Decitabine plus all-<i>trans</i> retinoic acid versus decitabine monotherapy for myelodysplastic syndromes with excess blasts: a multicenter, randomized controlled trial
作者：Xinping Zhou, Yanjuan Lin, Yan Gao, Zheng Ge, Huang Li, Jin Zhang, Hai Cheng, Guifang Ouyang, Fanjun Meng, Yulu Tian, Yuemin Kuang, Fengping Zhou, Lixia Sheng, Weimei Jin, Gaixiang Xu, Liya Ma, Li Ye, Chen Mei, Jian Li, Jie Jin, Hongyan Tong · 发表于：Haematologica · 年份：2025 · DOI：10.3324/haematol.2025.288526 · 被引用次数：2 · 研究领域：Acute Myeloid Leukemia Research、Retinoids in leukemia and cellular processes、Acute Lymphoblastic Leukemia research
Despite standard treatment with hypomethylating agents, the prognosis of patients with higher-risk myelodysplastic syndrome (MDS) remains poor. All-trans retinoic acid (ATRA) has demonstrated promising efficacy in unfit patients with acute myeloid leukemia. This multicenter controlled trial randomized (1:1) untreated patients with MDS with excess blasts (MDSEB) to ATRA plus decitabine (ATRA at 25 mg/m2/day in 2 divided daily doses throughout the 28-day cycle plus decitabine at 20 mg/m2 on days 1-5) or decitabine alone (20 mg/m2 on days 1-5). The primary endpoint was the overall response rate within four treatment cycles. A total of 227 patients were randomized. Four patients who did not commence therapy were excluded from the modified intention-to-treat (mITT) analysis. The median patient age was 62 years (range, 19-81). The overall response rate was 78% (86/110) in the ATRA group versus 51% (58/113) in the decitabine group (odds ratio =3.40; 95% confidence interval [CI]: 1.90-6.09; P<0.001). The ATRA group also had a higher complete remission rate (23% vs. 12%; odds ratio =2.05; 95% CI: 1.02-4.25; P=0.042). With a median follow-up of 30.1 months, progression-free survival (PFS) was 14.9 months in the ATRA group versus 10.5 months in the decitabine group (hazard ratio [HR]=0.70; 95% CI: 0.51-0.97; P=0.03). The overall survival was 23.0 months and 19.3 months, respectively (HR=0.77; 95% CI: 0.54-1.09; P=0.137). The two groups did not differ in grade 3 or higher hematological a...