Predicting declines in physical function in persons with multiple chronic medical conditions: what we can learn from the medical problem list

BACKGROUND: Primary care physicians are caring for increasing numbers of persons with comorbid chronic illness. Longitudinal information on health outcomes associated with specific chronic conditions may be particularly relevant in caring for these populations. Our objective was to assess the effect of certain comorbid conditions on physical well being over time in a population of persons with chronic medical conditions; and to compare these effects to that of hypertension alone. METHODS: We conducted a secondary analysis of 4-year longitudinal data from the Medical Outcomes Study. A heterogeneous population of 1574 patients with either hypertension alone (referent) or one or more of the following conditions: diabetes, coronary artery disease, congestive heart failure, respiratory illness, musculoskeletal conditions and/or depression were recruited from primary and specialty (endocrinology, cardiology or mental health) practices within HMO and fee-for-service settings in three U.S. cities. We measured categorical change (worse vs. same/better) in the SF-36(R) Health Survey physical component summary score (PCS) over 4 years. We used logistic regression analysis to determine significant differences in longitudinal change in PCS between patients with hypertension alone and those with other comorbid conditions and linear regression analysis to assess the contribution of the explanatory variables. RESULTS: Specific diagnoses of CHF, diabetes and/or chronic respiratory disease; or 4 or more chronic conditions, were predictive of a clinically significant decline in PCS. CONCLUSIONS: Clinical recognition of these specific chronic conditions or 4 or more of a list of chronic conditions may provide an opportunity for proactive clinical decision making to maximize physical functioning in these populations

Measurement properties of the Flu-Like Symptom Index from the Hepatitis Physical Symptom Severity Diary

Purpose Chronic Hepatitis C (CHC) Virus infection is a serious health issue in the US. Standard treatment involves peginterferon alpha and ribavirin, often associated with adverse side effects including flu-like symptoms. These adverse effects are common reasons for the discontinuation of treatment and therefore represent a major obstacle in the effective treatment of CHC. Methods The Hepatitis Physical Symptom Severity Diary, a newly developed patient-reported outcome mea-sure for assessing physical symptoms in CHC patients, was recently developed. It contains four questions addressing flu-like symptoms [the Flu-Like Symptom Index (FLSI)]. Measurement properties of the FLSI in CHC patients were assessed using data from two randomized clinical trials. Results Exploratory factor analysis using data from baseline and the last visit while on treatment supported a single-factor solution for the FLSI. Internal reliability and test–retest reliability are acceptable (Cronbach’s alpha range 0.73–0.81; intraclass correlation coefficient range 0.85–0.97), and correspondence to several similar con-structs was acceptable. The FLSI score was higher among those with investigator-reported flu-like symptoms (mean = 4.1) versus those without (1.4), although not statistically significant (p = 0.12). Responsiveness of the FLSI was moderate, as measured by standardized effect sizes and response means, and the minimum important difference (MID) was estimated at 2.5–3.0 points. Conclusions While additional research should be con-ducted to evaluate validity with more closely related con-structs and to utilize anchor-based methods for estimating the MID, data suggest that the FLSI has acceptable mea-surement properties and can be an effective tool in assessing flu-like symptoms in CHC patients

PRO development: rigorous qualitative research as the crucial foundation

Recently published articles have described criteria to assess qualitative research in the health field in general, but very few articles have delineated qualitative methods to be used in the development of Patient-Reported Outcomes (PROs). In fact, how PROs are developed with subject input through focus groups and interviews has been given relatively short shrift in the PRO literature when compared to the plethora of quantitative articles on the psychometric properties of PROs. If documented at all, most PRO validation articles give little for the reader to evaluate the content validity of the measures and the credibility and trustworthiness of the methods used to develop them. Increasingly, however, scientists and authorities want to be assured that PRO items and scales have meaning and relevance to subjects. This article was developed by an international, interdisciplinary group of psychologists, psychometricians, regulatory experts, a physician, and a sociologist. It presents rigorous and appropriate qualitative research methods for developing PROs with content validity. The approach described combines an overarching phenomenological theoretical framework with grounded theory data collection and analysis methods to yield PRO items and scales that have content validity

Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial

BACKGROUND: Cystic fibrosis (CF) is an inherited, rare autosomal recessive disease that results in chronically debilitating morbidities and high premature mortality. We evaluated how ivacaftor treatment affected CF symptoms, functioning, and well-being, as measured by the Cystic Fibrosis Questionnaire-Revised (CFQ-R), a widely-used patient-reported outcome (PRO) measure. METHODS: STRIVE, a double-blind, placebo-controlled randomized trial, evaluated ivacaftor (150 mg) in CF patients aged 12+ with the G551D-CFTR mutation for 48 weeks. Treatment effect analysis used a mixed-effects repeated measures model. Treatment benefit analyses applied the cumulative distribution function and a categorical analysis of change scores (“improvement,” “no change,” or “decline”). Content-based interpretation examined treatment effect on specific item responses. RESULTS: Data from 152 patients with a baseline CFQ-R assessment were analyzed. The treatment effect analysis favored treatment with ivacaftor over placebo on the Body Image, Eating, Health Perceptions, Physical Functioning, Respiratory, Social Functioning, Treatment Burden, and Vitality scales. Findings were supported by the analysis of categorical change. On all CFQ-R scales, the percentage of patients who improved was greater for ivacaftor. In the content-based analysis, the treatment benefit was characterized by better scores across a broad range of domains. CONCLUSIONS: Results illustrate broad benefits of ivacaftor treatment across many domains: respiratory symptoms, physical and social functioning, health perceptions, and vitality, as measured by the CFQ-R. The breadth of improvements reflects the systemic mechanism of action of ivacaftor compared to other therapies. Findings support the patient-reported value of ivacaftor treatment in this patient population. TRIAL REGISTRATION: ClinicalTrials.gov NCT00909532 ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (doi:10.1186/s12955-015-0293-6) contains supplementary material, which is available to authorized users

Predicting declines in physical function in persons with multiple chronic medical conditions: What we can learn from the medical problem list

<p>Abstract</p> <p>Background</p> <p>Primary care physicians are caring for increasing numbers of persons with comorbid chronic illness. Longitudinal information on health outcomes associated with specific chronic conditions may be particularly relevant in caring for these populations. Our objective was to assess the effect of certain comorbid conditions on physical well being over time in a population of persons with chronic medical conditions; and to compare these effects to that of hypertension alone.</p> <p>Methods</p> <p>We conducted a secondary analysis of 4-year longitudinal data from the Medical Outcomes Study. A heterogeneous population of 1574 patients with either hypertension alone (referent) or one or more of the following conditions: diabetes, coronary artery disease, congestive heart failure, respiratory illness, musculoskeletal conditions and/or depression were recruited from primary and specialty (endocrinology, cardiology or mental health) practices within HMO and fee-for-service settings in three U.S. cities. We measured categorical change (worse vs. same/better) in the SF-36^®Health Survey physical component summary score (PCS) over 4 years. We used logistic regression analysis to determine significant differences in longitudinal change in PCS between patients with hypertension alone and those with other comorbid conditions and linear regression analysis to assess the contribution of the explanatory variables.</p> <p>Results</p> <p>Specific diagnoses of CHF, diabetes and/or chronic respiratory disease; or 4 or more chronic conditions, were predictive of a clinically significant decline in PCS.</p> <p>Conclusions</p> <p>Clinical recognition of these specific chronic conditions or 4 or more of a list of chronic conditions may provide an opportunity for proactive clinical decision making to maximize physical functioning in these populations.</p

Health-related quality of life in chronic hepatitis C: impact of disease and treatment response. The Interventional Therapy Group

Hepatitis C infects nearly 4 million Americans. Most have chronic hepatitis C (CHC), which progresses to cirrhosis in about 20% of patients. Interferon treatment leads to transient responses in about 40% of patients and apparent eradication of infection in 7% to 40% of patients. In this report, we document the impact of CHC on health-related quality of life (HQL), and changes in HQL among treatment responders. Three hundred twenty-four CHC patients from 10 countries who had relapsed after responding to interferon-alfa therapy were randomized to monotherapy (IFN alfa-2b + placebo) or combination therapy (IFN alfa-2b + ribavirin), treated for 24 weeks, and followed up for 24 weeks. HQL was assessed using the Hepatitis Quality of Life Questionnaire (HQLQ), containing the generic SF-36 Health Survey, three additional generic scales, and two hepatitis-specific scales. Before treatment, CHC patients were impaired in 5 of 8 SF-36 concepts (physical functioning, role-physical, general health, vitality, and social functioning) in comparison with matched population norms. Sustained virological response (SVR) to treatment yielded improvements on three generic scales (vitality, social functioning, and health distress) and the CHC-specific health distress scale. Overall response to treatment (SVR plus histological improvement) yielded the same pattern of improvements with additional gains in generic general health and CHC-specific limitations. Successful treatment of CHC improved HQL as measured by both CHC-specific and generic measures of functional health and well being