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August 14, 1987

New Hope for Neurofibromatosis? The Mast Cell Connection

JAMA. 1987;258(6):823. doi:10.1001/jama.1987.03400060099039

Neurofibromatosis is a common and progressive illness.1,2 The spectrum of disease varies from the mildest of formes frustes to highly destructive and disfiguring syndromes. (It now seems that the most famous putative severe example, that of Joseph Merrick, "The Elephant Man," was not neurofibromatosis at all.3) The primary, if not the only, treatment has been surgery.

In the August issue of the American Medical Association's Archives of Dermatology,4 Riccardi presents his early experience in treating neurofibromatosis with ketotifen, a mast cell stabilizer.5 During a three-year period, 15 patients received oral ketotifen. The conditions of 11 patients improved in one or more of the following variables: pain, itching, or the general feeling of well-being. In addition, most of these patients also had an apparent decrease in neurofibroma size, or a slowing of tumor growth, and a decrease in the profuse bleeding that accompanies surgery in this illness.