Abstract
BackgroundAlpha-1-Antitrypsin Deficiency (AATD) is an economically unexplored genetic disease.MethodsDirect and indirect costs (based on self-reported information on healthcare utilization) and health-related quality of life (HRQL, as assessed by SGRQ, CAT, and EQ-5D-3 L) were compared between 131 AATD patients (106 with, 25 without augmentation therapy (AT)) and 2,049 COPD patients without AATD participating in the COSYCONET COPD cohort. The medication costs of AT were excluded from all analyses to reveal differences associated with morbidity profiles. The association of AATD (with/without AT) with costs or HRQL was examined using generalized linear regression modelling (GLM) adjusting for age, sex, GOLD grade, BMI, smoking status, education and comorbidities.ResultsAdjusted mean direct annual costs were €6,099 in AATD patients without AT, €7,117 in AATD patients with AT (excluding costs for AT), and €7,460 in COPD patients without AATD. AATD with AT was significantly associated with higher outpatient (+273%) but lower inpatient (−35%) and medication costs (−10%, disregarding AT) compared with COPD patients without AATD. There were no significant differences between groups regarding indirect costs and HRQL.ConclusionApart from AT costs, AATD patients tended to have lower, though not significant, overall costs and similar HRQL compared to COPD patients without AATD. AT was not associated with lower costs or higher HRQL.Trial registrationNCT01245933
Highlights
Alpha-1-Antitrypsin Deficiency (AATD) is an economically unexplored genetic disease
As there is an ongoing controversy about the efficacy of augmentation therapy (AT) therapy [1, 15], we examined differences regarding direct and indirect costs and health-realted quality of life (HRQL) between augmented and nonaugmented Alpha-1-antitrypsin deficiency (AATD) patients
For Global Initiative for Chronic Obstructive Pulmonary Disease (GOLD) staging, reference values according to the Global Lung Initiative (GLI) were used [18]. 450 subjects with a Tiffeneau index above 70% at baseline examination were excluded from this analysis (Fig. 1)
Summary
Alpha-1-Antitrypsin Deficiency (AATD) is an economically unexplored genetic disease. Alpha-1-antitrypsin (AAT) is an anti-protease that protects tissues – mainly lung parenchyma – from damage by enzymes released from inflammatory cells. The prevalence of deficiency related alleles (PI*ZZ and PI*SZ) in the world population is estimated to be 0.1% for the PI*ZZ and 0.7% for the PI*SZ genotype [7]. COPD patients with underlying AATD differ from COPD patients without this deficiency e.g. with regard to smoking history, comorbidities and exercise-related desaturation in arterial blood [10, 11]. Comorbidities, which have been identified as a major driver of excess costs as well as of loss in health-realted quality of life (HRQL) in COPD patients [13, 14], may differ between patients with and without AATD
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
