Maria-Magdalena Balp

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.

A network meta-analysis of the efficacy of inhaled antibiotics for chronic Pseudomonas infections in cystic fibrosis

BACKGROUND Various inhaled antibiotics are currently used for treating chronic Pseudomonas aeruginosa lung infection in cystic fibrosis (CF) patients, however their relative efficacies are unclear. We compared the efficacy of the inhaled antibiotics tobramycin (TIP, TIS-T, TIS-B), colistimethate sodium (colistin) and aztreonam lysine for inhalation (AZLI) based on data from randomised controlled trials. METHODS In the base case, efficacies of antibiotics were compared using a network meta-analysis of seven trials including change from baseline in forced expiratory volume in 1 second (FEV(1)) % predicted, P. aeruginosa sputum density and acute exacerbations. RESULTS The tobramycin preparations, AZLI and colistin, showed comparable improvements in efficacy in terms of FEV1% predicted at 4 weeks; the difference in % change from baseline (95%CrI) for TIP was compared to TIS-T (-0.55, -3.5;2.4), TIS-B (-0.64, -7.1;5.7), AZLI (3.64, -1.0;8.3) and colistin (5.77, -1.2;12.8). CONCLUSION We conclude that all studied antibiotics have comparable efficacies for the treatment of chronic P. aeruginosa lung infection in CF.

Validation of the Treatment Satisfaction Questionnaire for Medication in patients with cystic fibrosis

BACKGROUND Our objective was to confirm the measurement properties of the Treatment Satisfaction Questionnaire with Medication (TQSM) in patients with cystic fibrosis (CF) receiving inhaled antibiotics. METHODS The TSQM was included in the EAGER study, a clinical trial comparing a nebulized and a dry powder device for inhaled tobramycin in a CF population with chronic Pseudomonas aeruginosa (Pa) lung infection, aged 6 years and above (N=553). Reliability and validity of the questionnaire were investigated using Cronbachs α and multitrait-multimethod approach. RESULTS The TSQM demonstrated very good reliability and construct validity: all Cronbachs α were above 0.86 and all items met the convergent and discriminant validity criteria. In multivariate regressions, higher patient satisfaction and lower perceived impact of side-effects were associated with better treatment compliance. CONCLUSIONS The TSQM showed very good measurement properties that strongly support its use to assess satisfaction of patients with CF taking inhaled antibiotics.

Systematic review of treatments for chronic spontaneous urticaria with inadequate response to licensed first‐line treatments

Patients with chronic spontaneous urticaria (CSU) are sometimes unresponsive to nonsedating, second‐generation, H1 antihistamines; this study summarizes published clinical evidence for patients who remain symptomatic despite treatment.

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

IntroductionAssessing 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.MethodsLatent growth models (LGMs) were applied to longitudinal data from three randomized, Phase 3 clinical trials investigating the efficacy and safety of omalizumab in CSU.ResultsA 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).ConclusionEvidence 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.

Omalizumab substantially improves dermatology-related quality of life in patients with chronic spontaneous urticaria

Chronic spontaneous/idiopathic urticaria (CSU/CIU) has substantial detrimental effects on health‐related quality of life (HRQoL) with an effect comparable to or worse than many other skin diseases.

Patient-reported impact of chronic urticaria compared with psoriasis in theUnited States

Abstract Purpose: Data are lacking on the burden of chronic idiopathic urticaria (CIU) versus other dermatologic conditions. This analysis compared the burden of chronic urticaria (CU, proxy for CIU) with psoriasis. Methods: Data from CU (N = 747) and psoriasis patients (N = 5107) came from 2010 to 2012 US National Health and Wellness Surveys. Outcomes included SF-12v2/SF-36v2 mental and physical component summary scores (MCS and PCS, respectively) and other health/activity-related measures. Results: MCS score was 44.7 for CU, and 48.2, 44.7 and 44.3 for mild/moderate/severe psoriasis, respectively (US norm = 50). PCS score was 43.8 for CU, and 46.5, 44.1 and 40.3 for mild/moderate/severe psoriasis. Health utility score was 0.67 for CU, and 0.72, 0.67 and 0.65 for mild/moderate/severe psoriasis. More CU patients reported depression (39%), anxiety (42%) and sleep difficulties (50%) than psoriasis patients (any severity). Overall work impairment was 29% for CU, and 19%, 26% and 31% for mild/moderate/severe psoriasis. Activities impairment was 39% for CU, and 28%, 37% and 43% for mild/moderate/severe psoriasis. CU and psoriasis patients had frequent healthcare visits. Conclusions: Patients with CU had impaired mental/physical health and work/non-work activities, similar to moderate-to-severe psoriasis patients. Results suggest that better disease management of CU is needed. This analysis should also reflect the significant burden of CIU.

