Aims: Understanding the added value of patient-reported outcomes (PROs) to traditional clinical endpoints (e.g. survival or response to therapy) in randomized controlled trials (RCTs) is critical to better inform clinical decision-making. We assessed concordance between the results of clinical endpoints and PROs in RCTs, and evaluated how authors summarized treatment recommendations based on the combined datasets. Methods: A systematic review in PubMed/Medline and the Cochrane Library identified cancer RCTs with a PRO endpoint published from January 2004 to December 2016 in the top four cancer sites: breast, colorectal, lung and prostate cancer. Two independent reviewers recorded information on trial characteristics, PROs and clinical endpoints and level of concordance. For clinical endpoints, we defined the study outcome as “better” or “worse” if more than half of the endpoints of the RCT reported in the publication favored the experimental or the control arm, respectively. For PROs, we defined “better” or “worse” if more than half of the specific scales/domains of PRO instruments used favored the experimental or the control arm, respectively. Final treatment recommendations, reported by authors themselves, were also extracted. Results: Overall, 480 RCTs were identified and, based on predefined criteria, 417 RCTs were included in the concordance analysis. Overall survival (19.4%) and progression-free survival (14.1%) were the most frequent clinical primary endpoints. In one third of the studies, PROs supported traditional clinical endpoints favouring the experimental treatment. In 23.7% of cases both clinical and PROs yielded similar results for each treatment group. However, in the large majority of studies (76.3%) PROs provided information that contrasted with the clinical findings and thus yielded additional information on overall treatment effectiveness. Details are provided in Table 1. When there were no differences in clinical endpoints between treatment arms, and PRO favoured the experimental arm, final trial recommendations basically always included discussion of PRO findings. Conclusions: In the large majority of RCTs that we evaluated, PROs contributed information enabling a more comprehensive evaluation of overall treatment effectiveness of the treatments being tested

Concordance between patient reported and clinical outcomes in randomized controlled trials (RCTs) of cancer treatment / Efficace, Fabio; Aaronson, Neil K.; Sparano, Francesco; Sprangers, Mirjam; Fayers, Peter; Pusic, Andrea L.; Anota, Amelie; Cottone, Francesco; Rees, Jonathan; Deliu, Nina; Piciocchi, Alfonso; La Sala, Edoardo; Haas, Alissa; Kieffer, Jacobien M.; Wang, Wenna; Pezold, Mike; Fuzesi, Sarah; Isharwal, Sumit; Yeung, John; Wan, Chonghua; Blazeby, Jane. - In: QUALITY OF LIFE RESEARCH. - ISSN 1573-2649. - (2017), pp. 103-103. ((Intervento presentato al convegno 24th Annual Conference of the International Society for Quality of Life Research (ISOQOL) tenutosi a Philadelphia (PA), USA [10.1007/s11136-017-1658-6].

Concordance between patient reported and clinical outcomes in randomized controlled trials (RCTs) of cancer treatment

Fabio Efficace;Francesco Cottone;Nina Deliu;Alfonso Piciocchi;
2017

Abstract

Aims: Understanding the added value of patient-reported outcomes (PROs) to traditional clinical endpoints (e.g. survival or response to therapy) in randomized controlled trials (RCTs) is critical to better inform clinical decision-making. We assessed concordance between the results of clinical endpoints and PROs in RCTs, and evaluated how authors summarized treatment recommendations based on the combined datasets. Methods: A systematic review in PubMed/Medline and the Cochrane Library identified cancer RCTs with a PRO endpoint published from January 2004 to December 2016 in the top four cancer sites: breast, colorectal, lung and prostate cancer. Two independent reviewers recorded information on trial characteristics, PROs and clinical endpoints and level of concordance. For clinical endpoints, we defined the study outcome as “better” or “worse” if more than half of the endpoints of the RCT reported in the publication favored the experimental or the control arm, respectively. For PROs, we defined “better” or “worse” if more than half of the specific scales/domains of PRO instruments used favored the experimental or the control arm, respectively. Final treatment recommendations, reported by authors themselves, were also extracted. Results: Overall, 480 RCTs were identified and, based on predefined criteria, 417 RCTs were included in the concordance analysis. Overall survival (19.4%) and progression-free survival (14.1%) were the most frequent clinical primary endpoints. In one third of the studies, PROs supported traditional clinical endpoints favouring the experimental treatment. In 23.7% of cases both clinical and PROs yielded similar results for each treatment group. However, in the large majority of studies (76.3%) PROs provided information that contrasted with the clinical findings and thus yielded additional information on overall treatment effectiveness. Details are provided in Table 1. When there were no differences in clinical endpoints between treatment arms, and PRO favoured the experimental arm, final trial recommendations basically always included discussion of PRO findings. Conclusions: In the large majority of RCTs that we evaluated, PROs contributed information enabling a more comprehensive evaluation of overall treatment effectiveness of the treatments being tested
24th Annual Conference of the International Society for Quality of Life Research (ISOQOL)
quality of life; patient-reported outcomes
04 Pubblicazione in atti di convegno::04c Atto di convegno in rivista
Concordance between patient reported and clinical outcomes in randomized controlled trials (RCTs) of cancer treatment / Efficace, Fabio; Aaronson, Neil K.; Sparano, Francesco; Sprangers, Mirjam; Fayers, Peter; Pusic, Andrea L.; Anota, Amelie; Cottone, Francesco; Rees, Jonathan; Deliu, Nina; Piciocchi, Alfonso; La Sala, Edoardo; Haas, Alissa; Kieffer, Jacobien M.; Wang, Wenna; Pezold, Mike; Fuzesi, Sarah; Isharwal, Sumit; Yeung, John; Wan, Chonghua; Blazeby, Jane. - In: QUALITY OF LIFE RESEARCH. - ISSN 1573-2649. - (2017), pp. 103-103. ((Intervento presentato al convegno 24th Annual Conference of the International Society for Quality of Life Research (ISOQOL) tenutosi a Philadelphia (PA), USA [10.1007/s11136-017-1658-6].
File allegati a questo prodotto
File Dimensione Formato  
Efficace_concordance_2017.pdf

solo gestori archivio

Tipologia: Versione editoriale (versione pubblicata con il layout dell'editore)
Licenza: Tutti i diritti riservati (All rights reserved)
Dimensione 85.85 kB
Formato Adobe PDF
85.85 kB Adobe PDF   Visualizza/Apri   Richiedi una copia

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/1288226
Citazioni
  • ???jsp.display-item.citation.pmc??? ND
  • Scopus 0
  • ???jsp.display-item.citation.isi??? 0
social impact