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Novel approaches in the treatment of systemic mastocytosis
Article first published online: 31 AUG 2006
Copyright © 2006 American Cancer Society
Volume 107, Issue 7, pages 1429–1439, 1 October 2006
How to Cite
Quintas-Cardama, A., Aribi, A., Cortes, J., Giles, F. J., Kantarjian, H. and Verstovsek, S. (2006), Novel approaches in the treatment of systemic mastocytosis. Cancer, 107: 1429–1439. doi: 10.1002/cncr.22187
- Issue published online: 18 SEP 2006
- Article first published online: 31 AUG 2006
- Manuscript Accepted: 28 JUN 2006
- Manuscript Revised: 21 JUN 2006
- Manuscript Received: 19 MAY 2006
- systemic mastocytosis;
- tyrosine kinase inhibitors;
In the absence of curative options, therapy for aggressive forms of systemic mastocytosis (SM) has relied in the use of cytoreductive agents, mainly interferon-α (IFN-α) and cladribine. However, responses are transient and only occur in a subset of patients. Gain-of-function mutations at codon 816 of the KIT protooncogene lead to constitutively active Kit receptor molecules, which are central to the pathogenesis of SM. Recent advances in the understanding of the molecular underpinnings of SM have led to the development of small molecules targeting mutant Kit tyrosine kinase isoforms that significantly have widened the range of therapeutic options for patients with SM. Some of these promising agents, such as dasatinib, AMN107, and PKC412, currently are under investigation in clinical trials whereas, others are at different stages of preclinical development. In addition, monoclonal antibodies directed to neoplastic mast cell-restricted surface antigens constitute a viable option for the treatment of SM that warrants further investigation. Cancer 2006. © 2006 American Cancer Society.