Psychometric Properties and Meaningful Change Thresholds of the Vitiligo Area Scoring Index.

Importance: Defining meaningful improvement using the Total Vitiligo Area Scoring Index (T-VASI) and the Facial VASI (F-VASI) aids interpretation of findings from clinical trials evaluating vitiligo treatments; however, clear and clinically meaningful thresholds have not yet been established.

Objective: To assess concept validity and measurement performance of the T-VASI and F-VASI in patients with nonsegmental vitiligo and to identify meaningful change thresholds.

Design, Settings, And Participants: This mixed-methods study consisted of a secondary analysis of a phase 2 multicenter double-blind dose-ranging randomized clinical trial and embedded qualitative interviews conducted at 35 sites in Canada, France, Japan, and the US.

Qualitative assessment of the suitability of the Dysphagia Symptom Questionnaire to monitor dysphagia in children aged 7-10 years with eosinophilic esophagitis.

Background: The Dysphagia Symptom Questionnaire (DSQ) is a patient-reported outcome measure that assesses the frequency and severity of dysphagia in patients with eosinophilic esophagitis (EoE); however, it has only been validated for use in patients with EoE aged 11-40 years. This study examined the content validity of the DSQ and its usability on an electronic handheld device in children aged 7-10 years with EoE.

Methods: In this qualitative, observational cohort study, participants were recruited to partake in two rounds of interviews.

Qualitative Interviews to Assess the Content Validity and Usability of the Electronic Raynaud Diary in Patients with Systemic Sclerosis.

Objective: To better understand the symptoms and impacts of Raynaud phenomenon (RP) in patients with systemic sclerosis (SSc) and to evaluate the content validity and usability of a new electronic patient-reported outcome (PRO) measure for RP: the Raynaud Diary.

Methods: The Raynaud Diary was developed as a daily eDiary for assessing the number and duration of symptomatic Raynaud attacks; worst pain, numbness, tingling, and discomfort in the fingers; and overall disease severity, captured using the Raynaud's Condition Score. The Raynaud Diary was debriefed in two waves of qualitative interviews with adults with self-reported RP secondary to SSc.

Conversations and Reactions Around Severe Hypoglycemia (CRASH) Study: Results From People With Diabetes and Caregivers in the United States.

A survey was conducted in eight countries to examine conversations around, and experiences and treatments during, severe hypoglycemia among people with diabetes and caregivers of people with diabetes. This article reports a subgroup analysis from the United States involving 219 people with diabetes and 210 caregivers. Most respondents (79.

Conversations and Reactions Around Severe Hypoglycaemia (CRASH): Spanish results of a global survey of people with type 1 diabetes or insulin-treated type 2 diabetes and caregivers.

Introduction: Information on experience/management of severe hypoglycaemic events (SHEs) among people with insulin-treated diabetes (PWD) and caregivers (CGs) providing care to PWD was sought.

Materials And Methods: An online cross-sectional survey was conducted in eight countries.

Inclusion Criteria: PWD (aged≥18 years; self-reported type 1 [T1D] or insulin-treated type 2 [T2D] diabetes; experienced ≥1 SHE [hypoglycaemia requiring external assistance] in past 3 years); CGs (layperson aged ≥18 years; caring for PWD meeting all criteria above except age [≥4 years]).

Impact and burden of acid sphingomyelinase deficiency from a patient and caregiver perspective.

Acid sphingomyelinase deficiency (ASMD), historically known as Niemann-Pick disease (NPD) types A, A/B, and B, is a rare, progressive, potentially fatal lysosomal storage disease with a spectrum of phenotypes. Little is known about how ASMD symptoms affect the lives of patients and their caregivers. In a cross-sectional qualitative study conducted in the US and UK, and in collaboration with the National Niemann-Pick Disease Foundation (US) and Niemann-Pick UK, we investigated the symptom experience of patients with ASMD types B and A/B and explored how the disease impacts their and their caregivers' lives.

Measuring health-related quality of life in patients with rare disease.

Background: There has been a growing emphasis on health-related quality of life (HRQoL) as an important outcome in rare disease drug development, although its assessment may be useful outside the drug development context, including in clinical applications or natural history studies. Central to assessing quality of life in health research is utilizing outcome measures that capture symptoms and impacts of the disease and treatment that are important and relevant to patients. Identifying and implementing valid and reliable tools to measure HRQoL in rare diseases poses unique challenges that often require creative solutions.

The Patient Experience of Premenopausal Women Treated with Bremelanotide for Hypoactive Sexual Desire Disorder: RECONNECT Exit Study Results.

