Reduced-intensity conditioning with combined haploidentical and cord blood transplantation results in rapid engraftment, low GVHD, and durable remissions
Reduced-intensity conditioning with combined haploidentical and cord blood transplantation results in rapid engraftment, low GVHD, and durable remissions
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DOI：
10.1182/blood-2011-08-372508
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发表时间：
2011-12-08
期刊：
影响因子：
20.3
通讯作者：
van Besien, Koen
中科院分区：
文献类型：
--
作者：
Liu, Hongtao;Rich, Elizabeth S.;van Besien, Koen
我们对45例患者进行了一项前瞻性研究，研究对象为降低强度预处理（RIC）和移植无关脐带血（UCB）和来自一个半相合家庭成员的CD 34（+）干细胞。中位年龄为50岁;体重为80 kg。58%的人患有活动性疾病。在第11天（四分位距[IQR]，9-15）发生了神经元植入，在第19天（IQR，15-33）发生了血小板植入。在大多数患者中，早期单倍体相合植入被100天的UCB持久植入所取代，并定期持续少量宿主和/或单倍体造血。第100天的单倍嵌合体百分比与单倍CD 34剂量相关（P = .003）。急性GVHD（aGVHD）的累积发生率为25%，慢性GVHD（cGVHD）为5%。1年生存率为55%，无进展生存率（PFS）为42%，非复发死亡率（NRM）为28%，复发率为30%。RIC和单脐带移植导致中性粒细胞和血小板的快速植入，aGVHD和cGVHD的发生率低，迟发性机会性感染的频率低，输血需求减少，住院时间缩短，以及有希望的长期结局。UCB细胞剂量对造血恢复时间无影响。因此，UCB选择可以优先匹配，并且可以为大多数患者快速确定更好匹配的供体。本研究在http://clinicaltrials.gov注册为NCI临床试验编号NCT 00943800。（血。2011;118（24）：6438-6445）
We conducted a 45 patient prospective study of reduced-intensity conditioning (RIC) and transplantation of unrelated umbilical cord blood (UCB) and CD34(+) stem cells from a haploidentical family member. Median age was 50 years; weight was 80 kg. Fifty-eight percent had active disease. Neutrophil engraftment occurred at 11 days (interquartile range [IQR], 9-15) and platelet engraftment at 19 days (IQR, 15-33). In the majority of patients, early haploidentical engraftment was replaced by durable engraftment of UCB by 100 days, with regular persistence of minor host and/or haplo-hematopoiesis. Percentage of haplochimerism at day 100 correlated with the haplo-CD34 dose (P = .003). Cumulative incidence of acute GVHD (aGVHD) was 25% and chronic GVHD (cGVHD) was 5%. Actuarial survival at 1 year was 55%, progression-free survival (PFS) was 42%, nonrelapse mortality (NRM) was 28%, and relapse was 30%. RIC and haplo-cord transplantation results in fast engraftment of neutrophils and platelets, low incidences of aGVHD and cGVHD, low frequency of delayed opportunistic infections, reduced transfusion requirements, shortened length of hospital stay, and promising long-term outcomes. UCB cell dose had no impact on time to hematopoietic recovery. Therefore, UCB selection can prioritize matching, and better matched donors can be identified rapidly for most patients. This study is registered at http://clinicaltrials.gov as NCI clinical trial no. NCT00943800. (Blood. 2011;118(24):6438-6445)