Vibor Milunovic, Rogulj Inga Mandac, Ostojic Slobodanka Kolonic
Lombardi Comprehensive Cancer Centre, Georgetown University, 3970 Reservoir Road NW E501, 20007 Washington D.C., United States.
Division of Hematology, Clinical Hospital Center, Zagreb. Croatia.
Cardiovasc Hematol Disord Drug Targets. 2017 Jul 4;17(1):38-51. doi: 10.2174/1871529X16666161229155608.
Immune thrombocytopenia (ITP) in adulthood is characterized by chronic relapsing course. Despite the efficacious first line treatment (corticosteroid, intravenous immunoglobulin), majority of patients will enter the chronic phase warranting another treatment approach. Until recently, splenectomy performed in ITP chronic phase represented the standard of care with longterm remissions in more than 70% of patients, but it has never been tested in clinical trials. However, with the advances of our understanding of ITP pathophysiology and the shifting focus on megakaryocyte impairment, novel drugs were introduced in the treatment paradigm, mainly trombopoietin receptor agonists (TPO-RAs); romiplostim and eltrombopag.
These TPO-RAs were tested in randomized controlled trials resulting in adequate platelet response with few side effects and less need for additional therapy leading to approval of corresponding regulatory agencies and wide acceptance by hematological community, but however TPO-RAs must be taken continuously to maintain the response. With their onset, the rate of splenectomy in chronic ITP has diminished in modern era.
The main aim behind conducting this review is to evaluate the pros and cons of splenectomy compared to TPO-RAs treatment in order to provide the critical overview which may help the practicing clinician in managing often challenging cases of chronic ITP.
成人免疫性血小板减少症(ITP)的特点是慢性复发性病程。尽管一线治疗(皮质类固醇、静脉注射免疫球蛋白)有效,但大多数患者会进入慢性期,需要另一种治疗方法。直到最近,ITP慢性期进行的脾切除术仍是标准治疗方法,超过70%的患者可实现长期缓解,但从未在临床试验中进行过验证。然而,随着我们对ITP病理生理学认识的进步以及对巨核细胞损伤关注重点的转移,新型药物被引入治疗模式,主要是血小板生成素受体激动剂(TPO-RAs);罗米司亭和艾曲泊帕。
这些TPO-RAs在随机对照试验中进行了测试,结果显示血小板反应良好,副作用少,且较少需要额外治疗,从而获得了相应监管机构的批准并被血液学界广泛接受,但TPO-RAs必须持续服用以维持反应。随着它们的出现,现代慢性ITP患者的脾切除率有所下降。
进行本综述的主要目的是评估脾切除术与TPO-RAs治疗相比的利弊,以便提供关键概述,帮助临床医生处理慢性ITP常见的棘手病例。