Prolonged thrombocytopenia after hematopoietic stem cell transplantation (HSCT) is a strong risk factor for transplantation-related morbidity and mortality, and no standard treatment guideline exists. Thrombopoietin receptor agonists (TPO-RAs), eltrombopag and romiplostim, increases the platelet production, and are being increasingly used in various conditions with thrombocytopenia. In this review, we present an overview of these TPO-RAs and review their efficacy and safety in prolonged post-HSCT thrombocytopenia. Through a systematic literature search, we identified 25 reports describing their use for this indication. Thirteen reports (8 case series and 5 case reports) described the use of eltrombopag in 78 patients with prolonged isolated thrombocytopenia (PIT) and 43 patients with secondary failure of platelet recovery (SFPR). A consistent and durable response with a rise in platelet counts >50 × 10 9/L for 7 consecutive days without platelet transfusion was seen in 85 of 121 patients (overall response rate [ORR], 70%). The responders included 56 patients with PIT (ORR for PIT, 72%) and 29 patients with SFPR (ORR for SFPR, 67%). No serious grade 3 or 4 adverse effects were reported. Similarly, 12 reports (6 case series and 6 case reports) described the use of romiplostim in prolonged post-HSCT thrombocytopenia (in 17 patients with PIT and 32 patients with SFPR). Response with the increment of platelet count was described in 40 out of 49 patients (ORR, 82%). Among the responders, 10 patients had PIT (ORR for PIT, 59%) and 30 patients had SFPR (ORR for SFPR, 94%). Our data show that TPO-RAs have an overall favorable response rate for both PIT and SFPR with a reasonable safety profile. However, given the lack of control groups, study heterogeneity, and the potential publication bias, these results should be interpreted with caution.

中文翻译：

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造血干细胞移植后延长血小板减少症中血小板生成素受体激动剂的使用。

造血干细胞移植（HSCT）后血小板减少症是与移植相关的发病率和死亡率的重要危险因素，目前尚无标准的治疗指南。血小板生成素受体激动剂（TPO-RAs），Eltrombopag和romiplostim，增加血小板生成，并越来越多地用于各种血小板减少症。在这篇综述中，我们概述了这些TPO-RA，并综述了它们在延长HSCT后血小板减少症中的功效和安全性。通过系统的文献检索，我们确定了25份描述其用于此适应症的报告。13份报告（8例系列和5例报告）描述了Eltrombopag在78例长期孤立性血小板减少症（PIT）和43例继发性血小板恢复失败（SFPR）患者中的使用。121名患者中有85名患者连续7天获得了持续且持久的应答，血小板计数增加> 50×10 9 / L，无血小板输注（总应答率[ORR]为70％）。响应者包括56例PIT患者（PIT为ORR，占72％）和29例SFPR患者（对于SFPR，ORR，占67％）。没有严重的3级或4级不良反应的报道。同样，有12个报告（6个病例系列和6个病例报告）描述了romiplostim在延长的HSCT后血小板减少症（17例PIT患者和32例SFPR患者）中的使用。在49位患者中有40位（ORR，82％）描述了随着血小板计数增加而引起的反应。在应答者中，有10例患者发生PIT（PIT为ORR，占59％），有30例患有SFPR（对于SFPR为ORR，占94％）。我们的数据表明，TPO-RA对PIT和SFPR总体上具有良好的响应率，并且具有合理的安全性。但是，由于缺乏对照组，研究异质性和潜在的出版偏见，应谨慎解释这些结果。