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Untreated Childhood Medulloblastoma

MedGen UID:
852504
•Concept ID:
C0338341
•
Finding

Definition

A finding of medulloblastoma in childhood that has not been treated. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVUntreated Childhood Medulloblastoma

Professional guidelines

PubMed

Assessment of Puberty and Hypothalamic-Pituitary-Gonadal Axis Function After Childhood Brain Tumor Treatment.
Rosimont M, Kariyawasam D, Samara-Boustani D, Giani E, Beltrand J, Bolle S, Fresneau B, Puget S, Sainte-Rose C, Alapetite C, Pinto G, Touraine P, Piketty ML, Brabant S, Abbou S, Aerts I, Beccaria K, Bourgeois M, Roujeau T, Blauwblomme T, Rocco FD, Thalassinos C, Rigaud C, James S, Busiah K, Simon A, Bourdeaut F, Lemelle L, Guerrini-Rousseau L, Orbach D, Doz F, Dufour C, Grill J, Polak M, Briceño LG
J Clin Endocrinol Metab 2023 Aug 18;108(9):e823-e831. doi: 10.1210/clinem/dgad097. PMID: 36810692
Combination of Ribociclib with BET-Bromodomain and PI3K/mTOR Inhibitors for Medulloblastoma Treatment In Vitro and In Vivo.
Jonchere B, Williams J, Zindy F, Liu J, Robinson S, Farmer DM, Min J, Yang L, Stripay JL, Wang Y, Freeman BB, Yu J, Shelat AA, Rankovic Z, Roussel MF
Mol Cancer Ther 2023 Jan 3;22(1):37-51. doi: 10.1158/1535-7163.MCT-21-0896. PMID: 36318650Free PMC Article
Combination of Ribociclib and Gemcitabine for the Treatment of Medulloblastoma.
Pribnow A, Jonchere B, Liu J, Smith KS, Campagne O, Xu K, Robinson S, Patel Y, Onar-Thomas A, Wu G, Stewart CF, Northcott PA, Yu J, Robinson GW, Roussel MF
Mol Cancer Ther 2022 Aug 2;21(8):1306-1317. doi: 10.1158/1535-7163.MCT-21-0598. PMID: 35709750Free PMC Article

Recent clinical studies

Etiology

Dynamic profiling of medulloblastoma surfaceome.
Bakhshinyan D, Suk Y, Kuhlmann L, Adile AA, Ignatchenko V, Custers S, Gwynne WD, Macklin A, Venugopal C, Kislinger T, Singh SK
Acta Neuropathol Commun 2023 Jul 10;11(1):111. doi: 10.1186/s40478-023-01609-7. PMID: 37430373Free PMC Article
Assessment of Puberty and Hypothalamic-Pituitary-Gonadal Axis Function After Childhood Brain Tumor Treatment.
Rosimont M, Kariyawasam D, Samara-Boustani D, Giani E, Beltrand J, Bolle S, Fresneau B, Puget S, Sainte-Rose C, Alapetite C, Pinto G, Touraine P, Piketty ML, Brabant S, Abbou S, Aerts I, Beccaria K, Bourgeois M, Roujeau T, Blauwblomme T, Rocco FD, Thalassinos C, Rigaud C, James S, Busiah K, Simon A, Bourdeaut F, Lemelle L, Guerrini-Rousseau L, Orbach D, Doz F, Dufour C, Grill J, Polak M, Briceño LG
J Clin Endocrinol Metab 2023 Aug 18;108(9):e823-e831. doi: 10.1210/clinem/dgad097. PMID: 36810692
Evaluation of an EZH2 inhibitor in patient-derived orthotopic xenograft models of pediatric brain tumors alone and in combination with chemo- and radiation therapies.
Qi L, Lindsay H, Kogiso M, Du Y, Braun FK, Zhang H, Guo L, Zhao S, Injac SG, Baxter PA, Su JM, Xiao S, Erickson SW, Earley EJ, Teicher B, Smith MA, Li XN
Lab Invest 2022 Feb;102(2):185-193. Epub 2021 Nov 20 doi: 10.1038/s41374-021-00700-8. PMID: 34802040Free PMC Article
Effectiveness of rhGH treatment on adult height in GH-deficient childhood survivors of medulloblastoma.
Ciaccio M, Gil S, Guercio G, Vaiani E, Alderete D, Palladino M, Warman DM, Rivarola MA, Belgorosky A
Horm Res Paediatr 2010;73(4):281-6. Epub 2010 Mar 9 doi: 10.1159/000284393. PMID: 20215775
Infratentorial pediatric brain tumors: the value of new imaging modalities.
Schneider JF, Viola A, Confort-Gouny S, Ayunts K, Le Fur Y, Viout P, Bennathan M, Chapon F, Figarella-Branger D, Cozzone P, Girard N
J Neuroradiol 2007 Mar;34(1):49-58. doi: 10.1016/j.neurad.2007.01.010. PMID: 17316798

