Decitabine plus all-trans retinoic acid versus decitabine monotherapy for myelodysplastic syndromes with excess blasts: a multicenter, randomized controlled trial.

All-trans retinoic acid (ATRA) has demonstrated promising efficacy in unfit patients with acute myeloid leukemia.

全反式维甲酸（ATRA）在不适合接受强化疗的急性髓细胞性白血病患者中显示出有希望的疗效。

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).

这项多中心对照试验将未经治疗的骨髓增生异常综合征伴有过多原始细胞(MDSEB)患者随机分为两组（1:1），一组接受全反式维甲酸加去甲基化剂（ATRA 25 mg/m2/天，分两次口服，整个28天周期内，加上去甲基化剂20 mg/m2，第1-5天使用），另一组仅使用去甲基化剂（20 mg/m2，第1-5天使用）。

The primary endpoint was the overall response rate within four treatment cycles.

主要终点是在四个治疗周期内的总反应率。

A total of 227 patients were randomized.

共有227名患者被随机分配。

Four patients who did not commence therapy were excluded from the modified intention-to-treat(mITT) analysis.

未开始治疗的四名患者被排除在改良意向治疗(mITT)分析之外。

The median patient age was 62 years (range, 19-81).

患者的中位年龄为62岁（范围，19-81岁）。

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).

ATRA组的总缓解率为78%（86/110），而地西他滨组为51%（58/113）（比值比=3.40；95%置信区间[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).

ATRA组的完全缓解率也更高（23%对比12%；优势比=2.05；95%置信区间：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).

中位随访时间为30.1个月，ATRA组的无进展生存期（PFS）为14.9个月，而地西他滨组为10.5个月（风险比[HR]=0.70；95%置信区间：0.51-0.97；P=0.03）。

The overall survivalwas 23.0 months and 19.3 months, respectively (HR=0.77; 95% CI: 0.54-1.09; P=0.137).

总生存期分别为23.0个月和19.3个月（HR=0.77；95% 置信区间：0.54-1.09；P=0.137）。

The two groups did not differ in grade 3 or higher hematological adverse events.

两组在3级或更高级别的血液不良事件上没有差异。

In conclusion, adding ATRA to decitabine increased the overall response rate and prolonged PFS in adult patients with MDS-EB without increasing hematological toxicity.

总之，在成人MDS-EB患者中，添加ATRA到地西他滨治疗提高了总体反应率并延长了无进展生存期，且没有增加血液学毒性。

This study was registered at Chinese Clinical Trial Registry (www.chictr.org.cn, identifier: ChiCTR1800018307).

本研究已在中国临床试验注册中心注册（注册网址：www.chictr.org.cn，注册号：ChiCTR1800018307）。