scholarly journals Development of a preliminary conceptual model of the patient experience of chronic kidney disease: a targeted literature review and analysis

2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Jennifer E. Flythe ◽  
Niklas Karlsson ◽  
Anna Sundgren ◽  
Paul Cordero ◽  
Amanda Grandinetti ◽  
...  
Keyword(s):  
Chronic Kidney Disease ◽  
Kidney Disease ◽  
Literature Review ◽  
Conceptual Model ◽  
Patient Experience ◽  
Patient Reported Outcome ◽  
Dietary Habit ◽  
Patient Reported ◽  
Emotional Strain ◽  
Pro Instruments

Abstract Background Patient-reported outcome (PRO) instruments should capture the experiences of disease and treatment that patients consider most important in order to inform patient-centred care and product development. The aim of this study was to develop a preliminary conceptual model of patient experience in chronic kidney disease (CKD) based on a targeted literature review and to characterize existing PRO instruments used in CKD. Methods PubMed, EMBASE and Cochrane databases and recent society meetings were searched for publications reporting signs/symptoms and life impacts of CKD. Concepts identified in the literature review were used to develop a preliminary conceptual model of patient experience of CKD, overall, and within patient subpopulations of differing CKD causes, severities and complications. PRO instruments, identified from PRO databases, CKD literature and CKD clinical trials, were assessed for content validity, psychometric strength and coverage of concepts in the literature review. Results In total, 100 publications met criteria for analysis; 56 signs/symptoms and 37 life impacts of CKD were identified from these sources. The most frequently mentioned signs/symptoms were pain/discomfort (57% of publications) and tiredness/low energy/lethargy/fatigue (42%); the most commonly reported life impacts were anxiety/depression (49%) and decrements in physical functioning (43%). Signs/symptoms and life impacts varied across the subpopulations and were more frequent at advanced CKD stages. The preliminary conceptual model grouped signs/symptoms into seven domains (pain/discomfort; energy/fatigue; sleep-related; gastrointestinal-related; urinary-related; skin−/hair−/nails-related; and other) and life impacts into six domains (psychological/emotional strain; cognitive impairment; dietary habit disruption; physical function decrements; interference with social relationships; and other). Eleven PRO instruments were considered to be promising for use in CKD; all had limitations. Conclusions Although preliminary, the proposed conceptual model highlights key PROs for people with CKD and is intended to spur development of more tailored PRO instruments to assess these concepts.

scholarly journals Understanding the patient experience in hepatocellular carcinoma: a qualitative patient interview study

2021 ◽  
Author(s):  
Nikunj Patel ◽  
Joshua Maher ◽  
Xandra Lie ◽  
Chad Gwaltney ◽  
Afsaneh Barzi ◽  
...  
Keyword(s):  
Hepatocellular Carcinoma ◽  
Drug Development ◽  
Conceptual Model ◽  
Patient Experience ◽  
Outcome Measurement ◽  
Qualitative Interviews ◽  
Patient Reported Outcome ◽  
Weight Changes ◽  
Patient Reported ◽  
Pro Instruments

Abstract Purpose This study aimed to elucidate the patient experience of hepatocellular carcinoma (HCC) to guide patient-centered outcome measurement in drug development. Methods Patients with HCC participated in qualitative interviews to elicit disease-related signs/symptoms and impacts, using discussion guides developed from literature searches and discussions with oncologists. Interview participants rated the disturbance of their experiences (0–10 scale). A conceptual model was developed and mapped against patient-reported outcome (PRO) instruments identified from database reviews. Results Interviews were conducted with 25 individuals with HCC (68% were men; median age: 63 years; 12% Barcelona clinic liver cancer (BCLC) stage A; 32% stage B; and 56% stage C) in the USA. Fifty-one HCC-related concepts were identified from the interviews and were grouped into eight sign/symptom categories (eating behavior/weight changes; extremities [arms, legs]; fatigue and strength; gastrointestinal; pain; sensory; skin; other) and four impact categories (emotional; physical; cognitive function; other) for the conceptual model. The most prevalent and disturbing experiences across the disease stages were fatigue/lack of energy and emotional impacts such as frustration, fear, and depression. Abdominal pain and skin-related issues were particularly common and disturbing in individuals with HCC stage C. The EORTC QLQ-C30 and HCC18 were identified as commonly used PRO instruments in HCC studies and captured the relevant signs/symptoms associated with the patient experience. Conclusion Patients with HCC reported a range of signs/symptoms and impacts that negatively affect daily functioning and quality of life. Including PRO measures in HCC clinical trials can provide meaningful patient perspectives during drug development.

scholarly journals Development of a conceptual model and patient-reported outcome measures for assessing symptoms and functioning in patients with heart failure

2020 ◽  
Vol 29 (10) ◽  
pp. 2835-2848
Author(s):  
Olga Moshkovich ◽  
Katy Benjamin ◽  
Katie Hall ◽  
Ryan Murphy ◽  
Robyn von Maltzahn ◽  
...  
Keyword(s):  
Heart Failure ◽  
Literature Review ◽  
Conceptual Model ◽  
Phase 1 ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Cognitive Testing ◽  
Expert Interviews ◽  
Cognitive Changes ◽  
Patient Reported

