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Malignancy in Neurofibromatosis Type 1

Partners Center for Human Genetics, Harvard Medical School, Boston, Massachusetts, USA

Correspondence: Bruce R. Korf, M.D., Ph.D., Medical Director, Partners Center for Human Genetics, Associate Professor of Neurology (Pediatrics), Harvard Medical School, 77 Avenue Louis Pasteur, Suite 642, Boston, Massachusetts 02115, USA. Telephone: 617-525-5750; Fax 617-525-5757; e-mail: bkorf{at}partners.org

Neurofibromatosis type 1 (NF1) represents a major risk factor for development of malignancy, particularly malignant peripheral nerve sheath tumors (MPNST), optic gliomas, other gliomas, and leukemias. The oncologist will see NF1 patients referred for treatment of malignancy, and should be alert to the possibility of undiagnosed NF1 among patients with cancer. Brain tumors tend to have a more indolent course in NF1 than in the general population, and hence are best managed conservatively. MPNST, in contrast, do not respond to standard chemotherapy or radiation therapy. The most effective treatment of MPNST appears to be early diagnosis and surgery, but early diagnosis is hampered by frequent occurrence within preexisting large tumors, making new growth or change difficult to detect. New insights into pathogenesis now offer hope of development of specific methods of treatment with reduced toxicity and more precise molecular targeting. There is an urgent need, however, to develop methods to measure tumor growth and monitor outcomes, develop preclinical drug screening systems, and further explore the pathogenesis of the disorder to determine whether mechanisms other than Ras regulation may be important in pathogenesis.

Key Words. Neurofibromatosis type 1 • Malignant peripheral nerve sheath tumors • Central nervous system

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