Liu Hien Duong, Ahn Kwang Woo, Hu Zhen-Huan, Hamadani Mehdi, Nishihori Taiga, Wirk Baldeep, Beitinjaneh Amer, Rizzieri David, Grunwald Michael R, Sabloff Mitchell, Olsson Richard F, Bajel Ashish, Bredeson Christopher, Daly Andrew, Inamoto Yoshihiro, Majhail Navneet, Saad Ayman, Gupta Vikas, Gerds Aaron, Malone Adriana, Tallman Martin, Reshef Ran, Marks David I, Copelan Edward, Gergis Usama, Savoie Mary Lynn, Ustun Celalettin, Litzow Mark R, Cahn Jean-Yves, Kindwall-Keller Tamila, Akpek Gorgun, Savani Bipin N, Aljurf Mahmoud, Rowe Jacob M, Wiernik Peter H, Hsu Jack W, Cortes Jorge, Kalaycio Matt, Maziarz Richard, Sobecks Ronald, Popat Uday, Alyea Edwin, Saber Wael
Department of Hematology and Medical Oncology, Cleveland Clinic Taussig Cancer Institute, 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 May;23(5):767-775. doi: 10.1016/j.bbmt.2017.01.078. Epub 2017 Jan 20.
Allogeneic hematopoietic cell transplantation (HCT) is potentially curative for patients with chronic myelomonocytic leukemia (CMML); however, few data exist regarding prognostic factors and transplantation outcomes. We performed this retrospective study to identify prognostic factors for post-transplantation outcomes. The CMML-specific prognostic scoring system (CPSS) has been validated in subjects receiving nontransplantation therapy and was included in our study. From 2001 to 2012, 209 adult subjects who received HCT for CMML were reported to the Center for International Blood and Marrow Transplant Research. The median age at transplantation was 57 years (range, 23 to 74). Median follow-up was 51 months (range, 3 to 122). On multivariate analyses, CPSS scores, Karnofsky performance status (KPS), and graft source were significant predictors of survival (P = .004, P = .01, P = .01, respectively). Higher CPSS scores were not associated with disease-free survival, relapse, or transplantation-related mortality. In a restricted analysis of subjects with relapse after HCT, those with intermediate-2/high risk had a nearly 2-fold increased risk of death after relapse compared to those with low/intermediate-1 CPSS scores. Respective 1-year, 3-year, and 5-year survival rates for low/intermediate-1 risk subjects were 61% (95% confidence interval [CI], 52% to 72%), 48% (95% CI, 37% to 59%), and 44% (95% CI, 33% to 55%), and for intermediate-2/high risk subjects were 38% (95% CI, 28% to 49%), 32% (95% CI, 21% to 42%), and 19% (95% CI, 8% to 29%). We conclude that higher CPSS score at time of transplantation, lower KPS, and a bone marrow graft are associated with inferior survival after HCT. Further investigation of CMML disease-related biology may provide insights into other risk factors predictive of post-transplantation outcomes.
异基因造血细胞移植(HCT)对慢性粒单核细胞白血病(CMML)患者具有潜在的治愈作用;然而,关于预后因素和移植结果的数据很少。我们进行了这项回顾性研究,以确定移植后结果的预后因素。CMML特异性预后评分系统(CPSS)已在接受非移植治疗的受试者中得到验证,并纳入我们的研究。2001年至2012年,209名接受CMML-HCT的成年受试者被报告至国际血液和骨髓移植研究中心。移植时的中位年龄为57岁(范围23至74岁)。中位随访时间为51个月(范围3至122个月)。多因素分析显示,CPSS评分、卡诺夫斯基功能状态(KPS)和移植物来源是生存的显著预测因素(P分别为0.004、0.01、0.01)。较高的CPSS评分与无病生存、复发或移植相关死亡率无关。在对HCT后复发的受试者进行的有限分析中,与低/中-1 CPSS评分的受试者相比,中-2/高风险的受试者复发后死亡风险增加近2倍。低/中-1风险受试者的1年、3年和5年生存率分别为61%(95%置信区间[CI],52%至72%)、48%(95%CI,37%至59%)和44%(95%CI,33%至55%),中-2/高风险受试者的生存率分别为38%(95%CI,28%至49%)、32%(95%CI,21%至42%)和19%(95%CI,8%至29%)。我们得出结论,移植时较高的CPSS评分、较低的KPS和骨髓移植物与HCT后较差的生存率相关。对CMML疾病相关生物学的进一步研究可能会为预测移植后结果的其他风险因素提供见解。