Publications by authors named "Florian S Gutzwiller"

33 Publications

Use of the Evaluating Respiratory Symptoms™ in COPD as an Outcome Measure in Clinical Trials: A Rapid Systematic Review.

Chronic Obstr Pulm Dis 2021 Oct;8(4):551-571

Pulmonary and Critical Care Medicine, Department of Medicine, University Medical Centre Giessen and Marburg, Philipps-Universität Marburg, Germany and Member of the German Centre for Lung Research (DZL), Marburg, Germany.

Rationale: Patients with chronic obstructive pulmonary disease (COPD) struggle with respiratory symptoms that impair their daily activities and quality of life. Understanding a treatment's ability to relieve symptoms requires precise assessment. The Evaluating Respiratory Symptoms in COPD (E-RS:COPD) was developed to quantify respiratory symptoms in clinical trials. This study aimed to better understand how trials use this patient-reported outcome measure as an endpoint, as well as its responsiveness and performance relative to other outcome measures.

Objectives: To summarize the use of the E-RS:COPD in pharmacological trials since its qualification by regulatory authorities.

Methods: A rapid systematic literature review, using key biomedical databases to identify English language full-text publications of randomized controlled clinical trials (RCTs) that included the E-RS:COPD as an endpoint (2010-2020). Two investigators independently screened the publications and extracted data.

Measurements And Main Results: Of 219 screened records, 28 full-text publications were included, and data from 17 reporting 20 unique double-blind RCTs were synthesized. The E-RS:COPD was positioned as a primary or secondary endpoint in six publications (35%), and served as an exploratory or additional endpoint in 11 (65%). Statistically significant E-RS:COPD treatment effects versus placebo/comparator were found in 13 of the 14 publications reporting symptom results. E-RS:COPD effects corresponded well with other outcome measures (e.g., St George's Respiratory Questionnaire [SGRQ] and forced expiratory volume 1 second [FEV]). Two publications reported the number of responders.

Conclusions: E-RS:COPD is sensitive to treatment effects in clinical trials testing drug therapies. Presentation of trial results should include responder analyses to facilitate interpretation and application of results.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.15326/jcopdf.2021.0235DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8686855PMC
October 2021

Social Media Use in COPD Patients in Germany and Switzerland.

Pneumologie 2021 Aug 7;75(8):583-591. Epub 2021 May 7.

Pforzheim University, School of Engineering, Pforzheim, Germany.

Use of social media and the Internet has changed the information-seeking behaviour and exchange of experience and information by patients. Passive observation of such online interaction between patients (social media listening) is conducted in order to understand the burden of the disease, symptom perception, and expectations from a patient perspective. For most conditions, it remains to be established how representative the social media user community is for the overall patient population. In this study, we describe internet and social media use in a population of 570 COPD patients from Germany and Switzerland. This study population is a good representation of the overall patient population in Germany and Switzerland with regards to socioeconomic data. Patients were analyzed in an exploratory fashion whether usage of the Internet to obtain disease-specific information and exchanging on COPD via social media is associated with or is independent from certain socioeconomic criteria. About three-fourths of patients indicated using the Internet to search information about COPD and about a third of patients indicated using social media to exchange with others about their disease. Results indicated that among the patients using the Internet to seek information and among those sharing information via social media, patients with very severe COPD (GOLD stage 4) were overrepresented versus milder forms of the disease. Similarly, patients with more advanced educational background were also overrepresented in the groups using social media and Internet in relation to COPD. Differences in mean age were statistically significant, but surprisingly small between social media users and non-users. No relationship with regards to social media and Internet use for COPD were observed for domiciling situation and sex.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1055/a-1481-0037DOI Listing
August 2021

A multistakeholder Delphi consensus core outcome set for clinical trials in moderate-to-severe asthma (coreASTHMA).

Ann Allergy Asthma Immunol 2021 07 27;127(1):116-122.e7. Epub 2021 Mar 27.

Center for Medical Technology Policy, Baltimore, Maryland.

Background: Treatments for long-term control of asthma have improved and include a promising but expensive class of biologic therapies. However, the clinical trials evaluating these and other novel treatments have used a variety of different outcomes to evaluate efficacy. The evolution of asthma care calls for a re-examination of outcomes that are most important to patients and other stakeholders.

Objective: To develop a core set of outcomes to be measured in phase 3 and phase 4 clinical drug trials in patients with moderate-to-severe asthma.

Methods: We used a robust and in-depth multistakeholder consensus process bringing together patients, clinicians, regulators, payers, health technology assessors, researchers, and product developers to reach consensus on outcomes. We used a modified Delphi method to reach consensus, an approach adapted from the Core Outcome Measures in Effectiveness Trials Initiative aligned with contemporary methodological standards for core outcome set development.

Results: The following outcomes were included in the final core set: severe asthma exacerbation, change in asthma control, asthma-specific or severe asthma-specific quality of life, asthma-specific hospital stay (ie, >24-hour stays at any level of care) or admission, and asthma-specific emergency department visit.

Conclusion: These 5 outcomes represent a minimum set of core outcomes for use in phase 3 and phase 4 clinical drug trials in moderate-to-severe asthma. Consistent collection of these outcomes as minimum, independent of whether additional heterogeneous primary or secondary outcomes are included, will allow for meaningful comparisons of the effect of asthma therapies across clinical trials.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.anai.2021.03.022DOI Listing
July 2021

The Impact of Exacerbation Frequency on Clinical and Economic Outcomes in Swedish COPD Patients: The ARCTIC Study.

Int J Chron Obstruct Pulmon Dis 2021;16:701-713. Epub 2021 Mar 18.

IQVIA Solutions, Real World Evidence Solutions, Copenhagen, Denmark.

Purpose: The aim of this study was to assess the association between exacerbation frequency and clinical and economic outcomes in patients with COPD.

Patients And Methods: Electronic medical record data linked to National Health Registries were collected from COPD patients at 52 Swedish primary care centers (2000-2014). The outcomes analyzed were exacerbation rate, mortality, COPD treatments, lung function and healthcare costs during the follow-up period. Based on the exacerbation rate two years before index date, the patients were initially classified into three groups, either 0, 1 or ≥2 exacerbations per year. After the index date, the classification into exacerbation groups was updated each year based on the exacerbation rate during the last year of follow-up. A sensitivity analysis was conducted excluding patients with asthma diagnosis from the analysis.

Results: In total 18,586 COPD patients were analyzed. A majority of the patients (60-70%) who either have had no exacerbation or frequent exacerbations (≥2/year) during the pre-index period remained in their group (ie, with 0 or ≥2 annual exacerbations) during up to 11 years of follow-up. Compared with having no exacerbation, mortality was higher in patients having 1 (HR; 2.06 [1.93-2.20]) and ≥2 (4.58 [4.33-4.84]) exacerbations at any time during the follow-up. Lung function decline was more rapid in patients with frequent exacerbations and there was an almost linear relationship between exacerbations frequency and mortality. Total healthcare costs were higher in the frequent exacerbation group (≥2/year) than in patients with no or one exacerbation annually (p<0.0001 for both). The results did not differ from the main analysis after exclusion of patients with a concurrent asthma diagnosis.

