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Elsevier, The Lancet Oncology, 12(13), p. 1218-1224

DOI: 10.1016/s1470-2045(12)70414-x

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Imatinib mesylate for plexiform neurofibromas in patients with neurofibromatosis type 1: A phase 2 trial

This paper is available in a repository.
This paper is available in a repository.

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Abstract

Plexiform neurofibromas are slow-growing chemoradiotherapy-resistant tumours arising in patients with neurofibromatosis type 1 (NF1). Currently, there are no viable therapeutic options for patients with plexiform neurofibromas that cannot be surgically removed because of their proximity to vital body structures. We undertook an open-label phase 2 trial to test whether treatment with imatinib mesylate can decrease the volume burden of clinically significant plexiform neurofibromas in patients with NF1.