The level of patient-reported outcome reporting in randomised controlled trials of brain tumour patients

A systematic review

Linda Dirven* (Corresponding Author), Martin J.B. Taphoorn, Jaap C. Reijneveld, Jane Blazeby, Marc Jacobs, Andrea Pusic, Roger Stupp, Peter Fayers, Fabio Efficace, EORTC Quality of Life Group (Patient Reported Outcome Measurements Over Time In ONcology-PROMOTION Registry)

*Corresponding author for this work

Research output: Contribution to journalReview article

28 Citations (Scopus)

Abstract

Background: To determine the net clinical benefit of a new treatment strategy, information on both survival and patient-reported outcomes (PROs) is required. However, to make an adequately informed decision, PRO evidence should be of sufficiently high quality. Objective: To investigate the methodological quality of PRO reporting in randomised controlled trials (RCTs) in patients with brain tumours, and to assess the proportion of studies that should impact clinical decision-making. 


Methods: We conducted a systematic literature search in several databases covering January 2004 to March 2012. We selected relevant RCTs and retrieved the following data: (1) basic trial demographics and PRO characteristics, (2) quality of PRO reporting and (3) risk of bias. Studies that should impact clinical decision-making based on their methodological robustness were analysed systematically.


Results: We identified 14 RCTs, representing over 3000 glioma patients. Only two RCTs (14%) satisfied sufficiently many key methodological criteria to provide high-quality PRO evidence, and should therefore impact clinical decision-making. Important methodological limitations in other studies were lack of reporting of the extent (43%) and reasons (86%) of missing data and statistical approaches to handle this (71%). PRO results were not interpreted in 79% of the studies and clinical significance was not discussed in 86%. Studies with high-quality PRO evidence generally showed lower risk of bias. 


Conclusions: Investigators involved in brain tumour research should pay special attention to methodological challenges identified in current work. The level of PRO reporting should continue to improve in order to facilitate a critical appraisal of study results.

Original languageEnglish
Pages (from-to)2432-2448
Number of pages17
JournalEuropean Journal of Cancer
Volume50
Issue number14
Early online date14 Jul 2014
DOIs
Publication statusPublished - Sep 2014

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Brain Neoplasms
Randomized Controlled Trials
Patient Reported Outcome Measures
Glioma
Research Personnel
Demography
Databases
Survival
Research
Clinical Decision-Making

Keywords

  • Brain tumour
  • Clinical decision-making
  • Clinical trial
  • Patient-reported outcome
  • Quality of life

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

Dirven, L., Taphoorn, M. J. B., Reijneveld, J. C., Blazeby, J., Jacobs, M., Pusic, A., ... EORTC Quality of Life Group (Patient Reported Outcome Measurements Over Time In ONcology-PROMOTION Registry) (2014). The level of patient-reported outcome reporting in randomised controlled trials of brain tumour patients: A systematic review. European Journal of Cancer, 50(14), 2432-2448. https://doi.org/10.1016/j.ejca.2014.06.016

The level of patient-reported outcome reporting in randomised controlled trials of brain tumour patients : A systematic review. / Dirven, Linda (Corresponding Author); Taphoorn, Martin J.B.; Reijneveld, Jaap C.; Blazeby, Jane; Jacobs, Marc; Pusic, Andrea; Stupp, Roger; Fayers, Peter; Efficace, Fabio; EORTC Quality of Life Group (Patient Reported Outcome Measurements Over Time In ONcology-PROMOTION Registry).

In: European Journal of Cancer, Vol. 50, No. 14, 09.2014, p. 2432-2448.

