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Seo C, Hu S, King-Kallimanis BL, Chang AE, Regnault A, Miller AS, Kaur MN. Understanding data visualization techniques in qualitative studies used to develop and validate patient-reported outcome measures: a targeted literature review. Qual Life Res 2025:10.1007/s11136-025-03964-5. [PMID: 40279025 DOI: 10.1007/s11136-025-03964-5] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Accepted: 03/24/2025] [Indexed: 04/26/2025]
Abstract
PURPOSE Qualitative data that reflects patients' experiences are the foundation of any patient-reported outcome measure (PROM) development and validation study; however, there is limited understanding of the type of data visualization techniques that facilitate communication of this data. The goal of this targeted literature review was to investigate data visualization methods that have been used in published PROM development and validation literature to report qualitative results. METHODS A literature search in OVID via MEDLINE was conducted among the top 10 non-disease-specific journals publishing PROM qualitative development and validation studies. Studies that reported qualitative methods to develop/validate a PROM and included data visualization in the form of tables or figures were included. Article characteristics and data visualization types were extracted. RESULTS A total of 185 articles were included in data extraction. Most articles (n = 109, 59.1%) included figures (n = 172, average 2 relevant figures per article) in the form of hierarchy/flowcharts (n = 124, 72.1%) and bar charts (n = 29, 16.9%). Information reported in figures included depiction of conceptual frameworks (n = 112, 65.1%) and concept frequency (n = 40, 24.4%). Most articles (n = 152, 81.7%) included tables (n = 307, average 2 relevant tables per article). Information reported in tables included concept frequency (n = 133, 43.3%) and cognitive debriefing and revisions (n = 91, 29.6%). CONCLUSION Data visualization techniques used to report qualitative results in the identified PROM qualitative development and validation studies were heterogeneous, and many studies did not utilize any data visualization techniques. This study will inform the development of guidance for using data visualizations to report qualitative PROM research.
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Affiliation(s)
- Caroline Seo
- BioPharmaceuticals Medical Evidence, AstraZeneca, Gaithersburg, MD, USA.
| | - Sophia Hu
- Patient-Reported Outcomes, Value, and Experience (PROVE) Center, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA
| | | | - Ashley E Chang
- Division of Plastic Surgery, Department of Surgery, University of Pennsylvania, Philadelphia, PA, USA
| | | | - Amitai S Miller
- Patient-Reported Outcomes, Value, and Experience (PROVE) Center, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA
| | - Manraj N Kaur
- Patient-Reported Outcomes, Value, and Experience (PROVE) Center, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA
- Harvard Medical School, Boston, MA, USA
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Regueiro M, Fischer M, Bossuyt P, McGinnis K, Protic M, Hunter Gibble T, Panni T, Chan LS, Hibi T, Rubin DT. Mirikizumab Sustained Impact on Fatigue in Patients with Moderately to Severely Active Crohn's Disease in the Phase 2 AMAG Study. Inflamm Bowel Dis 2025; 31:432-441. [PMID: 39093640 PMCID: PMC11808575 DOI: 10.1093/ibd/izae166] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 01/02/2024] [Indexed: 08/04/2024]
Abstract
BACKGROUND Fatigue is a burdensome, under-recognized, multidimensional symptom experienced by patients with Crohn's disease (CD). We evaluated the impact of mirikizumab on fatigue and the association between changes in select patient-reported outcomes and clinical measures with changes in fatigue from baseline to week 104 (W104). METHODS Patients (N = 191) were randomized (2:1:1:2) to receive placebo (PBO), 200 mg, 600 mg, or 1000 mg of mirikizumab, administered intravenously (IV) every 4 weeks at W0, W4, and W8. Patients who achieved ≥1 point improvement in Simple Endoscopic Score for Crohn's Disease (SES-CD) and received mirikizumab at W12 (rerandomized maintenance cohort) were rerandomized to continue induction IV treatment assignment (IV-C) or received 300 mg of mirikizumab subcutaneously (SC) until W52. Nonrandomized maintenance cohort had endoscopic nonimprovers (1000 mg) and PBO patients (PBO/1000 mg) who received 1000 mg of mirikizumab until W52. Subjects from the maintenance period with clinical benefit received 300 mg SC Q4W from W52 to W104. The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) questionnaire was used to assess fatigue, and the FACIT-F associations were assessed using Pearson correlation coefficient. RESULTS At W12, mirikizumab groups reported improved FACIT-F scores compared with PBO, and improvement was maintained through W52 and W104. Changes in FACIT-F at W52 and W104 had strong correlations with changes at the same time point in quality of life (QoL) scores but lacked correlations with changes in inflammatory biomarkers. CONCLUSIONS Mirikizumab treatment significantly improved fatigue in patients with moderately to severely active CD, which was sustained to W104. The improvement in fatigue was correlated with improvement in clinical measures and was strongly correlated with improvement in QoL.
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Affiliation(s)
- Miguel Regueiro
- Department of Gastroenterology, Hepatology and Nutrition, Cleveland Clinic, Cleveland, OH, USA
| | - Monika Fischer
- Division of Gastroenterology and Hepatology, Indiana University, Indianapolis, IN, USA
| | - Peter Bossuyt
- Imelda GI Clinical Research Centre, Imelda General Hospital, Bonheiden, Belgium
| | | | | | | | | | | | - Toshifumi Hibi
- Kitasato Institute Hospital Center for Advanced Inflammatory Bowel Disease Research and Treatment, Minato-ku, Tokyo, Japan
| | - David T Rubin
- Inflammatory Bowel Disease Center, University of Chicago Medicine, Chicago, IL, USA
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Bootsma L, Vollebregt PF, van Bodegraven AA, van der Horst D, Han-Geurts IJM, Felt-Bersma RJF. Identification of spontaneous patient-reported complaints related to perianal fistula in patients with Crohn's disease. Colorectal Dis 2024; 26:2057-2068. [PMID: 39448386 DOI: 10.1111/codi.17207] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 01/17/2024] [Revised: 08/16/2024] [Accepted: 09/09/2024] [Indexed: 10/26/2024]
Abstract
AIM To identify patient-reported complaints affecting quality of life in Crohn's disease patients with a perianal fistula, and to compare differences between subgroups. METHOD A questionnaire was distributed to 1667 patients from the Dutch Crohn's and Colitis Patients' organization, those patients with Crohn's disease and perianal fistula were included. Patients were asked to report (using free text) their most important fistula-related complaints affecting their quality of life. All responses were structurally analyzed and categorized. Data comparisons were made between subgroups: women versus men, patients with versus without current presence of a seton, and patients aged ≤40 versus >40 years. RESULTS Of 743 respondents (44.6%), 123 patients with Crohn's disease and perianal fistula were included (92 women, median age 41 years [IQR 34-56] and 36 with seton). A total of 776 complaints were allocated to 36 categories, with 19 reported in >10% of patients. Perianal fistula-related complaints affected nearly all patients (95.9%). Impact on psychological status (71.7% vs. 29.0%; p < 0.0001) and on sexual activities (37.0% vs. 16.1%; p = 0.003) were more common in women than men. Younger patients more often reported insecurity (38.7% vs. 18.0%; p = 0.026), shame (29.0% vs. 11.5%; p = 0.024), and impact on sexual activities (40.3% vs. 23.0%; p = 0.048) than older patients. Patients with a seton more frequently reported self-experienced malodour (50.0% vs. 23.0%; p = 0.005), physical activity limitations (41.7% vs. 19.5%; p = 0.014), and work/study impact (22.2% vs. 5.7%; p = 0.019). CONCLUSION We identified 19 perianal fistula-related complaints reported by >10% of patients. These complaints may guide improvement of current outcome measures.
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Affiliation(s)
- Lars Bootsma
- Proctos Kliniek, Bilthoven, The Netherlands
- Utrecht University, Utrecht, The Netherlands
| | - Paul F Vollebregt
- Department of Gastroenterology and Hepatology, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands
| | - Adriaan A van Bodegraven
- Department of Gastroenterology, Geriatrics, Internal and Intensive Care Medicine (COMIK), Zuyderland Medical Center, Sittard, the Netherlands
| | | | | | - Richelle J F Felt-Bersma
- Proctos Kliniek, Bilthoven, The Netherlands
- Department of Gastroenterology and Hepatology, Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands
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Obekpa EO, McCurdy SA, Gallardo KR, Rodriguez SA, Cazaban CG, Brown HS, Yang JJ, Wilkerson JM. Characteristics and quality of life of people living with comorbid disorders in substance use recovery residences. Front Public Health 2024; 12:1412934. [PMID: 39628806 PMCID: PMC11611819 DOI: 10.3389/fpubh.2024.1412934] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/06/2024] [Accepted: 10/30/2024] [Indexed: 12/06/2024] Open
Abstract
Background Opioid use disorder (OUD) is associated with significant morbidity and mortality; however, research on physical and mental health comorbidities and health-related quality of life (HRQoL) among people taking medication for OUD (MOUD) and living in recovery residences is sparse. We investigated the prevalence of comorbidities and examined which EQ-5D-5L HRQoL dimensions are most affected by these comorbidities. Methods Data were collected from 358 residents living in 14 Texas-based recovery residences from April 2021 to June 2023. The EQ-5D-5L descriptive system comprises five dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression). Each dimension has five levels of perceived problems, dichotomized into "No problems" (level 1) and "Any problems" (levels 2-5) for analyses. Cross-sectional analyses of residents' characteristics, comorbidities (categorized as mental health disorders or association with major body systems), and EQ-5D-5L dimensions were conducted using Chi-squared or Student t-tests. Multivariable logistic regression models were used to estimate the odds ratios (ORs) and 95% confidence intervals (CIs). Results The mean [SD] age of residents was 36.0 [8.9]. Most residents were non-Hispanic White (68.7%), male (59.7%), unemployed (66.3%), and engaged in polysubstance use (75.4%). The most frequently reported comorbidities were mental health (26.5%), respiratory (26.3%), neurological (19.3%), cardiovascular (18.2%), and musculoskeletal (17.0%) disorders. The most reported HRQoL problems were anxiety/depression (75.8%) and pain/discomfort (53.2%). In the unadjusted regression models, all comorbidities, except mental health (negative association) and digestive (no association) disorders, were positively associated with HRQoL problems. The usual activities dimension was the most affected by comorbidities, followed by mobility and pain/discomfort. Increasing age was positively associated with cardiovascular disorders (aOR = 1.06; 95% CI = 1.03-1.10), musculoskeletal disorders (aOR = 1.03; 95% CI = 1.00-1.06), mobility problems (aOR = 1.05; 95% CI = 1.01-1.09), and pain/discomfort problems (aOR = 1.02; 95% CI = 1.00-1.05). Illicit drug use was positively associated with mobility problems (aOR = 3.36; 95% CI = 1.20-9.45). Neurological (aOR = 2.71; 95% CI = 1.38-5.33) and musculoskeletal (aOR = 2.57; 95% CI = 1.25-5.29) disorders were positively associated with pain/discomfort problems. MOUD duration was negatively associated with mental health disorders (aOR = 0.14; 95% CI = 0.08-0.22) but not HRQoL. Conclusions Comorbidities significantly predict HRQoL among individuals with OUD. Our findings highlight the need for an integrated care model to treat OUD and comorbidities to sustain recovery and improve health and HRQoL.
