Trial reportThe New England journal of medicine2023
BRAF-MEK Inhibition in Newly Diagnosed Papillary Craniopharyngiomas.
Registry-linked trialOpen access · greenAbstract readClinical Trial, Phase II
In one paragraph
Trial report in The New England journal of medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03224767 (Phase II Trial of BRAF/MEK Inhibitors in Papillary Craniopharyngiomas), which is not on this map. Cited by 84 papers, 7 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
84citing papers in PubMed, 7 pooled it
25.5field-weighted citation impact, top 1% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Phase II Trial of BRAF/MEK Inhibitors in Papillary Craniopharyngiomas
Who cites it
84 citing papers in PubMed, 7 syntheses or guidelines pooled it, 121 citations in OpenAlex.
- BRAF-targeted therapy in non-melanomatous BRAF-mutant tumours: a systematic review of broad but histology-modulated efficacy.International journal of clinical oncology · 2026Pooled it
- Pituitary stalk biopsy - A systematic review of the safety and efficacy of stalk lesion biopsy.Pituitary · 2026Pooled it
- Efficacy and safety of BRAF-MEK dual inhibition in BRAF-V600E mutated papillary craniopharyngioma: a systematic review.Journal of neuro-oncology · 2025Pooled it
- Modern treatment of craniopharyngioma to improve outcomes: evidence of a change of paradigm.Endocrine · 2025Pooled it
- The spectrum of radiation therapy options for craniopharyngioma: a systematic review.Journal of neuro-oncology · 2025Pooled it
- Fractionated stereotactic radiotherapy in craniopharyngiomas: A systematic review and single arm meta-analysis.Journal of neuro-oncology · 2024Pooled it
- Advancing Craniopharyngioma Management: A Systematic Review of Current Targeted Therapies and Future Perspectives.International journal of molecular sciences · 2024Pooled it
- High-precision radiotherapy achieves excellent long-term control and preserves function in pediatric craniopharyngioma-Subset analysis of a randomized trial.Neuro-oncology · 2025Trial
- Molecular therapy for papillary craniopharyngioma: a multi-institutional analysis of practice patterns across the RAPID Consortium.Journal of neuro-oncology · 2026Article
- Hypothalamic syndrome and acquired hypothalamic obesity: from fragmented care to integrated multidisciplinary management.Pituitary · 2026Review
- The Modular Cup-in-Cup Construct in Revision Total Hip Arthroplasty.Arthroplasty today · 2026Article
- Early Findings from the Multicenter RAPID Consortium on Papillary Craniopharyngiomas.Journal of neurological surgery. Part B, Skull base · 2026Article
- Gamma Knife treatment of papillary craniopharyngioma: a single-center study of tumor control and predictors of progression.Radiation oncology (London, England) · 2026Article
- Benefits and challenges of adding BKM120 to a BI-3406 plus trametinib combination therapy.BMC cancer · 2026Article
- Cancer treatment-related cognitive impairment.Neuro-oncology advances · 2026Review
- Current Approaches and Therapeutic Strategies for Hypothalamic Syndrome in Patients with Childhood-onset Craniopharyngioma.Journal of clinical research in pediatric endocrinology · 2026Review
- Clinical responses to trastuzumab deruxtecan in molecularly defined ependymoma.NPJ precision oncology · 2026Article
- Development and validation of a predictive model for hypothalamic obesity following craniopharyngioma resection.BMC endocrine disorders · 2026Article
- Quantitative BRAF p.V600E mutation monitoring in cerebrospinal fluid cell-free DNA reflects therapeutic response to BRAF/MEK inhibitors in papillary craniopharyngioma: a report of two cases.Acta neuropathologica communications · 2026Article
- Targeted therapies in adolescent and young adult patients with central nervous system tumors.Neuro-oncology advances · 2026Review
24 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
Authors and funding
28 authors at 9 institutions in 2 countries.
