Alpha-1 Antitrypsin Deficiency-Associated Clinical Manifestations and Healthcare Resource Use in the United States

Abstract

Pulmonary events (PEs) associated with alpha-1 antitrypsin deficiency (AATD) can have a severe clinical course and increase healthcare resource use (HRU). However, AATD-associated HRU and healthcare costs have not been extensively described. This study describes and compares realworld HRU and healthcare costs among US patients with severe (requiring hospitalization after AATD-related PE) versus nonsevere AATD clinical course. Administrative healthcare claims for patients with a second primary AATD diagnosis between 6/1/2008 and 12/31/2017 were analyzed from 2 databases (requiring continuous enrollment 6 months preceding diagnosis). Patient baseline characteristics and AATD-associated PE incidence rates, HRU, and healthcare costs during follow-up were compared in patients with severe versus nonsevere AATD. Of 5109 patients with a second AATD diagnosis, 2674 (severe, n ¼ 711 [26.6%]; nonsevere, n ¼ 1963 [73.4%]) had !1 AATD-associated PE. PE incidence per 100 person-years was higher in patients with severe versus nonsevere AATD. Annual incidences (mean ± SD) of emergency department (1.2 ± 5.7 vs. 0.4 ± 1.2), inpatient (1.3 ± 2.5 vs. 0.1 ± 0.5), and outpatient (10.3 ± 15.9 vs. 5.7 ± 13.2) visits were higher in patients with severe versus nonsevere AATD. Median (interquartile range) annual costs were also higher for patients with severe versus nonsevere AATD for emergency department ($185

Disease burden associated with alpha-1 antitrypsin deficiency: systematic and structured literature reviews

“…Estimates of the total direct healthcare cost of AATD reviewed here came mainly from the USA and suggested median annual costs of USD 9753 excluding AAT therapy [43]. In Europe, annual costs were approximately EUR 1000 higher for patients who received AAT therapy versus those who did not [75].…”

“…Many studies in this review included <100 patients, which is expected given the rarity of AATD, and study follow-up also tended to be relatively short, with many prospective studies assessing patients for ⩽1 year. Only the larger registries [25,26,122] or retrospective, population-based studies [43,60,64,109] were able to include a robust sample size and consider a longer time frame. Moreover, many studies were published only as conference abstracts, particularly those discussing the clinical burden (20 out of 40 studies) and economic burden (nine out of 21), limiting the details available for analysis.…”

“…Of the 38 included studies, all reported data regarding specific morbidities in patients with AATD including pulmonary, liver and skin disease; overall, 50 different morbidities were reported (table 1) [23][24][25][26][27][28][29][30]. Pulmonary morbidity was commonly reported, and included COPD with a prevalence of 36.3-75.8% [25,26,33,43,49], emphysema (14.4-53.8%) [29,43,46,47] and bronchiectasis (3.8-73.1%) (table 1) [29,[45][46][47].…”

“…Resource use overall was higher in patients with AATD diagnosed with COPD versus those with general COPD [75,[106][107][108] and those with more severe disease [43]. AlphaNet's Disease Management and Prevention Program in the USA reported greater resource use ( primary care, lung specialist visits and hospitalisation) for patients with PiSZ versus PiZZ genotypes who had lung disease, but the authors noted that this may have been due to the latter having better adherence to management recommendations and maintaining a healthier lifestyle [44].…”

“…The annual direct medical cost for patients with AATD in the USA was estimated to be USD 127 537 for users of AAT therapy versus USD 15 874 for nonusers [105]. A second US study reported that the median annual total healthcare costs were USD 9753, and the median total medical costs were USD 4927 and total pharmacy costs were USD 2063, although the proportion of patients receiving AAT therapy was not reported [43] (table 5). In Germany, the mean annual direct medical costs per patient were EUR 6099 for users of AAT therapy versus EUR 7117 for nonusers, excluding costs for AAT therapy [75].…”