BMJ oncology最新文献

{"title":"Association of pre-existing cardiovascular disease with administration of fluoropyrimidine chemotherapy in patients with gastrointestinal malignancies.","authors":"Aderonke Temilade Abiodun, Chengsheng Ju, Catherine A Welch, Jennifer Lai, Freya Tyrer, Pinkie Chambers, Lizz Paley, Sally Vernon, John Deanfield, Mark de Belder, Mark Rutherford, Paul C Lambert, Sarah Slater, Kai Keen Shiu, Li Wei, Michael D Peake, David Adlam, Charlotte Manisty","doi":"10.1136/bmjonc-2024-000323","DOIUrl":"10.1136/bmjonc-2024-000323","url":null,"abstract":"<p><strong>Objective: </strong>Fluoropyrimidine chemotherapy is a first-line treatment for many gastrointestinal (GI) cancers, however, cardiotoxicity concerns may limit administration in patients with pre-existing cardiovascular disease (CVD). This study investigated the association of pre-existing CVD with use of fluoropyrimidine chemotherapy in tumour-eligible GI cancer patients.</p><p><strong>Methods and analysis: </strong>National cancer registry data from the Virtual Cardio-Oncology Research Initiative from England between 2014 and 2018 was used to identify GI cancer patients eligible to receive fluoropyrimidine chemotherapy. Linkage to Hospital Episode Statistics and CVD registry data were used to ascertain prior CVD and outcomes. Primary outcome was first administration of fluoropyrimidine chemotherapy following cancer diagnosis. Cox proportional hazard models determined HR and 95% CIs for the association between initiation of fluoropyrimidine treatment and prior CVD.</p><p><strong>Results: </strong>112 726 eligible patients were identified (median age 71 years (IQR 62-80), 39.7% female). 33 026 (29.3%) had pre-existing CVD. 73 392 (65.1%) patients had a diagnosis of colorectal, 23 208 (20.6%) oesophageal, 14 788 (13.1%) gastric and 1338 (1.2%) small bowel cancer. Individuals with pre-existing CVD had a 27% reduced rate of receiving fluoropyrimidine chemotherapy (HR, 0.73; 95% CI 0.70 to 0.75) on multivariable analysis. Significantly reduced rates of fluoropyrimidine administration were found across all subtypes of pre-existing CVD.</p><p><strong>Conclusions: </strong>GI cancer patients with all types of pre-existing CVD are less likely to receive fluoropyrimidine chemotherapy despite eligibility. This suggests widespread caution regarding administration of fluoropyrimidines across this population; further research is needed to assess whether such conservatism is justified.</p>","PeriodicalId":72436,"journal":{"name":"BMJ oncology","volume":"3 1","pages":"e000323"},"PeriodicalIF":0.0,"publicationDate":"2024-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11347681/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143070024","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Vitamin B6 status and chronic chemotherapy-induced peripheral neuropathy: a prospective cohort study among patients with non-metastatic colorectal cancer receiving oxaliplatin-based chemotherapy.","authors":"Lisanne Renting, Nienke R K Zwart, Per Magne Ueland, Adrian McCann, Arve Ulvik, Henk K van Halteren, Floor J E Lubberman, Renate M Winkels, Ellen Kampman, Dieuwertje E Kok","doi":"10.1136/bmjonc-2024-000462","DOIUrl":"10.1136/bmjonc-2024-000462","url":null,"abstract":"<p><strong>Objective: </strong>Chronic chemotherapy-induced peripheral neuropathy (CIPN) is a long-lasting side-effect of oxaliplatin. Vitamin B6 might play a role in the pathogenesis of CIPN. Therefore, we investigated associations between plasma vitamin B6 markers and the occurrence and severity of chronic CIPN in patients with non-metastatic colorectal cancer (CRC).</p><p><strong>Methods and analysis: </strong>242 patients with CRC receiving oxaliplatin-based chemotherapy were included. Blood samples were collected at diagnosis (ie, before chemotherapy), and 6 and 12 months after diagnosis (ie, during and after chemotherapy, respectively). Pyridoxal 5'-phosphate (PLP), pyridoxal (PL) and xanthurenic acid:3-hydroxykynurenine (XA:HK) ratio were measured as vitamin B6 markers using liquid chromatography tandem mass spectrometry. Chronic CIPN was assessed 12 months after diagnosis using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-CIPN twenty-item scale questionnaire. Prevalence ratios (PRs) and restricted cubic splines (RCSs) were used to assess associations with chronic CIPN occurrence, and linear regressions were used to assess associations with chronic CIPN severity. Analyses were adjusted for age, sex, smoking, alcohol consumption, diabetes and timing of chemotherapy (neoadjuvant/adjuvant/both).