Background:The current standard therapy for pediatric optic pathway/hypothalamic glioma (OPHG) is systemic anticancer therapy (SAT) over surgery and radiotherapy. Nevertheless, recurrent radiological or clinical tumor progression after SAT forms a considerable challenge. Sporadic OPHGs are considered to have a higher tendency toward progression after first-line systemic anticancer therapy (SAT) compared to neurofibromatosis type-1-associated (NF1) OPHGs.Methods:The objective of this study was to conduct a national retrospective cohort analysis of children who received various treatments for a progressive OPHG, involving the hypothalamus and/or chiasm and/or optic radiations. The study aimed to examine the differences in clinical course and the range of treatment modalities applied to both sporadic and NF1-associated OPHGs between 1995 and 2020. Additionally, we sought to identify risk factors for 3- and 5-year progression following first- and second-order SAT.Results:In total, 136 children received treatment, of whom 49 of 136 (36.0%) had NF1. Within a median of 7.5 years (range: 0.1–23.8 years) of follow-up, sporadic OPHGs received more treatments compared to NF1-associated OPHGs (median of 2 (range: 1–8) vs. median of 1 (range: 1–7) (p< 0.01)). Nine children with sporadic OPHGs (6.6%) died. Of 112 children (82.4%) receiving SAT, 92% received combined first-line vincristine and carboplatin. These children had a 3- and 5-year progression-free survival of 61.8% (95% CI: 51.0–72.6%) and 48.4% (95% CI: 38.0–58.8%), respectively. Sporadic OPHGs had a higher rate of second progression (p< 0.01). Starting first-line vincristine and carboplatin at an age below one year was the only independent risk factor for progression.Conclusions:In this national historic cohort of pediatric OPHGs, four out of five children received SAT. Sporadic OPHGs received a higher number of various SATs compared to NF1-associated OPHGs, but the sporadic appearance of OPHGs was not an independent risk factor for progression after combined vincristine and carboplatin, as ‘age below one year at the start’ was the only factor.
背景：目前儿童视路/下丘脑胶质瘤（OPHG）的标准治疗方案是全身性抗癌治疗（SAT），而非手术或放疗。然而，SAT后出现的影像学或临床肿瘤进展复发构成了重大挑战。与1型神经纤维瘤病相关（NF1）的OPHG相比，散发性OPHG被认为在一线全身性抗癌治疗（SAT）后具有更高的进展倾向。 方法：本研究旨在对1995年至2020年间因累及下丘脑和/或视交叉和/或视辐射的进展性OPHG接受过各种治疗的儿童进行一项全国性回顾性队列分析。研究目标包括：比较散发性与NF1相关OPHG的临床病程差异及所采用治疗方式的种类，并识别一线和二级SAT后3年及5年进展的风险因素。 结果：共有136名儿童接受了治疗，其中49人（36.0%）患有NF1。在中位随访7.5年（范围：0.1-23.8年）期间，散发性OPHG患者接受的治疗次数显著多于NF1相关OPHG患者（中位数2次（范围：1-8）对比中位数1次（范围：1-7），p<0.01）。9名散发性OPHG患儿（6.6%）死亡。在接受SAT的112名儿童（82.4%）中，92%接受了一线长春新碱联合卡铂方案治疗。这些患儿的3年无进展生存率为61.8%（95% CI：51.0-72.6%），5年无进展生存率为48.4%（95% CI：38.0-58.8%）。散发性OPHG的二次进展率更高（p<0.01）。一岁以下开始一线长春新碱联合卡铂治疗是进展的唯一独立风险因素。 结论：在这项全国性儿童OPHG历史队列研究中，五分之四的患儿接受了SAT。与NF1相关OPHG相比，散发性OPHG接受了更多种类的SAT治疗，但散发性OPHG本身并非长春新碱联合卡铂治疗后进展的独立风险因素，因为“开始治疗时年龄小于一岁”是唯一的风险因素。
肿瘤(癌症)患者之家