The importance of patient-reported outcomes: a call for their comprehensive integration in cardiovascular clinical trials, 2014, Anker et al

Anker, S D, Agewall, S, Borggrefe M et al, 2014, The importance of patient-reported outcomes: a call for their comprehensive integration in cardiovascular clinical trials, European Heart Journal, Volume 35, Issue 30, 7 August 2014, Pages 2001–2009, https://doi.org/10.1093/eurheartj/ehu205

https://pubmed.ncbi.nlm.nih.gov/24904027/

Abstract

Patient-reported outcomes (PROs), such as symptoms, health-related quality of life (HRQOL), or patient perceived health status, are reported directly by the patient and are powerful tools to inform patients, clinicians, and policy-makers about morbidity and 'patient suffering', especially in chronic diseases.

Patient-reported outcomes provide information on the patient experience and can be the target of therapeutic intervention. Patient-reported outcomes can improve the quality of patient care by creating a holistic approach to clinical decision-making; however, PROs are not routinely used as key outcome measures in major cardiovascular clinical trials. Thus, limited information is available on the impact of cardiovascular therapeutics on PROs to guide patient-level clinical decision-making or policy-level decision-making.

Cardiovascular clinical research should shift its focus to include PROs when evaluating the efficacy of therapeutic interventions, and PRO assessments should be scientifically rigorous.

The European Society of Cardiology and other professional societies can take action to influence the uptake of PRO data in the research and clinical communities. This process of integrating PRO data into comprehensive efficacy evaluations will ultimately improve the quality of care for patients across the spectrum of cardiovascular disease.

 

I thought it could be worthwhile to have a closer look at a particular claim of some defenders of research on the Lightning Process and similarly badly designed intervention studies -- the claim that there is a general shift to use patient reported outcomes (PROs) in "areas with a tradition of using more objective outcome".

The paper by Anker et al is referenced by Kjetil Gundro Brurberg on the Mental Elf Blog in his defense of the PACE trial [1] against the criticism published in the Journal of Health Psychology's special edition. (See @Kalliope 's post here.)

Brurberg writes:

"PROMs are often more important to patients than objectively assessed outcomes, and researchers should therefore avoid devaluing PROMs on a general basis.

"PROMs are increasingly used in areas with a tradition of using more objective outcome (Anker et al, 2014), and they may even have long-term prognostic importance (Norekvål et al, 2010).

"One would therefore expect some degree of correlation between reliable and valid patient reported outcomes and objectively assessed activity levels, as substantiated by recent validation studies (Mahieu et al, 2016).

I only had a quick look at Anker et al, but to me it doesn't seem they state that it's fine to only use subjective outcomes in open label trials?

[1] Brurberg, KG, 2017, A PACE-gate or an editorial without perspectives?, https://www.nationalelfservice.net/...ce-gate-or-an-editorial-without-perspectives/

 

I think Brurberg's other references don't support the idea that subjective outcomes are sufficient in open label trials either?

Mahieu MA, Ahn GE, Chmiel JS, Dunlop DD, Helenowski IB, Semanik P, Song J, Yount S, Chang RW, Ramsey-Goldman R. Fatigue, patient reported outcomes, and objective measurement of physical activity in systemic lupus erythematosus. Lupus. 2016 Oct;25(11):1190-9. doi: 10.1177/0961203316631632. Epub 2016 Feb 10. PMID: 26869353; PMCID: PMC4980272.

Forum thread here.

Norekvål TM, Fridlund B, Rokne B, Segadal L et al. (2010) Patient reported outcomes as predictors of 10-year survival in women after acute myocardial infarction. Health Quality of Life Outcomes. 8: DOI: 10.1186/1477-7525-8-140.