Multicenter biologic assignment trial comparing reduced-intensity allogeneic hematopoietic cell transplant to hypomethylating therapy or best supportive care in patients aged 50 to 75 with intermediate-2 and high-risk myelodysplastic syndrome: Blood and Marrow Transplant Clinical Trials Network #1102 study rationale, design, and methods.
Multicenter biologic assignment trial comparing reduced-intensity allogeneic hematopoietic cell transplant to hypomethylating therapy or best supportive care in patients aged 50 to 75 with intermediate-2 and high-risk myelodysplastic syndrome: Blood and Marrow Transplant Clinical Trials Network #1102 study rationale, design, and methods.
复制标题
DOI：
10.1016/j.bbmt.2014.06.010
复制
发表时间：
2014-10
影响因子：
4.3
通讯作者：
Cutler, Corey S.
中科院分区：
文献类型：
--
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.
降低强度预处理方案（RIC）的引入使得向患有骨髓增生异常综合征（MDS）的老年患者提供异基因造血细胞移植（alloHCT）成为可能。然而，与新型非移植疗法相比，alloHCT的相对风险和益处仍然是相当大的不确定性的来源。我们将进行一项前瞻性生物分配试验，根据供体可用性比较RIC alloHCT与非移植治疗。主要结局是3年总生存率。次要结局包括无白血病生存率、生活质量和成本效益。将在大约3年内入组400例患者。计划的亚组分析将评估关键的生物学问题，如年龄和对低甲基化药物的反应对治疗效果的影响。这项研究的结果可能为被认为是alloHCT候选人的老年MDS患者制定新的护理标准。
The introduction of reduced intensity conditioning regimens (RIC) made it possible to offer allogeneic hematopoietic cell transplantation (alloHCT) to older patients with myelodysplastic syndromes (MDS). However, the relative risks and benefits of alloHCT compared to novel non-transplant therapies continue to be the source of considerable uncertainty. We will perform a prospective biologic assignment trial to compare RIC alloHCT to non-transplant therapies based on donor availability. Primary outcome is 3-year overall survival. Secondary outcomes include leukemia-free survival, quality of life, and cost-effectiveness. Four hundred patients will be enrolled over roughly 3 years. Planned subgroup analyses will evaluate key biologic questions, such as the impact of age & response to hypomethylating agents on treatment effects. Findings from this study potentially may set a new standard of care for older MDS patients who are considered candidates for alloHCT.
登录
查看更多内容
影响因子：
20.3
作者：
Itzykson, Raphael;Thepot, Sylvain;Fenaux, Pierre
通讯作者：
Fenaux, Pierre
影响因子：
6.2
作者：
Ma, Xiaomei;Does, Monique;Mayne, Susan T.
通讯作者：
Mayne, Susan T.
影响因子：
45.3
作者：
Luebbert, Michael;Suciu, Stefan;Wijermans, Pierre W.
通讯作者：
Wijermans, Pierre W.
影响因子：
158.5
作者：
Cassileth, PA;Harrington, DP;Wiernik, PH
通讯作者：
Wiernik, PH
影响因子：
2.7
作者：
Logan, Brent;Leifer, Eric;Geller, Nancy
通讯作者：
Geller, Nancy