BACKGROUND: The NFLymSI-18 is a patient-reported outcome measure comprised of the highest priority symptoms, emotional concerns, treatment side effects, and other concerns identified by lymphoma patients and oncologists. This study assessed the content validity of the NFLymSI-18 for patients with indolent B-cell non-Hodgkin\'s lymphoma (iNHL), with a focus on the Disease-Related Symptoms Physical (DRS-P) subscale.
METHODS: Patients with a confirmed iNHL diagnosis who had received one or more lines of treatment were recruited during clinic visits. Patients described their symptoms, treatment side effects, and emotional concerns related to iNHL in a semi-structured interview. Qualitative data were analyzed using NVivo10.
RESULTS: Data saturation was obtained by the 18th interview. Most participants (67%) had follicular lymphoma. 28% of participants had marginal zone lymphoma, and one participant had lymphoplasmacytoid lymphoma/Waldenström macroglobulinemia. Mean age of the 18 participants was 67 years. 56% of the sample was male. Most participants (67%) had a college or advanced degree. When asked to describe their iNHL symptoms, patients most often discussed swelling (n = 14), fatigue (n = 11), and pain (n = 8). The following symptoms were mentioned by three patients each: anxiety, appetite loss, rash, sleep disruption, trouble breathing, and malaise. Mapping of NFLymSI-18 content to these concerns showed the instrument includes all those most frequently mentioned symptoms.
CONCLUSIONS: This study supports the content validity of the NFLymSI-18, including its DRS-P Subscale, for patients with iNHL. The instrument shows strong validity for the most referenced symptoms of swelling, fatigue, and pain. The diversity of additional symptoms reported by patients is consistent with the heterogeneous symptomology of iNHL.

BACKGROUND: Limitations in physical functioning are common in patients with late-stage chronic kidney disease (CKD) and can greatly affect their lives. Using patient interviews, this study reports experiences associated with physical functioning limitations for patients with late-stage CKD.
METHODS: A preliminary conceptual model on concepts relevant to physical functioning limitations in patients with CKD was developed using data from a targeted literature review (patients with CKD stages IV-V) and previous interviews (patients with CKD stages IIIa-IIIb). The preliminary conceptual model informed a semi-structured interview guide designed to capture experiences of physical functioning limitations in patients with CKD. Patients with CKD stages IV-V who were not receiving dialysis were interviewed; their responses were used to develop a comprehensive conceptual model summarizing their experiences associated with physical functioning limitations.
RESULTS: A total of 25 patients with CKD stage IV (n = 19) or V (n = 6) were interviewed. Based on patient responses, the reported concepts were grouped into one of six categories: physical functioning limitations/difficulties, behavioural impacts, activity participation restrictions, symptoms attributed to physical functioning limitations, impacts on sleep and emotional functioning impacts related to physical functioning limitations. Twenty-three patients reported concepts associated with physical functioning limitations, most frequently \'walking up and down stairs\' (83%) and \'walking distances\' (74%). All 23 patients also reported behavioural impacts, including \'need to rest/subsequent periods of rest\' (100%) and \'participation in fewer activities\' (91%). As well as summarizing the reported concepts, the comprehensive conceptual model shows how concepts may relate to one another; for example, challenging symptoms or difficulty completing tasks can lead to changes in patient behaviour such as purposely reducing or avoiding activities.
CONCLUSIONS: This study found that patients with late-stage CKD not receiving dialysis who experience physical functioning limitations report a range of impacts on their daily lives. The comprehensive conceptual model summarizes the concepts reported and the relationships between them, providing a holistic understanding of how patients with late-stage CKD are affected by physical functioning limitations. Infographic available for this article. INFOGRAPHIC.

