Phase I study of thalidomide for the treatment of plexiform neurofibroma in neurofibromatosis 1.
case_series · Level IV
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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
- Head and Neck Neoplasms
- Neoplasms, Multiple Primary
- Neurofibroma, Plexiform
- Neurofibromatosis 1
- Pelvic Neoplasms
- Spinal Neoplasms
- Thalidomide