific demographic and clinical characteristics.
Using data collected between 1998 and 2011 for the Centers for Disease Control and Prevention's Universal Data Collection project, we analysed data on 2428 males with moderate to severe HB aged 2-79 years who were seen at 135 federally funded haemophilia treatment centres.
Prevalence of prophylactic treatment in our sample was 35% among children and youth (ages 2-19) and 14% among adults (age 20 and older). Increased HB prophylaxis use was significantly associated with younger age (<40 years), Hispanic ethnicity, severe disease and self-infusion, while decreased use was associated with above-normal body mass index (BMI) in adults. Health care coverage was vital, although type of coverage did not appear to influence access.
Our analysis confirms previous reports of lower prevalence of prophylaxis use among individuals with HB compared to those with haemophilia A and adds to the body of knowledge regarding treatment patterns among a historically understudied population.
在安全的因子浓缩物可获得且在经济上可行的国家，对于因子活性分别为正常水平的 1-5%和<1%的中重度血友病（血友病 A）患者，预防治疗被认为是最佳治疗方法。由于难以获得足够大的样本量，历史上，在血友病 B（HB）患者人群中，预防性治疗并未得到很好的研究。
本研究在美国一个较大的 HB 患者样本中，考察了预防治疗的使用情况及其与特定人口统计学和临床特征的关系。
使用美国疾病控制与预防中心的通用数据收集项目在 1998 年至 2011 年期间收集的数据，我们分析了在 135 家联邦资助的血友病治疗中心就诊的 2428 名年龄在 2-79 岁的中重度 HB 男性的数据。
在我们的样本中，儿童和青少年（2-19 岁）中预防性治疗的患病率为 35%，成年（20 岁及以上）中为 14%。HB 预防治疗使用率的增加与年龄较小（<40 岁）、西班牙裔、重度疾病和自我输注显著相关，而在成年中，较高的体重指数（BMI）与预防治疗使用率降低相关。医疗保健覆盖至关重要，尽管覆盖类型似乎并不影响获得治疗的机会。
我们的分析证实了之前的报告，即与血友病 A 患者相比，HB 患者预防治疗的使用率较低，并增加了对历史上研究较少的人群的治疗模式的了解。
