1.Ramaswamy, V, Remke, M, Bouffet, E, et al. Risk stratification of childhood medulloblastoma in the molecular era: the current consensus. Acta Neuropathol. 2016;131(6):821–31.
2.Polkinghorn, WR, Tarbell, NJ. Medulloblastoma: tumorigenesis, current clinical paradigm, and efforts to improve risk stratification. Nat Clin Pract Oncol. 2007;4:295–304.
3.Ramaswamy, V, Northcott, PA, Taylor, MD. FISH and chips: the recipe for improved prognostication and outcomes for children with medulloblastoma. Cancer Genet. 2011;204:577–88.
4.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;7:813–20.
5.Jones, DT, Jager, N, Kool, M, et al. Dissecting the genomic complexity underlying medulloblastoma. Nature. 2012;488:100–5.
6.Bautista, F, Fioravantti, V, de Rojas, T, et al. Medulloblastoma in children and adolescents: a systematic review of contemporary phase I and II clinical trials and biology update. Cancer Med. 2017;6:2606–24.
7.Gilman, AL, Jacobsen, C, Bunin, N, et al. Phase I study of tandem high-dose chemotherapy with autologous peripheral blood stem cell rescue for children with recurrent brain tumors: a Pediatric Blood and MarrowTransplant Consortium study. Pediatr Blood Cancer. 2011;57:506–13.
8.Kramer, K, Pandit-Taskar, N, Humm, JL, et al. A phase II study of radioimmunotherapy with intraventricular (131) I-3F8 for medulloblastoma. Pediatr Blood Cancer. 2018;65:1–10.
9.Akyuz, C, Demir, HA, Varan, A, Yalcin, B, Kutluk, T, Buyukpamukcu, M. Temozolomide in relapsed pediatric brain tumors: 14 cases from a single center. Childs Nerv Syst. 2012;28:111–5.
10.Massimino, M, Gandola, L, Spreafico, F, et al. No salvage using high-dose chemotherapy plus/minus reirradiation for relapsing previously irradiated medulloblastoma. Int J Radiat Oncol Biol Phys. 2009;73:1358–63.
11.De Sio, L, Milano, GM, Castellano, A, et al. Temozolomide in resistant or relapsed pediatric solid tumors. Pediatr Blood Cancer. 2006;47:30–6.
12.Bouffet, E, Doz, F, Demaille, MC, et al. Improving survival in recurrent medulloblastoma: earlier detection, better treatment or still an impasse? Br J Cancer. 1998;77:1321–6.
13.Balter-Seri, J, Mor, C, Shuper, A, Zaizov, R, Cohen, IJ. Cure of recurrent medulloblastoma: the contribution of surgical resection at relapse. Cancer. 1997;79:1241–7.
14.Chamberlain, MC, Kormanik, PA. Chronic oral VP-16 for recurrent medulloblastoma. Pediatr Neurol. 1997;17:230–4.
15.Crafts, DC, Levin, VA, Edwards, MS, Pischer, TL, Wilson, CB. Chemotherapy of recurrent medulloblastoma with combined procarbazine, CCNU, and vincristine. J Neurosurg. 1978;49:589–92.
16.Baruchel, S, Sharp, JR, Bartels, U, et al. A Canadian paediatric brain tumour consortium (CPBTC) phase II molecularly targeted study of imatinib in recurrent and refractory paediatric central nervous system tumours. Eur J Cancer. 2009;45:2352–9.
17.Beutler, D, Avoledo, P, Reubi, JC, et al. Three-year recurrence-free survival in a patient with recurrent medulloblastoma after resection, high-dose chemotherapy, and intrathecal Yttrium-90-labeled DOTA0-D-Phe1-Tyr3-octreotide radiopeptide brachytherapy. Cancer. 2005;103:869–73.
18.Taylor, MD, Northcott, PA, Korshunov, A, et al. Molecular subgroups of medulloblastoma: the current consensus. Acta Neuropathol. 2012;123:465–72.
19.Cho, YJ, Tsherniak, A, Tamayo, P, et al. Integrative genomic analysis of medulloblastoma identifies a molecular subgroup that drives poor clinical outcome. J Clin Oncol. 2011;29:1424–30.
20.Ramaswamy, V, Remke, M, Bouffet, E, et al. Recurrence patterns across medulloblastoma subgroups: an integrated clinical and molecular analysis. Lancet Oncol. 2013;14:1200–7.
21.ClinicalTrials.gov [Internet]. Reduced craniospinal radiation therapy and chemotherapy in treating younger patients with newly diagnosed WNT-driven medulloblastoma, 18th ed. Bethesda, MD: National Library of Medicine (US); 2018.
22.ClinicalTrials.gov [Internet]. A clinical and molecular risk-directed therapy for newly diagnosed medulloblastoma. Bethseda, MD: National Library of Medicine (US); 2019.
23.ClinicalTrials.gov [Internet]. A study assessing the feasibility of a surgery and chemotherapy-only in children with Wnt positive medulloblastoma. Bethseda, MD: National Library of Medicine; 2018.
24.Schwalbe, EC, Lindsey, JC, Nakjang, S, et al. Novel molecular subgroups for clinical classification and outcome prediction in childhood medulloblastoma: a cohort study. Lancet Oncol. 2017;18:958–71.
25.Robinson, GW, Rudneva, VA, Buchhalter, I, et al. Risk-adapted therapy for young children with medulloblastoma (SJYC07): therapeutic and molecular outcomes from a multicentre, phase 2 trial. Lancet Oncol. 2018;19:768–84.
