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Novel Approaches for Systemic Mastocytosis.
Luke Fletcher1,2, Uma Borate1,2
1Knight Cancer Institute.
Current Opinion in Hematology
|January 30, 2019
Summary
New therapies are evolving for advanced systemic mastocytosis, a rare blood cancer. While midostaurin offers some improvement, newer tyrosine kinase inhibitors show promise for complete remission in systemic mastocytosis patients.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Systemic mastocytosis is a rare clonal neoplastic disease of mast cells.
- Recent advancements have introduced novel therapeutic agents targeting specific mutations.
Purpose of the Study:
- To review the pathophysiology of systemic mastocytosis.
- To summarize recent clinical trials and drug development for systemic mastocytosis, focusing on advanced subtypes.
Main Methods:
- Literature review of recent clinical trials and drug development in systemic mastocytosis.
- Focus on targeted therapies, particularly tyrosine kinase inhibitors.
Main Results:
- Midostaurin, FDA-approved in 2017, shows a 60% response rate but limited complete remissions.
- Avapritinib and DCC-2618, targeting KIT mutations, demonstrate promising early clinical trial data, with avapritinib showing potential for complete remissions.
Conclusions:
- Systemic mastocytosis therapies are rapidly evolving.
- Further research into oncogenic mutations and targeted therapies may improve complete response rates and durable remissions.
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