Qualitative interviews to improve patient-reported outcome measures in late-onset Pompe disease: the patient perspective

Abstract Background Late-onset Pompe Disease (LOPD) is a rare, heterogeneous disease manifested by a range of symptoms varying in severity. Research establishing the frequency of these symptoms and their impact on patients’ daily lives is limited. The objective of this study was to develop a conceptual model that captures the most relevant symptoms and functional limitations experienced by patients with LOPD, to inform the development of new patient-reported outcome (PRO) tools. Methods A preliminary conceptual model was constructed following a literature review and revised through interviews with expert clinicians to identify important and relevant concepts regarding symptoms and impacts of LOPD. This preliminary model informed the development of a qualitative patient interview guide, which was used to gather the patient perspective on symptoms and impacts relating to LOPD or its treatment (including symptom/impact frequency and levels of disturbance). Patient interviews aided further refinement of the conceptual model. The findings from the patient interviews were triangulated with the literature review and clinician interviews to identify the most relevant and significant effects of LOPD from the patient perspective. Results Muscle weakness, fatigue, pain, and breathing difficulties (especially while lying down) were the most common and highly disturbing symptoms experienced by patients. Limitations associated with mobility (e.g., difficulty rising from a sitting position, getting up after bending) and activities of daily living, (e.g., reduced ability to participate in social/family activities or work/study) were the most frequently reported impacts with the highest levels of disturbance on the patient’s daily life. These identified symptoms and impacts were included in the new conceptual model of disease. Conclusions This qualitative patient interview study, also informed by a literature review and clinician interviews, identified the most frequent and relevant symptoms and the functional impact of LOPD on patients. The study interviews also captured the patient-preferred language to describe symptoms and impacts of LOPD. The results from this study can be used to develop future PRO instruments that are tailored to the specific symptoms and impacts experienced by patients with LOPD.

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

Quality of Life Research ◽

10.1007/s11136-021-02903-4 ◽

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.

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

Quality of Life Research ◽

10.1007/s11136-020-02537-y ◽

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.

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

Journal of Crohn s and Colitis ◽

10.1093/ecco-jcc/jjab076.243 ◽

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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Development of a patient-reported outcome measure (PROM) and change measure for use in early recovery following hip or knee replacement

Journal of Patient-Reported Outcomes ◽

10.1186/s41687-020-00262-1 ◽

2020 ◽

Vol 4 (1) ◽

Author(s):

Louise H. Strickland ◽

David W. Murray ◽

Hemant G. Pandit ◽

Crispin Jenkinson

Keyword(s):

Knee Replacement ◽

Healthcare Professionals ◽

Outcome Measure ◽

Qualitative Interviews ◽

Patient Reported Outcome ◽

Early Recovery ◽

Patient Reported Outcome Measure ◽

Patient Interviews ◽

Patient Reported ◽

Change Measure

Abstract Background Hip and knee replacement are effective procedures for end-stage arthritis that has not responded to medical management. However, until now, there have been no validated, patient-reported tools to measure early recovery in this growing patient population. The process of development and psychometric evaluation of the Oxford Arthroplasty Early Recovery Score (OARS), a 14-item patient-reported outcome measure (PROM) measuring health status, and the Oxford Arthroplasty Early Change Score (OACS) a 14-item measure to assess change during the first 6 weeks following surgery is reported. Patients and methods A five-phased, best practice, iterative approach was used. From a literature based starting point, qualitative interviews with orthopaedic healthcare professionals, were then performed ascertaining if and how clinicians would use such a PROM and change measure. Analysis of in-depth patient-interviews in phase one identified important patient-reported factors in early recovery which were used to provide questionnaire themes. In Phase two, candidate items from Phase One interviews were generated and pilot questionnaires developed and tested. Exploratory factor analysis with item reduction and final testing of the questionnaires was performed in phase three. Phase Four involved validation testing. Results Qualitative interviews (n = 22) with orthopaedic healthcare professionals, helped determine views of potential users, and guide structure. In Phase One, factors from patient interviews (n = 30) were used to find questionnaire themes and generate items. Pilot questionnaires were developed and tested in Phase Two. Items were refined in the context of cognitive debrief interviews (n = 34) for potential inclusion in the final tools. Final testing of questionnaire properties with item reduction (n = 168) was carried out in phase three. Validation of the OARS and OACS was performed in phase four. Both measures were administered to consecutive patients (n = 155) in an independent cohort. Validity and reliability were assessed. Psychometric testing showed positive results, in terms of internal consistency and sensitivity to change, content validity and relevance to patients and clinicians. In addition, these measures have been found to be acceptable to patients throughout early recovery with validation across the 6 week period. Conclusions These brief, easy-to-use tools could be of great use in assessing recovery pathways and interventions in arthroplasty surgery.

