ANALYSIS OF PULMONARY-RELATED CLINICAL MANIFESTATIONS AND HEALTH RESOURCE UTILIZATION IN PATIENTS WITH SEVERE AND NON-SEVERE ALPHA-1 ANTITRYPSIN DEFICIENCY IN THE UNITED STATES

Abstract

SESSION TITLE: Monday Electronic Posters 1 SESSION TYPE: Original Inv Poster Discussion PRESENTED ON: 10/21/2019 02:30 PM - 03:15 PM PURPOSE: Pulmonary manifestations (PM) of alpha-1 antitrypsin deficiency (AATD) can inflict a severe clinical course for patients and require increased healthcare resource use (HRU). This study aimed to describe the real-world burden of AATD with PM in the U.S. METHODS: Patient-level healthcare claims from the Truven Health MarketScan® database were retrospectively analyzed. Patients with a second primary diagnosis of AATD between 6/1/2008-12/31/2017 and continuous enrollment in the 6 months preceding the second diagnosis date were included. Patient characteristics and AATD-associated PM events of Chronic Obstructive Pulmonary Disease (COPD), chronic bronchitis, emphysema and bronchiectasis were assessed among all identified patients. All-cause HRU was assessed separately among patients with severe PM (≥1 event with hospitalization within 1 calendar day) and non-severe PM (events with no hospitalization within 1 calendar day). Incidence rates (IR) and associated 95% confidence intervals (CI) of the defined PM and HRU were measured from day 1 after the date of hospital discharge for severe events and the date of first PM event claim for non-severe events to the end of data or continuous eligibility. IR were summarized descriptively and compared using standardized differences (d). RESULTS: Of 5109 cases, 2674 (52.3%) had ≥1 of the defined PM events. Severe AATD patients (N=711) were older (mean age 57.9 vs 52.8 years; d=0.37) vs non-severe patients (N=1963), had a greater baseline morbidity (higher mean CCI score [2.2 vs 1.4; d=0.497]), had a higher prevalence of baseline COPD (75.8% vs 56.9%; d=0.41) and emphysema (33.8% vs 23.8%; d=0.22), and increased hospitalization frequency (mean 0.7 vs 0.13; d=0.70). Pulmonary exacerbations were the most frequent manifestation. IR for severe exacerbations was 522.4 (95% CI: 485.4-562.2) and 231.6 (221.6-242.1) for non-severe per 100 person-years. The IR for severe and non-severe COPD, chronic bronchitis, emphysema, and bronchiectasis were: 234.5 [216.8-253.6] and 82.3 [78.2-86.6], 65.9 [60.4-71.8] and 26.6 [24.8-28.4], 46.8 [42.5-51.6] and 20.4 [18.9-22.0], and 7.9 [6.6-9.5] and 4.3 [3.7-5.0] per 100 person-years respectively. Patients with severe PE had intensified HRU based on significantly higher mean annual frequencies of emergency room (1.2 vs 0.5; d=0.16), inpatient (1.3 vs 0.08; d=0.68), outpatient (10.3 vs 6.4; d=0.28), other visits (4.0 vs 2.0; d=0.21), and significantly longer hospitalizations (mean, 8.8 days vs 5.4 days; d=0.49). CONCLUSIONS: Patients with severe AATD-related PM requiring hospitalization are substantially burdened by more frequent events, more severe clinical course and higher HRU. CLINICAL IMPLICATIONS: These findings characterize patients with pulmonary-related AATD and highlight the importance of strategies to reduce the frequency and severity of PM through disease modification. DISCLOSURES: Employee relationship with Takeda Pharmaceuticals Please note: >$100000 Added 03/18/2019 by Emily Brouwer, source=Web Response, value=Stocks Employee relationship with Takeda Please note: >$100000 Added 03/18/2019 by Emily Brouwer, source=Web Response, value=Salary Employee relationship with Takeda Pharmaceuticals Please note: $1-$1000 Added 03/15/2019 by Corey Joseph, source=Web Response, value=I am an employee of Takeda Employee relationship with Takeda Pharmaceutical Company Limited Please note: >$100000 Added 03/12/2019 by Ernesto Mayen Herrera, source=Web Response, value=Salary