Phipps S, Dunavant M, Srivastava D K, Bowman L, Mulhern R K
Division of Behavioral Medicine, Department of Biostatistics, St Jude Children's Research Hospital, Memphis, TN, USA.
J Clin Oncol. 2000 Mar;18(5):1004-11. doi: 10.1200/JCO.2000.18.5.1004.
To evaluate cognitive and academic functioning in survivors of pediatric bone marrow transplants (BMTs) at 1 and 3 years after a BMT.
In a prospective, longitudinal design, patients underwent a comprehensive battery of neurocognitive measures before admission for transplantation and at 1, 3, and 5 years after a BMT. This article describes a cohort of 102 survivors with follow-up data available for 1 year after a BMT, including 54 survivors with follow-up available for 3 years. This represents the largest cohort of pediatric BMT survivors yet reported in a prospective study.
In the cohort as a whole, there were no significant changes on global measures of intelligence (intelligence quotient [IQ]) and academic achievement at either 1 or 3 years after a BMT, despite adequate power to detect an IQ change of three points or greater. Likewise, performance on specific tests of neuropsychologic function remained stable. No significant differences were observed between patients whose conditioning regimen included total-body irradiation (TBI) and those whose did not. The primary predictor of neurocognitive outcome was patient age, with younger patients more likely to show declines over time. The subset of patients who were less than 3 years of age at the time of transplantation seemed to be particularly vulnerable to cognitive sequelae.
The use of BMTs with or without TBI entails minimal risk of late neurocognitive sequelae in patients who are 6 years of age or older at the time of transplantation. However, patients who are less than 6 years of age at the time of transplantation, and particularly those less than 3 years of age, seem to be at some risk of cognitive declines.
评估儿童骨髓移植（BMT）幸存者在BMT后1年和3年时的认知及学业功能。
采用前瞻性纵向设计，患者在移植入院前以及BMT后1年、3年和5年接受一系列全面的神经认知测量。本文描述了一组102名幸存者，他们有BMT后1年的随访数据，其中54名幸存者有3年的随访数据。这是前瞻性研究中报道的最大一组儿童BMT幸存者。
在整个队列中，BMT后1年或3年时，智力（智商[IQ]）和学业成绩的整体测量指标均无显著变化，尽管有足够的检验效能来检测3分及以上的IQ变化。同样，神经心理功能特定测试的表现保持稳定。接受包含全身照射（TBI）的预处理方案的患者与未接受该方案的患者之间未观察到显著差异。神经认知结果的主要预测因素是患者年龄，年龄较小的患者随着时间推移更有可能出现下降。移植时年龄小于3岁的患者亚组似乎特别容易出现认知后遗症。
对于移植时6岁及以上的患者，无论是否使用TBI进行BMT，后期发生神经认知后遗症的风险都极小。然而，移植时年龄小于6岁的患者，尤其是小于3岁的患者，似乎存在一定的认知功能下降风险。
