Gerds Aaron T, Woo Ahn Kwang, Hu Zhen-Huan, Abdel-Azim Hisham, Akpek Gorgun, Aljurf Mahmoud, Ballen Karen K, Beitinjaneh Amer, Bacher Ulrike, Cahn Jean-Yves, Chhabra Saurabh, Cutler Corey, Daly Andrew, DeFilipp Zachariah, Gale Robert Peter, Gergis Usama, Grunwald Michael R, Hale Gregory A, Hamilton Betty Ky, Jagasia Madan, Kamble Rammurti T, Kindwall-Keller Tamila, Nishihori Taiga, Olsson Richard F, Ramanathan Muthalagu, Saad Ayman A, Solh Melhem, Ustun Celalettin, Valcárcel David, Warlick Erica, Wirk Baldeep M, Kalaycio Matt, Alyea Edwin, Popat Uday, Sobecks Ronald, Saber Wael
Hematology and Medical Oncology, Taussig Cancer Institute, Cleveland Clinic, Cleveland, Ohio.
Center for International Blood and Marrow Transplant Research, Department of Medicine, Medical College of Wisconsin, Milwaukee, Wisconsin; Division of Biostatistics, Institute for Health and Society, Medical College of Wisconsin, Milwaukee, Wisconsin.
Biol Blood Marrow Transplant. 2017 Jun;23(6):971-979. doi: 10.1016/j.bbmt.2017.03.014. Epub 2017 Mar 10.
For patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation, umbilical cord blood transplantation (UCBT) has become an acceptable alternative donor source in the absence of a matched sibling or unrelated donor. To date, however, there have been few published series dedicated solely to describing the outcomes of adult patients with myelodysplastic syndrome (MDS) who have undergone UCBT. Between 2004 and 2013, 176 adults with MDS underwent UCBT as reported to the Center for International Blood and Marrow Transplant Research. Median age at the time of transplantation was 56 years (range, 18-73 years). The study group included 10% with very low, 23% with low, 19% with intermediate, 19% with high, and 13% with very high-risk Revised International Prognostic Scoring System (IPSS-R) scores. The 100-day probability of grade II-IV acute graft-versus-host disease (GVHD) was 38%, and the 3-year probability of chronic GVHD was 28%. The probabilities of relapse and transplantation-related mortality (TRM) at 3 years were 32% and 40%, respectively, leading to a 3-year disease-free survival (DFS) of 28% and an overall survival (OS) of 31%. In multivariate analysis, increasing IPSS-R score at the time of HCT was associated with inferior TRM (P = .0056), DFS (P = .018), and OS (P = .0082), but not with GVHD or relapse. The presence of pretransplantation comorbidities was associated with TRM (P = .001), DFS (P = .02), and OS (P = .001). Reduced-intensity conditioning was associated with increased risk of relapse (relative risk, 3.95; 95% confidence interval, 1.78-8.75; P < .001), and although a higher proportion of myeloablative UCBTs were performed in patients with high-risk disease, the effect of conditioning regimen intensity was the same regardless of IPSS-R score. For some individuals who lack a matched sibling or unrelated donor, UCBT can result in long-term DFS; however, the success of UCBT in this population is hampered by a high rate of TRM.
对于接受异基因造血细胞移植的血液系统恶性肿瘤患者，在没有匹配的同胞或无关供者的情况下，脐带血移植（UCBT）已成为一种可接受的替代供者来源。然而，迄今为止，很少有专门发表的系列研究仅描述接受UCBT的成年骨髓增生异常综合征（MDS）患者的结局。2004年至2013年期间，向国际血液和骨髓移植研究中心报告的176例成年MDS患者接受了UCBT。移植时的中位年龄为56岁（范围18 - 73岁）。研究组中，修订国际预后评分系统（IPSS - R）评分为极低危的占10%，低危的占23%，中危的占19%，高危的占19%，极高危的占13%。II - IV级急性移植物抗宿主病（GVHD）的100天发生率为38%，慢性GVHD的3年发生率为28%。3年时复发和移植相关死亡率（TRM）的发生率分别为32%和40%，导致3年无病生存率（DFS）为28%，总生存率（OS）为31%。在多变量分析中，造血细胞移植（HCT）时IPSS - R评分增加与较差的TRM（P = 0.0056）、DFS（P = 0.018）和OS（P = 0.0082）相关，但与GVHD或复发无关。移植前合并症的存在与TRM（P = 0.001）、DFS（P = 0.02）和OS（P = 0.001）相关。减低强度预处理与复发风险增加相关（相对风险，3.95；95%置信区间，1.78 - 8.75；P < 0.001），尽管较高比例的清髓性UCBT是在高危疾病患者中进行的，但预处理方案强度的影响与IPSS - R评分无关。对于一些缺乏匹配同胞或无关供者的个体，UCBT可导致长期DFS；然而，该人群中UCBT的成功受到高TRM发生率的阻碍。
