Costs of Medical Care Among Augmentation Therapy Users and Non-Users with Alpha-1 Antitrypsin Deficiency in the United States

Abstract

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

“…Consistent with other studies that have reported that approximately 13% of patients with AATD receive augmentation therapy [16,17], only 15.1% of patients in the current study had been prescribed augmentation therapy. From a clinical perspective, the low utilization of a1-PI augmentation therapy could be partially accounted for by the multiple medical conditions for which augmentation therapy is not recommended by Sandhaus et al, including having certain genotypes of AATD, having lung disease and currently smoking, having emphysema or bronchiectasis but not airflow obstruction, being treated for liver disease due to AATD, having undergone liver transplantation, and the presence of liver disease [20].…”

“…From a clinical perspective, the low utilization of a1-PI augmentation therapy could be partially accounted for by the multiple medical conditions for which augmentation therapy is not recommended by Sandhaus et al, including having certain genotypes of AATD, having lung disease and currently smoking, having emphysema or bronchiectasis but not airflow obstruction, being treated for liver disease due to AATD, having undergone liver transplantation, and the presence of liver disease [20]. From an economic perspective, the cost of augmentation therapy is high, with 1 report indicating that the difference in annual costs between recipients and nonrecipients of augmentation therapy exceeds $100,000 in the United States; 75% of that cost difference is attributable to augmentation therapy [16]. It is noteworthy, however, that a higher percentage of patients with a severe AATD clinical course received augmentation therapy.…”

“…A severe clinical course of AATD can lead to increased healthcare resource use (HRU) and healthcare costs [15][16][17]. In a retrospective study examining HRU among Medicare beneficiaries, higher percentages of patients diagnosed with AATD compared with COPD visited the emergency room (ER; 58.4% vs. 42.5%) and had inpatient visits (58.0% vs. 19.5%) [17].…”

“…The American Thoracic Society/European Respiratory Society and the Chronic Obstructive Pulmonary Disease Foundation recommend augmentation therapy with a1-protease inhibitors (PIs) or nonspecific therapy for the treatment of some individuals with AATD-associated pulmonary issues [19,20]. Published reports indicate that roughly 13% of patients with AATD receive augmentation therapy [16,17], signaling that it may be underutilized.…”

“…Together, the delayed and low rates of AATD diagnosis [6,7,21,22], high HRU burden of AATD [15][16][17], and low utilization of augmentation therapy [16,17] emphasize the need for improved diagnosis and treatment of AATD. Such improvements may mitigate the burden of AATD on HRU and healthcare costs.…”