Shin Seung-Hwan, Kim Jung-Ho, Jeon Young-Woo, Yoon Jae-Ho, Yahng Seung-Ah, Lee Sung-Eun, Choi Yun-Suk, Kim Dae-Young, Lee Jung-Hee, Lee Seok, Kim Hee-Je, Min Chang-Ki, Lee Jong-Wook, Lee Kyoo-Hyung, Min Woo-Sung, Kim Yoo-Jin, Lee Je-Hwan
Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
Department of Hematology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea.
Biol Blood Marrow Transplant. 2015 Feb;21(2):342-9. doi: 10.1016/j.bbmt.2014.10.031. Epub 2014 Nov 20.
Even with the recent optimization of haploidentical stem cell transplantation (SCT), its role for patients with myelodysplastic syndrome (MDS) or acute myeloid leukemia evolving from MDS (sAML) should be validated. We analyzed the outcomes of consecutive 60 patients with MDS or sAML who received T cell-replete haploidentical SCT after reduced-intensity conditioning with fludarabine, busulfan, and rabbit antithymocyte globuline ± 800 cGy total body irradiation. Patients achieved a rapid neutrophil engraftment after a median of 12 days (range, 8 to 23) and an early immune reconstitution without high incidences of acute graft-versus-host disease (GVHD) II to IV and chronic GVHD (36.7% and 48.3%, respectively). After a median follow-up of 4 years, incidence of relapse and nonrelapse mortality and rate of overall survival and disease-free survival was 34.8%, 23.3%, 46.8%, and 41.9%, respectively. In multivariate analysis, the disease status at peak was a significant predictor for relapse (lower-risk MDS versus higher-risk MDS or sAML; hazard ratio [HR], 5.69; 95% confidence interval [CI], 1.45 to 22.29; P = .013) and disease-free survival (HR, 4.44; 95% CI, 1.14 to 17.34; P = .032). Chronic GVHD was an additional significant predictor for relapse (no versus yes; HR, 2.87; 95% CI, 1.03 to 7.51; P = .043). Our T cell-replete haploidentical SCT may be a feasible option for patients with MDS and sAML without conventional donors.
即使近期单倍体相合干细胞移植（SCT）已得到优化，但其在骨髓增生异常综合征（MDS）患者或由MDS演变而来的急性髓系白血病（sAML）患者中的作用仍需验证。我们分析了连续60例MDS或sAML患者的治疗结果，这些患者在接受氟达拉滨、白消安和兔抗胸腺细胞球蛋白进行减低强度预处理并联合或不联合800 cGy全身照射后，接受了T细胞充足的单倍体相合SCT。患者在中位12天（范围8至23天）后实现快速中性粒细胞植入，且早期免疫重建，急性移植物抗宿主病（GVHD）II至IV级和慢性GVHD的发生率均不高（分别为36.7%和48.3%）。中位随访4年后，复发率、非复发死亡率、总生存率和无病生存率分别为34.8%、23.3%、46.8%和41.9%。多因素分析显示，疾病高峰期的疾病状态是复发（低危MDS与高危MDS或sAML相比；风险比[HR]，5.69；95%置信区间[CI]，1.45至22.29；P = 0.013）和无病生存（HR，4.44；95%CI，1.14至17.34；P = 0.032）的重要预测因素。慢性GVHD是复发的另一个重要预测因素（无与有相比；HR，2.87；95%CI，1.03至7.51；P = 0.043）。我们的T细胞充足的单倍体相合SCT对于没有传统供体的MDS和sAML患者可能是一种可行的选择。
