Elsayed Abdelrahman, Elsayed Basant, Elmarasi Mohamed, Elsabagh Ahmed Adel, Elsayed Engy, Elmakaty Ibrahim, Yassin Mohamed
Department of Medical Education, Hamad Medical Corporation, Doha, Qatar.
College of Medicine, Qatar University, Doha, Qatar.
Immunotargets Ther. 2024 Sep 13;13:461-486. doi: 10.2147/ITT.S463384. eCollection 2024.
Hematopoietic cell transplantation (HCT) is a well-established procedure that has become a therapeutic mainstay for various hematological conditions. Prolonged thrombocytopenia following HCT is associated with a significant risk of morbidity and mortality, yet no universally recognized treatment protocol exists for such a complication. First-generation thrombopoietin receptor (TpoR) agonists as well as second-generation agents are known for their role in enhancing platelet production, and their use is expanding across various thrombocytopenic conditions. Therefore, we conducted this comprehensive review of the literature to provide an updated evaluation of the use of TpoR agonists and explore their efficacy and safety in the treatment of extended post-HCT thrombocytopenia. The literature search was conducted using PubMed database from 1996 through December 2023, using a predefined strategy with medical subject headings terms. We identified 64 reports on the utility of TpoR agonists, five of them were randomized controlled trials and the rest were retrospective observational studies and case series, with a total number of 1730 patients. Second-generation TpoR agonists appear more convenient than subcutaneous recombinant human thrombopoietin (rhTpo) as they can be orally administered and exhibit similar efficacy in platelet recovery, as indicated by recent trial results. Among these agents, avatrombopag, unlike eltrombopag, does not require any dietary restrictions, which could be more favorable for patients. However, eltrombopag remains the most extensively studied agent. TpoR agonists had promising effects in the treatment of post-HCT thrombocytopenia with a good safety profile so far, highlighting the potential benefit of their use.
造血细胞移植（HCT）是一种成熟的治疗方法，已成为多种血液系统疾病的主要治疗手段。HCT后长期血小板减少与显著的发病和死亡风险相关，但对于这种并发症尚无普遍认可的治疗方案。第一代血小板生成素受体（TpoR）激动剂以及第二代药物因其在提高血小板生成方面的作用而闻名，并且它们在各种血小板减少症中的应用正在不断扩大。因此，我们对文献进行了全面综述，以提供对TpoR激动剂使用的最新评估，并探讨其在治疗HCT后持续性血小板减少症中的疗效和安全性。文献检索使用PubMed数据库，时间跨度为1996年至2023年12月，采用预定义的医学主题词策略。我们确定了64篇关于TpoR激动剂效用的报告，其中5篇是随机对照试验，其余为回顾性观察研究和病例系列，患者总数为1730例。近期试验结果表明，第二代TpoR激动剂似乎比皮下注射重组人血小板生成素（rhTpo）更方便，因为它们可以口服给药，并且在血小板恢复方面具有相似的疗效。在这些药物中，阿伐曲泊帕与艾曲泊帕不同，不需要任何饮食限制，这对患者可能更有利。然而，艾曲泊帕仍然是研究最广泛的药物。到目前为止，TpoR激动剂在治疗HCT后血小板减少症方面具有良好的效果和安全性，凸显了其使用的潜在益处。