Diagnosis

Assessment of Puberty and Hypothalamic-Pituitary-Gonadal Axis Function After Childhood Brain Tumor Treatment.
Rosimont M, Kariyawasam D, Samara-Boustani D, Giani E, Beltrand J, Bolle S, Fresneau B, Puget S, Sainte-Rose C, Alapetite C, Pinto G, Touraine P, Piketty ML, Brabant S, Abbou S, Aerts I, Beccaria K, Bourgeois M, Roujeau T, Blauwblomme T, Rocco FD, Thalassinos C, Rigaud C, James S, Busiah K, Simon A, Bourdeaut F, Lemelle L, Guerrini-Rousseau L, Orbach D, Doz F, Dufour C, Grill J, Polak M, Briceño LG
J Clin Endocrinol Metab 2023 Aug 18;108(9):e823-e831. doi: 10.1210/clinem/dgad097. PMID: 36810692
Nifurtimox Is Effective Against Neural Tumor Cells and Is Synergistic with Buthionine Sulfoximine.
Du M, Zhang L, Scorsone KA, Woodfield SE, Zage PE
Sci Rep 2016 Jun 10;6:27458. doi: 10.1038/srep27458. PMID: 27282514Free PMC Article
Identification of pediatric brain neoplasms using Raman spectroscopy.
Leslie DG, Kast RE, Poulik JM, Rabah R, Sood S, Auner GW, Klein MD
Pediatr Neurosurg 2012;48(2):109-17. Epub 2012 Nov 15 doi: 10.1159/000343285. PMID: 23154646
Identification and characterisation of childhood cerebellar tumours by in vivo proton MRS.
Davies NP, Wilson M, Harris LM, Natarajan K, Lateef S, Macpherson L, Sgouros S, Grundy RG, Arvanitis TN, Peet AC
NMR Biomed 2008 Oct;21(8):908-18. doi: 10.1002/nbm.1283. PMID: 18613254
Preclinical development of camptothecin derivatives and clinical trials in pediatric oncology.
Vassal G, Pondarré C, Boland I, Cappelli C, Santos A, Thomas C, Lucchi E, Imadalou K, Pein F, Morizet J, Gouyette A
Biochimie 1998 Mar;80(3):271-80. doi: 10.1016/s0300-9084(98)80009-6. PMID: 9615866

Therapy

Combination of Ribociclib and Gemcitabine for the Treatment of Medulloblastoma.
Pribnow A, Jonchere B, Liu J, Smith KS, Campagne O, Xu K, Robinson S, Patel Y, Onar-Thomas A, Wu G, Stewart CF, Northcott PA, Yu J, Robinson GW, Roussel MF
Mol Cancer Ther 2022 Aug 2;21(8):1306-1317. doi: 10.1158/1535-7163.MCT-21-0598. PMID: 35709750Free PMC Article
Evaluation of an EZH2 inhibitor in patient-derived orthotopic xenograft models of pediatric brain tumors alone and in combination with chemo- and radiation therapies.
Qi L, Lindsay H, Kogiso M, Du Y, Braun FK, Zhang H, Guo L, Zhao S, Injac SG, Baxter PA, Su JM, Xiao S, Erickson SW, Earley EJ, Teicher B, Smith MA, Li XN
Lab Invest 2022 Feb;102(2):185-193. Epub 2021 Nov 20 doi: 10.1038/s41374-021-00700-8. PMID: 34802040Free PMC Article
scRNA-seq in medulloblastoma shows cellular heterogeneity and lineage expansion support resistance to SHH inhibitor therapy.
Ocasio JK, Babcock B, Malawsky D, Weir SJ, Loo L, Simon JM, Zylka MJ, Hwang D, Dismuke T, Sokolsky M, Rosen EP, Vibhakar R, Zhang J, Saulnier O, Vladoiu M, El-Hamamy I, Stein LD, Taylor MD, Smith KS, Northcott PA, Colaneri A, Wilhelmsen K, Gershon TR
Nat Commun 2019 Dec 20;10(1):5829. doi: 10.1038/s41467-019-13657-6. PMID: 31863004Free PMC Article
Effectiveness of rhGH treatment on adult height in GH-deficient childhood survivors of medulloblastoma.
Ciaccio M, Gil S, Guercio G, Vaiani E, Alderete D, Palladino M, Warman DM, Rivarola MA, Belgorosky A
Horm Res Paediatr 2010;73(4):281-6. Epub 2010 Mar 9 doi: 10.1159/000284393. PMID: 20215775
Preclinical development of camptothecin derivatives and clinical trials in pediatric oncology.
Vassal G, Pondarré C, Boland I, Cappelli C, Santos A, Thomas C, Lucchi E, Imadalou K, Pein F, Morizet J, Gouyette A
Biochimie 1998 Mar;80(3):271-80. doi: 10.1016/s0300-9084(98)80009-6. PMID: 9615866