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Affiliation(s)
- Elizabeth O. Obekpa
- UTHealth Houston School of Public Health, Institute of Implementation Science, Houston, TX, United States
| | - Sheryl A. McCurdy
- Department of Health Promotion and Behavioral Sciences, UTHealth Houston School of Public Health, Houston, TX, United States
| | - Kathryn R. Gallardo
- Department of Health Promotion and Behavioral Sciences, UTHealth Houston School of Public Health, Houston, TX, United States
| | - Serena A. Rodriguez
- Department of Health Promotion and Behavioral Sciences, UTHealth Houston School of Public Health, Houston, TX, United States
| | - Cecilia Ganduglia Cazaban
- Department of Management, Policy, and Community Health, UTHealth Houston School of Public Health, Houston, TX, United States
| | - H. Shelton Brown
- Department of Management, Policy, and Community Health, The University of Texas School of Public Health, Austin, TX, United States
| | - James J. Yang
- Department of Biostatistics and Data Science, UTHealth Houston School of Public Health, Houston, TX, United States
| | - J. Michael Wilkerson
- Department of Health Promotion and Behavioral Sciences, UTHealth Houston School of Public Health, Houston, TX, United States
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Khan S, Sebastian SA, Parmar MP, Ghadge N, Padda I, Keshta AS, Minhaz N, Patel A. Factors influencing the quality of life in inflammatory bowel disease: A comprehensive review. Dis Mon 2024; 70:101672. [PMID: 38143196 DOI: 10.1016/j.disamonth.2023.101672] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/26/2023]
Abstract
Inflammatory bowel disease (IBD) is a group of chronic relapsing disorders, including Crohn's disease (CD) and ulcerative colitis (UC), which affects an increasing number of people worldwide. In the last few decades, the scientific world has witnessed many developments in IBD management by controlling debilitating symptoms and remaining in remission for more protracted periods. Even so, we still have a large population suffering from active IBD. An individual's quality of life (QoL) can be severely affected by IBD, like any other chronic illness. In this article, we have reviewed factors influencing the QoL in IBD patients, including chronic pain, diet, physical activity, and psychological factors like depression, anxiety, and stress symptoms. We also discussed the mechanisms of diet-microbial-immune system interaction, currently available dietary therapies for active CD and UC, and early psycho-social interventions that can reduce the disease burden and improve QoL in IBD patients.
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Affiliation(s)
- Samina Khan
- University of Alberta Hospital, Edmonton, Alberta, Canada
| | | | | | - Nitin Ghadge
- Independent Researcher, Albany, NY, United States of America
| | - Inderbir Padda
- Richmond University Medical Center/Mount Sinai, Staten Island, NY, United States of America
| | | | - Naofel Minhaz
- Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh
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King K, McGuinness S, Watson N, Norton C, Chalder T, Czuber-Dochan W. What Do We Know about Medication Adherence Interventions in Inflammatory Bowel Disease, Multiple Sclerosis and Rheumatoid Arthritis? A Scoping Review of Randomised Controlled Trials. Patient Prefer Adherence 2023; 17:3265-3303. [PMID: 38111690 PMCID: PMC10725835 DOI: 10.2147/ppa.s424024] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Figures] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 06/02/2023] [Accepted: 09/13/2023] [Indexed: 12/20/2023] Open
Abstract
Purpose Between 53% and 75% of people with inflammatory bowel disease, 30%-80% with rheumatoid arthritis, and up to 50% with multiple sclerosis do not take medications as prescribed to maintain remission. This scoping review aimed to identify effective adherence interventions for inflammatory bowel disease, but with few studies found, multiple sclerosis and rheumatoid arthritis were included to learn lessons from other conditions. Methods Full and pilot randomised controlled trials testing medication adherence interventions for inflammatory bowel disease, multiple sclerosis, and rheumatoid arthritis conducted between 2012 and 2021 were identified in six electronic databases. Results A total of 3024 participants were included from 24 randomised controlled trials: 10 pilot and 14 full studies. Eight investigated inflammatory bowel disease, 12 rheumatoid arthritis, and four multiple sclerosis. Nine studies (37.5%) reported significantly improved medication adherence, all involving tailored, personalised education, advice or counselling by trained health professionals, with five delivered face-to-face and 1:1. Quality of effective interventions was mixed: five rated high quality, two medium and two low quality. Interventions predominantly using technology were likely to be most effective. Secondary tools, such as diaries, calendars and advice sheets, were also efficient in increasing adherence. Only 10 interventions were based on an adherence theory, of which four significantly improved adherence. Conclusion Tailored, face-to-face, 1:1 interactions with healthcare professionals were successful at providing personalised adherence support. Accessible, user-friendly technology-based tools supported by calendars and reminders effectively enhanced adherence. Key components of effective interventions should be evaluated and integrated further into clinical practice if viable, whilst being tailored to inflammatory conditions.
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Affiliation(s)
- Kathryn King
- Florence Nightingale Faculty Nursing, Midwifery and Palliative Care, King’s College London, London, UK
| | - Serena McGuinness
- Florence Nightingale Faculty Nursing, Midwifery and Palliative Care, King’s College London, London, UK
| | - Natalie Watson
- Florence Nightingale Faculty Nursing, Midwifery and Palliative Care, King’s College London, London, UK
| | - Christine Norton
- Florence Nightingale Faculty Nursing, Midwifery and Palliative Care, King’s College London, London, UK
| | - Trudie Chalder
- Department of Psychological Medicine, Institute of Psychiatry, Psychology and Neuroscience, King’s College London, London, UK
| | - Wladyslawa Czuber-Dochan
- Florence Nightingale Faculty Nursing, Midwifery and Palliative Care, King’s College London, London, UK
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Maspero M, Hull T. Patient-Reported Outcomes in Colorectal Surgery. Clin Colon Rectal Surg 2023; 36:240-251. [PMID: 37223227 PMCID: PMC10202545 DOI: 10.1055/s-0043-1761607] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 05/25/2023]
Abstract
Given the increased life expectancy and improvements in the treatment of colorectal patients, the success of a treatment course can no longer be determined only by objective outcomes. Health care providers ought to take into consideration the impact an intervention will have on the quality of life of patients. Endpoints that take into account the patient's perspective are defined as patient-reported outcomes (PROs). PROs are assessed through patient-reported outcome measures (PROMs), usually in the form of questionnaires. PROs are especially important in colorectal surgery, whose procedures can often be associated with some degree of postoperative functional impairment. Several PROMs are available for colorectal surgery patients. However, while some scientific societies have offered recommendations, there is no standardization in the field and PROMs are seldom implemented in clinical practice. The routine use of validated PROMs can guarantee that functional outcomes are followed over time; this way, they can be addressed in case of worsening. This review will provide an overview of the most commonly used PROMs in colorectal surgery, both generic and disease specific, as well as a summary of the available evidence in support of their routine utilization.
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Affiliation(s)
- Marianna Maspero
- Department of Colon and Rectal Surgery, Cleveland Clinic Foundation, Cleveland, Ohio
| | - Tracy Hull
- Department of Colon and Rectal Surgery, Cleveland Clinic Foundation, Cleveland, Ohio
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Systemic Diseases with Oral Manifestations and Their Impact on Health-Related Quality of Life. SERBIAN JOURNAL OF EXPERIMENTAL AND CLINICAL RESEARCH 2022. [DOI: 10.2478/sjecr-2021-0074] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 01/02/2023] Open
Abstract
Abstract
Health-related quality of life is a multidimensional concept established to evaluate the physical, psychological, and social impacts of health conditions on individuals’ well-being. Various tools for measuring health-related quality of life can be categorized into two subsets: generic and disease–specific instruments. The oral cavity can be stricken by a broad range of local and systemic diseases and their systemic treatment modalities. The most common systemic illnesses associated with oral lesions are hematologic disorders, endocrinopathies, neurological disorders, gastrointestinal conditions, mucocutaneous and rheumatic diseases, and neoplastic processes. Their manifestations in the oral cavity are, in most cases, rather nonspecific but should not be overlooked. Oral health is one of the most important parts of overall health, thus it has been proposed that poor oral health may affect health–related quality of life. The presence of oral manifestations of systemic diseases has a negative impact on the daily functioning of patients, decreasing their overall well-being. This article will review the most common systemic diseases with oral manifestations and their impact on the health–related quality of life. Oral health researchers should put a stronger emphasis on the patient-reported quality of life as a primary outcome in future clinical trials. The significance of this area has still not been widely understood in the current dental literature even though it could help improve patients’ health-related quality of life.
