Symptom Experience and Content Validity of the Psoriasis Symptom Scale (PSS) in Patients with Generalized Pustular Psoriasis (GPP).

Introduction: We sought to understand key symptoms of generalized pustular psoriasis (GPP) and to confirm the relevance to patients and content validity of the Psoriasis Symptom Scale (PSS) in GPP.

Methods: A targeted literature review and clinical expert interviews were conducted as background research. Patients were interviewed individually (involving concept elicitation and cognitive interviews), and a separate patient workshop was conducted to determine disease-specific symptoms of importance.

People With Diabetes and Caregivers Prefer Rescue Glucagon Treatment With a Wider Storage Temperature Range and a Nasal Administration, When Efficacy is Similar: A Discrete Choice Experiment in Spain.

Background: Conventional injectable glucagon (IG) and nasal glucagon (NG), both having similar efficacy, are two options for the emergency treatment of severe hypoglycemia in Spain. This study elicited the effect of changes in key attributes on preferences for NG and IG medication profiles of people with diabetes and caregivers in Spain.

Methods: The relative attribute importance (RAI) that participants placed on glucagon preparation, preparation and administration time, delivery method, recovery time, device size, storage temperature, and headache risk was estimated from an online discrete choice experiment.

Establishing an alternative accommodation for stable hospitalised antepartum patients: barriers and challenges.

Background: Patients in remote communities who risk premature delivery require transfer to a tertiary care centre for obstetric and neonatal care. Following stabilisation, many patients are candidates for outpatient management but cannot be discharged to their home communities due to lack of neonatal intensive care unit (ICU) support.

Problem: Without outpatient accommodation proximal to neonatal ICU, these patients face prolonged hospitalisation-an expensive option with medical, social and psychological consequences.

Patient-Reported Disease Burden and Unmet Therapeutic Needs in Atopic Dermatitis.

Background: Atopic dermatitis (AD) is a common, chronic, relapsing, inflammatory skin disease causing a variety of dermatologic signs and symptoms, affecting patient’s quality of life. While treatment options are available, they are of variable effectiveness. This study sought to characterize patient-reported AD signs and symptoms, flare, and associated bother, by disease severity and control.

Patients' preferences for delaying metastatic castration-resistant prostate cancer: Combining health state and treatment valuation.

Purpose: Men with castration-resistant prostate cancer (CRPC) experience disease progression at different rates. The purpose of this study was to quantify the strength of patient preferences for delaying prostate cancer progression utilizing a discrete choice experiment (DCE) and valuing 3 health states in the continuum of CRPC.

Patients And Methods: Men with CRPC, recruited from US patient panels, completed a cross-sectional web-based survey.

Reliability, validity, and the ability to detect change of the Psoriasis Symptom Scale (PSS) in patients with plaque psoriasis.

The primary objective of the study was to evaluate the measurement properties of the patient-reported four-item Psoriasis Symptom Scale (PSS).: Analysis of phase-III data on the efficacy of risankizumab to assess psychometric characteristics of the PSS in patients with moderate-to-severe psoriasis. PSS items had a good range of symptom severity coverage.

Adultification of Black Children in Pediatric Anesthesia.

Background: Unconscious racial bias in anesthesia care has been shown to exist. We hypothesized that black children may undergo inhalation induction less often, receive less support from child life, have fewer opportunities to have a family member present for induction, and receive premedication with oral midazolam less often.

Methods: We retrospectively collected data on those <18 years of age from January 1, 2012 to January 1, 2018 including age, sex, race, height, weight, American Society of Anesthesiologists (ASA) physical status, surgical service, and deidentified anesthesiology attending physician.

Burden of renal angiomyolipomas associated with tuberous sclerosis complex: results of a patient and caregiver survey.

