Qualitative interview study of patient-reported symptoms, impacts and treatment goals of patients with obstructive hypertrophic cardiomyopathy.

Objective: Hypertrophic cardiomyopathy (HCM), including obstructive HCM (oHCM), is the most common inherited cardiomyopathy causing lifestyle-limiting symptoms. Data are lacking about patients' perspectives on the daily impact of their symptoms. This qualitative interview study was conducted to better understand patients' experiences with oHCM.

Content validity of the Leicester Cough Questionnaire in adults with refractory or unexplained chronic cough: a qualitative interview study.

Background: Chronic cough, a cough lasting >8 weeks, includes refractory chronic cough (RCC) and unexplained chronic cough (UCC). Patient-reported outcome (PRO) measures are needed to better understand chronic cough impacts that matter most to patients. The 19-item Leicester Cough Questionnaire (LCQ), an existing PRO measure of chronic cough, assesses impacts of cough across physical, psychological, and social domains.

Determinants of Bladder Care at Night in a Subacute Ward for Aged Patients: An Observational Study.

Purpose: The purpose of this study was to evaluate how bladder care at night correlates to patients' mobility status.

Design: This was an observational study.

Subjects And Setting: The sample comprises 63 aged care subacute patients who were observed over 3 consecutive nights in an inpatient subacute aged care ward located in Melbourne, Australia.

Understanding the patient experience and treatment benefits in patients with non-small-cell lung cancer with brain metastasis.

Background: Despite the high prevalence of brain metastases (BM) secondary to non-small-cell lung cancer (NSCLC) (NSCLC/BM), patients' experiences (symptoms and impacts) are not fully understood. This study sought to understand the patient experience with NSCLC/BM and identify a patient-reported outcome (PRO) measure fit to capture the most important NSCLC/BM symptoms and impacts.

Methods: A targeted literature review was completed; the National Comprehensive Cancer Network (NCCN)/Functional Assessment of Cancer Therapy-Brain Symptom Index, 24-item version (NFBrSI-24) was identified as a relevant measure that assessed the core symptoms and impacts associated with NSCLC/BM.

Interview-Based Patient- and Caregiver-Reported Experiences of Hunger and Improved Quality of Life with Setmelanotide Treatment in Bardet-Biedl Syndrome.

Introduction: Bardet-Biedl syndrome (BBS) is a rare genetic disease associated with hyperphagia, a pathologic insatiable hunger, due to impaired signaling in the melanocortin-4 receptor (MC4R) pathway. The impact of hyperphagia on the lives of patients with BBS and their families has not been fully characterized.

Methods: Patients with BBS or their caregivers who participated in clinical trials of the MC4R agonist setmelanotide (NCT03013543 and NCT03746522) were included in this qualitative study.

The Bladder at Night during Hospitalisation: Towards optimal care for elderly patients with nocturia.

Objective: This study aimed to describe the characteristics of nocturia in older hospitalised patients and to explore knowledge, beliefs and experiences associated with night toileting while in hospital in order to identify unmet care needs.

Methods: A multisite mixed methods cross-sectional study of older hospitalised adults who were admitted for ≥2 days was conducted using a standardised researcher-administered questionnaire. An additional cohort 16 older hospitalised adults with nocturia >twice per night were interviewed to understand the experience and impact of nocturia during hospitalisation.

Patient and caregiver preferences on treatment attributes for atopic dermatitis.

Background: The expanding number of potential treatment options for atopic dermatitis (AD) highlights the need to better understand the treatment preferences of individuals with AD.

Objective: This study identified attributes that most greatly influenced treatment preferences of adults/adolescents/caregivers of children with mild/moderate/severe AD.

Methods: Adults (≥18 years), adolescents (12-17 years), and caregivers of children (2-11 years) with mild, moderate, or severe AD in the United States (US) and United Kingdom (UK) participated in semistructured interviews.

Development of the impact of weight on daily activities questionnaire: A patient-reported outcome measure.

While patient-reported outcome measures are available to evaluate health-related quality of life and functioning in obesity, existing measures do not evaluate the impact of excess weight and weight loss on the ability to perform regularly occurring daily activities. Three iterative sets of qualitative interviews were conducted in two countries (United States, n = 23; United Kingdom, n = 23) with individuals with body mass index ≥30 kg/m to inform development of the Impact of Weight on Daily Activities Questionnaire (IWDAQ) for use in clinical trials to evaluate daily activity limitations associated with excess weight. Candidate concepts were selected based on the literature, expert opinion, and previously conducted qualitative research, after which the draft IWDAQ was developed and tested.

Treatment Mode Preferences in Psoriatic Arthritis: A Qualitative Multi-Country Study.

Objective: Qualitative research exploring patient preferences regarding the mode of treatment administration for psoriatic arthritis (PsA) is limited. We report patient preferences and their reasons across PsA treatment modes.

