Hansford Jordan R, Huang Jie, Endersby Raelene, Dodgshun Andrew J, Li Bryan K, Hwang Eugene, Leary Sarah, Gajjar Amar, Von Hoff Katja, Wells Olivia, Wray Alison, Kotecha Rishi S, Raleigh David R, Stoller Schuyler, Mueller Sabine, Schild Steven E, Bandopadhayay Pratiti, Fouladi Maryam, Bouffet Eric, Huang Annie, Onar-Thomas Arzu, Gottardo Nicholas G
Children's Cancer Center, Royal Children's Hospital, Melbourne, Victoria, Australia.
Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Neurooncol Adv. 2022 Apr 14;4(1):vdac056. doi: 10.1093/noajnl/vdac056. eCollection 2022 Jan-Dec.
Pineoblastoma is a rare brain tumor usually diagnosed in children. Given its rarity, no pineoblastoma-specific trials have been conducted. Studies have included pineoblastoma accruing for other embryonal tumors over the past 30 years. These included only occasional children with pineoblastoma, making clinical features difficult to interpret and determinants of outcome difficult to ascertain.
Centrally or independently reviewed series with treatment and survival data from North American and Australian cases were pooled. To investigate associations between variables, Fisher's exact tests, Wilcoxon-Mann-Whitney tests, and Spearman correlations were used. Kaplan-Meier plots, log-rank tests, and Cox proportional hazards models were used in survival analyses.
We describe a pooled cohort of 178 pineoblastoma cases from Children's Oncology Group (n = 82) and institutional series (n = 96) over 30 years. Children <3 years of age have significantly worse survival compared to older children, with 5-year progression-free survival (PFS) and overall survival (OS) estimates of 13.5 ± 5.1% and 16.2 ± 5.3%, respectively, compared with 60.8 ± 5.6% and 67.3 ± 5.0% for ≥3 years old (both < .0001). Multivariable analysis showed male sex was associated with worse PFS in children <3 years of age (hazard ratio [HR] 3.93, 95% CI 1.80-8.55; = .0006), suggestive of sex-specific risks needing future validation. For children ≥3 years of age, disseminated disease at diagnosis was significantly associated with an inferior 5-year PFS of 39.2 ± 9.7% (HR 2.88, 95% CI 1.52-5.45; = .0012) and 5-year OS of 49.8 ± 9.1% (HR 2.87, 95% CI 1.49-5.53; = .0016).
Given the rarity of this tumor, prospective, collaborative international studies will be vital to improving the long-term survival of these patients.
松果体母细胞瘤是一种罕见的脑肿瘤，通常在儿童中被诊断出来。鉴于其罕见性，尚未开展针对松果体母细胞瘤的特异性试验。在过去30年中，一些研究纳入了因其他胚胎性肿瘤而确诊的松果体母细胞瘤病例。但这些研究中仅有少数松果体母细胞瘤患儿，这使得临床特征难以解读，预后的决定因素也难以确定。
汇总了北美和澳大利亚病例的经中心或独立审核的系列研究，其中包含治疗和生存数据。为研究变量之间的关联，使用了Fisher精确检验、Wilcoxon-Mann-Whitney检验和Spearman相关性分析。生存分析采用Kaplan-Meier曲线、对数秩检验和Cox比例风险模型。
我们描述了一个30年间来自儿童肿瘤协作组（n = 82）和机构系列研究（n = 96）的178例松果体母细胞瘤病例的汇总队列。与年龄较大的儿童相比，3岁以下儿童的生存率显著更差，其5年无进展生存率（PFS）和总生存率（OS）估计分别为13.5±5.1%和16.2±5.3%，而3岁及以上儿童分别为60.8±5.6%和67.3±5.0%（两者均P <.0001）。多变量分析显示，男性性别与3岁以下儿童较差的PFS相关（风险比[HR] 3.93，95%置信区间1.80 - 8.55；P =.0006），提示存在性别特异性风险，有待未来验证。对于3岁及以上儿童，诊断时的播散性疾病与较差的5年PFS显著相关，为39.2±9.7%（HR 2.88，95%置信区间1.52 - 5.45；P =.0012），5年OS为49.8±9.1%（HR 2.87，95%置信区间1.49 - 5.53；P =.0016）。
鉴于这种肿瘤的罕见性，前瞻性、协作性的国际研究对于提高这些患者的长期生存率至关重要。
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