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Avapritinib for Systemic Mastocytosis.

Prithviraj Bose1, Srdan Verstovsek1

  • 1Department of Leukemia, University of Texas MD Anderson Cancer Center, Houston, TX, USA.

Expert Review of Hematology
|July 22, 2021
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Summary

Avapritinib effectively targets advanced systemic mastocytosis (AdvSM) by inhibiting the KIT D816V mutation. This targeted therapy shows a 75% response rate in AdvSM, offering a new treatment option for this rare myeloid neoplasm.

Keywords:
Avapritinibadvanced smaggressive sm (asm)c-findingsindolent smkit d816vmast cell leukemia (mcl)midostaurinsm with an associated hematologic neoplasm (sm-ahn)systemic mastocytosis (sm)

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Area of Science:

  • Hematology
  • Oncology
  • Pharmacology

Background:

  • Systemic mastocytosis (SM) is a rare myeloid neoplasm.
  • Approximately 95% of SM cases are driven by activating KIT mutations, commonly D816V.
  • SM classifications include indolent (ISM), smoldering (SSM), and advanced (AdvSM), with AdvSM having a poor prognosis.

Purpose of the Study:

  • To provide an overview of systemic mastocytosis (SM).
  • To discuss avapritinib, a KIT inhibitor, including its discovery, pharmacodynamics, pharmacokinetics, and regulatory status.
  • To review the safety and efficacy of avapritinib in patients with advanced and non-advanced SM.

Main Methods:

  • Review of avapritinib's chemistry and discovery.
  • Analysis of pharmacodynamic and pharmacokinetic data.
  • Evaluation of safety and efficacy profiles in clinical studies.

Main Results:

  • Avapritinib demonstrated a 75% response rate in evaluable patients with advanced SM (AdvSM).
  • Significant reductions in mast cell burden and disease markers were observed.
  • Early results in indolent/smoldering SM (ISM/SSM) are promising.
  • Avapritinib is approved in the US for AdvSM.

Conclusions:

  • Avapritinib is a highly effective targeted therapy for KIT D816V-driven advanced SM.
  • Potential side effects include cognitive impairment and intracranial hemorrhage, especially with thrombocytopenia.
  • Avapritinib represents a significant advancement in treating advanced systemic mastocytosis.