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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
PubMed
Abstract

Insights

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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