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Armoon B, Fleury MJ, Bayat AH, Bayani A, Mohammadi R, Griffiths MD. Quality of life and its correlated factors among patients with substance use disorders: a systematic review and meta-analysis. Arch Public Health 2022; 80:179. [PMID: 35927697 PMCID: PMC9351239 DOI: 10.1186/s13690-022-00940-0] [Citation(s) in RCA: 12] [Impact Index Per Article: 4.0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/23/2022] [Accepted: 07/25/2022] [Indexed: 11/23/2022] Open
Abstract
Background Patients with substance use disorder (SUD) usually report lower quality of life (QoL) than other patients and as much as patients with other mental disorders. The present study investigated variables associated with QoL domains among patients with SUD. Methods Studies in English published before December 1st 2021, were searched for on PubMed, Scopus, Cochrane, and Web of Science to identify primary studies on factors associated with QoL domains among patients with SUD. After reviewing for study duplicates, the full-texts of selected papers were assessed for eligibility using PECO (Participants, Exposures, Comparison and Outcome) criteria: (a) participants: patients with SUD; (b) exposures: sociodemographic factors, clinical, and service use variables; (c) comparison: patient groups without SUD; and (d) outcomes: four domains of QoL (physical, mental, social, and environmental domains). Three researchers recorded the data independently using predefined Excel spreadsheets. The Newcastle–Ottawa Scale (NOS) was used for assessing risk of bias and rated each study in terms of exposure, outcome, and comparability. Pooled odds ratios (ORs) and β coefficient were utilized at a 95% confidence level, and because sampling methods differed between studies’ pooled estimates, a random effects model was utilized. Results After the assessment of over 10,230 papers, a total of 17 studies met the eligibility criteria. Five studies (1260 participants) found that patients with SUD who were older were less likely to have a good physical Qol (OR = 0.86, 95% CI = 0.78, 0.95). Two studies (1171 participants) indicated that patients with SUD who were homeless were less likely to have a good environmental Qol (β = -0.47, p = 0.003). However, a better mental QoL was observed in four studies (1126 participants) among those receiving support from their family or friends (social networks) (OR = 1.05, 95% CI = 1.04, 1.07). Two studies (588 participants) showed that those using cocaine were less likely to have a good mental QoL (OR = 0.83, 95% CI = 0.75, 0.93). Two studies (22,534 participants) showed that those using alcohol were less likely to have a good physical QoL (β = -2.21, p = 0.001). Two studies (956 participants) showed that those having severe substance use disorders were less likely to have a good mental (β = -5.44, p = 0.002) and environmental (β = -0.59, p = 0.006) QoL respectively. Four studies (3515 participants) showed that those having mental disorders were less likely to have a good physical QoL (β = -1.05, p = 0.001), and another three studies (1211 participants) that those having mental disorders were less likely to have a good mental QoL (β = -0.33, p = 0.001). Finally, two studies (609 and 682 participants) showed that individuals who experienced trauma symptoms or mental disorders were less likely to have good social and environmental QoL, respectively (OR = 0.78, 95% CI = 0.61, 1.00) and (OR = 0.92, 95% CI = 0.9, 0.94). Conclusions The findings suggest the need for mental health services to improve the QOL among patients with SUD but further study is needed. Cocaine may cause behavioral changes which can increase the possibility of reckless and suicidal behaviors. Therefore, identifying cocaine user access, adherence, and satisfaction with treatment is recommended as an important component of adaptive functioning. Interventions that help patients with SUD get support from people within their social networks who support their recovery are also essential to their QoL. Supplementary Information The online version contains supplementary material available at 10.1186/s13690-022-00940-0.
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Bissenbakker K, Møller A, Brodersen JB, Jønsson ABR. Conceptualisation of a measurement framework for Needs-based Quality of Life among patients with multimorbidity. J Patient Rep Outcomes 2022; 6:83. [PMID: 35895139 PMCID: PMC9329502 DOI: 10.1186/s41687-022-00489-0] [Citation(s) in RCA: 4] [Impact Index Per Article: 1.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/21/2021] [Accepted: 07/13/2022] [Indexed: 11/13/2022] Open
Abstract
Background Multimorbidity is both an individual and societal problem. For society, patients with multimorbidity increase healthcare costs. For the individual, living with multimorbidity is complex, and there is an inverse relationship between a patient's Quality of Life (QoL) and their number of chronic conditions. Numerous intervention studies target these problems, yet there is no multimorbidity-specific patient-reported outcome measure (PROM) developed specifically for this group with adequate measurement properties to assess QoL. This study explores what overall needs regarding QoL are affected by living with multimorbidity through qualitative interviews. With this, we conceptualise Needs-based QoL specifically for this group, ensuring high content validity (regarding relevance and comprehensiveness) of using the Needs-based approach to measure their QoL. This is essential as this preliminary study leads to the development of the MultiMorbidity Questionnaire (MMQ), a PROM measuring QoL among patients with multimorbidity. Methods This study draws upon qualitative interviews with fifteen patients with multimorbidity based on a semi-structured interview guide following the Needs-based approach. This approach allowed the patients to cover needs relevant for their QoL in relation to the complexities of living with multimorbidity. The transcribed interviews were thematically analysed, inspired by Braun and Clarke’s reflexive approach.
Results Analysis of the interviews resulted in the construction of six intertwined domains relevant to patients with multimorbidity, covering their Needs-based QoL; “Physical ability”, “Self-determination”, “Security”, “Partner and social life”, “Self-image”, and “Personal finances”. “Physical limitations” and “Personal finances” were stated as core needs implicating the other domains. Conclusion This study shows six intertwined overall domains relevant for patients with multimorbidity regarding their Needs-based QoL; “Physical ability”, “Self-determination”, “Security”, “Partner and social life”, “Self-image”, and “Personal finances”. These needs are relevant in a Danish context, with a generally high standard of living. Based on this conceptual framework of Need-based QoL for patients with multimorbidity, we will develop items for a new patient-reported outcome measure called the MMQ. Supplementary Information The online version contains supplementary material available at 10.1186/s41687-022-00489-0.
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Heaney A, McKenna SP, Hagell P. Evaluation of the Unidimensional Fatigue Impact Scale (U-FIS) in Crohn's Disease: The Importance of Local Item Dependency. J Nurs Meas 2021; 30:JNM-D-20-00116. [PMID: 34518414 DOI: 10.1891/jnm-d-20-00116] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/25/2022]
Abstract
BACKGROUND AND PURPOSE The Unidimensional Fatigue Impact Scale (U-FIS) was developed for use in a multiple sclerosis population. The aim was to determine whether the U-FIS is a valid tool for measuring the impact of fatigue in Crohn's disease (CD). METHOD CD patients completed the U-FIS as part of a validation study of the Crohn's Life Impact Questionnaire (CLIQ). Data were analyzed according to Rasch measurement theory (RMT). RESULTS Two hundred sixty-one completed U-FIS questionnaires were available for analysis. After rescoring the items to resolve disordered thresholds, all 22 items showed acceptable RMT fit. However, there was considerable local item dependency (LID). CONCLUSION The U-FIS did not provide unidimensional measurement in a sample of CD patients due to high levels of LID. Combining the three FIS outcomes into a single measure was not justified.
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Affiliation(s)
| | - Stephen P McKenna
- Galen Research Ltd, Manchester, UK; University of Manchester, Manchester, UK
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Role of Quality of Life as Endpoint for Inflammatory Bowel Disease Treatment. INTERNATIONAL JOURNAL OF ENVIRONMENTAL RESEARCH AND PUBLIC HEALTH 2021; 18:ijerph18137159. [PMID: 34281095 PMCID: PMC8296948 DOI: 10.3390/ijerph18137159] [Citation(s) in RCA: 25] [Impact Index Per Article: 6.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Subscribe] [Scholar Register] [Received: 05/05/2021] [Revised: 06/18/2021] [Accepted: 07/01/2021] [Indexed: 02/06/2023]
Abstract
Inflammatory bowel diseases (IBDs) are chronic disabling conditions, characterized by an unpredictable course with flare-ups and periods of remission, that frequently affect young people and require lifelong medical follow-up and treatment. For years, the main endpoints of IBD treatment had been clinical remission and response, followed by biomarker normalization and mucosal healing. In the last decades, different therapies have been proved to be effective to treat IBD and the use of patient reported outcome (PRO) have become more relevant. Therefore, health-related quality of life (HRQoL) that has been defined as the value assigned to the duration of life influenced by physical and mental health, has been suggested as an important endpoint for IBD management since multiple studies have shown that IBD impairs it, both physically and psychologically. Thus, HRQoL has been included as an outcome in numerous studies evaluating different IBD therapies, both clinical trials and real-life studies. It has been assessed by using both generic and specific disease tools, and most treatments used in clinical practice have been demonstrated to improve HRQoL. The relevance of HRQoL as an endpoint for new drugs is going to increase and its management and improvement will also improve the prognosis of IBD patients.
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Patient-Reported Outcomes in Inflammatory Bowel Disease: A Measurement of Effect in Research and Clinical Care. GASTROENTEROLOGY INSIGHTS 2021. [DOI: 10.3390/gastroent12020020] [Citation(s) in RCA: 6] [Impact Index Per Article: 1.5] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Indexed: 12/19/2022] Open
Abstract
The measurement of outcomes is key in evaluating healthcare or research interventions in inflammatory bowel disease (IBD). In patient-centred care, patient-reported outcome measures (PROMs) are central to this evaluation. In this review, we provide an overview of validated, adult disease-specific PROMs developed for use in IBD. Our aim is to assist clinicians and researchers in selection of PROMs to measure outcomes in their patient cohort. The Consensus-based Standards for the Selection of Health Measurement Instruments database of systematic reviews was the primary resource used to identify PROMs used in IBD. Search terms were ‘Crohn’s disease’, ‘ulcerative colitis’, and ‘IBD’. Seven systematic reviews were identified from this search. In addition, the publication by the IBD Core Outcome Set Working Group was used to identify further PROMs. Three systematic reviews were excluded as they did not meet the inclusion criteria. From the five included systematic reviews, we identified 21 PROMs and their shortened versions. In conclusion, it does not appear that any one PROM is entirely suitable for both research and clinical practice. Overall, the IBDQ-32 is most widely used in research but has the limitation of cost, whereas the IBD-Control has been recommended in the clinical core outcome set.
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14
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McKenna SP, Heaney A, Langley PC. Fundamental Outcome Measurement: Selecting Patient Reported Outcome Instruments and Interpreting the Data they Produce. Innov Pharm 2021; 12:10.24926/iip.v12i2.3911. [PMID: 34345514 PMCID: PMC8326699 DOI: 10.24926/iip.v12i2.3911] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/18/2022] Open
Abstract
Over the past 40 years literally thousands of generic and disease specific patient reported outcome (PRO) instruments have been developed. While most were developed for a specific study and were never used again, there is still the question of how manufacturers and others should select a PRO instrument for a study. These studies may be clinical pivotal trials or observational tracking studies to support therapy response. Formulary committees also need to be able to interpret PRO data to make decisions about whether to accept claims for therapy response. It is possible to argue that the many different approaches to outcome measurement have resulted from the lack of agreed methodologies. However, a more likely explanation is that the authors have failed to apply the axioms of fundamental measurement when creating their measures. The result is a plethora of ordinal PRO instruments that inform little about the impact of interventions. Clinical trials rarely report PRO data. Where they do, analyses are generally restricted to (for example) changes in the experimental group's scores. Comparisons between the treatment and placebo groups or between active groups are infrequently reported, most likely due to the failure of the instrument to show differences or changes in outcome. This is unfortunate as it means no assessment is made of the value that patients gain from the intervention. This commentary is intended to make researchers and formulary committees aware of the issues that need to be addressed when selecting PRO instruments for a study or evaluating publications and claims for therapy response. The latter is crucial as reported data influence the selection of medicines and healthcare products. In the latter case a particular concern is with PRO claims embedded in simulation models.