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A novel patient-reported outcome instrument assessing the symptoms of paroxysmal nocturnal hemoglobinuria, the PNH-SQ

Journal of Patient-Reported Outcomes ◽

10.1186/s41687-021-00376-0 ◽

2021 ◽

Vol 5 (1) ◽

Author(s):

R. Paola Daly ◽

Jessica J. Jalbert ◽

Shannon Keith ◽

Tara Symonds ◽

Jamile Shammo

Keyword(s):

Abdominal Pain ◽

Conceptual Model ◽

Paroxysmal Nocturnal Hemoglobinuria ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Response Options ◽

Patient Interview ◽

Outcome Instrument ◽

Patient Reported ◽

Bothersome Symptom

Abstract Background Patient-reported outcome measures (PROs) used to measure symptoms of patients with paroxysmal nocturnal hemoglobinuria (PNH) in trials do not measure PNH symptoms comprehensively and do not assess daily fluctuations in symptoms. Following a literature review and consultation with a PNH expert, we drafted the PNH Symptom Questionnaire (PNH-SQ) and a patient-centric conceptual model of PNH symptoms and impacts. We then interviewed 15 patients with PNH to assess comprehensiveness of symptom capture from the patient perspective and to cognitively debrief the PNH-SQ. Patient interview data were also used to finalize the PNH conceptual model. Results Participants mentioned 27 signs or symptoms of PNH spontaneously or after being probed; 93% reported experiencing ≥ 1 PNH symptom. Concept saturation was reached for all PNH symptoms. Further, interviews confirmed the instrument captured the most common PNH symptoms, including fatigue (87%), abdominal pain (60%), and difficulty swallowing (47%), with fatigue ranked as the most bothersome symptom. The interviews demonstrated that participants understood the items of the PNH-SQ (90–100%); considered the symptoms relevant (> 50– > 90%); the recall period appropriate (> 80–100%); and the response options suitable (> 80–100%). Participants also suggested changes regarding item redundancy and relevance; this feedback was used to finalize the instrument. Conclusions The finalized PNH-SQ assesses the presence and severity of 10 symptoms—abdominal pain, chest discomfort, difficulty sleeping, difficulty swallowing, difficulty thinking clearly, fatigue, headache, muscle weakness, pain in the legs or back, and shortness of breath—over 24 h. The PNH-SQ is a content-valid questionnaire suitable for assessing daily symptom presence and severity in PNH clinical trials.

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The BODY-Q Cellulite Scale: A Development and Validation Study

Aesthetic Surgery Journal ◽

10.1093/asj/sjaa100 ◽

2020 ◽

Author(s):

Anne F Klassen ◽

Manraj N Kaur ◽

Claire E E de Vries ◽

Lotte Poulsen ◽

Trisia Breitkopf ◽

...

