Indelicato Daniel J, Bradley Julie A, Sandler Eric S, Aldana Philipp R, Sapp Amy, Gains Jennifer E, Crellin Adrian, Rotondo Ronny L
Department of Radiation Oncology, University of Florida College of Medicine, Jacksonville, Florida.
Department of Pediatric Hematology/Oncology, Nemours Children's Health System, Jacksonville, Florida.
Pediatr Blood Cancer. 2017 Dec;64(12). doi: 10.1002/pbc.26654. Epub 2017 May 24.
International, multidisciplinary care of children with central nervous system (CNS) tumors presents unique challenges. The aim of this study is to report patient outcomes of U.K. children referred for proton therapy to a North American facility.
From 2008 to 2016, 166 U.K. children with approved CNS tumors were treated with proton therapy at a single academic medical center in the United States. Median age was 7 years (range, 1-19). Median follow-up was 2.6 years.
The 3-year actuarial overall survival (OS) and local control (LC) rates were 96% and 91%, respectively, for the overall group, 92% and 85% for the ependymoma subgroup (n = 57), 95% and 88% for the low-grade glioma subgroup (n = 54), and 100% and 100%, respectively, for the craniopharyngioma subgroup (n = 45). Cyst expansion was observed in 13 patients, including one case resulting in visual impairment. Serious side effects included new-onset seizures in three patients (1.8%), symptomatic vasculopathy in three patients (1.8%), and symptomatic brainstem necrosis in one patient (0.6%).
In this cohort of British children referred overseas for proton therapy, disease control does not appear compromised, toxicity is acceptable, and improvement in long-term function is anticipated in survivors owing to the reduced brain exposure afforded by proton therapy.
对中枢神经系统（CNS）肿瘤患儿进行国际多学科护理面临着独特的挑战。本研究旨在报告被转诊至北美机构接受质子治疗的英国儿童的患者治疗结果。
2008年至2016年期间，166名经批准患有中枢神经系统肿瘤的英国儿童在美国一家学术医疗中心接受了质子治疗。中位年龄为7岁（范围1 - 19岁）。中位随访时间为2.6年。
整个队列的3年精算总生存率（OS）和局部控制率（LC）分别为96%和91%，室管膜瘤亚组（n = 57）为92%和85%，低级别胶质瘤亚组（n = 54）为95%和88%，颅咽管瘤亚组（n = 45）分别为100%和100%。13名患者出现囊肿扩大，其中1例导致视力受损。严重副作用包括3名患者（1.8%）出现新发癫痫、3名患者（1.8%）出现有症状的血管病变以及1名患者（0.6%）出现有症状的脑干坏死。
在这一被转诊至海外接受质子治疗的英国儿童队列中，疾病控制似乎未受影响，毒性可接受，并且由于质子治疗减少了对脑部的照射，预计幸存者的长期功能会有所改善。
