Department of Hematology and Oncology, University of Florida, Gainesville, Florida.
Pharmacy Department, Florida Hospital, Orlando, Florida.
Biol Blood Marrow Transplant. 2019 Jul;25(7):1320-1324. doi: 10.1016/j.bbmt.2019.01.027. Epub 2019 Jan 30.
Thrombocytopenia after allogeneic hematopoietic stem cell transplantation (allo-SCT) can pose significant problems in management of patients. Eltrombopag is a small-molecule thrombopoietin receptor agonist that has been approved for use in immune thrombocytopenic purpura and aplastic anemia; but its use after allo-SCT is limited. Between 2014 and 2017, we treated 13 patients with eltrombopag for poor platelet engraftment without evidence of relapse at the time of initiation, including 6 patients with primary platelet engraftment failure and 7 with secondary platelet engraftment failure. Eltrombopag was started at an initial dose of 25 or 50 mg per day, and dose adjustments were made in accordance with the manufacturer's recommendation. The cumulative incidence of platelet recovery to ≥50,000/μL without the need for transfusion for at least 7 days was defined as response. The overall response rate was 62% (n = 8). Of the 6 patients with primary isolated platelet failure, 3 (50%) responded, and of the 7 patients with secondary platelet failure, 5 (71%) responded. The median time to response was 33 days (range, 11 to 68 days). In addition, no significant differences in platelet recovery were noted in patients with adequate and decreased bone marrow megakaryocytic reserve (60% and 67%, respectively). Although eltrombopag was well tolerated, and no patient discontinued treatment because of adverse events, only 3 patients were alive at the end of the observation period, with relapse and graft-versus-host disease accounting for majority of the deaths. This suggested that despite the relatively good overall response rate to eltrombopag, inadequate platelet engraftment is a harbinger of poor outcome in allo-SCT.
异基因造血干细胞移植（allo-SCT）后血小板减少症可能给患者的管理带来重大问题。艾曲泊帕是一种小分子血小板生成素受体激动剂，已被批准用于治疗免疫性血小板减少性紫癜和再生障碍性贫血；但其在 allo-SCT 后的应用受到限制。2014 年至 2017 年，我们用艾曲泊帕治疗了 13 例无复发证据的血小板植入不良患者，包括 6 例原发性血小板植入失败和 7 例继发性血小板植入失败。艾曲泊帕起始剂量为每天 25 或 50 毫克，剂量调整根据制造商的建议进行。血小板恢复至≥50,000/μL 且至少 7 天无需输血的累积发生率定义为反应。总反应率为 62%（n=8）。在 6 例原发性孤立性血小板减少症患者中，有 3 例（50%）有反应，在 7 例继发性血小板减少症患者中，有 5 例（71%）有反应。反应的中位时间为 33 天（范围为 11 至 68 天）。此外，在骨髓巨核细胞储备充足和减少的患者中，血小板恢复无显著差异（分别为 60%和 67%）。虽然艾曲泊帕耐受性良好，且无患者因不良事件而停止治疗，但在观察期末仅 3 例患者存活，复发和移植物抗宿主病是导致大多数患者死亡的原因。这表明，尽管艾曲泊帕总体反应率相对较好，但血小板植入不良是 allo-SCT 预后不良的预示因素。
