Background: The outcomes of patients with advanced non-small cell lung cancer (NSCLC) treated in phase I clinical trials have not been systematically analyzed.
Methods: We reviewed the records of consecutive patients with advanced/metastatic NSCLC who were treated in the Phase I Clinical Trials Program at MD Anderson from August 2004 to May 2009.
Results: Eighty-five patients (51 men, 34 women) treated on various phase I protocols were identified. The median age was 62 years (range, 30-85). The median number of previous systemic therapies was two (range, 0-5). A partial response was observed in eight patients (9.5%) and stable disease lasting >4 months was observed in 16 patients (19%). The median overall survival time was 10.6 months and median progression-free survival (PFS) time was 2.8 months, which was 0.6 months shorter than the median PFS of 3.4 months following prior second-line therapy. Factors predicting longer survival in the univariate analysis were an Eastern Cooperative Oncology Group performance status (PS) score of 0-1, no prior smoking, two or fewer organ systems involved, a hemoglobin level ≥ 12 g/dL, liver metastases, a history of thromboembolism, and a platelets count > 440 × 10(9)/L. In the multivariate analysis, a PS score of 0-1 and history negative for smoking predicted longer survival. Sixty-two (73%) patients had grade ≤ 2 toxicity, and there were no treatment-related deaths.
Conclusion: Phase I clinical trials were well tolerated by selected patients with advanced NSCLC treated at M.D. Anderson. Nonsmokers and patients with a good PS survived longer. PFS in our population was shorter in smokers/ex-smokers and patients with a PS score of 2. It is reasonable to refer pretreated patients with a good PS to phase I clinical trials.