美金刚是某些接受脑放射治疗（RT）以减少认知影响的成年人的标准药物，但尚不清楚儿科患者是否可以服用、耐受美金刚和/或受益于美金刚。在这项前瞻性单臂可行性研究中，我们假设接受中枢神经系统 (CNS) 放疗的儿科患者能够耐受美金刚并具有良好的依从性。 4 至 18 岁患有原发性 CNS 恶性肿瘤的患者（不包括世界卫生组织 4 级星形细胞瘤、胶质母细胞瘤） ) 接受颅内放疗的患者符合资格。在 RT 期间和之后进行为期 6 个月的美金刚疗程，在 4 周内以 5 mg 增量调整剂量，目标是基于体重的最大剂量（0.4 mg/kg，最接近的 5 mg），每天两次不超过 10 mg。主要终点是 RT 后 1 个月测量的 80% 患者的药物依从率达到 80%。次要目标包括 3 个月和 6 个月时美金刚的可行性。2020 年至 2022 年期间招募了 18 名患者，并接受了美金刚联合 RT 治疗。该研究提前结束，以避免与 3 期随机儿童肿瘤学组研究 AACL2031 竞争。没有满足预定义的停止规则。一名患者因使用改变认知的物质而退出，留下 17 名患者可供分析。一名患者在服用一剂美金刚后因恶心而停用美金刚。对于其余 16 名患者，在 RT 后 1 个月时，药物完成率中位数为 100%（范围为 74%-100%；n = 9/17，100% 依从性），其中 15/16 (94%)坚持率>80%。在次要终点 RT 后 3 个月和 6 个月时间点，中位依从率分别为 100%（范围，55%-100%）和 96%（范围，33%-100%）。 1 至 2 级疲劳、头痛和恶心是最常见的毒性事件，至少可能与研究药物 (n = 27) 有关，没有可归因的 3 级事件。美金刚是一种可行、安全且耐受性良好的补充药物儿童中枢神经系统恶性肿瘤的多学科治疗。等待“匿名审查”的结果来确定美金刚在该人群中的价值。版权所有 © 2024 Elsevier Inc. 保留所有权利。

Memantine is standard in certain adults receiving brain radiation therapy (RT) to decrease cognitive impacts, but it is unknown whether pediatric patients can take, tolerate, and/or benefit from memantine. In this prospective single-arm feasibility study, we hypothesized that pediatric patients receiving central nervous system (CNS) RT would tolerate memantine with good adherence.Patients aged 4 to 18 years with a primary CNS malignancy (excluding World Health Organization grade 4 astrocytoma, glioblastoma) receiving intracranial RT were eligible. A 6-month memantine course was given during and after RT, with dose titration in 5 mg increments over 4 weeks targeting a weight-based maximum (0.4 mg/kg to the closest 5 mg), not to exceed 10 mg twice a day. The primary endpoint was to achieve 80% drug adherence rate in 80% of patients measured 1 month after RT. Secondary objectives included memantine feasibility at 3 and 6 months.Eighteen patients enrolled from 2020 to 2022 and were prescribed memantine with RT. The study closed early to avoid competing with the phase 3 randomized Children's Oncology Group study AACL2031. No predefined stopping rules were met. One patient withdrew for cognition-altering substance use, leaving 17 patients available for analysis. One patient discontinued memantine after one dose due to nausea. For the remaining 16 patients, there was a median of 100% pill completion rate (range, 74%-100%; n = 9/17 with 100% adherence) at 1 month after RT, with 15/16 (94%) with adherence rates >80%. At the 3- and 6-month post-RT time points for secondary endpoints, the median adherence rates were 100% (range, 55%-100%) and 96% (range, 33%-100%), respectively. Grade 1 to 2 fatigue, headache, and nausea were the most common toxicity events, at least possibly related to the study drug (n = 27), without attributable grade 3+ events.Memantine is a feasible, safe, and well-tolerated addition to multimodality treatment for pediatric CNS malignancies. Results of "Anonymized for Review" are awaited to define the value of memantine in this population.Copyright © 2024 Elsevier Inc. All rights reserved.