We conducted a retrospective population-based, matched cohort study using the National Health Insurance Research Database to estimate healthcare resource utilisation (HRU) and costs in patients with newly diagnosed AL amyloidosis in Taiwan. Cases were matched 10:1 by age, sex, and area of residence to patients without AL amyloidosis (comparators) randomly selected from the database during the same time period. Annual all-cause HRU and costs for 3 years were quantified. AL amyloidosis-attributable costs were obtained by subtracting all-cause HRU costs incurred by comparators from cases. The mean age of all patients was 60.78 years and 59.07% were male. Co-morbidities were more frequent in cases than comparators. By 6 months after diagnosis, 12.1% of cases had died versus 0.9% of comparators. In the first year, cases had 103% more outpatient visits, 177% more emergency room visits, were hospitalised 4-times more frequently, and spent 5.5-times more days in hospital than comparators, and total healthcare costs were > sixfold higher. Costs incurred during the first year after diagnosis accounted for 55% of the 3-year cumulative cost. High HRU costs associated with delayed diagnosis and end-organ damage indicate a need for earlier diagnosis and more effective treatments for AL amyloidosis.
Introduction
Amyloidosis is a chronic, incurable disease caused by the accumulation of misfolded proteins that disrupt cellular activities and impair organ functioning. More than 30 proteins are known to form amyloid deposits, of which the most common are light chains (AL amyloidosis), caused by the production of excessive monoclonal immunoglobulin light chains by abnormal plasma cells1. The early symptoms of AL amyloidosis are frequently insidious and non-specific, leading to delays in diagnosis of up to several years2,3. As a result, many patients have advanced disease with heart, kidney and/or liver involvement at diagnosis, which correlates poorly with prognosis4,5.
Because of diagnostic delay or missed diagnosis, precise estimates about the incidence and prevalence of AL amyloidosis are difficult to make. The incidence has been estimated as 10 per million in Western countries and approximately 8.4 per million in Taiwan6,7. Treatment is based on chemotherapy directed against clonal plasma cells using combinations of steroids, cyclophosphamide, and novel agents including bortezomib, daratumumab, thalidomide, lenalidomide, pomalidomide. Autologous stem cell transplant (ACST) can induce hematologic remission and prolong survival8, however fewer than 25% of patients are transplant eligible due to underlying cardiac disease3. Treatment selection is determined by the extent of organ involvement, the presence of co-morbidities, and the presence or absence of the t(11;14) translocation which is associated with lower response rates to bortezomib therapy3,9.
Survival can be a matter of months in patients who present with advanced disease and extensive cardiac involvement, whereas patients diagnosed at an early stage can survive for more than 10 years10. The burden of disease for patients with AL amyloidosis is substantial—the heart is involved in 50–70% of patients, usually presenting as heart failure11. Approximately one-third of patients with renal involvement will eventually require dialysis, and a proportion of patients require organ transplant in order to tolerate chemotherapy11. A systematic review of the literature published in 2017 found that quality of life is significantly affected in patients with AL amyloidosis11.
Information about the baseline healthcare-related costs of AL amyloidosis is needed to inform health technology assessments for new treatments. Studies using healthcare claims databases in the United States (US) have reported that the total all-cause healthcare cost in the first year after a diagnosis of AL amyloidosis was US $122,18012. The cost of healthcare in the first year in patients with relapsed/refractory AL amyloidosis was US $139,14313. Outside of the US there has been little published about the cost of healthcare in patients diagnosed with AL amyloidosis11, and the burden of AL amyloidosis on healthcare systems in Asia is not known.
To bridge the gap in knowledge about the real-world economic burden of AL amyloidosis we conducted a population-based study using a national health insurance claims database to examine the healthcare resource utilisation (HRU) and costs associated with AL amyloidosis in Taiwan. AL amyloidosis-attributable HRU and costs were assessed by comparing outcomes in patients with and without AL amyloidosis.