The DOI matches its Crossref record; 33 of the 33 references it cites were found in a registry or an index.
Vismodegib Exerts Targeted Efficacy Against Recurrent Sonic Hedgehog–Subgroup Medulloblastoma: Results From Phase II Pediatric Brain Tumor Consortium Studies PBTC-025B and PBTC-032
Open accessAbstract
Purpose: Two phase II studies assessed the efficacy of vismodegib, a sonic hedgehog (SHH) pathway inhibitor that binds smoothened (SMO), in pediatric and adult recurrent medulloblastoma (MB). Patients and Methods: Adult patients enrolled onto PBTC-025B and pediatric patients enrolled onto PBTC-032 were treated with vismodegib (150 to 300 mg/d). Protocol-defined response, which had to be sustained for 8 weeks, was confirmed by central neuroimaging review. Molecular tests to identify patterns of response and insensitivity were performed when tissue was available. Results: A total of 31 patients were enrolled onto PBTC-025B, and 12 were enrolled onto PBTC-032. Three patients in PBTC-025B and one in PBTC-032, all with SHH-subgroup MB (SHH-MB), exhibited protocol-defined responses. Progression-free survival (PFS) was longer in those with SHH-MB than in those with non-SHH–MB, and prolonged disease stabilization occurred in 41% of patient cases of SHH-MB. Among those with SHH-MB, loss of heterozygosity of PTCH1 was associated with prolonged PFS, and diffuse staining of P53 was associated with reduced PFS. Whole-exome sequencing identified mutations in SHH genes downstream from SMO in four of four tissue samples from nonresponders and upstream of SMO in two of four patients with favorable responses. Conclusion: Vismodegib exhibits activity against adult recurrent SHH-MB but not against recurrent non-SHH–MB. Inadequate accrual of pediatric patients precluded conclusions in this population. Molecular analyses support the hypothesis that SMO inhibitor activity depends on the genomic aberrations within the tumor. Such inhibitors should be advanced in SHH-MB studies; however, molecular and genomic work remains imperative to identify target populations that will truly benefit.
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Cites
Molecular subgroups of medulloblastoma: the current consensus
Taylor, M. D., Northcott, P. A., Korshunov, A., Remke, M., Cho, Y.-J., Clifford, S. C., Eberhart, C. G., Parsons, D. W., Rutkowski, S., Gajjar, A., et al. (2012) · Acta Neuropathologica · doi:10.1007/s00401-011-0922-z · Cited by 1,936
Self-citationMedulloblastoma Comprises Four Distinct Molecular Variants
Northcott, P. A., Korshunov, A., Witt, H., Hielscher, T., Eberhart, C. G., Mack, S., Bouffet, E., Clifford, S. C., Hawkins, C. E., French, P., et al. (2011) · Journal of Clinical Oncology · doi:10.1200/jco.2009.27.4324 · Cited by 1,339
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Self-citationTreatment of Medulloblastoma with Hedgehog Pathway Inhibitor GDC-0449
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Subgroup-specific structural variation across 1,000 medulloblastoma genomes
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Self-citationRausch, T., Jones, D. T. W., Zapatka, M., Stütz, A. M., Zichner, T., Weischenfeldt, J., Jäger, N., Remke, M., Shih, D., Northcott, P. A., et al. (2012) · Cell · doi:10.1016/j.cell.2011.12.013 · Cited by 865
Novel mutations target distinct subgroups of medulloblastoma
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Self-citationDissecting the genomic complexity underlying medulloblastoma
Jones, D. T. W., Jäger, N., Kool, M., Zichner, T., Hutter, B., Sultan, M., Cho, Y.-J., Pugh, T. J., Hovestadt, V., Stütz, A. M., et al. (2012) · Nature · doi:10.1038/nature11284 · Cited by 839
Smoothened Mutation Confers Resistance to a Hedgehog Pathway Inhibitor in Medulloblastoma
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Genome Sequencing of SHH Medulloblastoma Predicts Genotype-Related Response to Smoothened Inhibition
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Cited by
Risk stratification of childhood medulloblastoma in the molecular era: the current consensus
Ramaswamy, V., Remke, M., Bouffet, E., Bailey, S., Clifford, S. C., Doz, F., Kool, M., Dufour, C., Vassal, G., Milde, T., et al. (2016) · Acta Neuropathologica · doi:10.1007/s00401-016-1569-6 · Cited by 654
Self-citationTargeting Cancer Stemness in the Clinic: From Hype to Hope
Saygin, C., Matei, D., Majeti, R., Reizes, O., & Lathia, J. D. (2019) · Cell Stem Cell · doi:10.1016/j.stem.2018.11.017 · Cited by 517
Medulloblastomics revisited: biological and clinical insights from thousands of patients
Hovestadt, V., Ayrault, O., Swartling, F. J., Robinson, G. W., Pfister, S. M., & Northcott, P. A. (2020) · Nature Reviews Cancer · doi:10.1038/s41568-019-0223-8 · Cited by 271
Self-citationMedulloblastoma: From Molecular Subgroups to Molecular Targeted Therapies
Wang, J., Garancher, A., Ramaswamy, V., & Wechsler-Reya, R. J. (2018) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-070815-013838 · Cited by 113
Role of imaging in the era of precision medicine
Giardino, A., Gupta, S., Olson, E., Sepulveda, K., Lenchik, L., Ivanidze, J., Rakow-Penner, R., Patel, M. J., Subramaniam, R. M., & Ganeshan, D. (2017) · Academic Radiology · doi:10.1016/j.acra.2016.11.021 · Cited by 87
Metadata
- Authors
- Robinson, G. W., Orr, B. A., Wu, G., Gururangan, S., Lin, T., Qaddoumi, I., Packer, R. J., Goldman, S., Prados, M. D., Desjardins, A., Chintagumpala, M., Takebe, N., Kaste, S. C., Rusch, M., Allen, S. J., Onar-Thomas, A., Stewart, C. F., Fouladi, M., Boyett, J. M., Gilbertson, R. J., Curran, T., Ellison, D. W., & Gajjar, A.
- Venue
- Journal of Clinical Oncology
- Published
- 2015-08-20
- Volume
- 33
- Issue
- 24
- Pages
- 2646-2654
- ISSN
- 0732-183X (ISSN-L)
- 1527-7755
- OpenAlex
- W2111433192
- Cited by
- 448
- References
- 33
- FWCI
- 18.40
- Type
- journal article
- Language
- en
- Open access
- bronze
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- CoChat. (2026). Citation check: Vismodegib Exerts Targeted Efficacy Against Recurrent Sonic Hedgehog–Subgroup Medulloblastoma: Results From Phase II Pediatric Brain Tumor Consortium Studies PBTC-025B and PBTC-032. https://app.cochat.ai/papers/10.1200/jco.2014.60.1591
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- Robinson, G. W., Orr, B. A., Wu, G., Gururangan, S., Lin, T., Qaddoumi, I., Packer, R. J., Goldman, S., Prados, M. D., Desjardins, A., Chintagumpala, M., Takebe, N., Kaste, S. C., Rusch, M., Allen, S. J., Onar-Thomas, A., Stewart, C. F., Fouladi, M., Boyett, J. M., . . . Gajjar, A. (2015). Vismodegib Exerts Targeted Efficacy Against Recurrent Sonic Hedgehog–Subgroup Medulloblastoma: Results From Phase II Pediatric Brain Tumor Consortium Studies PBTC-025B and PBTC-032. Journal of Clinical Oncology. https://doi.org/10.1200/jco.2014.60.1591
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