The Lancet Oncology最新文献

{"title":"Using patient-reported outcomes and health-related quality of life data in regulatory decisions on cancer treatment: highlights from an EMA-EORTC workshop","authors":"Madeline Pe, Caroline Voltz-Girolt, Jill Bell, Vishal Bhatnagar, Jan Bogaerts, Christopher Booth, Juan Garcia Burgos, Joseph C Cappelleri, Corneel Coens, Pierre Demolis, Harald Enzmann, Johannes M Giesinger, Alexandra Gilbert, Mogens Groenvold, Paul Kluetz, Claire Piccinin, Douwe Postmus, Chantal Quinten, Bettina Ryll, Maxime Sasseville, Peter Mol","doi":"10.1016/s1470-2045(25)00150-0","DOIUrl":"https://doi.org/10.1016/s1470-2045(25)00150-0","url":null,"abstract":"<h2>Section snippets</h2><section><section><h2>Well-defined PRO research objectives</h2>A commonly shared view across various international stakeholders was that PROs intended to provide quantitative assessment of clinical outcomes should be treated like any other endpoint that is included in the evaluation of a cancer treatment. Thus, an important first step is to clearly describe the research questions that PROs can address to support the evaluation of cancer treatments. PROs are not an outcome but a way to measure an outcome; therefore, it is important to identify in the</section></section><section><section><h2>Role of submitted PRO data for decision making</h2>Further optimisation in the use of PROs is necessary to fully leverage the insights patient-generated data can provide.4, 5</section></section><section><section><h2>Support overall benefit–risk evaluation</h2>PROs can quantify symptoms and functional aspects of how patients experience and respond to their treatment and can complement traditional clinical endpoints such as overall survival, progression-free survival, and tumour response measures. PRO data can reflect treatment efficacy (ie, improvement in disease-related symptoms) or harms (ie, emergence of symptomatic adverse events and their impact on functioning). By incorporating these additional outcomes into clinical trials, a more holistic</section></section><section><section><h2>Further characterise tolerability</h2>One research objective that is relevant across early phase and late phase clinical cancer trials is to characterise safety and tolerability. It has been proposed that a complete understanding of tolerability should include direct measurement from the patient on how they are feeling and functioning when on treatment.<sup>6</sup> For example, patient-reported symptomatic adverse events can complement standard safety reporting by clinicians. Understanding treatment tolerability can help corroborate or refine</section></section><section><section><h2>Product information and label</h2>A common goal for commercial sponsors is to use PROs to support medicines' approval, labelling, or marketing claims of treatment benefit. However, methodological issues, PRO data quality (including high rates of missing data or asymmetric missing data), and the question of what makes a clinically relevant PRO result have often prevented their inclusion in the product label (eg, EU Summary of Product Characteristics).<sup>1</sup> Development of PRO standards to address these methodological issues is</section></section><section><section><h2>PROs, including HRQOL, are a crucial endpoint from the HTA's and payers' perspectives</h2>PROs can help assess the overall value of a new treatment by considering patient-reported experiences. A crucial consideration for HTA decisions is that the data can address questions on comparative effectiveness versus standard of care. In some health-care systems, PROs also inform cost–benefit considerations of alterna","PeriodicalId":22865,"journal":{"name":"The Lancet Oncology","volume":"6 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143832146","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Use of patient-reported outcomes to inform symptom and functional outcomes in cancer drug regulatory decisions: challenges and future directions","authors":"Francesco Pignatti, Peter Mol, Chantal Quinten, Douwe Postmus, Anja Schiel, Maxime Sasseville, Shun Tezuka, Vishal Bhatnagar, Paul Kluetz","doi":"10.1016/s1470-2045(25)00151-2","DOIUrl":"https://doi.org/10.1016/s1470-2045(25)00151-2","url":null,"abstract":"No Abstract","PeriodicalId":22865,"journal":{"name":"The Lancet Oncology","volume":"13 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143832277","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Living within our means: trouble ahead for England's cancer planning","authors":"Mark Lawler, Pat Price, Richard