eprintid: 10202698 rev_number: 9 eprint_status: archive userid: 699 dir: disk0/10/20/26/98 datestamp: 2025-01-03 17:02:03 lastmod: 2025-01-03 17:02:03 status_changed: 2025-01-03 17:02:03 type: article metadata_visibility: show sword_depositor: 699 creators_name: Lee, Siow Ming creators_name: Hewish, Madeleine creators_name: Ahmed, Samreen creators_name: Papadatos-Pastos, Dionysis creators_name: Karapanagiotou, Eleni creators_name: Blackhall, Fiona creators_name: Ford, Amy creators_name: Young, Robin creators_name: Garcia, Angel creators_name: Arora, Arvind creators_name: Hollingdale, Abigail creators_name: Ahmad, Tanya creators_name: Forster, Martin creators_name: Greystoke, Alastair creators_name: Bremner, Fion creators_name: Rudd, Robin creators_name: Farrelly, Laura creators_name: Vaja, Simran creators_name: Hackshaw, Allan title: 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 ispublished: pub divisions: UCL divisions: B02 divisions: C10 divisions: D19 divisions: H01 keywords: Autophagy inhibitor, Chemotherapy, Concurrent and maintenance treatment, Hydroxychloroquine, Randomised trial, Small cell lung cancer note: © 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/). abstract: 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. date: 2025-01-17 date_type: published publisher: Elsevier BV official_url: https://doi.org/10.1016/j.ejca.2024.115162 oa_status: green full_text_type: pub language: eng primo: open primo_central: open_green verified: verified_manual elements_id: 2344341 doi: 10.1016/j.ejca.2024.115162 medium: Print-Electronic pii: S0959-8049(24)01769-6 lyricists_name: Hackshaw, Allan lyricists_name: Lee, Siow-Ming lyricists_name: Vaja, Simran lyricists_id: AHACK11 lyricists_id: MSLEE39 lyricists_id: SVAJA98 actors_name: Hackshaw, Allan actors_id: AHACK11 actors_role: owner full_text_status: public publication: European Journal of Cancer volume: 215 article_number: 115162 event_location: England issn: 0959-8049 citation: Lee, Siow Ming; Hewish, Madeleine; Ahmed, Samreen; Papadatos-Pastos, Dionysis; Karapanagiotou, Eleni; Blackhall, Fiona; Ford, Amy; ... Hackshaw, Allan; + view all <#> Lee, Siow Ming; Hewish, Madeleine; Ahmed, Samreen; Papadatos-Pastos, Dionysis; Karapanagiotou, Eleni; Blackhall, Fiona; Ford, Amy; Young, Robin; Garcia, Angel; Arora, Arvind; Hollingdale, Abigail; Ahmad, Tanya; Forster, Martin; Greystoke, Alastair; Bremner, Fion; Rudd, Robin; Farrelly, Laura; Vaja, Simran; Hackshaw, Allan; - view fewer <#> (2025) 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. European Journal of Cancer , 215 , Article 115162. 10.1016/j.ejca.2024.115162 <https://doi.org/10.1016/j.ejca.2024.115162>. Green open access document_url: https://discovery.ucl.ac.uk/id/eprint/10202698/3/Hackshaw_1-s2.0-S0959804924017696-main.pdf