Burden of Systemic Light-Chain (AL) Amyloidosis by Mayo Stage: Results From an Electronic Health Record (EHR) Linked To Claims Analysis
Author(s)
Jeffrey A. Thompson, MS, PhD1, Pedro A. Laires, PhD1, Ankita Gupta, MS2, Julia Catini, PharmD, MBA1;
1Alexion, AstraZeneca Rare Disease, Boston, MA, USA, 2Optum, Eden Prairie, MN, USA
1Alexion, AstraZeneca Rare Disease, Boston, MA, USA, 2Optum, Eden Prairie, MN, USA
Presentation Documents
OBJECTIVES: Light-chain amyloidosis (AL) is a rare hematologic disorder in which misfolded light-chain fibrils produced by clonal plasma cells aggregate, deposit, and cause organ dysfunction. Limited evidence exists on the real-world cost and healthcare resource utilization (HCRU) amongst AL patients when stratified by Mayo 2004 European Modification disease-severity staging system. The objective of this study was to address this gap.
METHODS: A retrospective analysis was conducted using deidentified patient records from Optum's Clinical EHR database that included adults with ≥2 AL diagnosis (ICD-10-CM: E85.81) or AL mentions in physician’s notes (≥30 days apart). NT-proBNP and troponin were then operationalized to assign European Modification Mayo stage most proximal to first-identified index AL diagnosis. Patient records were then deterministically linked to payer-agnostic claims, where costs/HCRU were assessed during the maximum follow-up time post-index while patients had continuous medical and prescription enrollment. Outcomes included all-cause/AL-related: per-patient-per-month (PPPM) total, medical, and prescription costs, as well as mean inpatient, outpatient, office, and emergency-room visits. Results were stratified by all patients, AL-treated patients, all patients with cardiac-involvement, and AL-treated patients with cardiac-involvement.
RESULTS: 215 patients with linked EHR+claims data and an identifiable Mayo Stage were included: 53-Stage 1, 79-Stage 2, 31-Stage 3a, and 52-Stage 3b. The highest costs were seen in patients receiving treatment, where Stage 3a patients had PPPM all-cause/AL-related total, medical, and prescription mean costs of $26,338/$14,116; $15,601/$4,772; and $10,737/$9,345; respectively. Treated Stage 3b patients had PPPM all-cause/AL-related total, medical, and prescription mean costs of $19,354/$11,357; $11,255/$4,453 and $8,099/$7,286; respectively. Amongst the Stage 3a patients 19/23(83%) and 17/23(74%), and amongst Stage 3b patients, 27/35(77%) and 20/35(57%), had at least one all-cause/AL-related inpatient hospitalization during follow-up, respectively.
CONCLUSIONS: AL is a severe condition with high burden amongst patients with higher levels of disease severity, supporting the continued need for therapeutic advances to help treat these patient populations.
METHODS: A retrospective analysis was conducted using deidentified patient records from Optum's Clinical EHR database that included adults with ≥2 AL diagnosis (ICD-10-CM: E85.81) or AL mentions in physician’s notes (≥30 days apart). NT-proBNP and troponin were then operationalized to assign European Modification Mayo stage most proximal to first-identified index AL diagnosis. Patient records were then deterministically linked to payer-agnostic claims, where costs/HCRU were assessed during the maximum follow-up time post-index while patients had continuous medical and prescription enrollment. Outcomes included all-cause/AL-related: per-patient-per-month (PPPM) total, medical, and prescription costs, as well as mean inpatient, outpatient, office, and emergency-room visits. Results were stratified by all patients, AL-treated patients, all patients with cardiac-involvement, and AL-treated patients with cardiac-involvement.
RESULTS: 215 patients with linked EHR+claims data and an identifiable Mayo Stage were included: 53-Stage 1, 79-Stage 2, 31-Stage 3a, and 52-Stage 3b. The highest costs were seen in patients receiving treatment, where Stage 3a patients had PPPM all-cause/AL-related total, medical, and prescription mean costs of $26,338/$14,116; $15,601/$4,772; and $10,737/$9,345; respectively. Treated Stage 3b patients had PPPM all-cause/AL-related total, medical, and prescription mean costs of $19,354/$11,357; $11,255/$4,453 and $8,099/$7,286; respectively. Amongst the Stage 3a patients 19/23(83%) and 17/23(74%), and amongst Stage 3b patients, 27/35(77%) and 20/35(57%), had at least one all-cause/AL-related inpatient hospitalization during follow-up, respectively.
CONCLUSIONS: AL is a severe condition with high burden amongst patients with higher levels of disease severity, supporting the continued need for therapeutic advances to help treat these patient populations.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE258
Topic
Economic Evaluation
Disease
SDC: Cardiovascular Disorders (including MI, Stroke, Circulatory), SDC: Rare & Orphan Diseases, SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)