Conclusion: In addition to faster lung function decline and increased mortality, frequent exacerbations in COPD patients imply a significant economic burden.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.2147/COPD.S297943DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7987259PMC
June 2021

Inhaled corticosteroids and the risk of type 2 diabetes among Swedish COPD patients.

NPJ Prim Care Respir Med 2020 10 20;30(1):47. Epub 2020 Oct 20.

Integrative Toxicology, The National Institute of Environmental Medicine, Karolinska Institutet, Stockholm, Sweden.

This study reports the association of ICS use and the risk of type 2 diabetes mellitus (T2DM) in Swedish patients with COPD using data from real-world, primary care settings. A total of 7078 patients with COPD were included in this analysis and the 5-year cumulative incidence rate per 100,000 person years was 1506.9. The yearly incidence rate per 100,000 person years ranged from 850 to 1919. Use of ICS especially at a high dose in patients with COPD was related to an increased risk of T2DM.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1038/s41533-020-00207-7DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7576803PMC
October 2020

Is the St. George's Respiratory Questionnaire an Appropriate Measure of Symptom Severity and Activity Limitations for Clinical Trials in COPD? Analysis of Pooled Data from Five Randomized Clinical Trials.

Int J Chron Obstruct Pulmon Dis 2020 8;15:2103-2113. Epub 2020 Sep 8.

Novartis Pharma AG, Basel, Switzerland.

Purpose: The objective of this study was to examine the psychometric properties of the St. George's Respiratory Questionnaire (SGRQ) in patients with chronic obstructive pulmonary disease (COPD) using Rasch measurement theory (RMT) analyses.

Materials And Methods: RMT analysis was conducted on the baseline SGRQ data from five multi-national, Phase III randomized trials investigating a fixed-dose combination of a long-acting β2-agonist and a long-acting muscarinic antagonist in COPD patients. Analysis was performed for the SGRQ "Symptoms" and "Activity" domains. An exploratory analysis was also conducted using the different specific symptoms as defined in the reconceptualization of the SGRQ "Symptoms" domain. Differential item functioning (DIF) analysis was performed for geographical regions on the "Activity" domain, in order to explore cross-cultural validity of the SGRQ.

Results: Overall, the SGRQ "Activity" domain showed good measurement property, but two items ("Sitting or lying still making feel breathless" and "Playing sports or game making feel breathless") showed very high fit residuals. The SGRQ "Symptoms" domain demonstrated good targeting; however, two items showed disordered thresholds ("Coughed" and "Brought up phlegm"). In an exploratory RMT analysis, measures for "Cough and Sputum", "Breathing difficulties" or "Wheezing attacks" showed unsatisfactory measurement properties with poor reliability (person separation index = 0.35, 0.66 and 0.16, respectively) and targeting issues. The examination of cross-cultural performances of the SGRQ "Activity" items showed a great variability in the responses to these items in different global regions.

Conclusion: Our results indicated that SGRQ may not be an appropriate instrument to measure symptom severity or activity limitations in patients with COPD. Hence, there is a need to develop other relevant PRO instruments that can be used in conjunction with SGRQ to provide a holistic assessment of the health status of COPD patients in clinical research.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.2147/COPD.S261919DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7490086PMC
June 2021

Osteoporosis and fracture risk associated with inhaled corticosteroid use among Swedish COPD patients: the ARCTIC study.

Eur Respir J 2021 02 17;57(2). Epub 2021 Feb 17.

Intergrative Toxicology, National Institute of Environmental Medicine, Karolinska Institute, Stockholm, Sweden.

The effect of inhaled corticosteroids (ICS) on the risk of osteoporosis and fracture in patients with chronic obstructive pulmonary disease (COPD) remains uncertain. The aim of this study was to assess this risk in patients with COPD.Electronic medical record data linked to National Health Registries were collected from COPD patients and matched reference controls at 52 Swedish primary care centres from 2000 to 2014. The outcomes analysed were the effect of ICS on all fractures, fractures typically related to osteoporosis, recorded osteoporosis diagnosis, prescriptions of drugs for osteoporosis and a combined measure of any osteoporosis-related event. The COPD patients were stratified by the level of ICS exposure.A total of 9651 patients with COPD and 59 454 matched reference controls were analysed. During the follow-up, 19.9% of COPD patients had at least one osteoporosis-related event compared with 12.9% of reference controls (p<0.0001). Multivariate analysis in the COPD population demonstrated a dose-effect relationship, with high-dose ICS being significantly associated with any osteoporosis-related event (risk ratio 1.52 (95% CI 1.24-1.62)), while the corresponding estimate for low-dose ICS was 1.27 (95% CI 1.13-1.56) compared with COPD patients not using ICS. A similar dose-related adverse effect was found for all four of the specific osteoporosis-related events: all fractures, fractures typically related to osteoporosis, prescriptions of drugs for osteoporosis and diagnosis of osteoporosis.We conclude that patients with COPD have a greater risk of bone fractures and osteoporosis, and high-dose ICS use increased this risk further.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1183/13993003.00515-2020DOI Listing
February 2021

Patient-Reported Outcomes (PROs) in COPD Clinical Trials: Trends and Gaps.

Int J Chron Obstruct Pulmon Dis 2020 23;15:1789-1800. Epub 2020 Jul 23.

Respiratory Medicine Department, University of Ioannina, Ioannina, Greece.

Key characteristics of chronic obstructive pulmonary disease (COPD) that significantly affect health-related quality of life (HRQoL) include chest symptoms, dyspnea, cough, sputum production, and exacerbations. Additional areas of impact are sleep, fatigue, emotional well-being, social functioning, and coping. Patient-reported outcomes (PROs) are essential to evaluate symptoms, impact of symptoms on activities of daily living, and treatment response. This review summarizes COPD-specific PRO endpoints from randomized controlled trials of approved and commonly used COPD drugs. A search conducted in "ClinicalTrials.gov" to identify COPD clinical trials (only completed Phase III and IV) incorporating PRO endpoints yielded a total of 104 clinical trials for inclusion in this analysis. Both symptom-based and HRQoL-specific PRO measures were reported. Several COPD-specific PRO measures are available; however, the St. George's Respiratory Questionnaire (SGRQ) and the Baseline and Transition Dyspnea Indexes (BDI/TDI) were reported in the majority of the studies. Results reflected a gap in terms of full coverage of key impacted areas from a patient's perspective. Methodological issues identified in this review related to scoring of instruments require careful consideration, as these challenges may limit the complete assessment of drug benefits. Selection of PRO measures aligned with the expected treatment benefit of a drug in a clinical trial should reflect patients' perspective holistically.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.2147/COPD.S235845DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7398869PMC
June 2021

Clinical Impact and Healthcare Resource Utilization Associated with Early versus Late COPD Diagnosis in Patients from UK CPRD Database.

