Spigel, D RVicente, DCiuleanu, T EGettinger, SPeters, SHorn, LAudigier-Valette, CPardo Aranda, NJuan-Vidal, OCheng, YZhang, HShi, MLuft, AWolf, JAntonia, SNakagawa, KFairchild, JBaudelet, CPandya, DDoshi, PChang, HReck, M2023-02-092023-02-092021-02-01http://hdl.handle.net/10668/17105Patients with relapsed small-cell lung cancer (SCLC) have few treatment options and dismal survival. Phase I/II data show activity of nivolumab in previously treated SCLC. CheckMate 331 is a randomized, open-label, phase III trial of nivolumab versus standard chemotherapy in relapsed SCLC. Patients with relapse after first-line, platinum-based chemotherapy were randomized 1 : 1 to nivolumab 240 mg every 2 weeks or chemotherapy (topotecan or amrubicin) until progression or unacceptable toxicity. Primary endpoint was overall survival (OS). Overall, 284 patients were randomized to nivolumab and 285 to chemotherapy. Minimum follow-up was 15.8 months. No significant improvement in OS was seen with nivolumab versus chemotherapy [median OS, 7.5 versus 8.4 months; hazard ratio (HR), 0.86; 95% confidence interval (CI), 0.72-1.04; P = 0.11]. A survival benefit with nivolumab was suggested in patients with baseline lactate dehydrogenase ≤ upper limit of normal and in those without baseline liver metastases. OS (nivolumab versus chemotherapy) was similar in patients with programmed death-ligand 1 combined positive score ≥1% versus Nivolumab did not improve survival versus chemotherapy in relapsed SCLC. No new safety signals were seen. In exploratory analyses, select baseline characteristics were associated with improved OS for nivolumab.enAttribution-NonCommercial-NoDerivatives 4.0 Internationalhttp://creativecommons.org/licenses/by-nc-nd/4.0/PD-1biomarkersimmunotherapysmall-cell lung cancerAntineoplastic Combined Chemotherapy ProtocolsHumansLung NeoplasmsNeoplasm Recurrence, LocalNivolumabProgression-Free SurvivalSmall Cell Lung Carcinomaresearch article33539946open access10.1016/j.annonc.2021.01.0711569-8041http://www.annalsofoncology.org/article/S0923753421000995/pdf