Psychometric validation of a patient-reported single-item assessment of 'Good Day Bad Day' in a neurogenic orthostatic hypotension population treated with droxidopa.

BACKGROUND: Symptoms of neurogenic orthostatic hypotension (nOH), including lightheadedness/dizziness, presyncope, syncope, and falls, can lead to impaired functional ability and reduced quality of life. Because the severity and frequency of nOH symptoms fluctuate, it may be difficult for patients to accurately quantify the effect of symptoms on their daily lives using available outcome measures. A new single-item instrument, the 'Good Day Bad Day,' was developed, and its psychometric validity was assessed in patients with nOH. METHODS: Data from a 6-month, prospective, observational cohort study of patients with nOH who were newly initiating droxidopa treatment were used. Patients were asked to quantify the number of good and bad days in the previous 7 days and responded to other validated patient-reported outcomes instruments. The concurrent and discriminant validities and the stability of the Good Day Bad Day instrument were assessed. RESULTS: A total of 153 patients were included in the analysis (mean [SD] age, 62.3 [17] years). Change in the number of good days moderately correlated with improvements in other patient-reported outcomes (rho value range, -0.38 to -0.61). When data were examined categorically (low vs high symptom severity), the mean number of good days was higher in subgroups representing low symptom severity across measures at 1, 3, and 6 months (all P ≤ 0.01). CONCLUSIONS: The Good Day Bad Day instrument provided good discrimination at baseline and over time and may aid in assessment of the effects of nOH symptoms on patients.

Second opinions, multiple physician appointments, and overlapping prescriptions in the paediatric population: A systematic literature review.

OBJECTIVES: Doctor shopping, double doctoring, and overlapping prescriptions are often used as synonyms for multiple physician appointments in the same disease episode. Such behaviours translate into poor patient satisfaction and patient-doctor communication as well as abuse or misuse of drugs, increasing health care costs and resulting in negative health consequences. This systematic review of the literature was conducted to identify factors that drive doctor-shopping behaviour in children's caregivers. METHODS: The search was conducted in PubMed and grey literature and was based on the following search terms: included doctor or physician shopping, drug seeking, double doctoring, children, and combinations of those. Overall, 500 records were identified, of which 11 were selected for qualitative synthesis. Data extracted considered definitions of doctor shopping, co-morbidities, and target population characteristics. RESULTS: Definitions of doctor shopping were inconsistent. The frequency of doctor shopping was high for acute illnesses and ranged from 53% in children with a fever in Hong Kong to 18% at an emergency department in Canada. The incidence of this phenomenon was low when taking into account addictive drugs and was rated at 0.02% to 0.3% in the full paediatric population. This phenomenon was more prevalent in children younger than 1 year, in children with attention-deficit hyperactivity disorder (ADHD) and co-morbid psychiatric conditions, and in those whose caregivers themselves had psychiatric conditions. It was more frequent in cases with an acute disease (eg, fever, gastroenteritis, and urinary tract infection) than a chronic disease (eg, asthma). CONCLUSIONS: The knowledge about doctor shopping by children's caregivers is limited, despite that this is a frequent behaviour. There is a need for further research covering a broader range of diseases. The causes and consequences of doctor shopping should be sought as well to investigate its relation to health care regulations and possibility to reduce unnecessary medical resource utilization.

Measuring the health-related quality of life in young children: how far have we come?

The importance of understanding the impact of disease and treatment on children's Health-Related Quality of Life (HRQoL) has given rise to an increasing use of child self-report and observer or proxy instruments. In this article, we review the status quo and challenges of HRQoL measurement specific to children under five. A number of HRQoL questionnaires exist for use with children and/or proxies, and both guidelines and reviews have been published on paediatric HRQoL. However, none address the challenges of measurement for children under five, for whom proxy measures should be used. In reality, there is significant heterogeneity in the cut-off age for self-report questionnaires. Recommendations are that proxies should be used for observable concepts, but not for concepts that require interpretation. Some research has been undertaken on dimensions/concepts in paediatric HRQoL questionnaires. However, no HRQoL models have been developed specifically for children, and heterogeneity in questionnaire dimensions underlines that there is no clear grasp of what HRQoL means in paediatric populations. There is a need to carry out research in order to develop theoretical models of HRQoL that are specific to children at different developmental stages, in order to evaluate and support new and existing measures for paediatric HRQoL and their use in clinical practice as well as clinical trials.

