Evaluation of symptoms in respiratory syncytial virus infection in adults: psychometric evaluation of the Respiratory Infection Intensity and Impact Questionnaire™ symptom scores.

Background: The Respiratory Infection Intensity and Impact Questionnaire (RiiQ™) is a patient-reported outcome measure designed to assess symptoms and impacts of respiratory syncytial virus (RSV) infection. This study evaluated the construct validity, reliability, and responsiveness of the RiiQ™ Respiratory and Systemic Symptoms Scale scores.

Methods: Prospective data were analyzed from a total of 1795 participants, including from non-hospitalized patients with acute respiratory infection (ARI) and no coinfections enrolled in a Phase 2b RSV vaccine study (RSV-positive: n = 60; RSV-negative: n = 1615), and two observational studies of patients hospitalized with RSV (n = 20; n = 100).

Development and evaluation of the Acromegaly Symptom Diary.

Background: Patient-reported outcome (PRO) measures are important to consider when evaluating treatments, yet there are no PRO measures for patients with acromegaly that have been developed in accordance with US Food and Drug Administration guidance. Acromegaly is a rare, chronic condition caused by hypersecretion of growth hormone. Disease activity is monitored by measurement in serum of growth hormone and insulin-like growth factor-I.

Comparison of anchor-based methods for estimating thresholds of meaningful within-patient change using simulated PROMIS PF 20a data under various joint distribution characteristic conditions.

Purpose: To compare the performance of anchor-based methods for estimating thresholds of meaningful within-patient change (i.e., individual change) of clinical outcome assessments in conditions reflecting data characteristics of small- to medium-sized clinical trials.

The Prader-Willi Syndrome Anxiousness and Distress Behaviors Questionnaire: Development and Psychometric Validation.

Objectives: To facilitate the development of new therapies for Prader-Willi syndrome (PWS), we sought to develop a reliable and valid assessment of anxiousness and distress, common characteristics that have a significant negative impact on individuals with PWS and their families.

Methods: The PWS Anxiousness and Distress Behaviors Questionnaire (PADQ) was developed with extensive input from clinical experts, as well as caregivers of individuals with PWS, who participated in iterative sets of qualitative interviews. The psychometric properties of the PADQ were subsequently demonstrated in a cross-sectional evaluation using data from the Global PWS Registry provided by > 400 caregivers and confirmed using data from a phase 3 clinical trial of an oxytocin analogue (intranasal carbetocin, LV-101).

Validation of the Alopecia Areata Patient Priority Outcomes (AAPPO) Questionnaire in Adults and Adolescents with Alopecia Areata.

Introduction: Individuals with alopecia areata (AA) may experience significant impacts on their health-related quality of life. The novel Alopecia Areata Patient Priority Outcomes (AAPPO) questionnaire has been developed to assess hair loss signs, emotional symptoms, and activity limitations associated with AA. The objective of this study was to evaluate psychometric properties and establish scoring of the AAPPO in adults and adolescents with AA.

Development and Validation Clinician and Patient Reported Photonumeric Scales to Assess Buttocks Cellulite Severity.

Background: The Clinician Reported Photonumeric Cellulite Severity Scale (CR-PCSS) and Patient Reported PCSS (PR-PCSS) are newly developed tools for assessing cellulite severity.

Objective: To report on the reliability, validity, and ability to detect a change in cellulite severity on the buttocks of adult women with the CR-PCSS and PR-PCSS.

Materials And Methods: Content validity of both scales was established through concept elicitation and cognitive interviews.

Diagnostic and clinical experience of patients with pantothenate kinase-associated neurodegeneration.

Background: Pantothenate kinase-associated neurodegeneration (PKAN) is an autosomal recessive neurodegenerative disorder with brain iron accumulation (NBIA).

Objectives: To assess PKAN diagnostic pathway, history, and burden across the spectrum of PKAN severity from patient and/or caregiver perspectives.

Methods: Caregivers of patients (n = 37) and patients themselves (n = 2) were interviewed in a validation study of the PKAN-Activities of Daily Living (ADL) scale.

Developing a preference-based utility scoring algorithm for the Psoriasis Area Severity Index (PASI).

It is challenging to identify health state utilities associated with psoriasis because generic preference-based measures may not capture the impact of dermatological symptoms. The Psoriasis Area Severity Index (PASI) is one of the most commonly used psoriasis rating scales in clinical trials. The purpose of this study was to develop a utility scoring algorithm for the PASI.

