Szychot Elwira, Seunarine Kiran, Mankad Kshitij, Thust Steffi, Clark Chris, Gaze Mark N, Michalski Antony
Department of Clinical Studies, The Institute of Cancer Research, London, United Kingdom.
Department of Paediatric Oncology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom.
Pediatr Blood Cancer. 2017 Nov;64(11). doi: 10.1002/pbc.26619. Epub 2017 May 16.
The introduction of aggressive chemo-radiotherapy regimens has improved overall survival in children with primitive neuroectodermal tumours (PNET). However, these combinations may result in neurotoxicity. Previously reported magnetic resonance imaging abnormalities in children receiving intensive sequential chemotherapy, hyperfractionated accelerated radiotherapy (HART) and high-dose thiotepa prompted us to investigate the degree of brain volume loss and patients' functional status after therapy.
We retrospectively reviewed clinico-radiological data of children with PNET treated in this way at our centre.
We studied 14 children treated between December 2009 and April 2013. Data were not complete for one child. Performance status was severely restricted in four children, and mildly to moderately impaired in 7 of the 13 children. Eleven of 13 children showed mild-to-severe generalised neuroparenchymal atrophy, in 7 of whom neuroparenchymal volume loss was moderate to severe. Of these seven, six had received high-dose thiotepa. There was no correlation between brain volume loss and Lansky performance status. However, unexpected neurotoxicities, such as symptoms of transverse myelitis, were observed.
Measurement of brain volume loss in patients treated with HART and high-dose thiotepa may not be sufficient to predict function. However, correlation of brain volume loss due to late neurotoxicity with performance decline may be more obvious over longer period of follow-up. The combination of HART and myeloablative courses of thiotepa is associated with severe neurotoxicity and subsequent decline in performance status in a significant proportion of patients.
积极的放化疗方案的引入提高了原始神经外胚层肿瘤（PNET）患儿的总生存率。然而，这些联合治疗可能会导致神经毒性。先前报道，接受强化序贯化疗、超分割加速放疗（HART）和高剂量硫代磷酸三乙酯治疗的儿童出现磁共振成像异常，这促使我们研究治疗后脑容量损失程度和患者的功能状态。
我们回顾性分析了在本中心接受此类治疗的PNET患儿的临床放射学数据。
我们研究了2009年12月至2013年4月期间接受治疗的14名儿童。有一名儿童的数据不完整。4名儿童的体能状态严重受限，13名儿童中有7名轻度至中度受损。13名儿童中有11名表现出轻度至重度的广泛性神经实质萎缩，其中7名神经实质体积损失为中度至重度。在这7名儿童中，有6名接受过高剂量硫代磷酸三乙酯治疗。脑容量损失与兰斯基体能状态之间没有相关性。然而，观察到了意外的神经毒性，如横贯性脊髓炎症状。
对于接受HART和高剂量硫代磷酸三乙酯治疗的患者，测量脑容量损失可能不足以预测功能。然而，在更长的随访期内，晚期神经毒性导致的脑容量损失与功能下降之间的相关性可能会更明显。HART与硫代磷酸三乙酯清髓疗程的联合治疗与严重的神经毒性以及相当一部分患者随后的体能状态下降有关。
