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From the Department of Neurology (Drs. Gupta and Cohen), Brain Tumor Institute (Dr. Cohen), Radiology (Dr. Ruggieri), and Cancer Center (Dr. Cohen), Cleveland Clinic Foundation, OH; Department of Neurology (Dr. Packer), Childrens National Medical Center, Washington, DC; and Department of Neurology (Dr. Phillips), Childrens Hospital of Philadelphia, PA.
Address correspondence and reprint requests to Dr. Bruce H. Cohen, S-71, Department of Neurology, Cleveland Clinic Foundation, 9500 Euclid Avenue, Cleveland, OH 44195; e-mail: cohenb1{at}ccf.org
An open-label phase I trial of thalidomide (TL) in 20 patients with neurofibromatosis 1 (NF1) treated symptomatic plexiform neurofibroma (PNF). TL was well tolerated in doses up to 200 mg/d. Adverse reactions included transient somnolence in four, evanescent rash in two, and reversible mild peripheral neuropathy in two patients. Four patients showed less than 25% reduction in the tumor size. TL may have a role in the treatment of PNF and should be explored in a larger controlled study, possibly using higher doses of TL.
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