A Scale to Assess Activities of Daily Living in Pantothenate Kinase-Associated Neurodegeneration.

Objective: Pantothenate kinase-associated neurodegeneration (PKAN) is an autosomal-recessive, neurodegenerative disorder with a mixed-motor phenotype caused by a defective PanK2 enzyme, for which there are few adequate treatment options. Clinimetrically sound measures of patient-reported outcomes are necessary to facilitate therapeutic development for this debilitating disease. This study's objective was to develop such a scale and assess its clinimetric properties.

Psychometric Evaluation of a Novel Instrument Assessing the Impact of Migraine on Physical Functioning: The Migraine Physical Function Impact Diary.

Objective: The objective of this study was to evaluate the measurement properties of the Migraine Physical Function Impact Diary (MPFID), a novel patient-reported outcome (PRO) measure for assessing the impact of migraine on physical functioning.

Methods: In a prospective, observational study, adults with episodic migraine (EM) or chronic migraine (CM) used an eDiary to complete the MPFID (assessing daily impacts of migraine on physical function) and a headache diary (capturing migraine days, migraine pain intensity, and migraine interference) each day, and other PRO instruments related to migraine. Item-level evaluation, item response theory (IRT), and exploratory factor analysis (EFA) methods were applied to identify domains, select final MPFID items, and develop scoring procedures.

Assessing the impact of multiple sclerosis disease activity and daclizumab HYP treatment on patient-reported outcomes: Results from the SELECT trial.

Background: The SELECT study demonstrated superior effects of daclizumab high-yield process (DAC HYP) to placebo in key endpoints in patients with relapsing and remitting multiple sclerosis (RRMS).

Objective: To assess the impact of DAC HYP and disease activity on health-related quality of life (HRQoL) using data from this study.

Methods: HRQoL was assessed at baseline, 12, 24, and 52 weeks using the Multiple Sclerosis Impact Scale (MSIS-29), the 12-items Short Form Health Survey, and the EuroQoL-5 Dimensions.

Patient-Reported Symptoms over 48 Weeks in a Randomized, Open-Label, Phase 3b Non-inferiority Trial of Adults with HIV Switching to Coformulated Elvitegravir, Cobicistat, Emtricitabine, and Tenofovir DF Versus Continuation of Ritonavir-Boosted Protease Inhibitor with Emtricitabine and Tenofovir DF.

Background: Coformulated elvitegravir, cobicistat, emtricitabine, and tenofovir disoproxil fumarate (EVG/COBI/FTC/TDF; Stribild(®)) is a recommended integrase inhibitor-based regimen in treatment guidelines from the US Department of Health and Human Services and the British HIV Association. The purpose of this analysis was to determine the change in patient-reported symptoms over time among HIV-infected adults who switch to Stribild(®) versus those continuing on a protease inhibitor (PI) with FTC/TDF.

Methods: A secondary analysis was conducted on the STRATEGY-PI study (GS-US-236-0115, ClinicalTrials.

Patient-Reported Symptoms Over 48 Weeks in a Randomized, Open-Label, Phase IIIb Non-Inferiority Trial of Adults with HIV Switching to Co-Formulated Elvitegravir, Cobicistat, Emtricitabine, and Tenofovir DF versus Continuation of Non-Nucleoside Reverse Transcriptase Inhibitor with Emtricitabine and Tenofovir DF.

Background: Co-formulated elvitegravir, cobicistat, emtricitabine, and tenofovir disoproxil fumarate (EVG/COBI/FTC/TDF; Stribild(®)) is a guideline-recommended regimen for HIV treatment-naïve patients and a switch option for virologically suppressed patients.

Objective: The purpose of this analysis was to understand how HIV patients' symptoms change after switching to Stribild(®) versus continuing a regimen consisting of a non-nucleoside reverse transcriptase inhibitor (NNRTI) with emtricitabine and tenofovir disoproxil fumarate.

Methods: A secondary analysis was conducted of the STRATEGY-NNRTI study (GS-US-236-0121), a randomized, open-label, phase IIIb trial of HIV-infected adults who were taking an NNRTI plus FTC/TDF and were randomly assigned (2:1) either to Stribild(®) ('switch') or to continue on their existing regimen ('no-switch').

