Impact of Tafamidis on Health-Related Quality of Life in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial)
In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial, tafamidis significantly reduced all-cause mortality and cardiovascular-related hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is associated with a significant burden of disease; further analys...
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Veröffentlicht in: | The American journal of cardiology 2021-02, Vol.141, p.98-105 |
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description | In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial, tafamidis significantly reduced all-cause mortality and cardiovascular-related hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is associated with a significant burden of disease; further analysis of patient-reported quality of life will provide additional data on the efficacy of tafamidis. In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial, 441 adult patients with ATTR-CM were randomized (2:1:2) to tafamidis 80 mg, tafamidis 20 mg, or placebo for 30 months, with pooled tafamidis (80 mg and 20 mg) compared with placebo. Change in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) domain scores, EQ-5D-3L scores, and patient global assessment, were prespecified exploratory end points. A greater proportion of patients improved KCCQ-OS score at month 30 with tafamidis (41.8%) versus placebo (21.4%). Tafamidis significantly reduced the decline in all 4 KCCQ-OS domains (p |
doi_str_mv | 10.1016/j.amjcard.2020.10.066 |
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ATTR-CM is associated with a significant burden of disease; further analysis of patient-reported quality of life will provide additional data on the efficacy of tafamidis. In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial, 441 adult patients with ATTR-CM were randomized (2:1:2) to tafamidis 80 mg, tafamidis 20 mg, or placebo for 30 months, with pooled tafamidis (80 mg and 20 mg) compared with placebo. Change in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) domain scores, EQ-5D-3L scores, and patient global assessment, were prespecified exploratory end points. A greater proportion of patients improved KCCQ-OS score at month 30 with tafamidis (41.8%) versus placebo (21.4%). Tafamidis significantly reduced the decline in all 4 KCCQ-OS domains (p <0.0001 for all), and in EQ-5D-3L utility (0.09 [confidence interval 0.05 to 0.12]; p <0.0001) and EQ visual analog scale (9.11 [confidence interval 5.39 to 12.83]; p <0.0001) scores at month 30 versus placebo. A larger proportion of tafamidis-treated patients reported their patient global assessment improved at month 30 (42.3% vs 23.8% with placebo). In conclusion, tafamidis effectively reduced the decline in patient-reported outcomes, providing further insight into its efficacy in health-related quality of life in patients with ATTR-CM.</description><identifier>ISSN: 0002-9149</identifier><identifier>EISSN: 1879-1913</identifier><identifier>DOI: 10.1016/j.amjcard.2020.10.066</identifier><identifier>PMID: 33220323</identifier><language>eng</language><publisher>United States: Elsevier Inc</publisher><subject>Activities of Daily Living ; Aged ; Aged, 80 and over ; Amyloid ; Amyloid Neuropathies, Familial - drug therapy ; Amyloid Neuropathies, Familial - physiopathology ; Benzoxazoles - therapeutic use ; Cardiomyopathies - drug therapy ; Cardiomyopathies - physiopathology ; Cardiomyopathy ; Cardiovascular diseases ; Clinical trials ; Confidence intervals ; Cost of Illness ; Domains ; Female ; Heart failure ; Humans ; Male ; Patient Reported Outcome Measures ; Patients ; Placebos ; Quality of Life ; Questionnaires ; Self Efficacy ; Social Participation ; Tariffs ; Transthyretin</subject><ispartof>The American journal of cardiology, 2021-02, Vol.141, p.98-105</ispartof><rights>2021 The Authors</rights><rights>Copyright © 2020. Published by Elsevier Inc.