Lucas John T, Ladra Matthew M, MacDonald Shannon M, Busse Paul M, Friedmann Alison M, Ebb David H, Marcus Karen J, Tarbell Nancy J, Yock Torunn I
Department of Radiation Oncology, Wake Forest School of Medicine, Winston-Salem, North Carolina.
Provision Center for Proton Therapy, Knoxville, Tennessee.
Pediatr Blood Cancer. 2015 Sep;62(9):1523-8. doi: 10.1002/pbc.25494. Epub 2015 Mar 27.
Esthesioneuroblastoma (EN) of the paranasal sinus comprises less than 3% of tumors of in pediatric and adolescent patients [1]. The collective adult literature indicates a critical role for radiotherapy in attaining cure [2], yet pediatric outcome data is limited. Radiation in pediatric patients with EN can cause significant morbidity due to the proximity of critical structures. Proton radiotherapy offers a potential dosimetric benefit that may improve long-term survival and toxicity outcomes in the pediatric population [3].
We retrospectively identified eight patients treated for EN with proton radiotherapy from 2000-2013. Times to event clinical endpoints are summarized using the Kaplan-Meier methods and are from the date of radiotherapy completion. Toxicities are reviewed and graded according to CTCAE v. 4.0.
Median follow up was 4.6 years for survivors (range 0.8-9.4 years). The 4 year overall survival was 87.5%. Four of eight patients (one elective) had comprehensive neck radiotherapy. No local or regional failures were observed. Two patients failed distantly with diffuse leptomeningeal disease and intraparenchymal brain metastases, at 0.6 and 1.3 months respectively. Four patients developed radiation related late toxicities including endocrine dysfunction, two cases of grade 2 retinopathy and one case of grade 3 optic neuropathy.
In a limited cohort, proton radiotherapy appears to provide excellent locoregional disease control even in those patients with locally advanced disease and intracranial extension. Distant failure determined overall survival in our cohort. Toxicities were acceptable given disease location and extent.
鼻窦嗅神经母细胞瘤（EN）在儿童和青少年患者的肿瘤中占比不到3%[1]。成人相关文献表明放疗在实现治愈方面起着关键作用[2]，但儿科的预后数据有限。由于关键结构位置临近，儿童EN患者接受放疗会导致显著的发病率。质子放疗具有潜在的剂量学优势，可能改善儿科患者的长期生存和毒性结局[3]。
我们回顾性确定了2000年至2013年期间接受质子放疗的8例EN患者。采用Kaplan-Meier方法总结至事件临床终点的时间，时间从放疗完成之日起计算。根据CTCAE v. 4.0对毒性进行评估和分级。
幸存者的中位随访时间为4.6年（范围0.8 - 9.4年）。4年总生存率为87.5%。8例患者中有4例（1例为选择性）接受了全颈放疗。未观察到局部或区域复发。2例患者分别在0.6个月和1.3个月时出现远处转移失败，表现为弥漫性软脑膜疾病和脑实质内脑转移。4例患者出现放疗相关的晚期毒性反应，包括内分泌功能障碍、2例2级视网膜病变和1例3级视神经病变。
在一个有限的队列中，质子放疗似乎能提供出色的局部区域疾病控制，即使是那些局部晚期疾病和颅内扩展的患者。远处转移失败决定了我们队列中的总生存率。考虑到疾病的位置和范围，毒性反应是可接受的。
