Haploidentical versus Matched-Sibling Transplant in Adults with Philadelphia-Negative High-Risk Acute Lymphoblastic Leukemia: A Biologically Phase III Randomized Study
Haploidentical versus Matched-Sibling Transplant in Adults with Philadelphia-Negative High-Risk Acute Lymphoblastic Leukemia: A Biologically Phase III Randomized Study
复制标题
DOI：
10.1158/1078-0432.ccr-15-2335
复制
发表时间：
2016-07-15
影响因子：
11.5
通讯作者：
Huang, Xiao-Jun
中科院分区：
文献类型：
--
作者：
Wang, Yu;Liu, Qi-Fa;Huang, Xiao-Jun
目的：尽管配对同胞供者（MSD）造血干细胞移植（HSCT）在急性淋巴细胞白血病（ALL）成人患者首次完全缓解（CR 1）的治疗中具有既定作用，但单倍体相合供者（HID）HSCT作为缓解后治疗对这部分患者的影响尚未确定。在一项疾病特异性、生物学III期随机、多中心研究中比较了HID或MSD的移植结局。在2010年7月和2013年12月之间，210例费城阴性高风险急性淋巴细胞白血病的CR 1患者被分配到接受未经操作的HID（121例）或MSDs HSCT（89例）根据捐助者的可用性意向治疗（ITT）的基础上。因此，210例患者中的186例（88%）最终从MSD（n = 83）或HID（n = 103）移植。根据ITT原则，HID组和MSD组的3年无病生存率（DFS）与CR相比无差异[61%，95%置信区间（CI），52%-70%; vs. 60%，CI，49%-71%; P = 0.91]，两组之间的DFS也无差异（68%，CI，58%-78%; vs. 64%，CI，52%-76%; P = 0.56），累计非复发死亡率为13%（可信区间，7%-19%）和11%（CI，4%-18%; P = 0.84），复发率为18%（CI，10%-26%）和24%（CI，14%-34%; P = 0.30）。对于CR 1的费城阴性高危ALL患者，单倍相合HSCT的结局与MSD-HSCT相似。这种移植可能是一个有效的替代缓解后治疗的高危急性淋巴细胞白血病患者CR 1缺乏相同的供体。(C)2016年AACR。
Purpose: Although matched-sibling donor (MSD) hematopoietic stem-cell transplantation (HSCT) has an established role in the management of adults with acute lymphoblastic leukemia (ALL) in first complete remission (CR1), the effect of haploidentical donor (HID) HSCT as post-remission treatment for this portion of patients is not defined.Experimental Design: Transplantation outcomes from HIDs or MSDs were compared in a disease-specific, biologically phase III randomized, multicenter study. Between July 2010 and December 2013, 210 patients with Philadelphia-negative high-risk ALL in CR1 were assigned to undergo unmanipulated HIDs (121 patients) or MSDs HSCT (89 patients) according to donor availability on an intent-to-treat (ITT) basis.Results: Overall, 24 of the 210 patients had lost transplant eligibility. Therefore, 186 of 210 (88%) patients were finally transplanted from MSD (n = 83) or HID (n = 103). Based on the ITT principle, the 3-year disease-free survival (DFS) did not differ between HID and MSD groups [61%, 95% confidence interval (CI), 52%-70%; vs. 60%, CI, 49%-71%; P = 0.91] from CR, neither did DFS differ between the two groups (68%, CI, 58%-78%; vs. 64%, CI, 52%-76%; P = 0.56) from time of the graft, with cumulative incidence of nonrelapse mortality of 13% (CI, 7%-19%) and 11% (CI, 4%-18%; P = 0.84) and relapse rates of 18% (CI, 10%-26%) and 24% (CI, 14%-34%; P = 0.30), respectively.Conclusions: Haploidentical HSCT achieves outcomes similar to those of MSD-HSCT for Philadelphia-negative high-risk ALL patients in CR1. Such transplantation could be a valid alternative as post-remission treatment for high-risk ALL patients in CR1 lacking an identical donor. (C) 2016 AACR.