Decitabine plus all-<i>trans</i> retinoic acid versus decitabine monotherapy for myelodysplastic syndromes with excess blasts: a multicenter, randomized controlled trial
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 (MDS-EB) 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 4 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 [OR] 3.40; 95% confidence interval [CI] 1.90-6.09; p.
Authors
- Jie Jin (ORCID: https://orcid.org/0000-0002-8166-9915)
- Liya Ma (ORCID: https://orcid.org/0009-0000-6438-411X)
- Yuemin Kuang
- Yulu Tian
- Yanjuan Lin (ORCID: https://orcid.org/0000-0001-8953-7549)
- Gaixiang Xu (ORCID: https://orcid.org/0000-0002-4932-5813)
- Fengping Zhou
- Guifang Ouyang (ORCID: https://orcid.org/0000-0002-3411-3666)
- Li Ye (ORCID: https://orcid.org/0000-0002-9246-5520)
- Chen Mei (ORCID: https://orcid.org/0000-0001-8071-8175)
- Jin Zhang (ORCID: https://orcid.org/0000-0001-5416-0964)
- Huang Li (ORCID: https://orcid.org/0000-0003-4669-3466)
- Weimei Jin
- Xinping Zhou
- Fanjun Meng (ORCID: https://orcid.org/0000-0001-5731-1203)
- Lixia Sheng
- Jian Li (ORCID: https://orcid.org/0009-0002-8613-4669)
- Hongyan Tong
- Zheng Ge
- Hai Cheng
- Yan Gao (ORCID: https://orcid.org/0000-0003-0192-8786)
Institutions
- Qingdao University (CN)
- Fujian Medical University (CN)
- Xuzhou Medical College (CN)
- Ruijin Hospital (CN)
- Sir Run Run Shaw Hospital (CN)
- Zhongda Hospital Southeast University (CN)
- Affiliated Hospital of Xuzhou Medical College (CN)
- Affiliated Hospital of Qingdao University (CN)
- Jinhua Academy of Agricultural Sciences (CN)
- Fujian Provincial Hospital (CN)
- Ningbo University Affiliated Hospital (CN)
- Jinhua Central Hospital (CN)
- Ningbo First Hospital (CN)
- First Affiliated Hospital Zhejiang University (CN)
- Zhejiang University (CN)
Publication Details
- Journal
- Haematologica
- Published
- 2025-11-20
- DOI
- https://doi.org/10.3324/haematol.2025.288526
- Primary Topic
- Acute Myeloid Leukemia Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- National Natural Science Foundation of China
- National Health Commission of the People's Republic of China