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References for Chemotherapy for Tumors in the Nervous System of Children

This page was last updated on June 28th, 2022

  1. Allen JC: A phase II trial of preirradiation carboplatin in newly diagnosed germinoma of the central nervous system. Cancer 74: 940–944, 1994
  2. Sands SA, Oberg JA, Gardner SL, et al: Neuropsychological functioning of children treated with intensive chemotherapy followed by myeloablative consolidation chemotherapy and autologous hematopoietic cell rescue for newly diagnosed CNS tumors: an analysis of the Head Start II survivors. Pediatr Blood Cancer 54:429-436, 2010
  3. Ater JL, Zhou T, Holmes E, et al: Randomized study of two chemotherapy regimens for treatment of low-grade glioma in young children: a report from the Children’s Oncology Group . J Clin Oncol 30:2641–2647, 2012
  4. Bartels U, Laperriere N, Bouffet E, et al: Intracystic therapies for cystic craniopharyngioma in childhood. Front Endocrinol (Lausanne) 27:39, 2012
  5. Bouffet E, Jakacki R, Goldman S, et al: Phase II study of weekly vinblastine in recurrent or refractory pediatric low-grade glioma. J Clin Oncol 30:1358-1363, 2012
  6. Cavalheiro S, Dastoli PA, Silva NS, et al: Use of interferon alpha in intratumoral chemotherapy for cystic craniopharyngioma. Childs Nerv Syst 21:719-724, 2005
  7. 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 22:4881-4887, 2004
  8. Chintagumpala M, Hassall T, Palmer S, et al: A pilot study of risk-adapted radiotherapy and chemotherapy in patients with supratentorial PNET. Neuro Oncol 11:33-40, 2008
  9. Cohen BH, Packer RJ, Siegel KR, et al: Brain tumors in children under 2 years: treatment, survival and long-term prognosis. Pediatr Neurosurg 19:171-179, 1993
  10. Dean RL, Emerich DF, Hasler BP, et al: Cereport® (RMP-7) increases carboplatin levels in brain tumors after pretreatment with dexamethasone. Neuro Oncol 1: 268-274, 1999
  11. Dhall G, Grodman H, Ji L, et al: Outcome of children less than three years old at diagnosis with non-metastatic medulloblastoma treated with chemotherapy on the “Head Start” I and II protocols. Pediatr Blood Cancer 50:1169-75, 2008
  12. Dunkel IJ, Finlay JL: High-dose chemotherapy with autologous stem cell rescue for brain tumors. Crit Rev Oncol Hematol 41:197-204, review, 2002
  13. Ertel I, Boesal C : Adjuvant chemotherapy of high grade astrocytomas in children: Radiation therapy with or without CCNU, vincristine, and prednisone. Pediatr Neurosci 12:272-282, 1985-1986
  14. Evans AE, Jenkin RD, Sposto R, et al: The treatment of medulloblastoma. Results of a prospective randomized trial of radiation therapy with and without CCNU, vincristine, and prednisone. J Neurosurg 72:572-582, 1990
  15. Fangusaro J, Finlay J, Sposto R, et al: Intensive chemotherapy followed by consolidative myeloablative chemotherapy with autologous hematopoietic cell rescue (AuHCR) in young children with newly diagnosed supratentorial primitive neuroectodermal tumors (sPNETs): report of the Head Start I and II experience. Pediatr Blood Cancer 50:312-318, 2008
  16. Finlay JL, Dhall G, Boyett JM, et al: Myeloablative chemotherapy with autologous bone marrow rescue in children and adolescents with recurrent malignant astrocytoma: outcome compared with conventional chemotherapy: a report from the Children’s Oncology Group. Pediatr Blood Cancer 51:806-811, 2008
  17. Garvin JH, Selch MT, Holmes E, et al: Phase II study of pre-irradiation chemotherapy for childhood intracranial ependymoma. Children’s Cancer Group protocol 9942: A report from the Children’s Oncology Group. Pediatr Blood Cancer 59:1183-1189, 2012
  18. Geyer JR, Finlay JL, Boyett JM, et al: Survival of infants with malignant astrocytomas. A report from the Children’s Cancer Group. Cancer 75:1045-1050, 1995
  19. Grundy RG, Wilne SA, Weston CL, et al: Primary post-operative chemotherapy without radiotherapy for intracranial ependymoma in children: the UKCCSG/SIOP prospective study. Lancet Oncol 8:696-705, 2007
  20. Hegi ME, Diserens AC, Gorlia T, et al: MGMT gene silencing and benefit from temozolomide in glioblastoma. N Engl J Med 352:997-1003, 2005
  21. Hukin J, Siffert J, Velasquez L, et al: Leptomeningeal dissemination in children with progressive low-grade neuroepithelial tumors. Neuro Oncol 4:253-260, 2002
  22. Steinbok P, Hukin J: Intracystic treatments for craniopharyngioma. Neurosurg Focus 28 (4): E13, 2010
  23. Hukin J, Steinbok P, Lafay-Cousin L, et al: Intracystic bleomycin therapy for craniopharyngioma in children: the Canadian experience. Cancer 109:2124-2131, 2007
