ArticleNeuro-oncology2017
A phase I trial of the MEK inhibitor selumetinib (AZD6244) in pediatric patients with recurrent or refractory low-grade glioma: a Pediatric Brain Tumor Consortium (PBTC) study.
2 registry-linked trialsOpen access · bronzeAbstract readClinical Trial, Phase I
In one paragraph
Article in Neuro-oncology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 191 papers, 3 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
191citing papers in PubMed, 3 pooled it
19.6field-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.
NCT03326388 phase1 / phase2completednot on this mapstarted 2019, after this paper: background citation
A Paediatric Phase I/II Study Of Intermittent Dosing Of The Mek-1 Inhibitor Selumetinib In Children With Neurofibromatosis Type-1 And Inoperable Plexiform Neurofibroma And/Or Progressive Optic Pathway Glioma
NCT05555550 early_phase1recruitingnot on this mapstarted 2026, after this paper: background citation
Evaluation of 18F-Fluciclovine PET-MRI as a Biomarker of Response in Pediatric and Young Adult Patients With Low Grade Gliomas (LGG)
Who cites it
191 citing papers in PubMed, 3 syntheses or guidelines pooled it, 316 citations in OpenAlex.
- Rebound regrowth phenomenon in patients with pediatric low-grade gliomas treated with MAPK inhibitors - a systematic review.Oncology reviews · 2026Pooled it
- Pediatric low-grade brainstem glioma: a review of current diagnosis and treatment paradigms in the molecular era.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2025Pooled it
- Evidence-Based Recommendations for Education Provided to Patients and Families Regarding the Adverse Events of ALK and MEK Inhibitors: A Systematic Review From the Children's Oncology Group.Journal of pediatric hematology/oncology nursingPooled it
- A phase 2 PBTC study of selumetinib for recurrent/progressive pediatric low-grade glioma: Strata 2, 5, and 6 with long-term outcomes on strata 1, 3, and 4.Neuro-oncology · 2025Trial
- The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial.Nature medicine · 2024Trial
- Long-term safety and efficacy of selumetinib in children with neurofibromatosis type 1 on a phase 1/2 trial for inoperable plexiform neurofibromas.Neuro-oncology · 2023Trial
- Selumetinib in children with neurofibromatosis type 1 and asymptomatic inoperable plexiform neurofibroma at risk for developing tumor-related morbidity.Neuro-oncology · 2022Trial
- Phase II Study of Selumetinib in Children and Young Adults With Tumors Harboring Activating Mitogen-Activated Protein Kinase Pathway Genetic Alterations: Arm E of the NCI-COG Pediatric MATCH Trial.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2022Trial
- A phase II trial of selumetinib in children with recurrent optic pathway and hypothalamic low-grade glioma without NF1: a Pediatric Brain Tumor Consortium study.Neuro-oncology · 2021Trial
- Selumetinib in Children with Inoperable Plexiform Neurofibromas.The New England journal of medicine · 2020Trial
- Selumetinib in paediatric patients with BRAF-aberrant or neurofibromatosis type 1-associated recurrent, refractory, or progressive low-grade glioma: a multicentre, phase 2 trial.The Lancet. Oncology · 2019Trial
- A phase II study of eribulin in recurrent or refractory osteosarcoma: A report from the Children's Oncology Group.Pediatric blood & cancer · 2019Trial
- Management of adverse events in patients with pediatric low-grade glioma treated with MAPK-directed therapies: Delphi consensus recommendations and unmet needs.Neuro-oncology practice · 2026Article
- RAS/MAPK inhibition in pediatric gliomas.Neuro-oncology advances · 2026Review
- Review
- Pediatric Optic Pathway Gliomas: Diagnosis, Management, and Outcomes.International ophthalmology clinics · 2026Review
- MEK inhibition in adult patients with pilocytic astrocytomas.NPJ precision oncology · 2026Article
- Molecular convergence enables precision medicine for pediatric low grade gliomas.Discover oncology · 2026Review
- Current Perspectives on Pediatric Low-Grade Gliomas: Focus on BRAF and MEK Inhibition.Brain tumor research and treatment · 2026Review
- Report from the National Pediatric Cancer Foundation - infantile glioma and other non-embryonal central nervous system tumors: evolving molecular advances and current treatment landscape.Frontiers in oncology · 2026Review
131 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
21 authors at 10 institutions in 1 country.
