Abstract

A unique Danish mutation, Leu111Met (methionine-for-leucine substitution at amino acid number 111), of cardiac phenotype causing transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM) has been identified to at least three unrelated Danish families with almost 100% penetration. Healthy carriers convert to patients after a median of 10 years at a median age of 47.5 years. If left untreated patients die from cardiac failure. No specific medical treatment exists. At first evidence of ATTRm patients are offered a liver transplant and 33.3% further a heart transplant (Statistics Denmark 2019). The objective of this study was to estimate the Danish lifetime hospital costs associated with treatment of ATTRm, before and after carriers convert to patients, respectively. We developed a model for Danish lifetime hospital costs associated with ATTRm including cost of monitoring and diagnosis carriers, transplants, post-transplant treatment and hospital admissions post-diagnosis (inpatient, outpatient and ambulatory). Since there are no published Danish healthcare data on hospital admissions for the specific Danish Leu111Met population, we rely on conservative estimates of frequencies from clinical experts. Carriers are followed closely 10 years prior to diagnosis and have an average life-expectancy of 18.5 years from diagnosis. Estimates of the cost of diagnosis, transplantations and hospitalizations were based on relevant publicly available Danish reference costs (DRG rates). The average cost of monitoring and diagnosing a carrier was US$ 24,304. Heart(33.3%) and liver transplant(100%) were the single largest cost drivers and amounts to an average of US$ 270,664 per patient. The total hospital costs after a carrier convert to patient is US$ 103,294 per patient. The total cumulative lifetime (28.5 years) hospital cost associated with ATTRm was US$ 398,262. Caring for ATTRm patients incurs a significant economic burden to the secondary health care system. This analysis is conservative and limited by excluding primary care sector costs (medication) and productivity loss.

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