单份脐带血与双份脐带血移植治疗血液系统恶性肿瘤的比较
One-unit versus two-unit cord-blood transplantation for hematologic cancers.
机构信息
From the University of Minnesota Medical School, Minneapolis (J.E.W., M.R.V.); Medical College of Wisconsin, Milwaukee (M.E., D.M., M.M.H.); EMMES Corporation, Rockville, MD (S.C., Y.W.); BC Children's Hospital, Vancouver (K.R.S.), and University of Manitoba, Winnipeg (D.A.W.) - both in Canada; Children's Hospital of Philadelphia, Philadelphia (N.B.); Fred Hutchinson Cancer Research Center, Seattle (C.D.); Indiana University, Indianapolis (P.H.); University of Michigan, Ann Arbor (E.P.); University of California San Francisco Benioff Children's Hospital Oakland, Oakland (M.W.); and Duke University, Durham, NC (J.K.).
出版信息
N Engl J Med. 2014 Oct 30;371(18):1685-94. doi: 10.1056/NEJMoa1405584.
BACKGROUND
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.
METHODS
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.
RESULTS
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.
CONCLUSIONS
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. (Funded by the National Heart, Lung, and Blood Institute and the National Cancer Institute; ClinicalTrials.gov number, NCT00412360.).
背景
据估计，已有 3 万人将脐带血用作造血干细胞的来源进行了 30000 次移植。由于单个脐带血单位中的造血细胞数量有限，因此不能用于体重较大的受者，这导致造血恢复延迟和死亡率升高。因此，我们假设两个脐带血单位中具有更多数量的造血细胞，与移植后更好的转归相关。
方法
2006 年 12 月 1 日至 2012 年 2 月 24 日，共有 224 名年龄在 1 至 21 岁之间的血液系统恶性肿瘤患者，按照统一的清髓性预处理方案和移植物抗宿主病（GVHD）的免疫预防方案，随机分为两组：双份脐带血移植组（111 例）和单份脐带血移植组（113 例）。主要终点是 1 年总生存率。
结果
两组在年龄、性别、自我报告种族（白种人与非白种人）、表现状态、供受者 HLA 匹配程度以及移植时疾病类型和状态方面相匹配。双份和单份脐带血移植组患者的 1 年总生存率分别为 65%（95%可信区间，56 至 74）和 73%（95%可信区间，63 至 80）（P=0.17）。两组在无病生存率、中性粒细胞恢复、移植相关死亡率、复发、感染、免疫重建和 II 至 IV 级急性 GVHD 方面的结果也相似。然而，单份脐带血移植组患者的血小板恢复更好，且 III 级和 IV 级急性和广泛慢性 GVHD 的发生率更低。
结论
我们发现，在患有血液系统恶性肿瘤的儿童和青少年中，单份和双份脐带血移植后的生存率相似；然而，单份脐带血移植与更好的血小板恢复和更低的 GVHD 风险相关。（由美国国立心肺血液研究所和美国国立癌症研究所资助；ClinicalTrials.gov 注册号：NCT00412360。）
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