A 10-year-long ongoing complete response to vismodegib as first-line treatment in adult metastatic medulloblastoma

Mduma E, Awuor A, Lugina EL. Adult medulloblastoma: a case report. J Med Case Rep. 2022;16:330. DOI: https://doi.org/10.1186/s13256-022-03531-3

Fang FY, Rosenblum JS, Ho WS, Heiss JD. New developments in the pathogenesis, therapeutic targeting, and treatment of pediatric medulloblastoma. Cancers (Basel). 2022;14:2285. DOI: https://doi.org/10.3390/cancers14092285

Juraschka K, Taylor MD. Medulloblastoma in the age of molecular subgroups: a review. J Neurosurg Pediatr. 2019;24:353–63. DOI: https://doi.org/10.3171/2019.5.PEDS18381

Franceschi E, Hofer S, Brandes AA, Frappaz D, Kortmann RD, Bromberg J, et al. EANO-EURACAN clinical practice guideline for diagnosis, treatment, and follow-up of post-pubertal and adult patients with medulloblastoma. Lancet Oncol. 2019;20:e715–28. DOI: https://doi.org/10.1016/S1470-2045(19)30669-2

Majd N, Penas-Prado M. Updates on management of adult medulloblastoma. Curr Treat Options Oncol. 2019;20:64. DOI: https://doi.org/10.1007/s11864-019-0663-0

Gatto L, Franceschi E, Tosoni A, Di Nunno V, Bartolini S, Brandes AA. Molecular targeted therapies: time for a paradigm shift in medulloblastoma treatment? Cancers (Basel). 2022;14:333. DOI: https://doi.org/10.3390/cancers14020333

Kian W, Roisman LC, Goldstein IM, Abo-Quider A, Samueli B, Wallach N, et al. Vismodegib as first-line treatment of mutated sonic hedgehog pathway in adult medulloblastoma. JCO Precis Oncol. 2020;4:PO.19.00264. DOI: https://doi.org/10.1200/PO.19.00264

Gould SE, Low JA, Marsters JC, Jr., Robarge K, Rubin LL, de Sauvage FJ, et al. Discovery and preclinical development of vismodegib. Exp Opin Drug Discov. 2014;9:969–84. DOI: https://doi.org/10.1517/17460441.2014.920816

Jamieson C, Martinelli G, Papayannidis C, Cortes JE. Hedgehog pathway inhibitors: a new therapeutic class for the treatment of acute myeloid leukemia. Blood Cancer Discov. 2020;1:134–45. DOI: https://doi.org/10.1158/2643-3230.BCD-20-0007

Dagklis A, Demeyer S, De Bie J, Radaelli E, Pauwels D, Degryse S, et al. Hedgehog pathway activation in T-cell acute lymphoblastic leukemia predicts response to SMO and GLI1 inhibitors. Blood. 2016;128:2642–54. DOI: https://doi.org/10.1182/blood-2016-03-703454

Li Y, Song Q, Day BW. Phase I and phase II sonidegib and vismodegib clinical trials for the treatment of paediatric and adult MB patients: a systemic review and meta-analysis. Acta Neuropathol Commun. 2019;7:123. DOI: https://doi.org/10.1186/s40478-019-0773-8

Garcia EP, Minkovsky A, Jia Y, Ducar MD, Shivdasani P, Gong X, et al. Validation of OncoPanel: a targeted next-generation sequencing assay for the detection of somatic variants in cancer. Arch Pathol Lab Med. 2017;141:751–8. DOI: https://doi.org/10.5858/arpa.2016-0527-OA

Asklund T, Henriksson R, Axelsson J, Bergström Å, Kasper M, Ögren M, et al. Early and persisting response to vismodegib in a patient with bone metastasizing medulloblastoma. Acta Oncol. 2013;52:862–6. DOI: https://doi.org/10.3109/0284186X.2012.724537

Rudin CM, Hann CL, Laterra J, Yauch RL, Callahan CA, Fu L, et al. Treatment of medulloblastoma with hedgehog pathway inhibitor GDC-0449. N Engl J Med. 2009;361:1173–8. DOI: https://doi.org/10.1056/NEJMoa0902903

Climans SA, Macdonald DR, Sutherland DE, Mason WP. Prolonged response to vismodegib in a patient with systemic medulloblastoma metastases. BMJ Case Rep. 2020;13(10):e236406. DOI: https://doi.org/10.1136/bcr-2020-236406

Lou E, Schomaker M, Wilson JD, Ahrens M, Dolan M, Nelson AC. Complete and sustained response of adult medulloblastoma to first-line sonic hedgehog inhibition with vismodegib. Cancer Biol Ther. 2016;17:1010–16. DOI: https://doi.org/10.1080/15384047.2016.1220453

Frappaz D, Barritault M, Montané L, Laigle-Donadey F, Chinot O, Le Rhun E, et al. MEVITEM-a phase I/II trial of vismodegib + temozolomide vs temozolomide in patients with recurrent/refractory medulloblastoma with Sonic Hedgehog pathway activation. Neuro Oncol. 2021;23:1949–60. DOI: https://doi.org/10.1093/neuonc/noab087

Robinson GW, Orr BA, Wu G, Gururangan S, Lin T, Qaddoumi I, et al. Vismodegib exerts targeted efficacy against recurrent sonic hedgehog-subgroup medulloblastoma: results from phase II pediatric brain tumor consortium studies PBTC-025B and PBTC-032. J Clin Oncol. 2015;33:2646–54. DOI: https://doi.org/10.1200/JCO.2014.60.1591