Patient and clinician preferences for biologic treatments for severe uncontrolled asthma: a discrete choice experiment (DCE).

Objectives: To estimate the preferences of patients with asthma and asthma-treating clinicians for attributes of biologic treatments, to compare patients' and clinicians' preferences, and to better understand the reasons for their preferences.

Methods: Adults with moderate-to-severe asthma and clinicians who treat asthma in the US completed a cross-sectional, online survey including a discrete choice experiment (DCE) that consisted of seven attributes spanning treatment efficacy, risk and convenience. Marginal utilities were estimated using a mixed logit model, and relative attribute importance scores calculated.

Characterizing health-related quality of life and identifying disease predictors among patients suspected of having long COVID: an analysis of COMET-ICE clinical trial data.

Introduction: Long COVID affects health-related quality of life (HRQoL). Here, we investigate the extent to which symptoms experienced during the acute phase of COVID-19 are significant predictors of the presence of long COVID at 12 weeks.

Methods: Post-hoc analysis of COMET-ICE trial data, which assessed sotrovimab vs.

Treatment preferences of patients with relapsed or refractory multiple myeloma in the United States, United Kingdom, Italy, Germany, France, and Spain: results from a discrete choice experiment.

Introduction: Newer treatment options for relapsed/refractory multiple myeloma (RRMM) with efficacy and safety profiles that differ from traditional therapies have facilitated personalized management strategies to optimize patient outcomes. In the context of such personalized management, understanding how treatment characteristics influence patients' preferences is essential. This study assessed patients' preferences for RRMM treatment attributes and determined trade-offs between potential benefits, administration procedures, and adverse effects.

Estimating meaningful change for The Impact of Weight on Self-Perception (IW-SP) questionnaire among people with type 2 diabetes.

Purpose: The Impact of Weight on Self-perception Questionnaire (IW-SP) is a three-item patient-reported outcome measure (PROM) instrument assessing the impact of body weight on self-perception. To date no published threshold for meaningful change exists. The objective of this study was to estimate the minimal important change (MIC) for the IW-SP among people with type 2 diabetes.

The Impact of Substantial Improvements in HbA1c and Weight Loss on the Medication Preferences of People with Type 2 Diabetes.

Purpose: To quantify the preferences of people with type 2 diabetes (T2D) for treatment attributes of a glucose-dependent insulinotropic polypeptide (GIP)/glucagon-like peptide-1 (GLP-1) receptor agonist (RA) versus an injectable GLP-1 RA medication profile.

Patients And Methods: Injection-naive people taking oral medications for T2D in the US and UK completed a web survey including a discrete choice experiment to quantify patients' preferences for five treatment attributes: delivery system, frequency of nausea, frequency of hypoglycemia, HbA1c reduction, and weight reduction. Attributes and levels were based on head-to-head clinical trial data of tirzepatide 5mg, 10mg, and 15mg versus semaglutide 1mg.

Content validity and psychometric properties of the inFLUenza Patient-Reported Outcome Plus (FLU-PRO Plus) instrument in patients with COVID-19.

Purpose: A well-defined and reliable patient-reported outcome instrument for COVID-19 is important for assessing symptom severity and supporting research studies. The InFLUenza Patient-Reported Outcome (FLU-PRO) instrument has been expanded to include loss of taste and smell in the FLU-PRO Plus, to comprehensively cover COVID-19 symptoms. Our studies were designed to evaluate and validate the FLU-PRO Plus among patients with COVID-19.

Pexidartinib Provides Modest Pain Relief in Patients With Tenosynovial Giant Cell Tumor: Results From ENLIVEN.

Background: The double-blind, randomized, placebo-controlled phase 3 study of orally administered PLX3397 in patients with pigmented villonodular synovitis or giant cell tumor of the tendon sheath (ENLIVEN) showed that pexidartinib provides a robust objective tumor response in adults with tenosynovial giant cell tumors (TGCT) not amenable to improvement with surgery. Based on these results, in 2019, pexidartinib received accelerated approval in the United States in this population as a breakthrough therapy under an orphan drug designation. However, the ability of pexidartinib to relieve pain in ENLIVEN was not fully detailed, and the relationship between pain relief and objective tumor response was not described.

