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Table 1 Characteristics of the 16 articles involving patients and/or experts to justify patient relevance of outcomes

From: Patient-relevant outcomes: what are we talking about? A scoping review to improve conceptual clarity

Authors Objective/ motivation of article Type of article/ study Terms used synonymously Indicator disease/intervention Outcomes considered patient-relevant [instrument, if applicable]
Blome et al. 2009 [11] Development and validation of a specific version of the German patient benefit index (PBI-S) to be used in the treatment of pruritus (PBI-P) Mixed methods study involving patients and experts 5 terms:
- patient-relevant benefit
- patient-relevant treatment benefit
- patient preference
- patient-reported benefit
- patient-relevant outcome
Pruritus In decreasing order of importance, i.a.:
- no longer experience itching
- find a clear diagnosis and therapy
- have confidence in therapy
- be free of pain
- no longer have a burning sensation on the skin
- be able to sleep better
- be less dependent on doctor and clinic visits
Blome et al. 2014 [12] Development and validation of a specific version of the German patient benefit index (PBI-S) to be used in the treatment of lymphedema and lipedema (PBI-L) Mixed methods study involving patients and experts 3 terms:
- patient-relevant outcome
- patient-relevant benefit
- patient-relevant treatment benefit
Lymphedema, lipedema In decreasing order of importance, i.a.:
- experience less swelling and tension
- be free of pain
- avoid complications
- receive optimal hosiery (e.g., color, fit, prescription quantity)
- be less restricted in your ability to move around
- have no fear that the disease will become worse
- find a clear diagnosis and therapy
Cho et al. 2019 [13] Identification of patients‘and caregivers‘priorities for outcomes in trials on autosomal dominant polycystic kidney disease Mixed methods study involving patients only 4 terms:
- patient-important outcome
- patient-prioritised outcome
- patient-centered outcome
- patient-reported outcome
Autosomal dominant polycystic kidney disease In decreasing order of importance, i.a.:
- kidney function
- end-stage kidney disease
- survival
- cyst size/growth
- cyst pain/bleeding
- blood pressure
- ability to work
- cerebral aneurysm/stroke
- mobility/physical function
- fatigue
Daeter et al. 2018 [14] 1. Share the standard set of outcomes for coronary artery disease developed by Meetbaar Beter
2. Illustrate how the standard set is presented and published
Note: Meetbaar Beter aims to improve the quality of cardiovascular care in hospitals in the Netherlands by creating transparency on patient-relevant outcomes (Benchmarking)
Delphi approach among experts only 1 term:
- patient-relevant outcome
Coronary artery disease (overall) - long-term survival (≤5 years)
- 1-year mortality
- quality of life [SF-36]
- myocardial infarction (≤30 days)
Coronary artery bypass grafting - 120-day mortality
- surgical reexploration (≤30 days)
- cerebrovascular accident (≤72 h)
- Deep sternal wound infection (≤30 days)
- free of myocardial infarction
- free of coronary artery reintervention
Percutaneous coronary intervention - 30-day mortality
- urgent coronary artery bypass grafting (≤24 h)
- occurrence of target vessel revascularization (≤1 year)
Conservative treatment - chest pain (≤1 year)
- free of major adverse cardiac event
Dinglas et al. 2018 [15] Synthesis of the literature with regard to patient-important outcome for Intensive Care Unit survivors focusing on a research program on acute respiratory failure Synthesis, Delphi approach among patients only 1 term:
- patient-important outcome
Acute respiratory failure - survival
- physical function
- muscle and/or nerve function
- pulmonary function
- cognition
- mental health [HADS, IES-R]
- pain [EQ-5D pain question]
- health-related quality of life [EQ-5D, SF-36]
Eiring et al. 2016 [16] 1. Investigate the relative importance of patient-important outcomes in bipolar disorder
2. Construct a holistic taxonomy of patient-important outcomes
Mixed methods study involving patients only 2 terms:
- patient-important outcome
- patient-relevant outcome
Bipolar disorder In decreasing order of importance, i.a.:
- avoid serve depression
- avoid serve mania
- increase quality of life
- better functioning (school/work)
- better social functioning
Kinter et al. 2009 [17] 1. Identification of endpoints directly from patients with schizophrenia
2. Evaluation whether patients can express which endpoints matter to them
3. Ranking of the relevant endpoints
Mixed methods study involving patients only 5 terms:
- patient-relevant endpoint
- patient-relevant benefit
- patient endpoint
- patient preference
- patient-centered endpoint
Schizophrenia In decreasing order of importance, i.a.:
- clear thinking
- minimization of symptoms
- social activities
- daily activities
- supportive physician
Konkle et al. 2019 [18] Review of strengths and limitations of outcome measures used in hemophilia trials from a provider and patient perspective Review involving patients and experts 3 terms:
- patient-important outcome
- patient-relevant outcome
- patient-important clinical outcome
Hemophilia - frequency of bleeds
- factor activity level
- duration of expression
