Childhood Cancer Center, Skane University Hospital, Department of Clinical Sciences, Lund University, Lund, Sweden.
Institute for Cancer Outcomes and Survivorship, University of Alabama at Birmingham School of Medicine, Birmingham, Alabama, USA.
Cancer. 2023 Feb 15;129(4):624-633. doi: 10.1002/cncr.34575. Epub 2022 Dec 9.
A comprehensive assessment of morbidity after allogeneic bone marrow transplantation (BMT) performed in childhood remains understudied.
Seven hundred eighty-nine allogeneic BMT recipients who had survived ≥2 years after BMT performed between 1974 and 2014 at age <22 years and 690 siblings completed a 255-item survey self-reporting sociodemographics and chronic health conditions. A severity score (grade 3 [severe], 4 [life-threatening], or 5 [fatal]) was assigned to the conditions using Common Terminology Criteria for Adverse Events, version 5.0. For the BMT cohort, the cumulative incidence of chronic health conditions was calculated as a function of time from BMT. Proportional subdistribution hazards models were used to determine predictors of grade 3-5 conditions. Logistic regression was used to estimate the risk of grade 3-4 conditions in BMT recipients who were alive at the time of this study compared with siblings.
The median age at transplantation was 11.3 years (range, 0.4-22.0 years), and the median length of follow-up was 11.7 years (range, 2.0-45.3 years). The most prevalent primary diagnoses were acute lymphoblastic leukemia (30.7%), and acute myeloid leukemia/myelodysplastic syndrome (26.9%). At age 35 years, the cumulative incidence of a grade 3-4 condition was 53.8% (95% CI, 46.7%-60.3%). The adjusted odds ratio of a grade 3-4 condition was 15.1 in survivors (95% CI, 9.5-24.0) compared with siblings. The risk of a grade 3-5 condition increased with age at BMT (hazard ratio [HR], 1.03; 95% CI, 1.01-1.05) and was higher among females (HR, 1.27; 95% CI, 1.02-1.59), patients who received total body irradiation (HR, 1.71; 95% CI, 1.27-2.31), and those reporting chronic graft-versus-host disease (HR, 1.38; 95% CI, 1.09-1.74).
Two-year survivors of allogeneic BMT in childhood have an increased risk of grade 3-4 chronic health conditions compared with siblings, suggesting the need for long-term follow-up.
对于儿童异基因骨髓移植（BMT）后发病率的全面评估仍研究不足。
1974 年至 2014 年间，年龄<22 岁的 789 名异基因 BMT 受者在 BMT 后存活≥2 年，以及 690 名同胞完成了 255 项社会人口统计学和慢性健康状况的自我报告调查。使用不良事件通用术语标准，第 5.0 版（Common Terminology Criteria for Adverse Events，CTCAE v5.0）为这些疾病分配严重程度评分（3 级[严重]、4 级[危及生命]或 5 级[致命]）。对于 BMT 队列，慢性健康状况的累积发生率是作为从 BMT 开始的时间的函数计算的。比例亚分布风险模型用于确定 3-5 级疾病的预测因素。使用逻辑回归估计与同胞相比，在本研究时仍存活的 BMT 受者发生 3-4 级疾病的风险。
移植时的中位年龄为 11.3 岁（范围，0.4-22.0 岁），中位随访时间为 11.7 年（范围，2.0-45.3 年）。最常见的主要诊断为急性淋巴细胞白血病（30.7%）和急性髓系白血病/骨髓增生异常综合征（26.9%）。35 岁时，3-4 级疾病的累积发生率为 53.8%（95%CI，46.7%-60.3%）。幸存者（95%CI，9.5-24.0）发生 3-4 级疾病的调整比值比为 15.1。与同胞相比，女性（HR，1.27；95%CI，1.02-1.59）、接受全身照射（HR，1.71；95%CI，1.27-2.31）和报告慢性移植物抗宿主病（HR，1.38；95%CI，1.09-1.74）的患者发生 3-5 级疾病的风险更高。
与同胞相比，儿童异基因 BMT 的 2 年幸存者发生 3-4 级慢性健康状况的风险增加，表明需要长期随访。
