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

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 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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Affordance-Based Assessment Cuts Across The Dichotomy Of Subjective-Objective Outcome Measure At The Knee Joint

10.47363/jmhc/2021(3)144 ◽

2019 ◽

pp. 1-4

Author(s):

Wangdo Kim ◽

Keyword(s):

Outcome Measurement ◽

Patient Reported Outcome ◽

Assessment Process ◽

Active Sets ◽

Patient Reported ◽

Objective Outcomes ◽

Objective Outcome ◽

Subjective Outcomes ◽

Pro Instruments ◽

Inseparable Pair

There is increasing awareness of the need to include patient-reported outcome (PRO) instruments in evaluating the measurement of clinical outcomes, with an increasing focus placed on the patients’ perspective. Scientists have tried to link PROs with objective outcomes, providing unique information for managing patient care. Traditionally, objective and patient-reported outcomes (such as the Knee Injury and Osteoarthritis Outcome Score (KOOS)) are considered two distinct constructs, which cannot serve as a direct proxy for each other. Can Gibson´s affordance theory specify more about objective outcome measurements vs. subjective outcomes measurement and unify them? The present article develops a theoretical framework called entrainment of touch and posture that advocates the vis viva (living force) as the proper gauge for the dynamical action of a force, and that could explain “possibilities for action or affordance” during outcome measurement. We found that active touch and posture refer to what is ordinarily called touchingvariations in skin stimulation caused by surfaces are altered together by motor activity variations. This affordance of “walk-on-able” is worth noting because it is often neglected that locomotion and its surfaces form an inseparable pair. The assessment process can be viewed in terms of action possibilities provided by the active sets of organs residing that can obtain and utilize information about the tissue environments in which the grafts are to be located.

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

Orphanet Journal of Rare Diseases ◽

10.1186/s13023-021-02067-x ◽

2021 ◽

Vol 16 (1) ◽

Author(s):

Alaa Hamed ◽

Kristina An Haack ◽

Chad Gwaltney ◽

Eileen Baranowski ◽

Andrew Stewart ◽

...

Keyword(s):

Literature Review ◽

Conceptual Model ◽

Pompe Disease ◽

Patient Perspective ◽

Late Onset ◽

Qualitative Interviews ◽

Patient Reported Outcome ◽

Patient Interviews ◽

Patient Interview ◽

Patient Reported

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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Fundamental Outcome Measurement: Selecting Patient Reported Outcome Instruments and Interpreting the Data they Produce

INNOVATIONS in pharmacy ◽

10.24926/iip.v12i2.3911 ◽

2021 ◽

Vol 12 (2) ◽

pp. 17

Author(s):

Stephen P McKenna ◽

Alice Heaney ◽

Paul C Langley

Keyword(s):

Outcome Measurement ◽

Therapy Response ◽

Simulation Models ◽

Patient Reported Outcome ◽

Specific Patient ◽

Patient Reported ◽

The Many ◽

Reported Data ◽

The Impact ◽

Pro Instruments

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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