</p><p><strong>Results: </strong>Chronic CIPN was found in 80% (n=194) of patients. Higher PLP levels and XA:HK ratios during chemotherapy were associated with lower occurrence of chronic CIPN (PR_perdoubling 0.75, 95% CI 0.62 to 0.91 and P_RCS<0.05, respectively) and lower chronic CIPN severity (β_perdoubling -4.54, 95% CI -7.12 to -1.96 and β_perdoubling -6.30, 95% CI -9.53 to -3.07, respectively). No associations between PL levels and chronic CIPN were observed.</p><p><strong>Conclusion: </strong>Within this population, merely having PLP levels within the normal range, higher vitamin B6 status during chemotherapy was associated with lower occurrence and severity of chronic CIPN. Future research is warranted to investigate causality and the optimal vitamin B6 status during chemotherapy.</p>","PeriodicalId":72436,"journal":{"name":"BMJ oncology","volume":"3 1","pages":"e000462"},"PeriodicalIF":0.0,"publicationDate":"2024-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11347696/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143070005","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Choosing Wisely in oncology: are guidelines effective at preventing unnecessary diagnostics? The example of surveillance positron emission tomography for patients with localised colorectal cancer.","authors":"Daniel A Goldstein, Roi Tschernichovsky, Talish Razi, Keren Filosof, Idan Menashe, Ronen Arbel, Doron Netzer","doi":"10.1136/bmjonc-2024-000391","DOIUrl":"10.1136/bmjonc-2024-000391","url":null,"abstract":"<p><strong>Objective: </strong>Healthcare overuse is a major challenge for healthcare systems and patients worldwide. Professional guidelines such as the 'Choosing Wisely' guidelines have attempted to reduce specific examples of overuse. We examined the use of surveillance positron emission tomography CT (PETCT) in patients with colorectal cancer (CRC) treated with curative intent.</p><p><strong>Methods and analysis: </strong>We used the large Clalit Health Services dataset in Israel to identify patients with CRC who received adjuvant chemotherapy between January 2017 and December 2021. We examined the number of PETCTs performed for each patient.</p><p><strong>Results: </strong>We included 1799 patients in our study cohort. We distinguished localised from metastatic cases based on specific drugs administered or not administered during the follow-up period (ie, biologics). For the entire cohort, the median number of PETCTs performed per patient over the study period was 3364 (20.2%) patients underwent a single PETCT, 946 (52.6%) patients underwent ≥2 PETCTs and 25 patients underwent ≥10 PETCTs. If none or a single PETCT is considered 'guideline-concordant' during diagnosis and treatment of localised CRC, 69% of 4231 PETCTs performed were 'guideline-discordant'.</p><p><strong>Conclusion: </strong>Despite the professional guidelines recommending against routine PETCT to monitor for recurrence following curative-intent treatment of CRC, there remains a large volume of guideline-discordant PETCTs, constituting healthcare overuse of an expensive diagnostic procedure.</p>","PeriodicalId":72436,"journal":{"name":"BMJ oncology","volume":"3 1","pages":"e000391"},"PeriodicalIF":0.0,"publicationDate":"2024-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11347680/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143069659","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Recall of ibrutinib and issues with therapeutic approval.","authors":"Colton Lipfert, Myung Sun Kim, Alyson Haslam, Vinay K Prasad","doi":"10.1136/bmjonc-2024-000418","DOIUrl":"10.1136/bmjonc-2024-000418","url":null,"abstract":"","PeriodicalId":72436,"journal":{"name":"BMJ oncology","volume":"3 1","pages":"e000418"},"PeriodicalIF":0.0,"publicationDate":"2024-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11347682/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143069952","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Deciphering chemotherapy resistance: a novel apoptosis protein profile analysis in stage II colorectal cancer.","authors":"Zixiang Li, Leilei Fu","doi":"10.1136/bmjonc-2024-000460","DOIUrl":"10.1136/bmjonc-2024-000460","url":null,"abstract":"","PeriodicalId":72436,"journal":{"name":"BMJ oncology","volume":"3 1","pages":"e000460"},"PeriodicalIF":0.0,"publicationDate":"2024-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11347687/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143069872","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Integrated multiplex analysis of cell death regulators in stage II colorectal cancer suggests patients with 'persister' cell profiles fail to benefit from adjuvant chemotherapy.","authors":"Sanghee Cho, Elizabeth McDonough, John Graf, Jinru Shia, Canan Firat, Nil Urganci, Christine Surrette, Andreas Lindner, Manuela Salvucci, Anna Matveeva, Batuhan Kisakol, Anthony O'Grady, Mohammadreza Azimi, John P Burke, Deborah A McNamara, Simon McDade, Daniel B Longley, Jochen Hm Prehn, Fiona Ginty","doi":"10.1136/bmjonc-2024-000362","DOIUrl":"10.1136/bmjonc-2024-000362","url":null,"abstract":"<p><strong>Objective: </strong>Inducing tumour cell apoptosis is a primary objective of chemotherapy but, to date, there are no validated biomarkers of apoptosis sensitivity or resistance. Our objective was to image multiple apoptosis pathway proteins at single cell level and determine multi-protein associations with recurrence risk and chemotherapy response in patients with stage II colorectal cancer (CRC).</p><p><strong>Methods and analysis: </strong>Multiplexed imaging of 16 proteins in the intrinsic and extrinsic apoptosis pathways at single cell resolution on resected tissue from 194 patients with stage II CRC who either received adjuvant chemotherapy (n<i>=</i>108) or were treated with surgery only (n=86). K-means clustering of >600 000 cancer cells and cell level intensities of APAF1, procaspase-9, procaspase-3, XIAP, SMAC, BAX, BAK, BCL2, BCL-XL, MCL-1, procaspase-8, BID, FADD, FLIP, RIP3 and CIAP1 identified distinct cell cluster profiles.</p><p><strong>Results: </strong>Chemotherapy-treated patients with a higher percentage of cell clusters with low procaspase-3 and high XIAP had a higher risk of recurrence. This was validated in an independent cohort of adjuvant chemotherapy-treated high-risk patients with stage II CRC. We also applied two established system models of apoptosis initiation and execution to estimate cellular apoptosis sensitivity and show that these cell clusters do not appear to have impaired mitochondrial outer membrane permeabilisation sensitivity, but downstream procaspase-3 cleavage is compromised. This represents a key characteristic of drug-tolerant 'persister' cells.</p><p><strong>Conclusion: </strong>This study represents the most comprehensive analysis to date of apoptosis protein distribution at single cell level in CRC tumours. Our study identifies a subgroup of patients with stage II CRC with an apoptosis-resistant 'persister' cell profile who do not benefit from adjuvant chemotherapy.</p>","PeriodicalId":72436,"journal":{"name":"BMJ oncology","volume":"3 1","pages":"e000362"},"PeriodicalIF":0.0,"publicationDate":"2024-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11347685/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143069917","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Preconsultation compassion video to reduce anxiety among patients referred to a cancer centre: a randomised control trial.","authors":"Christine Winn, Generosa Grana, Anthony Mazzarelli, Andrea Nicholson, Kristine Mykulowycz, Chidinma Obiakor, Alicia Bair, Stephen Trzeciak, Brian Roberts","doi":"10.1136/bmjonc-2024-000427","DOIUrl":"10.1136/bmjonc-2024-000427","url":null,"abstract":"<p><strong>Objective: </strong>Anxiety is common among patients attending an initial oncology consultation. The objective of this trial was to test if an enhanced compassion video emailed to patients prior to their initial oncology consultation reduces anxiety compared with being sent an information-only introduction video.</p><p><strong>Methods and analysis: </strong>We conducted a randomised control trial at a single university-based cancer centre between May 2021 and October 2023. We enrolled adult patients scheduled for an initial cancer consultation. Subjects underwent simple 1:1 randomisation to receive either a standard introduction video or an enhanced compassion video via email. Investigators and subjects were blinded to allocation. The primary outcome was degree of anxiety on arrival to the initial oncology consultation, measured using the Hospital Anxiety and Depression scale (HADS).