Background: Tuberous sclerosis complex (TSC) is a rare genetic disorder characterized by benign tumors in multiple organs, including non-cancerous kidney lesions known as renal angiomyolipomas. This study's objective is to describe the age-stratified morbidity, treatment patterns, and health-related quality of life of TSC patients with renal angiomyolipomas in the United States. A cross-sectional, anonymous web-based survey was conducted with a convenience sample of TSC patients and caregivers identified through a patient advocacy organization.

MCDA swing weighting and discrete choice experiments for elicitation of patient benefit-risk preferences: a critical assessment.

Purpose: Multiple criteria decision analysis swing weighting (SW) and discrete choice experiments (DCE) are appropriate methods for capturing patient preferences on treatment benefit-risk trade-offs. This paper presents a qualitative comparison of the 2 methods.

Methods: We review and critically assess similarities and differences of SW and DCE based on 6 aspects: comprehension by study participants, cognitive biases, sample representativeness, ability to capture heterogeneity in preferences, reliability and validity, and robustness of the results.

Results of a Qualitative Study to Develop a Patient Reported Outcome Measure for Patients with 4 Subtypes of Soft Tissue Sarcoma.

Objective: The objective of this research was to develop a disease-specific symptom inventory for soft tissue sarcoma.

Methods: Literature review and clinical expert and patient interviews were conducted to determine disease-specific symptoms important to patients with one of the four STS subtypes. Clinical experts identified the most relevant STS symptom items from the item pool developed from literature review.

The content validity of the PSS in patients with plaque psoriasis.

Background: The primary objective of this study was to evaluate the content validity of the Psoriasis Symptom Scale (PSS), with a specific focus on understanding of the content of the PRO measure by conducting one-on-one interviews with patients with moderate to severe plaque psoriasis. This was a cross-sectional, qualitative study conducted with 20 patients with plaque psoriasis who participated in in-person, one-on-one interviews. Participants were asked to describe their psoriasis symptoms, completed the PSS, and were cognitively debriefed on its content.

Caring for Children With Tuberous Sclerosis Complex: What Is the Physical and Mental Health Impact on Caregivers?

Disease burden associated with tuberous sclerosis complex, a genetic disorder characterized by benign tumor growth including lesions in multiple organs, puts tremendous demands on families. This analysis examines the physical and mental health burden of tuberous sclerosis complex caregivers in the United States. An institutional review board-approved web-based survey of tuberous sclerosis complex caregivers collected information; descriptive analyses were conducted on age-based subgroups.

Tuberous sclerosis complex: a survey of health care resource use and health burden.

Purpose: Tuberous sclerosis complex (TSC) is a multiorgan, autosomal-dominant genetic disorder with incomplete penetrance.

Methods: This analysis of a web-based survey focuses on the clinical presentation, management, and associated burden of patients with TSC in the United States.

Results: A total of 676 TSC patients or caregivers responded.

The burden of subependymal giant cell astrocytomas associated with tuberous sclerosis complex: results of a patient and caregiver survey.

Tuberous sclerosis complex is a genetic disorder characterized by benign tumor growth including lesions in the ventricular system of the brain known as subependymal giant cell astrocytomas. This analysis focuses on the clinical presentation, management, and associated burden of subependymal giant cell astrocytomas in patients with tuberous sclerosis complex in the United States. An institutional review board-approved web-based survey of tuberous sclerosis complex patients and caregivers collected information, and descriptive analyses were conducted on age-based subgroups.

Development of a preference-based index from the National Eye Institute Visual Function Questionnaire-25.

Importance: Understanding how individuals value health states is central to patient-centered care and to health policy decision making. Generic preference-based measures of health may not effectively capture the impact of ocular diseases. Recently, 6 items from the National Eye Institute Visual Function Questionnaire-25 were used to develop the Visual Function Questionnaire-Utility Index health state classification, which defines visual function health states.

Vision-related functioning outcomes of dexamethasone intravitreal implant in noninfectious intermediate or posterior uveitis.

Purpose: To evaluate the effect of a single treatment with dexamethasone intravitreal implant (DEX implant) on patient-reported visual functioning in patients with noninfectious intermediate or posterior uveitis.

