Composite end point of graft-versus-host disease-free, relapse-free survival after allogeneic hematopoietic cell transplantation
Composite end point of graft-versus-host disease-free, relapse-free survival after allogeneic hematopoietic cell transplantation
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DOI：
10.1182/blood-2014-10-609032
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发表时间：
2015-02-19
期刊：
影响因子：
20.3
通讯作者：
Weisdorf, Daniel J.
中科院分区：
文献类型：
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作者：
Holtan, Shernan G.;DeFor, Todd E.;Weisdorf, Daniel J.
异基因造血细胞移植（HCT）的成功通常被评估为个体并发症，包括移植物抗宿主病（GVHD），复发或死亡，但没有一个因素可以完全表征治愈而不持续发病。我们研究了一个新的复合终点，即无GVHD/无复发生存期（GRFS），其中事件包括3-4级急性GVHD、需要全身治疗的慢性GVHD、复发或HCT后第一年死亡。在907例连续的明尼苏达大学同种异体HCT接受者中（2000-2012年），1年GRFS为31%（95%置信区间[Cl] 28-34）。回归分析显示年龄、疾病风险和供体类型显著影响GRFS。21岁以上的成人GRFS比儿童差2倍; GRFS在21岁以上没有差异。根据预处理强度、干细胞来源、疾病风险、年龄和移植年份进行调整后，HLA匹配的同胞供体骨髓产生最佳GRFS（51%，95%CI 46-66），而HLA匹配的同胞供体外周血干细胞显著较差（25%，95%CI 20-30，P = 0.01）。脐带血移植后的GRFS和来自匹配的无关供体的骨髓移植后的GRFS相似（分别为31%，95%CI 27-35和32%，95%CI 22-42）。由于GRFS测量了无持续发病率，代表了理想的HCT恢复，因此GRFS具有作为新疗法基准的新终点的价值。
The success of allogeneic hematopoietic cell transplantation (HCT) is typically assessed as individual complications, including graft-versus-host disease (GVHD), relapse, or death, yet no one factor can completely characterize cure without ongoing morbidity. We examined a novel composite end point of GVHD-free/relapse-free survival (G RFS) in which events include grade 3-4 acute GVHD, systemic therapy-requiring chronic GVHD, relapse, or death in the first post-HCT year. In 907 consecutive University of Minnesota allogeneic HCT recipients (2000-2012), 1-year GRFS was 31% (95% confidence interval [Cl] 28-34). Regression analyses showed age, disease risk, and donor type significantly influencing GRFS. Adults age 21+ had 2-fold worse GRFS vs children; GRFS did not differ beyond age 21. Adjusted for conditioning intensity, stem cell source, disease risk, age, and transplant year, HLA-matched sibling donor marrow resulted in the best GRFS (51%, 95% Cl 46-66), whereas HLA-matched sibling donor peripheral blood stem cells were significantly worse (25%, 95% Cl 20-30, P =.01). GRFS after umbilical cord blood transplants and marrow from matched unrelated donors were similar (31%, 95% CI 27-35 and 32%, 95% Cl 22-42, respectively). Because GRFS measures freedom from ongoing morbidity and represents ideal HCT recovery, GRFS has value as a novel end point for benchmarking new therapies.