Final survival and safety results for 21,064 non-small-cell lung cancer (NSCLC) patients who received compassionate use gefitinib in a U.S. expanded access program (EAP)

Abstract

7060 Background: Effective chemotherapy results in 1-year survival rates of approximately 30% in consenting patients with advanced and recurrent NSCLC who are able to tolerate chemotherapy. We present final analyses from >21,000 patients who received gefitinib, an epidermal growth factor receptor tyrosine kinase inhibitor in an EAP. Methods: Eligibility criteria included stage III/IV NSCLC patients who failed or were unable to tolerate chemotherapy. Patients received oral gefitinib 250 mg/day as long as treatment provided clinical benefit without unacceptable toxicity. Duration of therapy and survival were measured from the start of initial therapy to the last resupply date for ongoing patients or the date of last dose for withdrawn patients. For surviving patients who withdrew, periodic follow-up data were not collected, and patients were censored for survival at withdrawal until death was reported. Results: As of July 2003, 23,383 patients were enrolled and 21,064 received ≥ 1 dose of gefitinib. Of 21,064 patients, 9979 were women and 11,040 were men, 87.8% were white, median age was 67 years, and 72.7% had stage IV disease. Median survival was 5.3 months (CI, 5.1–5.5 mo) and 1-year survival was 29.9% (95% CI, 28.8–31.1). Better survival was seen in women, Asian/Oriental ethnic groups, and stage III patients. Of a 9501 patient subset who received gefitinib and had potential for a 1-year follow-up, 20.1% received drug for ≥ 6 months. In 2.3% of patients, a serious treatment-related adverse (AE) event was reported: 1.1% discontinued therapy due to a serious drug-related AE, and 0.3% had an investigator-assessed, drug-related death. Conclusions: One-year survival rates in over 20,000 patients treated with gefitinib on a compassionate basis are comparable to that obtained with chemotherapy in a second-line clinical setting. Reported safety was favorable when compared with chemotherapy. These findings are consistent with 2 large randomized phase II trials (IDEAL 1 and 2) and reported single institution EAP clinical experience. Author Disclosure Employment or Leadership Consultant or Advisory Stock Ownership Honoraria Research Funding Expert Testimony Other Remuneration AstraZeneca Pharmaceuticals Mutual funds