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

AbstractObjectiveTo 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.MethodsAnalyses 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.ResultsVariability in SGRQ Symptom scores was found suggesting subsets of patients with differential response to treatment. GMM analyses found subsets of non-responders in all trials. When the responders were analyzed separately from non-responders, there were increased treatment effects (e.g., symptoms score improvement over six months for whole groups: indacaterol=8–12 units, tiotropium=7 units, salmeterol=9 units, formoterol=11 units. Responder subgroup improvement: indacaterol=9–21 units, tiotropium=7 units, salmeterol=10 units, formoterol=20 units). Responders had significantly different baseline SGRQ Symptom scores, smoking history, age, and mMRC dyspnea scores than non-responders.ConclusionsPatients with COPD represent a heterogeneous population in terms of their reporting of symptoms and response to treatment. GMM analyses are able to identify sub-groups of responders and non-responders. Application of this methodology could be of value on other endpoints in COPD and in other disease areas

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. The goal of this study was to validate the SF-36 (version 2) for endometriosis. METHODS: Using data from two clinical trials (N = 252 and 198) of treatment for endometriosis, a full complement of psychometric analyses was performed. Additional instruments included a pain visual analog scale (VAS); a physician-completed questionnaire based on patient interview (modified Biberoglu and Behrman--B&B); clinical global impression of change (CGI-C); and patient satisfaction with treatment. RESULTS: Bodily pain (BP) and the Physical Component Summary Score (PCS) were correlated with the pain VAS at baseline and over time and the B&B at baseline and end of study. In addition, those who had the greatest change in BP and PCS also reported the greatest change on CGI-C and patient satisfaction with treatment. Other subscales showed smaller, but significant, correlations with change in the pain VAS, CGI-C, and patient satisfaction with treatment. CONCLUSIONS: The SF-36--particularly BP and the PCS--appears to be a valid and responsive measure for endometriosis and its treatment

Factors Associated with Revision Surgery after Internal Fixation of Hip Fractures

Background: Femoral neck fractures are associated with high rates of revision surgery after management with internal fixation. Using data from the Fixation using Alternative Implants for the Treatment of Hip fractures (FAITH) trial evaluating methods of internal fixation in patients with femoral neck fractures, we investigated associations between baseline and surgical factors and the need for revision surgery to promote healing, relieve pain, treat infection or improve function over 24 months postsurgery. Additionally, we investigated factors associated with (1) hardware removal and (2) implant exchange from cancellous screws (CS) or sliding hip screw (SHS) to total hip arthroplasty, hemiarthroplasty, or another internal fixation device. Methods: We identified 15 potential factors a priori that may be associated with revision surgery, 7 with hardware removal, and 14 with implant exchange. We used multivariable Cox proportional hazards analyses in our investigation. Results: Factors associated with increased risk of revision surgery included: female sex, [hazard ratio (HR) 1.79, 95% confidence interval (CI) 1.25-2.50; P = 0.001], higher body mass index (fo

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. Objective: To explore patients' use of, preferences for, and willingness to switch from prescription to over-the-counter (OTC) treatments for chronic urticaria. In addition, we compared treatment satisfaction with the treatment used most recently for their chronic urticaria. Methods: Data were collected by an online survey using members of international consumer survey panels representative of the general populations in France and Germany. Panelists with urticaria were selected based on answers to screening questions. Respondents were asked, in their native language, questions about their typical course of treatment (prescription vs OTC), satisfaction with treatment, troublesomeness of their typical outbreak, whether their condition had improved or become worse over the past 12 months, and their willingness to try a new treatment if one became available. Descriptive analyses were performed on selected variables. Groups were compared using difference of means tests. Correlations and multiple regressions were performed to assess predictors of satisfaction with treatment and likelihood of switching treatment. Results: The final study sample consisted of 405 patients. Patients who used prescription treatments were more satisfied and reported greater treatment benefits with their treatment than those using OTC treatments. Patients who felt that their condition had become worse over the past 12 months were more likely to consider switching treatments. Respondents who used Aerius (desloratadine) most recently for treating their symptoms reported greater satisfaction with treatment than did those who used other commonly used prescription treatments most recently. Conclusions: These results suggest that prescription treatments for chronic urticaria have a valuable place amongst the treatments offered for this very uncomfortable condition. Moreover, patients expressed preference for prescription treatments in large part because they felt that these treatments work.

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. The objective of this study was to assess the correlation between changes in patient-reported outcome measures (PROMs) of signs and symptoms, dermatologic quality of life (QoL), and urticaria-specific QoL. Methods Latent growth models (LGMs) were applied to longitudinal data from three randomized, Phase 3 clinical trials investigating the efficacy and safety of omalizumab in CSU. Results A near-perfect association between changes in signs and symptoms and changes in dermatologic and urticaria-specific QoLs was identified in each clinical trial when using LGMs (correlation coefficient range 0.88–0.92). Conclusion Evidence showed that changes in signs and symptoms are closely related to changes in HRQoL. However, analyses were performed on clinical trial results of an extremely effective treatment; a less effective treatment with much smaller changes over time may not show such close correlations. Results suggest that any of these PROMs may be used to understand changes in CSU

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

<p>Abstract</p> <p>Background</p> <p>To estimate utility values for different levels of migraine pain severity from a United Kingdom (UK) sample of migraineurs.</p> <p>Methods</p> <p>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.</p> <p>Results</p> <p>Utility scores for each health state were significantly different from 1.0 (no problems on any EQ-5D dimension) (p < 0.0001) and one another (p < 0.0001). The lowest mean utility, − 0.20 (95% confidence interval [CI]: -0.27 – -0.13), was for severe migraine pain. The smallest difference in mean utility was between mild and moderate migraine pain (0.13) and the largest difference in mean utility was between current health (without migraine) and severe migraine pain (1.07).</p> <p>Conclusions</p> <p>Results indicate that all levels of migraine pain are associated with significantly reduced utility values. As severity worsened, utility decreased and severe migraine pain was considered a health state worse than death. Results can be used in cost-utility models examining the relative economic value of therapeutic strategies for migraine in the UK.</p

Changing Relations: Newcomers and Established Residents in Garden City, Kansas

Garden City is located in southwest Kansas, 215 miles west of Wichita and 309 miles southeast of Denver, Colorado. At an elevation of approximately 2,900 feet, it rests amid a semiarid region of short grass and sandsage prairie. With an estimated population of 25,000, it is not only the Finney County seat but a trade and service center for small agricultural communities and unincorporated rural settlements in a five-state area of the southern High Plains (Garden City Planning Department 1989). What follows is an attempt by the Changing Relations Project--six sojourners--to understand and explain how Garden City has met and accommodated its newest arrivals