Dabrafenib as a diagnostic and therapeutic strategy for the non-surgical management of papillary craniopharyngioma Journal Article
| Authors: | Lin, A. L.; Tabar, V.; Young, R. J.; Geer, E. B. |
| Article Title: | Dabrafenib as a diagnostic and therapeutic strategy for the non-surgical management of papillary craniopharyngioma |
| Abstract: | Purpose: Papillary craniopharyngiomas can cause considerable morbidity due to mass effect and potential surgical complications. These tumors are known to harbor BRAF V600 mutations, which make them exquisitely sensitive to BRAF inhibitors. Methods: The patient is a 59 year old man with a progressive suprasellar lesion that was radiographically consistent with a papillary craniopharyngioma. He was consented to an Institution Review Board-approved protocol, which permits sequencing of cell free DNA in plasma and the collection and reporting of clinical data. Results: The patient declined surgical resection and was empirically treated with dabrafenib at 150 mg twice daily. Treatment response was demonstrated after 19 days, confirming the diagnosis. After achieving a near complete response after 6.5 months on drug, a decision was made to deescalate treatment to dabrafenib 75 mg twice daily with subsequent tumor stability for 2.5 months. Conclusion: Patients with a suspected papillary craniopharyngioma can be challenged with dabrafenib as a potentially effective diagnostic and therapeutic strategy, given that rapid regression with dabrafenib is only observed in tumors harboring a BRAF V600 mutation. Further work is needed to explore the optimal regimen and dose of the targeted therapy. © 2023, The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature. |
| Keywords: | adult; clinical article; middle aged; gene mutation; genetics; mutation; case report; drug withdrawal; nuclear magnetic resonance imaging; follow up; computer assisted tomography; tumor regression; pathology; fever; heart palpitation; targeted therapy; headache; craniopharyngioma; optic nerve; tumor growth; b raf kinase; optic chiasm; proto-oncogene proteins b-raf; hypophysis tumor; pituitary neoplasms; heart atrium flutter; dabrafenib; trametinib; braf inhibitor; high throughput sequencing; humans; human; male; article; circulating free dna; papillary craniopharyngioma |
| Journal Title: | Pituitary |
| Volume: | 26 |
| Issue: | 4 |
| ISSN: | 1386-341X |
| Publisher: | Springer |
| Date Published: | 2023-08-01 |
| Start Page: | 482 |
| End Page: | 487 |
| Language: | English |
| DOI: | 10.1007/s11102-023-01339-y |
| PUBMED: | 37428397 |
| PROVIDER: | scopus |
| PMCID: | PMC10766168 |
| DOI/URL: | |
| Notes: | The MSK Cancer Center Support Grant (P30 CA008748) is acknowledged in the PubMed record and PDF. Corresponding MSK author is Andrew L. Lin -- Source: Scopus |
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