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Immune thrombocytopenia - what are the new treatment options?

机译:免疫性血小板减少症-有哪些新的治疗选择?

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Introduction: Primary immune thrombocytopenia (ITP) is a minor disease for many patients; however, the natural history is variable and unpredictable. Many patients who are in otherwise good health can be left untreated, especially if their platelet counts are close to 20 x 109/l. The overall mortality rate for ITP is < 1 %, and the morbidity and mortality associated with treatment can be worse than the disease. Areas covered: ITP is not purely due to platelet destruction and in a significant proportion platelet production is sub-optimal; the review will cover the newer developments with the thrombopoietin receptor agonists (TPOrAs) of which two agents, romiplostim and eltrombopag, are now licensed and these will be placed in context to conventional treatment. Expert opinion: There is an increasing understanding of the natural history of ITP and an increasing evidence-based approach to the disease and its management. Treatment should be aimed at the patient with a bleeding risk and should minimise the risk of adverse effects while maximising the chances of response. Few, if any, treatments are curative and treatment strategies need to be designed in ITP that manage patients with what can be a life-long condition with periodic relapses.
机译:简介:原发性免疫性血小板减少症(ITP)对许多患者来说是一种轻度疾病;但是,自然历史是多变且不可预测的。许多原本身体状况良好的患者可能不予治疗,尤其是血小板计数接近20 x 109 / l时。 ITP的总死亡率<1%,与治疗相关的发病率和死亡率可能比疾病差。涵盖的领域:ITP并非纯粹是由于血小板破坏造成的,而且血小板产量在次优水平上是次优的;这篇综述将涵盖血小板生成素受体激动剂(TPOrAs)的最新进展,其中两种药物romiplostim和eltrombopag已获许可,并将与常规治疗结合使用。专家意见:人们对ITP的自然史有了越来越多的了解,并且对这种疾病及其治疗的循证方法越来越多。治疗应针对有出血风险的患者,并应最大程度地减少不良反应的风险,同时最大程度地增加发生反应的机会。如果有的话,很少有治疗方法可以治愈的,因此需要在ITP中设计治疗策略,以治疗可能会终生且周期性复发的患者。

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