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Thalidomide for neurofibromatosis

Posted May 30 2010 12:00am

Multiple plexiform fibromas are a hallmark of type 1 neurofibromatosis, which condition also presents with other findings and symptoms. It can appear as cafe-au-lait spots and bumps under the skin, or it can lead to complications that include blindness, scoliosis and disfigurement. Fibromas can cause pain and compromise other organs. Few treatments are available, although many novel agents are being studied. 


In regard to thalidomde, one study, termed a phase I study, with 20 patients had been performed. As per clincicaltrials.gov, not studies of this drug are registered for neurofibromatosis. Cleraly, the evidence is very preliminary.


Gupta, A., Cohen, B.H., Ruggieri, P., Packer, R.J., Phillips, P.C.

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

Neurology 2003 60: 130-132


V. C. Williams, J. Lucas, M. A. Babcock, D. H. Gutmann, B. Korf, and B. L. Maria

Neurofibromatosis Type 1 Revisited

Pediatrics, January 1, 2009; 123(1): 124 - 133. 

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