Department of Pediatrics, Division of Hematology-Oncology, University of California San Diego, La Jolla, California, USA (M.R.P., P.E.Z.); Peckham Center for Cancer and Blood Disorders, Rady Children's Hospital-San Diego, San Diego, California, USA.
Expert Rev Anticancer Ther. 2021 Sep;21(9):957-974. doi: 10.1080/14737140.2021.1932472. Epub 2021 Jun 8.
: Medulloblastoma, an embryonal small round blue cell tumor primarily arising in the posterior fossa, is the most common malignancy of the central nervous system in children and requires intensive multi-modality therapy for cure. Overall 5-year survival is approximately 75% in children with primary disease, but outcomes for relapsed disease are very poor. Recent advances have identified molecular subgroups with excellent prognosis, with 5-year overall survival rates >90%, and subgroups with very poor prognosis with overall survival rates <50%. Molecular subtyping has allowed for more sophisticated risk stratification of patients, but new treatments for the highest risk patients have not yet improved outcomes. Targeting cancer stem cells may improve outcomes, and several candidate targets and novel drugs are under investigation.: We discuss medulloblastoma epidemiology, biology, treatment modalities, risk stratification, and molecular subgroup analysis, links between subgroup and developmental biology, cancer stem cell biology in medulloblastoma including previously described cancer stem cell markers and proposed targeted treatments in the current literature.: The understanding of cancer stem cells in medulloblastoma will advance therapies targeting the most treatment-resistant cells within the tumor and therefore reduce the incidence of treatment refractory and relapsed disease.
: 髓母细胞瘤,一种主要起源于后颅窝的胚胎性小圆细胞蓝细胞瘤,是儿童中枢神经系统最常见的恶性肿瘤,需要强化的多模式治疗才能治愈。原发性疾病患儿的总体 5 年生存率约为 75%,但复发疾病的预后非常差。最近的进展确定了具有良好预后的分子亚群,5 年总生存率>90%,而预后非常差的亚群总生存率<50%。分子分型使得对患者进行更复杂的风险分层成为可能,但针对最高风险患者的新治疗方法尚未改善预后。针对癌症干细胞可能会改善预后,目前正在研究几种候选靶点和新型药物。: 我们讨论了髓母细胞瘤的流行病学、生物学、治疗方式、风险分层和分子亚组分析,以及亚组与发育生物学之间的联系,以及髓母细胞瘤中癌症干细胞生物学,包括先前描述的癌症干细胞标志物和目前文献中提出的靶向治疗。: 对髓母细胞瘤中癌症干细胞的理解将推进针对肿瘤内最具治疗抵抗性细胞的治疗方法,从而降低治疗抵抗和复发疾病的发生率。
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