Phase II study of imatinib in advanced chordoma

Silvia Stacchiotti*, Alessandra Longhi, Virginia Ferraresi, Giovanni Grignani, Alessandro Comandone, Roger Stupp, Alexia Bertuzzi, Elena Tamborini, Silvana Pilotti, Antonella Messina, Carlo Spreafico, Alessandro Gronchi, Paola Amore, Vincenza Vinaccia, Paolo Giovanni Casali

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

208 Scopus citations


Purpose: To explore the antitumor activity of imatinib in patients with advanced platelet-derived growth factor β (PDGFB)/PDGF receptor β (PDGFRB)-positive chordomas. Patients and Methods: In a collaborative Italian-Swiss, prospective, phase II clinical study conducted from November 2004 through April 2006, 56 patients with advanced PDGFB and/or PDGFRB chordoma received 800 mg/d of imatinib until progression. The primary end point was the overall tumor response rate (ORR), defined by RECIST. Secondary, exploratory end points included tissue response (ie, changes in tumor density or signal intensity/contrast enhancement, and/or [18F]-fluorodeoxyglucose positron emission tomography [PET] uptake), overall survival, progression-free survival (PFS), and pain score. Results: Among 50 patients evaluable by RECIST, the best response was one partial response (PR) obtained at 6 months (ORR, 2%). There were 35 patients with stable disease (SD, 70%) and a 64% clinical benefit rate (ie, RECIST complete response + PR + SD ≥ 6 months). A minor dimensional response (< 20%) was detected in nine patients. A maximum standard uptake value decrease ≥ 25% was observed in 10 (39%) of 26 patients evaluable for PET response at 3 months. Changes in the Brief Pain Inventory score were consistent with the response assessment. Median PFS (intention-to-treat population, 56 patients) was 9 months. No unexpected toxicities were observed. Conclusion: This is the largest phase II study in chordoma to date. It confirms anecdotal evidence that imatinib has antitumor activity in this orphan disease, and therefore, it is worth further investigation.

Original languageEnglish (US)
Pages (from-to)914-920
Number of pages7
JournalJournal of Clinical Oncology
Issue number9
StatePublished - Mar 20 2012

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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