Department of Hematology and Hematopoietic Stem Cell Transplantation, Yamanashi Prefectural Central Hospital, Kofu, Japan.
Hematology. 2022 Dec;27(1):360-366. doi: 10.1080/16078454.2022.2045725.
To explore the efficacy and safety of thrombopoietin receptor agonists (TPO-RAs) without anti-thymocyte globulin (ATG) in ATG-naïve patients with aplastic anemia (AA) in a real-world setting.
We retrospectively evaluated treatment outcomes in 45 consecutive ATG-naïve patients with AA who received TPO-RAs between 2017 and 2021 at our hospital.
ATG ineligibility was due to advanced age (≥ 70 years), = 22; not recommended under Japanese guidelines due to mild symptoms, = 13; patient preference, = 6; uncontrolled heart failure, = 2; uncontrolled diabetes mellitus, = 2; chronic renal failure, = 2; invasive aspergillosis, = 1. Twenty-eight patients (62%) achieved hematologic response in at least unilineage after 6 months' treatment, while 38 (84% in unilineage response-eligible patients) and four (25% in trilineage response-eligible patients) patients achieved at least unilineage and trilineage responses, respectively, at any point during the follow-up period. Five patients switched from eltrombopag to romiplostim because of adverse events or lack of efficacy, and two developed hematologic malignancies. Eltrombopag was effective even in elderly ATG-ineligible patients with severe AA. The 2-year overall survival rate was 84.3%, with a median 26.3-month follow-up. Time from diagnosis to eltrombopag treatment initiation tended to affect the response ( = 0.0727), but no factors that significantly predicted hematologic response were identified.
We found eltrombopag to be effective even in elderly ATG-naïve patients with severe AA, indicating that TPO-RA treatment should be considered in patients ineligible for ATG treatment because of age, complications, or severe AA.
在真实环境中，探索无抗胸腺细胞球蛋白（ATG）的血小板生成素受体激动剂（TPO-RA）在 ATG 初治的再生障碍性贫血（AA）患者中的疗效和安全性。
我们回顾性评估了 2017 年至 2021 年期间在我院接受 TPO-RA 治疗的 45 例 ATG 初治 AA 连续患者的治疗结局。
ATG 不适用的原因是年龄较大（≥70 岁），有 22 例；根据日本指南，由于症状较轻，不建议使用，有 13 例；患者偏好，有 6 例；未控制的心力衰竭，有 2 例；未控制的糖尿病，有 2 例；慢性肾衰竭，有 2 例；侵袭性曲霉病，有 1 例。28 例（62%）患者在 6 个月的治疗后至少在单一谱系中获得血液学反应，而 38 例（未达到单一谱系反应的患者中 84%）和 4 例（达到三谱系反应的患者中 25%）患者在随访期间的任何时间点至少达到单一谱系和三谱系反应。5 例患者因不良反应或疗效不佳而从艾曲波帕转为罗米司亭，2 例患者发生血液系统恶性肿瘤。即使在年龄较大 ATG 不适用的严重 AA 患者中，艾曲波帕也有效。2 年总生存率为 84.3%，中位随访时间为 26.3 个月。从诊断到艾曲波帕治疗开始的时间似乎会影响反应（ = 0.0727），但未发现可显著预测血液学反应的因素。
我们发现艾曲波帕即使在年龄较大 ATG 初治的严重 AA 患者中也有效，表明对于因年龄、并发症或严重 AA 而不适合 ATG 治疗的患者，应考虑 TPO-RA 治疗。