Methods: Patient eyes with noninfectious intermediate or posterior uveitis were randomized to a single treatment with DEX implant 0.70 mg (n=77), DEX implant 0.

Psychometric evaluation of the National Eye Institute Visual Function Questionnaire and Visual Function Questionnaire Utility Index in patients with non-infectious intermediate and posterior uveitis.

Objective: To evaluate the psychometric properties of the National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) and Visual Function Questionnaire Utility Index (VFQ-UI) in patients with non-infectious intermediate and posterior uveitis.

Methods: Secondary analysis of pooled data from a 26-week, multicenter, masked, randomized, sham-controlled Phase 3 clinical trial. Health-related quality of life was assessed using the NEI VFQ-25, the EQ-5D, and SF-36.

Normative comparison of patient-reported outcomes in patients with noninfectious uveitis.

Objective: To compare vision-related functioning and health-related quality of life of patients with noninfectious intermediate or posterior uveitis with those of the US general population and normal-vision reference groups.

Methods: Secondary analysis of health-related quality of life measures administered at baseline to patients with noninfectious intermediate or posterior uveitis participating in the HURON trial, a 26-week, multicenter, masked, randomized, sham-controlled trial of a dexamethasone intravitreal implant (n=224) was performed. Patient-reported outcome measures included the National Eye Institute Visual Function Questionnaire-25, the 36-Item Short-Form Health Survey, the Short Form-6 Dimensions, and the EuroQol-5D.

Health-related quality of life outcomes of adalimumab for patients with early rheumatoid arthritis: results from a randomized multicenter study.

Objective: Rheumatoid arthritis (RA) is associated with significant impairments in health-related quality of life (HRQOL). We evaluated patient-reported outcomes including HRQOL outcomes following adalimumab plus methotrexate (MTX) therapy in patients with early RA.

Methods: PREMIER was a phase III, multicenter, randomized, double-blind, active-comparator clinical trial in early RA.

Rasch analysis in the development of a simplified version of the National Eye Institute Visual-Function Questionnaire-25 for utility estimation.

Purpose: Preference-based health measures value how people feel about the desirability of a health state. Generic measures may not effectively capture the impact of vision loss from ocular diseases. Disease-targeted measures could address this limitation.

Psychometric characteristics of the short form 36 health survey and functional assessment of chronic illness Therapy-Fatigue subscale for patients with ankylosing spondylitis.

Background: We evaluated the psychometric characteristics of the Short Form 36 (SF-36) Health Survey and the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue subscale in patients with ankylosing spondylitis (AS).

Methods: We analyzed clinical and patient-reported outcome (PRO) data collected during 12-week, double-blind, placebo-controlled periods of two randomized controlled trials comparing adalimumab and placebo for the treatment of active AS. The Bath Ankylosing Spondylitis Disease Activity Index, Bath Ankylosing Spondylitis Functional Index, and other clinical measures were collected during the clinical trial.

Validation of the National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) in age-related macular degeneration.

Purpose: Patient-reported measures of visual function are increasingly incorporated into clinical trials of new treatments for age-related macular degeneration (AMD). Limited information is available regarding the associations between distance visual acuity (VA), reading speed, or contrast sensitivity and the National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) subscales judged relevant to these measures. This study's objective was to evaluate such associations along with questions on restricted activity days.

Observational, nonintervention, multicenter study for validation of the Bowel Function Index for constipation in European countries.

Objective: Constipation is a common adverse event of treatment with opioids for chronic non-malignant pain and may result in a considerable reduction in health-related quality of life. The aim of this study was to assess the psychometric properties of the Bowel Function Index (BFI) in european patients suffering from constipation secondary to opioid analgesic treatment for chronic, non-malignant pain.

Methods: This was a multinational study conducted at 15 clinical sites in the Czech Republic, Germany, Italy, and the United Kingdom.