ArticleNeuro-oncology advances
Selumetinib in pediatric patients with neurofibromatosis type 1 and plexiform neurofibroma: Propensity score analysis of SPRINT vs. natural history control arm.
Article in Neuro-oncology advances. 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 4 papers, 1 of them a synthesis that pooled it.
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.
Natural History Study and Longitudinal Assessment of Children, Adolescents, and Adults With Neurofibromatosis Type 1
A Phase I/II Study of the Mitogen Activated Protein Kinase (MEK) 1 Inhibitor Selumetinib (AZD6244; Hydrogen Sulfate in Children With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)
Who cites it
4 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Pooled it
- Therapeutic effects of selumetinib on diffuse neurofibroma and optic pathway glioma in neurofibromatosis type 1.Journal of neuro-oncology · 2026Trial
- Article
- Standalone NF1: guiding the transition from paediatric to adult care.Drugs in context · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background: Selumetinib is approved in children aged ≥ 2 years (USA) with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN) based on data from SPRINT (NCT01362803). This analysis estimated PN progression risk with selumetinib in SPRINT versus age-matched patients from the National Cancer Institute NF1 Natural History (NH) study (NCT00924196), accounting for differences in baseline characteristics using propensity score (PS) methods. Methods: Differences in baseline characteristics between the cohorts were assessed. PSs based on age, sex, race, weight, height, and PN (location, volume, and progression status) were used in 1:1 PS matching without replacement, stabilized inverse probability of treatment weighting (sIPTW), and 1:2 PS matching with replacement as sensitivity analyses. The effect of selumetinib (maximum follow-up 5.6 years) on progression risk was evaluated using univariate and multivariable Cox models (adjusted for baseline characteristics) of progression-free survival. Results: Before PS matching, patient baseline characteristics ( Conclusions: Reduction in risk of NF1-related PN progression with selumetinib was consistent with direct comparison and statistically significant, robust, and comparable across PS methods. Trial registration: ClinicalTrials.gov, NCT01362803. Registered May 27, 2011, https://clinicaltrials.gov/study/NCT01362803.
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What OpenQuestion holds
Registered trials
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.