Priscilla K BrastianosFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).ORCID 0000-0003-4470-8425
Erin TwohyFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Susan GeyerFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Elizabeth R GerstnerFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Timothy J KaufmannFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Shervin TabriziFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Brian KabatFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Julia ThieraufFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Michael W RuffFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Daniela A BotaFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
David A ReardonFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Adam L CohenFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Macarena I De La FuenteFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Glenn J LesserFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Jian CampianFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Pankaj K AgarwallaFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Priya KumthekarFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Bhupinder MannFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Shivangi VoraFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Michael KnoppFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
A John IafrateFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
William T CurryFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Daniel P CahillFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Helen A ShihFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Paul D BrownFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Sandro SantagataFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Fred G BarkerFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Evanthia GalanisFrom Massachusetts General Hospital Cancer Center, Harvard Medical School (P.K.B., E.R.G., S.T., J.T., A.J.I., W.T.C., D.P.C., H.A.S., F.G.B.), Dana-Farber Cancer Institute (D.A.R.), and Brigham and Women's Hospital, Harvard Program in Therapeutic Science, Dana-Farber Partners CancerCare (S.S.) - all in Boston; Alliance Statistics and Data Management Center (E.T., S.G., B.K.), Mayo Clinic (T.J.K., M.W.R., P.D.B., E.G.), Rochester, MN; UC Irvine-Chao Family Comprehensive Cancer Center, Orange, CA (D.A.B.); Huntsman Cancer Institute, University of Utah, Salt Lake City (A.L.C.); Sylvester Comprehensive Cancer Center, University of Miami Miller School of Medicine, Miami (M.I.D.L.F.); Wake Forest University School of Medicine, Winston-Salem, NC (G.J.L.); Washington University School of Medicine, St. Louis (J.C.); Rutgers Cancer Institute, New Brunswick, NJ (P.K.A.); Northwestern University, Chicago (P.K.); the Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, MD (B.M.); and Ohio State University Comprehensive Cancer Center, Columbus (S.V., M.K.).
Dana-Farber Cancer Institute · USHarvard University · USBrigham and Women's Hospital · USNorthwestern University · PHRutgers, The State University of New Jersey · USUniversity of California, Irvine · USUniversity of Miami · USUniversity of Utah · USWashington University in St. Louis · US
Funding
NRG Oncology Network Group Operations Center - GY9 BIQSFP Reports/BudgetsU10CA180868 · NCI · NRG ONCOLOGY FOUNDATION, INC. · PI NORMAN WOLMARK · 2014 to 2026
$206.8MMember Site CoreU10CA180821 · NCI · BRIGHAM AND WOMEN'S HOSPITAL · PI Evanthia Galanis · 2014 to 2026
$177.3MIROC: Enhancing local enrolling site radiological data capture capabilities for NCTN trialsU24CA180803 · NCI · AMERICAN COLLEGE OF RADIOLOGY · PI Thomas J. FitzGerald, MICHAEL V KNOPP · 2014 to 2026
$116.2MStatistics CoreU10CA180882 · NCI · MAYO CLINIC ROCHESTER · PI Sumithra Jay Mandrekar · 2014 to 2026
$115.0MWake Forest NCORP Research BaseUG1CA189824 · NCI · WAKE FOREST UNIVERSITY HEALTH SCIENCES · PI Emily Van Meter Dressler, GLENN J LESSER · 2014 to 2026
