Tyrosine Kinase Inhibitor (TKI) Therapy

Chordomas overexpress PDGFRA, PDGFRB, and KIT. Because of this finding, imatinib mesylate has been studied in adults with chordomas.[13,14]

In one study, 50 adults with chordomas were treated with imatinib and evaluated by Response Evaluation Criteria In Solid Tumors (RECIST) guidelines. One patient had a partial response and 28 additional patients had stable disease at 6 months.[14] The low rate of RECIST responses and the potentially slow natural course of the disease complicate the assessment of the efficacy of imatinib for chordoma.[14]

Other TKIs and combinations involving TKIs have been studied in adults.[15-17]

One multicenter French retrospective study reported five patients who had partial responses to treatment with either imatinib, sorafenib, or erlotinib. The median PFS was 36 months.[18]