Department of Hematology/Oncology, Kyungpook National University Hospital, Daegu, Korea.
Department of Hematology/Oncology, Chonnam National University Hwasun Hospital, Hwasun, Korea.
Korean J Intern Med. 2018 Nov;33(6):1194-1202. doi: 10.3904/kjim.2016.426. Epub 2017 Dec 15.
BACKGROUND/AIMS: This study evaluated the role of hypomethylating agents (HMA) compared to best supportive care (BSC) for patients with high or very-high (H/VH) risk myelodysplastic syndrome (MDS) according to the Revised International Prognostic Scoring System.
A total of 279 H/VH risk MDS patients registered in the Korean MDS Working Party database were retrospectively analyzed.
HMA therapy was administered to 205 patients (73.5%), including 31 patients (11.1%) who then received allogeneic hematopoietic cell transplantation (allo-HCT), while 74 patients (26.5%) received BSC or allo-HCT without HMA. The 3-year overall survival (OS) rates were 53.1% ± 10.7% for allo-HCT with HMA, 75% ± 21.7% for allo-HCT without HMA, 17.3% ± 3.6% for HMA, and 20.8% ± 6.9% for BSC groups (p < 0.001). In the multivariate analysis, only allo-HCT was related with favorable OS (hazard ratio [HR], 0.356; p = 0.002), while very poor cytogenetic risk (HR, 5.696; p = 0.042), age ≥ 65 years (HR, 1.578; p = 0.022), Eastern Cooperative Oncology Group performance status (ECOG PS) 2 to 4 (HR, 2.837; p < 0.001), and transformation to acute myeloid leukemia (AML) (HR, 1.901; p = 0.001) all had an adverse effect on OS.
For the H/VH risk group, very poor cytogenetic risk, age ≥ 65 years, ECOG PS 2 to 4, and AML transformation were poor prognostic factors. HMA showed no benefit in terms of OS when compared to BSC. Allo-HCT was the only factor predicting a favorable long-term outcome. The use of HMA therapy did not seem to have an adverse effect on the transplantation outcomes. However, the conclusion of this study should be carefully interpreted and proven by large scale research in the future.
背景/目的：本研究根据修订后的国际预后评分系统（Revised International Prognostic Scoring System），评估了低甲基化药物（HMA）与最佳支持治疗（BSC）相比，在高或极高（H/VH）风险骨髓增生异常综合征（MDS）患者中的作用。
回顾性分析了韩国 MDS 工作组数据库中登记的 279 例 H/VH 风险 MDS 患者。
205 例患者（73.5%）接受了 HMA 治疗，其中 31 例（11.1%）随后接受了异基因造血细胞移植（allo-HCT），而 74 例（26.5%）接受了 BSC 或无 HMA 的 allo-HCT。allo-HCT 联合 HMA 的 3 年总生存率（OS）为 53.1%±10.7%，allo-HCT 无 HMA 为 75%±21.7%，HMA 为 17.3%±3.6%，BSC 为 20.8%±6.9%（p<0.001）。多变量分析显示，只有 allo-HCT 与良好的 OS 相关（风险比[HR]，0.356；p=0.002），而非常差的细胞遗传学风险（HR，5.696；p=0.042）、年龄≥65 岁（HR，1.578；p=0.022）、东部合作肿瘤学组体能状态（ECOG PS）2 至 4 分（HR，2.837；p<0.001）和转化为急性髓系白血病（AML）（HR，1.901；p=0.001）均对 OS 有不良影响。
对于 H/VH 风险组，非常差的细胞遗传学风险、年龄≥65 岁、ECOG PS 2 至 4 分和 AML 转化是不良预后因素。与 BSC 相比，HMA 对 OS 没有益处。allo-HCT 是唯一预测长期预后良好的因素。HMA 治疗的使用似乎对移植结果没有不良影响。然而，本研究的结论应在未来通过大规模研究仔细解释和验证。
