Date of Award
Open Access Thesis
Master of Public Health (MPH)
School of Public Health
Background: Alpha-1 antitrypsin deficiency (AATD) is a rare disease caused by a genetic mutation in the SERPINA1 gene which causes low levels of or no working alpha-1 antitrypsin protein to be made. This results in a number of clinical manifestations, most prominently pulmonary issues such as emphysema. An intravenous infusion of human plasma-derived AAT (augmentation therapy) is sometimes prescribed to patients with emphysema, but there is a need for a greater understanding of the resulting economic burden in this patient population.
Methods: A retrospective observational cohort study was conducted using the PearlDiver Mariner Patient Claims Database to analyze healthcare costs of adult AATD patients with emphysema. Total costs for AATD-emphysema patients who have not taken augmentation therapy at any point were calculated. Augmentation therapy costs and non-therapy related healthcare costs were calculated for all augmentation therapy users, patients who received therapy on a weekly basis, and patients who received therapy monthly. Costs for each group were compared using nonparametric Wilcoxon rank sum tests, and a linear regression model was used to further assess the association between treatment frequency (weekly vs. monthly) and non-therapy related healthcare costs.
Results: Non-augmentation therapy users had a total average annual cost of $17,739, whereas all augmentation users had an average non-therapy cost of $23,753. Weekly augmentation therapy users had higher augmentation therapy costs compared to monthly users due to increased frequency but also still had significantly higher remaining non-therapy costs of $32,863 compared to monthly augmentation therapy users who had non-therapy costs of $23,696. Augmentation therapy users had a significantly higher average length of stay compared to non-users, though there was not a significant difference in average length of stay between the weekly and monthly augmentation therapy users.
Conclusions: Non-augmentation therapy users had a total average annual cost of $17,739, while all augmentation users had an average non-therapy cost of $23,753. Weekly augmentation therapy users had higher augmentation therapy costs due to increased frequency but also still had significantly higher remaining non-therapy costs of $32,863, whereas monthly augmentation therapy users had non-therapy costs of $23,696. Given the retrospective and observational nature of the study, it is possible that the association we observed between therapy frequency and resulting non-therapy healthcare costs is due to selection bias. Further research employing prospective study designs and approaches to assess for and mitigate potential selection bias is necessary in order to better understand how augmentation therapy influences economic burden for AATD patients with severe disease manifestations.
Raina, Nikita, "Healthcare Costs For Patients With Severe Alpha-1 Antitrypsin Deficiency Among Augmentation Therapy Users And Non-Users And Impact Of Therapy Frequency" (2023). Public Health Theses. 2330.
This Article is Open Access