Outcomes after Second Hematopoietic Stem Cell Transplantations in Pediatric Patients with Relapsed Hematological Malignancies
Outcomes after Second Hematopoietic Stem Cell Transplantations in Pediatric Patients with Relapsed Hematological Malignancies
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DOI：
10.1016/j.bbmt.2015.02.024
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发表时间：
2015-07-01
影响因子：
4.3
通讯作者：
Krance, Robert
中科院分区：
文献类型：
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作者：
Naik, Swati;Martinez, Caridad;Krance, Robert
造血干细胞移植(HCT)后恶性血液病复发与预后不良有关。第二次HCT是这些高危患者为数不多的治疗选择之一。对于接受第二次HCT的儿童，结果数据尤其有限。因此，我们进行了一项回顾性的单机构研究，并报告了与总存活率(OS)和复发相关的结果和预后变量，这些患者在2000年至2013年期间接受了第二次HCT。43名接受移植的患者中有11名存活下来，中位随访时间为49个月(范围从5个月到127个月)。整个队列的5年内发生OS的概率为24%。早期复发(6个月)的患者，5年OS分别为11%和34%(风险比[HR]，2.24；95%可信区间[CI]，1.21至4.93；P=.013)。第二次HCT时的活动性疾病也与整个队列中显著增加的复发风险相关(亚分布风险比[SHR]，2.36；P=.049)，复发是最常见的死亡原因(32例中23例；72%)。对34例白血病患者进行亚组分析，活动性疾病的存在与OS显著降低(SHR，2.28；95%CI，1.02至5.09；P=.044)和复发率显著增加(SHR，2.46；P=.046)有关。相比之下，潜在的疾病、供体来源、预适应方案或GVHD的发展并没有改变OS或复发率。因此，对于患有复发性血液系统恶性肿瘤的儿童，第二次HCT似乎是一种有用的治疗选择，最有可能使那些复发较晚且移植时疾病负担较低的人受益。(C)2015年美国血液和骨髓移植学会。
Relapse of hematological malignancies after hematopoietic stem cell transplantation (HCT) is associated with poor prognosis. A second HCT represents one of the few therapeutic options for these high-risk patients. For children undergoing second HCT, the outcome data are particularly limited. We, therefore, conducted a retrospective single-institution study and report the outcomes and prognostic variables associated with overall survival (OS) and relapse in 43 pediatric patients who underwent a second HCT between 2000 and 2013. Eleven of the 43 patients who underwent transplantation remain alive and disease-free at a median follow-up of 49 months (range, 5 to 127 months). The 5-year probability of OS for the entire cohort was 24%. Patients who had early relapse (6 months), with 5-year OS at 11% versus 34%, respectively (hazard ratio [HR], 2.24; 95% confidence interval [CI], 1.21 to 4.93; P = .013). Active disease at time of second HCT was also associated with a significantly increased risk of relapse (subdistribution hazard ratio [SHR], 2.36; P=.049) for the entire cohort and relapse was the most frequent cause of death (23 of 32; 72%). On subgroup analysis for the 34 patients with leukemia alone, presence of active disease was associated both with a significant decrease in OS (SHR, 2.28; 95% CI, 1.02 to 5.09; P = .044) and significant increase in the rate of relapse (SHR, 2.46; P = .046). By contrast, underlying disease, donor source, conditioning regimen, or development of GVHD did not modify OS or rate of relapse. Hence, a second HCT appears to be a useful therapeutic option in children with relapsed hematological malignancies that is most likely to benefit those individuals with late onset of relapse and with low disease burden at the time of transplantation. (C) 2015 American Society for Blood and Marrow Transplantation.