Unexpected rapid progression of metastatic adenoid cystic carcinoma during treatment with imatinib mesylate

Head Neck. 2005 Dec;27(12):1022-7. doi: 10.1002/hed.20274.


Background: There is a lack of effective treatment for metastatic adenoid cystic carcinoma (ACC), a usually indolent tumor. We studied the efficacy of imatinib mesylate, a potent inhibitor of KIT tyrosine kinase, in patients with KIT-positive metastatic ACC.

Method: Five patients with lung metastasis were treated in a pilot study with imatinib 400 mg by mouth twice a day. Mutations of c-kit and platelet-derived growth factor receptor (PDGFR)-alpha in tumors from these patients were analyzed.

Results: Disease progression was noted in three of five patients during the short treatment periods, ranging from 2 to 3 weeks. Three patients died of disease within 6 months. No detectable mutations were found in c-kit and PDGFR-alpha.

Conclusion: We observed an unexpected high progression rate of metastatic ACC within short periods during imatinib treatment. Use of imatinib to treat cancers without c-kit or PDGFR-alpha mutation should be approached with caution.

Publication types

  • Clinical Trial

MeSH terms

  • Administration, Oral
  • Adult
  • Antineoplastic Agents / administration & dosage
  • Antineoplastic Agents / adverse effects*
  • Benzamides
  • Carcinoma, Adenoid Cystic / drug therapy*
  • Carcinoma, Adenoid Cystic / genetics
  • Carcinoma, Adenoid Cystic / secondary
  • DNA Mutational Analysis
  • Disease Progression
  • Female
  • Head and Neck Neoplasms / genetics
  • Head and Neck Neoplasms / pathology*
  • Head and Neck Neoplasms / therapy
  • Humans
  • Imatinib Mesylate
  • Lung Neoplasms / drug therapy*
  • Lung Neoplasms / genetics
  • Lung Neoplasms / secondary
  • Male
  • Middle Aged
  • Pilot Projects
  • Piperazines / administration & dosage
  • Piperazines / adverse effects*
  • Pleural Effusion, Malignant / pathology
  • Protein-Tyrosine Kinases / antagonists & inhibitors
  • Pyrimidines / administration & dosage
  • Pyrimidines / adverse effects*


  • Antineoplastic Agents
  • Benzamides
  • Piperazines
  • Pyrimidines
  • Imatinib Mesylate
  • Protein-Tyrosine Kinases