- chronic pain
- healthcare resource use
- mental health
Lindsley et al. 2018 [19] Identification and prioritization of clinical questions and patient-relevant outcomes for research associated with the treatment of age-related macular degeneration Delphi approach among patients and experts 1 term:
- patient-important outcome
Age-related macular degeneration Highly important to patients:
- choroidal neovascularization
- development of advanced age-related macular degeneration
- retinal hemorrhage
- gain of vision
- slowing vision loss
- serious ocular events
Murad et al. 2011 [20] Assessment of preferences of patients with diabetes on how clinical trials should be conducted with regard to study design (pragmatic versus explanatory) and endpoints (surrogate versus patient-relevant) Cross-sectional study involving patients only 2 terms:
- patient-important outcome
- patient preference
Diabetes mellitus In decreasing order of importance, i.a.:
- end-stage renal disease
- stroke
- myocardial infarction
- blindness
- HbA1c control
- death
Nabbout et al. 2018 [21] Identification of a core set of patient- and caregiver-relevant concepts to be included in future clinical trials on dravet syndrome Qualitative study, Delphi approach among caregivers and experts 2 terms:
- patient- and caregiver-relevant outcome
- patient- and caregiver-relevant endpoint
Dravet syndrome - seizures
- expressive communication of the child
- receptive communication of the child
- daily activities of the caregiver
- social functioning of the caregiver
Sanderson et al. 2010 [22] Identification of treatment outcomes important to patients with rheumatoid arthritis receiving anti-tumor necrosis factor therapy Qualitative study involving patients only 6 terms:
- patient outcome
- patient priority treatment outcome
- patient priority outcome
- patients’ important treatment outcome
- patients’ important outcome
- patient-important outcome
Rheumatoid arthritis - rheumatoid arthritis under control (symptoms less, rheumatoid arthritis stable, medication effects)
- doing things (doing things, able to plan)
- emotional health (positive feelings, holistic identity, positive mental changes, better life)
- coping with illness (coping with rheumatoid arthritis, coping with health system)
- global outcomes (feeling well, return to a normal life, feeling (more) normal)
Sung et al. 2014 [23] Development of a comprehensive conceptual framework representing the relevant dimensions and outcomes important to women with pelvic organ prolapse Mixed methods study involving patients only 1 term:
- patient-important outcome
Pelvic organ prolapse In decreasing order of importance, i.a.:
- alleviation of physical bulge symptoms and associated discomfort
- improvement in physical function
- improvement in sexual function
- improvement in body image perception
- improvement in social function
Van der Elst et al. 2016 [24] Better comprehension of the perspective of patients with early rheumatoid arthritis on preferred health and treatment outcomes Qualitative study involving patients only 2 terms:
- patient-preferred outcome
- patient-preferred health and treatment outcome
Early rheumatoid arthritis - aspects of disease control, e.g., prevention or control of joint damage, less medication
- physical aspects, e.g., relief of pain and other physical symptoms, improved joint function and mobility
- aspects of participation, e.g., performing activities of daily living, engaging in work and/or leisure
- mental aspects, e.g., emotional well-being, life enjoyment
Wilson et al. 2019 [25] Synthesis of evidence in all outcome domains identified as important by patients undergoing unicompartmental or total knee replacement and outcome domains commonly used in other studies Systematic review & meta-analysis involving patients and referring to other studies 1 term:
- patient relevant outcome
Unicompartmental and total knee replacement in osteoarthritis - hospital admission impact: length of operation, length of hospital stay
- risk of early complications (myocardial infarction, stroke, venous thromboembolism, deep infection) or early mortality
- success of operation: range of movement achieved or kneeling ability, reduction in pain, improvement in function
- reoperation or revision rate
- rate of recovery: rate of return to work, rate of return to sporting activities
Van Veghel et al. 2016 [26] Presentation and discussion of the patient-relevant outcomes of Meetbaar Beter for coronary artery disease and aortic valve disease, focusing on the surgical procedures coronary artery bypass grafting, percutaneous coronary intervention, aortic valve replacement and transcatheter aortic valve implantation Database analysis involving experts only 3 terms:
- patient-relevant outcome
- patient-relevant health outcome
- patient-oriented outcome
Coronary artery disease (overall) - readmission due to myocardial infarction (≤30 days)
Coronary artery bypass grafting - 120-day mortality
- quality of life [SF-36]
Percutaneous coronary intervention - 1-year mortality
- Occurrence of target vessel revascularization (≤1 year)
Aortic valve replacement - 120-day mortality
- long-term survival
Transcatheter aortic valve implantation - 120-day mortality
- implantation of a new permanent pacemaker (≤30 days)