McCabe M A, Crowe H, Quinones R
Department of Hematology/Oncology, Children's National Medical Center, Washington, D.C., USA.
J Pediatr Hematol Oncol. 1997 Mar-Apr;19(2):145-50. doi: 10.1097/00043426-199703000-00010.
Bone marrow transplantation (BMT), particularly preparative regimens, may have a significant impact on the developing nervous system. However, the effects of various BMT regimens on children's growth and development have been poorly documented to date. Twins serve as ideal subjects to study the impact of medical treatment, since they control for nonmedical (genetic and environmental) influences upon neurodevelopmental outcome.
Two cases of monozygotic twins are presented to illustrate the impact of BMT regimens. Growth data and neurocognitive testing are presented for each patient (affected twin) in relation to his/her syngeneic BMT donor and case control (control twin).
These two cases illustrate the growth retardation that has been reported after BMT. However, changes in growth trends across twins appear to have begun after diagnosis, rather than after BMT per se. Comparisons of cognitive test results within these twin pairs illustrate learning problems in the affected twins. However, there was also evidence of learning anomalies in the unaffected twins.
Results underscore the importance of longitudinal assessment in order to identify the side effects of BMT regimens for children. Differences across the two cases highlight important research questions regarding variables associated with patients, disease, and treatment (e.g., age at the time of BMT, previous neurotoxic treatments, underlying disease) and emphasize the importance of controls in this line of research.
骨髓移植（BMT），尤其是预处理方案，可能会对发育中的神经系统产生重大影响。然而，迄今为止，各种BMT方案对儿童生长发育的影响鲜有文献记载。双胞胎是研究医学治疗影响的理想对象，因为他们可以控制非医学（遗传和环境）因素对神经发育结果的影响。
介绍两例同卵双胞胎病例，以说明BMT方案的影响。针对每位患者（受影响的双胞胎），给出了其与同基因BMT供体及病例对照（对照双胞胎）相关的生长数据和神经认知测试结果。
这两例病例说明了BMT后报告的生长发育迟缓情况。然而，双胞胎之间生长趋势的变化似乎在诊断后就已开始，而非在BMT本身之后。对这些双胞胎对认知测试结果的比较表明，受影响的双胞胎存在学习问题。然而，也有证据表明未受影响的双胞胎存在学习异常。
结果强调了纵向评估对于识别儿童BMT方案副作用的重要性。这两例病例的差异突出了关于与患者、疾病和治疗相关变量（例如BMT时的年龄、先前的神经毒性治疗、基础疾病）的重要研究问题，并强调了该研究领域中对照的重要性。