Sullivan","doi":"10.1016/s1470-2045(25)00202-5","DOIUrl":"https://doi.org/10.1016/s1470-2045(25)00202-5","url":null,"abstract":"No Abstract","PeriodicalId":22865,"journal":{"name":"The Lancet Oncology","volume":"40 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143832274","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Belzutifan for von Hippel-Lindau disease-associated renal cell carcinoma and other neoplasms (LITESPARK-004): 50 months follow-up from a single-arm, phase 2 study","authors":"Ramaprasad Srinivasan, Othon Iliopoulos, Kathryn E Beckermann, Vivek Narayan, Benjamin L Maughan, Stephane Oudard, Tobias Else, Jodi K Maranchie, Ane B Iversen, Jerry Cornell, Rodolfo F Perini, Yanfang Liu, W Marston Linehan, Eric Jonasch","doi":"10.1016/s1470-2045(25)00099-3","DOIUrl":"https://doi.org/10.1016/s1470-2045(25)00099-3","url":null,"abstract":"<h3>Background</h3>Hypoxia-inducible factor-2α inhibitor belzutifan is approved for von Hippel-Lindau disease-associated renal cell carcinoma, CNS haemangioblastomas, and pancreatic neuroendocrine tumours, based on previously published initial results from the LITESPARK-004 study. Updated results are presented here after a median follow-up of nearly 50 months.<h3>Methods</h3>In this single-arm, phase 2 study, participants were enrolled at 11 centres in Denmark, France, the UK, and the USA. Oral belzutifan 120 mg once daily was given to eligible adults aged 18 years or older with a diagnosis of von Hippel-Lindau disease (based on germline <em>VHL</em> alterations), at least one measurable renal cell carcinoma tumour, no tumour larger than 3 cm that necessitated immediate surgery, no metastatic disease, no previous systemic anticancer treatment, and an Eastern Cooperative Oncology Group performance status score of 0 or 1. The primary endpoint was the proportion of participants with an objective response in von Hippel-Lindau disease-associated renal cell carcinoma per Response Evaluation Criteria in Solid Tumours, version 1.1, determined by an independent review committee, and assessed in all participants who received at least one dose of belzutifan. This ongoing study is no longer recruiting and is registered at <span><span>ClinicalTrials.gov</span><svg aria-label=\"Opens in new window\" focusable=\"false\" height=\"20\" viewbox=\"0 0 8 8\"><path d=\"M1.12949 2.1072V1H7V6.85795H5.89111V2.90281L0.784057 8L0 7.21635L5.11902 2.1072H1.12949Z\"></path></svg></span>, <span><span>NCT03401788</span><svg aria-label=\"Opens in new window\" focusable=\"false\" height=\"20\" viewbox=\"0 0 8 8\"><path d=\"M1.12949 2.1072V1H7V6.85795H5.89111V2.90281L0.784057 8L0 7.21635L5.11902 2.1072H1.12949Z\"></path></svg></span>.<h3>Findings</h3>Between May 31, 2018, and Mar 29, 2019, 61 participants were enrolled; 36 (59%) were continuing treatment as of April 3, 2023. The median age of all enrolled participants was 41·0 years (IQR 29·0–51·0); 32 (52%) of 61 participants were male and 29 (48%) were female; most were White (n=55; 90%). Median study follow-up was 49·9 months (IQR 48·9–52·2). 41 (67%; 95% CI 54–79) of 61 participants with renal cell carcinoma had an objective response; seven (11%) had a complete response and 34 (56%) a partial response. 13 grade 3 treatment-related adverse events occurred in 11 (18%) participants (anaemia: seven [11%]; fatigue: three [5%]; urinary tract infection: one [2%]; hypoxia: one [2%]; and blister: one [2%]). None of the participants had a grade 4 or 5 treatment-related adverse event. Four (7%) participants had serious treatment-related adverse events (one participant each: anaemia, urinary tract infection, intracranial haemorrhage, and hypoxia).<h3>Interpretation</h3>Updated results support the use of belzutifan as systemic treatment for von Hippel-Lindau disease-associated renal cell carcinoma.<h3>Funding</h3>Merck Sharp & Dohme, a subsidiary of Merc","PeriodicalId":22865,"journal":{"name":"The Lancet Oncology","volume":"309 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143822729","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Belzutifan in von Hippel-Lindau disease","authors":"Chiara Ciccarese, Roberto Iacovelli, Giampaolo Tortora","doi":"10.1016/s1470-2045(25)00153-6","DOIUrl":"https://doi.org/10.1016/s1470-2045(25)00153-6","url":null,"abstract":"No Abstract","PeriodicalId":22865,"journal":{"name":"The Lancet Oncology","volume":"75 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143822733","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"NHS investment boosts cancer detection for 80 000 patients","authors":"Elizabeth