Lenalidomide Treatment for Thalidomide-refractory POEMS Syndrome: A Prospective Single-arm Clinical Trial

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Author(s)

    • Suichi Tomoki
    • Department of Neurology, Graduate School of Medicine, Chiba University, Japan
    • Suzuki Yo-ichi
    • Department of Neurology, Graduate School of Medicine, Chiba University, Japan
    • Tsuneyama Atsuko
    • Department of Neurology, Graduate School of Medicine, Chiba University, Japan
    • Nakamura Keigo
    • Department of Neurology, Graduate School of Medicine, Chiba University, Japan
    • Kuwabara Satoshi
    • Department of Neurology, Graduate School of Medicine, Chiba University, Japan
    • Misawa Sonoko
    • Department of Neurology, Graduate School of Medicine, Chiba University, Japan
    • Nagashima Kengo
    • Research Center for Medical and Health Data Science, The Institute of Statistical Mathematics, Japan
    • Sato Yasunori
    • Department of Preventive Medicine and Public Health, School of Medicine, Keio University, Japan
    • Iwai Yuta
    • Department of Neurology, Graduate School of Medicine, Chiba University, Japan
    • Sekiguchi Yukari
    • Department of Neurology, Graduate School of Medicine, Chiba University, Japan
    • Shibuya Kazumoto
    • Department of Neurology, Graduate School of Medicine, Chiba University, Japan
    • Amino Hiroshi
    • Department of Neurology, Graduate School of Medicine, Chiba University, Japan

Abstract

<p><b>Objective </b>A randomized controlled trial has shown the efficacy of thalidomide against Polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes (POEMS) syndrome; however, there are still refractory patients. We studied the effects of lenalidomide, a derivative of thalidomide, on patients refractory to thalidomide. </p><p><b>Methods </b>This prospective single-arm trial evaluated the safety and efficacy of lenalidomide plus dexamethasone in refractory or recurrent patients with POEMS syndrome. The regimen was administered as six 28-day cycles with lenalidomide on days 1-21 (15 mg in cycle 1, and 25 mg in cycle 2-6) plus dexamethasone once a week (20 mg). The primary endpoints were the rate of reduction in the serum vascular endothelial growth factor (VEGF) level at 24 weeks and the incidence of adverse events. This trial was registered with ClinicalTrial.gov, NCT02193698. </p><p><b>Results </b>Between July 2014 and December 2015, five men were enrolled. All patients had been refractory to thalidomide plus dexamethasone for more than 24 weeks. The mean rate of reduction in the serum VEGF level at 24 weeks was 59.6%±8.3% (p=0.0003). The mean serum VEGF level decreased from 2,466±771 pg/mL to 974±340 pg/mL. No serious adverse events were observed, and all patients completed six cycles treatment. </p><p><b>Discussion </b>Lenalidomide is a therapeutic option for thalidomide-refractory patients with POEMS syndrome. </p>

Journal

  • Internal Medicine

    Internal Medicine, 2020

    The Japanese Society of Internal Medicine

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