BACKGROUND: Wilson disease (WD) is a rare metabolic disorder of impaired copper transport manifesting in hepatic, neurological, and psychiatric symptoms. To evaluate the clinical symptoms of WD in clinical trials, a group of clinicians created the Unified Wilson Disease Rating Scale (UWDRS). Content validity of this scale has not been established. The aim of this study was to evaluate the content validity of the UWDRS Part II from the patient perspective.
METHODS: This study utilized multiple qualitative research methods including concept elicitation interviews, concept/instrument mapping, and cognitive debriefing interviews.
RESULTS: Concept elicitation interviews with a sample of patients with WD and one or more neurological signs/symptoms identified several signs, symptoms, and impacts related to neurological dysfunction, strengthening our understanding of the importance of the neurological aspects of the WD patient experience. Mapping neurological concepts to Part II and III items of the UWDRS showed complete coverage of all salient neurological concepts and near complete coverage of all neurological concepts reported by patients in concept elicitation interviews. Item debriefing of Part II of the UWDRS revealed that patients generally found the items clear and personally relevant to their experience with WD.
CONCLUSIONS: Overall, the findings from this study provide evidence for the content validity of the UWDRS Part II and supportive evidence for the content validity of Part III. The UWDRS should be used in conjunction with additional clinical outcomes assessments, specifically those evaluating the hepatic and psychiatric signs/symptoms of WD, to provide a comprehensive evaluation of the WD patient experience.

Herpes zoster (HZ) is a prevalent disease characterized by a painful rash. A multi‑country study was conducted to elicit public and physician knowledge, attitude, and practice (KAP) toward HZ disease and vaccination for the assessment of local factors influencing HZ vaccine perceptions in four Asian-Pacific countries/territories One-to-one qualitative interviews were conducted in 2022, among the public (people aged ≥ 50 years, adults with parents aged ≥ 50 years, zoster vaccine live-vaccinated individuals aged ≥ 50 years in Republic of Korea, and HZ patients; n = 78) and physicians (general practitioners and specialists; n = 24). Themes surrounding KAP toward HZ and HZ vaccination were summarized using a thematic analysis. A substantial knowledge gap related to HZ was observed among the public, including its causes, long-term impacts, and the at-risk population. There was a low perceived risk of HZ and low general awareness of HZ vaccine availability, although country/territory-specific differences existed. Fear of HZ-associated pain contributed toward vaccination intent among HZ patients and adults with parents aged ≥ 50 years. HZ-naïve adults who were encouraged to receive the vaccine by others were not motivated to do so due to optimism bias. Physicians were perceived to be a reliable source of information. However, physicians did not always proactively discuss HZ vaccination due to time constraints and a perceived need to prioritize other vaccinations including influenza and pneumococcal vaccines. Initiatives are needed to improve public awareness of HZ and its complications, in terms of overall impact on individuals and society, and highlight the important role of physicians in recommending vaccination.

BACKGROUND: Chronic ocular surface pain (COSP) is described as a persistent, moderate-to-severe pain at the ocular surface lasting more than 3 months. Symptoms of COSP have a significant impact on patients\' vision-dependent activities of daily living (ADL) and distal health-related quality of life (HRQoL). To adequately capture patient perspectives in clinical trials, patient-reported outcome (PRO) measures must demonstrate sufficient evidence of content validity in the target population. This study aimed to explore the patient experience of living with COSP and evaluate content validity of the newly developed Chronic Ocular Pain Questionnaire (COP-Q) for use in COSP clinical trials.
METHODS: Qualitative, combined concept elicitation (CE) and cognitive debriefing (CD) interviews were conducted with 24 patients experiencing COSP symptoms in the USA. Interviews were supplemented with real-time data collection via a daily diary app task in a subset of patients (n = 15) to explore the day-to-day patient experience. Three healthcare professionals (HCPs) from the USA, Canada, and France were also interviewed to provide a clinical perspective. CE results were used to further inform development of a conceptual model and to refine PRO items/response options. CD interviews assessed relevance and understanding of the COP-Q. Interviews were conducted across multiple rounds to allow item modifications and subsequent testing.
RESULTS: Eye pain, eye itch, burning sensation, eye dryness, eye irritation, foreign body sensation, eye fatigue, and eye grittiness were the most frequently reported symptoms impacting vision-dependent ADL (e.g., reading, using digital devices, driving) and wider HRQoL (e.g., emotional wellbeing, social functioning, work). COP-Q instructions, items, and response scales were understood, and concepts were considered relevant. Feedback supported modifications to instruction/item wording and confirmed the most appropriate recall periods.
CONCLUSIONS: Findings support content validity of the COP-Q for use in COSP populations. Ongoing research to evaluate psychometric validity of the COP-Q will support future use of the instrument in clinical trial efficacy endpoints.

