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Pilot Study of Gleevec/Imatinib Mesylate (STI 571, NSC 716051) in Neurofibromatosis (NF1) Patients with Plexiform Neurofibromas

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Technical Report,01 Apr 2009,31 Dec 2015

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Indiana University Indianapolis United States

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This is a second Pilot Study to determine the efficacy of Gleevec in neurofibromatosis NF1 patients with plexiform neurofibromas using new response assessment modalities with the secondary goals of assessing Gleevec toxicity, and characterizing markers of response. The rationale for this study arises from the response of human NF1 cells to Gleevec in vitro, as well as in patients including the experience in 37 NF1 patients treated with Gleevec in the initial pilot study. Twenty one patients were enrolled including 8 pediatric patients. Four were in evaluable and 17 were evaluable completing at least 6 months of treatment owing to the success of a novel dose escalation schedule for individual patients. Primary endpoint MRIs, and secondary endpoints safety monitoring, circulating cytokines and endothelial progenitor cell assays, and quality of life data were obtained and assays completed from evaluable patients. Proposed biopsies, pressure measures, and PET imaging were not indicated in the enrolled patients. Results from each of the secondary endpoint assays await final MRI measures which are in progress.

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  • Medicine and Medical Research

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