Economic Burden of Overall and Advanced Light Chain Amyloidosis: Results from a Claims Linked Electronic Health Record Database Analysis

Jeffrey A Thompson,1 Ankita Gupta,2 Julia Catini,3 Pedro A Laires4,5 1Global Health Outcomes and Economics Research, Alexion, AstraZeneca Rare Disease, Boston, MA, USA; 2Optum, New Delhi, India; 3Global Medical Affairs, Alexion, AstraZeneca Rare Disease, Wilmington, DE, USA; 4Epidemiology and Real World Science, Alexion, AstraZeneca Rare Disease, Barcelona, Spain; 5National School of Public Health, Public Health Research Center, Universidade NOVA de Lisboa, Lisbon, PortugalCorrespondence: Jeffrey A Thompson, Global Health Outcomes and Economics Research, Alexion, AstraZeneca Rare Disease, Boston, MA, 02210, USA, Tel +1-857-205-5537, Email jeffrey.thompson@alexion.comBackground: Real-world data on healthcare resource utilization (HCRU) and costs in light chain amyloidosis (AL) by disease severity are limited. This study evaluates HCRU and costs among patients with AL, particularly those with advanced stages.Methods: A retrospective analysis of deidentified patient records (January 2016 to March 2022) from Optum’s clinical electronic health record (EHR) database was conducted. Adult patients with ≥ 2 AL diagnoses or positive mentions of the disease in the physician’s notes (≥ 30 days apart) were included. Patients with a Mayo disease stage, including those with advanced disease (stages IIIa/IIIb), based on cardiac biomarkers (N-terminal pro-brain natriuretic peptide and troponin T), were identified most proximal to the index date (first-identified AL diagnosis date). Patient records were then linked to payer-agnostic claims. Data, including HCRU and costs, were assessed from the index date until the end of continuous enrollment or death (minimum 30-day follow-up) during the 12-month postindex period.Results: Among the 85 patients (75% aged ≥ 60 years; 65% males) included in the study, 60% had at least one condition-related inpatient hospitalization (66.7% with advanced cardiac involvement), 92.9% had an outpatient visit (54.4% with advanced cardiac involvement), 74.1% had an office visit (53.9% with advanced cardiac involvement), and 22.4% had an emergency room visit (68.4% with advanced cardiac involvement) within 12 months after diagnosis. The median (range) per-patient per-month (PPPM) condition-related total, medical, and drug costs for patients with advanced cardiac involvement (n = 47) were $7532 ($3336–$15,097), $2107 ($1079–$4618), and $4613 ($1664–$9488), respectively. The median (range) PPPM condition-related inpatient hospitalization costs for patients with advanced cardiac involvement (n = 34) was $462 ($77–$1839).Conclusion: This EHR-claims linked database study showed that patients with advanced disease face high economic burden, supporting the continued need for therapeutic advances to help manage this population.Keywords: light chain amyloidosis, healthcare resource utilization, Mayo stage IIIa, Mayo stage IIIb, economic burden