Goodden John, Pizer Barry, Pettorini Benedetta, Williams Dawn, Blair Jo, Didi Mohammed, Thorp Nicky, Mallucci Conor
Departments of Pediatric Neurosurgery.
J Neurosurg Pediatr. 2014 Jan;13(1):1-12. doi: 10.3171/2013.8.PEDS12546. Epub 2013 Oct 18.
Optic pathway/hypothalamic gliomas (OPHGs) are generally benign tumors situated in an exquisitely sensitive brain region. The location and natural history of OPHGs has led to much debate about optimal treatment. This paper revisits the role of and optimal timing of debulking surgery in OPHG.
This paper presents a series of cases managed by the neuro-oncology team at Alder Hey Children's Hospital and a single surgeon. Data were collected retrospectively for periods prior to 2009 and prospectively thereafter. Tailored treatment strategies were used, including observation and combinations of surgery, chemotherapy, and radiotherapy. Tumor control rates and outcomes are reviewed.
Forty-two patients were treated between 1998 and 2011. Their median age at diagnosis was 5 years 7 months. Nineteen patients were positive for neurofibromatosis Type 1 (NF1) and 23 patients were negative for NF1. The median duration of follow-up was 77 months (range 21.8-142.3 months). Presenting symptoms included visual impairment (in 50% of cases), headache (in 24%), and hypothalamic/pituitary dysfunction (in 29%). Twenty-two debulking procedures were performed in 21 patients. Four biopsies (3 open, 1 endoscopic) were also performed. The histological diagnosis was pilocytic astrocytoma in 21 patients and pilomyxoid astrocytoma in 2 patients. Ten patients (Group 1) had primary surgical debulking alone and were then observed. Four patients (Group 2) had surgical debulking, plus planned chemotherapy within 3 months. Seven patients (Group 3) required surgical debulking for progressive disease following a variety of treatments. Patient age had the greatest impact on subsequent tumor progression. In total, 13 patients received chemotherapy, 4 on initial presentation, 4 in combination with surgery, and 5 for further tumor progression. Five patients were treated with radiotherapy, 3 prior to referral to Alder Hey. Eleven patients required shunt insertion for hydrocephalus. Vision was stabilized for 74% of patients. The number of patients with hypothalamic/pituitary dysfunction increased from 12 at presentation to 16 by the end of treatment. The overall survival rate was 93%. Three patients died-1 from tumor progression, 1 from infective complications from tumor biopsy, and 1 from a spontaneous posterior fossa hemorrhage. NF1 was associated with improved outcome-fewer patients required active intervention and rates of visual impairment and/or or hypothalamic/pituitary dysfunction were lower.
Good long-term survival and functional outcomes can be achieved in children with OPHG. Tumor control was achieved through an individualized approach using surgery, chemotherapy, or radiotherapy in varied combinations. The authors aim to limit radiotherapy to cases involving older children in whom other therapies have failed, due to the well-described and often devastating late effects associated with midline cranial irradiation. Surgery has a clear role for diagnosis, tumor control, and relief of mass effect. In particular, primary surgical debulking of tumor (without adjuvant therapy) is safe and effective. Recent advances in intraoperative MRI may add value and need further assessment.
视路/下丘脑胶质瘤（OPHG）通常是位于极其敏感脑区的良性肿瘤。OPHG的位置和自然病史引发了关于最佳治疗方案的诸多争论。本文重新审视了OPHG减瘤手术的作用及最佳时机。
本文介绍了奥尔德希儿童医院神经肿瘤团队及一位外科医生管理的一系列病例。2009年之前的数据为回顾性收集，之后的数据为前瞻性收集。采用了量身定制的治疗策略，包括观察以及手术、化疗和放疗的联合应用。对肿瘤控制率和治疗结果进行了回顾。
1998年至2011年间共治疗了42例患者。他们诊断时的中位年龄为5岁7个月。19例患者1型神经纤维瘤病（NF1）呈阳性，23例患者NF1呈阴性。中位随访时间为77个月（范围21.8 - 142.3个月）。首发症状包括视力障碍（50%的病例）、头痛（24%）和下丘脑/垂体功能障碍（29%）。21例患者进行了22次减瘤手术。还进行了4次活检（3次开放活检，1次内镜活检）。组织学诊断为毛细胞型星形细胞瘤21例，黏液乳头型星形细胞瘤2例。10例患者（第1组）仅接受了初次手术减瘤，然后进行观察。4例患者（第2组）接受了手术减瘤，并在3个月内计划进行化疗。7例患者（第3组）在接受各种治疗后因疾病进展需要进行手术减瘤。患者年龄对随后的肿瘤进展影响最大。总共有13例患者接受了化疗，4例在初次就诊时接受化疗，4例与手术联合，5例用于进一步的肿瘤进展。5例患者接受了放疗，3例在转诊至奥尔德希之前接受放疗。11例患者因脑积水需要进行分流术。74%的患者视力得以稳定。下丘脑/垂体功能障碍患者的数量从就诊时的12例增加到治疗结束时的16例。总体生存率为93%。3例患者死亡——1例死于肿瘤进展，1例死于肿瘤活检的感染性并发症，1例死于自发性后颅窝出血。NF1与较好的预后相关——需要积极干预的患者较少，视力障碍和/或下丘脑/垂体功能障碍的发生率较低。
OPHG患儿可实现良好的长期生存和功能预后。通过使用手术、化疗或放疗的不同组合的个体化方法实现了肿瘤控制。作者旨在将放疗限制在其他治疗方法失败的大龄儿童病例中，因为中线颅脑照射具有明确且往往具有破坏性的晚期效应。手术在诊断、肿瘤控制和减轻占位效应方面具有明确作用。特别是，肿瘤的初次手术减瘤（无辅助治疗）是安全有效的。术中MRI的最新进展可能具有价值，需要进一步评估。