Research output: Contribution to journalReview article

Dirven, L, Taphoorn, MJB, Reijneveld, JC, Blazeby, J, Jacobs, M, Pusic, A, Stupp, R, Fayers, P, Efficace, F & EORTC Quality of Life Group (Patient Reported Outcome Measurements Over Time In ONcology-PROMOTION Registry) 2014, 'The level of patient-reported outcome reporting in randomised controlled trials of brain tumour patients: A systematic review', European Journal of Cancer, vol. 50, no. 14, pp. 2432-2448. https://doi.org/10.1016/j.ejca.2014.06.016
Dirven, Linda ; Taphoorn, Martin J.B. ; Reijneveld, Jaap C. ; Blazeby, Jane ; Jacobs, Marc ; Pusic, Andrea ; Stupp, Roger ; Fayers, Peter ; Efficace, Fabio ; EORTC Quality of Life Group (Patient Reported Outcome Measurements Over Time In ONcology-PROMOTION Registry). / The level of patient-reported outcome reporting in randomised controlled trials of brain tumour patients : A systematic review. In: European Journal of Cancer. 2014 ; Vol. 50, No. 14. pp. 2432-2448.
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abstract = "Background: To determine the net clinical benefit of a new treatment strategy, information on both survival and patient-reported outcomes (PROs) is required. However, to make an adequately informed decision, PRO evidence should be of sufficiently high quality. Objective: To investigate the methodological quality of PRO reporting in randomised controlled trials (RCTs) in patients with brain tumours, and to assess the proportion of studies that should impact clinical decision-making. Methods: We conducted a systematic literature search in several databases covering January 2004 to March 2012. We selected relevant RCTs and retrieved the following data: (1) basic trial demographics and PRO characteristics, (2) quality of PRO reporting and (3) risk of bias. Studies that should impact clinical decision-making based on their methodological robustness were analysed systematically.Results: We identified 14 RCTs, representing over 3000 glioma patients. Only two RCTs (14{\%}) satisfied sufficiently many key methodological criteria to provide high-quality PRO evidence, and should therefore impact clinical decision-making. Important methodological limitations in other studies were lack of reporting of the extent (43{\%}) and reasons (86{\%}) of missing data and statistical approaches to handle this (71{\%}). PRO results were not interpreted in 79{\%} of the studies and clinical significance was not discussed in 86{\%}. Studies with high-quality PRO evidence generally showed lower risk of bias. Conclusions: Investigators involved in brain tumour research should pay special attention to methodological challenges identified in current work. The level of PRO reporting should continue to improve in order to facilitate a critical appraisal of study results.",
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author = "Linda Dirven and Taphoorn, {Martin J.B.} and Reijneveld, {Jaap C.} and Jane Blazeby and Marc Jacobs and Andrea Pusic and Sala, {Edoardo La} and Roger Stupp and Peter Fayers and Fabio Efficace and {EORTC Quality of Life Group (Patient Reported Outcome Measurements Over Time In ONcology-PROMOTION Registry)}",
note = "Role of the funding source This paper stems from a larger project (i.e. Patient Reported Outcome Measurements Over Time In ONcology-PROMOTION Project) funded by a research grant from the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Group. Also, additional support for the conduct of the study was provided by the Italian Group for Adult Hematologic Diseases (GIMEMA). Acknowledgments The authors acknowledge Alessandro Perreca and Salvatore Soldati, from the GIMEMA, for their contribution to data management.",
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T1 - The level of patient-reported outcome reporting in randomised controlled trials of brain tumour patients

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AU - Dirven, Linda

AU - Taphoorn, Martin J.B.

AU - Reijneveld, Jaap C.

AU - Blazeby, Jane

AU - Jacobs, Marc

AU - Pusic, Andrea

AU - Sala, Edoardo La

AU - Stupp, Roger

AU - Fayers, Peter

AU - Efficace, Fabio

AU - EORTC Quality of Life Group (Patient Reported Outcome Measurements Over Time In ONcology-PROMOTION Registry)

N1 - Role of the funding source This paper stems from a larger project (i.e. Patient Reported Outcome Measurements Over Time In ONcology-PROMOTION Project) funded by a research grant from the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Group. Also, additional support for the conduct of the study was provided by the Italian Group for Adult Hematologic Diseases (GIMEMA). Acknowledgments The authors acknowledge Alessandro Perreca and Salvatore Soldati, from the GIMEMA, for their contribution to data management.

PY - 2014/9

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N2 - Background: To determine the net clinical benefit of a new treatment strategy, information on both survival and patient-reported outcomes (PROs) is required. However, to make an adequately informed decision, PRO evidence should be of sufficiently high quality. Objective: To investigate the methodological quality of PRO reporting in randomised controlled trials (RCTs) in patients with brain tumours, and to assess the proportion of studies that should impact clinical decision-making. Methods: We conducted a systematic literature search in several databases covering January 2004 to March 2012. We selected relevant RCTs and retrieved the following data: (1) basic trial demographics and PRO characteristics, (2) quality of PRO reporting and (3) risk of bias. Studies that should impact clinical decision-making based on their methodological robustness were analysed systematically.Results: We identified 14 RCTs, representing over 3000 glioma patients. Only two RCTs (14%) satisfied sufficiently many key methodological criteria to provide high-quality PRO evidence, and should therefore impact clinical decision-making. Important methodological limitations in other studies were lack of reporting of the extent (43%) and reasons (86%) of missing data and statistical approaches to handle this (71%). PRO results were not interpreted in 79% of the studies and clinical significance was not discussed in 86%. Studies with high-quality PRO evidence generally showed lower risk of bias. Conclusions: Investigators involved in brain tumour research should pay special attention to methodological challenges identified in current work. The level of PRO reporting should continue to improve in order to facilitate a critical appraisal of study results.

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