生存
标准风险患者的 5 年无进展生存比例约为 79% 至 83%。[20]Gajjar A, Chintagumpala M, Ashley D, et al. Risk-adapted craniospinal radiotherapy followed by high-dose chemotherapy and stem-cell rescue in children with newly diagnosed medulloblastoma (St Jude Medulloblastoma-96): long-term results from a prospective, multicentre trial. Lancet Oncol. 2006 Oct;7(10):813-20.http://www.ncbi.nlm.nih.gov/pubmed/17012043?tool=bestpractice.com[59]Packer RJ, Goldwein J, Nicholson HS, et al. Treatment of children with medulloblastomas with reduced-dose craniospinal radiation therapy and adjuvant chemotherapy: A Children's Cancer Group Study. J Clin Oncol. 1999 Jul;17(7):2127-36.http://www.ncbi.nlm.nih.gov/pubmed/10561268?tool=bestpractice.com[60]Packer RJ, Gajjar A, Vezina G, et al. Phase III study of craniospinal radiation therapy followed by adjuvant chemotherapy for newly diagnosed average-risk medulloblastoma. J Clin Oncol. 2006 Sep 1;24(25):4202-8.http://www.ncbi.nlm.nih.gov/pubmed/16943538?tool=bestpractice.com 但是高风险患者仅为 50%。[20]Gajjar A, Chintagumpala M, Ashley D, et al. Risk-adapted craniospinal radiotherapy followed by high-dose chemotherapy and stem-cell rescue in children with newly diagnosed medulloblastoma (St Jude Medulloblastoma-96): long-term results from a prospective, multicentre trial. Lancet Oncol. 2006 Oct;7(10):813-20.http://www.ncbi.nlm.nih.gov/pubmed/17012043?tool=bestpractice.com[37]Zeltzer PM, Boyett JM, Finlay JL, et al. Metastasis stage, adjuvant treatment, and residual tumor are prognostic factors for medulloblastoma in children: conclusions from the Children's Cancer Group 921 randomized phase III study. J Clin Oncol. 1999 Mar;17(3):832-45.http://www.ncbi.nlm.nih.gov/pubmed/10071274?tool=bestpractice.com[61]Packer RJ, Sutton LN, Elterman R, et al. Outcome for children with medulloblastoma treated with radiation and cisplatin, CCNU, and vincristine chemotherapy. J Neurosurg. 1994 Nov;81(5):690-8.http://www.ncbi.nlm.nih.gov/pubmed/7931615?tool=bestpractice.com[62]Miralbell R, Fitzgerald TJ, Laurie F, et al. Radiotherapy in pediatric medulloblastoma: quality assessment of Pediatric Oncology Group Trial 9031. Int J Radiat Oncol Biol Phys. 2006 Apr 1;64(5):1325-30.http://www.ncbi.nlm.nih.gov/pubmed/16413699?tool=bestpractice.com 对于婴儿,组织学数据提示 5 年无进展生存范围为 20% 至 40%,但是促结缔织增生亚型婴儿的疗效显著改善。[39]Geyer JR, Sposto R, Jennings M, et al. Multiagent chemotherapy and deferred radiotherapy in infants with malignant brain tumors: a report from the Children's Cancer Group. J Clin Oncol. 2005 Oct 20;23(30):7621-31.http://ascopubs.org/doi/full/10.1200/jco.2005.09.095http://www.ncbi.nlm.nih.gov/pubmed/16234523?tool=bestpractice.com[40]Duffner PK, Horowitz ME, Krischer JP, et al. The treatment of malignant brain tumors in infants and very young children: an update of the Pediatric Oncology Group experience. Neuro Oncol. 1999 Apr;1(2):152-61.http://www.ncbi.nlm.nih.gov/pubmed/11554387?tool=bestpractice.com[41]Grill J, Sainte-Rose C, Jouvet A, et al. Treatment of medulloblastoma with postoperative chemotherapy alone: an SFOP prospective trial in young children. Lancet Oncol. 2005 Aug;6(8):573-80.http://www.ncbi.nlm.nih.gov/pubmed/16054568?tool=bestpractice.com 一些研究提出了较为乐观的结论,脑室内应用甲氨蝶呤,或者大剂量化疗联合干细胞挽救可提高婴儿患者的生存率。应谨慎解释这些近期试验,因为样本量小,随访间期短,并且(在某些病例中)研究群体倾向于纳入有预后良好特征的患者,其数目比预期多。