Int J Chron Obstruct Pulmon Dis 2020 16;15:1729-1738. Epub 2020 Jul 16.

Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.

Purpose: Previous studies have shown that opportunities to diagnose chronic obstructive pulmonary disease (COPD) early are often missed in primary care. This retrospective study aimed to utilize secondary data from the United Kingdom (UK) healthcare system to understand the impact of early versus late diagnosis of COPD.

Patients And Methods: Newly diagnosed COPD patients were identified in the UK Clinical Practice Research Database from 2011 to 2014. Patients whose 5-year medical data before diagnosis revealed ≥3 counts of eight indicators of early COPD were deemed as late-diagnosed, whereas others were deemed as early-diagnosed. We assessed patients' characteristics; time-to-first, risk, and rates of exacerbation; and healthcare resource utilization (COPD-related clinic visits, Accident and Emergency visits, and hospitalizations) in late- versus early-diagnosed patients.

Results: Of 10,158 patients included in the study, 6783 (67%) were identified as late-diagnosed and 3375 (33%) as early-diagnosed. The median time-to-first exacerbation was shorter in late-diagnosed (14.5 months) versus early-diagnosed (29.0 months) patients, with a significant risk of exacerbation (hazard ratio 1.46 [95% confidence interval: 1.38-1.55]). Additionally, the exacerbation rate (per 100 person-years) over 3 years was higher in late (108.9) versus early (57.2) diagnosed patients. Late-diagnosed patients had a significantly higher rate of COPD hospitalizations (per 1000 patient years) compared with early-diagnosed patients during 2 and 3 years of follow-ups ( = 0.0165 and < 0.0001, respectively).

Conclusion: Results showed that a significant percentage of COPD patients in UK primary care are diagnosed late. A late COPD diagnosis is associated with a shorter time-to-first exacerbation and a higher rate and risk of exacerbations compared with early diagnosis. Additionally, late diagnosis of COPD is associated with a higher rate of COPD-related hospitalizations compared with early diagnosis.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.2147/COPD.S255414DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7371991PMC
June 2021

Modelling the Cost-Effectiveness of Indacaterol/Glycopyrronium versus Salmeterol/Fluticasone Using a Novel Markov Exacerbation-Based Approach.

Int J Chron Obstruct Pulmon Dis 2020 16;15:787-797. Epub 2020 Apr 16.

PT Health Economics Ltd, Sheffield, UK.

Purpose: Exacerbations drive outcomes and costs in chronic obstructive pulmonary disease (COPD). While patient-level (micro) simulation cost-effectiveness models have been developed that include exacerbations, such models are complex. We developed a novel, exacerbation-based model to assess the cost-effectiveness of indacaterol/glycopyrronium (IND/GLY) versus salmeterol/fluticasone (SFC) in COPD, using a Markov structure as a simplification of a previously validated microsimulation model.

Methods: The Markov model included three health states: infrequent or frequent exacerbator (IE or FE; ≤1 or ≥2 moderate/severe exacerbations in prior 12 months, respectively), or death. The model used data from the FLAME study and was run over a 10-year horizon. Cycle length was 1 year, after which patients remained in the same health state or transitioned to another. Analysis was conducted from a Swedish payer's perspective (Swedish healthcare costs, converted into Euros), with incremental costs and quality-adjusted life-years (QALYs) calculated (discounted 3% annually).

Results: At all post-baseline timepoints, IND/GLY was associated with more patients in the IE health state and fewer patients in the FE and dead states relative to SFC. Over a 10-year period, IND/GLY was associated with a cost saving of €1,887/patient, an incremental benefit of 0.142 QALYs, and an addition of 0.057 life-years, compared with SFC.

Conclusion: This Markov model represents a novel cost-effectiveness analysis for COPD, with simpler methodology than prior microsimulation models, while retaining exacerbations as drivers of disease progression. In patients with COPD with a history of exacerbations in the previous year, IND/GLY is a cost-effective treatment option compared with SFC.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.2147/COPD.S247156DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7174156PMC
June 2021

Gender differences among Swedish COPD patients: results from the ARCTIC, a real-world retrospective cohort study.

NPJ Prim Care Respir Med 2019 12 10;29(1):45. Epub 2019 Dec 10.

Department of Public Health and Caring Sciences, Family Medicine and Preventive Medicine, Uppsala University, Uppsala, Sweden.

The present study aimed to generate real-world evidence regarding gender differences among chronic obstructive pulmonary disease (COPD) patients, especially as regards the diagnosis and outcomes in order to identify areas for improvement and management and optimize the associated healthcare resource allocation. ARCTIC is a large, real-world, retrospective cohort study conducted in Swedish COPD patients and a matched reference population from 52 primary care centers in 2000-2014. The incidence of COPD, prevalence of asthma and other comorbidities, risk of exacerbations, mortality rate, COPD drug prescriptions, and healthcare resource utilization were analyzed. In total, 17,479 patients with COPD were included in the study. During the study period, COPD was more frequent among women (53.8%) and women with COPD experienced more exacerbations vs. men (6.66 vs. 4.66). However, the overall mortality rate was higher in men compared with women (45% vs. 38%), but no difference for mortality due to COPD was seen between genders over the study period. Women seemed to have a greater susceptibility to asthma, fractures, osteoporosis, rheumatoid arthritis, rhinitis, depression, and anxiety, but appeared less likely to have diabetes, kidney diseases, and cardiovascular diseases. Furthermore, women had a greater risk of COPD-related hospitalization and were likely to receive a significantly higher number of COPD drug prescriptions compared with men. These results support the need to reduce disease burden among women with COPD and highlight the role of healthcare professionals in primary care who should consider all these parameters in order to properly diagnose and treat women with COPD.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1038/s41533-019-0157-3DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6904454PMC
December 2019

Gathering Structured Patient Insight to Drive the PRO Strategy in COPD: Patient-Centric Drug Development from Theory to Practice.

Adv Ther 2020 01 9;37(1):17-26. Epub 2019 Nov 9.

Novartis Pharma AG, Basel, Switzerland.