A systematic literature review identifying associations between outcomes and quality of life (QoL) or healthcare resource utilization (HCRU) in schizophrenia.

AIMS: There have been no systematic literature reviews (SLRs) evaluating the identified association between outcomes (e.g. clinical, functional, adherence, societal burden) and Quality-of-Life (QoL) or Healthcare Resource Utilization (HCRU) in schizophrenia. The objective of this study was to conduct a SLR of published data on the relationship between outcomes and QoL or HCRU. MATERIALS AND METHODS: Electronic searches were conducted in Embase and Medline, for articles which reported on the association between outcomes and QoL or HCRU. Inclusion and exclusion criteria were applied to identify the most relevant articles and studies and extract their data. A summary table was developed to illustrate the strength of associations, based on p-values and correlations. RESULTS: One thousand and two abstracts were retrieved; five duplicates were excluded; 997 abstracts were screened and 95 references were retained for full-text screening. Thrirty-one references were included in the review. The most commonly used questionnaire, which also demonstrated the strongest associations (defined as a p < 0.0001 and/or correlation ±0.70), was the Positive and Negative Syndrome Scale (PANSS) associated with HCRU and QoL (the SF-36, the Schizophrenia Quality-of-Life questionnaire [S-QOL-18], the Quality-of-Life Scale [QLS]). Other robust correlations included the Clinical Global Impression-Severity (CGI-S) with QoL (EQ5D), relapse with HCRU, and remission with QoL (EQ5D). Lastly, functioning (Work Rehabilitation Questionnaire [WORQ] and Personal and Social Performance Scale [PSP]) was found to be associated to QoL (QLS and Subjective Well-being under Neuroleptics Questionnaire [SWN]). LIMITATIONS: This study included data from an 11-year period, and other instruments less frequently used may be further investigated. CONCLUSIONS: The evidence suggests that the PANSS is the clinical outcome that currently provides the most frequent and systematic associations with HCRU and QoL endpoints in schizophrenia.

Patient preference for chronic obstructive pulmonary disease (COPD) treatment inhalers: a discrete choice experiment in France.

OBJECTIVES: Understanding inhaler preferences may contribute to improving adherence in COPD patients and improving long-term outcomes. This study aims to identify and quantify preferences for convenience-related inhaler attributes in French moderate-to-severe COPD patients, with discrete choice experiment (DCE) methodology. METHODS: Attributes were defined from a literature search, clinician and patient interviews: shape, dose insertion, dose preparation, dose release, dose confirmation, dose counter and reusability. An online DCE was conducted in respondents with self-reported COPD stage 2-4 recruited through a panel. The study questionnaire included twelve choice scenarios per respondent and questions on patient characteristics, treatment and disease severity. Statistical analyses used a mixed logit regression model with random effects. Utility scores were estimated for four types of inhalers: Inhaler A - soft mist inhaler; Inhaler B - reusable soft mist inhaler; Inhaler C - multi-dose dry powder inhaler; and Inhaler D - single dose dry powder inhaler. RESULTS: The study was completed by 153 patients (50 females); respondents were 50.4 years old on average; 13 different inhaler devices were reported. The most preferred inhaler is L-shaped, has dose preparation with capsule insertion and a dose counter, and is reusable. Inhaler profiles A and B had the highest utilities (mean of 1.2533 and 0.9578 respectively) compared to inhaler C (0.6315) and D (0.2200). CONCLUSIONS: This study showed statistically significant results that the strongest drivers of preference in French users of inhalation devices for COPD are shape, dose counter and reusability. Convenience-related characteristics are important to patients and should be taken into account by clinicians prescribing these devices.

