Malignant solid neoplasm, SMO-mutated or PTCH-1-mutated (Hedgehog)
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Note: this is a new type of page for HemOnc.org, as most other regimen pages are tissue-specific. We will collect published information from basket trials on these pages, as well as regimens that are FDA approved.
Vismodegib monotherapy
Regimen
| Study | Evidence |
|---|---|
| Hainsworth et al. 2018 (MyPathway) | Phase IIa |
Eligibility criteria
- SMO p.W535L or other nonsynonymous mutations, or PTCH-1 loss-of-function mutations
Chemotherapy
- Vismodegib (Erivedge) 150 mg PO once per day
Continued until progression or intolerance
References
- MyPathway: Hainsworth JD, Meric-Bernstam F, Swanton C, Hurwitz H, Spigel DR, Sweeney C, Burris H, Bose R, Yoo B, Stein A, Beattie M, Kurzrock R. Targeted therapy for advanced solid tumors on the basis of molecular profiles: results from MyPathway, an open-label, phase IIa multiple basket study. J Clin Oncol. 2018 Feb 20;36(6):536-542. Epub 2018 Jan 10. link to original article contains verified protocol PubMed