Division of Hematology-Oncology, Department of Internal Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea; Division of Hematology-Oncology, Department of Internal Medicine, Pusan National University Yangsan Hospital, Pusan National University School of Medicine, Yangsan, Korea.
Acute Leukemia Center, Seoul St Mary's Hematology Hospital, College of Medicine, Catholic University of Korea, Seoul, Korea.
Clin Lymphoma Myeloma Leuk. 2019 Oct;19(10):656-664. doi: 10.1016/j.clml.2019.06.003. Epub 2019 Jun 27.
Decitabine has shown clinical benefits in patients with intermediate (INT)-2 or high-risk myelodysplastic syndrome (MDS), determined according to the International Prognostic Scoring System (IPSS), but the benefits have not been well demonstrated in patients with lower-risk (IPSS low or INT-1) disease. Recently, it was proposed that the prognosis for patients with IPSS lower-risk disease is heterogeneous, with a substantial proportion of these patients having poor survival.
This study included patients with IPSS lower-risk MDS from the DRAMA (An Observational Study for Dacogen Long-Term Treatment in Patients With Myelodysplastic Syndrome; NCT01400633) and DIVA (A Study for Dacogen Treatment in Patients With Myelodysplastic Syndrome; NCT01041846) studies, which were prospective observational studies on the efficacy and safety of decitabine treatment in patients with MDS. Using the Lower-Risk Prognostic Scoring System [LR-PSS], we classified IPSS lower-risk MDS. Patients in each LR-PSS category were divided according to overall response (OR) to decitabine treatment, and survival outcomes were compared.
One hundred sixteen patients were enrolled: LR-PSS category 1 (n = 12; 10.3%), category 2 (n = 56; 48.3%), and category 3 (n = 48; 41.4%). Survival outcomes differed among the 3 categories (P = .046). The overall survival according to OR showed a significant difference in total patients (P = .008) and category 3 patients (P = .003). We analyzed predictive factors for OR, but no variable was found to significantly affect OR.
Decitabine treatment showed a survival benefit in the higher-risk group of IPSS lower-risk MDS patients who responded to treatment, and classification using the LR-PSS category was helpful for this subgroup, indicating that decitabine treatment might alter the natural course of disease in these patients.
地西他滨已显示出在国际预后评分系统（IPSS）确定的中危（INT）-2 或高危骨髓增生异常综合征（MDS）患者中具有临床获益，但在低危（IPSS 低或 INT-1）疾病患者中并未得到很好的证实。最近，有人提出，低危 IPSS 疾病患者的预后存在异质性，其中相当一部分患者的生存情况较差。
本研究纳入了来自 DRAMA（地西他滨治疗骨髓增生异常综合征患者的观察性研究；NCT01400633）和 DIVA（地西他滨治疗骨髓增生异常综合征患者的研究；NCT01041846）研究的低危 MDS 患者，这两项研究是对地西他滨治疗 MDS 患者的疗效和安全性的前瞻性观察性研究。采用低危预后评分系统[LR-PSS]对 IPSS 低危 MDS 进行分类。根据对地西他滨治疗的总体反应（OR）将每个 LR-PSS 类别中的患者进行分组，并比较生存结果。
共纳入 116 例患者：LR-PSS 类别 1（n=12；10.3%）、类别 2（n=56；48.3%）和类别 3（n=48；41.4%）。3 个类别之间的生存结果存在差异（P=0.046）。根据 OR 计算的总生存率在所有患者（P=0.008）和类别 3 患者（P=0.003）中均存在显著差异。我们分析了 OR 的预测因素，但未发现任何变量对 OR 有显著影响。
地西他滨治疗在对治疗有反应的低危 MDS 患者的高危 IPSS 组中显示出生存获益，使用 LR-PSS 分类对该亚组具有帮助，表明地西他滨治疗可能改变这些患者的疾病自然病程。
