Imatinib in advanced chordoma: A retrospective case series analysis. Issue 17 (November 2015)

• Record Type: Journal Article
• Authors: Hindi, Nadia; Casali, Paolo G.; Morosi, Carlo; Messina, Antonella; Palassini, Elena; Pilotti, Silvana; Tamborini, Elena; Radaelli, Stefano; Gronchi, Alessandro; Stacchiotti, Silvia
• Is Part Of: European journal of cancer. Volume 51:Issue 17(2015:Nov.)
• Page Start: 2609
• Page End: 2614
• Publication Date: 2015-11
• Subjects: Chordoma -- Imatinib -- Sarcoma -- Chemotherapy -- Targeted therapy -- Metastasis; Cancer -- Periodicals; Neoplasms -- Periodicals; Cancer -- Périodiques; Cancer; Tumors; Electronic journals; Periodicals; Electronic journals; 616.994
• DOI: 10.1016/j.ejca.2015.07.038
• Languages: English
• ISSNs: 0959-8049

Abstract

Abstract: Introduction: Imatinib showed activity in 50 chordoma patients treated within a Phase II study. In that study, 70% of patients remained with stable disease (SD), median progression free survival (PFS) was 9 months and median overall survival (OS) was 34 months. We now report on a retrospective series of PDGFB/PDGFRB positive advanced chordoma patients treated with imatinib as a single agent within a compassionate-use programme at Istituto Nazionale Tumori, Milan, Italy (INT) between August 2002 and November 2010, when the programme was closed. Methods: 48 patients were consecutively treated with imatinib 800 mg/d. All patients had inoperable and progressive disease before starting imatinib. Demographics, treatment duration, toxicity and response rate by Response Evaluation Criteria in Solid Tumors (RECIST) were retrospectively recorded. Results: The median duration of therapy was 7 months (1–46.5). No patient is on therapy at present. 46 patients were evaluable for response. No partial responses were detected. Best response was: stable disease 34 (74%), progressive disease 12 (26%). At a median follow-up of 24.5 months (0.5–117), median PFS was 9.9 months (95% confidence interval (CI) 6.7–13). Eight patients (16.5%) remained on therapy >18 months and 10 patients (21%) remained progression-free >18 months. Median OS was 30 months (95% CI 20–40), with 24 (50%) patients dead at the time of the present analysis. Conclusions: We confirm the activity of imatinib in locally advanced and metastatic chordoma, in terms of >70% tumour growth arrest in previously progressive patients. Median duration of response lasted almost 10 months, with >20% of patients progression-free at 18+ months.