The Tardive Dyskinesia Impact Scale (TDIS), a novel patient-reported outcome measure in tardive dyskinesia: development and psychometric validation.

Background: Tardive dyskinesia (TD), a movement disorder in which patients experience abnormal involuntary movements, can have profound negative impacts on physical, cognitive, and psychosocial functioning. The Abnormal Involuntary Movement Scale (AIMS), a clinician-rated outcome, is considered the gold standard for evaluating treatment efficacy in TD clinical trials. However, it provides little information about the impacts of uncontrolled movements from a patient perspective and can be cumbersome to administer in clinical settings.

Development, psychometric evaluation, and initial feasibility assessment of a symptom tracker for use by patients with heart failure (HFaST).

Background: This study aimed to develop and provide a psychometric and feasibility pilot evaluation of the Heart Failure (HF) Symptom Tracker (HFaST), a new patient-reported tool designed to facilitate communication between patients and health care providers (HCPs) in routine clinical care. The HFaST enables patients to identify worsening HF symptoms, with a long-term goal of preventing hospitalizations or emergency room visits.

Methods: The HFaST was developed drawing on evidence from the literature, qualitatively with cognitive interviews (12 patient/caregiver and 8 HCPs), and evaluated quantitatively (psychometric, feasibility assessment).

Long-term health-related quality-of-life and symptom response profiles with arformoterol in COPD: results from a 52-week trial.

Background: Symptom severity is the largest factor in determining subjective health in COPD. Symptoms (eg, chronic cough, dyspnea) are associated with decreased health-related quality of life (HRQoL). We evaluated the impact of arformoterol on HRQoL in COPD patients, measured by St George's Respiratory Questionnaire (SGRQ).

Assessing Changes in Chronic Spontaneous/Idiopathic Urticaria: Comparisons of Patient-Reported Outcomes Using Latent Growth Modeling.

Introduction: Assessing the consequences of chronic spontaneous/idiopathic urticaria (CSU) requires the evaluation of health-related quality of life (HRQoL) associated with the severity of CSU signs and symptoms. It is important to understand how signs, symptoms, and HRQoL change over time in CSU. Evidence is lacking on how closely changes in signs and symptoms of CSU are related to changes in HRQoL.

EQ-5D Utilities in Chronic Spontaneous/Idiopathic Urticaria.

Objectives: To obtain utility estimates suitable for use in economic models for chronic spontaneous (idiopathic) urticaria (CSU).

Methods: Patient-level data from three randomized clinical trials-ASTERIA I, ASTERIA II and GLACIAL-were analysed. Health states were derived from the Urticaria Activity Score over 7 days (UAS7); higher scores denote greater activity.

Validation of the SF-36 in patients with endometriosis.

Objectives: Endometriosis presents with significant pain as the most common symptom. Generic health measures can allow comparisons across diseases or populations. However, the Medical Outcomes Study Short Form 36 (SF-36) has not been validated for this disease.

Impact of estradiol valerate/dienogest on work productivity and activities of daily living in women with heavy menstrual bleeding.

Objectives: To quantify the change in work productivity and activities of daily living in North American women with heavy menstrual bleeding (HMB) treated with estradiol valerate/dienogest (E2V/DNG; Qlaira(®)/Natazia(®)) compared to placebo.

Methods: Women in the United States and Canada, aged 20-53 years with an objective diagnosis of HMB and no recognizable anatomical pathology, were treated with E2V/DNG or placebo for seven cycles (196 days). Main outcome measures included work productivity (i.

Identifying differential responders and their characteristics in clinical trials: innovative methods for analyzing longitudinal data.

Objectives: To present a step-by-step example of the examination of heterogeneity within clinical trial data by using a growth mixture modeling (GMM) approach.

Methods: Secondary data from a longitudinal double-blind clinical drug study were used. Patients received enalapril or placebo and were followed for 2 years during the drug component, followed by a 3-year postdrug component.

Measuring patient experiences in Fabry disease: validation of the Fabry-specific Pediatric Health and Pain Questionnaire (FPHPQ).

Introduction: Common symptoms for children with Anderson-Fabry Disease (FD) such as acroparaesthesia and gastrointestinal manifestations can only be objectively assessed in patients using a valid instrument. To date, no such instrument exists.