Keyword(s):

Field Test ◽

Subcutaneous Tissue ◽

Measurement Theory ◽

Qualitative Interviews ◽

Test Sample ◽

Patient Reported Outcome ◽

The Body ◽

Patient Interviews ◽

Patient Reported ◽

The Impact

Abstract Background Cellulite is a localized metabolic disorder of the subcutaneous tissue. To measure the impact of cellulite and its treatment(s) on patients’ health-related quality of life, a psychometrically sound patient-reported outcome measure is needed. Objectives The authors sought to develop and field test a new BODY-Q cellulite scale to measure the appearance of cellulite. Methods Appearance-related codes from the original BODY-Q qualitative interviews were reexamined, and a set of cellulite-specific items was developed and refined through cognitive patient interviews (n = 10) and expert input (n = 17). This scale was field-tested in adults with cellulite through 2 crowdworking platforms. Rasch Measurement Theory analysis was employed to refine the scale and examine its psychometric properties. Results The field-test sample included 2129 participants. The 15-item scale was reduced in length to 11 items. Data from the sample fit the Rasch model (X2 [99] = 21.32, P = 0.06). All items had ordered thresholds and mapped out a targeted clinical hierarchy. The reliability statistics for the person separation index was 0.94 and for Cronbach’s alpha was 0.97. In terms of validity, worse scores on the cellulite scale were associated with being more bothered by how the cellulite looked overall, having more severe cellulite on the Patient-Reported Photo-numeric Cellulite Severity Scale, and having more self-reported cellulite and more areas of the body with cellulite. Conclusions The BODY-Q cellulite scale can be utilized to measure appearance of cellulite and provides a solid basis for future studies evaluating the impact of cellulite and its treatment.

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Identifying Salient Quality of Life Domains in Patients Living with High-Risk MDS and AML

Blood ◽

10.1182/blood-2021-150821 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 4982-4982

Author(s):

Anthony J. Messina ◽

Sabrina Figueiredo ◽

John H Powers ◽

Mary A Corcoran

Keyword(s):

Quality Of Life ◽

Conceptual Model ◽

Treatment Satisfaction ◽

Research Study ◽

Patient Reported Outcome ◽

Individual Factors ◽

Life Domains ◽

Patient Interviews ◽

Patient Reported

Abstract Background Valuing patients as the experts in their experience, how they live with myelodysplastic disease, and how to reframe assessments of efficacy, patient-reported outcomes (PROs) can complement traditional measurements of response. Therefore, when used correctly, PROs serve as a valued instrument to assess a patient's Quality of Survival (QoS) throughout the course of their treatment. This study aimed to address the need for improved quality of life measurement in MDS and AML research requires identifying instruments that are appropriate for this population and ones that accurately represent the goals, needs, and concerns of these patients. Therefore research efforts should be directed to providing confirmatory data of the suitability for utilizing existing patient-reported outcome measurements (PROMs) for this population or identify the need to develop and use highly sensitive disease-specific measurements when evaluating new treatments. Identification of representative, influential, patient-reported quality of life domains first needed to be established in order to direct discovery of appropriate PROMs for this population in this two part research study (part-one: patient interviews/surveys, part two: Delphi panel/identification of appropriate PROMs [forthcoming]). Methods Between January and June 2021, 17 participants (AML, n = 8; HR-MDS, n = 9) were enrolled in an IRB-research study participating in concept-elicitation, semistructured interviews and surveys aimed to identify the lived experience of those being treated for HR-MDS and AML. An interpretive descriptive methodology was chosen to produce coherent conceptual descriptions aimed to expose thematic patterns and commonalities that characterize individuals with HR-MDS and AML. Using these insights, a conceptual model was derived to establish the basis for PROM content validity evaluation and to improve the accuracy of the captured quality of life measurements of this patient population. Results Seven (7) patient-reported themes emerged from qualitative analysis; social wellbeing, physical wellbeing, emotional wellbeing, management of side effects, treatment satisfaction, autonomy, and individual factors. Treatment Satisfaction, Autonomy, and Individual Factors (such as education, financial wellbeing, incoming treatment goal, health status, outlook, and prognosis) were found to be the most salient related to quality of life and overall wellbeing. The conceptual model highlights the main areas of impact and the relationships among concepts. Significant overlap was observed between the influential domains identified amongst those with HR-MDS and AML. Surveys, which were administered after patient interviews, were analyzed for consistency, reliability (α=0.8405), and variable correlation. Conclusion A clear understanding of the influential health-related quality of life domains that make up the foundation of this patient population's experience has proven fundamental in the process of identification of appropriate PROMs to measure quality of life. The patient-reported influence treatment satisfaction (confidence in treatment/provider, quality of care, compassion received, provided support, knowledge of treatment, burden of care, information of prognosis, etc) has on a patient's quality of life may lead to a more holistic approach to patient-reported outcome item-banks and instrument selection. The importance of the maintenance of autonomy during and after treatment was apparent in the reported anxiety associated with the loss of the ability of self-care, and independence. Additionally, the absence of divergent themes amongst those with HR-MDS and AML further adds to the evidence that these sub-populations share similar patient experiences. Disclosures Powers: Arrevus, Celularity, Corbus, DaVolterra, Eicos, Eli Lilly, Evofem, Eyecheck, Fuji, Gilead, GSK group of companies, Johnson & Johnson, Microbion, Mustang, OPKO, Otsuka, Romark, Shiniogi, Vir: Consultancy.

