Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trial.

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Title: Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trial.
Authors: Gross AM; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA. andrea.gross@nih.gov., O'Sullivan Coyne G; Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Dombi E; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Tibery C; Clinical Research Directorate, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD, USA., Herrick WG; Clinical Pharmacodynamics Biomarker Program, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD, USA., Martin S; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Angus SP; Division of Hematology/Oncology, Department of Pediatrics, Indiana University School of Medicine, Riley Hospital for Children at Indiana University Health and Herman B. Wells Center for Pediatric Research, Indianapolis, IN, USA., Shern JF; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Rhodes SD; Division of Hematology/Oncology, Department of Pediatrics, Indiana University School of Medicine, Riley Hospital for Children at Indiana University Health and Herman B. Wells Center for Pediatric Research, Indianapolis, IN, USA., Foster JC; Biometric Research Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Rubinstein LV; Biometric Research Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Baldwin A; Clinical Research Directorate, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD, USA., Davis C; Division of Hematology/Oncology, Department of Pediatrics, Indiana University School of Medicine, Riley Hospital for Children at Indiana University Health and Herman B. Wells Center for Pediatric Research, Indianapolis, IN, USA., Dixon SAH; Division of Hematology/Oncology, Department of Pediatrics, Indiana University School of Medicine, Riley Hospital for Children at Indiana University Health and Herman B. Wells Center for Pediatric Research, Indianapolis, IN, USA., Fagan M; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Ong MJ; Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Wolters PL; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Tamula MA; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Reid O; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Sankaran H; Biometric Research Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Fang F; Division of Hematology/Oncology, Department of Pediatrics, Indiana University School of Medicine, Riley Hospital for Children at Indiana University Health and Herman B. Wells Center for Pediatric Research, Indianapolis, IN, USA., Govindharajulu JP; Clinical Pharmacodynamics Biomarker Program, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD, USA., Browne AT; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Kaplan RN; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Heisey K; Clinical Research Directorate, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD, USA., On TJ; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Xuei X; Division of Hematology/Oncology, Department of Pediatrics, Indiana University School of Medicine, Riley Hospital for Children at Indiana University Health and Herman B. Wells Center for Pediatric Research, Indianapolis, IN, USA., Zhang X; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Johnson BC; Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Parchment RE; Clinical Pharmacodynamics Biomarker Program, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD, USA., Clapp DW; Division of Hematology/Oncology, Department of Pediatrics, Indiana University School of Medicine, Riley Hospital for Children at Indiana University Health and Herman B. Wells Center for Pediatric Research, Indianapolis, IN, USA., Srivastava AK; Clinical Pharmacodynamics Biomarker Program, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD, USA., Doroshow JH; Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.; Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Chen AP; Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA., Widemann BC; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA. widemanb@mail.nih.gov.
Source: Nature medicine [Nat Med] 2025 Jan; Vol. 31 (1), pp. 105-115. Date of Electronic Publication: 2025 Jan 06.
Publication Type: Journal Article; Clinical Trial, Phase II; Research Support, N.I.H., Extramural; Research Support, N.I.H., Intramural; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Nature Publishing Company Country of Publication: United States NLM ID: 9502015 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1546-170X (Electronic) Linking ISSN: 10788956 NLM ISO Abbreviation: Nat Med Subsets: MEDLINE
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  Data: Selumetinib in adults with NF1 and inoperable plexiform neurofibroma: a phase 2 trial.
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  Data: <searchLink fieldCode="AU" term="%22Gross+AM%22">Gross AM</searchLink>; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA. andrea.gross@nih.gov.<br /><searchLink fieldCode="AU" term="%22O'Sullivan+Coyne+G%22">O'Sullivan Coyne G</searchLink>; Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Dombi+E%22">Dombi E</searchLink>; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Tibery+C%22">Tibery C</searchLink>; Clinical Research Directorate, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD, USA.<br /><searchLink fieldCode="AU" term="%22Herrick+WG%22">Herrick WG</searchLink>; Clinical Pharmacodynamics Biomarker Program, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD, USA.<br /><searchLink fieldCode="AU" term="%22Martin+S%22">Martin S</searchLink>; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Angus+SP%22">Angus SP</searchLink>; Division of Hematology/Oncology, Department of Pediatrics, Indiana University School of Medicine, Riley Hospital for Children at Indiana University Health and Herman B. 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Wells Center for Pediatric Research, Indianapolis, IN, USA.<br /><searchLink fieldCode="AU" term="%22Fagan+M%22">Fagan M</searchLink>; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Ong+MJ%22">Ong MJ</searchLink>; Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Wolters+PL%22">Wolters PL</searchLink>; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Tamula+MA%22">Tamula MA</searchLink>; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Reid+O%22">Reid O</searchLink>; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Sankaran+H%22">Sankaran H</searchLink>; Biometric Research Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Fang+F%22">Fang F</searchLink>; Division of Hematology/Oncology, Department of Pediatrics, Indiana University School of Medicine, Riley Hospital for Children at Indiana University Health and Herman B. Wells Center for Pediatric Research, Indianapolis, IN, USA.<br /><searchLink fieldCode="AU" term="%22Govindharajulu+JP%22">Govindharajulu JP</searchLink>; Clinical Pharmacodynamics Biomarker Program, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD, USA.<br /><searchLink fieldCode="AU" term="%22Browne+AT%22">Browne AT</searchLink>; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Kaplan+RN%22">Kaplan RN</searchLink>; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Heisey+K%22">Heisey K</searchLink>; Clinical Research Directorate, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD, USA.<br /><searchLink fieldCode="AU" term="%22On+TJ%22">On TJ</searchLink>; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Xuei+X%22">Xuei X</searchLink>; Division of Hematology/Oncology, Department of Pediatrics, Indiana University School of Medicine, Riley Hospital for Children at Indiana University Health and Herman B. Wells Center for Pediatric Research, Indianapolis, IN, USA.<br /><searchLink fieldCode="AU" term="%22Zhang+X%22">Zhang X</searchLink>; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Johnson+BC%22">Johnson BC</searchLink>; Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Parchment+RE%22">Parchment RE</searchLink>; Clinical Pharmacodynamics Biomarker Program, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD, USA.<br /><searchLink fieldCode="AU" term="%22Clapp+DW%22">Clapp DW</searchLink>; Division of Hematology/Oncology, Department of Pediatrics, Indiana University School of Medicine, Riley Hospital for Children at Indiana University Health and Herman B. Wells Center for Pediatric Research, Indianapolis, IN, USA.<br /><searchLink fieldCode="AU" term="%22Srivastava+AK%22">Srivastava AK</searchLink>; Clinical Pharmacodynamics Biomarker Program, Frederick National Laboratory for Cancer Research, Leidos Biomedical Research, Inc., Frederick, MD, USA.<br /><searchLink fieldCode="AU" term="%22Doroshow+JH%22">Doroshow JH</searchLink>; Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.; Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Chen+AP%22">Chen AP</searchLink>; Division of Cancer Treatment and Diagnosis, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Widemann+BC%22">Widemann BC</searchLink>; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA. widemanb@mail.nih.gov.
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  Data: <searchLink fieldCode="JN" term="%229502015%22">Nature medicine</searchLink> [Nat Med] 2025 Jan; Vol. 31 (1), pp. 105-115. <i>Date of Electronic Publication: </i>2025 Jan 06.
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