We illustrate our experience of gathering patient insights on the most patient-relevant symptoms in chronic obstructive pulmonary disease (COPD) via a structured and systematic approach towards 'patient-centric' drug development, leveraging recent advances in digital technologies using online platforms. The four-step approach comprised the following: literature search, social media listening (SML) study, online bulletin board (OBB) exercise, and design of an online patient preference study (PPS). The initial online studies (SML and OBB) revealed that, besides dyspnoea and exacerbations, patients perceive cough and mucus production as equally important aspects of disease management for COPD. To further build and quantify patients' understanding of the importance of these symptoms, an online patient preference survey is underway. Based on these findings, we have elected to include the Cough and Sputum Assessment Questionnaire or CASA-Q, a validated instrument to collect patient-reported outcomes (PRO), besides the use of the COPD assessment test or CAT to assess the severity and impact of COPD in drug development studies for COPD. Additionally, to capture movement and sleep disturbance, we consider the inclusion of actigraphy as a digital evidence-capture end point. Lastly, in a phase II trial, a survey questionnaire on incontinence will be administered to evaluate the importance of this issue among patients. We believe that integrating insights derived from "online" studies (SML, OBB, and PPS) into drug development offers an opportunity to truly listen to patients' voices in early product design ensuring relevance of end points selected for the clinical trial program. This approach also has the potential to complement conventional qualitative and quantitative data collection requirements for PRO instrument development. While awaiting final guidance from the US Food and Drug Administration, or FDA, the recently released draft documents on collecting representative patients' input reference social media as a tool to collect qualitative patient preference data and these developments suggest that patient preference data can influence future clinical trial design, end point selection, and regulatory reviews.Funding: Novartis Pharma AG, Basel.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s12325-019-01134-xDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6979452PMC
January 2020

Impact of cough and mucus on COPD patients: primary insights from an exploratory study with an Online Patient Community.

Int J Chron Obstruct Pulmon Dis 2019 24;14:1365-1376. Epub 2019 Jun 24.

Novartis Pharma AG, Basel, Switzerland.

Qualitative research provides real-life information on patients' condition and facilitates informed design of future clinical studies. We used Online Communities as a qualitative research tool to evaluate the effect of cough and mucus on COPD patients. Two 2-week Online Communities were run in parallel in the UK and in the USA, including COPD patients with persistent cough and excessive mucus. Patients anonymously posted their responses to pre-assigned tasks, supervised and guided by a trained moderator. Five themes around the impact of cough and mucus were explored with new questions posted every 2-3 days. On the final day, high-level conclusions were shared with patients for feedback. Data were analyzed following the principles of grounded theory. Twenty COPD patients (UK, n=10; USA, n=10) participated in the Online Communities. We found that cough and mucus disrupted COPD patients' lives at functional, emotional, social and economic levels. Patients created daily rituals and adjusted their lifestyle to cope with the impact of these symptoms. Patients identified themselves with our conclusions and saw the Online Community as an effective forum to share their experiences. Findings of our study add to the body of evidence on the negative impact of COPD symptoms and unmet needs of these patients.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.2147/COPD.S202580DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6599966PMC
February 2020

Evaluation of exacerbations and blood eosinophils in UK and US COPD populations.

Respir Res 2019 Aug 7;20(1):178. Epub 2019 Aug 7.

Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.

Background: Blood eosinophil counts and history of exacerbations have been proposed as predictors of patients with chronic obstructive pulmonary disease (COPD) who may benefit from triple therapy (inhaled corticosteroid, long-acting β-agonist and long-acting muscarinic antagonist).

Methods: In a retrospective cohort analysis we examined the profiles of COPD patients from the UK Clinical Practice Research Datalink (CPRD) and US Optum Clinformatics™ Data Mart (Optum) databases with reference to exacerbation frequency and blood eosinophil distribution.

Results: Of the 31,437 (CPRD) and 383,825 (Optum) patients with COPD, 15,364 (CPRD) and 139,465 (Optum) met the eligibility criteria and were included. Among patients with ≥2 exacerbations and available eosinophil counts in the baseline period (CPRD, n = 3089 and Optum, n = 13414), 17.0 and 13.3% respectively had eosinophil counts ≥400 cells/μL. Patients with ≥2 exacerbations or eosinophil count ≥400 cells/μL during first year, exacerbated at least once (CPRD, 82.8% vs Optum, 80.6%) or continued to have eosinophil count ≥300 cells/μL (76.8% vs 76.5%), respectively in the follow-up year. In both years, a higher variability in the number of exacerbations and eosinophil count was observed in patients with one exacerbation and eosinophil counts between 300 and 400 cells/μL; patients with eosinophil count < 150 cells/μL had the lowest variability. Approximately 10% patients had both ≥2 exacerbations and eosinophil count ≥300 cells/μL across the databases.

Conclusion: A high variability in blood eosinophil counts over two consecutive years was observed in UK and US patients with COPD and should be considered while making treatment decisions. A small proportion of COPD patients had frequent exacerbations and eosinophil count ≥300 cells/μL.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1186/s12931-019-1130-yDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6686508PMC
August 2019

Impact of COPD diagnosis timing on clinical and economic outcomes: the ARCTIC observational cohort study.

Int J Chron Obstruct Pulmon Dis 2019 13;14:995-1008. Epub 2019 May 13.

Public Health and Caring Sciences, Family Medicine and Preventive Medicine, Uppsala University, Uppsala, Sweden.

Assess the clinical and economic consequences associated with an early versus late diagnosis in patients with COPD. In a retrospective, observational cohort study, electronic medical record data (2000-2014) were collected from Swedish primary care patients with COPD. COPD indicators (pneumonia, other respiratory diseases, oral corticosteroids, antibiotics for respiratory infections, prescribed drugs for respiratory symptoms, lung function measurement) registered prior to diagnosis were applied to categorize patients into those receiving early (2 or less indicators) or late diagnosis (3 or more indicators registered >90 days preceding a COPD diagnosis). Outcome measures included annual rate of and time to first exacerbation, mortality risk, prevalence of comorbidities and health care utilization. More patients with late diagnosis (n=8827) than with early diagnosis (n=3870) had a recent comorbid diagnosis of asthma (22.0% vs 3.9%; <0.0001). Compared with early diagnosis, patients with late diagnosis had a higher exacerbation rate (hazard ratio [HR] 1.89, 95% confidence interval [CI]: 1.83-1.96; <0.0001) and shorter time to first exacerbation (HR 1.61, 95% CI: 1.54-1.69; <0.0001). Mortality was not different between groups overall but higher for late versus early diagnosis, after excluding patients with past asthma diagnosis (HR 1.10, 95% CI: 1.02-1.18; =0.0095). Late diagnosis was also associated with higher direct costs than early diagnosis. Late COPD diagnosis is associated with higher exacerbation rate and increased comorbidities and costs compared with early diagnosis. The study highlights the need for accurate diagnosis of COPD in primary care in order to reduce exacerbations and the economic burden of COPD.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.2147/COPD.S195382DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6526023PMC
January 2020

Differences in COPD Exacerbation Risk Between Women and Men: Analysis From the UK Clinical Practice Research Datalink Data.

Chest 2019 10 16;156(4):674-684. Epub 2019 May 16.

Novartis Pharma AG, Basel, Switzerland; Heart Center Freiburg University, Cardiology and Angiology I, Faculty of Medicine, Freiburg, Germany.