Treatments for type 2 diabetes vary widely in their complexity. The simplicity or complexity of a treatment regimen may have an impact on patient preference, treatment adherence, and health outcomes. The purpose of this qualitative study was to develop two draft patient-reported outcome instruments focusing on patients\' experience with simplicity and complexity of treatment for type 2 diabetes.
The instruments were developed in a series of steps: gather information to support development of a concept elicitation interview guide (literature review and expert interviews), concept elicitation interviews with patients (N = 30), cognitive interviews with patients (N = 20), and a translatability assessment.
In concept elicitation interviews, patients with type 2 diabetes reported a range of treatment attributes that influence their perceptions of treatment simplicity and complexity, such as injection devices, medication preparation, dose timing, dose frequency, ease of taking the correct dose, flexibility of dose schedule, remembering to take medication, and food requirements. Two draft questionnaires were developed based on the literature review, expert interviews, and concept elicitation interviews with patients. Revisions were made to these draft instruments based on qualitative interviews with patients and translatability assessment.
The qualitative research conducted in this study supports the content validity of two newly developed instruments, the Simplicity of Diabetes Treatment Questionnaire (Sim-Q) and the Simplicity of Diabetes Treatment Questionnaire-Comparison (Sim-Q-Comp), designed to assess the simplicity and complexity of diabetes treatment from the patient\'s perspective.
Treatments for type 2 diabetes vary widely in their complexity, and previous research suggests that simpler treatments may have benefits for patients, such as better medication adherence and improved glycemic control. Despite the benefits of treatment simplicity, there are limited options for assessing simplicity of treatment from the patient perspective. This study was designed to develop two patient-reported outcome measures that assess simplicity and complexity of treatment for type 2 diabetes. Thirty patients with type 2 diabetes reported a range of treatment attributes that influence their perceptions of treatment simplicity and complexity. These attributes included injection devices, medication preparation, dose timing, dose frequency, ease of taking the correct dose, flexibility of dose schedule, and food requirements. Two questionnaires were developed based on literature review, expert interviews, and patient interviews (one questionnaire for rating a single treatment, and another questionnaire for comparing two treatments). Revisions were made to the draft instruments based on feedback from 20 additional participants and a translatability assessment. The resulting instruments are called the Simplicity of Diabetes Treatment Questionnaire (Sim-Q) and Simplicity of Diabetes Treatment Questionnaire-Comparison (Sim-Q-Comp). Future research with more patients is needed to further examine the psychometric properties of the questionnaires.

