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评估风险因素:血友病抑制剂的预防。

Assessing risk factors: prevention of inhibitors in haemophilia.

机构信息

Service d'Hématologie Pédiatrique, Hôpital d'Enfants La Timone, Université de la Méditerranée, Marseille, France.

出版信息

Haemophilia. 2010 Mar;16 Suppl 2:10-5. doi: 10.1111/j.1365-2516.2009.02197.x.

Abstract

The formation of antibodies against factor VIII or factor IX that inhibit replacement therapy is currently the most serious treatment-related complication faced by patients with haemophilia. This review highlights non-modifiable and modifiable risk factors that determine the development of these antibodies. The non-modifiable risk factors include patient genotype for haemophilia, immunogenotype, ethnicity and positive family history. Age, intensity of treatment and the type of clotting factor administered are identified as modifiable risk factors. These risk factors are likely to be identified more accurately in forthcoming prospective randomized controlled trials and current patient registries. Through a more complete picture of a patient's overall risk profile, individually tailored treatment schedules might be developed that could minimize the incidence of inhibitor formation and thus maximize therapeutic benefit.

摘要

针对凝血因子 VIII 或凝血因子 IX 的抗体的形成会抑制替代疗法,这是目前血友病患者面临的最严重的与治疗相关的并发症。本综述强调了决定这些抗体产生的不可变和可改变的危险因素。不可变的危险因素包括血友病患者的基因型、免疫基因型、种族和阳性家族史。年龄、治疗强度和所给予的凝血因子类型被确定为可改变的危险因素。这些危险因素很可能在即将进行的前瞻性随机对照试验和当前的患者登记册中更准确地识别。通过更全面地了解患者的整体风险概况,可以制定个性化的治疗方案,最大限度地减少抑制剂形成的发生率,从而最大限度地提高治疗效果。

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