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Affiliation(s)
| | | | - Paul C Langley
- University of Minnesota, Minnesota MN USA; Maimon Research, Tucson AZ USA
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15
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Abstract
The quality adjusted life year (QALY) has serious problems related to its failure to adhere to measurement theory. If a QALY is to be meaningful, the utility score that translates time spent to an equivalent time spent in so-called perfect health must have ratio properties (i.e., it must support multiplication). Multiattribute utility scores (e.g. those generated by the EQ-5D-5L) fail to meet this standard. The multiattribute instruments produce ordinal scores that lack a true zero and they generate negative values. The manifest deficiencies of multiattribute utility instruments render them unfit, not only as a measure of therapy response but also in generating QALY claims. After 30 years of belief in their use, utilities and QALYs are clearly analytical dead ends. The purpose of this commentary is to demonstrate a coherent way forward in health technology assessment by focusing, not on clinical attributes as surrogates for quality of life, but on measures that are based on a conceptual model describing patient value in terms of need-fulfilment. Building on an extensive, yet often overlooked literature, need-based measures that fit Rasch Measurement Theory criteria are converted from ordinal scores to interval scores to evaluate response to therapy. These measures meet the requirements of single attribute fundamental measurement which is the standard in the physical sciences. It is proposed that a translation from a Rasch interval scale (defined by logits) can be transformed to a bounded ratio scale. Need based Quality of Life (N-QOL) scales bounded by 0 (where no needs are fulfilled) to 1 (where all needs are fulfilled) form such scales. The N-QOL supports the full range of arithmetic operations. Multiattribute utilities and mathematically invalid QALYs can be put to one side as unfortunate historical curiosities in favor of a disease or target population specific N-QOL scale. Such a scale has the required properties to evaluate disease specific response to therapy This can also support N-QOL adjusted life years with a need- fulfillment life year (NALY) metric with ratio properties.
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Affiliation(s)
- Paul C Langley
- College of Pharmacy, University of Minnesota, Minnesota MN USA; Maimon Research, Tucson AZ USA
| | - Stephen P McKenna
- Population Health, University of Manchester, Manchester UK; Galen Research, Manchester UK
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Peyrin-Biroulet L, Louis E, Loftus EV, Lacerda A, Zhou Q, Sanchez Gonzalez Y, Ghosh S. Quality of Life and Work Productivity Improvements with Upadacitinib: Phase 2b Evidence from Patients with Moderate to Severe Crohn's Disease. Adv Ther 2021; 38:2339-2352. [PMID: 33755884 PMCID: PMC8107073 DOI: 10.1007/s12325-021-01660-7] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/21/2020] [Accepted: 02/08/2021] [Indexed: 12/30/2022]
Abstract
Introduction In the phase 2 CELEST study, positive efficacy results were obtained with the Janus kinase 1 inhibitor upadacitinib for adult patients with moderate to severe Crohn’s disease. We present the health-related quality of life and work productivity improvement results with upadacitinib from CELEST. Methods CELEST (NCT02365649) was a double-blind study where patients were randomized 1:1:1:1:1:1 in the 16-week induction period to placebo or upadacitinib 3 mg twice daily (BID), 6 mg BID, 12 mg BID, 24 mg BID, or 24 mg once daily (QD). Patients completing the induction period were re-randomized 1:1:1 to receive upadacitinib 3 mg BID, 12 mg BID, or 24 mg QD for 36 weeks or 3 mg BID, 6 mg BID, or 12 mg BID (after amendment). Inflammatory Bowel Disease Questionnaire (IBDQ), European Quality of Life-5 Dimensions visual analog scale (EQ-5D VAS), and Work Productivity and Activity Impairment (WPAI) questionnaire outcomes were assessed at baseline and Weeks 8, 16, and 52. Results At Week 16, a significant percentage (P ≤ 0.05) of patients receiving upadacitinib 6-mg BID dose or higher achieved IBDQ response (IBDQ score change ≥ 16 points; 49%–57% for upadacitinib vs. 24% for placebo) and IBDQ remission, except 24 mg QD (IBDQ score ≥ 170; 26%–39% for upadacitinib vs. 11% for placebo). Greater improvements in IBDQ total score, EQ-5D VAS, and activity impairment from baseline (P ≤ 0.1) versus placebo were also observed. Larger improvements (P ≤ 0.1) in IBDQ response and total score and EQ-5D VAS were observed at Week 8 with 6 and 24 mg BID versus placebo, with improvements for all dosages maintained or greater at Week 52 for IBDQ, EQ-5D VAS, and WPAI endpoints, in particular for the 12-mg BID group. Conclusion Improvements in health-related quality of life and work productivity were achieved and sustained with upadacitinib in patients with moderate to severe Crohn’s disease. Trial Registration ClinicalTrials.gov identifier, NCT02365649. Supplementary Information The online version contains supplementary material available at 10.1007/s12325-021-01660-7.
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Affiliation(s)
- Laurent Peyrin-Biroulet
- Department of Gastroenterology, Nancy University Hospital, Nancy, France.
- Université de Lorraine, Nancy, France.
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Horton MC, Oyebode J, Clare L, Megson M, Shearsmith L, Brayne C, Kind P, Hoare Z, Al Janabi H, Hewison V, Tennant A, Wright P. Measuring Quality of Life in Carers of People With Dementia: Development and Psychometric Evaluation of Scales measuring the Impact of DEmentia on CARers (SIDECAR). THE GERONTOLOGIST 2021; 61:e1-e11. [PMID: 31688902 PMCID: PMC8023371 DOI: 10.1093/geront/gnz136] [Citation(s) in RCA: 11] [Impact Index Per Article: 2.8] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 06/06/2019] [Indexed: 02/07/2023] Open
Abstract
BACKGROUND AND OBJECTIVES A 2008 European consensus on research outcome measures in dementia care concluded that measurement of carer quality of life (QoL) was limited. Three systematic reviews (2012, 2017, and 2018) of dementia carer outcome measures found existing instruments wanting. In 2017, recommendations were published for developing reliable measurement tools of carers' needs for research and clinical application. The aim of this study was to develop a new instrument to measure the QoL of dementia carers (family/friends). METHODS Items were generated directly from carers following an inductive needs-led approach. Carers (n = 566) from 22 English and Welsh locations then completed the items and comparator measures at three time points. Rasch, factor, and psychometric (reliability, validity, responsiveness, and minimally important differences [MIDs]) analyses were undertaken. RESULTS Following factor analysis, the pool of 70 items was refined to three independent scales: primary SIDECAR-D (direct impact of caring upon carer QOL, 18 items), secondary SIDECAR-I (indirect impact, 10 items), and SIDECAR-S (support and information, 11 items). All three scales satisfy Rasch model assumptions. SIDECAR-D, I, S psychometrics: reliability (internal ≥ .70; test-retest ≥ .85); convergent validity (as hypothesized); responsiveness (effect sizes: D: moderate; I and S: small); MIDs (D = 9/100, I = 10/100, S = 11/100). DISCUSSION AND IMPLICATIONS SIDECAR scales demonstrate robust measurement properties, meeting COSMIN quality standards for study design and psychometrics. SIDECAR provides a theoretically based needs-led QoL profile specifically for dementia carers. SIDECAR is free for use in public health, social care, and voluntary sector services, and not-for-profit organizations.
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Affiliation(s)
- Mike C Horton
- Psychometric Laboratory for Health Sciences, University of Leeds, UK
| | - Jan Oyebode
- Centre for Applied Dementia Studies, University of Bradford, UK
| | - Linda Clare
- Centre for Research in Ageing and Cognitive Health, University of Exeter, UK
| | - Molly Megson
- Leeds Institute of Medical Research at St James’s, University of Leeds, UK
| | - Leanne Shearsmith
- Leeds Institute of Medical Research at St James’s, University of Leeds, UK
| | - Carol Brayne
- Cambridge Institute of Public Health, University of Cambridge, UK
| | - Paul Kind
- Leeds Institute of Health Sciences, University of Leeds, UK
| | - Zoe Hoare
- School of Health Sciences, Bangor University, UK
| | - Hareth Al Janabi
- Institute of Applied Health Research, University of Birmingham, UK
| | | | - Alan Tennant
- Psychometric Laboratory for Health Sciences, University of Leeds, UK
| | - Penny Wright
- Centre for Research in Ageing and Cognitive Health, University of Exeter, UK
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18
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McKenna SP, Heaney A. Setting and maintaining standards for patient-reported outcome measures: can we rely on the COSMIN checklists? J Med Econ 2021; 24:502-511. [PMID: 33759686 DOI: 10.1080/13696998.2021.1907092] [Citation(s) in RCA: 13] [Impact Index Per Article: 3.3] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Indexed: 12/13/2022]
Abstract
As test-developers we have often been troubled by published reviews of patient-reported outcome measures (PROMs). Too often minor issues are judged important while other reviews exclude the best measures available. Perhaps this led several groups to make recommendations for evaluating the quality of PROMs. The COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist is the latest set of recommendations. While reviewing the COSMIN literature and reviews conducted using their recommendations several concerns became apparent. The checklist is not evidence-based, relying on the opinion of researchers experienced in health-related quality of life. PROMs measuring other types of outcomes are inadequately covered by the checklist. COSMIN choose to focus on Classical Test Theory and the checklists are not appropriate for use with PROMs developed using modern measurement. Such an approach only obstructs progress in the field of outcome measurement. The retrospective nature of the evaluations also penalizes new PROMs. While the checklists imply that composite, ordinal level measurement is acceptable, crucial aspects of instrument development and quality are excluded. Reviews based on the COSMIN checklist produce contradictory conclusions and fail to provide evidence to support the recommendations. These problems suggest that the checklists themselves lack reliability and validity. It is also clear that several reviewers lack the expertise to apply the checklists. Researchers require a good grounding in instrument development and psychometrics to produce quality reviews. The science of modern PROM development is still in an early phase. Few available PROMs have sufficient quality, limiting the need for complex reviews. Standards need to be agreed for high quality outcome measurement. The issue is who should set these standards? Most published reviews merely scratch the surface and lack essential detail. All reviews of PROMs should be treated with caution, irrespective of whether the COSMIN checklist was employed.
