Abstract
The strong association of systemic mastocytosis (SM) with mutations and constitutive activation of the receptor tyrosine kinase KIT in >90% of patients has led to great interest in investigating the clinical activity and safety of tyrosine kinase inhibitors (TKI) in these neoplasms. These agents were (a) either already approved for other hematologic neoplasms and demonstrated in vitro activity toward mutated forms of KIT, for example, imatinib, nilotinib or dasatinib; or (b) applied to patients with SM because of activity against KIT in vitro as part of a broader multikinase inhibitory profile, for example, midostaurin; or c) developed as specific KIT inhibitors, for example, avapritinib, ripretinib, or masitinib. Midostaurin and imatinib have been approved for specific indications by the regulatory health authorities, while avapritinib, ripretinib, and masitinib are still being investigated in clinical trials. Due to the low overall response rates, nilotinib and dasatinib are no longer actively pursued in SM.
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Jawhar, M., Gotlib, J., Reiter, A. (2020). Tyrosine Kinase Inhibitors in Systemic Mastocytosis. In: Akin, C. (eds) Mastocytosis. Springer, Cham. https://doi.org/10.1007/978-3-030-27820-5_15
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DOI: https://doi.org/10.1007/978-3-030-27820-5_15
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