Dengue is the most prevalent arboviral infection causing an estimated 50-60 million cases of febrile illness globally per year, exacting considerable disease burden. Few instruments exist to assess the patient illness experience, with most based on healthcare provider assessment, lacking standardization in timepoints and symptom assessment. This study aimed to evaluate the content validity of the novel \'Dengue Virus Daily Diary (DENV-DD)\', designed to measure symptom intensity and disease burden within outpatient infant to adult populations.
The Dengue Illness Index Report Card was used as a foundation to create the DENV-DD, consisting of patient- and observer-reported outcome (PRO/ObsRO) instruments. In two South American dengue-endemic communities, qualitative combined concept elicitation and cognitive debriefing interviews were conducted among individuals and caregivers of children with symptomatic laboratory-confirmed dengue. Interviews were conducted across two rounds allowing DENV-DD modifications. A small-scale quantitative assessment of the DENV-DD was also conducted with data from an independent Dengue Human Infection Model (DHIM) to generate early evidence of feasibility of DENV-DD completion, instrument performance and insight into the sign/symptom trajectory over the course of illness.
Forty-eight participants were interviewed (20 adults, 20 older children/adolescents with their caregivers, 8 caregivers of younger children). A wide spectrum of signs/symptoms lasting 3-15 days were reported with fever, headache, body ache/pain, loss of appetite, and body weakness each reported by > 70% participants. DENV-DD instructions, items and response scales were understood, and items were considered relevant across ages. DHIM data supported feasibility of DENV-DD completion.
Findings demonstrate content validity of the DENV-DD (PRO/ObsRO instruments) in dengue-endemic populations. Psychometric and cultural validity studies are ongoing to support use of the DENV-DD in clinical studies.
Dengue is the most common viral infection transmitted to humans by mosquitos, and affects an estimated 50–60 million individuals globally per year. However, there are few resources for understanding and capturing the patient experience of dengue throughout illness. Most research studies are based on healthcare provider assessment, which lack consistency in terms of assessment time points and the signs/symptoms assessed. The ‘Dengue Illness Index Report Card (DII-RC)’ was used as a foundation to create the new ‘Dengue Virus Daily Diary (DENV-DD)’ to better capture the patient experience of symptom intensity and dengue disease burden for the duration of illness. Forty-eight individuals and caregivers of younger children from Peru and Ecuador who recently had symptomatic dengue were interviewed to understand the patient experience over the time of illness and to test whether the DENV-DD is understood by patients and caregivers and includes all relevant and important signs/symptoms and health-related quality of life impacts. Nine individuals with active dengue infection also completed the DENV-DD daily for 28-days as part of a clinical study. We found that > 70% of patients experienced fever, headache, body ache/pain, loss of appetite and body weakness. The DENV-DD instructions, questions and response option(s) were well understood, feasible to complete and the concepts assessed by the DENV-DD were relevant to the dengue experience. Our study adds to the understanding of the dengue illness experience and supports the DENV-DD for use in future dengue studies as an assessment of signs/symptoms throughout the duration of illness.

Background: Systemic lupus erythematosus (SLE) is a chronic autoimmune disease that can lead to irreversible organ damage (OD). Data describing the patient burden of OD, as compared with SLE without OD, are limited. Objective: To develop a comprehensive conceptual model describing the burden experienced by patients living with SLE-associated OD. Methods: There were three phases to this qualitative study. First, a targeted literature review was conducted to inform a draft conceptual model. Second, key opinion leaders (KOLs) were interviewed to assess the draft conceptual model and help shape patient interview materials. Third, patients of different demographic backgrounds from across the United States were interviewed individually to gather their perspectives on living with SLE-associated OD. Data from concept elicitation interviews with KOLs and patients were coded and analyzed using NVivo software to identify the key concepts of the overall patient burden of SLE-associated OD. Findings from the KOL and patient interviews were used to finalize the conceptual model. Results: KOLs highlighted that SLE-associated OD carried a higher rate of mortality than SLE alone. Participants with SLE-associated OD (n = 40) experienced detrimental impacts across 4 areas of their lives: physical, cognitive, psychosocial functioning, and economic and work-related well-being. Physical impacts were described by all participants, often affecting their ability to perform everyday tasks. Many also described deterioration of cognitive functioning. Almost all participants experienced emotional impacts and challenges to their relationships and social lives resulting from living with SLE-associated OD. Additionally, SLE-associated OD imposed an economic burden including increased healthcare costs. SLE-associated OD had a more severe and debilitating impact on all aspects of the patient\'s quality of life than SLE prior to OD development, including further limitations in activities of daily living after the development of OD. Discussion: Study findings guided the development of a comprehensive conceptual model that fully represents the patient experience of living with SLE-associated OD, highlighting the additional burden of OD when compared with SLE alone. Conclusions: The conceptual model will inform improvements in disease management, which may result in better patient outcomes and aid development of clinical outcome assessments of disease burden.