Background: Historically, COPD has been considered to affect mostly older men with a history of smoking; however, in recent times, its prevalence and mortality rates have steadily increased among women.

Objectives: The goal of this study was to systematically assess differences in COPD expression between women and men in UK primary care clinics who were newly diagnosed with COPD.

Methods: This retrospective cohort study compared women and men with an incident diagnosis of COPD by using electronic medical records data from the Clinical Practice Research Datalink and linked Hospital Episode Statistics data. The overall study period was between January 1, 2006, and February 28, 2016; patients with an incident diagnosis of COPD between January 1, 2010, and February 28, 2015, were analyzed.

Results: A cohort of 22,429 patients were identified as incident patients and included in the study; 48% of patients with COPD were women. The risk of first moderate or severe exacerbation was 17% greater in women than in men (hazard ratio, 1.17; 95% CI, 1.12-1.23), with a median time to first exacerbation of 504 days for women and 637 days for men. These differences were more prominent in the younger age group (≥ 40 years to < 65 years), as well as in Global Initiative for Chronic Obstructive Lung Disease 2016 groups B, C, and D and in individuals with moderate to severe airflow obstruction. The annual rate of moderate or severe exacerbations was higher in women compared with men in the first, second, and third year of follow-up.

Conclusions: These results highlight the unmet need for appropriate identification and management of women with COPD in clinical practice.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.chest.2019.04.107DOI Listing
October 2019

Patients' perspectives on COPD: findings from a social media listening study.

ERJ Open Res 2019 Feb 11;5(1). Epub 2019 Feb 11.

Novartis Pharma AG, Basel, Switzerland.

We utilised social media listening (SML) to obtain patients' perspectives on symptoms, diagnosis and comorbidities associated with chronic obstructive pulmonary disease (COPD) and its impact on patients' quality of life (QoL). A comprehensive search on social media platforms was performed for English language content posted between July 2016 and January 2018 using COPD-related terms. Social Studio, a social media data aggregator tool, was used to capture relevant records. The content was manually curated to analyse and map psychological aspects with descriptive statistics applied on aggregated findings. A total of 849 posts from patients or caregivers ("patient insights") were considered for the analysis, corresponding to postings of 695 unique individuals. Based on 734 mentions of symptoms from 849 posts by potential patients/caregivers, cough (27%), mucus (25%) and shortness of breath (21%) were the most frequent; analysis by perceived COPD severity indicated these to be common across all severities. Difficulty in mucus clearance (24% of 268 mentions) and sadness (40% of 129 mentions) were top among the aspects impacting physical and emotional QoL, respectively. SML from patients with COPD indicated that relief from cough, mucus production and shortness of breath would be the most desirable aspects of disease management from a patient's perspective.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1183/23120541.00128-2018DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6368996PMC
February 2019

Real-world retrospective cohort study ARCTIC shows burden of comorbidities in Swedish COPD versus non-COPD patients.

NPJ Prim Care Respir Med 2018 09 10;28(1):33. Epub 2018 Sep 10.

Department of Public Health and Caring Sciences, Family Medicine and Preventive Medicine, Uppsala University, Uppsala, Sweden.

This study aimed to generate real-world evidence to assess the burden of comorbidities in COPD patients, to effectively manage these patients and optimize the associated healthcare resource allocation. ARCTIC is a large, real-world, retrospective cohort study conducted in Swedish COPD patients using electronic medical record data collected between 2000 and 2014. These patients were studied for prevalence of various comorbidities and for association of these comorbidities with exacerbations, mortality, and healthcare costs compared with an age-, sex-, and comorbidities-matched non-COPD reference population. A total of 17,479 patients with COPD were compared with 84,514 non-COPD reference population. A significantly higher prevalence of various comorbidities was observed in COPD patients 2 years post-diagnosis vs. reference population, with the highest percentage increase observed for cardiovascular diseases (81.8% vs. 30.7%). Among the selected comorbidities, lung cancer was relatively more prevalent in COPD patients vs. reference population (relative risk, RR = 5.97, p < 0.0001). Ischemic heart disease, hypertension, depression, anxiety, sleep disorders, osteoporosis, osteoarthritis, and asthma caused increased mortality rates in COPD patients. Comorbidities that were observed to be significantly associated with increased number of severe exacerbations in COPD patients included heart failure, ischemic heart disease, depression/anxiety, sleep disorders, osteoporosis, lung cancer, and stroke. The cumulative healthcare costs associated with comorbidities over 2 years after the index date were observed to be significantly higher in COPD patients (€27,692) vs. reference population (€5141) (p < 0.0001). The data support the need for patient-centered treatment strategies and targeted healthcare resource allocation to reduce the humanistic and economic burden associated with COPD comorbidities.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1038/s41533-018-0101-yDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6131165PMC
September 2018

 Identifying the associated risks of pneumonia in COPD patients: ARCTIC an observational study.

Respir Res 2018 Sep 10;19(1):172. Epub 2018 Sep 10.

Karolinska Institutet, Solna, Sweden.

Background: Inhaled corticosteroids (ICS) are associated with an increased risk of pneumonia in patients with chronic obstructive pulmonary disease (COPD). Other factors such as severity of airflow limitation and concurrent asthma may further raise the possibility of developing pneumonia. This study assessed the risk of pneumonia associated with ICS in patients with COPD.

Methods: Electronic Medical Record data linked to National Health Registries were collected from COPD patients and matched reference controls in 52 Swedish primary care centers (2000-2014). Levels of ICS treatment (high, low, no ICS) and associated comorbidities were assessed. Patients were categorized by airflow limitation severity.

Results: A total of 6623 patients with COPD and 48,566 controls were analyzed. Patients with COPD had a more than 4-fold increase in pneumonia versus reference controls (hazard ratio [HR] 4.76, 95% confidence interval [CI]: 4.48-5.06). ICS use increased the risk of pneumonia by 20-30% in patients with COPD with forced expiratory volume in 1 s ≥ 50% versus patients not using ICS. Asthma was an independent risk factor for pneumonia in the COPD population. Multivariate analysis identified independent predictors of pneumonia in the overall population. The highest risk of pneumonia was associated with high dose ICS (HR 1.41, 95% CI: 1.23-1.62).

Conclusions: Patients with COPD have a greater risk of pneumonia versus reference controls; ICS use and concurrent asthma increased the risk of pneumonia further.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1186/s12931-018-0868-yDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6131919PMC
September 2018

Capturing Exacerbations of Chronic Obstructive Pulmonary Disease with EXACT. A Subanalysis of FLAME.

Am J Respir Crit Care Med 2019 01;199(1):43-51

5 National Heart and Lung Institute, Imperial College London, London, United Kingdom.

Rationale: Chronic obstructive pulmonary disease exacerbations accelerate lung function decline, reduce quality of life, and increase mortality. A subset of patients (n = 457) from the FLAME (Effect of Indacaterol Glycopyrronium vs. Fluticasone Salmeterol on COPD Exacerbations) study used the Exacerbations of COPD Tool (EXACT) to capture symptom-defined exacerbations.