Gourd","doi":"10.1016/s1470-2045(25)00218-9","DOIUrl":"https://doi.org/10.1016/s1470-2045(25)00218-9","url":null,"abstract":"No Abstract","PeriodicalId":22865,"journal":{"name":"The Lancet Oncology","volume":"108 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143819981","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Bortezomib, melphalan, and prednisone with or without daratumumab in transplant-ineligible patients with newly diagnosed multiple myeloma (ALCYONE): final analysis of an open-label, randomised, multicentre, phase 3 trial","authors":"Maria-Victoria Mateos, Jesus San-Miguel, Michele Cavo, Kenshi Suzuki, Andrzej Jakubowiak, Stefan Knop, Chantal Doyen, Paulo Lucio, Zsolt Nagy, Ludek Pour, Sebastian Grosicki, Andre Crepaldi, Anna Marina Liberati, Philip Campbell, Sung-Soo Yoon, Genadi Iosava, Tomoaki Fujisaki, Mamta Garg, Mai Ngo, Eva G Katz, Meletios A Dimopoulos","doi":"10.1016/s1470-2045(25)00018-x","DOIUrl":"https://doi.org/10.1016/s1470-2045(25)00018-x","url":null,"abstract":"<h3>Background</h3>In the phase 3 ALCYONE study, the addition of daratumumab to bortezomib, melphalan, and prednisone (D-VMP) significantly improved outcomes in transplant-ineligible patients with newly diagnosed multiple myeloma. Here, we present results from the final analysis of ALCYONE.<h3>Methods</h3>ALCYONE was an international, multicentre, randomised, open-label, active-controlled, phase 3 trial in adults aged 18 years or older with newly diagnosed multiple myeloma who were ineligible for high-dose chemotherapy with autologous stem-cell transplantation, because of their age (≥65 years) or presence of substantial comorbidities, and had an Eastern Cooperative Oncology Group performance status of 0–2. Patients were enrolled between Feb 9, 2015, and July 14, 2016, and were randomly assigned (1:1) by randomly permuted blocks using an interactive web-based randomisation system to receive bortezomib, melphalan, and prednisone (VMP) alone or D-VMP, with randomisation stratified by International Staging System disease stage, geographical region, and age. Patients received up to nine 6-week cycles of subcutaneous bortezomib (1·3 mg/m<sup>2</sup> of body surface area, twice per week on weeks 1, 2, 4, and 5 of cycle 1 and once weekly on weeks 1, 2, 4, and 5 of cycles 2–9), oral melphalan (9 mg/m<sup>2</sup>, once daily on days 1–4 of each cycle), and oral prednisone (60 mg/m<sup>2</sup>, once daily on days 1–4 of each cycle). Patients in the D-VMP group also received intravenous daratumumab at a dose of 16 mg/kg once weekly during cycle 1, once every 3 weeks in cycles 2–9, and once every 4 weeks thereafter until disease progression, unacceptably toxicity, or the end of study. The primary endpoint, progression-free survival, has been previously reported. The ALCYONE study has completed; presented here are final analyses for selected secondary endpoints related to overall survival, depth of response, subsequent therapy, and safety. The intention-to-treat population was the primary analysis population (including for overall survival), defined as all patients who were randomly assigned to study treatment. The safety population, consisting of patients who received any dose of study treatment, was used in safety analyses. This trial is registered with <span><span>ClinicalTrials.gov</span><svg aria-label=\"Opens in new window\" focusable=\"false\" height=\"20\" viewbox=\"0 0 8 8\"><path d=\"M1.12949 2.1072V1H7V6.85795H5.89111V2.90281L0.784057 8L0 7.21635L5.11902 2.1072H1.12949Z\"></path></svg></span>, <span><span>NCT02195479</span><svg aria-label=\"Opens in new window\" focusable=\"false\" height=\"20\" viewbox=\"0 0 8 8\"><path d=\"M1.12949 2.1072V1H7V6.85795H5.89111V2.90281L0.784057 8L0 7.21635L5.11902 2.1072H1.12949Z\"></path></svg></span>.<h3>Findings</h3>In total, 706 patients were enrolled and randomly assigned to receive D-VMP (n=350) or VMP (n=356). Baseline characteristics were balanced between the two treatment groups; most participants were female (379 [54%] of 706 ","PeriodicalId":22865,"journal":{"name":"The Lancet Oncology","volume":"5 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143813914","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"US funding cuts impact cancer care and research","authors":"Sharmila Devi","doi":"10.1016/s1470-2045(25)00208-6","DOIUrl":"https://doi.org/10.1016/s1470-2045(25)00208-6","url":null,"abstract":"No Abstract","PeriodicalId":22865,"journal":{"name":"The Lancet Oncology","volume":"108 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143775965","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"WHO