Exploring the patient experience of chronic hepatitis D (CHD) and assessment of content validity of the Hepatitis Quality of Life Questionnaire and (HQLQv2) and the Fatigue Severity Scale (FSS).

Background: Chronic hepatitis D (CHD) is the most severe form of viral hepatitis, which results in accelerated progression to cirrhosis and poor prognosis compared with other hepatitis infections, impacting patients' health-related quality of life (HRQoL). To adequately capture patient perspectives of new hepatitis D virus (HDV) treatments in clinical trials, patient-reported outcome (PRO) measures that are valid and assess key concepts relevant to the patient are needed. This study aimed to explore the patient experience of CHD and evaluate the content validity of the Hepatitis Quality of Life Questionnaire (HQLQv2) and the Fatigue Severity Scale (FSS) for use in an HDV population.

Understanding the Patient Experience of Advanced Parkinson's Disease: Qualitative Research with Patients and Expert Clinicians to Identify Symptoms and Associated Health-Related Quality of Life Impacts.

Introduction: Parkinson's disease (PD) is a progressive neurodegenerative condition, characterized by motor symptoms (MS) and non-motor symptoms (NMS). Currently, there is no global definition of advanced PD, but those in advanced stages experience diverse and severe symptomatology, and significant impacts to their health-related quality of life (HRQoL). This qualitative interview study aimed to explore the experience of symptoms and HRQoL impacts of advanced PD from the patient and clinician perspective and identify key concepts for assessment in PD clinical trials.

Patient Experience of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): A Qualitative Exploration of Signs, Symptoms, and Health-Related Quality of Life Impacts.

Introduction: Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare type of autoimmune neuropathy, characterized by signs of distal and proximal weakness of the upper and lower limbs, sensory dysfunction, absent or diminished tendon reflexes, and symptoms of numbness, tingling, pain, and fatigue. These signs/symptoms can lead to difficulty walking, climbing stairs, and reduced manual dexterity. Detailed qualitative exploration of the patient experience of CIDP, notably signs/symptoms, its impacts on health-related quality of life, and treatment experience is limited.

Qualitative In-trial Interviews: Methods, Challenges, and Best Practice.

Qualitative in-trial interviews with clinical trial participants are a means of providing rich, in-depth patient experience data to supplement and complement data captured by clinical outcome assessments and other clinical trial efficacy endpoints. Such in-trial interview data can be used to build understanding of disease and treatment experiences, evaluate content validity of clinical outcome assessments, aid interpretation of scores and meaningful changes, inform trial design feasibility and operational considerations, and provide supportive evidence regarding safety, efficacy, and effectiveness. Despite the rapid growth of in-trial interviews as part of clinical development programs in the pharmaceutical industry in recent years, published guidelines regarding the methods, conduct, and implementation of in-trial interviews are scarce.

Development and validation of a quality of life and treatment satisfaction measure in canine osteoarthritis.

Introduction: Canine osteoarthritis (OA) causes pain and mobility impairment. This can reduce dog quality of life (QoL), owner QoL and owners' satisfaction with, and adherence to, treatments. No existing canine OA-specific instrument assesses all three impacts.

Qualitative interviews of patients with COPD and muscle weakness enrolled in a clinical trial evaluating a new anabolic treatment: patient perspectives of disease experience, trial participation and outcome assessments.

Background: Chronic obstructive pulmonary disease (COPD) and muscle weakness can cause impaired physical function, significantly impacting patients' health-related quality of life (HRQoL). Loss of muscle strength is usually assessed through clinical and performance outcome (PerfO) assessments, which consists of tasks performed in a standardized manner, providing evidence of a patient's functional ability. However, evidence documenting the patient experience of COPD and muscle weakness is limited.

Qualitative exploration of the visual function impairments and impacts on vision-dependent activities of daily living in Retinitis Pigmentosa and Leber Congenital Amaurosis: content validation of the ViSIO-PRO and ViSIO-ObsRO measures.

Background: Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) are rare inherited retinal degenerative disorders. The associated visual impairments have significant impacts on patients' vision-dependent activities of daily living (ADL), mobility, and distal health-related quality of life (HRQoL). To adequately capture patient and caregiver perspectives in clinical trials, patient and observer-reported outcome instruments must demonstrate sufficient evidence of content validity in the target population.

Development of Novel Patient-Reported Outcome (PRO) and Observer-Reported Outcome (ObsRO) Instruments in Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA): ViSIO-PRO and ViSIO-ObsRO.

Introduction: Retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) are rare inherited retinal degenerative disorders resulting in visual impairments and impacts on patients' vision-dependent activities of daily living (ADL), mobility and distal health-related quality of life (HRQoL). This study aimed to conduct qualitative research to understand the patient experience of RP/LCA across genotypes and inform development of patient- and observer-reported outcome (PRO/ObsRO) instruments in RP/LCA.

Methods: Research activities included a qualitative literature review and review of existing visual function PRO instruments in RLBP1 RP, and concept elicitation (CE) and cognitive debriefing (CD) interviews of existing PRO instruments with patients with RLBP1 RP, expert clinicians, and payers.

Psychometric Validation of the ViSIO-PRO and ViSIO-ObsRO in Retinitis Pigmentosa and Leber Congenital Amaurosis.

Introduction: Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) are rare inherited retinal degenerative disorders. The Visual Symptom and Impact Outcomes patient-reported outcome (ViSIO-PRO) and observer-reported outcome (ViSIO-ObsRO) instruments were developed in this population to assess visual function symptoms and impacts on vision-dependent activities of daily living (ADL) and distal health-related quality of life (HRQoL). This study aimed to explore the psychometric properties of the ViSIO-PRO and ViSIO-ObsRO in RP/LCA.

