Inadequate health-related quality of life assessment and reporting in phase III clinical trials of immune checkpoint inhibitors in solid cancers: A systematic review

Servetto, Alberto; Salomone, Fabio; Costanzo, Fabrizio Di; Iuliano, Rossella; Marandino, Laura; Napolitano, Fabiana; Santaniello, Antonio; Placido, Pietro De; Placido, Sabino De; Maïo, Massimo Di; Formisano, Luigi; Bianco, Roberto

doi:10.1016/j.critrevonc.2022.103649

Cited by 14 publications

(12 citation statements)

References 26 publications

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“…This is particularly evident for trials testing immunotherapy, as previously shown by a dedicated analysis. 15 In this study, immunotherapy studies confirmed a high rate of inclusion of QoL among endpoints, but a very disappointing rate of presence of QoL data in the primary publication.…”

Section: Discussionmentioning

confidence: 52%

“…For each study, details about publication (journal, year, first author, date of definitive and ahead-of-print publication, availability of online supplemental material and/ or study protocol) were collected. Impact factor (IF) corresponding to the year of publication was considered, according to the Journal of Citation Reports, and papers were conventionally divided into three IF categories: low (<15), intermediate (15)(16)(17)(18)(19)(20)(21)(22)(23)(24)(25)(26)(27)(28)(29)(30) and high (>30). These cutoffs were based on the IQR of the studies included in the 2012-2016 analysis, and for homogeneity, we maintained the same cut-offs for the 2017-2021 period.…”

Section: Methodsmentioning

confidence: 99%

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Time trends in health-related quality of life assessment and reporting within publications of oncology randomised phase III trials: a meta-research study

Marandino

Trastu

Ghisoni

et al. 2023

bmjonc

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ObjectiveTo assess time trends in the inclusion of health-related quality of life (QoL) among study endpoints and in the reporting of QoL results in study publications, randomised phase III oncology trials published between 2017 and 2021 were compared with the trials published in the previous 5 years.Methods and analysisAll issues published between 2012 and 2021 by 11 major journals were handsearched for primary publications of phase III trials in adult patients with solid tumours. Trials published in 2017–2021 were compared with trials published in 2012–2016 for three endpoints: (1) proportion of publications including QoL among endpoints out of all the eligible publications; (2) proportion of publications presenting QoL results out of those including QoL among endpoints and (3) proportion of publications presenting QoL data out of all the eligible publications.Results388 publications between 2017 and 2021 were eligible and compared with 446 publications between 2012 and 2016. QoL was included among endpoints in 67.8% of trials in 2017–2021 vs 52.9% in 2012–2016 (univariate OR 1.87, 95% CI 1.41 to 2.48, p<0.001). QoL results were available in 52.1% in 2017–2021 vs 62.3% in 2012–2016 of primary publications of trials including QoL among endpoints (OR 0.66, 95% CI 0.46 to 0.94, p=0.02). Overall, QoL was analysed and presented in 35.3% of primary publications in 2017–2021 vs 33.0% in 2012–2016 (OR 1.11, 95% CI 0.83 to 1.48, p=0.48).ConclusionsThe proportion of oncology trials including QoL among endpoints increased in 2017–2021 compared with 2012–2016. However, the proportion of primary publications reporting QoL results remains suboptimal.

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Section: Discussionmentioning

confidence: 52%

Section: Methodsmentioning

confidence: 99%

Time trends in health-related quality of life assessment and reporting within publications of oncology randomised phase III trials: a meta-research study

Marandino

Trastu

Ghisoni

et al. 2023

bmjonc

Self Cite

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“…Unfortunately, a substantial proportion of published RCTs (both for solid tumors and hematologic malignancies) have failed to report adequate information about PRO methodology, thereby precluding the uptake of PRO results by the scientific community and the impact of these data on real-world practice. 27,53,[124][125][126][127] As PROs are often secondary endpoints, it is not uncommon to see this data being disclosed in a separate manuscript (other than the primary trial publication reporting efficacy and safety results). Although this strategy may be acceptable and may have the advantage of allowing sufficient space to provide all necessary methodological reporting, including appropriateness of analyses and interpretation of data, a timely PRO report is essential to allow readers to understand the overall value of a new drug being tested in an RCT.…”

