Division of Pediatric Hematology and Oncology, Masonic Children's Hospital, University of Minnesota, Minneapolis, Minnesota.
Department of Pediatric Hematology and Oncology, Children's Minnesota Hematology Oncology, Minneapolis, Minnesota.
Pediatr Blood Cancer. 2018 Oct;65(10):e27290. doi: 10.1002/pbc.27290. Epub 2018 Jun 22.
Aplastic anemia (AA) is characterized by multilineage cytopenias and bone marrow hypocellularity. Severe AA can be treated with immunosuppressive therapy (IST) and/or allogeneic hematopoietic stem cell transplantation. The thrombopoietin agonist eltrombopag has been shown to induce hematopoietic recovery and transfusion independence in adults with refractory and relapsed AA. Recently, upfront eltrombopag therapy in patients with AA in combination with IST has shown efficacy. Data for its use without concurrent IST in pediatric patients with AA remain sparse. Here we report two pediatric patients with AA not meeting severe criteria who achieved hematologic response with upfront eltrombopag monotherapy.
再生障碍性贫血（AA）的特征是多系细胞减少和骨髓细胞减少。严重的 AA 可以用免疫抑制治疗（IST）和/或异基因造血干细胞移植治疗。血小板生成素激动剂艾曲波帕已被证明可诱导成人难治性和复发性 AA 患者的造血恢复和输血独立性。最近，艾曲波帕与 IST 联合用于 AA 患者的一线治疗显示出疗效。其在不联合 IST 的儿科 AA 患者中的应用数据仍然很少。在这里，我们报告了两名不符合严重标准的 AA 儿科患者，他们使用艾曲波帕单药治疗获得了血液学反应。
