低危骨髓增生异常综合征患者对红细胞生成刺激剂反应的预测。
Prediction of Response to Erythropoiesis Stimulating Agents in Low-Risk Myelodysplastic Syndromes.
机构信息
Department of Medicine, Faculty of Medicine, Chulalongkorn University, King Chulalongkorn Memorial Hospital, Bangkok, Thailand.
Research Unit in Translational Hematology, Chulalongkorn University, Bangkok, Thailand.
出版信息
Asian Pac J Cancer Prev. 2021 Dec 1;22(12):4037-4042. doi: 10.31557/APJCP.2021.22.12.4037.
INTRODUCTION
Erythropoiesis stimulating agents (ESAs) represents the principal treatments for anemia in patients with lower-risk myelodysplastic syndromes (MDS). Pre-treatment erythropoietin (EPO) level and previous blood transfusion requirement are the two major predictors for response to ESAs. However, most evidence was derived from Western countries whereas there have been limited data in patients with Asian background.
METHODS
We retrospectively collected data on patients with low-risk MDS who received ESAs. Erythroid response was evaluated according to IWG 2006 criteria. MDS subtypes, r-IPSS, baseline hemoglobin (Hb), ESAs dosage and erythropoietin level were reviewed from medical records. Gene mutations were analyzed in patients' blood or bone marrow at diagnosis by 40-gene myeloid panel targeted sequencing. Clinical and laboratory parameters were compared between erythroid responder and non-responder groups.
RESULTS
A total of 47 patients were recruited in the study. The median age at diagnosis of the patients in this cohort was 77 years (IQR, 70-83) and 44.7% were male. The median revised international prognostic scoring system (R-IPSS) score of patients was 2.5. Response rate to ESAs was 46.8% (22/47). Median EPO level in responders was significantly lower than non-responders (27.7 vs. 59.1 U/L, p=0.02). Median ESAs dosage in responder group was 30,000 units per week. Cytogenetic abnormalities were detected in 27.3% and 24% of the responder and non-responder groups, respectively. Of 22 patients with available 40 gene mutation targeted sequencing, ASXL1, IDH2 and TET2 represented the 3 most common mutations and were found in 22%, 22% and 17%, respectively. There were no differences in cytogenetic abnormalities and gene mutations between groups. Patients who responded to ESAs showed a higher 5-year overall survival (OS) compared to non-responders (5-year OS 75% vs. 60.9%; p=0.008).
CONCLUSION
We conclude that a low serum EPO level is a predictive factor for responsiveness to ESAs in Asian patients with low-risk MDS.
简介
红细胞生成刺激剂（ESA）是治疗低危骨髓增生异常综合征（MDS）患者贫血的主要方法。治疗前的促红细胞生成素（EPO）水平和既往输血需求是预测 ESA 反应的两个主要因素。然而，大多数证据来自西方国家，而亚洲背景患者的数据有限。
方法
我们回顾性收集了接受 ESA 治疗的低危 MDS 患者的数据。根据 IWG 2006 标准评估红细胞反应。从病历中回顾 MDS 亚型、r-IPSS、基线血红蛋白（Hb）、ESA 剂量和 EPO 水平。在诊断时，通过 40 基因髓系靶向测序分析患者血液或骨髓中的基因突变。比较红细胞反应者和非反应者组的临床和实验室参数。
结果
本研究共纳入 47 例患者。该队列患者的中位诊断年龄为 77 岁（IQR，70-83），44.7%为男性。患者的中位修订国际预后评分系统（R-IPSS）评分为 2.5。ESA 反应率为 46.8%（22/47）。反应者的 EPO 水平中位数明显低于非反应者（27.7 与 59.1 U/L，p=0.02）。反应者组 ESA 剂量中位数为 30000 单位/周。在反应者和非反应者组中，分别检测到 27.3%和 24%的细胞遗传学异常。在 22 名有可用 40 基因突变靶向测序的患者中，ASXL1、IDH2 和 TET2 分别代表 3 种最常见的突变，分别为 22%、22%和 17%。两组间细胞遗传学异常和基因突变无差异。对 ESA 有反应的患者 5 年总生存率（OS）高于无反应者（5 年 OS 75%与 60.9%；p=0.008）。
结论
我们得出结论，低血清 EPO 水平是亚洲低危 MDS 患者对 ESA 反应的预测因素。
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