Tang Xiangfeng, Chen Jing, Fang Jianpei, Sun Xin, Qin Mao Quan, Li Junhui, Zhu Yiping, Luan Zuo
Department of Pediatrics, Navy General Hospital, Beijing, China.
Pediatr Transplant. 2015 Jun;19(4):413-21. doi: 10.1111/petr.12475. Epub 2015 Apr 22.
In a multicenter study, we have conducted a retrospective study on 73 pediatric AML patients who were primary refractory or in greater than CR1 and investigated MSD (or MMSD) (n = 20), URD (n = 23), and UCB (n = 30) HCT between January 1998 and October 2009. The median day to neutrophil engraftment was similar in all groups. The median day to platelet engraftment was longer in the UCB group. The number of HLA mismatch was higher in the UCB group (p = 0.034); however, the cumulative incidence of grade III-IV aGVHD was not different among all groups (p = 0.125); furthermore, cGVHD was lower in the UCB group (p = 0.078). The risk of relapse did not differ among all groups (RR = 1.28, p = 0.125), but the patients of MSD (or MMSD) grafts had a trend of higher risk recurrence. Sixty-two patients survived with a median follow-up of 58.2 months. Five-yr LFS was 73.1%, 59.8%, and 59.6% for URD, UCB, and MSD (or MMDS), respectively (p = 0.426). Five-yr LFS in CR1 was 68.9%, with a significantly better result compared to 41.7% in CR2 (p = 0.025). Our comparisons suggest that pediatric AML patients receiving UCB had a higher early TRM, a lower cGVHD rate, and a similar long-term survival. The outcome of URD and UCB is comparable to that of a suitable sibling for pediatric AML.
在一项多中心研究中,我们对73例原发性难治性或处于大于CR1阶段的儿童急性髓系白血病(AML)患者进行了回顾性研究,并调查了1998年1月至2009年10月期间进行单倍体相合造血干细胞移植(MSD,或单倍体相合无关供者造血干细胞移植MMSD)(n = 20)、非血缘供者造血干细胞移植(URD,n = 23)和脐血造血干细胞移植(UCB,n = 30)的情况。所有组中性粒细胞植入的中位天数相似。脐血组血小板植入的中位天数更长。脐血组人类白细胞抗原(HLA)错配数更高(p = 0.034);然而,所有组中III - IV级急性移植物抗宿主病(aGVHD)的累积发生率并无差异(p = 0.125);此外,脐血组慢性移植物抗宿主病(cGVHD)发生率更低(p = 0.078)。所有组之间复发风险无差异(相对风险RR = 1.28,p = 0.125),但单倍体相合造血干细胞移植(或单倍体相合无关供者造血干细胞移植)移植物的患者有更高复发风险的趋势。62例患者存活,中位随访时间为58.2个月。非血缘供者造血干细胞移植、脐血造血干细胞移植和单倍体相合造血干细胞移植(或单倍体相合无关供者造血干细胞移植)的5年无病生存率(LFS)分别为73.1%、59.8%和%59.6(p = 0.426)。CR1阶段的5年无病生存率为68.9%,与CR2阶段的41.7%相比结果显著更好(p = 0.025)。我们的比较表明,接受脐血造血干细胞移植的儿童AML患者早期移植相关死亡率更高,慢性移植物抗宿主病发生率更低,长期生存率相似。非血缘供者造血干细胞移植和脐血造血干细胞移植的结果与适合的同胞供者移植治疗儿童AML的结果相当。