HEALTHCARE AND ECONOMIC BURDEN OF ALPHA-1 ANTITRYPSIN DEFICIENCY (AATD)-RELATED EMPHYSEMA VERSUS NON-AATD EMPHYSEMA IN THE UNITED STATES (US): A MATCHED COHORT STUDY USING REAL-WORLD CLAIMS DATA
Author(s)
Gerasimos Konidaris, MSc1, SHRAVAN S G, BE, MBA2, Kalyani Hawaldar, MS3, Gandarvaka Miles, PHD, MPH3.
1Sanofi, Reading, United Kingdom, 2Sanofi, Hyderabad, India, 3Sanofi, Cambridge, MA, USA.
1Sanofi, Reading, United Kingdom, 2Sanofi, Hyderabad, India, 3Sanofi, Cambridge, MA, USA.
OBJECTIVES: This study compared all-cause and respiratory-related healthcare resource utilisation (HCRU) and costs between AATD-related emphysema and non-AATD emphysema cohorts in the US.
METHODS: A retrospective matched cohort study was performed using administrative claims from the MerativeTM MarketScan® Research Databases (January 2016-June 2024). Adult patients with non-AATD emphysema were matched to those with AATD-related emphysema on propensity score and exact-matched (3:1) on age, sex, insurance type at index (first observed emphysema diagnosis), and calendar time. The outcomes included all-cause and respiratory-related HCRU and costs. Mean differences (MDs) in the number of visits and non-zero costs (per-patient-per-year [PPPY] and 95% confidence intervals [CIs]) were calculated using negative binomial regression and generalised linear modelling with a gamma distribution and log link, respectively.
RESULTS: The matched cohort included 384 patients with AATD-related emphysema and 1,152 patients with non-AATD emphysema (mean [standard deviation] age: 58.00 [11.40] years). Baseline asthma prevalence was higher in AATD-related emphysema cohort than non-AATD emphysema cohort (28.4% versus 15.9%; standardised MD: 0.31). During follow-up, patients with AATD-related emphysema had more all-cause outpatient visits (MD PPPY [95% CI]: 4.81 [2.75, 7.03]) and fewer emergency room (ER) visits (MD PPPY [95% CI]: −0.29 [−0.49, −0.08]), on average, versus patients with non-AATD emphysema. All-cause healthcare costs were higher in AATD-related emphysema cohort than non-AATD emphysema cohort (MD non-zero costs PPPY [95% CI]: $36,870.40 [$24,810.02, $49,235.70]). Respiratory-related hospitalisation (MD PPPY [95% CI]: 0.12 [0.04, 0.21]), outpatient visits (MD PPPY [95% CI]: 4.05 [3.36, 4.79]), ER visits (MD PPPY [95% CI]: 0.29 [0.15, 0.45]), and healthcare costs (MD non-zero costs PPPY [95% CI]: $16,391.30 [$8,653.11, $25,509.52]) were higher in AATD-related emphysema cohort than non-AATD emphysema cohort.
CONCLUSIONS: This study demonstrated substantial HCRU and economic burden in AATD-related emphysema versus non-AATD emphysema in the US. Earlier diagnosis and improved treatment may offer better disease control and reduce healthcare burden.
METHODS: A retrospective matched cohort study was performed using administrative claims from the MerativeTM MarketScan® Research Databases (January 2016-June 2024). Adult patients with non-AATD emphysema were matched to those with AATD-related emphysema on propensity score and exact-matched (3:1) on age, sex, insurance type at index (first observed emphysema diagnosis), and calendar time. The outcomes included all-cause and respiratory-related HCRU and costs. Mean differences (MDs) in the number of visits and non-zero costs (per-patient-per-year [PPPY] and 95% confidence intervals [CIs]) were calculated using negative binomial regression and generalised linear modelling with a gamma distribution and log link, respectively.
RESULTS: The matched cohort included 384 patients with AATD-related emphysema and 1,152 patients with non-AATD emphysema (mean [standard deviation] age: 58.00 [11.40] years). Baseline asthma prevalence was higher in AATD-related emphysema cohort than non-AATD emphysema cohort (28.4% versus 15.9%; standardised MD: 0.31). During follow-up, patients with AATD-related emphysema had more all-cause outpatient visits (MD PPPY [95% CI]: 4.81 [2.75, 7.03]) and fewer emergency room (ER) visits (MD PPPY [95% CI]: −0.29 [−0.49, −0.08]), on average, versus patients with non-AATD emphysema. All-cause healthcare costs were higher in AATD-related emphysema cohort than non-AATD emphysema cohort (MD non-zero costs PPPY [95% CI]: $36,870.40 [$24,810.02, $49,235.70]). Respiratory-related hospitalisation (MD PPPY [95% CI]: 0.12 [0.04, 0.21]), outpatient visits (MD PPPY [95% CI]: 4.05 [3.36, 4.79]), ER visits (MD PPPY [95% CI]: 0.29 [0.15, 0.45]), and healthcare costs (MD non-zero costs PPPY [95% CI]: $16,391.30 [$8,653.11, $25,509.52]) were higher in AATD-related emphysema cohort than non-AATD emphysema cohort.
CONCLUSIONS: This study demonstrated substantial HCRU and economic burden in AATD-related emphysema versus non-AATD emphysema in the US. Earlier diagnosis and improved treatment may offer better disease control and reduce healthcare burden.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
RWD103
Topic
Economic Evaluation, Patient-Centered Research, Real World Data & Information Systems
Topic Subcategory
Health & Insurance Records Systems
Disease
Rare & Orphan Diseases, Respiratory-Related Disorders (Allergy, Asthma, Smoking, Other Respiratory)