Hypoactive sexual desire disorder (HSDD) has a significant negative impact on women's overall health and relationships with their partners. Primary analyses from the RECONNECT clinical trials demonstrated statistically significant and clinically meaningful improvements in sexual desire and related distress with bremelanotide relative to placebo in premenopausal women with HSDD. Exit surveys and patient interviews were conducted to evaluate the impact of HSDD and bremelanotide treatment from the patient's perspective.

Psychometric assessment of the PROMIS Fatigue Short Form 6a in women with moderate-to-severe endometriosis-associated pain.

Background: Endometriosis is a common problem in women of reproductive age and has impacts on health-related quality of life and productivity. Fatigue is an important part of the burden of endometriosis, it is not often included as an endpoint in clinical trials.

Objectives: The study assessed the psychometric properties of the PROMIS Fatigue Short Form 6a in women with moderate-to-severe endometriosis-associated pain.

Responsiveness and thresholds for clinically meaningful changes in worst pain numerical rating scale for dysmenorrhea and nonmenstrual pelvic pain in women with moderate to severe endometriosis.

Objective: To evaluate the utility, responsiveness, and thresholds for clinically meaningful change of a numerical rating scale for worst pain associated with dysmenorrhea (NRS-DYS) and nonmenstrual pelvic pain (NRS-NMPP) in women with moderate to severe endometriosis-associated pain.

Design: Analysis of data from two phase III randomized clinical trials (EM-I [NCT01620528] and EM-II [NCT01931670]).

Setting: Not applicable.

Clinically Meaningful Reduction in Dyspareunia Is Associated With Significant Improvements in Health-Related Quality of Life Among Women With Moderate to Severe Pain Associated With Endometriosis: A Pooled Analysis of Two Phase III Trials of Elagolix.

Background: Dyspareunia experienced by women diagnosed with endometriosis is associated with a decreased health-related quality of life (HRQoL).

Aim: We evaluated the relationship of clinically meaningful improvements in dyspareunia with HRQoL changes among women with endometriosis.

Methods: This was a post hoc analysis of pooled data from the phase III ELARIS-I and ELARIS-II clinical trials.

Documenting the Journey of Patients with Eosinophilic Esophagitis and the Impact of the Disease on Patients and their Caregivers: A Cross-sectional, Qualitative Research Study.

Introduction: Eosinophilic esophagitis (EoE) is a chronic, immune-mediated esophageal disease for which there is currently no approved treatment in the USA and only one approved therapy in Europe. EoE can significantly affect the lives of patients and caregivers; however, little has been published about patients' experiences from symptom onset to diagnosis and treatment.

Methods: This was a cross-sectional, qualitative research study.

Evaluating functional disability in clinical trials of lisdexamfetamine dimesylate in binge eating disorder using the Sheehan Disability Scale.

Objectives: This study examined Sheehan Disability Scale (SDS) performance in binge eating disorder (BED) and explored relationships between SDS and BED outcomes using data from three placebo-controlled lisdexamfetamine (LDX) studies (two short-term, dose-optimized studies and one double-blind, randomized-withdrawal study) in adults with Diagnostic and Statistical Manual of Mental Disorders, fourth edition, text revision (DSM-IV-TR)-defined BED.

Methods: Analyses evaluated the psychometric properties of the SDS.

Results: Confirmatory factor analysis supported a unidimensional total score in the short-term studies, with internal consistency (Cronbach's α) being 0.

Health-Related Quality of Life Improvements in Patients With Endometriosis Treated With Elagolix.

Objective: To evaluate the effects of elagolix on clinically meaningful improvements in health-related quality of life (HRQOL) measured by the EHP-30 (Endometriosis Health Profile-30).

Methods: Data from two phase III trials of elagolix for moderate to severe pain associated with endometriosis were pooled and analyzed as three groups: placebo, elagolix 150 mg once daily, or elagolix 200 mg twice daily. Patients were administered the EHP-30 questionnaire at baseline, and at months 1, 3, and 6 of treatment.

Responsiveness Evaluation and Recommendation for Responder Thresholds for Endometriosis Health Profile-30: Analysis of Two Phase III Clinical Trials.

To evaluate the responsiveness of the Endometriosis Health Profile-30 (EHP-30) and ascertain score changes that are indicative of response to treatment. A analysis of two Phase III, double-blind, placebo-controlled, randomized clinical trials among women with moderate-to-severe endometriosis-associated pain (Elaris Endometriosis I and II [EM-I and EM-II]). EHP-30 core items and sexual relationship module were administered at day 1, month 3 (M3), and month 6 (M6) to monitor patient-reported impacts of endometriosis-related pain.

Achieving clinically meaningful response in endometriosis pain symptoms is associated with improvements in health-related quality of life and work productivity: analysis of 2 phase III clinical trials.

