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Outcome selection and role of patient reported outcomes in contemporary cardiovascular trials: systematic review.

Rahimi K, Malhotra A, Banning AP, Jenkinson C - BMJ (2010)

Bottom Line: Systematic review.Medline and Embase.This was largely explained by inappropriate use of composite outcomes and inadequate measurement of harms.

View Article: PubMed Central - PubMed

Affiliation: Department of Cardiology, Oxford Heart Centre, John Radcliffe Hospital, Oxford OX3 9DU, UK. kazem.rahimi@orh.nhs.uk

ABSTRACT

Objectives: To systematically assess the type of outcomes selected and the prevalence of patient reported outcomes in contemporary cardiovascular trials and to quantify any misuse or underuse of patient reported outcomes using a specially developed tool that would allow estimation of the relevance of such outcomes to clinical decision making.

Design: Systematic review.

Data sources: Medline and Embase.

Study selection: Randomised controlled trials of the treatment for or prevention of cardiovascular disease published in 10 leading general medical and cardiology journals from January 2005 to December 2008.

Results: Primary outcomes were patient important (death, morbidity, or patient reported outcomes) in only 93 of 413 trials (23%, SE 2%), whereas another 92 (22%, SE 2%) combined these outcomes with other less important ones into a composite. Sixty five trials (16%; SE 2%) used at least one instrument to measure patient reported outcomes, mostly in trials where such information would have been important or crucial for clinical decision making (52 trials). Patient reported outcomes were judged to be of little incremental value to a large number of, mostly explanatory, cardiovascular trials (152 trials). However, many trials in which patient reported outcomes would have been important or crucial for clinical decision making did not report such outcomes (122 of 174 trials, 70%). These included several trials that primarily aimed to improve symptoms or functional status, trials that tested interventions with a considerable potential for causing harm (mainly bleeding) that were not meaningfully measured, and trials with composite outcomes that were dominated by outcomes of questionable importance to patients.

Conclusions: Despite a continued rise in the reporting of patient reported outcomes with no evidence for their misuse in more recent cardiovascular trials, they seem to be still underused once their relevance to clinical decision making has been taken into account. This was largely explained by inappropriate use of composite outcomes and inadequate measurement of harms.

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Fig 1 Search retrieval and study selection process
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fig1: Fig 1 Search retrieval and study selection process

Mentions: Figure 1 summarises the retrieval and selection process for studies. The combined searches yielded 2374 reports, of which 1376 were excluded on the basis of titles and abstracts. The remaining 998 reports were allocated a unique random number with the use of a random sequence generator.10 We assessed in full text the first 500 articles according to this random list for their eligiblity, and extracted relevant data from eligible trials. Of these, 87 did not meet the eligibility criteria. We used an electronic data extraction form to capture pivotal information from each trial. We categorised reported outcomes and individual components of any composite outcomes into patient important outcomes (death, major morbidity, patient reported outcome) or intermediate outcomes—that is, those not immediately or directly important to patients, such as biochemical, physiological, or surrogate outcomes. The categorisation of outcome was based on previously published ranking systems,11 12 with more emphasis on the methods of outcome measurement (see also web extra appendix 2). From the trials evaluated, we assessed those that were reporting patient reported outcomes to see whether a previously validated instrument was used or whether psychometric properties were reported or referenced in the article. All data were extracted in duplicate. The results and disagreements were discussed until consensus was reached.


Outcome selection and role of patient reported outcomes in contemporary cardiovascular trials: systematic review.

Rahimi K, Malhotra A, Banning AP, Jenkinson C - BMJ (2010)

Fig 1 Search retrieval and study selection process
© Copyright Policy - open-access
Related In: Results  -  Collection

License 1 - License 2
Show All Figures
getmorefigures.php?uid=PMC2967478&req=5

fig1: Fig 1 Search retrieval and study selection process
Mentions: Figure 1 summarises the retrieval and selection process for studies. The combined searches yielded 2374 reports, of which 1376 were excluded on the basis of titles and abstracts. The remaining 998 reports were allocated a unique random number with the use of a random sequence generator.10 We assessed in full text the first 500 articles according to this random list for their eligiblity, and extracted relevant data from eligible trials. Of these, 87 did not meet the eligibility criteria. We used an electronic data extraction form to capture pivotal information from each trial. We categorised reported outcomes and individual components of any composite outcomes into patient important outcomes (death, major morbidity, patient reported outcome) or intermediate outcomes—that is, those not immediately or directly important to patients, such as biochemical, physiological, or surrogate outcomes. The categorisation of outcome was based on previously published ranking systems,11 12 with more emphasis on the methods of outcome measurement (see also web extra appendix 2). From the trials evaluated, we assessed those that were reporting patient reported outcomes to see whether a previously validated instrument was used or whether psychometric properties were reported or referenced in the article. All data were extracted in duplicate. The results and disagreements were discussed until consensus was reached.

Bottom Line: Systematic review.Medline and Embase.This was largely explained by inappropriate use of composite outcomes and inadequate measurement of harms.

View Article: PubMed Central - PubMed

Affiliation: Department of Cardiology, Oxford Heart Centre, John Radcliffe Hospital, Oxford OX3 9DU, UK. kazem.rahimi@orh.nhs.uk

ABSTRACT

Objectives: To systematically assess the type of outcomes selected and the prevalence of patient reported outcomes in contemporary cardiovascular trials and to quantify any misuse or underuse of patient reported outcomes using a specially developed tool that would allow estimation of the relevance of such outcomes to clinical decision making.

Design: Systematic review.

Data sources: Medline and Embase.

Study selection: Randomised controlled trials of the treatment for or prevention of cardiovascular disease published in 10 leading general medical and cardiology journals from January 2005 to December 2008.

Results: Primary outcomes were patient important (death, morbidity, or patient reported outcomes) in only 93 of 413 trials (23%, SE 2%), whereas another 92 (22%, SE 2%) combined these outcomes with other less important ones into a composite. Sixty five trials (16%; SE 2%) used at least one instrument to measure patient reported outcomes, mostly in trials where such information would have been important or crucial for clinical decision making (52 trials). Patient reported outcomes were judged to be of little incremental value to a large number of, mostly explanatory, cardiovascular trials (152 trials). However, many trials in which patient reported outcomes would have been important or crucial for clinical decision making did not report such outcomes (122 of 174 trials, 70%). These included several trials that primarily aimed to improve symptoms or functional status, trials that tested interventions with a considerable potential for causing harm (mainly bleeding) that were not meaningfully measured, and trials with composite outcomes that were dominated by outcomes of questionable importance to patients.

Conclusions: Despite a continued rise in the reporting of patient reported outcomes with no evidence for their misuse in more recent cardiovascular trials, they seem to be still underused once their relevance to clinical decision making has been taken into account. This was largely explained by inappropriate use of composite outcomes and inadequate measurement of harms.

Show MeSH
Related in: MedlinePlus