</p><p><strong>Results: </strong>Of 1005 subjects randomised to the standard video and 1038 to the enhanced compassion video, 183 and 179 subjects completed the HADS-anxiety in each group, respectively. Only 25% reported watching their assigned video. There was no difference in degree of anxiety between the standard or compassion video groups using intention to treat analysis (median (IQR) 7 (4-10) vs 7 (4-10), p value=0.473)) or per-protocol analysis (limited to subjects who reported watching the video) (median (IQR) 7 (4-10) (n=45) vs 7 (5-10) (n=46), p value=0.997).</p><p><strong>Conclusion: </strong>Receiving an enhanced compassion video did not reduce anxiety compared with a standard introduction video. Given 25% of subjects reported watching their assigned video, future research should focus on identifying interventions at the point-of-care to reduce anxiety.</p><p><strong>Trial registration number: </strong>NCT04503681.</p>","PeriodicalId":72436,"journal":{"name":"BMJ oncology","volume":"3 1","pages":"e000427"},"PeriodicalIF":0.0,"publicationDate":"2024-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11347698/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143069934","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Preclinical study and parallel phase II trial evaluating antisense STAT3 oligonucleotide and checkpoint blockade for advanced pancreatic, non-small cell lung cancer and mismatch repair-deficient colorectal cancer.","authors":"Chad Tang, Genevieve P Hartley, Coline Couillault, Ying Yuan, Heather Lin, Courtney Nicholas, Anupallavi Srinivasamani, James Dai, Ecaterina E Ileana Dumbrava, Siqing Fu, Daniel D Karp, Aung Naing, Sarina A Piha-Paul, Jordi Rodon Ahnert, Shubham Pant, Vivek Subbiah, Timonthy A Yap, Apostolia M Tsimberidou, Paola Guerrero, Sarah Dhebat, Theresa Proia, Michael A Curran, David S Hong","doi":"10.1136/bmjonc-2023-000133","DOIUrl":"10.1136/bmjonc-2023-000133","url":null,"abstract":"<p><strong>Objective: </strong>To evaluate signal transducer and activator of transcription 3 (STAT3) inhibition we conducted a co-clinical trial testing danvatirsen, a STAT3 antisense oligonucleotide (ASO) and checkpoint inhibition in conjunction with preclinical experiments.</p><p><strong>Methods and analysis: </strong>Orthotopically implanted pancreatic cancer (pancreatic adenocarcinoma (PDAC)) was treated with STAT3 ASO with immune checkpoint inhibition. Tumour infiltrating immune cell populations were characterised via flow cytometry. In vitro experiments evaluated STAT3 inhibition in pancreatic stellate cells (PSCs) and myeloid-derived suppressor cells (MDSCs).A phase II trial employing a Simon II stage design tested the clinical efficacy of danvatirsen and durvalumab in non-small cell lung cancer (NSCLC), PDAC and mismatch repair-deficient colorectal cancer (MRD CRC). The primary objective was 4-month disease control rate (DCR).</p><p><strong>Results: </strong>In vivo studies identified improvement in survival of PDAC implanted mice treated with STAT3 ASO and checkpoint inhibition. Within tumour-infiltrating lymphocytes there was expansion of CD4 and PD-1+ CD8 populations with STAT3 ASO.Thirty-seven patients (29 PDAC, 7 NSCLC and 1 MRD CRC) from a single institution started treatment on trial between April 2017 and March 2020. No objective responses were observed. Four of six (66.7%, 95% CI 22.3% to 95.7%) NSCLC and 4 of 23 (17.4%, 95% CI 5% to 38.8%) PDAC patients exhibited 4-month DCR. Follow-up in vitro studies revealed an anti-inflammatory and pro-tumour effect of STAT3 ASO mediated by PSCs and MDSCs distinct from ablation of STAT3.</p><p><strong>Conclusion: </strong>Although durvalumab and danvatirsen met the primary endpoint, no objective responses were observed. A rationale for the lack of objective responses is danvatirsen-induced myeloid immune suppression.</p><p><strong>Trial registration number: </strong>NCT02983578.