Methods: In this global, cross-sectional, qualitative study, interviews were conducted with adult patients with PsA in Brazil, France, Germany, Italy, Spain, the UK, and the US.

Patient and physician preferences for ulcerative colitis treatments in the United States.

This study aimed to elicit patient and physician preferences for ulcerative colitis (UC) treatments in the United States (US). The following UC treatment attributes included in the discrete-choice experiment (DCE) were identified during qualitative interviews with both patients and physicians: time to symptom improvement, chance of long-term symptom control, risks of serious infection and malignancy, mode and frequency of administration, and need for steroids. The DCE survey instruments were developed and administered to patients and physicians.

Validation of a new measure of quality of life in obesity trials: Impact of Weight on Quality of Life-Lite Clinical Trials Version.

The Impact of Weight on Quality of Life-Lite (IWQOL-Lite) is widely used in evaluations of weight-loss interventions, including pharmaceutical trials. Because this measure was developed using input from individuals undergoing intensive residential treatment, the IWQOL-Lite may include concepts not relevant to clinical trial populations and may be missing concepts that are relevant to these populations. An alternative version, the IWQOL-Lite Clinical Trials Version (IWQOL-Lite-CT), was developed and validated according to the US Food and Drug Administration's (FDA's) guidance on patient-reported outcomes.

Does successful treatment of overactive bladder improve co-morbidities in patients with nocturia?

Objective: To investigate whether treatment of overactive bladder (OAB), one comorbidity of nocturia, could reduce waking to void and improve other co-existing symptoms.

Methods: A prospective cohort study was conducted at Royal Melbourne Hospital. Participants received 12 weeks of standard treatment, including lifestyle interventions and pharmacotherapy.

Development of the Painful Periods Screening Tool for endometriosis.

Objectives: Diagnostic delay is common in endometriosis. There is an unmet need for a symptom-based, patient-completed screening tool to facilitate discussions between patients and physicians about potential endometriosis symptoms. The objective of this study was to develop and assess the patient-completed Painful Periods Screening Tool (PPST) to assess the presence of potential endometriosis symptoms.

Questions to ask a patient with nocturia.

Background: Patients may not raise nocturia as a concern as they mistakenly consider the symptom to be a normal part of ageing. Nocturia is associated with significant morbidity and is likely to be a marker of poor health.

Objective: This paper provides questions to guide diagnosis, evaluation and individualised treatment of nocturia.

Exploring the Diabetic Gastroparesis Patient Experience: Patient Exit Interviews.

Introduction: To improve understanding of the diabetic gastroparesis (DGP) patient experience and inform the patient-reported outcome measurement strategy for future trials in DGP, qualitative interviews were conducted with participants in a phase 2 clinical trial of a novel DGP treatment.

Methods: Trial participants were invited to participate in interviews at both the pretreatment visit (PTV) and the end-of-treatment visit (EOTV). The interviews were conducted by experienced qualitative researchers and followed a semistructured interview guide.

Understanding the Patient Experience with Carcinoid Syndrome: Exit Interviews from a Randomized, Placebo-controlled Study of Telotristat Ethyl.

Purpose: Telotristat ethyl, an oral tryptophan hydroxylase inhibitor, is intended to treat carcinoid syndrome by reducing serotonin production. Telotristat ethyl was evaluated in TELESTAR, a Phase III study for patients who had carcinoid syndrome with at least 4 bowel movements (BMs) per day and who were receiving somatostatin analogue therapy. This interview substudy was conducted to provide insight into the patient experience in TELESTAR and to help understand whether reductions in BM frequency (the primary end point) and other symptoms were clinically meaningful.

TANGO - a screening tool to identify comorbidities on the causal pathway of nocturia.

Objectives: To develop a robust screening metric for use in identifying non-lower urinary tract comorbidities pertinent to the multidisciplinary assessment of patients with nocturia.

Methods: Variables having a significant risk association with nocturia of greater than once per night were identified. Discriminating items from validated and reliable tools measuring these comorbidities were identified.

Clinical trials in irritable bowel syndrome: a review.

Irritable bowel syndrome (IBS) is one of the most common gastrointestinal disorders and it is characterized by episodes of abdominal pain and altered bowel functions. The specific bowel disturbances of diarrhea, constipation or an alternation between the two defines the IBS subtypes of diarrhea-predominant, constipation-predominant, and mixed or alternating IBS. Because of the abnormalities in bowel states associated with each IBS subtype, it is not likely that one agent would successfully treat all three subtypes.

Urgency as an Endpoint in Irritable Bowel Syndrome.

Background: The choice of endpoints is crucial for proper evaluation of agents in clinical trials of irritable bowel syndrome (IBS). In a recently published draft guidance for IBS from the United States Food and Drug Administration (FDA), urgency was not considered an appropriate primary endpoint. The FDA's position is that it is not clear how patients with diarrhea-predominant IBS (D-IBS) "define or describe urgency".