Avapritinib in unresectable or metastatic gastrointestinal stromal tumor with PDGFRA exon 18 mutation: safety and efficacy

Expert Rev Anticancer Ther. 2021 Oct;21(10):1081-1088. doi: 10.1080/14737140.2021.1963235. Epub 2021 Aug 22.


Introduction: Avapritinib (formerly known as BLU-285) is an orally available type I tyrosine kinase inhibitor that, in 2020, obtained regulatory approval for the treatment of patients with gastrointestinal stromal tumors (GISTs) harboring a primary mutation in PDGFRA exon 18, including the PDGFRA D842V mutation.

Areas covered: Herein, we comprehensively review the available efficacy and safety data on avapritinib, with the final goal of providing practical knowledge to both sarcoma and community-based oncologists for the correct management of this rare GIST subpopulation with this novel therapy.

Expert opinion: The approval of avapritinib in GIST is a milestone in precision oncology, as this is the first agent ever demonstrating unequivocal antitumoral activity in GIST driven by the multi-resistant PDGFRA D842V mutation. The safety profile is manageable and tolerability-guided dose adjustment is recommended to manage treatment-related adverse events without compromising efficacy. Based on its unprecedented activity, avapritinib should be considered as first-line therapy for GIST patients harboring this mutation. We strongly recommend to determine KIT/PDGFRA genotype in order to identify the different GIST molecular subtypes and guide treatment decision.

Keywords: Avapritinib; BLU-285; GIST; PDGFRA; imatinib; sarcoma; treatment; tyrosine kinase inhibitor.

MeSH terms

  • Antineoplastic Agents* / adverse effects
  • Exons
  • Gastrointestinal Stromal Tumors* / drug therapy
  • Gastrointestinal Stromal Tumors* / genetics
  • Gastrointestinal Stromal Tumors* / pathology
  • Humans
  • Mutation
  • Precision Medicine
  • Proto-Oncogene Proteins c-kit / genetics
  • Proto-Oncogene Proteins c-kit / therapeutic use
  • Pyrazoles
  • Pyrroles
  • Receptor, Platelet-Derived Growth Factor alpha / genetics
  • Triazines


  • Antineoplastic Agents
  • Pyrazoles
  • Pyrroles
  • Triazines
  • avapritinib
  • Proto-Oncogene Proteins c-kit
  • Receptor, Platelet-Derived Growth Factor alpha