Avapritinib in the treatment of PDGFRA exon 18 mutated gastrointestinal stromal tumors

Future Oncol. 2020 Aug;16(22):1639-1646. doi: 10.2217/fon-2020-0348. Epub 2020 Jun 10.


Gastrointestinal stromal tumors (GIST) can be molecularly classified based on different subtypes including mutations in KIT and PDGFRA. Patients with PDGFRA mutations are an important subgroup that commonly arise in the stomach and are associated with a more indolent disease course. Importantly, the most common PDGFRA molecular subtype, the D842V mutation in exon 18 of the gene which alters the activation loop, is imatinib insensitive in in vitro studies. Poor responses to imatinib have been seen clinically compared with PDGFRA exon 18 non-D842V-mutated GIST. Avapritinib (BLU-285) is a potent KIT and PDGFRA-specific tyrosine kinase inhibitor which has shown >90% response rates in patients with PDGFRA exon 18 D842V-mutated GIST. Results from the Phase I trial of avapritinib have indicated that this drug should be the standard of care for patients with PDGFRA exon 18 D842V-mutated GIST.

Keywords: BLU-285; D842V; GIST; PDGFRA; avapritinib.

Publication types

  • Review

MeSH terms

  • Exons*
  • Gastrointestinal Neoplasms / drug therapy*
  • Gastrointestinal Neoplasms / genetics
  • Gastrointestinal Stromal Tumors / drug therapy*
  • Gastrointestinal Stromal Tumors / genetics
  • Humans
  • Imatinib Mesylate / therapeutic use
  • Mutation*
  • Pyrazoles / therapeutic use*
  • Pyrroles / therapeutic use*
  • Receptor, Platelet-Derived Growth Factor alpha / antagonists & inhibitors
  • Receptor, Platelet-Derived Growth Factor alpha / genetics*
  • Standard of Care
  • Triazines / therapeutic use*


  • Pyrazoles
  • Pyrroles
  • Triazines
  • avapritinib
  • Imatinib Mesylate
  • Receptor, Platelet-Derived Growth Factor alpha