Department of Radiation Oncology, Massachusetts General Hospital, Boston, Massachusetts.
Department of Clinical Oncology, Aswan University, Aswan, Egypt.
Int J Radiat Oncol Biol Phys. 2021 Aug 1;110(5):1480-1487. doi: 10.1016/j.ijrobp.2021.02.045. Epub 2021 Mar 1.
Radiation therapy (RT) is used for pediatric craniopharyngioma in the definitive, adjuvant, or salvage settings. Proton RT may be useful owing to tumor proximity to eloquent anatomy. We report clinical outcomes for a large cohort treated with proton therapy.
We conducted a retrospective review of pediatric patients (≤21 years) treated with surgery and proton therapy for craniopharyngioma between August 2002 and October 2018. Clinical characteristics, treatment course, and outcomes were recorded. Acute toxicity was graded using Common Terminology Criteria for Adverse Events, version 5.0. Late toxicity was assessed using neuroendocrine, neuro-ophthalmologic, and neuropsychological testing.
Among 77 patients, median age at diagnosis was 8.6 years (range, 1.3-20); median age at radiation was 9.6 years (range, 2.3-20.5). Most common presenting symptoms were headache (58%), visual impairment (55%), and endocrinopathy (40%). Patients underwent a median of 2 surgical interventions (range, 1-7) before protons. At initial surgery, 18% had gross total resection, 60% had subtotal resection, and 22% had biopsy/cyst decompression. Median RT dose was 52.2 Gy (relative biologic effectiveness). Common acute toxicities were headache (29%), fatigue (35%), and nausea/vomiting (12%). Only 4% developed any acute grade 3 toxicity. Nine patients experienced cyst growth requiring replanning or surgical decompression. At a median of 4.8 years from RT (range, 0.8-15.6), there were 6 local failures and 3 deaths, 2 related to disease progression. Effect of tumor and treatment contributed to late toxicity including Moyamoya syndrome (13%), visual impairment (40%), and endocrine deficiency requiring hormone replacement (94%). Subclinical decline in functional independence and adaptive skills in everyday life was detected at follow-up.
Surgery and proton therapy results in excellent disease control for pediatric craniopharyngioma. Severe acute toxicity is rare. Late toxicities from tumor, surgery, and radiation remain prevalent. Endocrine and ophthalmology follow-up is necessary, and neuropsychological testing may identify patients at risk for treatment-related cognitive and adaptive functioning changes.
放射治疗（RT）用于儿童颅咽管瘤的明确、辅助或挽救性治疗。由于肿瘤靠近重要解剖结构，质子 RT 可能有用。我们报告了一大群接受质子治疗的患者的临床结果。
我们对 2002 年 8 月至 2018 年 10 月期间接受手术和质子治疗颅咽管瘤的儿童患者（≤21 岁）进行了回顾性研究。记录了临床特征、治疗过程和结果。急性毒性采用不良事件常用术语标准，版本 5.0 进行分级。迟发性毒性采用神经内分泌、神经眼科和神经心理学测试进行评估。
在 77 例患者中，中位诊断年龄为 8.6 岁（范围，1.3-20 岁）；中位放疗年龄为 9.6 岁（范围，2.3-20.5 岁）。最常见的首发症状为头痛（58%）、视力障碍（55%）和内分泌功能障碍（40%）。患者在接受质子治疗前中位进行了 2 次手术干预（范围，1-7 次）。在初次手术中，18%为大体全切除，60%为次全切除，22%为活检/囊肿减压。中位放疗剂量为 52.2 Gy（相对生物效应）。常见的急性毒性包括头痛（29%）、疲劳（35%）和恶心/呕吐（12%）。仅有 4%发生任何 3 级急性毒性。9 例患者出现囊肿生长，需要重新计划或手术减压。从放疗开始中位时间为 4.8 年（范围，0.8-15.6 年），有 6 例局部复发和 3 例死亡，其中 2 例与疾病进展有关。肿瘤和治疗的影响导致迟发性毒性，包括烟雾病（13%）、视力障碍（40%）和需要激素替代的内分泌缺乏（94%）。在随访中发现，日常功能独立性和适应性技能的亚临床下降。
手术和质子治疗为儿童颅咽管瘤提供了极好的疾病控制。严重的急性毒性罕见。肿瘤、手术和放疗引起的迟发性毒性仍然普遍存在。需要进行内分泌和眼科随访，神经心理学测试可能会发现因治疗相关认知和适应性功能变化而处于风险中的患者。