Adoption of cardiovascular risk reduction guidelines: a cluster-randomized trial.

Background And Objectives: Cardiovascular disease (CVD) and underlying atherosclerosis begin in childhood and are related to CVD risk factors. This study evaluates tools and strategies to enhance adoption of new CVD risk reduction guidelines for children.

Methods: Thirty-two practices, recruited and supported by 2 primary care research networks, were cluster randomized to a multifaceted controlled intervention.

Planning in middle childhood: early predictors and later outcomes.

Data from 1,364 children and families who participated in the National Institute of Child Health and Human Development's Study of Early Child Care and Youth Development were analyzed to track the early correlates and later academic outcomes of planning during middle childhood. Maternal education, through its effect on parenting quality when children were 54 months old, predicts their concurrent performance on sustained attention, inhibition, and short-term verbal memory tests. This performance predicts planning in first grade, which predicts third-grade reading and mathematics attainment, but not the rate of growth in academic skills from first to fifth grades.

Implementation of the NHLBI integrated guidelines for cardiovascular health and risk reduction in children and adolescents: rationale and study design for young hearts, strong starts, a cluster-randomized trial targeting body mass index, blood pressure, and tobacco.

Background: Cardiovascular disease (CVD) and the underlying atherosclerosis begin in childhood, and their presence and intensity are related to known cardiovascular disease risk factors. Attention to risk factor control in childhood has the potential to reduce subsequent risk of CVD.

Objective: The Young Hearts Strong Starts Study was designed to test strategies facilitating adoption of the National, Heart, Lung and Blood Institute supported Integrated Guidelines for Cardiovascular Health and Risk Reduction in Children and Adolescents.

Patterns of developmental change in infants' nighttime sleep awakenings from 6 through 36 months of age.

Nighttime sleep awakenings and infant and family characteristics were measured longitudinally in more than 1,200 infants when the infants were 6, 15, 24, and 36 months old. By 6 months of age, the majority of children slept through the night, awakening their mothers only about once or twice per week. However, not all children followed this predominant developmental pattern.

Longitudinal study of resilience and mental health in Marines leaving military service.

Background: Despite a growing emphasis on psychological outcomes following military combat, few longitudinal studies have examined the combined role of risk and protective factors in the mental health of combat-exposed military personnel. This study characterizes the impact of resilience scale scores and combat exposure on mental health outcomes among Marines after separating from military service, along with intra-individual changes in mental health status.

Methods: Data were collected from longitudinal surveys of 475 active duty Marines attending a random sample of mandatory Transition Assistance Program workshops before leaving the military and responding to follow-up mail or web surveys an average of 6 months after returning to civilian life.

Cost-effectiveness analysis of motivational interviewing with feedback to reduce drinking among a sample of college students.

Objective: This study evaluated the costs and cost-effectiveness of combining motivational interviewing with feedback to address heavy drinking among university freshmen.

Method: Microcosting methods were used in a prospective cost and cost-effectiveness study of a randomized trial of assessment only (AO), motivational interviewing (MI), feedback only (FB), and motivational interviewing with feedback (MIFB) at a large public university in the southeastern United States. Students were recruited and screened into the study during freshman classes based on recent heavy drinking.

Diagnostic validity of two instruments for assessing anxiety and depression among pregnant women in Hyderabad, Pakistan.

The objective of this study was to compare the diagnostic validity of two measures, the Aga Khan University Anxiety and Depression Scale (AKUADS) and the How I Feel scale, for assessing anxiety and depression among pregnant women in Pakistan. The sample included 200 pregnant women in Hyderabad, Pakistan. Using psychiatrist-administered Diagnostic and Statistical Manual of Mental Disorders-fourth edition (DSM-IV) criteria, 71 (36%) of the women were diagnosed with depression or anxiety or both.

Teachers' education, classroom quality, and young children's academic skills: results from seven studies of preschool programs.

In an effort to provide high-quality preschool education, policymakers are increasingly requiring public preschool teachers to have at least a Bachelor's degree, preferably in early childhood education. Seven major studies of early care and education were used to predict classroom quality and children's academic outcomes from the educational attainment and major of teachers of 4-year-olds. The findings indicate largely null or contradictory associations, indicating that policies focused solely on increasing teachers' education will not suffice for improving classroom quality or maximizing children's academic gains.