The Benefits of Safely Achieving Near Normoglycemia from the Perspective of People with Type 2 Diabetes: A Quantitative Survey Study.

Purpose: To understand the perspectives of people with type 2 diabetes (T2D) on safely reaching near normoglycemia, defined as a glycated hemoglobin A1c (HbA1c) value of <6%. HbA1c indicates the average blood sugar level over the past few months.

Patients And Methods: This cross-sectional online quantitative survey of people with T2D asked about the current impacts of T2D, the anticipated benefits of safely achieving near normoglycemia among participants with a current HbA1c ≥6%, or the actual benefits of safely achieving near normoglycemia among participants who had an HbA1c <6%.

Patient Preferences for Multi-Cancer Early Detection (MCED) Screening Tests.

Background: Emerging blood-based multi-cancer early detection (MCED) tests can detect a variety of cancer types across stages with a range of sensitivity, specificity, and ability to predict the origin of the cancer signal. However, little is known about the general US population's preferences for MCED tests.

Objective: To quantify preferences for MCED tests among US adults aged 50-80 years using a discrete choice experiment (DCE).

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.

The Relationship Between Patient-Reported Severity of Hair Loss and Health-Related Quality of Life and Treatment Patterns Among Patients with Alopecia Areata.

Introduction: Alopecia areata (AA) is an autoimmune disease characterized by hair loss. Patients with AA experience a range of social and emotional impacts, and the lack of effective treatments and multiple affected locations can deepen the burden of illness. The objective of the current study was to assess health-related quality of life (HRQL) among patients with AA, and to evaluate the relationship between patient-reported AA severity, HRQL and treatment patterns.

Patient and Physician Preferences for Regimen Attributes for the Treatment of HIV in the United States and Canada.

A long-acting injectable (LAI) antiretroviral therapy (ART) regimen is now available as a treatment option for virologically suppressed adults with HIV-1. This study assessed preference for a LAI regimen using an online survey of virally suppressed people living with HIV (PLWH) and physicians treating HIV in the US and Canada. Preference was elicited in a discrete choice experiment (DCE) with three choice options (switch to a LAI regimen, switch to another daily oral ART regimen, or stay on their current daily oral ART regimen) and four treatment attributes.

German version of the Hypogonadism Impact of Symptoms Questionnaire: Psychometric properties and association with testosterone levels.

In German, the 'Aging Males' Symptom Scale (AMS)' is available for the assessment of symptoms of hypogonadism in men of advanced age. An English questionnaire named 'Hypogonadism Impact of Symptoms' (HIS-Q), applicable also in young males has recently been developed in the United States. We intended to: (1) evaluate the psychometric properties of the German translation of the short form of the HIS-Q (HIS-Q-SF-D); (2) explore the association of individual patient scores with their respective serum testosterone levels.

Development and validation of a novel patient-reported outcome measure in people with episodic migraine and chronic migraine: The Activity Impairment in Migraine Diary.

Objective: To evaluate the content validity and psychometric properties of the Activity Impairment in Migraine Diary (AIM-D).

Background: Measuring treatment effects on migraine impairment requires a psychometrically sound patient-reported outcome (PRO) measure developed consistent with U.S.

Results from Phase I Extension Study Assessing Pexidartinib Treatment in Six Cohorts with Solid Tumors including TGCT, and Abnormal CSF1 Transcripts in TGCT.

Purpose: To assess the response to pexidartinib treatment in six cohorts of adult patients with advanced, incurable solid tumors associated with colony-stimulating factor 1 receptor (CSF1R) and/or KIT proto-oncogene receptor tyrosine kinase activity.

Patients And Methods: From this two-part phase I, multicenter study, pexidartinib, a small-molecule tyrosine kinase inhibitor that targets CSF1R, KIT, and FMS-like tyrosine kinase 3 (FLT3), was evaluated in six adult patient cohorts (part 2, extension) with advanced solid tumors associated with dysregulated CSF1R. Adverse events, pharmacokinetics, and tumor responses were assessed for all patients; patients with tenosynovial giant cell tumor (TGCT) were also evaluated for tumor volume score (TVS) and patient-reported outcomes (PRO).

The Burden of Type 2 Diabetes and the Value of Achieving Near Normoglycemia from the Patient Perspective.

