Koo Jane, Silverman Stacy, Nuechterlein Brandon, Keating Amy K, Verneris Michael R, Foreman Nicholas K, Mulcahy Levy Jean M
Department of Pediatric Hematology/Oncology/Bone Marrow Transplant, University of Colorado, Children's Hospital Colorado, Aurora, CO, USA.
The Morgan Adams Foundation Pediatric Brain Tumor Research Program, Children's Hospital Colorado, Aurora, CO, USA.
Bone Marrow Transplant. 2019 Oct;54(10):1605-1613. doi: 10.1038/s41409-019-0479-3. Epub 2019 Feb 19.
High-dose chemotherapy with autologous hematopoietic stem cell transplantation (autoHSCT) is a well-established treatment for pediatric central nervous system (CNS) tumors. Given the risks of toxicity and infection, pediatric autoHSCT has been historically performed on hospitalized children. As our practice evolved, some patients were transplanted as outpatients. We performed a retrospective cohort analysis of 37 patients who received 90 transplant procedures (49 outpatient and 41 inpatient) at Children's Hospital Colorado. The most common primary diagnosis was medulloblastoma (51.4%). Of the patients transplanted as outpatients, 69.4% were admitted for fever and neutropenia and had a median time to hospitalization of day +6, with fever and neutropenia being the most common reasons for admission. The median time to neutrophil engraftment was the same in both cohorts, 11 days. Median time to platelet engraftment was 13 days (8-82 days) vs 16 days (8-106 days) (p = 0.0008). At day +100, the transplant-related mortality (TRM) was 0% for both the cohorts. At a median follow-up of 1.7 years, overall survival (OS) for all patients was 66.1% and TRM was 0% for both the cohorts. Outpatient autoHSCT for properly selected children with CNS tumors is safe and effective.
大剂量化疗联合自体造血干细胞移植（autoHSCT）是治疗小儿中枢神经系统（CNS）肿瘤的一种成熟疗法。鉴于毒性和感染风险，小儿autoHSCT在历史上一直是在住院儿童中进行的。随着我们实践的发展，一些患者作为门诊患者接受了移植。我们对科罗拉多儿童医院37例接受90次移植手术（49例门诊和41例住院）的患者进行了回顾性队列分析。最常见的原发性诊断是髓母细胞瘤（51.4%）。在作为门诊患者接受移植的患者中，69.4%因发热和中性粒细胞减少而入院，住院中位时间为+6天，发热和中性粒细胞减少是最常见的入院原因。两个队列中中性粒细胞植入的中位时间相同，均为11天。血小板植入的中位时间为13天（8 - 82天），而住院患者为16天（8 - 106天）（p = 0.0008）。在+100天时，两个队列的移植相关死亡率（TRM）均为0%。在中位随访1.7年时，所有患者的总生存率（OS）为66.1%，两个队列的TRM均为0%。对于经过适当选择的患有CNS肿瘤的儿童，门诊autoHSCT是安全有效的。
