Shaffer Brian C, Ahn Kwang Woo, Hu Zhen-Huan, Nishihori Taiga, Malone Adriana K, Valcárcel David, Grunwald Michael R, Bacher Ulrike, Hamilton Betty, Kharfan-Dabaja Mohamed A, Saad Ayman, Cutler Corey, Warlick Erica, Reshef Ran, Wirk Baldeep Mona, Sabloff Mitchell, Fasan Omotayo, Gerds Aaron, Marks David, Olsson Richard, Wood William Allen, Costa Luciano J, Miller Alan M, Cortes Jorge, Daly Andrew, Kindwall-Keller Tamila L, Kamble Rammurti, Rizzieri David A, Cahn Jean-Yves, Gale Robert Peter, William Basem, Litzow Mark, Wiernik Peter H, Liesveld Jane, Savani Bipin N, Vij Ravi, Ustun Celalettin, Copelan Edward, Popat Uday, Kalaycio Matt, Maziarz Richard, Alyea Edwin, Sobecks Ron, Pavletic Steven, Tallman Martin, Saber Wael
Brian C. Shaffer and Martin Tallman, Memorial Sloan Kettering Cancer Center; Adriana K. Malone, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai; Ran Reshef, Columbia University Medical Center, New York; Mark Litzow, Mayo Clinic Rochester; Jane Liesveld, University of Rochester Medical Center, Rochester; Peter H. Wiernik, Our Lady of Mercy Medical Center, Bronx, NY; Kwang Woo Ahn, Zhen-Huan Hu, and Wael Saber, Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WI; Taiga Nishihori and Mohamed A. Kharfan-Dabaja, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL; David Valcárcel, Hospital Vall d'Hebron, Barcelona, Spain; Michael R. Grunwald, Omotayo Fasan, and Edward Copelan, Levine Cancer Institute, Carolinas HealthCare System, Charlotte; William Allen Wood, University of North Carolina at Chapel Hill, Chapel Hill; David A. Rizzieri, Duke University Medical Center, Durham, NC; Ulrike Bacher, University of Medicine Göttingen, Göttingen, Germany; Betty Hamilton and Aaron Gerds, Cleveland Clinic Taussig Cancer Institute; Matt Kalaycio and Ron Sobecks, Cleveland Clinic Foundation, Cleveland; Basem William, Ohio State University Medical Center, Columbus, OH; Ayman Saad and Luciano J. Costa, University of Alabama at Birmingham, Birmingham, AL; Corey Cutler and Edwin Alyea, Dana-Farber Cancer Institute, Boston, MA; Erica Warlick and Celalettin Ustun, University of Minnesota Medical Center, Minneapolis, MN; Baldeep Mona Wirk, Seattle Cancer Care Alliance, Seattle, WA; Mitchell Sabloff, University of Ottawa and Ottawa Hospital Research Institute, Ottawa, Ontario; Andrew Daly, Tom Baker Cancer Center, Calgary, Alberta, Canada; David Marks, University Hospitals Bristol National Health Service Trust, Bristol; Robert Peter Gale, Imperial College London, London, United Kingdom; Richard Olsson, Karolinska Institutet, Stockholm, Sweden; Alan M. Miller, Baylor University Medical Center; Rammurti Kamble
J Clin Oncol. 2016 Jun 1;34(16):1864-71. doi: 10.1200/JCO.2015.65.0515. Epub 2016 Apr 4.
To develop a system prognostic of outcome in those undergoing allogeneic hematopoietic cell transplantation (allo HCT) for myelodysplastic syndrome (MDS).
We examined 2,133 patients with MDS undergoing HLA-matched (n = 1,728) or -mismatched (n = 405) allo HCT from 2000 to 2012. We used a Cox multivariable model to identify factors prognostic of mortality in a training subset (n = 1,151) of the HLA-matched cohort. A weighted score using these factors was assigned to the remaining patients undergoing HLA-matched allo HCT (validation cohort; n = 577) as well as to patients undergoing HLA-mismatched allo HCT.
Blood blasts greater than 3% (hazard ratio [HR], 1.41; 95% CI, 1.08 to 1.85), platelets 50 × 10(9)/L or less at transplantation (HR, 1.37; 95% CI, 1.18 to 1.61), Karnofsky performance status less than 90% (HR, 1.25; 95% CI, 1.06 to 1.28), comprehensive cytogenetic risk score of poor or very poor (HR, 1.43; 95% CI, 1.14 to 1.80), and age 30 to 49 years (HR, 1.60; 95% CI, 1.09 to 2.35) were associated with increased hazard of death and assigned 1 point in the scoring system. Monosomal karyotype (HR, 2.01; 95% CI, 1.65 to 2.45) and age 50 years or older (HR, 1.93; 95% CI, 1.36 to 2.83) were assigned 2 points. The 3-year overall survival after transplantation in patients with low (0 to 1 points), intermediate (2 to 3), high (4 to 5) and very high (≥ 6) scores was 71% (95% CI, 58% to 85%), 49% (95% CI, 42% to 56%), 41% (95% CI, 31% to 51%), and 25% (95% CI, 4% to 46%), respectively (P < .001). Increasing score was predictive of increased relapse (P < .001) and treatment-related mortality (P < .001) in the HLA-matched set and relapse (P < .001) in the HLA-mismatched cohort.
The proposed system is prognostic of outcome in patients undergoing HLA-matched and -mismatched allo HCT for MDS.
开发一种用于预测接受异基因造血细胞移植（allo HCT）治疗骨髓增生异常综合征（MDS）患者预后的系统。
我们研究了2000年至2012年间2133例接受HLA配型相合（n = 1728）或不相合（n = 405）allo HCT的MDS患者。我们使用Cox多变量模型在HLA配型相合队列的一个训练亚组（n = 1151）中确定死亡预后因素。使用这些因素的加权评分被分配给其余接受HLA配型相合allo HCT的患者（验证队列；n = 577）以及接受HLA配型不相合allo HCT的患者。
外周血原始细胞大于3%（风险比[HR]，1.41；95%可信区间[CI]，1.08至1.85）、移植时血小板≤50×10⁹/L（HR，1.37；95%CI，1.18至1.61）、卡氏功能状态评分低于90%（HR，1.25；95%CI，1.06至1.28）、综合细胞遗传学风险评分为不良或极不良（HR，1.43；95%CI，1.14至1.80）以及年龄30至49岁（HR，1.60；95%CI，1.09至2.35）与死亡风险增加相关，在评分系统中各得1分。单倍体核型（HR，2.01；95%CI，1.65至2.45）和年龄≥50岁（HR，1.93；95%CI，1.36至2.83）得2分。低（0至1分）、中（2至3分）、高（4至5分）和极高（≥6分）评分患者移植后3年总生存率分别为71%（95%CI，58%至85%）、49%（95%CI，42%至56%）、41%（95%CI，31%至51%）和25%（95%CI，4%至46%）（P <.001）。评分增加预示着HLA配型相合组中复发风险增加（P <.001）和治疗相关死亡率增加（P <.001），以及HLA配型不相合队列中复发风险增加（P <.001）。
所提出的系统可预测接受HLA配型相合和不相合allo HCT治疗MDS患者的预后。
