← Back to all signals
RESEARCH PAPER ANALYSIS

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.

AI interpretation is pending for this paper.

Open original publication →
PMID40241281
JournalNeuro-oncology
Publication Date2025-10-14
Ingested2026-08-02 12:05 AM
EXECUTIVE SUMMARY

What the AI sees

Not AI summarized yet.

WHY IT MATTERS

Research significance

Pending deeper interpretation.

ABSTRACT

Source abstract

BACKGROUND: PBTC-029B was a phase 2 trial evaluating efficacy of selumetinib in children with recurrent/progressive low-grade glioma. We report results of strata 2, 5, and 6 with updated survivals for strata 1, 3, and 4. METHODS: Stratum 2 included recurrent/progressive pilocytic astrocytoma (PA) not associated with neurofibromatosis type-1 (NF1) that screened negative for the BRAF-KIAA1549 fusion and BRAFV600E mutation. Stratum 5 enrolled non-PA that screened positive for one of the BRAF aberrations. Stratum 6 enrolled children who consented to tissue screening, but there was an assay failure. For long-term survivals, stratum 1 included non-NF1 PA positive for one of the BRAF aberrations; stratum 3 included NF1-associated pLGG; and stratum 4 included non-NF1 optic pathway/hypothalamic tumors. RESULTS: Stratum 2: among 14 evaluable patients, there was 1 partial response (PR), 7 stable disease (SD), and 6 progressive disease (PD); the overall response rate (ORR) was 7.1%. Two-year progression-free survival (PFS)/overall survival (OS) were 57.1%/100%, respectively. Stratum 5: among 23 evaluable patients, there was 1 complete response (CR), 4 PR, 12 SD, and 6 PD; ORR was 21.7%. Two-year PFS/OS were 74.8%/100%, respectively. Stratum 6: among 26 evaluable patients, there were 7 PR, 14 SD, and 5 PD; ORR was 26.9%. Two-year PFS/OS were 72.0%/100%, respectively. The median follow-up for patients on strata 1, 3, and 4 without events are 60.4, 60.4, and 58.1 months, and 5-year PFS/OS were 30.8%/88.9%, 54.2%/100%, and 51.0%/100%, respectively. CONCLUSIONS: Selumetinib provided stability and responses across many pLGG subgroups, and some patients achieved prolonged disease control without additional therapy.

SUPPORTING PAPER SET

32 more papers to review

Ranked by current scoring engine
1 Hypothalamic obesity following craniopharyngioma surgery: What is the role of hypothalamic T2 signal changes in brain MRI? Neuro-oncology advances 61.9 2 Personalized Perioperative Music Listening: A Human-Centered Approach to Precision Anesthesia. Cureus 74.2 3 Adult Congenital Pulmonary Airway Malformation Presenting With a Large Cystic Lung Lesion: A Case Report. Cureus 47.5 4 Spinal Myeloid Sarcoma Revealing Acute Myeloid Leukemia in a Child: A Case Report. Cureus 54.3 5 A Non-enhanced CT Dataset for Differential Diagnosis of Pediatric Extracranial Germ Cell Tumors. Scientific data 57.0 6 Female cancers in adolescent and young adult women: global inequities and 2050 burden estimates from GLOBOCAN 2022. Frontiers in oncology 61.1 7 Knowledge of cervical cancer screening, barriers to awareness, and the role of technological interventions in improving screening uptake: a scoping review protocol. Frontiers in health services 78.8 8 Resources are associated with functional outcome and brain morphometry in childhood cancer survivors. Frontiers in oncology 63.9 9 Immunogenicity, efficacy, and safety of different dose schedules of human papillomavirus vaccines: a Bayesian network meta-analysis of randomized controlled trials. Frontiers in immunology 81.32 10 Case Report: Synchronous ALK-positive laryngeal inflammatory myofibroblastic tumor and EGFR-mutant lung adenocarcinoma in a child. Frontiers in pediatrics 48.5 11 Case Report: Anti-GQ1b antibody-positive overlap syndrome: a pediatric case involving the brainstem, spinal cord, and peripheral nerves. Frontiers in immunology 59.66 12 Hunger, satiety, and eating behavior after craniopharyngioma - narrative review on current management and future therapeutic perspectives. Frontiers in endocrinology 82.34 13 Awareness and Acceptance of the Human Papillomavirus (HPV) Vaccine and Associated Factors Among Parents of Adolescents (9-18 Years) at a Children's Hospital in Babol, Iran: A Cross-Sectional Study. Health science reports 63.7 14 Long-term complications after total body irradiation in children younger than 4 years transplanted for acute lymphoblastic leukemia. Bone marrow transplantation 62.4 15 Fertility and Birth Outcomes in Rheumatoid Arthritis: A Narrative Review. Rheumatology and therapy 63.6 16 Characteristics and outcome of T-cell/histiocyte-rich large B-cell lymphoma in childhood and adolescence: An international retrospective analysis. British journal of haematology 28.5 17 Benign and Malignant Conjunctival Lesions in Children. Seminars in ophthalmology 51.5 18 Clinical characteristics, management and outcomes in immune checkpoint inhibitor-induced central nervous system demyelinating disease. Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology 85.76 19 Preoperative Speech Impairments as Precursors of Posterior Fossa Syndrome in Children with Brain Tumours. Cerebellum (London, England) 65.9 20 Invasive Fusarium Infections in Immunocompromised Children: A 10-year Multicenter Retrospective Study and Literature Review. The Pediatric infectious disease journal 76.5 21 Rhabdomyosarcoma Mimicking Otomastoiditis in a Preschool Child with Facial Paralysis. Ear, nose, & throat journal 56.0 22 Cyst wall enhancement is associated with local recurrence after resection of intracranial hemangioblastoma: an exploratory retrospective cohort study. Journal of neuro-oncology 64.7 23 Correction to "Single-Cell RNA Sequencing Reveals the Heterogeneity in Differentiation Trajectory and Tumor Microenvironment Leading to More Aggressive Phenotypes of Papillary Thyroid Cancer in Children and Young Adult Patients". Advanced science (Weinheim, Baden-Wurttemberg, Germany) 47.5 24 Erratum. International archives of allergy and immunology 89.0 25 The Childhood Cancer and Leukemia International Consortium (CLIC): Expanding global collaboration in pediatric cancer etiology research. Cancer epidemiology 64.2 26 Aminosalicylate use Increases Corticosteroid Exposure and Decreases Biologic Durability in Pediatric Crohn's Disease. Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association 68.3 27 Inadequate Energy and Micronutrient Intakes Across Filipino Population Groups: Findings from the 2021 Expanded National Nutrition Survey (ENNS). The Journal of nutrition 67.5 28 Paediatric tracheal carcinoid with carinal involvement and nodal metastasis: challenging airway reconstruction with oncological clearance. BMJ case reports 56.0 29 Acute abdomen in pregnancy revealing sigmoid colon carcinoma at caesarean section. BMJ case reports 58.4 30 Archegonous cystic odontoma: expanding the spectrum of hybrid odontogenic lesions. BMJ case reports 56.0 31 Selective retinal arterial whitening in retinal astrocytic hamartoma in tuberous sclerosis. BMJ case reports 48.5 32 Appendicovesical fistula in a child with acute myeloid leukaemia. BMJ case reports 47.9
PATIENT-FRIENDLY SUMMARY

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.

For education only—not personal medical advice.

Pediatric cancer research intelligence graphic
PEDIATRIC CANCER VISUAL SYSTEM

Open the Research Intelligence Map

Explore the active pediatric oncology analysis view.

Expand Intelligence View →
Full Pediatric cancer research intelligence graphic