Factors associated with financial toxicity in patients with transthyretin amyloidosis: results from Amyloidosis Research Consortium’s treatment affordability patient and caregiver survey

Transthyretin (ATTR) amyloidosis is an ultimately fatal disease. While approved therapies slow disease progression, the cost of care can be significant for patients. The objective of this survey was to describe the factors associated with financial toxicity of ATTR amyloidosis and the economic burden of care and treatment. The Amyloidosis Research Consortium (ARC) conducted an online survey in the United States for patients with ATTR amyloidosis and their caregivers. Financial toxicity was assessed using the COmprehensive Score for Financial Toxicity (COST). Multivariate linear regression was used to identify factors associated with financial toxicity controlling for key sociodemographic and patient characteristics. Of 452 respondents, 249 (55%) reported financial toxicity. Respondents who reported financial toxicity predominantly had ATTRv amyloidosis, were younger, non-white, had a household income Patients with ATTR amyloidosis experience significant financial distress that is comparable to patients with cancer, and as a result, many patients delay treatment or pay for treatment using savings and/or borrowing money. African American respondents (vs white) were significantly at risk.转甲状腺素蛋白（Transthyretin, ATTR）淀粉样变性是一种最终可致命的疾病。尽管已有获批疗法可延缓疾病进展，但患者的护理成本仍较为高昂。本调查旨在明确与ATTR淀粉样变性患者的财务毒性及护理、治疗的经济负担相关的影响因素。淀粉样变性研究联盟（Amyloidosis Research Consortium, ARC）在美国针对ATTR淀粉样变性患者及其护理者开展了一项在线调查。财务毒性采用综合财务毒性评分（Comprehensive Score for Financial Toxicity, COST）进行评估。研究采用多元线性回归分析，在控制关键社会人口学特征与患者自身特征的前提下，识别与财务毒性相关的影响因素。在452名受访者中，249人（55%）报告存在财务毒性。存在财务毒性报告的受访者多为ATTRv淀粉样变性患者，年龄较轻、为非白人，且家庭收入[原文此处存在内容缺失]。ATTR淀粉样变性患者所经历的严重经济窘迫程度可与癌症患者相媲美，因此许多患者会延迟治疗，或通过储蓄及/或借贷来支付治疗费用。非裔美国受访者（相较于白人受访者）面临的财务毒性风险显著更高。