  24. Stewart CF, Iacono LC, Chintagumpala M, et al: Results of a phase II upfront window of pharmacokinetically guided topotecan in high-risk medulloblastoma and supratentorial primitive neuroectodermal tumor. J Clin Oncol 22:3357-3365, 2004
  25. Mason WP, Grovas A, Halpern S, et al: Intensive chemotherapy and bone marrow rescue for young children with newly diagnosed malignant brain tumors. J Clin Oncol 16:210-221, 1998
  26. Needle MN, Goldwein JW, Grass J, et al: Adjuvant chemotherapy for the treatment of intracranial ependymoma of childhood. Cancer 80:341-347, 1997
  27. Rutkowski S, Bode U, Deinlein F, et al: Treatment of early childhood medulloblastoma by postoperative chemotherapy alone. N Engl J Med 352:978-986, 2005
  28. Krueger DA, Care MM, Holland K, et al: Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis. N Engl J Med 363:1801-1811, 2010
  29. Jakacki RI, Burger PC, Zhou T, et al: Outcome of children with metastatic medulloblastoma treated with carboplatin during craniospinal radiotherapy: a Children’s Oncology Group Phase I/II study. J Clin Oncol 30:2648-2653, 2012
  30. Lafay-Cousin L, Hawkins C, Carret AS, et al: Central nervous system atypical teratoid rhabdoid tumors: the Canadian Paediatric Brain Tumour Consortium experience. Eur J Cancer 48:353-359, 2012
  31. Stupp R, Mason WP, van den Bent MJ, et al: Radiotherapy plus concomitant and adjuvant temozolomide for glioblastoma. N Engl J Med 352:987-996, 2005
  32. Liu AK, Macy ME, Foreman NK, et al: Bevacizumab as therapy for radiation necrosis in four children with pontine gliomas. Int J Radiation Oncology Biol Phys 75:1148–1154, 2009
  33. Linnebank M, Moskau S, Jurgens A, et al: Association of genetics variants of methionine metabolism with methotrexate-induced CNS white matter changes in patients with primary CNS lymphoma. Neuro Oncol 11:2-8, 2009
  34. Northcott PA, Dubuc AM, Pfister S, et al: Molecular subgroups of medulloblastoma. Expert Rev Neurother 12:871-884, 2012
  35. Packer RJ, Ater J, Allen J, et al: Carboplatin and vincristine chemotherapy for children with newly diagnosed progressive low-grade gliomas. Neurosurgery 86:747-754, 1997
  36. Packer RJ, Goldwein J, Nicholson HS, et al: Treatment of children with medulloblastoma with reduced-dose craniospinal radiation therapy and adjuvant chemotherapy: A Children’s Cancer Group Study. J Clin Oncol 17:2127-2136, 1999
  37. 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 24:4202-4208, 2006
  38. Pollack IF, Hamilton RL, Sobol RW, et al: O6-methylguanine-DNA methyltransferase expression strongly correlates with outcome in childhood malignant gliomas: results from the CCG-945 Cohort. J Clin Oncol 24:3431-3437, 2006
  39. Ross CJ, Katzov-Eckert H, Dubé MP, et al: Genetic variants in TPMT and COMT are associated with hearing loss in children receiving cisplatin chemotherapy. Nat Genet 41:1345-1349, 2009
  40. Sasazaki Y, Asami K, Utsumi J: Transient subacute encephalopathy induced by high-dose methotrexate treatment in children with acute lymphoblastic leukemia and malignant lymphoma. Gan To Kagaku Ryoho 19:1851-1857, 1992
  41. Strother D, Ashley D, Kellie SJ, et al: Feasibility of four consecutive high-dose chemotherapy cycles with stem-cell rescue for patients with newly diagnosed medulloblastoma or supratentorial primitive neuroectodermal tumor after craniospinal radiotherapy: results of a collaborative study. J Clin Oncol 19: 2696-2704, 2001
  42. Strunk T, Gottschalk S, Goepel W, et al: Subacute leukencephalopathy after low-dose intrathecal methotrexate in an adolescent heterozygous for the MTHFR C677T polymorphism. Med Pediatr Oncol 40:48-50, 2003
  43. Van den Bent MJ: Prevention of chemotherapy-induced neuropathy: leukemia inhibitory factor. Clin Cancer Res 11:1691-693, 2005
  44. Zacharoulis S, Levy A, Chi SN, et al: Outcome for young children newly diagnosed with ependymoma, treated with intensive induction chemotherapy followed by myeloablative chemotherapy and autologous stem cell rescue. Pediatr Blood Cancer 49:34-40, 2007
  45. Miesea F.R, Schusterb FR, Pierstorff K, et al: Magnetization transfer imaging provides no evidence of demyelination in methotrexate-induced encephalopathy. Am J Neuroradiol 32:E110 –E112, 2011
  46. Chi SN, Zimmerman MA, Yao X, et al: Intensive multimodality treatment for children with newly diagnosed CNS atypical teratoid rhabdoid tumor. J Clin Oncol 27:385-389, 2009
  47. Pollack IF, Jakacki RI, Butterfield LH, et al: Peptide vaccine therapy for childhood gliomas: interim results of a pilot study. Proceedings of the 103rd Annual Meeting of the American Association for Cancer Research. Philadelphia (PA): AACR. 2012, (Abstract LB131).