How strong should my anchor be for estimating group and individual level meaningful change? A simulation study assessing anchor correlation strength and the impact of sample size, distribution of change scores and methodology on establishing a true meaningful change threshold.

Purpose: Treatment benefit as assessed using clinical outcome assessments (COAs), is a key endpoint in many clinical trials at both the individual and group level. Anchor-based methods can aid interpretation of COA change scores beyond statistical significance, and help derive a meaningful change threshold (MCT). However, evidence-based guidance on the selection of appropriately related anchors is lacking.

Generation of evidence supporting the content validity of SF-36, FACIT-F, and LupusQoL, and novel patient-reported symptom items for use in patients with systemic lupus erythematosus (SLE) and SLE with lupus nephritis (LN).

Objective: SLE and lupus nephritis (LN) have significant impacts on the health-related quality of life of patients living with the condition, which are important to capture from the patient's perspective using patient-reported outcomes (PROs). The objectives of this study were to evaluate the content validity of PROs commonly used in SLE and LN (36-Item Short Form Health Survey (SF-36), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) and Lupus Quality of Life (LupusQoL), as well as novel PRO symptom severity items measuring skin rash, joint pain, joint stiffness and swelling of the legs and/or feet, in both populations.

Methods: Qualitative, semi-structured, cognitive interviews were conducted with 48 participants (SLE=28, LN=20).

Perception of impact of Dravet syndrome on children and caregivers in multiple countries: looking beyond seizures.

Aim: To assess the relevance and generalizability across countries of concepts of the impact of Dravet syndrome beyond seizures, as recognized by families.

Method: Caregivers of children with Dravet syndrome in four countries (Australia [n=8]; USA, UK, and Italy [all n=4]) participated in 1-hour qualitative telephone interviews, identifying key Dravet syndrome concepts. Interviews were recorded, transcribed, and, where necessary, translated into English for thematic analysis.

Psychometric evaluation of a patient-reported outcome measure in pancreatic exocrine insufficiency (PEI).

Background/objectives: Pancreatic exocrine insufficiency (PEI) is commonly caused by chronic pancreatitis (CP) or cystic fibrosis (CF). There are no PEI-specific patient-reported assessments of symptoms and impacts. The PEI Questionnaire (PEI-Q) was developed through qualitative research with PEI patients and expert clinical input.

Development and preliminary psychometric evaluation of an owner-completed measure of feline quality of life.

Due to improved healthcare and pet longevity, measurement of health-related quality of life (HRQoL) is increasingly important in companion animal medicine. The aim of this study was to develop and evaluate the content and psychometric properties of an owner-completed assessment of health and quality of life (QoL) in cats for use in general veterinary clinical practice. A 23-item feline QoL measure, drafted based on findings from an online survey completed by 45 pet owners, was revised following qualitative interviews with 10 pet owners of healthy cats to assess content validity.

Development and content validation of a preliminary core set of patient- and caregiver-relevant outcomes for inclusion in a potential composite endpoint for Dravet Syndrome.

Background: Dravet Syndrome (DS) is a rare developmental and epileptic encephalopathy characterized by multiple seizures, frequently prolonged and treatment refractory, with significant developmental disabilities and behavioral and psychiatric disorders. Patients with DS require intensive support and supervision from a caregiver, impacting significantly on both patients' and caregivers' lives. This study aimed to identify core concepts to measure the impact on both patients and caregivers in future DS clinical trials.

Qualitative Assessment of the Symptoms and Impact of Pancreatic Exocrine Insufficiency (PEI) to Inform the Development of a Patient-Reported Outcome (PRO) Instrument.

Background: Pancreatic exocrine insufficiency (PEI) affects patients with chronic pancreatitis (CP) and cystic fibrosis (CF) who produce insufficient digestive pancreatic enzymes. Common symptoms include steatorrhoea, diarrhea, and abdominal pain.

Objective: The objective of the study was to develop and test the content validity of a patient-reported outcome (PRO) instrument assessing PEI symptoms and their impact on health-related quality of life.

Development of a Consensus Statement for the Definition, Diagnosis, and Treatment of Acute Exacerbations of Idiopathic Pulmonary Fibrosis Using the Delphi Technique.

Introduction: There is a lack of agreed and established guidelines for the treatment of acute exacerbations of idiopathic pulmonary fibrosis (AE-IPF). This reflects, in part, the limited evidence-base underpinning the management of AE-IPF. In the absence of high-quality evidence, the aim of this research was to develop a clinician-led consensus statement for the definition, diagnosis and treatment of AE-IPF.

Methicillin resistance of staphylococci isolated from the skin of dogs with pyoderma.

Objective: To determine the methicillin-resistant profile of staphylococcal isolates from the skin of dogs with pyoderma.

Animals: 90 dogs with pyoderma.

Procedure: Staphylococci isolated from dogs with pyoderma were tested for susceptibility to methicillin by use of a standard disk diffusion test with oxacillin disks.

Effects of short-term trimethoprim-sulfamethoxazole administration on thyroid function in dogs.

Objective: To determine how rapidly trimethoprim-sulfamethoxazole affects serum total thyroxine (T4) and thyroid-stimulating hormone (TSH) concentrations in euthyroid dogs and how quickly hormone concentrations return to reference values following discontinuation of administration.

Design: Prospective study.

Animals: 7 healthy euthyroid dogs.