The Burden of Pruritus Associated With CKD: A Mixed Methods Analysis Among Patients Undergoing Dialysis.

Rationale & Objective: Despite its prevalence and distress to patients, chronic kidney disease-associated pruritus (CKD-aP) is poorly characterized, which may contribute to the condition's underdiagnosis and inadequate management. This study aimed to understand the symptom experience of patients with CKD-aP and the extent to which pruritus impacts their lives.

Study Design: Mixed methods study including one-on-one qualitative interviews and completion of the Skindex-10 Questionnaire (measuring itch-related quality of life).

Psychometric validation and meaningful change thresholds of the Worst Itching Intensity Numerical Rating Scale for assessing itch in patients with chronic kidney disease-associated pruritus.

Background: Chronic kidney disease-associated pruritus (CKD-aP) is characterized by persistent itch that often leads to substantially impaired quality of life. The Worst Itching Intensity Numerical Rating Scale (WI-NRS) is a single-item patient-reported outcome measure in which patients indicate the intensity of the worst itching they experienced over the past 24 h. Here, we evaluated the content validity and psychometric properties of the WI-NRS and confirmed the threshold of meaningful change in hemodialysis patients with moderate-to-severe CKD-aP.

Validation of the shortened Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS).

Background: The rare disease, Hunter Syndrome (mucopolysaccharidosis type II; MPS II), characterized by iduronate-2-sulfatase deficiency, has heterogeneous symptoms often including cognitive impairment (CI). To evaluate physical functioning and daily activity limitations of patients with MPS II, the multidomain shortened Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) questionnaire was previously developed and preliminarily validated. Here we gather data in a dedicated prospective longitudinal observational study using direct responses to the shortened instrument and assess its psychometric properties further.

Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report.

Background: Rare diseases (RDs) affect a small number of people within a population. About 5000 to 8000 distinct RDs have been identified, with an estimated 6% to 8% of people worldwide suffering from an RD. Approximately 75% of RDs affect children.

Psychometric evaluation of the 10-item Short Opiate Withdrawal Scale-Gossop (SOWS-Gossop) in patients undergoing opioid detoxification.

Introduction: The Short Opiate Withdrawal Scale (SOWS)-Gossop is a 10-item questionnaire developed to evaluate opioid withdrawal symptom severity. The scale was derived from the original 32-item Opiate Withdrawal Scale in order to reduce redundancy while providing an equally sensitive measure of opioid withdrawal symptom severity appropriate for research and clinical practice. The objective of this study was to examine the psychometric properties and provide score interpretation guidelines for the SOWS-Gossop 10-item version.

What do we know about asthma triggers? a review of the literature.

Objective: For patients with asthma, exacerbations and poor control can result from exposure to environmental triggers, such as allergens and air particulates. This study reviewed the international literature to determine whether a global checklist of common asthma triggers might be feasible for use as a research or management tool in clinical practice.

Methods: Literature published from 2002 to 2012 was identified through PubMed and EMBASE using the following search terms: asthma, asthma triggers, prevalence, among others.

The humanistic burden of hereditary angioedema: Impact on health-related quality of life, productivity, and depression.

Hereditary angioedema (HAE) is a rare, autosomal dominant disorder characterized by recurrent acute attacks of swelling of the larynx, abdomen, and periphery. This study was designed to assess the humanistic burden of illness associated with HAE. HAE burden was assessed via a web-based survey of patients that solicited information on attack characterization, treatment, side effects, pain, and functional and emotional burden of disease management.

Psychometric evaluation of the Medication Satisfaction Questionnaire (MSQ) to assess satisfaction with antipsychotic medication among schizophrenia patients.

The Medication Satisfaction Questionnaire (MSQ) is a single-item questionnaire which evaluates satisfaction with antipsychotic medication in schizophrenia patients. This study evaluated the reliability and validity for its use in research and clinical settings. Data pooled across treatment groups (control vs.

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.

Measurement of non-restorative sleep in insomnia: A review of the literature.

Objective: Non-restorative sleep (NRS) is a core symptom of insomnia, typically defined as a subjective feeling of being unrefreshed upon awakening. NRS symptoms have been less consistently studied than other symptoms of insomnia, and there is no consensus regarding measurement of NRS. Given its subjective nature, patient-reported outcome (PRO) instruments are important for evaluating NRS symptom severity and treatment-related changes.

Psychometric validation of two patient-reported outcome measures to assess symptom severity and changes in symptoms in hereditary angioedema.

Purpose: Hereditary angioedema (HAE) is a rare disorder characterized by highly variable, acute attacks of swelling at various anatomical locations. Clinical measures do not adequately assess the diversity of symptoms characteristic of an attack. Two disease-specific, patient-reported outcome measures were developed to comprehensively capture symptom severity and change: the Treatment Outcome Score (TOS) and the Mean Symptom Complex Severity (MSCS) score.

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.

Reliability, validity, and responsiveness of the daily sleep interference scale among diabetic peripheral neuropathy and postherpetic neuralgia patients.

To evaluate the psychometric characteristics of the Daily Sleep Interference Scale (DSIS) in patients with painful diabetic peripheral neuropathy (DPN) or postherpetic neuralgia (PHN), a post hoc secondary analysis of data from eight randomized clinical trials (four DPN and four PHN) was performed. Data were pooled within patient populations when assessment weeks were the same. The DSIS was completed by 1,124 DPN and 1,034 PHN patients.