Phase I study of thalidomide for the treatment of plexiform neurofibroma in neurofibromatosis 1

被引:65
作者
Gupta, A
Cohen, BH
Ruggieri, P
Packer, RJ
Phillips, PC
机构
[1] Cleveland Clin Fdn, Dept Neurol, Cleveland, OH 44195 USA
[2] Cleveland Clin Fdn, Brain Tumor Inst, Cleveland, OH 44195 USA
[3] Cleveland Clin Fdn, Ctr Canc, Cleveland, OH 44195 USA
[4] Childrens Natl Med Ctr, Washington, DC 20010 USA
[5] Childrens Hosp, Dept Neurol, Philadelphia, PA 19104 USA
关键词
D O I
10.1212/01.WNL.0000042321.94839.78
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
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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收藏
页码:130 / 132
页数:3
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