Yan Han, Zabih Veda, Bartels Ute, Das Sunit, Nathan Paul, Gupta Sumit
Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada.
Division of Hematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada.
Neurooncol Adv. 2022 Feb 9;4(1):vdac016. doi: 10.1093/noajnl/vdac016. eCollection 2022 Jan-Dec.
Medulloblastoma is a rare diagnosis among adolescents and young adults (AYA). Though prognostic factors and treatment are well characterized among children with medulloblastoma, equivalent data for AYA are sparse. We conducted a systematic review to identify predictors of survival among AYA with medulloblastoma.
We searched for primary studies of AYA (age 15-39 at diagnosis) with medulloblastoma in high-income countries within OVID MEDLINE, EMBASE, and EBM Reviews-Cochrane library databases from inception to August 2020. Patient demographics, primary outcomes, and univariate and multivariable data on all prognostic factors were collected from included studies. Prognosticators were characterized as patient, disease, or treatment-related.
We identified 18 articles. 5-year overall survival ranged between 40% and 89%, while disease-free survival ranged from 49% to 89%. Study quality was low as assessed by the Quality in Prognostic factor Studies tool. Though meta-analyses were not possible due heterogeneity, narrative summaries suggested that lower disease burden, superior postoperative functional status, and higher doses and larger fields of radiation were associated with improved survival. Reported chemotherapy regimens were heterogeneous in timing, agents, and relationship with radiation, precluding meaningful comparisons. Only one study included molecular subgroups for analysis, with the majority (76.5%) of tumors classified as Sonic Hedgehog (SHH).
Prognostication and treatment of AYA medulloblastoma is limited by a dearth of primary evidence and lack of specificity for patients aged 15-39. Dedicated prospective trials to delineate the benefit of various chemotherapy and radiation regimens are required in this population to identify prognosticators and ideal treatment regimens.
髓母细胞瘤在青少年和青年（AYA）中是一种罕见的诊断。尽管髓母细胞瘤患儿的预后因素和治疗方法已得到充分描述，但关于AYA的等效数据却很稀少。我们进行了一项系统综述，以确定AYA髓母细胞瘤患者生存的预测因素。
我们在OVID MEDLINE、EMBASE和循证医学评论 - 考克兰图书馆数据库中，检索了从数据库建立至2020年8月期间高收入国家中诊断年龄为15 - 39岁的AYA髓母细胞瘤的原始研究。从纳入研究中收集患者人口统计学资料、主要结局以及所有预后因素的单变量和多变量数据。将预后因素分为患者、疾病或治疗相关因素。
我们识别出18篇文章。5年总生存率在40%至89%之间，无病生存率在49%至89%之间。根据预后因素研究质量工具评估，研究质量较低。尽管由于异质性无法进行荟萃分析，但叙述性总结表明，较低的疾病负担、较好的术后功能状态以及更高剂量和更大照射野与生存率提高相关。报道的化疗方案在时间、药物以及与放疗的关系方面存在异质性，无法进行有意义的比较。只有一项研究纳入分子亚组进行分析，大多数（76.5%）肿瘤被分类为音猬因子（SHH）型。
AYA髓母细胞瘤的预后评估和治疗受到主要证据不足以及对15 - 39岁患者缺乏特异性的限制。该人群需要专门的前瞻性试验来描述各种化疗和放疗方案的益处，以确定预后因素和理想的治疗方案。