[42]Rutkowski S, Bode U, Deinlein F, et al. Treatment of early childhood medulloblastoma by postoperative chemotherapy alone. N Engl J Med. 2005 Mar 10;352(10):978-86.http://www.ncbi.nlm.nih.gov/pubmed/15758008?tool=bestpractice.com[44]Chi SN, Gardner SL, Levy AS, et al. Feasibility and response to induction chemotherapy intensified with high-dose methotrexate for young children with newly diagnosed high-risk disseminated medulloblastoma. J Clin Oncol. 2004 Dec 15;22(24):4881-7.http://ascopubs.org/doi/full/10.1200/jco.2004.12.126http://www.ncbi.nlm.nih.gov/pubmed/15611503?tool=bestpractice.com[45]Sung KW, Yoo KH, Cho EJ, et al. High-dose chemotherapy and autologous stem cell rescue in children with newly diagnosed high-risk or relapsed medulloblastoma or supratentorial primitive neuroectodermal tumor. Pediatr Blood Cancer. 2007 Apr;48(4):408-15.http://www.ncbi.nlm.nih.gov/pubmed/17066462?tool=bestpractice.com
预后因素
较差的预后因素包括年龄<3 岁,存在转移,[63]Rutkowski S, von Hoff K, Emser A, et al. Survival and prognostic factors of early childhood medulloblastoma: an international meta-analysis. J Clin Oncol. 2010 Nov 20;28(33):4961-8.http://ascopubs.org/doi/full/10.1200/jco.2010.30.2299http://www.ncbi.nlm.nih.gov/pubmed/20940197?tool=bestpractice.com 术后有至少 1.5 cm² 的肿瘤残留。大细胞表型代表肿瘤预后不良。[63]Rutkowski S, von Hoff K, Emser A, et al. Survival and prognostic factors of early childhood medulloblastoma: an international meta-analysis. J Clin Oncol. 2010 Nov 20;28(33):4961-8.http://ascopubs.org/doi/full/10.1200/jco.2010.30.2299http://www.ncbi.nlm.nih.gov/pubmed/20940197?tool=bestpractice.com 有力的证据提示 MYC 癌基因扩增(n-myc 或 c-myc)[17]Grotzer MA, Hogarty MD, Janss AJ, et al. MYC messenger RNA expression predicts survival outcome in childhood primitive neuroectodermal tumor/medulloblastoma. Clin Cancer Res. 2001 Aug;7(8):2425-33.http://www.ncbi.nlm.nih.gov/pubmed/11489822?tool=bestpractice.com[18]Lamont JM, McManamy CS, Pearson AD, et al. Combined histopathological and molecular cytogenetic stratification of medulloblastoma patients. Clin Cancer Res. 2004 Aug 15;10(16):5482-93.http://clincancerres.aacrjournals.org/content/10/16/5482.longhttp://www.ncbi.nlm.nih.gov/pubmed/15328187?tool=bestpractice.com 染色体 17p 缺失,[19]Batra SK, McLendon RE, Koo JS, et al. Prognostic implications of chromosome 17p deletions in human medulloblastomas. J Neurooncol. 1995;24(1):39-45.http://www.ncbi.nlm.nih.gov/pubmed/8523074?tool=bestpractice.com 与不良结局有关。β连环蛋白的状态证明Wnt/Wg通道激活作用似乎与特殊的良好临床表现相关。[20]Gajjar A, Chintagumpala M, Ashley D, et al. Risk-adapted craniospinal radiotherapy followed by high-dose chemotherapy and stem-cell rescue in children with newly diagnosed medulloblastoma (St Jude Medulloblastoma-96): long-term results from a prospective, multicentre trial. Lancet Oncol. 2006 Oct;7(10):813-20.http://www.ncbi.nlm.nih.gov/pubmed/17012043?tool=bestpractice.com[64]Ellison DW, Onilude OE, Lindsey JC, et al. beta-Catenin status predicts a favorable outcome in childhood medulloblastoma: the United Kingdom Children's Cancer Study Group Brain Tumour Committee. J Clin Oncol. 2005 Nov 1;23(31):7951-7.http://www.ncbi.nlm.nih.gov/pubmed/16258095?tool=bestpractice.com 尚未清楚确诊时间是否为一个预后因素,虽然一项研究提示症状出现至诊断的时间间隔与转移性疾病风险、生存率之间不存在显著关系。[65]Gerber NU, von Hoff K, von Bueren AO, et al. A long duration of the prediagnostic symptomatic interval is not associated with an unfavourable prognosis in childhood medulloblastoma. Eur J Cancer. 2012 Sep;48(13):2028-36.http://www.ncbi.nlm.nih.gov/pubmed/22153558?tool=bestpractice.com
复发
已经接受放疗的患者复发髓母细胞瘤的疗效较差,救治率为<10%,不论使用何种治疗方法。 已使用了多重化疗方案,但是罕见持久应答。 非弥漫转移的复发病例,可能通过多模式治疗,包括手术和局部病灶再放疗进行救治。