{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T14:26:20Z","timestamp":1787063180986,"version":"build-2736575974"},"reference-count":17,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2023,1,26]],"date-time":"2023-01-26T00:00:00Z","timestamp":1674691200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2023,1,26]],"date-time":"2023-01-26T00:00:00Z","timestamp":1674691200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"name":"Akcea Therapeutics"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Orphanet J Rare Dis"],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Hereditary transthyretin amyloidosis (ATTRv) is an ultra-rare, life-shortening disease with a high unmet need. This study examined ATTRv caregiver health-related quality of life (HRQoL) and productivity.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>A cross-sectional online survey, including EQ-5D-3L, Hospital anxiety and depression scale (HADS), and caregiver and patient characteristics questions, was developed to assess ATTRv caregiver burden. A companion general population survey collected EQ-5D-3L, HADS and chronic health conditions data. Caregiver-control group differences in HRQoL were assessed using t-tests and chi-square tests. Ordinary Least Squares regression was used to estimate the disutility of being a caregiver compared to controls stratified by patient ambulatory status.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Thirty-six caregivers and matched controls completed the survey (n\u2009=\u200972). The disease severity of patients they cared for was varied: 33% required no assistance walking, 58% required assistance with walking and 9% required a wheelchair\/were bedridden. On average, caregivers spent 6\u00a0h daily on practical care and 4\u00a0h daily on emotional support. Fifty-six percent indicated that they had changed their employment due to providing ATTRv care. Caregivers reported lower HRQoL, as indicated by lower EQ-5D 3L utility scores (M\u2009=\u20090.772, SD\u2009=\u20090.178 vs. M\u2009=\u20090.849, SD\u2009=\u20090.218) and higher HADS anxiety (9.3 vs. 6.1, p\u2009&lt;\u20090.01) and depression (7.6 vs. 4.4, p\u2009&lt;\u20090.01) scores, compared with matched controls. Caregivers were also more likely to report sleep problems (33% vs. 8%, p\u2009&lt;\u20090.01) and stress (42% vs. 0%, p\u2009&lt;\u20090.001) as chronic conditions than controls.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>The study results indicate that caring for a person with ATTRv can have a considerable negative impact on caregivers\u2019 HRQoL and productivity. The study findings provide important information for economic evaluations of ATTRv treatments.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1186\/s13023-022-02601-5","type":"journal-article","created":{"date-parts":[[2023,1,25]],"date-time":"2023-01-25T22:02:54Z","timestamp":1674684174000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":15,"title":["A survey exploring caregiver burden and health-related quality of life in hereditary transthyretin amyloidosis"],"prefix":"10.1186","volume":"18","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1025-2546","authenticated-orcid":false,"given":"Sarah","family":"Acaster","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Siu Hing","family":"Lo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandra","family":"Nestler-Parr","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2023,1,26]]},"reference":[{"issue":"1","key":"2601_CR1","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s41687-020-00273-y","volume":"5","author":"A Lovley","year":"2021","unstructured":"Lovley A, Raymond K, Guthrie SD, Pollock M, Sanchorawala V, White MK. Patient-reported burden of hereditary transthyretin amyloidosis on functioning and well-being. J Patient Rep Outcomes. 2021;5(1):1\u201310.","journal-title":"J Patient Rep Outcomes"},{"issue":"1","key":"2601_CR2","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s13023-021-01910-5","volume":"16","author":"J Gonz\u00e1lez-Moreno","year":"2021","unstructured":"Gonz\u00e1lez-Moreno J, Gaya-Barroso A, Losada-L\u00f3pez I, Rodr\u00edguez A, Bosch-Rovira T, Ripoll-Vera T, et al. Val50Met hereditary transthyretin amyloidosis: not just a medical problem, but a psychosocial burden. Orphanet J Rare Dis. 2021;16(1):1\u20137.","journal-title":"Orphanet J Rare Dis"},{"issue":"1","key":"2601_CR3","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s13023-021-01812-6","volume":"16","author":"L Magliano","year":"2021","unstructured":"Magliano L, Obici L, Sforzini C, Mazzeo A, Russo M, Cappelli F, et al. Psychosocial burden and professional and social support in patients with hereditary transthyretin amyloidosis (ATTRv) and their relatives in Italy. Orphanet J Rare Dis. 2021;16(1):1\u201311.","journal-title":"Orphanet J Rare Dis"},{"key":"2601_CR4","doi-asserted-by":"publisher","first-page":"349","DOI":"10.1007\/s40120-018-0106-z","volume":"7","author":"M Stewart","year":"2018","unstructured":"Stewart M, Shaffer S, Murphy B, Loftus J, Alvir J, Cicchetti M, et al. Characterizing the high disease burden of transthyretin amyloidosis for patients and caregivers. Neurol Ther. 2018;7:349\u201364.","journal-title":"Neurol Ther"},{"key":"2601_CR5","unstructured":"National Institute for Health and Care Excellence. Guide to the methods of technology appraisal 2013 [Internet]. London; 2013 Apr [cited 2022 Nov 30]. 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