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Refractory Medulloblastoma

MedGen UID:
1642493
•Concept ID:
C4684869
•
Neoplastic Process

Definition

Medulloblastoma that does not respond to treatment. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVRefractory Medulloblastoma

Professional guidelines

PubMed

Salvage therapy for progressive, treatment-refractory or recurrent pediatric medulloblastoma: a systematic review protocol.
Adile AA, Kameda-Smith MM, Bakhshinyan D, Banfield L, Salim SK, Farrokhyar F, Fleming AJ
Syst Rev 2020 Mar 4;9(1):47. doi: 10.1186/s13643-020-01307-8. PMID: 32127049Free PMC Article
Molecular stratifications, biomarker candidates and new therapeutic options in current medulloblastoma treatment approaches.
Menyhárt O, Győrffy B
Cancer Metastasis Rev 2020 Mar;39(1):211-233. doi: 10.1007/s10555-020-09854-1. PMID: 31970590Free PMC Article
Platinum compounds in children with cancer: toxicity and clinical management.
Ruggiero A, Trombatore G, Triarico S, Arena R, Ferrara P, Scalzone M, Pierri F, Riccardi R
Anticancer Drugs 2013 Nov;24(10):1007-19. doi: 10.1097/CAD.0b013e3283650bda. PMID: 23962902

Recent clinical studies

Etiology

Medulloblastoma: an Old Diagnosis with New Promises.
Szalontay L, Khakoo Y
Curr Oncol Rep 2020 Jul 9;22(9):90. doi: 10.1007/s11912-020-00953-4. PMID: 32643099
Phase II study of temozolomide and topotecan (TOTEM) in children with relapsed or refractory extracranial and central nervous system tumors including medulloblastoma with post hoc Bayesian analysis: A European ITCC study.
Le Teuff G, Castaneda-Heredia A, Dufour C, Jaspan T, Calmon R, Devos A, McHugh K, Leblond P, Frappaz D, Aerts I, Zwaan CM, Ducassou S, Chastagner P, Verschuur A, Corradini N, Casanova M, Rubie H, Riccardi R, Le Deley MC, Vassal G, Geoerger B; European consortium Innovative Therapies for Children with Cancer (ITCC)
Pediatr Blood Cancer 2020 Jan;67(1):e28032. Epub 2019 Oct 8 doi: 10.1002/pbc.28032. PMID: 31595663
Phase II study of irinotecan in combination with temozolomide (TEMIRI) in children with recurrent or refractory medulloblastoma: a joint ITCC and SIOPE brain tumor study.
Grill J, Geoerger B, Gesner L, Perek D, Leblond P, Cañete A, Aerts I, Madero L, de Toledo Codina JS, Verlooy J, Estlin E, Cisar L, Breazna A, Dorman A, Bailey S, Nicolin G, Grundy RG, Hargrave D; European Consortium Innovative Therapies for Children with Cancer (ITCC) and the European Society for Paediatric Oncology (SIOPE) brain tumor group
Neuro Oncol 2013 Sep;15(9):1236-43. Epub 2013 Jul 14 doi: 10.1093/neuonc/not097. PMID: 23857707Free PMC Article
Safety and toxicity of intrathecal liposomal cytarabine (Depocyte) in children and adolescents with recurrent or refractory brain tumors: a multi-institutional retrospective study.
Benesch M, Siegler N, Hoff Kv, Lassay L, Kropshofer G, Müller H, Sommer C, Rutkowski S, Fleischhack G, Urban C
Anticancer Drugs 2009 Oct;20(9):794-9. doi: 10.1097/CAD.0b013e32832f4abe. PMID: 19617818
A phase II study of the farnesyl transferase inhibitor, tipifarnib, in children with recurrent or progressive high-grade glioma, medulloblastoma/primitive neuroectodermal tumor, or brainstem glioma: a Children's Oncology Group study.
Fouladi M, Nicholson HS, Zhou T, Laningham F, Helton KJ, Holmes E, Cohen K, Speights RA, Wright J, Pollack IF; Children's Oncology Group
Cancer 2007 Dec 1;110(11):2535-41. doi: 10.1002/cncr.23078. PMID: 17932894