$57.9MTHE ALLIANCE NCTN BIOREPOSITORY AND BIOSPECIMEN RESOURCEU24CA196171 · NCI · WASHINGTON UNIVERSITY · PI Mine Cicek, Wendy Frankel · 2015 to 2026
$35.0MWASHINGTON UNIVERSITY / SITEMAN CANCER CENTER LEAD ACADEMIC SITEUG1CA233339 · NCI · WASHINGTON UNIVERSITY · PI NANCY L BARTLETT, Clifford Grant Robinson · 2019 to 2026
$11.3MOSU as a Network Lead Academic Participating Site for the NCI NCTNUG1CA233331 · NCI · OHIO STATE UNIVERSITY · PI John L. Hays, Dwight H. Owen · 2019 to 2026
$8.9MNCTN Lead Academic Participating Site at Dana-Farber/Partners Cancer CareUG1CA233180 · NCI · DANA-FARBER CANCER INST · PI Harold John Burstein · 2019 to 2026
$8.6MNCI, National Clinical Trials Network Lead Academic Participating Site (LAPS) UG1 (funded extension)UG1CA232760 · NCI · MAYO CLINIC ROCHESTER · PI Judy Caroline Boughey, Aaron Scott Mansfield · 2019 to 2026
$8.5MNorthwestern University Lead Academic Participating Site Supplement Year 2UG1CA233320 · NCI · NORTHWESTERN UNIVERSITY AT CHICAGO · PI BENSON, AL B, MATEI, DANIELA E · 2019 to 2025
$5.5MHuntsman Cancer Institute/ University of Utah Network Lead Academic Participating Site ApplicationUG1CA233178 · NCI · UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH · PI Matthew Poppe, Heloisa Prado Soares · 2019 to 2026
$4.9MNCI NIH HHS U10 CA180821NCI NIH HHS U10CA180821, U10CA180882, U24CA196171NCI NIH HHS U10 CA180868NCI NIH HHS U10CA180868NCI NIH HHS U10 CA180882NCI NIH HHS U24 CA180803NCI NIH HHS U24 CA196171NCI NIH HHS UG1 CA189824NCI NIH HHS UG1 CA232760NCI NIH HHS UG1CA232760, UG1CA233178, UG1CA233180, UG1CA23332NCI NIH HHS UG1 CA233178NCI NIH HHS UG1 CA233180NCI NIH HHS UG1 CA233320NCI NIH HHS UG1 CA233331NCI NIH HHS UG1CA233331, UG1CA233339, UG1CA189824NCI NIH HHS UG1 CA233339
Abstract
backgroundCraniopharyngiomas, primary brain tumors of the pituitary-hypothalamic axis, can cause clinically significant sequelae. Treatment with the use of surgery, radiation, or both is often associated with substantial morbidity related to vision loss, neuroendocrine dysfunction, and memory loss. Genotyping has shown that more than 90% of papillary craniopharyngiomas carry
methodsEligible patients who had papillary craniopharyngiomas that tested positive for
resultsOf the 16 patients in the study, 15 (94%; 95% confidence interval [CI], 70 to 100) had a durable objective partial response or better to therapy. The median reduction in the volume of the tumor was 91% (range, 68 to 99). The median follow-up was 22 months (95% CI, 19 to 30) and the median number of treatment cycles was 8. Progression-free survival was 87% (95% CI, 57 to 98) at 12 months and 58% (95% CI, 10 to 89) at 24 months. Three patients had disease progression during follow-up after therapy had been discontinued; none have died. The sole patient who did not have a response stopped treatment after 8 days owing to toxic effects. Grade 3 adverse events that were at least possibly related to treatment occurred in 12 patients, including rash in 6 patients. In 2 patients, grade 4 adverse events (hyperglycemia in 1 patient and increased creatine kinase levels in 1 patient) were reported; 3 patients discontinued treatment owing to adverse events.
conclusionsIn this small, single-group study involving patients with papillary craniopharyngiomas, 15 of 16 patients had a partial response or better to the BRAF-MEK inhibitor combination vemurafenib-cobimetinib. (Funded by the National Cancer Institute and others; ClinicalTrials.gov number, NCT03224767.).
Indexed as
Antineoplastic AgentsCraniopharyngiomaPituitary NeoplasmsAzetidinesDisease ProgressionHumansMitogen-Activated Protein Kinase KinasesPiperidinesProto-Oncogene Proteins B-rafRemission InductionVemurafenibAntineoplastic AgentsAzetidinesBRAF protein, humancobimetinibMitogen-Activated Protein Kinase KinasesPiperidinesProto-Oncogene Proteins B-rafVemurafenib
Identifiers
What OpenQuestion holds
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Registered trials
NCT03224767ClinicalTrials.gov
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.