Biologic Assignment Trial of Reduced-Intensity Hematopoietic Cell Transplantation Based on Donor Availability in Patients 50-75 Years of Age With Advanced Myelodysplastic Syndrome.
Biologic Assignment Trial of Reduced-Intensity Hematopoietic Cell Transplantation Based on Donor Availability in Patients 50-75 Years of Age With Advanced Myelodysplastic Syndrome.
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DOI：
10.1200/jco.20.03380
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发表时间：
2021-10-20
期刊：
影响因子：
--
通讯作者：
Cutler C
中科院分区：
文献类型：
--
作者：
Nakamura R;Saber W;Martens MJ;Ramirez A;Scott B;Oran B;Leifer E;Tamari R;Mishra A;Maziarz RT;McGuirk J;Westervelt P;Vasu S;Patnaik M;Kamble R;Forman SJ;Sekeres MA;Appelbaum F;Mendizabal A;Logan B;Horowitz M;Cutler C
异基因造血细胞移植（HCT）是骨髓增生异常综合征（MDS）唯一潜在的治愈性治疗方法，尽管它很少提供给老年患者。在老年高危MDS患者中，HCT相对于非HCT治疗的相对获益尚未确定。我们进行了一项多中心生物分配试验，在年龄50-75岁的中度-2或高风险新发MDS受试者中比较了低强度HCT与低甲基化治疗或最佳支持治疗。主要结局是3年时的总体生存概率。2014年1月至2018年11月期间，我们在34家临床试验机构入组了384例受试者。根据研究登记后90天内匹配供体的可用性，将受试者分配至供体组或非供体组。存活受试者的中位随访时间为34.2个月（范围：2.3-38个月）和26.9个月（范围：在意向治疗分析中，供体组3年的校正总生存率为47.9%（95% CI，41.3至54.1），而无供体组为26.6%（95% CI，18.4至35.6）（P = .0001），绝对差异为21.3%（95% CI，10.2至31.8）。与无供体组（20.6%; 95%CI，13.3 - 29.1; P = 0.003）相比，供体组3年无白血病生存率更高（35.8%; 95%CI，29.8 - 41.8）。在检查的所有亚组中均观察到生存获益。我们观察到，与无供体者相比，有匹配供体并接受低强度HCT的高危MDS老年受试者的生存率有显著优势。HCT应作为MDS管理计划的一个组成部分，纳入高危MDS的健康老年人。
Allogeneic hematopoietic cell transplantation (HCT) is the only potentially curative therapy for myelodysplastic syndromes (MDS), although it is infrequently offered to older patients. The relative benefits of HCT over non-HCT therapy in older patients with higher-risk MDS have not been defined. We conducted a multicenter biologic assignment trial comparing reduced-intensity HCT to hypomethylating therapy or best supportive care in subjects 50-75 years of age with intermediate-2 or high-risk de novo MDS. The primary outcome was overall survival probability at 3 years. Between January 2014 and November 2018, we enrolled 384 subjects at 34 centers. Subjects were assigned to the Donor or No-Donor arms according to the availability of a matched donor within 90 days of study registration. The median follow-up time for surviving subjects was 34.2 months (range: 2.3-38 months) in the Donor arm and 26.9 months (range: 2.4-37.2 months) in the No-Donor arm. In an intention-to-treat analysis, the adjusted overall survival rate at 3 years in the Donor arm was 47.9% (95% CI, 41.3 to 54.1) compared with 26.6% (95% CI, 18.4 to 35.6) in the No-Donor arm (P = .0001) with an absolute difference of 21.3% (95% CI, 10.2 to 31.8). Leukemia-free survival at 3 years was greater in the Donor arm (35.8%; 95% CI, 29.8 to 41.8) compared with the No-Donor arm (20.6%; 95% CI, 13.3 to 29.1; P = .003). The survival benefit was seen across all subgroups examined. We observed a significant survival advantage in older subjects with higher-risk MDS who have a matched donor identified and underwent reduced-intensity HCT, when compared with those without a donor. HCT should be included as an integral part of MDS management plans in fit older adults with higher-risk MDS.
影响因子：
4.3
作者：
Saber, Wael;Le Rademacher, Jennifer;Sekeres, Mikkael;Logan, Brent;Lewis, Moira;Mendizabal, Adam;Leifer, Eric;Appelbaum, Frederick R.;Horowitz, Mary M.;Nakamura, Ryotaro;Cutler, Corey S.
通讯作者：
Cutler, Corey S.