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Affiliation(s)
- Stephen P McKenna
- Galen Research, Manchester, UK
- School of Health Sciences, University of Manchester, Manchester, UK
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19
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Mokkink LB, Terwee CB, Bouter LM, Alonso JA, Patrick D, de Vet HCW. Reply to the concerns raised by McKenna and Heaney about COSMIN. J Med Econ 2021; 24:857-859. [PMID: 34166175 DOI: 10.1080/13696998.2021.1948231] [Citation(s) in RCA: 5] [Impact Index Per Article: 1.3] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Indexed: 12/23/2022]
Affiliation(s)
- Lidwine B Mokkink
- Department of Epidemiology and Data Science, Amsterdam Public Health Research Institute, Amsterdam UMC, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands
| | - Caroline B Terwee
- Department of Epidemiology and Data Science, Amsterdam Public Health Research Institute, Amsterdam UMC, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands
| | - Lex M Bouter
- Department of Epidemiology and Data Science, Amsterdam Public Health Research Institute, Amsterdam UMC, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands
- Department of Philosophy, Faculty of Humanities, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands
| | - Jordi A Alonso
- Health Services Research Unit, IMIM-Hospital del Mar Medical Research Institute, CIBER Epidemiología y Salud Pública (CIBERESP), Barcelona, Spain
| | - D Patrick
- Department of Health Services, University of Washington, Seattle, WA, USA
| | - Henrica C W de Vet
- Department of Epidemiology and Data Science, Amsterdam Public Health Research Institute, Amsterdam UMC, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands
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20
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Heaney A, Wilburn J, Rouse M, Langmead S, Blakeley JO, Huson S, McKenna SP. The development of the PlexiQoL: A patient-reported outcome measure for adults with neurofibromatosis type 1-associated plexiform neurofibromas. Mol Genet Genomic Med 2020; 8:e1530. [PMID: 33085177 PMCID: PMC7767563 DOI: 10.1002/mgg3.1530] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.6] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/13/2018] [Revised: 10/10/2018] [Accepted: 09/25/2020] [Indexed: 11/11/2022] Open
Abstract
Background To develop and validate a patient‐reported outcome (PRO) measure of quality of life (QoL), specific to patients with Neurofibromatosis Type 1 (NF1)‐associated plexiform neurofibromas (pNFs), suitable for use in clinical efficacy trials. The study was conducted in parallel in the UK and US. Methods Qualitative interviews were conducted with patients to generate questionnaire content. Face and content validity of the draft scale was assessed by cognitive debriefing interviews (CDIs). A postal validation survey was conducted to identify the final version of the questionnaire (the PlexiQoL), establish its unidimensionality, and assess its psychometric properties. Results Thematic analysis was performed on 42 interview transcripts. Thirty‐one CDIs revealed that patients found the draft scale to be comprehensible, relevant, and easy to complete. The postal validation survey was completed by 273 patients. Rasch analysis identified an 18‐item unidimensional scale that showed excellent internal consistency, reproducibility, and sensitivity to differences in patient‐perceived pNF severity, general health, and the use of pain medication. Conclusions The PlexiQoL is the first disease‐specific PRO assessing the ability of adults with NF‐1 associated pNFs to meet their basic human needs. Clinical trials are planned to assess the sensitivity to change of the PlexiQoL in people undergoing treatment for pNFs.
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Affiliation(s)
| | | | | | - Shannon Langmead
- Johns Hopkins Comprehensive Neurofibromatosis Clinic, Baltimore, MD, USA
| | - Jaishri O Blakeley
- Johns Hopkins Comprehensive Neurofibromatosis Clinic, Baltimore, MD, USA
| | - Susan Huson
- Manchester Centre for Genomic Medicine, Saint Mary's Hospital, Manchester, UK
| | - Stephen P McKenna
- Galen Research, Manchester, UK.,School of Health Sciences, The University of Manchester, Manchester, UK
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21
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Abstract
Composite measures that combine different types of indicators are widely used in medical research; to evaluate health systems, as outcomes in clinical trials and patient-reported outcome measurement. The potential advantages of such indices are clear. They are used to summarise complex data and to overcome the problem of evaluating new interventions when the most important outcome is rare or likely to occur far in the future. However, many scientists question the value of composite measures, primarily due to inadequate development methodology, lack of transparency or the likelihood of producing misleading results. It is argued that the real problems with composite measurement are related to their failure to take account of measurement theory and the absence of coherent theoretical models that justify the addition of the individual indicators that are combined into the composite index. All outcome measures must be unidimensional if they are to provide meaningful data. They should also have dimensional homogeneity. Ideally, a specification equation should be developed that can predict accurately how organisations or individuals will score on an index, based on their scores on the individual indicators that make up the measure. The article concludes that composite measures should not be used as they fail to apply measurement theory and, consequently, produce invalid and misleading scores.
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Affiliation(s)
- Stephen P McKenna
- Galen Research Ltd., Manchester, UK
- School of Health Sciences, University of Manchester, Manchester, UK
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22
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van Andel EM, Koopmann BDM, Crouwel F, Noomen CG, de Boer NKH, van Asseldonk DP, Mokkink LB. Systematic Review of Development and Content Validity of Patient-reported Outcome Measures in Inflammatory Bowel Disease: Do We Measure What We Measure? J Crohns Colitis 2020; 14:1299-1315. [PMID: 32211749 PMCID: PMC7493218 DOI: 10.1093/ecco-jcc/jjaa057] [Citation(s) in RCA: 14] [Impact Index Per Article: 2.8] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Indexed: 02/07/2023]
Abstract
BACKGROUND AND AIMS Patient-reported outcome measures are increasingly important in daily care and research in inflammatory bowel disease [IBD]. This study provides an overview of the content and content validity of IBD-specific patient-reported outcome measures on three selected constructs. METHODS Databases were searched up to May 2019 for development and/or content validity studies on IBD-specific self-report measures on health-related quality of life, disability, and self-report disease activity in adults. Evidence was synthesised on content validity in three aspects: relevance, comprehensiveness, and comprehensibility following the COnsensus-based Standards for the selection of health Measurement INstruments methodology. Questionnaire items were organised in themes to provide an overview of important aspects of these constructs. RESULTS For 14/44 instruments, 25 content validity studies were identified and 25/44 measures had sufficient content validity, the strongest evidence being of moderate quality, though most evidence is of low or very low quality. The Crohn's Life Impact Questionnaire and IBD questionnaire-32 on quality of life, the IBD-Control on disease activity, and the IBD Disability Index Self-Report and its 8-item version on disability, have the strongest evidence of sufficient relevance, comprehensiveness, and comprehensibility, ranging from moderate to very low quality. A fair number of recurring items themes, possibly important for the selected constructs, was identified. CONCLUSIONS The body of evidence for content validity of IBD-specific health-related quality of life, self-report disease activity, and disability self-report measures is limited. More content validity studies should be performed after reaching consensus on the constructs of interest for IBD, and studies should involve patients.
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Affiliation(s)
- Emma M van Andel
- Department of Gastroenterology and Hepatology, Noordwest Ziekenhuisgroep, Alkmaar, The Netherlands
| | - Brechtje D M Koopmann
- Department of Gastroenterology and Hepatology, Noordwest Ziekenhuisgroep, Alkmaar, The Netherlands
| | - Femke Crouwel
- Department of Gastroenterology and Hepatology, Noordwest Ziekenhuisgroep, Alkmaar, The Netherlands
| | - Casper G Noomen
- Department of Gastroenterology and Hepatology, Noordwest Ziekenhuisgroep, Alkmaar, The Netherlands
| | - Nanne K H de Boer
- Amsterdam UMC, Vrije Universiteit Amsterdam, Department of Gastroenterology and Hepatology, Amsterdam; Gastroenterology and Metabolism Research Institute, Amsterdam, The Netherlands
| | - Dirk P van Asseldonk
- Department of Gastroenterology and Hepatology, Noordwest Ziekenhuisgroep, Alkmaar, The Netherlands
| | - Lidwine B Mokkink
- Amsterdam UMC, Vrije Universiteit Amsterdam, Department of Epidemiology and Biostatistics; Amsterdam Public Health Research Institute, Amsterdam, The Netherlands
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Abstract
The QALY is an impossible construct; it defies common sense. It fails completely once we consider the axioms of fundamental measurement. Utilities as ordinal scales cannot be used to create QALYS. The QALY should never have been introduced to support the value assessment of pharmaceutical products and devices. The result is 30 years of QALY based assessments of pharmaceutical products and devices which are conceptually and technically wrong. They are a charade and will have contributed mistakenly to thousands of formulary decisions. In the search for a common metric to evaluate cost-effectiveness the impossibility of a QALY was overlooked. The result is a disaster, unfolding over decades. Our next steps must be to abandon the QALY paradigm and look ahead to a new value assessment framework.
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Affiliation(s)
- Paul C. Langley
- Adjunct Professor, College of Pharmacy, University of Minnesota
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24
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Langley PC. The National Pharmaceutical Council: Endorsing the Construction of Imaginary Worlds in Health Technology Assessment. PHARMACY 2020; 8:E119. [PMID: 32668706 PMCID: PMC7557741 DOI: 10.3390/pharmacy8030119] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Journal Information] [Subscribe] [Scholar Register] [Received: 06/04/2020] [Revised: 07/06/2020] [Accepted: 07/08/2020] [Indexed: 12/03/2022] Open
Abstract
All too often, organizations embrace standards for health technology assessment that fail to meet those of normal science. A value assessment framework has been endorsed that is patently in the realm of pseudoscience. If a value assessment framework is to be accepted, then claims for the value of competing products must be credible, evaluable and replicable. If not, for example, when the assessment relies on the construction of an imaginary lifetime incremental cost-per-quality-adjusted-life-year (QALY) world, then that assessment should be rejected. Such an assessment would fail one of the central roles of normal science: the discovery of new facts through an ongoing process of conjecture and refutation where provisional claims can be continually challenged. It is no good defending an endorsement of a value framework that fails expected standards on the grounds that it has been endorsed by professional groups and reflects decades of development. This is intellectually lazy. If this is the case, then the scientific revolution of the 17th century need not have happened. The purpose of this commentary is to consider the recommended standards for health technology assessment of the National Pharmaceutical Council (NPC), with particular reference to proposed methodological standards in value assessment and the commitment to mathematically impossible QALYs.