  48. Triscott J, Lee C, Foster C, et al: Personalizing the treatment of pediatric medulloblastoma: Polo-like kinase 1 as a molecular target in high-risk children. Cancer Res 73; 6734–6744, 2013
  49. Galanis E, Jaeckle KA, Maurer MJ, et al: Phase II trial of vorinostat in recurrent glioblastoma multiforme: a North Central Cancer Treatment Group study. J Clin Oncol 27: 2052-2058, 2009
  50. Li MH, Bouffet E, Hawkins CE, et al: Molecular genetics of supratentorial primitive neuroectodermal tumors and pineoblastoma. Neurosurg Focus 19 (5):E3, 2005
  51. Guidelines for platelet thresholds for pediatric hematology/oncology patients: Complete reference guide. Edmonton, CA: The C17 Guidelines Committee, 2011 ( [Accessed 25 January 2014]
  52. Comparison of radiation therapy regimens in combination with chemotherapy in treating young patients with newly diagnosed standard-risk medulloblastoma. Bethesda, MD: Children’s Oncology Group/National Cancer Institute, 2013 ( [Accessed 25 January 2014]
  53. Edwards M, Levin V, Wilson C: Brain tumor chemotherapy:An evaluation of agents in current use for phase II and III trials. Cancer Treat Rep 64:1179-1205, 1980.
  54. 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. Neurooncology April 1(2):152-161, 1999. 
  55. Massimino M, Spreafico F, Cefalo G et al : High Response Rate to Cisplatin/Etoposide Regimen in Childhood Low-Grade Glioma. Journal of Clinical Oncology, Vol 20, No 20 (October 15), 2002: pp 4209-4216
  56. Packer RJ, Jakacki R, Horn M et al: Objective Response of Multiply Recurrent Low-Grade Gliomas to Bevacizumab and Irinotecan. Pediatr Blood Cancer 2009;52:791–795
  57. Balmaceda C Heller G, Rosenblum M, et al:  Chemotherapy without irradiation–a novel approach for newly diagnosed CNS germ cell tumors: results of an international cooperative trial. The First International Central Nervous System Germ Cell Tumor Study.  J Clin Oncol 14:2908-2915, 1996
  58. Roberge D, Kun LE, Freeman CR: Intracranial germinoma: On whole ventricular irradiation. Pediatr Blood Cancer 44:358 –362, 2005
  59. Matsutani M; Japanese Pediatric Brain Tumor Study Group: Combined
    chemotherapy and radiation therapy for CNS germ cell tumors – the Japanese experience. J Neurooncol 2001;54:311–316, 2001
  60. Kellie SJ, Boyce H, Dunkel IJ, et al:  Primary chemotherapy for intracranial nongerminomatous germ cell tumors: Results of the Second International CNS Germ Cell Study Group Protocol.  J Clin Oncol 22:846-853,  2004
  61. Glass T, Cochrane DD, Rassekh SR, et al:  Growing teratoma syndrome in intracranial non-germinomatous germ cell tumors (iNGGCTs): a risk for secondary malignant transformation – a report of two cases.  Childs Nerv Syst Oct 12, 2013 (epub ahead of print)
  62. Kim JW, Kim WC, Cho JH, et al:  A multimodal approach including craniospinal irradiation improves the treatment outcome of high-risk intracranial nongerminomatous germ cell tumors.  Int J Radiat Oncol Biol Phys 84:625-631, 2012.
  63. Calaminus G, Kortmann R, Worch J, et al:  SIOP CNS GCT 96: final report of outcome of a prospective, multinational nonrandomized trial for children and adults with intracranial germinoma, comparing craniospinal irradiation alone with chemotherapy followed by focal primary site irradiation for patients with localized disease.  Neuro Oncol 15:788-796, 2013.
  64. Robertson PL, Jakacki R,  Hukin J, et al:  Multimodality therapy for CNS mixed malignant germ cell tumors (MMGCT): results of a phase II multi-institutional study.  J Neurooncol, published online 4 April, 2014

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