Department of Medicine, Østfold Hospital Trust Fredrikstad, Fredrikstad, Norway.
Semin Hematol. 2010 Jul;47(3):258-65. doi: 10.1053/j.seminhematol.2010.03.003.
Immune thrombocytopenia (ITP) is a disease characterized by accelerated platelet destruction and suboptimal platelet production. The latter concept has led to the exploration of new therapeutic options by focusing on the stimulation of platelet production as opposed to the traditional approach of immune suppression. Thrombopoietic agents act by stimulating the thrombopoietin receptor on the hematopoietic cells leading to stem cell differentiation, megakaryocyte proliferation, and platelet production. The last decade has witnessed the birth of second-generation thrombopoietic agents. Romiplostim and eltrombopag are the two agents that have been recently licensed. In randomized controlled trials, these agents have demonstrated unequivocal superiority over placebo in the treatment of ITP in splenectomized and in nonsplenectomized patients-an effect that seems to be durable while treatment continues and at an acceptable short-/intermediate-term safety profile. These agents represent a new therapeutic option in refractory ITP and in chronic ITP when splenectomy is contraindicated or needs to be deferred. However, the scope of therapeutic indications is expected to expand should long-term safety be confirmed in the ongoing studies. Several other agents are currently being investigated and are in preclinical and clinical development programs.
免疫性血小板减少症（ITP）是一种以血小板破坏加速和血小板生成欠佳为特征的疾病。后者的概念促使人们探索新的治疗选择，重点关注刺激血小板生成，而不是传统的免疫抑制方法。促血小板生成素制剂通过刺激造血细胞上的促血小板生成素受体，导致干细胞分化、巨核细胞增殖和血小板生成。过去十年见证了第二代促血小板生成素制剂的诞生。罗米司亭和艾曲泊帕是最近获得许可的两种药物。在随机对照试验中，这些药物在治疗脾切除和非脾切除患者的 ITP 方面，与安慰剂相比具有明确的优越性——这种疗效似乎在持续治疗期间和可接受的短期/中期安全性特征下是持久的。这些药物代表了难治性 ITP 和慢性 ITP 的新治疗选择，当脾切除术禁忌或需要推迟时。然而，如果正在进行的研究证实长期安全性，治疗适应症的范围预计将扩大。目前正在研究其他几种药物，并处于临床前和临床开发计划中。