Anuradha BanerjeeUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Regina I JakackiUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Arzu Onar-ThomasUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Shengjie WuUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Theodore NicolaidesUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Tina Young PoussaintUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Jason FangusaroUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Joanna PhillipsUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Arie PerryUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
David TurnerUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Michael PradosUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Roger J PackerUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Ibrahim QaddoumiUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Sridharan GururanganUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Ian F PollackUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Stewart GoldmanUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Lawrence A DoyleUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Clinton F StewartUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
James M BoyettUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Larry E KunUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Maryam FouladiUniversity of California San Francisco, San Francisco, California; Boston Children's Hospital, Boston, Massachusetts; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; St Jude Children's Research Hospital, Memphis, Tennessee; Lurie Children's Hospital, Chicago, Illinois; Children's National Medical Center, Washington, DC; Duke University Medical Center, Durham, North Carolina; Cancer Therapy Evaluation Program, National Cancer Institute, Bethesda, Maryland; Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Children's Hospital of Pittsburgh · USLurie Children's Hospital · USNational Cancer Institute · USSt. Jude Children's Research Hospital · USBoston Children's Hospital · USDuke University Hospital · USUniversity of California, San Francisco · USChildren's National · USCincinnati Children's Hospital Medical Center · USDuke Medical Center · US
Funding
Pediatric Brain Tumor Consortium (PBTC)UM1CA081457 · NCI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI Ira J Dunkel, Arzu Onar-Thomas · 2014 to 2026
$41.4MThe Pediatric Brain Tumor Consortium (PBTC)U01CA081457 · NCI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI BOYETT, JAMES MILFORD · 1999 to 2013
$29.2MNCI NIH HHS U01 CA081457NCI NIH HHS UM1 CA081457
Abstract
backgroundActivation of the mitogen-activated protein kinase pathway is important for growth of pediatric low-grade gliomas (LGGs). The aim of this study was to determine the recommended phase II dose (RP2D) and the dose-limiting toxicities (DLTs) of the MEK inhibitor selumetinib in children with progressive LGG.
methodsSelumetinib was administered orally starting at 33 mg/m2/dose b.i.d., using the modified continual reassessment method. Pharmacokinetic analysis was performed during the first course. BRAF aberrations in tumor tissue were determined by real-time polymerase chain reaction and fluorescence in situ hybridization.
resultsThirty-eight eligible subjects were enrolled. Dose levels 1 and 2 (33 and 43 mg/m2/dose b.i.d.) were excessively toxic. DLTs included grade 3 elevated amylase/lipase (n = 1), headache (n = 1), mucositis (n = 2), and grades 2-3 rash (n = 6). At dose level 0 (25 mg/m2/dose b.i.d, the RP2D), only 3 of 24 subjects experienced DLTs (elevated amylase/lipase, rash, and mucositis). At the R2PD, the median (range) area under the curve (AUC0-∞) and apparent oral clearance of selumetinib were 3855 ng*h/mL (1780 to 7250 ng × h/mL) and 6.5 L × h-1 × m-2 (3.4 to 14.0 L × h-1 × m-2), respectively. Thirteen of 19 tumors had BRAF abnormalities. Among the 5 (20%) of 25 subjects with sustained partial responses, all at the RP2D, 4 had BRAF aberrations, 1 had insufficient tissue. Subjects received a median of 13 cycles (range: 1-26). Fourteen (37%) completed all protocol treatment (26 cycles [n = 13], 13 cycles [n = 1]) with at least stable disease; 2-year progression-free survival at the RP2D was 69 ± SE 9.8%.
conclusionSelumetinib has promising antitumor activity in children with LGG. Rash and mucositis were the most common DLTs.
Indexed as
AdolescentBenzimidazolesBrain NeoplasmsChildChild, PreschoolDisease-Free SurvivalDose-Response Relationship, DrugFemaleGliomaHumansMaleMaximum Tolerated DoseMitogen-Activated Protein KinasesNeoplasm GradingNeoplasm Recurrence, LocalProtein Kinase InhibitorsAZD 6244BenzimidazolesMitogen-Activated Protein KinasesProtein Kinase Inhibitorslow-grade gliomaphase I trialselumetinib
Identifiers
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Read underepoch 390
Registered trials
NCT03326388ClinicalTrials.gov
NCT05555550ClinicalTrials.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.