One-unit versus two-unit cord-blood transplantation for hematologic cancers.
One-unit versus two-unit cord-blood transplantation for hematologic cancers.
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DOI：
10.1056/nejmoa1405584
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发表时间：
2014-10-30
期刊：
影响因子：
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通讯作者：
Blood and Marrow Transplant Clinical Trials Network
中科院分区：
文献类型：
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作者：
Wagner JE Jr;Eapen M;Carter S;Wang Y;Schultz KR;Wall DA;Bunin N;Delaney C;Haut P;Margolis D;Peres E;Verneris MR;Walters M;Horowitz MM;Kurtzberg J;Blood and Marrow Transplant Clinical Trials Network
估计有 30,000 例移植手术使用脐带血作为造血干细胞的来源。单个脐带血单位中的造血细胞数量有限，无法用于体重较大的受者，并导致造血恢复延迟和死亡率较高。因此，我们假设两个单位的脐带血中造血细胞数量较多与移植后结局的改善有关。 2006年12月1日至2012年2月24日期间，共有224名1至21岁的血液癌患者在统一的清髓预处理方案和移植物抗宿主病（GVHD）免疫预防后被随机分配接受双单位（111名患者）或单单位（113名患者）脐带血移植。主要终点是一年总生存率。治疗组在年龄、性别、自我报告的种族（白人与非白人）、体能状态、供体-受体 HLA 匹配程度以及移植时的疾病类型和状态方面进行匹配。双份和单份脐带血受者的 1 年总体生存率分别为 65%（95% 置信区间 [CI]，56 至 74）和 73%（95% CI，63 至 80）（P = 0.17）。两组在无病生存率、中性粒细胞恢复率、移植相关死亡、复发、感染、免疫重建和 II-IV 级急性 GVHD 率方面也观察到类似的结果。然而，在单个脐带血单位的接受者中观察到血小板恢复得到改善，并且 III 级和 IV 级急性和广泛慢性 GVHD 的发生率降低。我们发现，在患有血液系统癌症的儿童和青少年中，单单位和双单位脐带血移植后的存活率相似；然而，单单位脐带血移植与更好的血小板恢复和更低的 GVHD 风险相关。
Umbilical-cord blood has been used as the source of hematopoietic stem cells in an estimated 30,000 transplants. The limited number of hematopoietic cells in a single cord-blood unit prevents its use in recipients with larger body mass and results in delayed hematopoietic recovery and higher mortality. Therefore, we hypothesized that the greater numbers of hematopoietic cells in two units of cord blood would be associated with improved outcomes after transplantation. Between December 1, 2006, and February 24, 2012, a total of 224 patients 1 to 21 years of age with hematologic cancer were randomly assigned to undergo double-unit (111 patients) or single-unit (113 patients) cord-blood transplantation after a uniform myeloablative conditioning regimen and immunoprophylaxis for graft-versus-host disease (GVHD). The primary end point was 1-year overall survival. Treatment groups were matched for age, sex, self-reported race (white vs. nonwhite), performance status, degree of donor–recipient HLA matching, and disease type and status at transplantation. The 1-year overall survival rate was 65% (95% confidence interval [CI], 56 to 74) and 73% (95% CI, 63 to 80) among recipients of double and single cord-blood units, respectively (P = 0.17). Similar outcomes in the two groups were also observed with respect to the rates of disease-free survival, neutrophil recovery, transplantation-related death, relapse, infections, immunologic reconstitution, and grade II–IV acute GVHD. However, improved platelet recovery and lower incidences of grade III and IV acute and extensive chronic GVHD were observed among recipients of a single cord-blood unit. We found that among children and adolescents with hematologic cancer, survival rates were similar after single-unit and double-unit cord-blood transplantation; however, a single-unit cord-blood transplant was associated with better platelet recovery and a lower risk of GVHD.