Work Productivity and Activity Impairment Among Chronic Spontaneous/Idiopathic Urticaria Patients: ResultsFrom The First International Burden of Illness Study (Assure-Csu).

SPONTANEOUS/IDIOPATHIC URTICARIA PATIENTS: RESULTS FROM THE FIRST INTERNATIONAL BURDEN OF ILLNESS STUDY (ASSURE-CSU) Balp MM1, Chambenoit O2, Chiva-Razavi S2, Lynde C3, Sussman G4, Chapman-Rothe N5, Weller K6, Maurer M6, Koenders J7, Knulst AC8, Halliday A9, Alexopoulos ST9, Nakonechna A10, Grattan C11, Abuzakouk M12, Sweeney C13, Radder C13, Wolin D14, McBride D15, Hollis K13, Tian H16, Oude Elberink JNG17 1Novartis Pharma AG, Basel, Switzerland | 2Novartis Pharmaceuticals Canada Inc., Dorval, QC, Canada | 3Lynderm Research Inc., Toronto, ON, Canada | 41St Michael’s Hospital, University of Toronto, Toronto, ON, Canada | 5Novartis Pharma GmbH, Nuernberg, Germany | 6Charite Universitatsmedizin Berlin, Berlin, Germany | 7Novartis Pharma B.V., Arnhem, Netherlands | 8University Medical Center Utrecht, Utrecht, Netherlands, 9Novartis Pharmaceuticals UK Limited, Surrey, United Kingdom | 10Royal Liverpool and Broadgreen University Hospitals NHS Trust, United Kingdom | 11Norfolk and Norwich University Hospital, Norwich, United Kingdom, 12Cleveland Clinic Abu Dhabi, Al Maryah Island, Abu Dhabi, UAE | 13RTI Health Solutions, Research Triangle Park, NC, USA | 14RTI Health Solutions, Ann Arbor, MI, USA | 15RTI Health Solutions, Manchester, United Kingdom | 16Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA | 17University Medical Center Groningen, Groningen, Netherlands

332* Validation of the treatment satisfaction questionnaire for medication (TSQM) in cystic fibrosis (CF)

Objectives: To assess the measurement properties of the TSQM, a 14-item questionnaire measuring 4 dimensions related to treatment satisfaction (Effectiveness; Side-effects; Convenience; Global satisfaction) in CF. Methods: The TSQM is a generic treatment satisfaction instrument validated in several chronic diseases but not used before in CF. It was included in the EAGER study, a randomized open-label clinical trial comparing a nebulized and a dry powder device for inhaled tobramycin in a CF population aged 6 years and above. The two treatment arms (N= 553) were pooled together to conduct psychometric analyses. Construct validity was assessed using confirmatory factor analysis (CFA) and multi-trait analysis. Cronbach’s a were calculated to assess internal consistency reliability. Differential item functioning between responders who were assisted by a relative (patients below 12 years of age) and those who were not was sought for using logistic regression. Results: The quality of completion of the 14 TSQM items was excellent since all had less than 2% missing data. The CFA goodness-of-fit of the 4-dimension structure met commonly used standards (e.g. Goodness of Fit index = 0.92). In the multitrait analysis, all items met the convergent validity criterion and all but one met the discriminant validity. All Cronbach’s a were above 0.86, indicating very good reliability for all 4 dimensions. No item showed differential functioning related to mode of administration (alone vs. with the assistance of a relative). Conclusion: The TSQM showed excellent measurement properties that strongly support the use of this instrument to assess patient satisfaction with inhaled CF treatments.

Prevalence and clinical characteristics of chronic spontaneous urticaria in pediatric patients

Data on the prevalence and disease management of chronic urticaria (CU) and chronic spontaneous urticaria (CSU) in the pediatric population are scarce. This study assessed the prevalence of CU and CSU, and disease management among pediatric patients (0‐17 years).