Trial reportDrugs in R&D2018
Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis-1 with Everolimus: A Phase II, Open-Label, Single-Arm Trial.
Trial report in Drugs in R&D, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02332902 (Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1- CRAD001CUS232T), which is not on this map. Cited by 14 papers.
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.
Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1- CRAD001CUS232T
Who cites it
14 citing papers in PubMed, 24 citations in OpenAlex.
- Assessment and Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1: A Scoping Review.Neurology. Genetics · 2026Review
- Diagnosis, treatment management, and challenges in neurofibromatosis type 1: a case report with mixed features.Annals of medicine and surgery (2012) · 2026Article
- Neurofibromatosis Type 1 and the Search for Effective Tumor Therapies Using High-Throughput Drug Screening.Current oncology (Toronto, Ont.) · 2025Review
- Comparing 3D imaging devices for the measurement of cutaneous neurofibromas in patients with Neurofibromatosis Type 1.Skin research and technology : official journal of International Society for Bioengineering and the Skin (ISBS) [and] International Society for Digital Imaging of Skin (ISDIS) [and] International Society for Skin Imaging (ISSI) · 2024Article
- Neurofibromatosis type 1 system-based manifestations and treatments: a review.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2023Review
- Review
- Characterization of health concerns in people with neurofibromatosis type 1.Molecular genetics & genomic medicine · 2023Article
- [Treatment and progress of cutaneous neurofibroma].Zhongguo xiu fu chong jian wai ke za zhi = Zhongguo xiufu chongjian waike zazhi = Chinese journal of reparative and reconstructive surgery · 2022Review
- Insights into the Pathogenesis of NF1-Associated Neoplasms.JID innovations : skin science from molecules to population health · 2021Article
- Pharmacological Approaches in Neurofibromatosis Type 1-Associated Nervous System Tumors.Cancers · 2021Review
- Available Therapies for Patients with Neurofibromatosis-Related Nervous System Tumors.Current treatment options in oncology · 2020Review
- Translating current basic research into future therapies for neurofibromatosis type 1.British journal of cancer · 2020Review
- Disease burden and unmet needs in adults with neurofibromatosis type 1 and plexiform neurofibromas: A systematic literature review.Neuro-oncology advancesReview
- Clinical trials targeting neurofibromatoses-associated tumors: a systematic review.Neuro-oncology advancesReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 3 institutions in 1 country.
Funding
Abstract
backgroundCutaneous neurofibromas cause disfigurement and discomfort in individuals with neurofibromatosis type 1 (NF-1).
methodsThe primary objective of this phase II, open-label, single-arm trial was to assess whether orally administered everolimus reduced the surface volume of cutaneous neurofibromas in patients with NF-1.
resultsOf 22 patients who took the study drug, 17 completed the trial; 5 patients withdrew due to adverse events. Sixteen patients had photographs of sufficient quality for assessment of the primary outcome. A significant reduction in lesion surface volume, defined as an end of trial volume > 2 standard errors (SE) less than baseline volume, was observed for 4/31 lesions (13%) from 3/16 patients (19%). Additionally, a statistically significant absolute change in average height for paired lesions was observed (p = 0.048). Although not a prespecified outcome measure, a dramatic reduction in the size of 3 large plexiform neurofibromas with a cutaneous component was also noted and documented by measurement of maximum circumference or magnetic resonance imaging-based volumetric analysis. Adverse events were common in this trial, but no serious adverse events occurred.
conclusionsAlthough this was a small, exploratory trial that was not powered for significance, the reduction in surface volume observed in this study is noteworthy assuming that the natural course for untreated lesions is to maintain or increase in volume. Future studies are needed with larger study populations that incorporate longer durations of treatment and better standardization of volumetric measurements. Trial Registration ClinicalTrials.gov Identifier: NCT02332902.
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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.