Section: The Importance Of Accurate and Timely Reporting Of Pro Resultsmentioning

confidence: 99%

Toward a more patient‐centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS)

Efficace,

Buckstein,

Abel

et al. 2024

HemaSphere

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Notable treatment advances have been made in recent years for patients with myelodysplastic syndromes/neoplasms (MDS), and several new drugs are under development. For example, the emerging availability of oral MDS therapies holds the promise of improving patients' health‐related quality of life (HRQoL). Within this rapidly evolving landscape, the inclusion of HRQoL and other patient‐reported outcomes (PROs) is critical to inform the benefit/risk assessment of new therapies or to assess whether patients live longer and better, for what will likely remain a largely incurable disease. We provide practical considerations to support investigators in generating high‐quality PRO data in future MDS trials. We first describe several challenges that are to be thoughtfully considered when designing an MDS‐focused clinical trial with a PRO endpoint. We then discuss aspects related to the design of the study, including PRO assessment strategies. We also discuss statistical approaches illustrating the potential value of time‐to‐event analyses and their implications within the estimand framework. Finally, based on a literature review of MDS randomized controlled trials with a PRO endpoint, we note the PRO items that deserve special attention when reporting future MDS trial results. We hope these practical considerations will facilitate the generation of rigorous PRO data that can robustly inform MDS patient care and support treatment decision‐making for this patient population.

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“…Existing immunotherapy is based on immune checkpoint-specific treatment options for T cell antigen receptors; however, not all patients benefit from immunotherapy due to the immune heterogeneity of tumors ( 30 ). Most patients exhibit significant differences in response and resistance, suggesting that further analysis of the individual tissues and individual cells in tumor patients is necessary to develop specific immunotherapy strategies ( 31 ). Based on recent reports and sequencing data, immune cells can more systematically describe the molecular signatures of the immune system ( 32 - 34 ).…”

Section: Discussionmentioning

confidence: 99%

Immune characteristics and genetic markers of esophageal cancer by single-cell analysis: implications for immunotherapy

Xu¹,

Tseng²,

Zhang³

et al. 2023

J Thorac Dis

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Background Esophageal cancer (EC) is one of the most common cancers worldwide. The prognoses for patients with the same stage of EC can vary substantially. The progress of single-cell analysis technology has furthered the understanding of tumor heterogeneity. This paper aimed to apply single-cell analysis to explore the characteristics of the tumor environment of EC and provide a basis for personalized treatment. Methods The latest gene expression data and clinical follow-up information of single-cell sequencing results of EC samples were downloaded from The Cancer Genome Atlas (TCGA) Genomic Data Commons (GDC) Application Programming Interface (API). A differential gene function analysis of the immune infiltration signature agents in the tumor microenvironment (TME) was performed using bioinformatics analytical methods, and potential molecular targets were sought. Results We identified specific cell subsets in the EC and paracancerous samples, including panel cells, natural killer (NK) cells, exhausted cluster of differentiation (CD)8 + T cells, CD8 + memory T (Tcm) cells, and effector memory T (Tem) cells, including B cell enrichment in the cancer samples. Differences were detected between B cells and monocytes in stage II and III tumors, which may be related to RNA transcription and degradation. The CXCL8 protein was identified as a valid potential prognostic marker. Conclusions Cell groups with homogenous cell surface markers exhibit intercellular variations that exert a considerable effect on cell function. Our study will contribute to the understanding of the TME and cellular heterogeneity in EC patients and serve as a valuable resource for in-depth exploration of the pathogenesis of EC and the identification of potential therapeutic targets in the future.

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Inadequate health-related quality of life assessment and reporting in phase III clinical trials of immune checkpoint inhibitors in solid cancers: A systematic review

Cited by 14 publications

References 26 publications

Time trends in health-related quality of life assessment and reporting within publications of oncology randomised phase III trials: a meta-research study

Time trends in health-related quality of life assessment and reporting within publications of oncology randomised phase III trials: a meta-research study

Toward a more patient‐centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS)

Immune characteristics and genetic markers of esophageal cancer by single-cell analysis: implications for immunotherapy

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