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

Gupta, A; Cohen, B H; Ruggieri, P; Packer, R J; Phillips, P C · Neurology · 2003

case_series · Level IV

Where this comes from

Abstract

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.

Medical subject headings