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首页> 外文期刊>Blood coagulation & fibrinolysis: an international journal in haemostasis and thrombosis >Low-dose tertiary prophylactic therapy reduces total number of bleeds and improves the ability to perform activities of daily living in adults with severe haemophilia A: a single-centre experience from Beijing
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Low-dose tertiary prophylactic therapy reduces total number of bleeds and improves the ability to perform activities of daily living in adults with severe haemophilia A: a single-centre experience from Beijing

机译：低剂量三级预防性治疗可减少严重血友病成人的出血总数并提高其进行日常生活活动的能力A：北京的单中心体验

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摘要

Full-dose prophylaxis treatment for persons with haemophilia is not affordable in China due to its economic constraints, particularly in adults requiring higher clotting factor (CFC) doses. Low-dose tertiary prophylaxis for adults with severe haemophilia A (SHA) in Beijing became feasible and implemented when government insurance covering 85% CFC cost in Beijing began in December 2009. The aim of this study was to evaluate the benefits of low-dose tertiary prophylaxis in SHA adults. Analysis of data on 33 patients on low-dose tertiary prophylaxis (5-10IU/kg, two to three times per week) at the Haemophilia Treatment Center, Peking Union Medical College Hospital between December 2009 and December 2013. The 33 patients (age 18-60 years, mean 33.4) were on prophylaxis for 20.8 +/- 9.9 months (compared with prior on-demand therapy for 20.0 +/- 11.7 months). Prophylaxis resulted in significant decrease in annual bleeding rate (ABR, 11.8 +/- 7.6 vs. 41.5 +/- 20.7, 71.1% reduction, P<0.0001), and significant improvement in Functional Independence Score in Haemophilia (FISH) measurement reflecting improvement in self-care and mobility. Radiologic (Pettersson) joint score was neither improved nor deteriorated. Ten of the 33 patients originally wheel chair and bed-bound began to walk and function independently in their daily lives. Low-dose tertiary prophylaxis for adults with SHA in China is feasible and beneficial. Although the average ABR remained high, a significant improvement in self-care and mobility measured by FISH was observed. These promising clinical experiences form the basis for further formal studies with more defined therapeutic protocol and outcome measures for affordable prophylaxis regimens in haemophilia adults in China. Copyright (C) 2016 Wolters Kluwer Health, Inc. All rights reserved.
机译：由于经济上的限制，在中国无法对全血友病患者进行全剂量预防治疗，尤其是在需要更高凝血因子（CFC）剂量的成年人中。自2009年12月开始在北京开始针对覆盖重度CFC费用的85％的政府保险后，对成年人进行重度A级血友病预防在北京开始实施。本研究的目的是评估低剂量三级血友病的益处。 SHA成人的预防。 2009年12月至2013年12月在北京协和医院血友病治疗中心对33例低剂量三级预防（5-10IU / kg，每周2至3次）的数据进行分析。33例（18岁） -60年（平均33.4岁）接受了20.8 +/- 9.9个月的预防（与之前的按需治疗20.0 +/- 11.7个月相比）。预防措施导致年出血率显着降低（ABR，11.8 +/- 7.6与41.5 +/- 20.7，降低71.1％，P <0.0001），并且血友病（FISH）功能独立性评分的显着改善反映了血友病的改善。自我保健和机动性。放射学（Pettersson）关节评分既无改善也无恶化。在最初由轮椅和床约束的33位患者中，有10位开始在日常生活中独立行走和工作。在中国，对成年人SHA的低剂量三级预防是可行和有益的。尽管平均ABR值仍然很高，但通过FISH测得的自我保健和行动能力显着改善。这些有前途的临床经验为进一步的正式研究奠定了基础，并为中国血友病成人提供了更明确的治疗方案和可负担得起的预防方案的结果指标。版权所有（C）2016 Wolters Kluwer Health，Inc.保留所有权利。

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