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Conducting Patient Interviews Within a Clinical Trial Setting

10.3768/rtipress.2018.op.0054.1808 ◽

2018 ◽

Cited By ~ 1

Author(s):

Dana B. DiBenedetti ◽

T. Michelle Brown ◽

Carla Romano ◽

Claire Ervin ◽

Sandy Lewis ◽

...

Keyword(s):

Clinical Trial ◽

Qualitative Interviews ◽

Patient Reported Outcome ◽

Treatment Needs ◽

Clinical Trial Setting ◽

Patient Interviews ◽

Treatment Gaps ◽

Patient Reported ◽

Trial Participants ◽

Trial Setting

Qualitative data centered on patients’ experiences and perspectives typically go uncollected in clinical trial settings. Yet patients’ treatment experiences offer complementary insights and context on topics such as disease management, treatment gaps, and previous treatments outside of those gathered in traditional patient-reported outcome questionnaires. Qualitative interviews can capture patients’ perceptions of treatment needs, more fully explore meaningful changes experienced as a result of treatment, and reveal outcomes that are most important to patients. Asking patients detailed questions can provide insight into the “why” of a patient’s expressed thought or feeling. The inclusion of patient interviews within clinical trials is a relatively new and evolving field of research. This article delineates the types of data that may be collected during interviews with clinical trial participants and outlines two approaches to conducting qualitative research in the clinical trial setting, with a focus on maximizing the value of the resulting data.

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Development of a Cold Agglutinin Disease-Specific Patient-Reported Outcome Symptom Measure

Blood ◽

10.1182/blood-2021-147199 ◽

2021 ◽

Vol 138 (Supplement 1) ◽

pp. 4057-4057

Author(s):

Florence Joly ◽

Pronabesh Dasmahapatra ◽

Jun Su ◽

Dana DiBenedetti ◽

Katherine Kosa ◽

...

Keyword(s):