Objectives: To evaluate the effect of indacaterol/glycopyrronium versus salmeterol/fluticasone on symptom-defined exacerbations measured using EXACT, and to assess differences between these events and exacerbations requiring healthcare resource use (HCRU).

Methods: All patients in FLAME used an electronic diary to record and detect symptom deteriorations; HCRU-related exacerbations were confirmed by investigators. In patients using the EXACT questionnaire, the onset, recovery, and magnitude of symptom-defined exacerbations were identified by changes in total scores relative to baseline. We analyzed the annualized rate and time to first symptom-defined (EXACT) exacerbation and assessed differences between symptom-defined and HCRU events in terms of number, severity, and concordance.

Measurements And Main Results: A nonsignificant 17% reduction in the annualized rate of symptom-defined (EXACT) exacerbations (rate ratio, 0.83; 95% confidence interval [CI], 0.60-1.14; P = 0.242) and a numerically longer time to first symptom-defined exacerbation were observed with indacaterol/glycopyrronium versus salmeterol/fluticasone (hazard ratio, 0.76; 95% CI, 0.56-1.03; P = 0.075). These results were consistent with data from the overall FLAME population. Of the symptom-defined (EXACT) events, 23.5% corresponded to HCRU events, and 22.2% of HRCU events were captured by EXACT (κ index, 0.24; 95% CI, 0.15-0.33).

Conclusions: Regardless of the exacerbation definition used, our findings support the use of long-acting β agonists/long-acting muscarinic receptor antagonists as the preferred treatment option for patients at risk of future exacerbations. Clinical trial registered with www.clinicaltrials.gov (NCT01782326).
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1164/rccm.201801-0038OCDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6353015PMC
January 2019

Factors associated with appropriate inhaler use in patients with COPD - lessons from the REAL survey.

Int J Chron Obstruct Pulmon Dis 2018 26;13:695-702. Epub 2018 Feb 26.

Novartis Pharma AG, Basel, Switzerland.

Background: Nonadherence to medication and incorrect use of inhalers represent significant barriers to optimal disease management of patients with chronic obstructive pulmonary disease (COPD). Thus, health care professionals (HCPs) play a critical role in educating their patients on appropriate inhaler use and in ensuring medication adherence. However, many patients do not receive appropriate inhaler training or have not had their inhaler technique checked.

Methods: The Real-life Experience and Accuracy of inhaLer use (REAL) survey was a computer-assisted, telephonic survey consisting of 23 questions gathering real-world information on correct inhaler use, inhalation technique, device attributes, adherence, dosing accuracy, training, correct device use, ease of use, and factors that influence patient adherence in commercially available inhalers delivering COPD maintenance therapy. All results are based on patient-reported data.

Results: The survey was conducted between January 4, 2016 and February 2, 2016. A total of 764 patients using various inhalers (Breezhaler =186; Ellipta =191; Genuair =194; Respimat =201) with mild to very severe COPD, with a mean ± SD age 56±9.8 years, completed the survey. Patient self-reported adherence was significantly lower in younger patients compared to older patients (=0.020). Eighty-three percent of patients indicated that a demonstration (in-person) was "very helpful" versus 58% for video. Patient preferences for training methods were as follows: demonstration of inhaler use (83%), video (58%), instructions for use (51%), and leaflet (34%). Twenty-nine percent of patients had not been checked to see if they were using their device correctly by a HCP within the last two years. Patients who were checked were significantly more adherent than unchecked patients (=0.020). The majority of the patients using Breezhaler reported either being very confident or confident of having taken a full dose, which was higher than those using Genuair, Ellipta (α=0.05), and Respimat (α=0.05). Treatment adherence in the last 30 days was highest with Breezhaler followed by Respimat, Ellipta, and Genuair.

Conclusion: The REAL survey identified attributes that influenced patient adherence and optimal inhaler use. Predictive attributes that influence patient adherence which HCPs should be aware of include age and disease severity. Modifiable attributes which the HCP can influence include correct inhaler use training, choice of training methods, checking patient inhaler technique at subsequent visits, and device selection. Inhalers are integral in the effective management of patients with COPD; it is therefore important that patients use the inhaler correctly and have full confidence in the dosage.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.2147/COPD.S149404DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5834182PMC
October 2018

Economic burden of COPD in a Swedish cohort: the ARCTIC study.

Int J Chron Obstruct Pulmon Dis 2018 11;13:275-285. Epub 2018 Jan 11.

Department of Public Health and Caring Sciences, Family Medicine and Preventive Medicine, Uppsala University, Uppsala.

Background: We assessed direct and indirect costs associated with COPD in Sweden and examined how these costs vary across time, age, and disease stage in a cohort of patients with COPD and matched controls in a real-world, primary care (PC) setting.

Patients And Methods: Data from electronic medical records linked to the mandatory national health registers were collected for COPD patients and a matched reference population in 52 PC centers from 2000 to 2014. Direct health care costs (drug, outpatient or inpatient, PC, both COPD related and not COPD related) and indirect health care costs (loss of income, absenteeism, loss of productivity) were assessed.

Results: A total of 17,479 patients with COPD and 84,514 reference controls were analyzed. During 2013, direct costs were considerably higher among the COPD patient population (€13,179) versus the reference population (€2,716), largely due to hospital nights unrelated to COPD. Direct costs increased with increasing disease severity and increasing age and were driven by higher respiratory drug costs and non-COPD-related hospital nights. Indirect costs (~€28,000 per patient) were the largest economic burden in COPD patients of working age during 2013.

Conclusion: As non-COPD-related hospital nights represent the largest direct cost, management of comorbidities in COPD would offer clinical benefits and relieve the financial burden of disease.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.2147/COPD.S149633DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5769573PMC
September 2018

Indacaterol/glycopyrronium is cost-effective compared to salmeterol/fluticasone in COPD: FLAME-based modelling in a Swedish population.

Respir Res 2017 12 11;18(1):206. Epub 2017 Dec 11.

Respiratory and Intensive Care Medicine, Cochin Hospital (AP-HP) and University Paris Descartes, Paris, France.

Background: This study assessed the cost-effectiveness of indacaterol/glycopyrronium (IND/GLY) versus salmeterol/fluticasone (SFC) in chronic obstructive pulmonary disease (COPD) patients with moderate to very severe airflow limitation and ≥1 exacerbation in the preceding year.