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Affiliation(s)
- Paul C Langley
- College of Pharmacy, University of Minnesota, 308 SE Harvard St, Minneapolis, MN 55455, USA
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25
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McKenna SP, Rouse M, Heaney A, Hagell P. International Development of the Alzheimer's Patient Partners Life Impact Questionnaire (APPLIQue). Am J Alzheimers Dis Other Demen 2020; 35:1533317520951690. [PMID: 32851850 PMCID: PMC10624100 DOI: 10.1177/1533317520951690] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.6] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 02/06/2023]
Abstract
AIM Informal caregivers play a vital role in the care of people with Alzheimer's Disease (AD), yet caregiving is associated with caregivers' burden. The initial objective of the study was to develop a new outcome measure to assess quality of life (QoL) in AD caregivers. METHODS Informal (non-professional) caregivers providing 75% or more of the care activities for, and living in the same household as, a person with AD were invited to take part in the study. Qualitative interviews (N = 40) were conducted with AD caregivers in the UK and thematic analyses were applied to generate a pool of potential items. A draft questionnaire was produced and adapted for use in Italy, Spain, Germany and the US. In each of the 5 countries, cognitive debriefing interviews (N = 76) were conducted to determine the questionnaire's face and content validity, followed by a postal validation survey (N = 268). The data from these surveys were combined to reduce the number of items and assess the new questionnaire's psychometric properties. RESULTS Thematic analysis of the UK interview transcripts generated a draft questionnaire, which was successfully translated into each additional language. The items were well accepted and easy to complete. However, reanalysis of the qualitative interview data revealed that spousal and non-spousal caregivers identified different experiences of caregiving. A review of the item pool indicated that items were primarily targeted at spousal caregivers. Therefore, further analyses of the postal survey data included spousal caregivers only (n = 116). The results supported scaling assumptions (e.g., corrected item-total correlations ≥0.32), targeting (e.g., floor/ceiling effects <2.5%), internal consistency (α ≥0.93) and test-retest reliability (rs = 0.88) of the new questionnaire, according to classical test theory. Assessment of external construct validity yielded results in accordance with a priori expectations. QoL scores were most strongly related to scores on the emotional reactions sections of the Nottingham Health Profile and the General Well-Being Index. The new questionnaire was found to be capable of detecting meaningful differences between respondents; spousal caregivers had worse QoL when the person with AD was confused (p < .001), could not be left alone (p < .001), did not recognize the caregiver (p < .001), was incontinent (p < .05), and wandered around the house (p = .01). CONCLUSIONS The Alzheimer's Disease Patient Partners Life Impact Questionnaire (APPLIQue) is a questionnaire specific to spousal caregivers of people with AD. Data support its scaling assumptions and it exhibits excellent psychometric properties according to classical test theory. The questionnaire is recommended for use in intervention studies where the QoL of spousal caregivers is of interest.
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Affiliation(s)
- Stephen P. McKenna
- Galen Research Ltd, Manchester, United Kingdom
- School of Health Sciences, University of Manchester, Manchester, United Kingdom
| | | | | | - Peter Hagell
- The PRO-CARE Group, Faculty of Health Sciences, Kristianstad University, Kristianstad, Sweden
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Abstract
Patient-reported outcome measures (PROMs) are used to collect information directly from patients. They may cover several different types of outcomes ranging from symptoms, functioning, utility, satisfaction, through to quality-of-life (QoL). They generally consist of self-completed questionnaires that can be administered by means of hard copies or in a range of electronic formats. PROMs vary considerably in terms of the constructs they assess, the care with which they are developed, and their scientific quality. However, none of the PROMs available approach the quality of measurement achieved by measures/instruments used in physics. PROs are examples of latent variables. These are not directly observable, but can be inferred from, for example, responses to a questionnaire. The only measure of a latent variable that approaches the quality of measurement achieved by the physical sciences is the Lexile Framework for Reading. This framework is based on a construct theory that grew out of an analysis of several available reading measures. A specification equation was generated that was able to link the construct theory to scores obtained with the Lexile measure. A fundamental requirement of this quality of measurement is that the data collected with the model fit Rasch Measurement Theory (RMT). It is argued that PROM developers should aspire to match this level of measurement sophistication if their instruments are to provide valid insights into the impacts of disease and its treatment.
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Affiliation(s)
- Stephen P McKenna
- a Galen Research Ltd , Manchester , UK
- b School of Health Sciences, University of Manchester , Manchester , UK
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Abstract
All instruments designed to measure latent (unobservable) variables, such as patient-reported outcomes (PROs), have three major requirements; a coherent construct theory, a specification equation, and the application of an appropriate response model. The theory guides the selection of content for the questionnaire and the specification equation links the construct theory to scores produced with the instrument. For the specification equation to perform this role, the patient-reported outcome measure (PROM) must employ a response model that generates values for its individual items. The most commonly applied response model in PROM development is the Rasch model. To date this level of measurement sophistication has not been achieved in PRO measurement. Consequently, it is not possible to establish a PROM's true construct validity. However, the development of the Lexile Framework for Reading has demonstrated that such objective measurement is possible for latent variables. This article argues that higher quality PROM development is needed if meaningful and valid PRO measurement is to be achieved. It describes the current state of PROM development, shows that published reviews of PROMs adopt inappropriate criteria for judging their quality, and illustrates how the use of traditional PROMs can lead to incorrect (and possibly dangerous) conclusions being drawn about the efficacy of interventions.
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Affiliation(s)
- Stephen P McKenna
- a Galen Research Ltd , Manchester , UK
- b School of Health Sciences , University of Manchester , Manchester , UK
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Oyebode JR, Pini S, Ingleson E, Megson M, Horton M, Clare L, Al-Janabi H, Brayne C, Wright P. Development of an Item Pool for a Needs-Based Measure of Quality of Life of Carers of a Family Member with Dementia. PATIENT-PATIENT CENTERED OUTCOMES RESEARCH 2019; 12:125-136. [PMID: 30315554 DOI: 10.1007/s40271-018-0334-4] [Citation(s) in RCA: 7] [Impact Index Per Article: 1.2] [Reference Citation Analysis] [Abstract] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 11/29/2022]
Abstract
BACKGROUND AND OBJECTIVES This paper describes the development of an item pool for a needs-based self-report outcome measure of the impact of caring for a relative, friend or neighbour with dementia on carer quality of life. The aims are to give a detailed account of the steps involved and describe the resulting item pool. METHODS Seven steps were followed: generation of an initial item set drawing on 42 needs-led interviews with carers; a content and face validity check; assessment of psychometric potential; testing of response formats; pre-testing through cognitive interviews with 22 carers; administration rehearsal with two carers; and final review. RESULTS An initial set of 99 items was refined to a pool of 70 to be answered using a binary response format. Items were excluded due to overlap with others, ceiling effects, ambiguity, dependency on function of the person with dementia or two-part phrasing. Items retained covered a breadth of areas of impact of caring and were understandable and acceptable to respondents. CONCLUSIONS The resulting dementia carer-specific item pool reflects the accounts of a diverse sample of those who provide care for a person with dementia, allowing them to define the nature of the impact on their lives and resulting in a valid, acceptable set of items.
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Affiliation(s)
- Jan R Oyebode
- Centre for Applied Dementia Studies, Faculty of Health Studies, University of Bradford, Bradford, UK.
| | - Simon Pini
- Section of Patient Centred Outcome Research, Leeds Institute of Cancer and Pathology, University of Leeds, Leeds, UK
| | - Emma Ingleson
- Section of Patient Centred Outcome Research, Leeds Institute of Cancer and Pathology, University of Leeds, Leeds, UK
| | - Molly Megson
- Section of Patient Centred Outcome Research, Leeds Institute of Cancer and Pathology, University of Leeds, Leeds, UK
| | - Mike Horton
- Psychometric Laboratory for Health Sciences, Faculty of Medicine and Health, University of Leeds, Leeds, UK
| | - Linda Clare
- The Centre for Research in Ageing and Cognitive Health, Medical School, University of Exeter, Exeter, UK
| | - Hareth Al-Janabi
- Health Economics Unit, Institute of Applied Health Research, University of Birmingham, Birmingham, UK
| | - Carol Brayne
- Department of Public Health and Primary Care, University of Cambridge, Cambridge, UK
| | - Penny Wright
- Section of Patient Centred Outcome Research, Leeds Institute of Cancer and Pathology, University of Leeds, Leeds, UK
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Heaney A, Wilburn J, Langmead S, Blakeley J, Huson S, Jim C, McKenna SP. A qualitative study of the impact of plexiform neurofibromas on need fulfilment in adults with neurofibromatosis type 1. SAGE Open Med 2019; 7:2050312119829680. [PMID: 30800298 PMCID: PMC6378462 DOI: 10.1177/2050312119829680] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/28/2018] [Accepted: 01/15/2019] [Indexed: 11/16/2022] Open
Abstract
Objective To explore the impact of plexiform neurofibromas on the lives of adults with neurofibromatosis type 1. Background Neurofibromatosis type 1 is a complex neurogenetic syndrome that affects many aspects of health and functioning. A common manifestation of neurofibromatosis type 1 is plexiform neurofibromas, non-cancerous tumours that can cause disfigurement, pain and neurologic disability. Patient-reported outcome measures used in this condition have addressed symptoms and functional ability but not how the condition affects patients' lives, particularly, their ability to meet their human needs. Methods Unstructured qualitative interviews were conducted with adults with neurofibromatosis type 1-associated plexiform neurofibromas in the United Kingdom and United States. Interviewees were encouraged to describe how plexiform neurofibromas affected their ability to meet their needs. Interviews were audio-recorded and transcribed verbatim. The UK and US transcripts were combined and theoretical thematic analysis was conducted. Results In all, 42 interviews (United Kingdom = 20, United States = 22) were conducted. Transcripts revealed 696 statements on the impact of plexiform neurofibromas on need fulfilment. Five major themes emerged: appearance, relationships, independence, role fulfilment and pleasure. Conclusion Neurofibromatosis type 1-associated plexiform neurofibromas have a major effect on individuals' ability to meet their needs. An understanding of need fulfilment will complement information generated from traditional patient-reported outcome measures, particularly in a multi-faceted syndrome such as neurofibromatosis type 1.