Conceptual Model ◽

Patient Perspective ◽

Patient Reported Outcome ◽

Cold Agglutinin ◽

Cognitive Debriefing ◽

Shortness Of Breath ◽

Item Pool ◽

Cold Agglutinin Disease ◽

Publicly Traded ◽

Patient Reported

Abstract Introduction Cold agglutinin disease (CAD) is a rare autoimmune hemolytic anemia in which red blood cells are bound by cold agglutinin autoantibodies. In addition to cold-induced symptoms, patients with CAD experience symptoms of chronic anemia and hemolysis including fatigue, weakness, and shortness of breath (Swiecicki PL et al. Blood 2013;122(7):1114-21). The objectives of this study were to identify disease-related symptoms and impacts most important to patients with CAD, and to evaluate whether existing patient-reported outcome (PRO) measures are appropriate to assess relevant CAD concepts. Methods As part of a larger study, qualitative concept elicitation (CE) interviews were conducted to better understand the disease burden of CAD from the patient perspective. The results from the first set of interviews were reported by Su J et al. Blood 2020;136(1):29-30. Based on the identified symptoms and impacts, standard survey methodological principles were used to develop a draft item pool to address concepts of interest. Items were refined and evaluated during the second set of interviews, which included 3 rounds of 60-minute phone interviews with patients with CAD using a semi-structured interview guide that included a CE section and cognitive debriefing. Pooled results from both sets of interviews were used to develop a conceptual model of the symptoms and impacts of CAD. Physician advisors were also invited to review the concepts identified in the interviews and to select those that they believed were most relevant as part of an advisory board. A PubMed review of literature published between 2010-2020 was also conducted to identify existing PRO measures used in the assessment of symptoms, impacts, and/or quality of life and patient experiences in adults with CAD or similar disease areas. Identified PRO measures were assessed to determine the extent to which they captured CAD-specific concepts identified in the CE interviews. Results In total, 37 participants diagnosed with primary or secondary CAD took part in the interviews (Set 1, n=16; Set 2, n=21). Overall, the mean age of participants was 67.2 years (range: 35-87), and the majority were female (73%). The most frequently reported symptom of CAD was reactions to cold environments (n=36; 97%), e.g., cold or numb feet/hands, and skin discoloration. Other reported symptoms included fatigue (n=35; 95%), shortness of breath (n=28; 76%), and trouble with thinking/concentration (n=21; 57%). The most frequently reported unfavorable impact of CAD was on day-to-day activities (n=32; 87%). Other negative impacts included effects on enjoyable activities (n=29; 78%) e.g., gardening, physical health/activities (n=28; 76%), social/leisure life (n=26; 70%); and mood/emotions (n=24; 65%). Most participants (n=30; 81%) reported they had made lifestyle/behavioral changes to help limit their CAD symptoms, such as wearing extra clothing in places that were likely to be cold. A conceptual model of the symptoms and impacts of CAD was developed based on these data (Figure 1). Physician advisors (n=7) agreed that the patient-reported symptoms and impacts identified during the interviews were relevant concepts to CAD. Following a literature review, no existing PRO measure was found to adequately address concepts deemed critical to CAD from the patient perspective. Owing to the lack of an existing fit-for-purpose measure, 14 concepts were identified from the interviews to develop an initial draft item pool. During the cognitive debriefing in the second set of interviews, participants (n=21: Round 1 n=8; Round 2 n=7; Round 3 n=6) evaluated these concepts. After 3 rounds of interviews, the new item set yielded 11 items relating to: fatigue; cold sensitivity; dyspnea; wearing extra clothing; limited physical, social, and enjoyable activities; difficulty with usual activities; mood; frustration; and anxiety/stress. Participants reported these items were comprehensive of their experiences with CAD, easy to understand, and would be relevant to gaining a better understanding of individuals' experiences with CAD. Conclusion These results support the need for a novel PRO measure(s) that adequately addresses concepts critical to the measurement of CAD symptoms and impacts from the patient perspective. Figure 1 Figure 1. Disclosures Joly: Sanofi: Current Employment. Dasmahapatra: Sanofi: Current Employment, Current equity holder in publicly-traded company. Su: Sanofi: Current equity holder in publicly-traded company, Ended employment in the past 24 months; Astellas US LLC: Current Employment. DiBenedetti: RTI Health Solutions: Current Employment, Other: Employee of RTI Health Solutions, funded by Sanofi Genzyme to conduct the original work which this abstract is based on. Kosa: RTI Health Solutions: Current Employment. Hill: Novartis: Consultancy, Honoraria; Grifols: Consultancy, Honoraria; Sanofi: Consultancy; ReAlta: Consultancy; Alexion: Honoraria; Amgen: Honoraria; Argenx: Consultancy; Apellis: Consultancy, Honoraria.

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

BMC Nephrology ◽

10.1186/s12882-021-02440-9 ◽

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.

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