Phase I study of thalidomide for the treatment of plexiform neurofibroma in neurofibromatosis 1.
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.
Gupta
A
A
Department of Neurology, Cleveland Clinic Foundation, OH 44195, USA.
Cohen
B H
BH
Ruggieri
P
P
Packer
R J
RJ
Phillips
P C
PC
D000293:Adolescent; D000328:Adult; D020533:Angiogenesis Inhibitors / Q000009:adverse effects / Q000627:therapeutic use; D002648:Child; D015331:Cohort Studies; D003879:Dermatologic Agents / Q000009:adverse effects / Q000627:therapeutic use; D004305:Dose-Response Relationship, Drug; D003875:Drug Eruptions / Q000209:etiology; D005260:Female; D006258:Head and Neck Neoplasms / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D008297:Male; D009378:Neoplasms, Multiple Primary / Q000150:complications / Q000188:drug therapy*; D018318:Neurofibroma, Plexiform / Q000150:complications / Q000188:drug therapy*; D009456:Neurofibromatosis 1 / Q000150:complications / Q000188:drug therapy*; D010386:Pelvic Neoplasms / Q000150:complications / Q000188:drug therapy*; D010523:Peripheral Nervous System Diseases / Q000139:chemically induced; D012894:Sleep Stages / Q000187:drug effects; D013125:Spinal Neoplasms / Q000150:complications / Q000188:drug therapy*; D013792:Thalidomide / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
D020533:Angiogenesis Inhibitors; D003879:Dermatologic Agents; D013792:Thalidomide