Methods: A preliminary 40-item measure of symptoms and experience with FD, the Fabry-specific Paediatric Health and Pain Questionnaire (FPHPQ) was developed, but lacked a formal assessment of its measurement properties.

Impact of estradiol-valerate/dienogest on work productivity and activities of daily living in European and Australian women with heavy menstrual bleeding.

Background: The purpose of this study was to quantify the impact of estradiol-valerate/dienogest (E(2)V/DNG; Qlaira(®)/Natazia(®)) on work productivity and activities of daily living in European and Australian women with heavy menstrual bleeding.

Methods: Women aged 18-54 years with a confirmed diagnosis of heavy menstrual bleeding and no recognizable pathology were recruited across nine European countries (the Czech Republic, Finland, Germany, Hungary, The Netherlands, Poland, Sweden, UK, and Ukraine) and Australia. The women were randomized to receive either E(2)V/DNG (n = 149) or placebo (n = 82) for seven treatment cycles (196 days).

EQ-5D™-derived utility values for different levels of migraine severity from a UK sample of migraineurs.

Background: To estimate utility values for different levels of migraine pain severity from a United Kingdom (UK) sample of migraineurs.

Methods: One hundred and six migraineurs completed the EQ-5D to evaluate their health status for mild, moderate and severe levels of migraine pain severity for a recent migraine attack, and for current health defined as health status within seven days post-migraine attack. Statistical tests were used to evaluate differences in mean utility scores by migraine severity.

The Hunter syndrome-functional outcomes for clinical understanding scale (HS-FOCUS) questionnaire: evaluation of measurement properties.

Purpose: This study was to conduct the psychometric validation of the patient and parent versions of the Hunter syndrome-functional outcomes for clinical understanding scale (HS-FOCUS).

Methods: Data collected in a 53-week placebo-controlled multinational trial were used to evaluate item performance and reliability, validity, and ability to detect change of the six HS-FOCUS function domains.

Results: HS-FOCUS was completed by 49 patients above 12 years old and 84 parents.

Application of latent growth and growth mixture modeling to identify and characterize differential responders to treatment for COPD.

Objective: To explore the utility of applying growth mixture models (GMMs) in secondary analyses of clinical trials to identify sources of variability in data reported by patients with COPD.

Methods: Analyses were performed on data from two 6-month clinical trials comparing indacaterol and open-label tiotropium or blinded salmeterol and the first six months of a 12-month trial comparing indacaterol and blinded formoterol. Latent growth model (LGM) analyses were conducted to explore the response of the SGRQ Symptoms score from baseline to six months and GMM analyses were evaluated as a method to identify latent classes of differential responders.

Development of a frequent heartburn index.

Purpose: The aim of this study is to develop and validate a brief instrument for the measurement of overall psychosocial impact of frequent heartburn (heartburn experienced 2+ times weekly) in the general U.S. population, yielding a single, composite score.

The congestion quantifier five-item test for nasal congestion: refinement of the congestion quantifier seven-item test.

Background: Patients report that nasal congestion is the most bothersome symptom of allergic rhinitis (AR). Recently, a short, patient-reported congestion screener questionnaire, the Congestion Quantifier Seven-Item Test (CQ7), was developed to identify a level of congestion that may warrant patients seeking evaluation and possible treatment. We explored further item reduction of the CQ7 and examined the psychometric properties of this reduced set of items in a 15-day study of patients with confirmed AR or self-identified and clinician-confirmed congestion.

Use of latent growth curve models for assessing the effects of darbepoetin alfa on hemoglobin and fatigue.

Background: The relationship between darbepoetin alfa and fatigue in chemotherapy-induced anemia (CIA) patients is complex because of patients receiving transfusions and the mediating effect of hemoglobin. Latent growth models (LGMs) were used to examine simultaneously relationships among drug exposure, fatigue outcomes, covariates, and mediating factors.

Methods: Data from four CIA studies (AMG 20010145: small cell lung cancer, n=547; AMG 980297: lung cancer, n=288; AMG 20000161: lymphoproliferative malignancies, n=339; AMG 20030232: non-myeloid malignancies, n=320) were analyzed separately.

