Health utilities in individuals with alpha-1 antitrypsin deficiency with and without COPD from the EARCO registry.

چکیده اصلی

BACKGROUND: Data on health utilities in Alpha-1 antitrypsin deficiency (AATD) are scarce. This study aimed to evaluate health-related quality of life (HRQoL) using the EQ-5D in a cohort of AATD Pi*ZZ individuals from the European AATD Research Collaboration (EARCO) registry and to identify factors associated with impaired health utilities. METHODS: Observational, cross-sectional, multicenter study including AATD patients with Pi*ZZ genotype enrolled up to April 2025. HRQoL was assessed using the EQ-5D questionnaire (index and visual analogue scale (VAS)). Sociodemographic data, smoking history, respiratory symptoms, comorbidities, lung function and exacerbations were collected. Multivariable generalised linear models were used to identify factors independently associated with EQ-5D index scores in the overall population and in patients with AATD-COPD. RESULTS: A total of 1710 Pi*ZZ individuals were analysed with a mean age 55.2 ± 13.8 years, 51% were male and 49.6% had COPD. Mean EQ-5D index and VAS were 0.82 ± 0.22 and 62.9 ± 25.9, respectively. Patients with COPD had significantly worse EQ-5D index (0.75 ± 0.24 vs 0.90 ± 0.16) and VAS scores (56.3 ± 24.4 vs 70.4 ± 26.8; both p < 0.001). In multivariable analyses, higher symptom burden (CAT, mMRC), long-term oxygen therapy, being unemployed, exacerbations, COPD, bronchiectasis and lower educational level were independently associated with worse EQ-5D scores in Pi*ZZ subjects. CONCLUSIONS: HRQoL was substantially impaired in Pi*ZZ individuals, particularly in those with COPD. Symptom burden and exacerbations were stronger determinants of EQ-5D than lung function alone in all Pi*ZZ patients. Patients with AATD-associated COPD exhibited an HRQoL impairment comparable to that observed in usual COPD populations, but at a younger age.

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