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造血细胞移植合并症指数评分与儿童二次异基因造血细胞移植后的治疗相关死亡率和总生存率相关。

Hematopoietic Cell Transplantation-Comorbidity Index Score Is Correlated with Treatment-Related Mortality and Overall Survival following Second Allogeneic Hematopoietic Cell Transplantation in Children.

作者信息

Forlanini Federica, Zinter Matt S, Dvorak Christopher C, Bailey-Olson Mara, Winestone Lena E, Shimano Kristin A, Higham Christine S, Melton Alexis, Chu Julia, Kharbanda Sandhya

机构信息

Division of Pediatric Allergy, Immunology & Bone Marrow Transplantation, UCSF Benioff Children's Hospital, University of California, San Francisco, California; Department of Pediatrics, V. Buzzi Hospital, Università degli Studi di Milano, Milan, Italy.

Division of Pediatric Critical Care Medicine, UCSF Benioff Children's Hospital, University of California, San Francisco, California.

出版信息

Transplant Cell Ther. 2022 Mar;28(3):155.e1-155.e8. doi: 10.1016/j.jtct.2021.11.015. Epub 2021 Nov 28.

DOI:10.1016/j.jtct.2021.11.015
PMID:34848362
Abstract

Allogeneic hematopoietic cell transplantation (HCT) can lead to considerable complications and treatment-related mortality (TRM); therefore, a detailed assessment of risks is essential. The Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) can predict both TRM and overall survival (OS). Although the HCT-CI has been validated as a useful tool for first HCT, its potential utility for second HCT has not yet been investigated. Here we aimed to evaluate the utility of the HCT-CI score in assessing the risk of TRM and OS in the setting of a second allogeneic HCT. This was a retrospective analysis of all pediatric patients (age <21 years) who underwent a second allogeneic HCT at UCSF Benioff Children's Hospital San Francisco between 2008 and 2019. According to their HCT-CI, patients were classified as "low risk" with an HCT-CI of 0 or "intermediate-high risk" with an HCT-CI ≥1. A total of 59 patients were included in the study. Our primary endpoint was TRM, observed at 100 days, 180 days, 1 year, and last follow-up following HCT, and our secondary endpoint was OS at 1 year and at 5 years or last follow-up. We also evaluated outcomes of patients admitted to the pediatric intensive care unit based on the HCT-CI score. Seventy-six percent of patients had an HCT-CI of 0. The most frequent comorbidities were pulmonary, seen in 7 patients (12%; 95% CI, 5% to 23%), including 5 (71%) with moderate and 2 (29%) with severe comorbidities. The OS and the cumulative incidence of TRM at 1 year for the entire cohort were 81% (95% CI, 69% to 90%) and 12% (95% CI, 5% to 22%), respectively. The cumulative incidence of TRM and OS at 1 year showed a significant correlation with HCT-CI score; TRM was 4% (95% CI, 1% to 13%) for an HCT-CI of 0 versus 36% (95% CI, 13% to 60%) for an HCT-CI ≥1 (P < .001), and OS was 89% (95% CI, 75% to 99%) for an HCT-CI of 0 versus 57% (95% CI, 28% to 78%) for an HCT-CI ≥1 (P = .003). After adjusting for covariates, HCT-CI continued to be associated with both TRM (P = .004) and OS (P = .003). In addition, comparing patients with malignancies and nonmalignant disorders, disease-free-survival at last follow-up was higher in the nonmalignant disorder group and also was influenced by the HCT-CI score in each group (P = .0035). There also was a significant difference in outcomes of patients admitted to the pediatric intensive care unit; 15 patients (68%) with an HCT-CI of 0 were alive at last follow-up, compared with only two (22%) with an HCT-CI ≥1 (P = .016). HCT-CI has an impact on TRM and OS and may serve as a predictor of outcomes of second allogeneic transplantation. Although this study was conducted in a relatively small sample, it is the first to investigate the utility of the HCT-CI score in predicting outcomes after a second allogeneic HCT in pediatric recipients. © 2021 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc.

摘要

异基因造血细胞移植(HCT)可导致相当多的并发症和治疗相关死亡率(TRM);因此,详细评估风险至关重要。造血细胞移植合并症指数(HCT-CI)可预测TRM和总生存期(OS)。尽管HCT-CI已被验证为首次HCT的有用工具,但其在第二次HCT中的潜在效用尚未得到研究。在此,我们旨在评估HCT-CI评分在评估第二次异基因HCT情况下TRM和OS风险中的效用。这是一项对2008年至2019年期间在旧金山加州大学旧金山分校贝尼奥夫儿童医院接受第二次异基因HCT的所有儿科患者(年龄<21岁)的回顾性分析。根据HCT-CI,患者被分类为HCT-CI为0的“低风险”或HCT-CI≥1的“中高风险”。共有59名患者纳入研究。我们的主要终点是HCT后100天、180天、1年和末次随访时观察到的TRM,次要终点是1年和5年或末次随访时的OS。我们还根据HCT-CI评分评估了入住儿科重症监护病房患者的结局。76%的患者HCT-CI为0。最常见的合并症是肺部疾病,7例患者(12%;95%CI,5%至23%)出现,其中5例(71%)为中度合并症,2例(29%)为重度合并症。整个队列1年时的OS和TRM累积发生率分别为81%(95%CI,69%至90%)和12%(95%CI,5%至22%)。1年时TRM和OS的累积发生率与HCT-CI评分显著相关;HCT-CI为0时TRM为4%(95%CI,1%至13%),而HCT-CI≥1时为36%(95%CI,13%至60%)(P<.001),HCT-CI为0时OS为89%(95%CI,75%至99%),而HCT-CI≥1时为57%(95%CI,28%至78%)(P=.003)。在调整协变量后,HCT-CI继续与TRM(P=.004)和OS(P=.003)相关。此外,比较恶性肿瘤和非恶性疾病患者,非恶性疾病组末次随访时的无病生存期更高,且每组均受HCT-CI评分影响(P=.0035)。入住儿科重症监护病房患者的结局也存在显著差异;HCT-CI为0的15例患者(68%)在末次随访时存活,而HCT-CI≥1的患者仅2例(22%)存活(P=.016)。HCT-CI对TRM和OS有影响,可作为第二次异基因移植结局的预测指标。尽管本研究样本量相对较小,但它是首次研究HCT-CI评分在预测儿科受者第二次异基因HCT后结局中的效用。©2021美国移植和细胞治疗学会。由爱思唯尔公司出版。

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