Diagnosis

Sustained Survival Benefit in Recurrent Medulloblastoma by a Metronomic Antiangiogenic Regimen: A Nonrandomized Controlled Trial.
Peyrl A, Chocholous M, Sabel M, Lassaletta A, Sterba J, Leblond P, Nysom K, Torsvik I, Chi SN, Perwein T, Jones N, Holm S, Nyman P, Mörse H, Öberg A, Weiler-Wichtl L, Leiss U, Haberler C, Schmook MT, Mayr L, Dieckmann K, Kool M, Gojo J, Azizi AA, André N, Kieran M, Slavc I
JAMA Oncol 2023 Dec 1;9(12):1688-1695. doi: 10.1001/jamaoncol.2023.4437. PMID: 37883081Free PMC Article
MEVITEM-a phase I/II trial of vismodegib + temozolomide vs temozolomide in patients with recurrent/refractory medulloblastoma with Sonic Hedgehog pathway activation.
Frappaz D, Barritault M, Montané L, Laigle-Donadey F, Chinot O, Le Rhun E, Bonneville-Levard A, Hottinger AF, Meyronnet D, Bidaux AS, Garin G, Pérol D
Neuro Oncol 2021 Nov 2;23(11):1949-1960. doi: 10.1093/neuonc/noab087. PMID: 33825892Free PMC Article
Medulloblastoma: an Old Diagnosis with New Promises.
Szalontay L, Khakoo Y
Curr Oncol Rep 2020 Jul 9;22(9):90. doi: 10.1007/s11912-020-00953-4. PMID: 32643099
Safety and toxicity of intrathecal liposomal cytarabine (Depocyte) in children and adolescents with recurrent or refractory brain tumors: a multi-institutional retrospective study.
Benesch M, Siegler N, Hoff Kv, Lassay L, Kropshofer G, Müller H, Sommer C, Rutkowski S, Fleischhack G, Urban C
Anticancer Drugs 2009 Oct;20(9):794-9. doi: 10.1097/CAD.0b013e32832f4abe. PMID: 19617818
Activity of fotemustine in medulloblastoma and malignant glioma xenografts in relation to O6-alkylguanine-DNA alkyltransferase and alkylpurine-DNA N-glycosylase activity.
Vassal G, Boland I, Terrier-Lacombe MJ, Watson AJ, Margison GP, Vénuat AM, Morizet J, Parker F, Lacroix C, Lellouch-Tubiana A, Pierre-Kahn A, Poullain MG, Gouyette A
Clin Cancer Res 1998 Feb;4(2):463-8. PMID: 9516937

Therapy

Sustained Survival Benefit in Recurrent Medulloblastoma by a Metronomic Antiangiogenic Regimen: A Nonrandomized Controlled Trial.
Peyrl A, Chocholous M, Sabel M, Lassaletta A, Sterba J, Leblond P, Nysom K, Torsvik I, Chi SN, Perwein T, Jones N, Holm S, Nyman P, Mörse H, Öberg A, Weiler-Wichtl L, Leiss U, Haberler C, Schmook MT, Mayr L, Dieckmann K, Kool M, Gojo J, Azizi AA, André N, Kieran M, Slavc I
JAMA Oncol 2023 Dec 1;9(12):1688-1695. doi: 10.1001/jamaoncol.2023.4437. PMID: 37883081Free PMC Article
Medulloblastoma: an Old Diagnosis with New Promises.
Szalontay L, Khakoo Y
Curr Oncol Rep 2020 Jul 9;22(9):90. doi: 10.1007/s11912-020-00953-4. PMID: 32643099
Phase II study of temozolomide and topotecan (TOTEM) in children with relapsed or refractory extracranial and central nervous system tumors including medulloblastoma with post hoc Bayesian analysis: A European ITCC study.
Le Teuff G, Castaneda-Heredia A, Dufour C, Jaspan T, Calmon R, Devos A, McHugh K, Leblond P, Frappaz D, Aerts I, Zwaan CM, Ducassou S, Chastagner P, Verschuur A, Corradini N, Casanova M, Rubie H, Riccardi R, Le Deley MC, Vassal G, Geoerger B; European consortium Innovative Therapies for Children with Cancer (ITCC)
Pediatr Blood Cancer 2020 Jan;67(1):e28032. Epub 2019 Oct 8 doi: 10.1002/pbc.28032. PMID: 31595663
Phase II study of irinotecan in combination with temozolomide (TEMIRI) in children with recurrent or refractory medulloblastoma: a joint ITCC and SIOPE brain tumor study.
Grill J, Geoerger B, Gesner L, Perek D, Leblond P, Cañete A, Aerts I, Madero L, de Toledo Codina JS, Verlooy J, Estlin E, Cisar L, Breazna A, Dorman A, Bailey S, Nicolin G, Grundy RG, Hargrave D; European Consortium Innovative Therapies for Children with Cancer (ITCC) and the European Society for Paediatric Oncology (SIOPE) brain tumor group
Neuro Oncol 2013 Sep;15(9):1236-43. Epub 2013 Jul 14 doi: 10.1093/neuonc/not097. PMID: 23857707Free PMC Article
Bevacizumab and irinotecan in the treatment of children with recurrent/refractory medulloblastoma.
Aguilera DG, Goldman S, Fangusaro J
Pediatr Blood Cancer 2011 Mar;56(3):491-4. doi: 10.1002/pbc.22868. PMID: 21072819

