Trial reportAnnals of neurology2026
Trametinib in Adults with Neurofibromatosis Type 1-Related Symptomatic Plexiform Neurofibromas.
Trial report in Annals of neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers, 1 of them a synthesis that pooled it.
What it found
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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.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
2 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Pooled it
- Neurofibromatosis type 1-plexiform neurofibromas: Integrating treatment across pediatric and adult populations.Neuro-oncology · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveMitogen-activated protein kinase kinase inhibitors have shown promising results in treatment of plexiform neurofibromas in neurofibromatosis type 1 patients, but data in adults are limited. The aim of this phase 2 study was to investigate the efficacy and safety of trametinib in adults with neurofibromatosis type 1.
methodsThirty patients with a diagnosis of generalized or mosaic neurofibromatosis type 1 and a symptomatic inoperable plexiform neurofibroma were treated with 2mg trametinib per day. Primary outcome measures were partial response rate on tumor volume at cycle 12 and overall partial response rate. Partial response was defined as ≥20% decrease in tumor volume compared to baseline as measured with 3-dimensional volumetric analysis on magnetic resonance imaging every sixth cycle. Secondary outcome measures were pain, pain interference, quality of life, and toxicity.
resultsPartial response rate after cycle 12 was 47% (n = 14/30). Overall partial response rate was 50% (n = 15/30). Median best response was -22% decrease of tumor volume (range: -63 to +7%), and median time until best response was 12 cycles (range: 6-42 cycles). Pain score and pain interference were significantly decreased after 12 cycles of treatment. No change in quality of life was reported. Acneiform rash and fatigue were the most reported adverse events. Overall rate of treatment discontinuation because of adverse events was 40%.
interpretationWe observed a positive effect of trametinib on tumor volume and significant pain reduction in adults with plexiform neurofibromas. However, adverse events are common and frequently led to early termination of treatment. ANN NEUROL 2026;99:73-83.
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