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Affiliation(s)
| | | | - Shannon Langmead
- Johns Hopkins Comprehensive Neurofibromatosis Center, Baltimore, MD, USA
| | - Jaishri Blakeley
- Johns Hopkins Comprehensive Neurofibromatosis Center, Baltimore, MD, USA
| | - Susan Huson
- Manchester Centre for Genomic Medicine, Saint Mary's Hospital, Manchester, UK
| | - Carly Jim
- Department of Psychology, Manchester Metropolitan University, Manchester, UK
| | - Stephen P McKenna
- Galen Research, Manchester, UK.,School of Health Sciences, The University of Manchester, Manchester, UK
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Pini S, Ingleson E, Megson M, Clare L, Wright P, Oyebode JR. A Needs-led Framework for Understanding the Impact of Caring for a Family Member With Dementia. THE GERONTOLOGIST 2018; 58:e68-e77. [PMID: 29562360 PMCID: PMC5946854 DOI: 10.1093/geront/gnx148] [Citation(s) in RCA: 19] [Impact Index Per Article: 2.7] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/21/2017] [Accepted: 10/02/2017] [Indexed: 11/15/2022] Open
Abstract
Background and Objectives Approximately half the care for people with dementia is provided by families. It is therefore imperative that research informs ways of maintaining such care. In this study, we propose that a needs-led approach can provide a useful, novel means of conceptualizing the impact of caring on the lives of family carers. Our aim was to develop and present a needs-led framework for understanding how providing care impacts on carers’ fulfilment of needs. Design and Methods In this qualitative study, we conducted 42 semistructured interviews with a purposively diverse sample of family carers to generate nuanced contextualized accounts of how caring impacted on carers’ lives. Our inductive thematic analysis focused upon asking: “What need is being impacted here?” in order to generate a needs-led framework for understanding. Results Nine themes were widely endorsed. Each completed the sentence: “Being a carer impacts on fulfilling my need to/for….”: Freedom; feel close to my relative; feel in control of my life; be my own person; protect my relative; share/express my thoughts and feelings; take care of myself; feel connected to the people around me; get things done. Discussion and Implications These needs echo those from other research areas, with relational needs emerging as particularly central. The needs-led approach offers a perspective that is able to capture both stresses and positive aspects of caregiving. We recommend that clinical interviewing using Socratic questioning to discover human needs that are being impacted by caring would provide a valuable starting point for care planning.
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Affiliation(s)
- Simon Pini
- Patient Centred Outcome Research Group, St James's Institute of Oncology, University of Leeds, West Yorkshire, UK
| | - Emma Ingleson
- Patient Centred Outcome Research Group, St James's Institute of Oncology, University of Leeds, West Yorkshire, UK
| | - Molly Megson
- Patient Centred Outcome Research Group, St James's Institute of Oncology, University of Leeds, West Yorkshire, UK
| | | | - Penny Wright
- Patient Centred Outcome Research Group, St James's Institute of Oncology, University of Leeds, West Yorkshire, UK
| | - Jan R Oyebode
- Centre for Applied Dementia Studies, University of Bradford, Bradford, West Yorkshire, UK
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Elliott D. Developing outcome measures assessing wound management and patient experience: a mixed methods study. BMJ Open 2017; 7:e016155. [PMID: 29180591 PMCID: PMC5719294 DOI: 10.1136/bmjopen-2017-016155] [Citation(s) in RCA: 11] [Impact Index Per Article: 1.4] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 02/06/2017] [Revised: 07/13/2017] [Accepted: 07/19/2017] [Indexed: 11/17/2022] Open
Abstract
OBJECTIVES To develop outcome measures to assess practical management of primary surgical wounds and patient experience. DESIGN Mixed methods, including qualitative interviews and data extraction from published randomised controlled trials (RCTs). SETTING Two university-teaching NHS hospitals and three district NHS hospitals in the South West and Midlands regions of England. PARTICIPANTS Sixty-four patients and 15 healthcare professionals from abdominal general surgical specialities and obstetrics (caesarean section). METHODS Measures were developed according to standard guidelines to identify issues relevant to patients' experiences of surgical wounds and dressings, including analysis of existing RCT outcomes and semi-structured interviews. These were written into provisional questionnaire items for a single outcome measure. Cognitive interviews with patients and healthcare professionals assessed face validity, acceptability and relevance. Findings from interviews were regularly shared with the study team who suggested amendments to modify and reword items to improve understanding before further iterative testing with patients and healthcare professionals. RESULTS Analyses of existing RCT outcomes and interviews produced a total of 69 issues. Pretesting and iterative revision established the need for two separate measures. One measure addresses healthcare professionals' experience of wound management in two key areas: exudate and its impact, and allergic reactions to the dressing. The other measure addresses patients' experience of wounds in seven key areas: wound comfort, dressing removal, dressings to protect the wound, impact on daily activities, ease of movement, anxiety about the wound and satisfaction with dressing. Each measure took less than five min to complete and both were understood and acceptable to patients and healthcare professionals. CONCLUSION This in-depth study has developed two measures to assess practical management of primary surgical wounds and patient experience. Further work to test their validity, reliability and application to other settings is now required. TRIAL REGISTRATION NUMBER HTA - 12/200/04; Pre-results.
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Affiliation(s)
- Daisy Elliott
- Population Health Sciences, Bristol Medical School, University of Bristol, Canynge Hall, 39 Whatley Road, Bristol, BS8 2PS
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Chen XL, Zhong LH, Wen Y, Liu TW, Li XY, Hou ZK, Hu Y, Mo CW, Liu FB. Inflammatory bowel disease-specific health-related quality of life instruments: a systematic review of measurement properties. Health Qual Life Outcomes 2017; 15:177. [PMID: 28915891 PMCID: PMC5603012 DOI: 10.1186/s12955-017-0753-2] [Citation(s) in RCA: 67] [Impact Index Per Article: 8.4] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 06/29/2016] [Accepted: 09/07/2017] [Indexed: 12/16/2022] Open
Abstract
BACKGROUND This review aims to critically appraise and compare the measurement properties of inflammatory bowel disease (IBD)-specific health-related quality of life instruments. METHODS Medline, EMBASE and ISI Web of Knowledge were searched from their inception to May 2016. IBD-specific instruments for patients with Crohn's disease, ulcerative colitis or IBD were enrolled. The basic characteristics and domains of the instruments were collected. The methodological quality of measurement properties and measurement properties of the instruments were assessed. RESULTS Fifteen IBD-specific instruments were included, which included twelve instruments for adult IBD patients and three for paediatric IBD patients. All of the instruments were developed in North American and European countries. The following common domains were identified: IBD-related symptoms, physical, emotional and social domain. The methodological quality was satisfactory for content validity; fair in internal consistency, reliability, structural validity, hypotheses testing and criterion validity; and poor in measurement error, cross-cultural validity and responsiveness. For adult IBD patients, the IBDQ-32 and its short version (SIBDQ) had good measurement properties and were the most widely used worldwide. For paediatric IBD patients, the IMPACT-III had good measurement properties and had more translated versions. CONCLUSIONS Most methodological quality should be promoted, especially measurement error, cross-cultural validity and responsiveness. The IBDQ-32 was the most widely used instrument with good reliability and validity, followed by the SIBDQ and IMPACT-III. Further validation studies are necessary to support the use of other instruments.
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Affiliation(s)
- Xin-Lin Chen
- College of Basic Medical Science, Guangzhou University of Chinese Medicine, Guangzhou, China.
| | | | - Yi Wen
- The First Affiliated Hospital, The First Clinical College, Guangzhou University of Chinese Medicine, Guangzhou, China
| | - Tian-Wen Liu
- Guangdong Province Hospital of Chinese Medicine, Guangzhou University of Chinese Medicine, Guangzhou, China
| | - Xiao-Ying Li
- Jiangmen Wuyi Traditional Chinese Medicine Hospital, Jiangmen City, Guangdong Province, China
| | - Zheng-Kun Hou
- The First Affiliated Hospital, The First Clinical College, Guangzhou University of Chinese Medicine, Guangzhou, China
| | - Yue Hu
- College of Basic Medical Science, Guangzhou University of Chinese Medicine, Guangzhou, China
| | - Chuan-Wei Mo
- College of Basic Medical Science, Guangzhou University of Chinese Medicine, Guangzhou, China
| | - Feng-Bin Liu
- The First Affiliated Hospital, The First Clinical College, Guangzhou University of Chinese Medicine, Guangzhou, China.
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Wilburn J, McKenna SP, Heaney A, Rouse M, Taylor M, Culkin A, Gabe S, Burden S, Lal S. Development and validation of the Parenteral Nutrition Impact Questionnaire (PNIQ), a patient-centric outcome measure for Home Parenteral Nutrition. Clin Nutr 2017; 37:978-983. [PMID: 28446383 DOI: 10.1016/j.clnu.2017.04.004] [Citation(s) in RCA: 24] [Impact Index Per Article: 3.0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/07/2016] [Revised: 04/05/2017] [Accepted: 04/05/2017] [Indexed: 02/07/2023]
Abstract
BACKGROUND & AIMS Patients with Type 3 Intestinal Failure (IF) who need Home Parenteral Nutrition (HPN) face several clinical, psychological and social problems. The study was designed to produce and test the first patient-centric measure for HPN ('PNIQ: Parenteral Nutrition Impact Questionnaire'). The new measure focused on the extent to which patients were able to fulfil their human needs. METHODS Questionnaire content was derived from the analysis of transcripts of interviews conducted with UK HPN patients. Cognitive debriefing interviews (CDIs) were performed to ensure patients found the draft scale clear, relevant and accessible. Finally, a test--retest postal validation survey was conducted to reduce the number of items in the scale and to ensure that; it was unidimensional, reproducible and had construct validity. RESULTS The 30 interview transcripts were analysed to identify issues related to a wide range of needs. Fifteen CDIs showed that patients found the draft scale easy to complete and highly relevant. The postal survey included 233 patients on HPN recruited through two IF units. Items were rejected if they did not fit the Rasch model, had too similar content to other items or displayed differential item functioning related to age, gender or underlying mechanism of IF. A 20-item unidimensional scale was identified with high internal consistency (0.91) and test-retest reliability (0.92). Scores on PNIQ correlated moderately highly with social isolation, emotional reactions and energy level and were related to perceived interference on life of HPN. The underlying cause of IF did not influence the way the scale worked. CONCLUSIONS The PNIQ is a scientifically rigorous, unidimensional outcome measure that provides a complete assessment of the effect of HPN on everyday life. It will prove useful for measuring patient value in clinical practice and for determining outcome in clinical trials, audit, economic evaluations and outcomes-based reimbursement.