Methods: A previously published and validated patient-level simulation model was adapted using clinical data from the FLAME trial and real-world cost data from the ARCTIC study. Costs (total monetary costs comprising drug, maintenance, exacerbation, and pneumonia costs) and health outcomes (life-years (LYs), quality-adjusted life-years (QALYs)) were projected over various time horizons (1, 5, 10 years, and lifetime) from the Swedish payer's perspective and were discounted at 3% annually. Uncertainty in model input values was studied through one-way and probabilistic sensitivity analyses. Subgroup analyses were also performed.

Results: IND/GLY was associated with lower costs and better outcomes compared with SFC over all the analysed time horizons. Use of IND/GLY resulted in additional 0.192 LYs and 0.134 QALYs with cost savings of €1211 compared with SFC over lifetime. The net monetary benefit (NMB) was estimated to be €8560 based on a willingness-to-pay threshold of €55,000/QALY. The NMB was higher in the following subgroups: severe (GOLD 3), high risk and more symptoms (GOLD D), females, and current smokers.

Conclusion: IND/GLY is a cost-effective treatment compared with SFC in COPD patients with mMRC dyspnea grade ≥ 2, moderate to very severe airflow limitation, and ≥1 exacerbation in the preceding year.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1186/s12931-017-0688-5DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5725803PMC
December 2017

Cost Effectiveness of Sucroferric Oxyhydroxide Compared with Sevelamer Carbonate in the Treatment of Hyperphosphataemia in Patients Receiving Dialysis, from the Perspective of the National Health Service in Scotland.

Pharmacoeconomics 2015 Dec;33(12):1311-24

Institute of Pharmaceutical Medicine (ECPM), University of Basel, Klingelbergstrasse 61, 4056, Basel, Switzerland.

Background: Hyperphosphataemia is common and harmful in patients receiving dialysis. Treatment options include noncalcium-based phosphate binders such as sevelamer carbonate (SC) and sucroferric oxyhydroxide (PA21).

Objective: The aim of this study was to determine the health economic impact of PA21-based strategies compared with SC-based strategies, from the perspective of the Scottish National Health Service (NHS).

Methods: A Markov model was constructed based on data from a randomised clinical trial comparing PA21 and SC. Model input parameters were derived from published literature, national statistics and unpublished sources. Costs (price year 2012) and effects were discounted at 3.5 %. Analysis with a lifelong time horizon yielded the incremental cost-effectiveness ratio (ICER), expressed as cost or savings per quality-adjusted life-year (QALY) gained or forgone. Deterministic and probabilistic sensitivity analysis was performed to explore uncertainties around assumptions and model input parameters.

Results: In the base-case analysis, phosphorus reductions for PA21 and SC were 1.93 and 1.95 mg/dL. Average undiscounted survival was estimated to be 7.61 years per patient in both strategies. PA21 patients accrued less QALYs (2.826) than SC patients (2.835), partially due to differential occurrence of side effects. Total costs were ₤ 13,119 and ₤ 14,728 for PA21 and SC, respectively (difference per patient of ₤ 1609). By using PA21 versus SC, one would save ₤ 174,999 (or ₤ 123,463 when including dialysis and transplantation costs) for one QALY forgone. A scenario modelling the nonsignificant reduction in mortality (relative risk 0.714) observed in the trial yielded an ICER for PA21 of ₤ 22,621 per QALY gained. In probabilistic sensitivity analysis of the base-case, PA21 was dominant in 11 %, and at least cost-effective in 53 %, of iterations, using a threshold of ₤ 20,000 per QALY gained.

Conclusions: The use of PA21 versus SC in hyperphosphataemic patients being intolerant of calcium-based phosphate binders may be cost saving and yields only very limited disadvantages in terms of quality-adjusted survival. PA21 appears to be cost-effective from the perspective of the Scottish NHS.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1007/s40273-015-0320-9DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4661221PMC
December 2015

End-of-life costs of medical care for advanced stage cancer patients.

Vojnosanit Pregl 2015 Apr;72(4):334-41

Background/aim: Cancer, one of the leading causes of mortality in the world, imposes a substantial economic burden on each society, including Serbia. The aim of this study was to evaluate the major cancer cost drivers in Serbia.

Methods: A retrospective, in-depth, bottom-up analysis of two combined databases was performed in order to quantify relevant costs. End-of-life data were obtained from patients with cancer, who deceased within the first year of the established diagnose, including basic demographics, diagnosis, tumour histology, medical resource use and related costs, time and cause of death. All costs were allocated to one of the three categories of cancer health care services: primary care (included home care), hospital outpatient and hospital inpatient care.

Results: Exactly 114 patients were analyzed, out of whom a high percent (48.25%) had distant metastases at the moment of establishing the diagnosis. Malignant neoplasms of respiratory and intrathoracic organs were leading causes of morbidity. The average costs per patient were significantly different according to the diagnosis, with the highest (13,114.10 EUR) and the lowest (4.00 EUR) ones observed in the breast cancer and melanoma, respectively. The greatest impact on total costs was observed concerning pharmaceuticals, with 42% of share (monoclonal antibodies amounted to 34% of all medicines and 14% of total costs), followed by oncology medical care (21%), radiation therapy and interventional radiology (11%), surgery (90%), imaging diagnostics (9%) and laboratory costs (8%). CONCLUSION. Cancer treatment incurs high costs, especially for end-of-life pharmaceutical expenses, ensued from medical personnel tendency to improve such patients' quality of life in spite of nearing the end of life. Reimbursement policy on monoclonal antibodies, in particular at end-stage disease, should rely on cost-effectiveness evidence as well as documented clinical efficiency.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.2298/vsp1504334kDOI Listing
April 2015

A Cost-effectiveness Analysis of Ferric Carboxymaltose in Patients With Iron Deficiency and Chronic Heart Failure in Spain.

Rev Esp Cardiol (Engl Ed) 2015 Oct 28;68(10):846-51. Epub 2015 Jan 28.

Department of Heart Diseases, Wroclaw Medical University, Wroclaw, Poland.

Introduction And Objectives: Treatment with ferric carboxymaltose improves symptoms, functional capacity, and quality of life in patients with chronic heart failure and iron deficiency. The aim of this study was to assess the cost-effectiveness of ferric carboxymaltose treatment vs no treatment in these patients.

Methods: We used an economic model based on the Spanish National Health System, with a time horizon of 24 weeks. Patient characteristics and ferric carboxymaltose effectiveness (quality-adjusted life years) were taken from the Ferinject® Assessment in patients with IRon deficiency and chronic Heart Failure trial. Health care resource use and unit costs were taken either from Spanish sources, or from the above mentioned trial.

Results: In the base case analysis, patients treated with and without ferric carboxymaltose treatment acquired 0.335 and 0.298 quality-adjusted life years, respectively, representing a gain of 0.037 quality-adjusted life years for each treated patient. The cost per patient was €824.17 and €597.59, respectively, resulting in an additional cost of €226.58 for each treated patient. The cost of gaining 1 quality adjusted life year with ferric carboxymaltose was €6123.78. Sensitivity analyses confirmed the robustness of the model. The probability of ferric carboxymaltose being cost-effective (< €30 000 per quality-adjusted life year) and dominant (more effective and lower cost than no treatment) was 93.0% and 6.6%, respectively.