26.Kool, M, Korshunov, A, Remke, M, et al. Molecular subgroups of medulloblastoma: an international meta-analysis of transcriptome, genetic aberrations, and clinical data of WNT, SHH, Group 3, and Group 4 medulloblastomas. Acta Neuropathol. 2012;123:473–84.
27.Northcott, PA, Shih, DJ, Remke, M, et al. Rapid, reliable, and reproducible molecular sub-grouping of clinical medulloblastoma samples. Acta Neuropathol. 2012;123:615–26.
28.IBM Corp. IBM SPSS statistics for windows, Version 25.0. Armonk, NY: IBM Corp.; Released 2017.
29.Kopecky, AS, Khan, AJ, Pan, W, Drachtman, R, Parikh, RR. Outcomes and patterns of care in a nationwide cohort of pediatric medulloblastoma: factors affecting proton therapy utilization. Adv Radiat Oncol. 2017;2:588–96.
30.Stensvold, E, Krossnes, BK, Lundar, T, et al. Outcome for children treated for medulloblastoma and supratentorial primitive neuroectodermal tumor (CNS-PNET) – a retrospective analysis spanning 40 years of treatment. Acta Oncol. 2017;56:698–705.
31.Rickert, CH, Paulus, W. Epidemiology of central nervous system tumors in childhood and adolescence based on the new WHO classification. Childs Nerv Syst. 2001;17:503–11.
32.Koschmann, C, Bloom, K, Upadhyaya, S, Geyer, JR, Leary, SE. Survival after relapse of medulloblastoma. J Pediatr Hematol Oncol. 2016;38:269–73.
33.Dhellemmes, P, Demaille, MC, Lejeune, JP, Baranzelli, MC, Combelles, G, Torrealba, G. Cerebellar medulloblastoma: results of multidisciplinary treatment. Report of 120 cases. Surg Neurol. 1986;25:290–94.
34.Johnston, DL, Keene, D, Strother, D, et al. Survival following tumor recurrence in children with medulloblastoma. J Pediatr Hematol Oncol. 2018;40(3):e159–63.
35.Pietsch, T, Schmidt, R, Remke, M, et al. Prognostic significance of clinical, histopathological, and molecular characteristics of medulloblastomas in the prospective HIT2000 multicenter clinical trial cohort. Acta Neuropathol. 2014;128:137–49.
36.Ellison, DW, Kocak, M, Dalton, J, et al. Definition of disease-risk stratification groups in childhood medulloblastoma using combined clinical, pathologic, and molecular variables. J Clin Oncol. 2011;29:1400–7.
37.Chan, MY, Teo, WY, Seow, WT, Tan, AM. Epidemiology, management and treatment outcome of medulloblastoma in Singapore. Ann Acad Med Singapore. 2007;36:314–18.
38.Wetmore, C, Herington, D, Lin, T, Onar-Thomas, A, Gajjar, A, Merchant, TE. Reirradiation of recurrent medulloblastoma: does clinical benefit outweigh risk for toxicity? Cancer. 2014;120:3731–37.
39.Gottardo, NG, Hansford, JR, McGlade, JP, et al. Medulloblastoma down under 2013: a report from the third annual meeting of the International Medulloblastoma Working Group. Acta Neuropathol. 2014;127:189–201.
40.Louis, DN, Perry, A, Reifenberger, G, et al. The 2016 world health organization classification of tumors of the central nervous system: a summary. Acta Neuropathol. 2016;131:803–20.
41.Wang, X, Dubuc, AM, Ramaswamy, V, et al. Medulloblastoma subgroups remain stable across primary and metastatic compartments. Acta Neuropathol. 2015;129:449–57.
42.Hill, RM, Kuijper, S, Lindsey, JC, et al. Combined MYC and P53 defects emerge at medulloblastoma relapse and define rapidly progressive, therapeutically targetable disease. Cancer Cell. 2015;27:72–84.
43.Morrissy, AS, Garzia, L, Shih, DJ, et al. Divergent clonal selection dominates medulloblastoma at recurrence. Nature. 2016;529:351–57.
44.Friedrich, C, von Bueren, AO, von Hoff, K, et al. Treatment of young children with CNS-primitive neuroectodermal tumors/pineoblastomas in the prospective multicenter trial HIT 2000 using different chemotherapy regimens and radiotherapy. Neuro Oncol. 2013;15:224–34.
45.Taylor, MD, Liu, L, Raffel, C, et al. Mutations in SUFU predispose to medulloblastoma. Nat Genet. 2002;31:306–10.
46.Kieran, MW, Chisholm, J, Casanova, M, et al. Phase I study of oral sonidegib (LDE225) in pediatric brain and solid tumors and a phase II study in children and adults with relapsed medulloblastoma. Neuro Oncol. 2017;19:1542–52.
47.Graham, RTConley, S, Finlay, JL, AbdelBack, MS. MBCL-06. Successful salvage of desmoplastic nodular medulloblastoma patients treated on ACNS1211. Neurooncol. 2018;20:1.
48.Guerrini-Rousseau, L, Dufour, C, Varlet, P, et al. Germline SUFU mutation carriers and medulloblastoma: clinical characteristics, cancer risk, and prognosis. Neuro Oncol. 2018;20:1122–32.
49.Lafay-Cousin, L, Bouffet, E, Onar-Thomas, A, Billups, CA, Hawkins, C, Eberhart, C, Horbinski, C, Robinson, GW, Strother, DR, Heier, L, Souweidane, MM, Fouladi, M, Gajjar, A, Children Oncology Group. ACNS1221: a phase II study for the treatment of non metastatic desmoplastic medulloblastoma in children less than 4 years of age – a report of the Children Oncology Group. Journal of Clinical Oncology. 2017;35:10505–5.