Background: Endometriosis-related pain symptoms have a negative impact on health-related quality of life and productivity. In fact, as endometriosis-related symptom severity and the number of symptoms experienced increases, health-related quality of life decreases. Dysmenorrhea and nonmenstrual pelvic pain are prominent symptoms experienced by women with endometriosis and were shown to have improved with the oral, nonpeptide gonadotropin-releasing hormone antagonist, elagolix.

Psychometric assessment of the health-related productivity questionnaire (HRPQ) among women with endometriosis.

: Endometriosis impacts health-related quality of life. The objective was to assess the validity and responsiveness of the Health-Related Productivity Questionnaire, version 2 (HRPQ). : Outcome measures (Endometriosis Health Profile-30; pain scales; and global assessment) from Elaris Endometriosis I and II clinical trials (EM-I, EM-II) were used.

Impact of elagolix on work loss due to endometriosis-associated pain: estimates based on the results of two phase III clinical trials.

Objective: To estimate the impact of elagolix on work loss due to endometriosis-associated pain.

Design: Post hoc analysis of data from the Elaris I and II clinical trials.

Setting: Not applicable.

Impact of Respiratory Syncytial Virus-Confirmed Hospitalizations on Caregivers of US Preterm Infants.

This study assessed the impact of respiratory syncytial virus-confirmed hospitalizations (RSVH) on caregivers of high-risk preterm infants. Caregivers for infants born at 29 to 35 weeks' gestational age and hospitalized for confirmed RSV disease responded to measures of self-rated and perceived infant stress (1-7; 7 = very stressful), perceived infant health (0-100; 100 = best imaginable health), and productivity impairment. Data were collected at hospital discharge through 1 month post-discharge.

Experience of Symptoms and Disease Impact in Patients with Adenomyosis.

Background: Adenomyosis is a poorly understood, benign disease of the uterus.

Objective: In this study, patient interviews were conducted to characterize the symptoms and impact of adenomyosis.

Methods: This was a cross-sectional study in which women with adenomyosis were recruited from five US clinics and a health-related social network forum.

Qualitative interviews to identify burden of illness, impacts and costs associated with surgical site infections.

Aim: To gather qualitative data from patients on the burden, impacts and costs of surgical site infections (SSI) requiring second surgeries.

Patients & Methods: 15 adults with SSIs from spinal (n = 4), knee replacement (n = 3) or hip replacement (n = 8) surgery participated in a focus group or individual interview. Patients completed the PROMIS Physical Functioning (PF) Short Form 10A (PROMIS-PF).

Evaluating functional outcomes in adolescents with attention-deficit/hyperactivity disorder: development and initial testing of a self-report instrument.

Background: Engaging adolescents in decisions about their health may enhance their compliance with treatment and result in better health outcomes. Treatment outcomes in attention-deficit/hyperactivity disorder (ADHD) are rarely evaluated from the adolescents' point of view. There is also concern that adolescents with ADHD may not have insight about the impacts of their disease.

Health-related quality of life outcomes of docetaxel/carboplatin combination therapy vs. sequential therapy with docetaxel then carboplatin in patients with relapsed, platinum-sensitive ovarian cancer: results from a randomized clinical trial.

Objectives: A phase II clinical trial compared docetaxel in combination with carboplatin to sequential single agent docetaxel followed by carboplatin for treatment of recurrent platinum-sensitive ovarian, peritoneal, or tubal cancer. This manuscript reports prospectively collected health-related quality of life (HRQL).

Methods: Participants were randomized to either weekly docetaxel 30 mg/m(2)/days 1 and 8 and carboplatin AUC 6/day 1 every 3 weeks (cDC) or docetaxel 30 mg/m(2)/days 1 and 8, repeated every 3 weeks for 6 cycles followed by carboplatin AUC 6/day 1 every 3 weeks for 6 cycles or until disease progression (sDC).

Cost-effectiveness of combination versus sequential docetaxel and carboplatin for the treatment of platinum-sensitive, recurrent ovarian cancer.

Background: In a randomized controlled trial (RCT) of patients with recurrent, platinum-sensitive ovarian cancer, the combination weekly docetaxel and carboplatin was associated a with progression-free survival (PFS) of 13.7 months compared with 8.4 months for sequential, single-agent docetaxel followed by carboplatin.

Development and validation of the living with chronic obstructive pulmonary disease questionnaire.

Purpose: Available patient-reported outcome (PRO) measures for chronic obstructive pulmonary disease (COPD) focus primarily on impairment (symptoms) and activities (functioning). The purpose of the study was to develop a patient-based PRO measure for COPD that captures the overall everyday impact of living with COPD from the patient's perspective.

Methods: LCOPD items (Living with COPD Questionnaire) were generated from qualitative interviews in the U.