BRAF–MEK Inhibition in Newly Diagnosed Papillary Craniopharyngiomas

Genetic analysis has shown the presence of activating BRAF mutations in 94% of papillary craniopharyngiomas. A phase 2 study showed partial response or better in 15 of 16 previously untreated patients.

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Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2023-07, Vol.389 (2), p.118-126
Hauptverfasser:	Brastianos, Priscilla K., Twohy, Erin, Geyer, Susan, Gerstner, Elizabeth R., Kaufmann, Timothy J., Tabrizi, Shervin, Kabat, Brian, Thierauf, Julia, Ruff, Michael W., Bota, Daniela A., Reardon, David A., Cohen, Adam L., De La Fuente, Macarena I., Lesser, Glenn J., Campian, Jian, Agarwalla, Pankaj K., Kumthekar, Priya, Mann, Bhupinder, Vora, Shivangi, Knopp, Michael, Iafrate, A. John, Curry, William T., Cahill, Daniel P., Shih, Helen A., Brown, Paul D., Santagata, Sandro, Barker, Fred G., Galanis, Evanthia
Format:	Artikel
Sprache:	eng
Schlagworte:	Adverse events Antineoplastic Agents - adverse effects Antineoplastic Agents - therapeutic use Brain cancer Brain Tumor Brain tumors Clinical significance Complications Confidence intervals Craniopharyngioma - drug therapy Craniopharyngioma - genetics Creatine Creatine kinase Disease Progression Enrollments Genotyping Hematology Humans Hyperglycemia Hypothalamus MEK inhibitors Mitogen-Activated Protein Kinase Kinases - antagonists & inhibitors Mitogen-Activated Protein Kinase Kinases - genetics Morbidity Mutation Neurology Neurosurgery Oncology Patients Pituitary Pituitary Neoplasms - drug therapy Pituitary Neoplasms - genetics Proto-Oncogene Proteins B-raf - antagonists & inhibitors Proto-Oncogene Proteins B-raf - genetics Radiation therapy Remission Induction Surgery Treatments in Oncology Tumors Vemurafenib - adverse effects Vemurafenib - therapeutic use
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Beschreibung
Zusammenfassung:	Genetic analysis has shown the presence of activating BRAF mutations in 94% of papillary craniopharyngiomas. A phase 2 study showed partial response or better in 15 of 16 previously untreated patients.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa2213329