Abstract Purpose Heart failure (HF) is a common condition that places considerable burden on patients. We aimed to develop a patient-reported outcome (PRO) measure to assess the symptoms and impacts of HF. Methods Phase 1: a targeted literature review, expert interviews, and concept elicitation (CE) interviews with patients with HF (n = 26) were used to develop a conceptual model of the core symptoms and impacts of HF. To capture these concepts, three new fit-for-purpose PRO questionnaires were constructed in accordance with US Food and Drug Administration PRO guidance. Phase 2: three ‘waves’ of cognitive interviews were conducted with patients with HF (n = 28) to validate and refine the questionnaires. Results Three key symptoms—shortness of breath, oedema, and fatigue—were identified across the literature review, expert interviews and CE interviews. Several additional symptoms, cognitive changes and impacts of HF were reported in the CE interviews and included in the conceptual model. A 10-item symptom questionnaire (Heart Failure-Daily Symptom Diary) was constructed; cognitive testing showed that the final PRO measure was easy to understand/complete and relevant to patients with HF, confirming content validity. Two HF impact questionnaires were developed (Assessing Dyspnoea’s Impact on Mobility and Sleep and Heart Failure-Functional Status Assessment), but required refinement to ensure patient understanding. Conclusions Patient input contributed to the development of a PRO instrument for assessing physical and cognitive symptoms important to patients with HF using novel measurement strategies. Inclusion of daily metrics offers differentiation from other qualified instruments and may provide clinical insight for improving lifestyles. Additionally, two draft PRO measures may, after further validation, be useful to assess the impacts of HF.

scholarly journals Economic Modelling of Chronic Kidney Disease: A Systematic Literature Review to Inform Conceptual Model Design

2019 ◽  
Vol 37 (12) ◽  
pp. 1451-1468 ◽  
Author(s):  
Daniel M. Sugrue ◽  
Thomas Ward ◽  
Sukhvir Rai ◽  
Phil McEwan ◽  
Heleen G. M. van Haalen
Keyword(s):  
Chronic Kidney Disease ◽  
Kidney Disease ◽  
Literature Review ◽  
Conceptual Model ◽  
Systematic Literature Review ◽  
Economic Modelling ◽  
Model Design

scholarly journals Patient‐reported outcome measures and their utility in the management of patients with advanced chronic kidney disease

Nephrology ◽  
2019 ◽  
Vol 24 (8) ◽  
pp. 814-818 ◽  
Author(s):  
Kathryn Ducharlet ◽  
Vijaya Sundararajan ◽  
Jennifer Philip ◽  
Jennifer Weil ◽  
Nuala Barker ◽  
...  
Keyword(s):  
Chronic Kidney Disease ◽  
Kidney Disease ◽  
Outcome Measures ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Advanced Chronic Kidney Disease ◽  
Patient Reported

scholarly journals P116 Development of a new patient-reported outcome measure for patients with complex cryptoglandular fistulas

2021 ◽  
Vol 15 (Supplement_1) ◽  
pp. S208-S209
Author(s):  
J McCurdy ◽  
P Crooks ◽  
C Gwaltney ◽  
R Krupnick ◽  
K A Cadogan ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Literature Review ◽  
Conceptual Model ◽  
Rating Scale ◽  
Daily Life ◽  
Patient Reported Outcome ◽  
Cognitive Debriefing ◽  
Item Pool ◽  
Patient Reported

Abstract Background Complex cryptoglandular fistulas (CCF) arise from infected anal glands and are often associated with substantial morbidity and healthcare utilization. Standarized patient-reported outcome measures (PROMs) for CCF are lacking. Thus, we aimed to develop a disease-specific PROM to assess the burden of symptoms and their impact on daily life in patients with CCF. Methods To develop a conceptual model, a targeted literature review was conducted to identify symptoms and impacts on daily life associated with CCF. Existing PROMs relating to anal fistulas were also sought. Semi-structured interviews with colorectal surgeons (n=5) assessed clinical perspectives on patient experience of CCF. A draft item pool was developed based on the refined conceptual model and using the Quality of Life in Patients with Anal Fistula Questionnaire as a reference. The tool was refined through concept elicitation interviews on the symptoms and impacts of CCF, and cognitive debriefing on the interpretation, understanding and response to each item. Interviews were conducted until concept saturation was achieved and patient feedback suggested no further refinements were required. Results The literature review identified discharge, pain, faecal incontinence and bleeding as the most prevalent symptoms, and embarrassment as the most prevalent impact on daily life. Results from surgeon interviews were then used to revise the initial conceptual model. Twenty US adults (60% female; mean age, 49 years) with clinically confirmed CCF participated in interviews (four waves of n=5). Patients identified 10 salient symptoms and 11 salient impacts on daily life (salient defined as mention by ≥50% of patients and mean disturbance rating ≥5 on a 10-point scale). The final conceptual model included the main symptoms and treatment-related effects, and impacts on daily life. The draft item pool was refined, resulting in a final PROM consisting of 14 items (covering frequency and severity) relating to symptom domains – discharge, incontinence, pain, irritation, odour, abscess – and 6 items relating to health-related quality of life domains – functional, physical, psychological, social health. The PROM features a 7-day recall period, with responses mostly given on a 5-point verbal rating scale. Cognitive debriefing confirmed that the PROM was clear, easy to understand and relevant to patients’ experiences. Conclusion The 20-item CCF questionnaire (CCFQ-20) is a new PROM that has been developed and tested for content validity, following expert guidance and regulatory best practices. It addresses a comprehensive set of salient symptoms and impacts experienced by patients with CCF. Psychometric testing is required to fully evaluate this PROM. Sponsor: Takeda Pharmaceuticals USA, Inc.

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