Introduction: Following a review of patient-reported outcome (PRO) instruments in the literature, existing PRO instruments may not adequately capture the experience of receiving treatment for proliferative diabetic retinopathy (PDR). Therefore, this study aimed to develop a de novo instrument to comprehensively assess the patient experience of PDR. Methods: This qualitative, mixed-methods study comprised item generation for the Diabetic Retinopathy-Patient Experience Questionnaire (DR-PEQ), content validation in patients with PDR, and preliminary Rasch measurement theory (RMT) analyses. Adult patients with diabetes mellitus and PDR who received aflibercept and/or panretinal photocoagulation within 6 months of study initiation were eligible for participation. The preliminary DR-PEQ comprised four scales: Daily Activities, Emotional Impact, Social Impact, and Vision Problems. DR-PEQ items were generated using existing knowledge of patient experiences in PDR and conceptual gaps identified from existing PRO instruments. Patients indicated the level of difficulty conducting daily activities and frequency experiencing emotional impacts, social impacts, and vision problems attributed to diabetic retinopathy and its treatment in the past 7 days. Content validity was evaluated in two rounds of in-depth, semi-structured patient interviews. Measurement properties were investigated via RMT analyses. Results: The preliminary DR-PEQ comprised 72 items. Overall, mean (SD) patient age was 53.7 (14.7) years. Forty patients completed the first interview; of these, 30 completed the second interview. Patients reported that the DR-PEQ was easily understood and relevant to their experience. Minor revisions, including removal of the Social Impact scale and addition of a Treatment Experience scale, were implemented to generate 85 items spanning four scales: Daily Activities, Emotional Impact, Vision Problems, and Treatment Experience. RMT analyses provided preliminary evidence that the DR-PEQ performed as intended. Conclusion: The DR-PEQ evaluated a broad spectrum of symptoms, functional impacts, and treatment experiences relevant to patients with PDR. Additional analyses are warranted to evaluate psychometric properties in a larger patient population.

Objective: Agitation is a common neuropsychiatric symptom in patients with Alzheimer’s dementia, resulting in significant burden to patients and caregivers. This study was conducted to better understand caregiver perspectives on the frequency and severity of agitation behaviors, captured by the Cohen-Mansfield Agitation Inventory, and to assess what constitutes a meaningful change in these behaviors. Methods: This was a qualitative, non-interventional, descriptive study conducted in the United States. Semi-structured 1:1 interviews were completed with non- professional caregivers of patients with Alzheimer’s disease (AD) (i.e., family caregivers providing unpaid, informal care) who met the following inclusion criteria: ≥21 years old; caring for an individual with clinically confirmed AD; noticed agitation behaviors including emotional distress, excessive movements, verbal aggression, physical aggression; spent ≥4 days a week for ≥2 hours with the person with AD; and willing to participate in the virtual interview in English. Interview transcripts were analyzed thematically through detailed line-by-line inductive and deductive coding approaches using the ATLAS.ti software. Results: Thirty caregivers participated. Participants’ mean age was 64 (±13) years. Most participants were Caucasian/white (77%), non-Hispanic or Latinx (97%), and female (70%). Approximately half were providing care to their spouse (53%), and the remainder for a parent or parent-in-law. Several key themes on what constitutes ‘meaningful change’ emerged. All caregivers tied a meaningful change in agitation behaviors to a change in behavior frequency (becoming less frequent or stopping entirely). Beyond frequency, themes describing meaningful change included: change in behavior intensity; apparent intent to disturb or cause harm to self or others; potential to cause serious harm; amount of harm caused; more “normal” behavior; shorter episode duration; and less worry, frustration, or isolation. Additionally, a shift from verbally aggressive behavior to physically aggressive behavior was considered an escalation in agitation severity and meaningful worsening. Thus, unique to physical aggression, a meaningful change was described as a de-escalation to verbal aggression. Conclusion: Caregivers report several themes on what constitutes meaningful improvement or worsening of agitation behaviors in individuals with AD. A change in the frequency of agitation behaviors was consistently reported by caregivers as important, with decreased frequency perceived as a meaningful improvement.

Introduction: Few qualitative studies have explored the patient experience of daily life with proliferative diabetic retinopathy (PDR) and associated treatments. Herein, a conceptual model was developed to comprehensively examine symptoms, functional impacts, and treatment experiences in PDR.Methods: A qualitative, mixed-methods study comprising a literature search and semi-structured interviews with clinicians and patients was conducted. Published literature and online patient resources were searched to identify concepts relevant to patients, including symptoms, functional impacts, and treatment experiences of PDR. Semi-structured interviews with experienced clinicians were conducted to identify symptoms and impacts reported by patients with PDR and to receive feedback regarding concepts identified from the literature search. A preliminary conceptual model was then developed based on findings from the literature search and clinician interviews. Patients with PDR participated in two rounds of semi-structured interviews to identify additional concepts relevant to the patient experience in PDR and associated treatments, which informed revisions to the conceptual model. Saturation of patient interviews was assessed.Results: Findings from the literature search and clinician interviews yielded 109 concepts that were included in a preliminary conceptual model with three overarching domains: symptoms, impacts, and managing the disease. Clinicians confirmed concepts identified from the literature search. During interviews, patients reported a broad spectrum of symptoms (e.g., red vision); functional impacts relating to activities of daily living (e.g., reading), emotional functioning (e.g., loss of independence), and social functioning (e.g., problems recognizing faces); and treatment experiences (e.g., improves eye problems, no change) associated with PDR. Additional concepts elicited in patient interviews informed revisions to the conceptual model. Saturation was achieved in the patient sample.Conclusions: A wide variety of symptoms, functional impacts, and treatment experiences that significantly affect health-related quality of life were identified in patients with PDR. These insights are critical for understanding PDR symptomology and assessing treatment response.

To describe the Hi-DEF Scale validation study and characteristics of the study sample.

To describe findings from concept elicitation (CE) and cognitive debriefing (CD) interviews and inform the development of a novel patient-reported outcome (PRO) instrument, "Huntington's Disease Everyday Functioning (Hi-DEF)."