Prognosis

Apparent diffusion and fractional anisotropy of diffuse intrinsic brain stem gliomas.
Chen HJ, Panigrahy A, Dhall G, Finlay JL, Nelson MD Jr, Blüml S
AJNR Am J Neuroradiol 2010 Nov;31(10):1879-85. Epub 2010 Jul 1 doi: 10.3174/ajnr.A2179. PMID: 20595371Free PMC Article
Multiparametric differentiation of posterior fossa tumors in children using diffusion-weighted imaging and short echo-time 1H-MR spectroscopy.
Schneider JF, Confort-Gouny S, Viola A, Le Fur Y, Viout P, Bennathan M, Chapon F, Figarella-Branger D, Cozzone P, Girard N
J Magn Reson Imaging 2007 Dec;26(6):1390-8. doi: 10.1002/jmri.21185. PMID: 17968955
Valproic Acid prolongs survival time of severe combined immunodeficient mice bearing intracerebellar orthotopic medulloblastoma xenografts.
Shu Q, Antalffy B, Su JM, Adesina A, Ou CN, Pietsch T, Blaney SM, Lau CC, Li XN
Clin Cancer Res 2006 Aug 1;12(15):4687-94. doi: 10.1158/1078-0432.CCR-05-2849. PMID: 16899619
Preclinical development of camptothecin derivatives and clinical trials in pediatric oncology.
Vassal G, Pondarré C, Boland I, Cappelli C, Santos A, Thomas C, Lucchi E, Imadalou K, Pein F, Morizet J, Gouyette A
Biochimie 1998 Mar;80(3):271-80. doi: 10.1016/s0300-9084(98)80009-6. PMID: 9615866
Preradiation high-dose intravenous methotrexate with leucovorin rescue for untreated primary childhood brain tumors.
Allen JC, Walker R, Rosen G
J Clin Oncol 1988 Apr;6(4):649-53. doi: 10.1200/JCO.1988.6.4.649. PMID: 3258630

Clinical prediction guides

Single-fraction Radiation Treatment Dose Response in a Genetically Engineered Mouse Model of Medulloblastoma.
Tu KJ, Stewart CE, Williams NT, Ma Y, Luo L, Ghosh D, Weidenhammer LB, Floyd SR, Fan Y, Kirsch DG, Oldham M, Reitman ZJ
Radiat Res 2023 Dec 1;200(6):587-592. doi: 10.1667/RADE-23-00126.1. PMID: 37990957Free PMC Article
Systemic considerations with pigmented fundus lesions and retinal pigment epithelium hamartomas in Turcot syndrome.
Mokhashi N, Cai LZ, Shields CL, Benson WE, Ho AC
Curr Opin Ophthalmol 2021 Nov 1;32(6):567-573. doi: 10.1097/ICU.0000000000000798. PMID: 34456292
Apparent diffusion and fractional anisotropy of diffuse intrinsic brain stem gliomas.
Chen HJ, Panigrahy A, Dhall G, Finlay JL, Nelson MD Jr, Blüml S
AJNR Am J Neuroradiol 2010 Nov;31(10):1879-85. Epub 2010 Jul 1 doi: 10.3174/ajnr.A2179. PMID: 20595371Free PMC Article
Additive effects of 5-aza-2'-deoxycytidine and irradiation on clonogenic survival of human medulloblastoma cell lines.
Patties I, Jahns J, Hildebrandt G, Kortmann RD, Glasow A
Strahlenther Onkol 2009 May;185(5):331-8. Epub 2009 May 15 doi: 10.1007/s00066-009-1956-1. PMID: 19440673
Preclinical development of camptothecin derivatives and clinical trials in pediatric oncology.
Vassal G, Pondarré C, Boland I, Cappelli C, Santos A, Thomas C, Lucchi E, Imadalou K, Pein F, Morizet J, Gouyette A
Biochimie 1998 Mar;80(3):271-80. doi: 10.1016/s0300-9084(98)80009-6. PMID: 9615866

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