title: Phase II study of sorafenib in children with recurrent or progressive low-grade astrocytomas.
abstract: BACKGROUND
  Activation of the RAS-RAF-MEK-ERK signaling pathway is thought to be the key driver of pediatric low-grade astrocytoma (PLGA) growth. Sorafenib is a multikinase inhibitor targeting BRAF, VEGFR, PDGFR, and c-kit. This multicenter phase II study was conducted to determine the response rate to sorafenib in patients with recurrent or progressive PLGA.
  
  
  METHODS
  Key eligibility criteria included age ≥ 2 years, progressive PLGA evaluable on MRI, and at least one prior chemotherapy treatment. Sorafenib was administered twice daily at 200 mg/m(2)/dose (maximum of 400 mg/dose) in continuous 28-day cycles. MRI, including 3-dimensional volumetric tumor analysis, was performed every 12 weeks. BRAF molecular testing was performed on tumor tissue when available.
  
  
  RESULTS
  Eleven patients, including 3 with neurofibromatosis type 1 (NF1), were evaluable for response; 5 tested positive for BRAF duplication. Nine patients (82%) came off trial due to radiological tumor progression after 2 or 3 cycles, including 3 patients with confirmed BRAF duplication. Median time to progression was 2.8 months (95% CI, 2.1-31.0 months). Enrollment was terminated early due to this rapid and unexpectedly high progression rate. Tumor tissue obtained from 4 patients after termination of the study showed viable pilocytic or pilomyxoid astrocytoma.
  
  
  CONCLUSIONS
  Sorafenib produced unexpected and unprecedented acceleration of tumor growth in children with PLGA, irrespective of NF1 or tumor BRAF status. In vitro studies with sorafenib indicate that this effect is likely related to paradoxical ERK activation. Close monitoring for early tumor progression should be included in trials of novel agents that modulate signal transduction.
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Matthias A
authors.initials: MA
authors.lastname: Karajannis
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Geneviève
authors.initials: G
authors.lastname: Legault
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Michael J
authors.initials: MJ
authors.lastname: Fisher
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Sarah S
authors.initials: SS
authors.lastname: Milla
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Kenneth J
authors.initials: KJ
authors.lastname: Cohen
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Jeffrey H
authors.initials: JH
authors.lastname: Wisoff
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: David H
authors.initials: DH
authors.lastname: Harter
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Judith D
authors.initials: JD
authors.lastname: Goldberg
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Tsivia
authors.initials: T
authors.lastname: Hochman
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Amanda
authors.initials: A
authors.lastname: Merkelson
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Michael C
authors.initials: MC
authors.lastname: Bloom
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Angela J
authors.initials: AJ
authors.lastname: Sievert
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Adam C
authors.initials: AC
authors.lastname: Resnick
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Girish
authors.initials: G
authors.lastname: Dhall
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: David T W
authors.initials: DT
authors.lastname: Jones
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Andrey
authors.initials: A
authors.lastname: Korshunov
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Stefan M
authors.initials: SM
authors.lastname: Pfister
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Charles G
authors.initials: CG
authors.lastname: Eberhart
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: David
authors.initials: D
authors.lastname: Zagzag
authors.affiliation: NYU Comprehensive Neurofibromatosis Center, Division of Pediatric Hematology/Oncology, Department of Pediatrics and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (M.A.K., G.L., A.M., J.C.A.); Division of Oncology, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (M.J.F., A.J.S.); Department of Radiology, NYU Langone Medical Center, New York, New York (S.S.M., M.C.B.); The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland (K.J.C.); Division of Pediatric Neurosurgery, Department of Neurosurgery, NYU Langone Medical Center, New York, New York (J.H.W., D.H.H.); Division of Biostatistics, Department of Population Health and The Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (J.D.G., T.H.); Department of Neurosurgery, Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania (A.C.R); Division of Hematology/Oncology, Children's Hospital Los Angeles, Los Angeles, California (G.D.); German Cancer Research Center and University Hospital, Heidelberg, Germany (D.T.W.J., A.K., S.M.P.); Division of Neuropathology, Department of Pathology, Johns Hopkins University, Baltimore, Maryland (C.G.E.); Division of Neuropathology, Department of Pathology, Department of Neurosurgery and Laura and Isaac Perlmutter Cancer Center at NYU Langone Medical Center, New York, New York (D.Z.).
authors.forename: Jeffrey C
authors.initials: JC
authors.lastname: Allen
mesh_terms: D000293:Adolescent; D000818:Animals; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001254:Astrocytoma / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D001932:Brain Neoplasms / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D002675:Child, Preschool; D005260:Female; D006801:Humans; D053208:Kaplan-Meier Estimate; D020935:MAP Kinase Signaling System / Q000187:drug effects; D008279:Magnetic Resonance Imaging; D008297:Male; D051379:Mice; D041681:NIH 3T3 Cells; D009536:Niacinamide / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000627:therapeutic use; D010671:Phenylurea Compounds / Q000008:administration & dosage / Q000627:therapeutic use*; D047428:Protein Kinase Inhibitors / Q000008:administration & dosage / Q000627:therapeutic use; D000077157:Sorafenib; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D010671:Phenylurea Compounds; D047428:Protein Kinase Inhibitors; D009536:Niacinamide; D000077157:Sorafenib
keywords: low-grade glioma; pediatric low-grade astrocytoma; phase II trial; sorafenib