</p>","PeriodicalId":72436,"journal":{"name":"BMJ oncology","volume":"3 1","pages":"e000133"},"PeriodicalIF":0.0,"publicationDate":"2024-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11347683/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143069948","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Surgical management of skull base chordomas and chondrosarcomas: insights from a national cohort study.","authors":"Laurence J Glancz, Cathal John Hannan, Alexandros Vyziotis, Gillian M Potter, Rekha Siripurapu, Raj K Bhalla, Scott A Rutherford, Andrew Thomas King, Charlotte Hammerbeck-Ward, Adrian Crellin, Shermaine Pan, Rovel Colaco, Gillian A Whitfield, Omar N Pathmanaban","doi":"10.1136/bmjonc-2024-000386","DOIUrl":"10.1136/bmjonc-2024-000386","url":null,"abstract":"<p><strong>Objective: </strong>Skull base chordoma and chondrosarcoma are distinct sarcomas of the skull base but share significant therapeutic challenges due to their proximity to critical neurovascular structures, making surgical resection difficult. We sought to establish factors associated with outcome predictors in a national cohort of patients.</p><p><strong>Methods and analysis: </strong>Data for all patients referred with a diagnosis of skull base chordoma or chondrosarcoma from April 2017 to December 2022 were obtained. We performed analyses of data pertaining to the first cohort of patients treated in the UK with proton beam therapy (PBT) to determine factors associated with obtaining gross total resection (GTR) and adequate clearance of the brainstem and optic apparatus.</p><p><strong>Results: </strong>Of 230 patients with skull base chordoma or chondrosarcoma referred for PBT, 71% were accepted for PBT, with a wide regional variation between referring neurosurgical units (29%-93%). Of the first 75 consecutive patients treated with PBT, the only factor predictive of obtaining GTR was surgical resection at a unit with higher volumes of patients accepted for PBT (OR 1.32, 95% CI 1.11 to 1.63, p=0.004). Use of intraoperative MRI (OR 4.84, 95% CI 1.21 to 27.83, p=0.04) and resection at a higher volume unit (OR 1.29, 95% CI 1.07 to 1.64, p=0.013) were associated with increased rates of tumour clearance from the brainstem/optic apparatus.</p><p><strong>Conclusions: </strong>Treatment at a higher volume centre was a key determinant of the optimal surgical outcome in this cohort. These data support the management of skull base chordomas and chondrosarcomas in higher volume centres where multidisciplinary experience can be accumulated.</p>","PeriodicalId":72436,"journal":{"name":"BMJ oncology","volume":"3 1","pages":"e000386"},"PeriodicalIF":0.0,"publicationDate":"2024-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11347697/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143069957","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Health-related quality of life outcomes reporting associated with FDA approvals in haematology and oncology","authors":"Medhavi Gupta, O. Akhtar, B. Bahl, Angel Mier-Hicks, Kristopher Attwood, Kayla Catalfamo, B. Gyawali, P. Torka","doi":"10.1136/bmjonc-2024-000369","DOIUrl":"https://doi.org/10.1136/bmjonc-2024-000369","url":null,"abstract":"Health-related quality of life (HRQoL) outcomes are important in making clinical and policy decisions. This study aimed to examine the HRQoL reporting in cancer drug trials leading to Food and Drug Administration (FDA) approvals.This retrospective cohort study analysed HRQoL data for trials leading to FDA approvals between July 2015 and May 2020. Proportion of included trials that reported HRQoL, latency between FDA approval and first report of HRQoL data, HRQoL outcomes, and their correlation with OS (overall survival) and PFS (progression-free survival) were analysed.Of the 233 trials associated with 207 FDA approvals, HRQoL was reported in 50% of trials, of which only 42% had the data reported by the time of FDA approval. There were no changes in frequency of HRQoL reporting between 2015 and 2020. HRQoL data were first reported in the primary publication in only 30% trials. Of the 115 trials with HRQoL data available, HRQoL improved in 43%, remained stable in 53% and worsened in 4% of trials. Among the trials that led to FDA approvals based on surrogate endpoints (79%), HRQoL was reported in 45% and improved only in 18% trials. There was no association between OS and PFS benefit and HRQoL outcomes.Rates of HRQoL reporting were suboptimal in trials that led to FDA approvals with no improvements seen between 2015 and 2020. HRQoL reporting was often delayed and not presented in the primary publication. HRQoL reporting was further sparse in trials with approvals based on surrogate endpoints and HRQoL improved in only a minority of them.","PeriodicalId":72436,"journal":{"name":"BMJ oncology","volume":"52 10","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141696332","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}