Review of patient-reported outcome instruments measuring health-related quality of life and satisfaction in patients with type 2 diabetes treated with oral therapy.

OBJECTIVE: Treatments and their mode of administration may represent a burden for patients and can therefore impact their health-related quality of life (HRQL) or treatment/health satisfaction. Patients with type 2 diabetes mellitus (T2DM) can be treated with oral hypoglycemic agents (OHAs), injectable medications (such as insulin), or a combination of agents. This review aimed to identify patient-reported outcome (PRO) instruments measuring HRQL and/or satisfaction that could differentiate between oral medications based on medication related attributes such as efficacy, tolerability, weight loss, dosing frequency and pill burden. RESEARCH DESIGN AND METHODS: Medline, Embase, PsycINFO, Cochrane Library and the Patient-Reported Outcome and Quality of Life Questionnaires (PROQOLID) biomedical databases were searched to identify instruments and document their development methodology, content and psychometric properties (i.e. validity, reliability), responsiveness and ability to detect changes between treatments. RESULTS: Nineteen instruments were retained based on their potential to differentiate between OHAs. Ten instruments assessed HRQL, amongst which the Audit of Diabetes Dependent Quality of Life, Diabetes 39, Diabetes Health Profile and Impact of Weight on Quality of Life displayed good psychometric properties in T2DM populations and comprehensive HRQL content. Nine instruments assessed satisfaction. Both the Oral Hypoglycemic Agent Questionnaire (OHAQ) and Diabetes Medication Satisfaction (DiabMedSat) Questionnaire have highly relevant content regarding drug attributes. The OHAQ is specific to oral treatment and the DiabMedSat includes HRQL items. The Diabetes Treatment Satisfaction Questionnaire is a standard instrument that is extensively used and provides conclusive results in studies of patients with T2DM. CONCLUSIONS: Very few of the existing PRO instruments are specific to OHAs. Despite satisfaction instruments being recommended to differentiate between OHAs in studies of T2DM based on medication attributes, we find that none of the existing instruments appear to be useful in detecting differences between treatments, therefore limiting their use in clinical and observational research.

A literature review to explore the link between treatment satisfaction and adherence, compliance, and persistence.

PURPOSE: To explore the published evidence on the link between treatment satisfaction and patients' compliance, adherence, and/or persistence. METHODS: Articles published from January 2005 to November 2010 assessing compliance, adherence, or persistence and treatment satisfaction were identified through literature searches in Medline, Embase, and PsycInfo. Abstracts were reviewed by two independent researchers who selected articles for inclusion. The main attributes of each study examining the link between satisfaction and adherence, compliance, or persistence were summarized. RESULTS: The database searches yielded 1278 references. Of the 281 abstracts that met the inclusion criteria, 20 articles were retained. In the articles, adherence and compliance were often used interchangeably and various methods were used to measure these concepts. All showed a positive association between treatment satisfaction and adherence, compliance, or persistence. Sixteen studies demonstrated a statistically significant link between satisfaction and compliance or persistence. Of these, ten demonstrated a significant link between satisfaction and compliance, two showed a significant link between satisfaction and persistence, and eight demonstrated a link between either a related aspect or a component of satisfaction (eg, treatment convenience) or adherence (eg, intention to persist). An equal number of studies aimed at explaining compliance or persistence according to treatment satisfaction (n = 8) and treatment satisfaction explained by compliance or persistence (n = 8). Four studies only reported correlation coefficients, with no hypothesis about the direction of the link. The methods used to evaluate the link were varied: two studies reported the link using descriptive statistics, such as percentages, and 18 used statistical tests, such as Spearman's correlation or logistic regressions. CONCLUSION: This review identified few studies that evaluate the statistical association between satisfaction and adherence, compliance, or persistence. The available data suggested that greater treatment satisfaction was associated with better compliance and improved persistence, and with lower regimen complexity or treatment burden.