Comparison of Symptom Burden/Toxicity, Neurocognitive Change, and Functional Outcomes in Pediatric Brain Tumor Patients Treated with Proton vs. Photon Radiotherapy.

项目概要 在美国，大多数接受癌症治疗的儿童都将实现长期生存，并且生存率具有独特性 这个不断增长的人口所面临的挑战。儿童脑肿瘤幸存者尤其面临以下风险： 神经认知障碍、教育困难、社会问题和医疗残疾。颅脑辐射 治疗是一种重要的救生治疗方法，但会导致认知能力下降。质子束辐射 疗法（PBRT）是儿科脑肿瘤治疗中最有前途的最新进展之一。拟议的 PBRT 的医学优势在于质子束辐射传输的精确性，可最大程度地沉积 剂量到临床目标，同时最大限度地减少对周围组织的辐射。通过减少健康大脑的剂量 与传统的光子或组织相比，PBRT 可以更好地保护认知功能并减轻症状负担 X 射线照射 (XRT) 可提高生存的功能独立性。 使用基于模型的加速纵向队列比较设计，我们将比较症状 从辐射开始起多个数据点的负担/毒性、神经认知变化和功能结果 与 XRT 相比，接受 PBRT 治疗的患者的晚期生存情况。提出以下目标： (1) 比较儿科脑肿瘤患者和幸存者不同放疗类型的症状负担和毒性，(2) 比较不同 RT 类型的神经认知结果随时间的变化，(3) 比较不同 RT 类型的功能结果 按 RT 类型划分的早期和晚期生存率，以及 (4) 检查症状负担/毒性之间的关系， 神经认知功能和功能结果作为 RT 类型的函数。 该提案与 NCI 的目标一致，即“减少癌症及其相关疾病的长期不良影响”。 儿童的治疗”以及“改善癌症患者、幸存者及其家人的生活质量”。 神经认知迟发效应导致许多人在教育、社会和职业方面受到严重限制 幸存者，极大地影响他们的生活质量和长期功能独立性。需要研究 确定哪些治疗最能限制与症状负担和术后相关的痛苦 治疗神经认知能力下降。我们的结果将具有临床价值，可以及时比较 PBRT 和 XRT 组之间的症状、神经认知变化和功能结果将指导 临床医生和家属了解 PBRT 后预期的一系列结果。

PROJECT SUMMARY Most children treated for cancer in the US will achieve long-term survival, and survivorship presents unique challenges for this growing population. Pediatric brain tumor survivors, in particular, are at risk for neurocognitive impairments, educational difficulties, social problems, and medical disabilities. Cranial radiation therapy is an essential lifesaving treatment but is associated with cognitive decline. Proton beam radiation therapy (PBRT) is one of the most promising recent advances in pediatric brain tumor treatment. The proposed medical advantage of PBRT lies in the precision of radiation delivery with proton beams, depositing maximum dose to clinical targets while minimizing radiation to surrounding tissues. By reducing dose to healthy brain tissue, PBRT may spare cognitive functioning and reduce symptom burden better than conventional photon or x-ray irradiation (XRT) leading to greater functional independence in survivorship. Using a model-based, accelerated longitudinal cohort comparison design, we will compare symptom burden/toxicity, neurocognitive change, and functional outcomes at multiple data points from start of radiation through late survivorship in patients treated with PBRT versus XRT. The following aims are proposed: (1) to compare symptom burden and toxicity by RT type in pediatric brain tumor patients and survivors, (2) to compare change in neurocognitive outcomes over time by RT type, (3) to compare functional outcomes in early and late survivorship by RT type, and (4) to examine relations among symptom burden/toxicity, neurocognitive function, and functional outcomes as a function of RT type. This proposal is consistent with NCI’s objective to “reduce the long-term adverse effects of cancer and its treatment” in children and to “improve the quality of life for cancer patients, survivors, and their families.” Neurocognitive late effects lead to significant educational, social, and occupational limitations for many survivors, greatly affecting their quality of life and functional independence long-term. Research is needed to determine which treatments are best able to limit the suffering associated with symptom burden and post- treatment neurocognitive decline. Our results will have clinical value, providing a timely comparison of symptoms, neurocognitive changes, and functional outcomes between PBRT and XRT groups that will guide clinicians and families on the range of outcomes to expect after PBRT.