Oral topotecan demonstrates clinical activity in relapsed non-small cell lung cancer. Results from an open-label, phase III study (387) comparing oral topotecan to intravenous docetaxel

7017 Background: Oral topotecan has demonstrated activity in NSCLC as a single agent and in combination (Clin Cancer Res, 2000). Study objective was to develop an oral therapy with a similar risk:benefit profile to IV docetaxel. Methods: In this open-label phase III study, pretreated patients (pts) with stage III/IV NSCLC stratified for time to progression (TTP) from 1st-line treatment (≤90 days, >90 days), stage (locally advanced, metastatic), gender, and performance status (PS) (0/1, 2) were randomized to oral topotecan 2.3 mg/m2/d, d1–5, q21d or IV docetaxel 75 mg/m2/d, d1 with dexamethasone premedication, q21d. Primary endpoint was 1-yr survival. Secondary endpoints were overall survival, TTP, response rate, QOL (measured using LCSS), and tolerability. Assuming a 37% 1-yr survival rate, analysis was powered to exclude an absolute 10% margin of noninferiority. Pts were treated until disease progression and monitored ≥1 yr to record poststudy therapy and survival. Results: Between 10/2001 and 4/2003, 82...