Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis
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Background: Hereditary transthyretin amyloidosis polyneuropathy (ATTRv-PN) is a rare life-threatening disease that imposes considerable mortality and morbidity associated with increased costs, high social support and productivity losses. This study aims to estimate the societal costs and burden of ATTRv-PN. Methods: A cost-of-illness (COI) and burden of disease model were specified from a societal perspective, using a prevalence-based approach. ⋯ A total of 2056 DALYs were lost, 26% due to disability and 74% due to death. Conclusions: Annual costs and burden of ATTRv-PN were considerable but within the range of other rare diseases. Policies and public interventions to prevent and reduce the burden of disease should be prioritised, since patients experience excess morbidity, mortality and total costs will likely increase in the future.
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Clinical Trial
The utility of repeat kidney biopsy in systemic immunoglobulin light chain amyloidosis.
Background: The diagnostic utility of repeat kidney biopsy in AL amyloidosis patients in complete (CR) or very good partial hematologic response (VGPR) but with renal organ relapse is not clear. Methods: We present eight patients with AL amyloidosis who had a repeat kidney biopsy performed. Results: AL amyloidosis was initially diagnosed by a kidney biopsy. ⋯ Amyloid deposits persist in the kidneys even after successful hematologic treatment and it is impossible to differentiate between new versus old amyloid deposits, which makes performing a repeat kidney biopsy unnecessary in most cases. Demonstration of amyloid deposits on repeat kidney biopsy would not aid in the decision making regarding re-initiation of hematologic treatment. A kidney biopsy should be considered only in cases when a specific alternative diagnosis is suspected.