Use of, satisfaction with, and willingness to switch prescription and over-the-counter treatments for chronic urticaria: an online survey.

Background: : Chronic urticaria (hives) affects a sizeable number of people worldwide, perhaps as much as 3%. It is often accompanied by angioedema. The negative effects of urticaria and angioedema, and any adverse effects of treatment, can result in significant patient burden.

Relative strength of relationships of nasal congestion and ocular symptoms with sleep, mood and productivity.

Background: Nasal congestion associated with allergic rhinitis has been shown to be the most bothersome symptom. Ocular symptoms may be troublesome to patients as well.

Objective: To estimate the relative strength of relationships of nasal congestion and ocular symptoms associated with allergic rhinitis with patient-reported outcomes of sleep quality; practical problems; somnolence; impairment at work, class, activities; and mood.

Optimal recall periods for patient-reported outcomes: challenges and potential solutions.

Objectives: As the role and importance of patient-reported outcomes (PROs) increase, the validity and reliability of PRO measures come under greater scientific and regulatory scrutiny. One key issue is selecting the 'most appropriate' recall period for capturing PROs in clinical trials. This paper draws on survey research, health-specific literature, and results from clinical trials to summarize factors that can influence recall and provide guidance on selecting an optimal recall period.

Using the congestion quantifier seven-item test to assess change in patient symptoms and their impact.

Generally, nasal congestion is reported to be the most troublesome symptom of allergic rhinitis and has been shown to have negative effects on sleep, productivity, work, and school performance. A new patient-reported outcome (PRO) tool called the Congestion Quantifier Seven-Item Test (CQ7) was developed and shown to be a reliable and valid screening tool for nasal congestion. The purpose of this study was to assess the CQ7 as a tool for clinical use that can monitor changes in nasal congestion or determine if patients are showing meaningful improvement in symptoms.

Analyzing growth and change: latent variable growth curve modeling with an application to clinical trials.

Objective: Typical methods of analyzing data from clinical trials have shortcomings, notably comparisons of group means, use of change scores from pre- and post-treatment assessments, ignoring intervening assessments, and focusing on direct effects of treatment. A comparison of group means disregards the likelihood that individuals have different trajectories of change. Moreover, change scores ignore intervening assessments that may provide useful information about change.

Development and validation of the Congestion Quantifier seven-item test (CQ7): a screening tool for nasal congestion.

Objective: This study aimed to develop and validate a short, simple, patient-completed instrument for identifying patients with congestion in a 15-day study. Allergic rhinitis (AR) is the most common allergic condition worldwide, with congestion as one of the most salient symptoms. Nevertheless, there is no short screening tool designed specifically to identify congestion that can help patients make decisions about seeking treatment.

Measuring functional performance in patients with COPD: a discussion of patient-reported outcome measures.

Background: Chronic obstructive pulmonary disease (COPD) is a chronic condition and a major public health concern. Moreover, its prevalence is increasing. COPD commonly affects patient performance of daily activities that people perform in order to meet basic needs, fulfill usual roles, and maintain their health and well-being.

Relationship of nasal congestion with sleep, mood, and productivity.

Objective: To explore the impact of nasal congestion alone relative to a full set of allergic rhinitis (AR) symptoms on sleep, fatigue, daytime somnolence, and work and school productivity in a 15-day prospective, naturalistic study.

Research Design And Methods: Patients (N = 404) received a clinical exam to confirm congestion and assess its possible causes, including confirmed allergic rhinitis. They completed a battery of patient-reported outcomes (PROs) that assess the impact of nasal congestion and morning AR symptoms on patients' reports of sleep, daytime sleepiness, fatigue, and work, school, and activity impairment.

Longitudinal analysis of the reciprocal effects of self-assessed global health and depressive symptoms.

This study examined whether a reciprocal relationship exists between measures of self-assessed global health and depressive symptoms, net of covariates that included chronic illness, functional disability, education, income, gender, race, and age. Analyses of five waves of data from the Rand version of the Health and Retirement Survey (N=7,475), using an autoregressive, cross-lagged panel design, indicated that self-assessed overall health had a modest but statistically significant and consistent effect on depressive symptoms. In contrast, the level of depressive symptoms had a statistically nonsignificant effect on self-assessed health.