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资源有限国家中造血干细胞移植后儿童急性白血病幸存者长期随访的挑战:巴西单中心报告。

The challenge of long-term follow-up of survivors of childhood acute leukemia after hematopoietic stem cell transplantation in resource-limited countries: A single-center report from Brazil.

机构信息

Bone Marrow Transplantation Unit, Federal University of Paraná, Curitiba, Brazil.

Pediatric Transplant and Cellular Therapy, Duke University, Durham, NC, USA.

出版信息

Pediatr Transplant. 2020 Jun;24(4):e13691. doi: 10.1111/petr.13691. Epub 2020 Apr 4.

DOI:10.1111/petr.13691
PMID:32246550
Abstract

With the number of long-term HSCT survivors steadily increasing, attention needs to be focused on the late complications and quality of life. We therefore analyzed the outcome of 101 pediatric patients (<18 years old at the time of HSCT) transplanted for acute leukemia between 1981 and 2015 at Complexo Hospital de Clínicas, Federal University of Paraná, Brazil, and who survived at least two years after HSCT. The median follow-up was 5.9 years (2.0-29.0); median age at follow-up was 17.5 years (2.98-39.0). The 5-year cumulative incidence of relapse was 27.5% (95% CI 18.6%-36.4%). Two-year cumulative incidence of chronic GVHD was 21.8% (95% CI 13.7%-29.8%). Of the 101 patients, 72 patients (71.3%) presented with late effects. Those surviving longer after HSCT experienced more complications. Patients who received TBI-based regimen developed more late effects (P = .013) and more endocrinological complications (P = .024). Endocrinological complications were the most common late sequelae found in this study. For childhood survivors, quality of life was not influenced by age (at HSCT or at last visit), time from HSCT, gender, donor, or GVHD. For survivors that no longer were children, only age at last visit impacted financial domain measures, irrespective of gender, donor, or GVHD. The current study confirms the high burden late complications after pediatric HSCT have on the survivors and underlines the importance of extended follow-up.

摘要

随着长期 HSCT 幸存者人数的稳步增加,需要关注晚期并发症和生活质量。因此,我们分析了巴西联邦大学帕拉那联邦大学综合医院 1981 年至 2015 年间为急性白血病接受移植的 101 例儿科患者(HSCT 时年龄<18 岁)的结果,这些患者在 HSCT 后至少存活 2 年。中位随访时间为 5.9 年(2.0-29.0);随访时的中位年龄为 17.5 岁(2.98-39.0)。5 年累积复发率为 27.5%(95%CI 18.6%-36.4%)。2 年累积慢性 GVHD 发生率为 21.8%(95%CI 13.7%-29.8%)。在 101 例患者中,72 例(71.3%)出现晚期效应。HSCT 后存活时间较长的患者发生更多并发症。接受 TBI 为基础方案的患者发生更多晚期效应(P=0.013)和更多内分泌并发症(P=0.024)。内分泌并发症是本研究中发现的最常见的晚期后遗症。对于儿童幸存者,生活质量不受 HSCT 时年龄(年龄或上次就诊时年龄)、HSCT 后时间、性别、供体或 GVHD 的影响。对于不再是儿童的幸存者,只有上次就诊时的年龄会影响财务领域的措施,而与性别、供体或 GVHD 无关。本研究证实了儿科 HSCT 后晚期并发症对幸存者的负担很重,并强调了延长随访的重要性。

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