EGFR tyrosine kinase inhibitors in non-small cell lung cancer: Report of a 3-year compassionate use experience with gefitinib in stage IIIB/IV outpatients

Background: Since March 2002, 240 patients with advanced stage IIIB/IV non-small cell lung cancer (NSCLC) have been treated inside a compassionate use program with oral gefitinib ( 250 mg o.d.). Patients and Methods: Within a 36-month observation period, 32 patients who showed either partial remission or confirmed disease stabilization for at least 3 months, were retrospectively analyzed. Results: Our reported 4% overall response rate matches with literature data for an unselected patient population. The median progression-free survival (PFS) was 9 months, the median overall survival ( OS) since detection of advanced disease was 21 months. Interestingly, no relevant differences in PFS were observed for chemo-naive and second/third-line patients, whereas a reduced benefit in terms of response rate, PFS and OS was found for forth-line patients. 12 patients survived for at least 2 years since manifestation of their advanced disease. A slight benefit regarding cough and performance status conservation was observed. Mild to modest skin reactions and diarrhea were the adverse events reported most often. Grade 3 toxicities were rarely observed. Conclusions: The retrospective evaluation of therapy results from compassionate use patients helps clinicians to better evaluate new drugs in practice. EGFR tyrosine kinase ( TK) inhibitors require ongoing intensive evaluation to optimize their clinical use in NSCLC.