Introduction: Type 2 diabetes (T2D) is extremely burdensome to people with T2D and associated with impaired health-related quality of life. This study explores the impact of T2D and potentially relevant outcomes for new therapies using a unique approach to in-depth qualitative interviews where people with T2D are asked to think about their future with T2D.

Methods: A cross-sectional qualitative interview study among people with T2D from the USA and UK.

Pexidartinib improves physical functioning and stiffness in patients with tenosynovial giant cell tumor: results from the ENLIVEN randomized clinical trial.

Background and purpose - The ENLIVEN trial showed that, after 25 weeks, pexidartinib statistically significantly reduced tumor size more than placebo in patients with symptomatic, advanced tenosynovial giant cell tumor (TGCT) for whom surgery was not recommended. Here, we detail the effect of pexidartinib on patient-reported physical function and stiffness in ENLIVEN.Patients and methods - This was a planned analysis of patient-reported outcome data from ENLIVEN, a double-blinded, randomized phase 3 trial of adults with symptomatic, advanced TGCT treated with pexidartinib or placebo.

Patient-Reported Outcomes and Impact of Type 2 Diabetes: A Cross-Sectional Study in the Kingdom of Saudi Arabia.

Background: The Kingdom of Saudi Arabia (KSA) has the second highest prevalence of type 2 diabetes mellitus (T2DM) in the Middle East. There is a paucity of research on the experiences and treatment preferences of patients with T2DM in KSA. This study explored Saudi patients' health-related quality of life, eating habits, experiences during Ramadan, and preference between two glucagon-like peptide-1 receptor agonist (GLP-1 RA) treatment devices.

Patients' preferences for once-daily oral versus once-weekly injectable diabetes medications: The REVISE study.

Aims: To understand patient preferences for once-daily oral versus once-weekly injectable type 2 diabetes mellitus (T2DM) medication administration profiles, and reasons for their preferences.

Materials And Methods: The REVISE study, a cross-sectional online survey of 600 participants with T2DM (United Kingdom, n = 300; United States, n = 300), elicited general preferences for once-daily oral versus once-weekly injectable diabetes medications, and reasons for the preference. Participants then viewed two videos describing the administration procedures for injectable dulaglutide and oral semaglutide, based on the product instructions for use.

Psychometric properties of a custom Patient-Reported Outcomes Measurement Information System (PROMIS) physical function short form and worst stiffness numeric rating scale in tenosynovial giant cell tumors.

Purpose: The purpose of this study was to evaluate the psychometric properties of the PROMIS-Physical Function (PF) and Worst Stiffness Numeric Rating Scale (NRS) among patients with tenosynovial giant cell tumors (TGCT).

Methods: Measurement properties of the customized lower extremity (LE) and upper extremity (UE) PROMIS-PF scales and Worst Stiffness NRS were assessed using data from the Phase 3 ENLIVEN trial (n = 120). Anchor- and distribution-based analyses were utilized to derive a responder threshold for meaningful change over time.

Qualitative study of patients with venous malformations: symptom experiences and content validity of patient-reported outcome measures.

Purpose: To determine important symptoms and functional effects of venous malformations (VMs) to assess the content validity of commonly used patient-reported outcome (PRO) measures for use with VM patients.

Methods: This cross-sectional, qualitative study involved cognitive interviews with participants with VM aged ≥ 14 years. From February to June 2016, 11 participants (8 female) with a mean (± standard deviation) age of 31 ± 15 years were recruited from three clinical sites.

Patient and physician preferences for attributes of biologic medications for severe asthma.

Objective: Despite the increased availability of biologic treatments indicated for severe asthma, patient and physician preferences for these medications remains largely unknown. The purpose of this study was to understand perceptions of biologic therapies, barriers to care with biologic medications, and preferences for biologic therapy attributes.

Methods: This mixed-methods study involved quantitative surveys and qualitative telephone interviews with patients and physicians from the United States.

Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial.

Background: Tenosynovial giant cell tumour (TGCT), a rare, locally aggressive neoplasm, overexpresses colony-stimulating factor 1 (CSF1). Surgery is standard with no approved systemic therapy. We aimed to evaluate pexidartinib, a CSF1 receptor inhibitor, in patients with TGCT to provide them with a viable systemic treatment option, especially in cases that are not amenable to surgical resection.