Department of Hematology, The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology, Suzhou, China.
Institute of Blood and Marrow Transplantation, Suzhou, China.
J Hematol Oncol. 2018 Aug 16;11(1):103. doi: 10.1186/s13045-018-0649-6.
Poor graft function (PGF) is a life-threatening complication after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Current treatment strategies include the use of growth factors, CD34-selected stem cell boost, mesenchymal stem cell transfusion, and second allo-HSCT, but these treatments are not effective in all patients. Eltrombopag, an oral thrombopoietin receptor agonist, which showed promising results in severe aplasia anemia, may be an alternative choice for PGF patients. Therefore, we treated 12 patients who responded poorly to standard treatments for secondary PGF after allo-HSCT with eltrombopag. The median duration was 116 (35-1000) days from transplantation to PGF diagnosis and 59 (30-180) days from PGF diagnosis to eltrombopag treatment. Eltrombopag was started at a dose of 25 mg/d for 3 days and then increased to 50 or 75 mg/d. Median treatment duration was 8 (2-23) weeks. Ten patients (83.3%) responded to the treatment: 8 achieved complete response (CR), and the remaining 2 achieved partial response. In the 10 responding subjects, median platelet count was 18 (5-27) × 10/L vs 74 (30-117) × 10/L prior to and after treatment. Neutrophil count was 0.51 (0.28-0.69) × 10/L vs 1.84 (0.78-4.90) × 10/L. Hemoglobin was 88 (63-123) vs 101 (78-134) g/L. In the 8 patients who achieved CR, the time from eltrombopag initiation to achieving CR was 29 (10-49) days; the response lasted until the last follow-up in all 8 CR subjects (10-18 months). The 12-month overall survival rate was 83.3%. There was no treatment-related mortality and no evidence of cataract, thrombosis, or any other grade 3/4 toxicities.
移植物功能不良（PGF）是异基因造血干细胞移植（allo-HSCT）后的一种危及生命的并发症。目前的治疗策略包括使用生长因子、CD34 选择的干细胞增强、间充质干细胞输注和第二次 allo-HSCT，但这些治疗方法并非对所有患者都有效。血小板生成素受体激动剂艾曲波帕在严重再生障碍性贫血中显示出良好的效果，可能是 PGF 患者的另一种选择。因此，我们用艾曲波帕治疗了 12 例 allo-HSCT 后对标准治疗反应不佳的继发性 PGF 患者。从移植到 PGF 诊断的中位时间为 116（35-1000）天，从 PGF 诊断到艾曲波帕治疗的中位时间为 59（30-180）天。艾曲波帕起始剂量为 25mg/d，连用 3 天，然后增至 50 或 75mg/d。中位治疗持续时间为 8（2-23）周。10 例患者（83.3%）对治疗有反应：8 例达到完全缓解（CR），其余 2 例达到部分缓解。在 10 例有反应的患者中，血小板计数中位数为 18（5-27）×10/L，治疗前后分别为 74（30-117）×10/L。中性粒细胞计数中位数为 0.51（0.28-0.69）×10/L，治疗前后分别为 1.84（0.78-4.90）×10/L。血红蛋白中位数为 88（63-123）g/L，治疗前后分别为 101（78-134）g/L。在 8 例达到 CR 的患者中，从艾曲波帕开始到达到 CR 的时间为 29（10-49）天；在所有 8 例 CR 患者中，反应持续到最后一次随访（10-18 个月）。12 个月总生存率为 83.3%。无治疗相关死亡，无白内障、血栓形成或任何其他 3/4 级毒性的证据。
