采用笔形束扫描质子疗法治疗儿童和青少年原发性脑肿瘤的临床结果和生活质量。
Clinical outcomes and quality of life in children and adolescents with primary brain tumors treated with pencil beam scanning proton therapy.
机构信息
Department of Radiation Oncology, Geneva University Hospital, Geneva, Switzerland.
Department of Radiation Oncology, University College London Hospitals, London, United Kingdom.
出版信息
Pediatr Blood Cancer. 2020 Dec;67(12):e28465. doi: 10.1002/pbc.28465. Epub 2020 Sep 9.
BACKGROUND
Long-term treatment-related toxicity may substantially impact well-being, quality of life (QoL), and health of children/adolescents with brain tumors (CBTs). Strategies to reduce toxicity include pencil beam scanning (PBS) proton therapy (PT). This study aims to report clinical outcomes and QoL in PBS-treated CBTs.
PROCEDURE
We retrospectively reviewed 221 PBS-treated CBTs aged <18 years. Overall-free (OS), disease-free (DFS), and late-toxicity-free survivals (TFS), local control (LC) and distant (DC) brain/spinal control were calculated using Kaplan-Meier estimates. Prospective QoL reports from 206 patients (proxies only ≤4 years old [yo], proxies and patients ≥5 yo) were descriptively analyzed. Median follow-up was 51 months (range, 4-222).
RESULTS
Median age at diagnosis was 3.1 years (range, 0.3-17.7). The main histologies were ependymoma (n = 88; 39.8%), glioma (n = 37; 16.7%), craniopharyngioma (n = 22; 10.0%), atypical teratoid/rhabdoid tumor (ATRT) (n = 21; 9.5%) and medulloblastoma (n = 15; 6.8%). One hundred sixty (72.4%) patients received chemotherapy. Median PT dose was 54 Gy(relative biological effectiveness) (range, 18.0-64.8). The 5-year OS, DFS, LC, and DC (95% CI) were 79.9% (74-85.8), 65.2% (59.8-70.6), 72.1% (65.4-78.8), and 81.8% (76.3-87.3), respectively. Late PT-related ≥G3 toxicity occurred in 19 (8.6%) patients. The 5-year ≥G3 TFS was 91.0% (86.3-95.7). Three (1.4%) secondary malignancies were observed. Patients aged ≤3 years at PT (P = .044) or receiving chemotherapy (P = .043) experienced more ≥G3 toxicity. ATRT histology independently predicted distant brain failure (P = .046) and death (P = .01). Patients aged ≥5 years self-rated QoL higher than their parents (proxy assessment). Both reported lower social functioning and cognition after PT than at baseline, but near-normal long-term global well-being. QoL was well below normal before and after PT in children ≤4 years.
CONCLUSIONS
The outcome of CBTs was excellent after PBS. Few patients had late ≥G3 toxicity. Patients aged <5 years showed worse QoL and toxicity outcomes.
背景
长期治疗相关的毒性可能会极大地影响患有脑肿瘤（CBT）的儿童/青少年的幸福感、生活质量（QoL）和健康。降低毒性的策略包括铅笔束扫描（PBS）质子治疗（PT）。本研究旨在报告 PBS 治疗的 CBT 患者的临床结果和 QoL。
过程
我们回顾性分析了 221 例接受 PBS 治疗的年龄<18 岁的 CBT 患者。使用 Kaplan-Meier 估计计算总生存期（OS）、无疾病生存期（DFS）和晚期毒性无生存期（TFS）、局部控制（LC）和远处（DC）脑/脊髓控制的情况。对 206 例患者（仅<4 岁的代理，患者和代理≥5 岁）前瞻性 QoL 报告进行了描述性分析。中位随访时间为 51 个月（范围为 4-222）。
结果
诊断时的中位年龄为 3.1 岁（范围为 0.3-17.7）。主要组织学类型为室管膜瘤（n=88；39.8%）、神经胶质瘤（n=37；16.7%）、颅咽管瘤（n=22；10.0%）、非典型畸胎瘤/横纹肌样瘤（ATRT）（n=21；9.5%）和髓母细胞瘤（n=15；6.8%）。160 例（72.4%）患者接受了化疗。中位 PT 剂量为 54 Gy（相对生物效应）（范围为 18.0-64.8）。5 年 OS、DFS、LC 和 DC（95%CI）分别为 79.9%（74-85.8）、65.2%（59.8-70.6）、72.1%（65.4-78.8）和 81.8%（76.3-87.3）。19 例（8.6%）患者发生晚期 PT 相关≥G3 毒性。5 年≥G3 TFS 为 91.0%（86.3-95.7）。观察到 3 例（1.4%）继发性恶性肿瘤。PT 时年龄≤3 岁的患者（P=0.044）或接受化疗的患者（P=0.043）经历了更多的≥G3 毒性。ATRT 组织学独立预测远处脑失败（P=0.046）和死亡（P=0.01）。年龄≥5 岁的患者自我报告的 QoL 高于其父母（代理评估）。两组患者在 PT 后比基线时的社会功能和认知能力均下降，但长期整体健康状况接近正常。≤4 岁的儿童在 PT 前后的 QoL 均明显低于正常水平。
结论
CBT 经 PBS 治疗后疗效极佳。少数患者发生晚期≥G3 毒性。年龄<5 岁的患者 QoL 和毒性结果更差。
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