Prognosis

Sustained Survival Benefit in Recurrent Medulloblastoma by a Metronomic Antiangiogenic Regimen: A Nonrandomized Controlled Trial.
Peyrl A, Chocholous M, Sabel M, Lassaletta A, Sterba J, Leblond P, Nysom K, Torsvik I, Chi SN, Perwein T, Jones N, Holm S, Nyman P, Mörse H, Öberg A, Weiler-Wichtl L, Leiss U, Haberler C, Schmook MT, Mayr L, Dieckmann K, Kool M, Gojo J, Azizi AA, André N, Kieran M, Slavc I
JAMA Oncol 2023 Dec 1;9(12):1688-1695. doi: 10.1001/jamaoncol.2023.4437. PMID: 37883081Free PMC Article
MEVITEM-a phase I/II trial of vismodegib + temozolomide vs temozolomide in patients with recurrent/refractory medulloblastoma with Sonic Hedgehog pathway activation.
Frappaz D, Barritault M, Montané L, Laigle-Donadey F, Chinot O, Le Rhun E, Bonneville-Levard A, Hottinger AF, Meyronnet D, Bidaux AS, Garin G, Pérol D
Neuro Oncol 2021 Nov 2;23(11):1949-1960. doi: 10.1093/neuonc/noab087. PMID: 33825892Free PMC Article
Medulloblastoma: an Old Diagnosis with New Promises.
Szalontay L, Khakoo Y
Curr Oncol Rep 2020 Jul 9;22(9):90. doi: 10.1007/s11912-020-00953-4. PMID: 32643099
Phase II study of temozolomide and topotecan (TOTEM) in children with relapsed or refractory extracranial and central nervous system tumors including medulloblastoma with post hoc Bayesian analysis: A European ITCC study.
Le Teuff G, Castaneda-Heredia A, Dufour C, Jaspan T, Calmon R, Devos A, McHugh K, Leblond P, Frappaz D, Aerts I, Zwaan CM, Ducassou S, Chastagner P, Verschuur A, Corradini N, Casanova M, Rubie H, Riccardi R, Le Deley MC, Vassal G, Geoerger B; European consortium Innovative Therapies for Children with Cancer (ITCC)
Pediatr Blood Cancer 2020 Jan;67(1):e28032. Epub 2019 Oct 8 doi: 10.1002/pbc.28032. PMID: 31595663
Activity of fotemustine in medulloblastoma and malignant glioma xenografts in relation to O6-alkylguanine-DNA alkyltransferase and alkylpurine-DNA N-glycosylase activity.
Vassal G, Boland I, Terrier-Lacombe MJ, Watson AJ, Margison GP, Vénuat AM, Morizet J, Parker F, Lacroix C, Lellouch-Tubiana A, Pierre-Kahn A, Poullain MG, Gouyette A
Clin Cancer Res 1998 Feb;4(2):463-8. PMID: 9516937

Clinical prediction guides

MEVITEM-a phase I/II trial of vismodegib + temozolomide vs temozolomide in patients with recurrent/refractory medulloblastoma with Sonic Hedgehog pathway activation.
Frappaz D, Barritault M, Montané L, Laigle-Donadey F, Chinot O, Le Rhun E, Bonneville-Levard A, Hottinger AF, Meyronnet D, Bidaux AS, Garin G, Pérol D
Neuro Oncol 2021 Nov 2;23(11):1949-1960. doi: 10.1093/neuonc/noab087. PMID: 33825892Free PMC Article
Phase II study of temozolomide and topotecan (TOTEM) in children with relapsed or refractory extracranial and central nervous system tumors including medulloblastoma with post hoc Bayesian analysis: A European ITCC study.
Le Teuff G, Castaneda-Heredia A, Dufour C, Jaspan T, Calmon R, Devos A, McHugh K, Leblond P, Frappaz D, Aerts I, Zwaan CM, Ducassou S, Chastagner P, Verschuur A, Corradini N, Casanova M, Rubie H, Riccardi R, Le Deley MC, Vassal G, Geoerger B; European consortium Innovative Therapies for Children with Cancer (ITCC)
Pediatr Blood Cancer 2020 Jan;67(1):e28032. Epub 2019 Oct 8 doi: 10.1002/pbc.28032. PMID: 31595663
Safety and toxicity of intrathecal liposomal cytarabine (Depocyte) in children and adolescents with recurrent or refractory brain tumors: a multi-institutional retrospective study.
Benesch M, Siegler N, Hoff Kv, Lassay L, Kropshofer G, Müller H, Sommer C, Rutkowski S, Fleischhack G, Urban C
Anticancer Drugs 2009 Oct;20(9):794-9. doi: 10.1097/CAD.0b013e32832f4abe. PMID: 19617818
A phase II study of the farnesyl transferase inhibitor, tipifarnib, in children with recurrent or progressive high-grade glioma, medulloblastoma/primitive neuroectodermal tumor, or brainstem glioma: a Children's Oncology Group study.
Fouladi M, Nicholson HS, Zhou T, Laningham F, Helton KJ, Holmes E, Cohen K, Speights RA, Wright J, Pollack IF; Children's Oncology Group
Cancer 2007 Dec 1;110(11):2535-41. doi: 10.1002/cncr.23078. PMID: 17932894
Activity of fotemustine in medulloblastoma and malignant glioma xenografts in relation to O6-alkylguanine-DNA alkyltransferase and alkylpurine-DNA N-glycosylase activity.
Vassal G, Boland I, Terrier-Lacombe MJ, Watson AJ, Margison GP, Vénuat AM, Morizet J, Parker F, Lacroix C, Lellouch-Tubiana A, Pierre-Kahn A, Poullain MG, Gouyette A
Clin Cancer Res 1998 Feb;4(2):463-8. PMID: 9516937

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