Eltrombopag for the Treatment of Refractory Pure RBC Aplasia after Major ABO Incompatible Hematopoietic Stem Cell Transplantation
Eltrombopag for the Treatment of Refractory Pure RBC Aplasia after Major ABO Incompatible Hematopoietic Stem Cell Transplantation
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DOI：
10.1016/j.bbmt.2018.04.022
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发表时间：
2018-08-01
影响因子：
4.3
通讯作者：
Bruno, Benedetto
中科院分区：
文献类型：
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作者：
Busca, Alessandro;Dellacasa, Chiara;Bruno, Benedetto
纯红细胞再生障碍性贫血(PRCS)是异基因造血干细胞移植(HSCT)后公认的并发症。有许多治疗方法可供选择，包括促红细胞生成素、利妥昔单抗、硼替佐米、血浆置换、免疫吸附、供体淋巴细胞输注、间充质干细胞、抗胸腺细胞球蛋白和大剂量类固醇；然而，治疗结果往往各不相同，有时会导致令人失望的结果。在这篇简短的文章中，我们报告了2例主要ABO血型不合HSCT后PRCA患者的经验，他们对多种治疗干预措施耐药，最终受益于eltrombopg的治疗，eltrombopg是一种美国食品和药物管理局批准的血小板生成素类似物，用于治疗免疫抑制剂无效或不符合HSCT条件的免疫性血小板减少性紫癜或严重再生障碍性贫血。这两名患者的数据显示，对于那些没有从多次治疗中受益的患者，eltrombopg对HSCT后红系再生障碍性贫血和输血依赖是有效的。此外，电疗耐受性良好，只有一过性的血小板增多需要调整剂量，没有克隆进化的证据。根据在这2例患者中获得的阳性结果，我们认为埃托巴可能对PRCA等单核细胞减少症有良好的疗效。在很大一部分患者中进行进一步研究是强制性的，以证实这些初步结果。(C)2018年，爱思唯尔公司代表美国血液和骨髓移植学会出版。
Pure RBC aplasia (PRCS) is a well-recognized complication after allogeneic hematopoietic stem cell transplantation (HSCT). Many therapeutic options are available to treat this condition, including erythropoietin, rituximab, bortezomib, plasma exchange, immunoadsorption, donor lymphocyte infusion, mesenchymal stem cells, antithymocyte globulin, and high-dose steroids; however, treatment outcomes are often variable and can sometimes lead to disappointing results. In this brief article we report our experience with 2 patients with PRCA after major ABO-incompatible HSCT who were resistant to multiple therapeutic interventions and who eventually benefited from treatment with eltrombopag, a thrombopoietin mimetic approved by the US Food and Drug Administration for the treatment of patients with immune thrombocytopenic purpura or severe aplastic anemia refractory to immunosuppressive agents or not eligible for HSCT. Data from these 2 patients show that eltrombopag was effective in treating erythroid aplasia and transfusion dependence after HSCT in patients who did not benefit from multiple previous treatments. Moreover, eltrombopag was well tolerated, with only a transient thrombocytosis requiring dose adjustment and no evidence of clonal evolution. Based on the positive results obtained in these 2 patients, we suggest that eltrombopag may have a favorable effect on unilineage cytopenias such as PRCA. Further studies in a large proportion of patients are mandatory to confirm these preliminary results. (C) 2018 Published by Elsevier Inc. on behalf of the American Society for Blood and Marrow Transplantation.