Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis-1 with Everolimus: A Phase II, Open-Label, Single-Arm Trial View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2018-10-03

AUTHORS

John M. Slopis, Octavio Arevalo, Cynthia S. Bell, Adelaide A. Hebert, Hope Northrup, Roy F. Riascos, Joshua A. Samuels, Keri C. Smith, Patti Tate, Mary Kay Koenig

ABSTRACT

BACKGROUND: Cutaneous neurofibromas cause disfigurement and discomfort in individuals with neurofibromatosis type 1 (NF-1). METHODS: The 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. RESULTS: Of 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. CONCLUSIONS: Although 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. More... »

PAGES

295-302

References to SciGraph publications

  • 2017-02-23. Neurofibromatosis type 1 in NATURE REVIEWS DISEASE PRIMERS
  • 2004-05. The tor pathway: a target for cancer therapy in NATURE REVIEWS CANCER
  • 2012-04-29. Molecular Therapies for Tuberous Sclerosis and Neurofibromatosis in CURRENT NEUROLOGY AND NEUROSCIENCE REPORTS
  • 2017-02-23. Neurofibromatosis type 1 in NATURE REVIEWS DISEASE PRIMERS
  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1007/s40268-018-0248-6

    DOI

    http://dx.doi.org/10.1007/s40268-018-0248-6

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1107372748

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/30284154


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