Spontaneous intracranial hypotension (SIH) is an underdiagnosed and debilitating condition caused by a spinal cerebrospinal fluid (CSF) leak. Although SIH can lead to substantial morbidity and disability, little data exists about patients\' perspectives. Without hearing directly from patients, our understanding of the full experience of having SIH is limited, as is our ability to identify and use appropriate patient-reported outcome measures (PROMs) within clinical care and research. The purpose of this study was to conduct qualitative interviews with confirmed SIH patients to fully describe their experiences and identify relevant concepts to measure.
Patients were recruited from an SIH specialty clinic at a large, U.S.-based healthcare center. Patients undergoing an initial consultation who were ≥ 18 years old, English-speaking, met the International Classification of Headache Disorders-3 criteria for SIH, and had a brain MRI with contrast that was positive for SIH were eligible to participate. During semi-structured qualitative interviews with a trained facilitator, participants were asked to describe their current SIH symptoms, how their experiences with SIH had changed over time, and the aspects of SIH that they found most bothersome. Analysts reviewed the data, created text summaries, and wrote analytic reports.
Fifteen participants completed interviews. Common symptoms reported by patients included headache, tinnitus, ear fullness/pressure/pain, and neck or interscapular pain. Patients reported that their symptoms worsened over the course of their day and with activity. The most bothersome aspect of SIH was disruption to daily activities and limits to physical activities/exercise, which were severe. With regard to symptoms, the most bothersome and impactful included physical pain and discomfort (including headache), as well as fatigue.
Patients reported a diverse set of symptoms that were attributed to SIH, with devastating impacts on functioning and high levels of disability. Researchers considering use of PROMs for SIH should consider inclusion of both symptom scales and aspects of functioning, and future work should focus on evaluating the validity of existing measures for this patient population using rigorous qualitative and quantitative methods in diverse samples. Additionally, these data can be used to assist clinicians in understanding the impacts of SIH on patients.

BACKGROUND: Psoriasis is a common autoimmune dermatologic condition which has a pronounced negative impact on patient quality of life and disease burden. Currently, there are a number of treatments available for psoriasis, with differences in efficacy, mechanism of action, mode of administration, adverse effects, and tolerability. However, a reliable, validated patient-reported instrument to address patient expectations and of psoriasis treatment has not been developed. This project was undertaken with the aim of developing a fit-for-purpose self-reported instrument to inform patient expectations and preferences of psoriasis treatments.
METHODS: Two studies, both utilizing qualitative and quantitative methods, were conducted in patients within the entire spectrum of psoriasis severity. In Study 1, a group concept mapping (GCM) exercise was conducted with dermatologists and moderate-to-severe psoriasis patients to identify concepts important in the treatment of psoriasis. In Study 2, a preliminary Treatment Acceptability Questionnaire (TAQ) was developed using GCM-derived concepts from Studies 1 and 2, followed by cognitive debriefing (CD) telephone interviews of the preliminary TAQ. In Study 2, another GCM exercise was conducted with mild and newly diagnosed psoriasis patients. Psychometric analyses were performed on the TAQ to evaluate validity and reliability.
RESULTS: The Study 1 GCM exercise generated 43 concepts from moderate-to-severe psoriasis patients (n = 20) and dermatologists (n = 10). In Study 2, 37 GCM concepts were generated from mild and newly diagnosed psoriasis patients (n = 20). From the 2 GCM exercises, 28 concepts were selected to form the preliminary TAQ; CD interviews indicated strong understanding and relevance of TAQ items among patients with disease ranging from mild to severe. The final TAQ consisted of 20 items; psychometric analysis demonstrated strong validity and reliability of the TAQ.
CONCLUSIONS: The TAQ is a novel psychometrically validated patient-reported instrument to inform healthcare providers of patients\' expectations of and preferences for treatment of their psoriasis and can help in shared decision making between patients and physicians.