Levitch Cara F, Malkin Benjamin, Latella Lauren, Guerry Whitney, Gardner Sharon L, Finlay Jonathan L, Sands Stephen A
Department of Psychiatry and Behavioral Sciences and Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York, New York, USA.
Graduate School of Education, Fordham University, Bronx, New York, USA.
Neurooncol Pract. 2021 May 18;8(5):609-619. doi: 10.1093/nop/npab028. eCollection 2021 Oct.
The Head Start treatment protocols have focused on curing young children with brain tumors while avoiding or delaying radiotherapy through using a combination of high-dose, marrow-ablative chemotherapy and autologous hematopoietic cell transplantation (AuHCT). Late effects data from treatment on the Head Start II (HS II) protocol have previously been published for short-term follow-up (STF) at a mean of 39.7 months post-diagnosis. The current study examines long-term follow-up (LTF) outcomes from the same cohort.
Eighteen HS II patients diagnosed with malignant brain tumors <10 years of age at diagnosis completed a neurocognitive battery and parents completed psychological questionnaires at a mean of 104.7 months' post-diagnosis.
There was no significant change in Full Scale IQ at LTF compared to baseline or STF. Similarly, most domains had no significant change from STF, including verbal IQ, performance IQ, academics, receptive language, learning/memory, visual-motor integration, and externalizing behaviors. Internalizing behaviors increased slightly at LTF. Clinically, most domains were within the average range, except for low average mathematics and receptive language. Additionally, performance did not significantly differ by age at diagnosis or time since diagnosis. Of note, children treated with high-dose methotrexate for disseminated disease or atypical teratoid/rhabdoid tumor displayed worse neurocognitive outcomes.
These results extend prior findings of relative stability in intellectual functioning for a LTF period. Ultimately, this study supports that treatment strategies for avoiding or delaying radiotherapy using high-dose, marrow-ablative chemotherapy and AuHCT may decrease the risk of neurocognitive and social-emotional declines in young pediatric brain tumor survivors.
“启智计划”治疗方案专注于治愈患有脑肿瘤的幼儿，同时通过联合使用大剂量、清髓性化疗和自体造血细胞移植（AuHCT）来避免或延迟放疗。先前已发表了“启智计划II（HS II）”方案治疗的晚期效应数据，用于诊断后平均39.7个月的短期随访（STF）。本研究调查了同一队列的长期随访（LTF）结果。
18名诊断时年龄小于10岁的HS II恶性脑肿瘤患者在诊断后平均104.7个月完成了一套神经认知测试，家长完成了心理问卷。
与基线或短期随访相比，长期随访时全量表智商没有显著变化。同样，大多数领域与短期随访相比没有显著变化，包括言语智商、操作智商、学业、接受性语言、学习/记忆、视觉运动整合和外化行为。内化行为在长期随访时略有增加。临床上，除了数学和接受性语言处于低平均水平外，大多数领域都在平均范围内。此外，诊断时的年龄或诊断后的时间对表现没有显著差异。值得注意的是，接受大剂量甲氨蝶呤治疗播散性疾病或非典型畸胎样/横纹肌样肿瘤的儿童神经认知结果较差。
这些结果扩展了先前关于长期随访期间智力功能相对稳定的研究结果。最终，本研究支持使用大剂量、清髓性化疗和AuHCT避免或延迟放疗的治疗策略可能会降低小儿脑肿瘤幸存者神经认知和社会情感衰退的风险。
