SynthesisCNS drugs2026
Disease Stabilization with MEK Inhibitors in NF1-Associated Plexiform Neurofibromas: A Systematic Review and Meta-Analysis with Subgroup Analyses by Age and Study Design.
Synthesis in CNS drugs, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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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.
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Authors and funding
6 authors.
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Abstract
backgroundPlexiform neurofibromas (PN) represent a significant cause of morbidity among patients diagnosed with neurofibromatosis type 1 (NF1). MEK inhibitors continue to be developed as targeted therapies by inhibiting the mitogen-activated protein kinase pathway to treat PN; nonetheless to this day, therapeutic responses have varied across different patient populations and clinical contexts, and the overall efficacy and tolerability of these agents remain incompletely characterized.
objectiveWe aimed to systematically evaluate the efficacy and safety of MEK inhibitor therapy in patients with NF1-associated PN and to evaluate differences among key subgroups based on the most contemporary metadata.
methodsA comprehensive search was performed across electronic databases to identify studies that reported outcomes related to MEK inhibitor therapy in NF1-associated PN. Pooled proportions were calculated using a random-effects meta-analysis with logit transformation. Outcomes assessed included objective response rate, disease control rate, disease progression rate, and grade ≥ 3 adverse events.
resultsA total of 23 studies comprising 769 patients were included. The pooled objective response rate was estimated at 56% (95% confidence interval [CI] 46-65; I
conclusionsThe use of MEK inhibitors is associated with high rates of disease control and minimal tumor progression in patients with NF1-related PN, with consistent effects observed across clinical trial and real-world environments. Although tumor reduction occurs in some patients, the predominant therapeutic benefit appears to be sustained disease stabilization, with response variability noted among different age groups and study designs.
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