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血友病患者中抑制剂的负担。

The burden of inhibitors in haemophilia patients.

作者信息

Walsh Christopher E, Jiménez-Yuste Víctor, Auerswald Guenter, Grancha Salvador

机构信息

Victor Jiménez-Yuste, Hospital Universitario La Paz - Hematology, Paseo de la Castellana 261 Apostol Santiago 61 1 J, Madrid 28017, Spain, Tel.: +34 619452698, Fax: +34 917277226, E-mail:

出版信息

Thromb Haemost. 2016 Aug 31;116 Suppl 1:S10-7. doi: 10.1160/TH16-01-0049. Epub 2016 Aug 16.

Abstract

The burden of disease in haemophilia patients has wide ranging implications for the family and to society. There is evidence that having a current inhibitor increases the risk of morbidity and mortality. Morbidity is increased by the inability to treat adequately and its consequent disabilities, which then equates to a poor quality of life compared with non-inhibitor patients. The societal cost of care, or `burden of inhibitors', increases with the ongoing presence of an inhibitor. Therefore, it is clear that successful eradication of inhibitors by immune tolerance induction (ITI) is the single most important milestone one can achieve in an inhibitor patient. The type of factor VIII (FVIII) product used in ITI regimens varies worldwide. Despite ongoing debate, there is in vitro and retrospective clinical evidence to support the use of plasma-derived VWF-containing FVIII concentrates in ITI regimens in order to achieve early and high inhibitor eradication success rates.

摘要

血友病患者的疾病负担对家庭和社会有着广泛的影响。有证据表明,当前存在抑制物会增加发病和死亡风险。由于无法进行充分治疗及其导致的残疾,发病率会增加,与无抑制物患者相比,这等同于生活质量较差。随着抑制物的持续存在,护理的社会成本,即“抑制物负担”会增加。因此,很明显,通过免疫耐受诱导(ITI)成功根除抑制物是抑制物患者能够实现的最重要的单一里程碑。ITI方案中使用的凝血因子VIII(FVIII)产品类型在全球范围内各不相同。尽管仍有争议,但有体外和回顾性临床证据支持在ITI方案中使用含血浆源性血管性血友病因子(VWF)的FVIII浓缩物,以实现早期和高抑制物根除成功率。

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