TY - JOUR TI - Hydroxychloroquine in combination with platinum doublet chemotherapy as first-line treatment for extensive-stage small cell lung cancer (Study 15): A randomised phase II multicentre trial VL - 215 AV - public Y1 - 2025/01/17/ N1 - © 2024 The Authors. Published by Elsevier Ltd. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/). ID - discovery10202698 N2 - BACKGROUND: Most patients with small-cell lung cancer (SCLC) present with extensive-stage (ES) disease and have a poor prognosis despite achieving high initial response rates to platinum-based doublet chemotherapy. This study evaluated whether adding hydroxychloroquine (HCQ) to chemotherapy could improve outcomes. METHODS: This was a randomised multicentre phase II trial. Eligible patients had untreated ES-SCLC, a performance status 0-2 and measurable disease. Patients were randomly assigned (1:1 ratio) to HCQ (400 mg orally twice daily) plus carboplatin-gemcitabine or carboplatin-etoposide alone. Chemotherapy was administered for up to six cycles, with HCQ given concurrently and then as single agent for up to 30 months. Primary endpoint was PFS, aiming for a hazard ratio (HR) of 0.70. RESULTS: 72 patients were randomised (36 HCQ+chemotherapy and 36 chemotherapy alone). Median HCQ treatment duration was 4.4 months. HCQ did not improve PFS (HR 1·12 95 %CI 0·69-1.84; p = 0·64), with a median of 5.7 months (HCQ+chemotherapy) versus 6.2 months (chemotherapy). The corresponding median OS were 8.9 and 10.2 months (HR 0.83, 95 %CI 0.48-1.45, p = 0.52). Fewer patients in the HCQ arm completed four cycles of chemotherapy due to adverse events (64 % vs. 81 %). Grade ? 3 adverse events were higher in the HCQ+chemotherapy arm (83.3 % vs. 27.8 %), primarily anaemia, neutropenia, and thrombocytopenia, partly due to the initially higher gemcitabine dose used CONCLUSIONS: Combining HCQ with platinum doublet chemotherapy did not improve PFS or OS outcomes for ES-SCLC, resulting in more patients stopping chemotherapy due to increased adverse events. When considered alongside other randomised studies of HCQ in cancer, the evidence collectively indicates a limited role for HCQ as a therapeutic option. SN - 0959-8049 PB - Elsevier BV UR - https://doi.org/10.1016/j.ejca.2024.115162 JF - European Journal of Cancer KW - Autophagy inhibitor KW - Chemotherapy KW - Concurrent and maintenance treatment KW - Hydroxychloroquine KW - Randomised trial KW - Small cell lung cancer A1 - Lee, Siow Ming A1 - Hewish, Madeleine A1 - Ahmed, Samreen A1 - Papadatos-Pastos, Dionysis A1 - Karapanagiotou, Eleni A1 - Blackhall, Fiona A1 - Ford, Amy A1 - Young, Robin A1 - Garcia, Angel A1 - Arora, Arvind A1 - Hollingdale, Abigail A1 - Ahmad, Tanya A1 - Forster, Martin A1 - Greystoke, Alastair A1 - Bremner, Fion A1 - Rudd, Robin A1 - Farrelly, Laura A1 - Vaja, Simran A1 - Hackshaw, Allan ER -