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Affiliation(s)
| | - Stephen P McKenna
- School of Health Sciences, University of Manchester, Oxford Road, Manchester M13 9PL, UK
| | | | | | - Michael Taylor
- Intestinal Failure Unit (H8), Salford Royal NHS Foundation Trust, Salford M6 8HD, UK
| | - Alison Culkin
- Lennard Jones Intestinal Failure Unit, St Mark's Hospital, Middlesex HA1 3UJ, UK
| | - Simon Gabe
- Lennard Jones Intestinal Failure Unit, St Mark's Hospital, Middlesex HA1 3UJ, UK
| | - Sorrel Burden
- School of Health Sciences, University of Manchester, Oxford Road, Manchester M13 9PL, UK; Intestinal Failure Unit (H8), Salford Royal NHS Foundation Trust, Salford M6 8HD, UK
| | - Simon Lal
- Intestinal Failure Unit (H8), Salford Royal NHS Foundation Trust, Salford M6 8HD, UK
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Niv G, Bar Josef S, Ben Bassat O, Avni I, Lictenstein L, Niv Y, Barnoy S. Quality of life and uncertainty in Crohn's disease. Qual Life Res 2017; 26:1609-1616. [PMID: 28181069 DOI: 10.1007/s11136-017-1509-5] [Citation(s) in RCA: 10] [Impact Index Per Article: 1.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Accepted: 01/17/2017] [Indexed: 01/01/2023]
Abstract
INTRODUCTION Crohn's disease impairs patients' perception of health and has a negative impact on health-related quality of life. Although it is apparent that uncertainty is a significant factor that decreases quality of life, it has never been studied in patients with Crohn's disease. The objective of the present study was to examine the association between level of certainty, self-epistemic authority, Internet information gathering habits, and health-related quality of life. METHODS A cross-sectional study of 105 Crohn's disease patients was conducted. Data were collected using a questionnaire composed of five parts: (1) demographic and clinical information; (2) health-related quality of life; (3) level of certainty; (4) self-epistemic authority; and (5) Internet information gathering habits regarding Crohn's disease. RESULTS A significant positive correlation was demonstrated between levels of certainty and health-related quality of life. Self-epistemic authority correlated positively with certainty, while information gathering via the Internet was related to decreased certainty. Multiple regression analysis for factors associated with health-related quality of life showed a positive association with the level of certainty, while negative associations were found between Internet information seeking and disease activity with the quality of life. CONCLUSION Level of certainty proved an important variable associated with health-related quality of life in Crohn's disease patients. Improving patients' self-epistemic authority can increase certainty and, thus, improve health-related quality of life.
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Affiliation(s)
- Galia Niv
- Rabin Medical Center, Petah Tikva, Israel
| | - Simona Bar Josef
- Gastroenterology Department, Rabin Medical Center, Petah Tikva, Israel
| | - Ofer Ben Bassat
- Gastroenterology Department, Rabin Medical Center, Petah Tikva, Israel
| | - Irit Avni
- Gastroenterology Department, Rabin Medical Center, Petah Tikva, Israel
| | - Lev Lictenstein
- Gastroenterology Department, Rabin Medical Center, Petah Tikva, Israel
| | - Yaron Niv
- Gastroenterology Department, Rabin Medical Center, Petah Tikva, Israel.,Sackler Faculty of Medicine, Tel-Aviv University, Tel Aviv-Yafo, Israel
| | - Sivia Barnoy
- Nursing Department, Sackler Faculty of Medicine, Tel-Aviv University, Tel Aviv-Yafo, Israel.
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Wilburn J, Twiss J, Kemp K, McKenna SP. A qualitative study of the impact of Crohn's disease from a patient's perspective. Frontline Gastroenterol 2017; 8:68-73. [PMID: 28839887 PMCID: PMC5369426 DOI: 10.1136/flgastro-2015-100678] [Citation(s) in RCA: 26] [Impact Index Per Article: 3.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 01/04/2016] [Revised: 04/12/2016] [Accepted: 04/15/2016] [Indexed: 02/04/2023] Open
Abstract
OBJECTIVE To understand how the lives of people with Crohn's disease (CD) are affected. Most research in CD has focused on symptoms and functioning rather than on how these outcomes influence quality of life (QoL). DESIGN As part of a study to develop a CD-specific patient-reported outcome measure, qualitative interviews were conducted with patients from Manchester Royal Infirmary to determine how CD affects QoL. The needs-based model was adopted for the study. The interviews, which took the form of focused conversations covering all aspects of the impact of CD and its treatment, were audio-recorded. Theoretical thematic analysis of the transcripts identified needs affected by CD. RESULTS Thirty patients (60% female) aged 25-68 years were interviewed. Participants had experienced CD for between 2 and 40 years. Nearly 1300 statements relating to the impact of CD were identified. Thirteen main need themes were identified: nutrition, hygiene, continence, freedom from infection, security, self-esteem, role, attractiveness, relationships, intimacy, clear-mindedness, pleasure and autonomy. CONCLUSIONS The findings from the interviews indicate that CD has a major impact on need-fulfilment. Such issues should be addressed in CD audit, clinical trials and when evaluating clinical practice.
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Affiliation(s)
| | | | - Karen Kemp
- Department of Gastroenterology, Manchester Royal Infirmary, Manchester, UK
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Rouse M, Twiss J, McKenna SP. Co-calibrating quality-of-life scores from three pulmonary disorders: implications for comparative-effectiveness research. J Med Econ 2016; 19:596-603. [PMID: 26824603 DOI: 10.3111/13696998.2016.1148700] [Citation(s) in RCA: 5] [Impact Index Per Article: 0.6] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Indexed: 11/09/2022]
Abstract
Background Efficient use of health resources requires accurate outcome assessment. Disease-specific patient-reported outcome (PRO) measures are designed to be highly relevant to patients with a specific disease. They have advantages over generic PROs that lack relevance to patient groups and miss crucial impacts of illness. It is thought that disease-specific measurement cannot be used in comparative effectiveness research (CER). The present study provides further evidence of the value of disease-specific measures in making valid comparisons across diseases. Methods The Asthma Life Impact Scale (ALIS, 22 items), Living with Chronic Obstructive Pulmonary Disease (LCOPD, 22 items) scale, and Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR, 25 items) were completed by 140, 162, and 91 patients, respectively. The three samples were analyzed for fit to the Rasch model, then combined into a scale consisting of 58 unique items and re-analyzed. Raw scores on the three measures were co-calibrated and a transformation table produced. Results The scales fit the Rasch model individually (ALIS Chi(2) probability value (p-Chi(2)) = 0.05; LCOPD p-Chi(2 )=( )0.38; CAMPHOR p-Chi(2 )=( )0.92). The combined data also fit the Rasch model (p-Chi(2 )=( )0.22). There was no differential item functioning related to age, gender, or disease. The co-calibrated scales successfully distinguished between perceived severity groups (p < 0.001). Limitations The samples were drawn from different sources. For scales to be co-calibrated using a common item design, they must be based on the same theoretical construct, be unidimensional, and have overlapping items. Conclusions The results showed that it is possible to co-calibrate scores from disease-specific PRO measures. This will permit more accurate and sensitive outcome measurement to be incorporated into CER. The co-calibration of needs-based disease-specific measures allows the calculation of γ scores that can be used to compare directly the impact of any type of interventions on any diseases included in the co-calibration.
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Affiliation(s)
- M Rouse
- a Galen Research Ltd , Manchester , UK
| | - J Twiss
- a Galen Research Ltd , Manchester , UK
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Beaudart C, Biver E, Reginster JY, Rizzoli R, Rolland Y, Bautmans I, Petermans J, Gillain S, Buckinx F, Van Beveren J, Jacquemain M, Italiano P, Dardenne N, Bruyere O. Development of a self-administrated quality of life questionnaire for sarcopenia in elderly subjects: the SarQoL. Age Ageing 2015; 44:960-6. [PMID: 26433796 PMCID: PMC4621234 DOI: 10.1093/ageing/afv133] [Citation(s) in RCA: 92] [Impact Index Per Article: 9.2] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/16/2014] [Accepted: 07/22/2015] [Indexed: 12/25/2022] Open
Abstract
BACKGROUND The impact of sarcopenia on quality of life is currently assessed by generic tools. However, these tools may not detect subtle effects of this specific condition on quality of life. OBJECTIVE The aim of this study was to develop a sarcopenia-specific quality of life questionnaire (SarQoL, Sarcopenia Quality of Life) designed for community-dwelling elderly subjects aged 65 years and older. SETTINGS Participants were recruited in an outpatient clinic in Liège, Belgium. SUBJECTS Sarcopenic subjects aged 65 years or older. METHODS The study was articulated in the following four stages: (i) Item generation-based on literature review, sarcopenic subjects' opinion, experts' opinion, focus groups; (ii) Item reduction-based on sarcopenic subjects' and experts' preferences; (iii) Questionnaire generation-developed during an expert meeting; (iv) Pretest of the questionnaire-based on sarcopenic subjects' opinion. RESULTS The final version of the questionnaire consists of 55 items translated into 22 questions rated on a 4-point Likert scale. These items are organised into seven domains of dysfunction: Physical and mental health, Locomotion, Body composition, Functionality, Activities of daily living, Leisure activities and Fears. In view of the pretest, the SarQoL is easy to complete, independently, in ∼10 min. CONCLUSIONS The first version of the SarQoL, a specific quality of life questionnaire for sarcopenic subjects, has been developed and has been shown to be comprehensible by the target population. Investigations are now required to test the psychometric properties (internal consistency, test-retest reliability, divergent and convergent validity, discriminant validity, floor and ceiling effects) of this questionnaire.
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Affiliation(s)
- Charlotte Beaudart
- Public Health, Epidemiology and Health Economics, University of Liège, Liège 4000, Belgium
| | - Emmanuel Biver
- Division of Bone Diseases, Faculty of Medicine, Geneva University Hospitals, Geneva, Switzerland
| | - Jean-Yves Reginster
- Public Health, Epidemiology and Health Economics, University of Liège, Liège 4000, Belgium
| | - René Rizzoli
- Division of Bone Diseases, Faculty of Medicine, Geneva University Hospitals, Geneva, Switzerland
| | - Yves Rolland
- Clinical Gerontology, CHU Toulouse, Toulouse, France
| | - Ivan Bautmans
- Frailty in Ageing Research Department, Vrije Universiteit Brussel, Brussels, Belgium
| | | | | | - Fanny Buckinx
- Public Health, Epidemiology and Health Economics, University of Liège, Liège 4000, Belgium
| | - Julien Van Beveren
- Department of French and Romance Languages and Literatures, Faculty of Philosophy and Letters, University of Liège, Liège, Belgium
| | - Marc Jacquemain
- Institute of Human and Social Sciences, University of Liège, Liège, Belgium
| | - Patrick Italiano
- Institute of Human and Social Sciences, University of Liège, Liège, Belgium
| | - Nadia Dardenne
- Public Health, Epidemiology and Health Economics, University of Liège, Liège 4000, Belgium
| | - Olivier Bruyere
- Public Health, Epidemiology and Health Economics, University of Liège, Liège 4000, Belgium
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