Conclusions: Treatment with ferric carboxymaltose in patients with chronic heart failure and iron deficiency, with or without anemia, is cost-effective in Spain.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.rec.2014.10.010DOI Listing
October 2015

Determinants of quality of life of patients with heart failure and iron deficiency treated with ferric carboxymaltose: FAIR-HF sub-analysis.

Int J Cardiol 2013 Oct 17;168(4):3878-83. Epub 2013 Jul 17.

Institute of Pharmaceutical Medicine/ECPM, Universität Basel, Basel, Switzerland. Electronic address:

Background: Heart failure (HF) is a burden to patients and health care systems. The objectives of HF treatment are to improve health related quality of life (HRQoL) and reduce mortality and morbidity. We aimed to evaluate determinants of health-related quality of life (HRQoL) in patients with iron deficiency and HF treated with intravenous (i.v.) iron substitution or placebo.

Methods: A randomised, double-blind, placebo-controlled trial (n = 459) in iron-deficient chronic heart failure (CHF) patients with or without anaemia studied clinical and HRQoL benefits of i.v. iron substitution using ferric carboxymaltose (FCM) over a 24-week trial period. Multivariate analysis was carried out with various clinical variables as independent variables and HRQoL measures as dependent variables.

Results: Mean change from baseline of European Quality of Life - 5 Dimensions (EQ-5D) (value set-based) utilities (on a 0 to 100 scale) at week 24 was 8.91 (i.v. iron) and 0.68 (placebo; p < 0.01). In a multivariate analysis excluding baseline HRQoL, a higher exercise tolerance and i.v. iron substitution positively influenced HRQoL, whereas impaired renal function and a history of stroke had a negative effect. The level of HRQoL was also influenced by country of residence. When baseline HRQoL was factored in, the multivariate model remained stable.

Conclusion: In this study, i.v. iron substitution, exercise tolerance, stroke, country of residence and renal function influenced measures of HRQoL in patients with heart failure and iron deficiency.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.ijcard.2013.06.045DOI Listing
October 2013

Health economic assessment of ferric carboxymaltose in patients with iron deficiency and chronic heart failure based on the FAIR-HF trial: an analysis for the UK.

Eur J Heart Fail 2012 Jul 11;14(7):782-90. Epub 2012 Jun 11.

Institute of Pharmaceutical Medicine/ECPM, Universität Basel, CH-4056 Basel, Switzerland.

Aims: The purpose of this study was to evaluate the cost-effectiveness of iron repletion using intravenous (i.v.) ferric carboxymaltose (FCM) in chronic heart failure (CHF) patients with iron deficiency with or without anaemia. Cost-effectiveness was studied from the perspective of the National Health Service in the UK.

Methods And Results: A model-based cost-effectiveness analysis was used to compare iron repletion with FCM with no iron treatment. Using data from the FAIR-HF trial and publicly available sources and publications, per patient costs and clinical effectiveness of FCM were estimated compared with placebo. Cost assessment was based on study drug and administration costs, cost of CHF treatment, and hospital length of stay. The incremental cost-effectiveness ratio (ICER) of FCM use was expressed as cost per quality-adjusted life year (QALY) gained, and sensitivity analyses were performed on the base case. The time horizon of the analysis was 24 weeks. Mean QALYs were higher in the FCM arm (difference 0.037 QALYs; bootstrap-based 95% confidence interval 0.017-0.060). The ICER of FCM compared with placebo was €4414 per QALY gained for the FAIR-HF dosing regimen. Sensitivity analyses confirmed the base case result to be robust.

Conclusion: From the UK payers' perspective, managing iron deficiency in CHF patients using i.v. FCM was cost-effective in this analysis. The base case ICER was clearly below the threshold of €22 200-€33 300/QALY gained (£20 000-£30 000) typically used by the UK National Institute for Health and Clinical Excellence and proved to be robust in sensitivity analysis. Improved symptoms and better quality of life contributed to this result.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1093/eurjhf/hfs083DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3380546PMC
July 2012

A questionnaire on treatment satisfaction and disease specific knowledge among patients with acute coronary syndrome. I: Are treatment satisfaction and disease specific knowledge continuous latent traits?

Patient Educ Couns 2012 Mar 29;86(3):360-5. Epub 2011 Jun 29.

Carinthia University of Applied Sciences, Feldkirchen, Austria.

Objective: To assess psychometric properties of a questionnaire covering treatment satisfaction and disease-specific knowledge among patients discharged after treatment for acute coronary syndrome.

Methods: Comparative scaling of the questionnaire using latent class analysis (LCA) and exploratory factor analysis (EFA) in a consecutive sample of 2015 patients.

Results: LCA revealed four qualitatively differing patterns of patients' knowledge and five distinct patterns of treatment satisfaction. EFA for patients' knowledge identified four uncorrelated "dimensions". Patient satisfaction was scored in a two-factor solution despite proven heterogeneity of persons.

Conclusions: LCA was helpful to identify classes of patients that cannot be scaled according to a latent trait model.

Practice Implications: Specific patterns of insufficient disease-specific knowledge and satisfaction requiring specific interventions became visible.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.pec.2011.06.003DOI Listing
March 2012

A questionnaire on treatment satisfaction and disease specific knowledge among patients with acute coronary syndrome. II: Insights for patient education and quality improvement.

Patient Educ Couns 2012 Mar 29;86(3):366-71. Epub 2011 Jun 29.

Department of Healthcare Management, Carinthia University of Applied Sciences, Feldkirchen, Austria.

Objective: Secondary prevention for coronary heart disease is achieved by pharmaceutical control of risk factors and patients' own self management behaviour. To comply with longterm treatment patients need sufficient knowledge of their condition and should be satisfied with acute care. Therefore a questionnaire measuring both issues was constructed.

Methods And Results: Latent class analysis applied in a sample of over 2000 patients suffering from acute coronary syndrome revealed 4 configurative patterns of knowledge and 5 distinct patterns of (dis-)satisfaction. Nearly 50% of all patients displayed insufficient knowledge upon discharge. Deficits clustered around misinformation on necessary lifestyle changes versus dysfunctional strategies for future emergency situations. Satisfaction and disease specific knowledge were interrelated in complex patterns.

Conclusions: Disease specific knowledge and satisfaction with treatment proved to be psychometrically valid indicators of the quality of the treatment process that might also have an impact on outcome.

Practice Implications: A validated questionnaire is ready for routine administration after discharge of patients with acute coronary syndrome from acute hospital care. Patient education efforts and quality improvement in treatment centres might be effectively monitored using this questionnaire.
View Article and Find Full Text PDF

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.pec.2011.06.004DOI Listing
March 2012
-->