Classification of Tumours: evolution of a global resource in the molecular era","authors":"Jennelle C Hodge, George J Netto, Bharat Rekhi, Wendy A Cooper, Michael Eden, Andrew S Field, Vicky Goh, James G Kench, Joseph D Khoury, Katia R M Leite, Zhiyong Liang, Daichi Maeda, Miguel Reyes-Múgica, Brian Rous, Aleš Ryška, Shahin Sayed, Antonia Sepulveda, Chanjuan Shi, Gary Tse, Peter Schirmacher, Dilani Lokuhetty","doi":"10.1016/s1470-2045(24)00709-5","DOIUrl":"https://doi.org/10.1016/s1470-2045(24)00709-5","url":null,"abstract":"No Abstract","PeriodicalId":22865,"journal":{"name":"The Lancet Oncology","volume":"12 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143745370","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"First-line talazoparib plus enzalutamide versus placebo plus enzalutamide for metastatic castration-resistant prostate cancer: patient-reported outcomes from the randomised, double-blind, placebo-controlled, phase 3 TALAPRO-2 trial","authors":"Nobuaki Matsubara, Arun A Azad, Neeraj Agarwal, Fred Saad, Ugo De Giorgi, Jae Young Joung, Peter C C Fong, Robert J Jones, Stefanie Zschäbitz, Jan Oldenburg, Neal D Shore, Curtis Dunshee, Joan Carles, Andre P Fay, Paul Cislo, Jane Chang, Cynthia G Healy, Alexander Niyazov, Karim Fizazi","doi":"10.1016/s1470-2045(25)00030-0","DOIUrl":"https://doi.org/10.1016/s1470-2045(25)00030-0","url":null,"abstract":"<h3>Background</h3>Patients with metastatic castration-resistant prostate cancer have poor prognoses, underscoring the need for novel therapeutic strategies. First-line talazoparib plus enzalutamide significantly improved radiographic progression-free survival compared with placebo plus enzalutamide in men with metastatic castration-resistant prostate cancer in the phase 3 TALAPRO-2 study. We aimed to evaluate patient-reported outcomes in the all-comers cohort of TALAPRO-2, which included patients with and without alterations in homologous recombination repair (HRR) genes.<h3>Methods</h3>TALAPRO-2 is a randomised, double-blind, placebo-controlled, phase 3 trial conducted at 223 hospitals, cancer centres, and medical centres in 26 countries worldwide. Eligible participants were male patients aged 18 years or older (≥20 years in Japan) who were receiving ongoing androgen deprivation therapy, had asymptomatic or mildly symptomatic metastatic castration-resistant prostate cancer, Eastern Cooperative Oncology Group performance status of 0 or 1, and had not received previous life-prolonging systemic therapy for castration-resistant prostate cancer or metastatic castration-resistant prostate cancer. Patients were randomly assigned (1:1) using a centralised interactive web response system and a permuted block size of 4 to oral talazoparib 0·5 mg once daily or placebo, plus oral enzalutamide 160 mg once daily. The funder, patients, and investigators were masked to allocation of talazoparib or placebo; enzalutamide was open-label. Stratification factors were HRR gene alteration status (deficient vs non-deficient or unknown) and previous treatment with docetaxel or abiraterone, or both (yes vs no) in the castration-sensitive setting. The primary endpoint was radiographic progression-free survival by blinded independent central review and has been reported previously. Patient-reported outcomes were assessed as secondary endpoints in the patient-reported outcomes population, which comprised patients from the intention-to-treat population with a baseline patient-reported outcome assessment followed by at least one post-baseline patient-reported outcome assessment. Patient-reported outcomes included mean change from baseline in patient-reported pain symptoms (per Brief Pain Inventory-Short Form [BPI-SF]); global health status/quality of life (GHS/QoL), overall cancer and prostate cancer-specific functioning and symptoms (per European Organisation for Research and Treatment of Cancer [EORTC] Core Quality of Life Questionnaire [QLQ-C30] and Quality of Life Questionnaire-Prostate [QLQ-PR25]); and general health status (per EQ-5D-5L). Time to deterioration in patient-reported pain symptoms (per BPI-SF), and time to definitive deterioration in patient-reported GHS/QoL (per EORTC QLQ-C30) and prostate cancer-specific urinary symptoms (per EORTC-QLQ-PR25) were the other secondary endpoints. This study is registered with <span><span>ClinicalTrials.gov</span><svg aria-l","PeriodicalId":22865,"journal":{"name":"The Lancet Oncology","volume":"19 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143745531","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}