Allogeneic hematopoietic cell transplantation (HCT) has the capacity to cure numerous malignant and nonmalignant disorders. A dreaded complication is graft failure (GF), as it puts patients at high risk of infection and disease relapse. There are few contemporary data on the risks, outcomes, and economic burden of GF in pediatric patients. In this study, we address this gap by focusing on 14 years of transplant at our single center, for which data are compared in 2 time periods: 2005 to 2010 (n = 146) and 2011 to 2018 (n = 144). In the 290 patients studied, the median age was 9.33 years, and 50.3% had malignant versus nonmalignant disease. Cell source included bone marrow (51%), cord blood (19.7%), unmanipulated peripheral blood stem cells (PBSCs; 12.1%), and CD34-selected PBSCs (17.2%). Twenty-one percent of patients had reduced-intensity conditioning (RIC), and 54.8% of transplants were fully HLA matched. Most patients received serotherapy with rabbit anti-thymocyte globulin (39.3%) or alemtuzumab (42.8%). The incidence of neutropenic and non-neutropenic GF (NGF and NNGF) was 6.6% and 3.8%, respectively. Multivariate analysis demonstrated alemtuzumab (odds ratio [OR], 6.256, P < .001) was the main variable associated with a higher rate of GF in both time periods, whereas RIC (OR, 11.8, P < .001) and cell source (CD34-selected PBSCs; OR, 4.22, P = .04) showed period-specific effects. Specifically, from time periods 1 to 2, cord blood transplants were discontinued at our center, with a concomitant increase in CD34-selected grafts and a shift from more episodes of NGF to NNGF. Overall survival was 69% in the entire HCT cohort and 50% among patients with GF. Survival among GF patients improved from time periods 1 to 2 (20% versus 80%, P = .001), potentially due to a higher incidence of NNGF and increased ability to perform stem cell boosts from the same donor once cord blood transplants were phased out. Inpatient length of stay was consistently higher for patients with GF. Similar trends were seen for inpatient costs, although improvements were seen in our entire HCT population over time. In summary, GF remains a significant challenge in pediatric HCT and poses an economic burden on the health care system.

同种异体造血细胞移植（HCT）具有治愈众多恶性和非恶性疾病的能力。可怕的并发症是移植失败（GF），因为它使患者处于感染和疾病复发的高风险中。关于儿科患者的GF的风险，结果和经济负担的当代数据很少。在本研究中，我们通过集中研究单个中心的14年移植来解决这一差距，并比较了两个时间段的数据：2005年至2010年（n = 146）和2011年至2018年（n = 144）。在研究的290名患者中，中位年龄为9.33岁，其中恶性与非恶性疾病的比例为50.3％。细胞来源包括骨髓（51％），脐带血（19.7％），未处理的外周血干细胞（PBSC; 12.1％）和CD34选择的PBSC（17.2％）。21％的患者进行了降低强度调节（RIC），并且54.8％的移植物完全符合HLA。大多数患者接受兔抗胸腺细胞球蛋白（39.3％）或alemtuzumab（42.8％）的血清疗法。中性粒细胞减少和非中性粒细胞减少（NGF和NNGF）的发生率分别为6.6％和3.8％。多因素分析显示alemtuzumab（赔率[OR]为6.256，P <.001）是与两个时期中较高的GF率相关的主要变量，而RIC（OR，11.8，P <.001）和细胞来源（CD34选择的PBSCs; OR，4.22，P  = .04） ）显示了特定时期的效果。具体来说，从第1到第2阶段，我们中心停止了脐带血移植，伴随CD34选择的移植物增加，并且从更多的NGF发作转变为NNGF。整个HCT队列的总生存率为69％，GF患者的总生存率为50％。GF患者的生存时间从第1到第2阶段有所改善（20％对80％，P = 0.001），这可能是因为一旦淘汰了脐血移植，NNGF的发病率就会增加，并且来自同一供体的干细胞升压能力会增强。GF患者的住院时间持续较高。尽管随着时间的推移，我们整个HCT人群的住院费用都有所改善，但住院费用也呈现出类似的趋势。总而言之，GF在小儿HCT中仍然是一项重大挑战，并给医疗保健系统带来了经济负担。