</rights><rights>2021. The Authors</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c440t-e98268a97352637eaec79f74b2b0d1a90c2b0ede570f640fc7ec438c3fe0be5c3</citedby><cites>FETCH-LOGICAL-c440t-e98268a97352637eaec79f74b2b0d1a90c2b0ede570f640fc7ec438c3fe0be5c3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://www.proquest.com/docview/2479989257?pq-origsite=primo$$EHTML$$P50$$Gproquest$$H</linktohtml><link.rule.ids>314,780,784,3550,27924,27925,45995,64385,64387,64389,72469</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/33220323$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Hanna, Mazen</creatorcontrib><creatorcontrib>Damy, Thibaud</creatorcontrib><creatorcontrib>Grogan, Martha</creatorcontrib><creatorcontrib>Stewart, Michelle</creatorcontrib><creatorcontrib>Gundapaneni, Balarama</creatorcontrib><creatorcontrib>Patterson, Terrell A.</creatorcontrib><creatorcontrib>Schwartz, Jeffrey H.</creatorcontrib><creatorcontrib>Sultan, Marla B.</creatorcontrib><creatorcontrib>Maurer, Mathew S.</creatorcontrib><title>Impact of Tafamidis on Health-Related Quality of Life in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial)</title><title>The American journal of cardiology</title><addtitle>Am J Cardiol</addtitle><description>In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial, tafamidis significantly reduced all-cause mortality and cardiovascular-related hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is associated with a significant burden of disease; further analysis of patient-reported quality of life will provide additional data on the efficacy of tafamidis. In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial, 441 adult patients with ATTR-CM were randomized (2:1:2) to tafamidis 80 mg, tafamidis 20 mg, or placebo for 30 months, with pooled tafamidis (80 mg and 20 mg) compared with placebo. Change in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) domain scores, EQ-5D-3L scores, and patient global assessment, were prespecified exploratory end points. A greater proportion of patients improved KCCQ-OS score at month 30 with tafamidis (41.8%) versus placebo (21.4%). Tafamidis significantly reduced the decline in all 4 KCCQ-OS domains (p <0.0001 for all), and in EQ-5D-3L utility (0.09 [confidence interval 0.05 to 0.12]; p <0.0001) and EQ visual analog scale (9.11 [confidence interval 5.39 to 12.83]; p <0.0001) scores at month 30 versus placebo. A larger proportion of tafamidis-treated patients reported their patient global assessment improved at month 30 (42.3% vs 23.8% with placebo). In conclusion, tafamidis effectively reduced the decline in patient-reported outcomes, providing further insight into its efficacy in health-related quality of life in patients with ATTR-CM.</description><subject>Activities of Daily Living</subject><subject>Aged</subject><subject>Aged, 80 and over</subject><subject>Amyloid</subject><subject>Amyloid Neuropathies, Familial - drug therapy</subject><subject>Amyloid Neuropathies, Familial - physiopathology</subject><subject>Benzoxazoles - therapeutic use</subject><subject>Cardiomyopathies - drug therapy</subject><subject>Cardiomyopathies - physiopathology</subject><subject>Cardiomyopathy</subject><subject>Cardiovascular diseases</subject><subject>Clinical trials</subject><subject>Confidence intervals</subject><subject>Cost of Illness</subject><subject>Domains</subject><subject>Female</subject><subject>Heart failure</subject><subject>Humans</subject><subject>Male</subject><subject>Patient Reported Outcome Measures</subject><subject>Patients</subject><subject>Placebos</subject><subject>Quality of Life</subject><subject>Questionnaires</subject><subject>Self Efficacy</subject><subject>Social Participation</subject><subject>Tariffs</subject><subject>Transthyretin</subject><issn>0002-9149</issn><issn>1879-1913</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2021</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>8G5</sourceid><sourceid>ABUWG</sourceid><sourceid>AFKRA</sourceid><sourceid>AZQEC</sourceid><sourceid>BENPR</sourceid><sourceid>CCPQU</sourceid><sourceid>DWQXO</sourceid><sourceid>GNUQQ</sourceid><sourceid>GUQSH</sourceid><sourceid>M2O</sourceid><recordid>eNqFkU1v1DAQhiMEotvCTwBZ4tIesvgjieMTqlZAK63EhxZxtLzOWOvIiRfbqZRfxV_E0S4IeuE0npnnnRn5LYpXBK8JJs3bfq2GXqvQrSmmS22Nm-ZJsSItFyURhD0tVhhjWgpSiYviMsY-p4TUzfPigjFKMaNsVfy8H45KJ-QN2imjBtvZiPyI7kC5dCi_glMJOvRlUs6mecG21gCyI_qskoUxRfTdpgPaBTXGdJgDpNy7HWbnbYc2-T7rh9kfVe6haxP8gNIB_tqV6X-1jzQbZ0erlcuUVe7mRfHMKBfh5TleFd8-vN9t7srtp4_3m9ttqasKpxJES5tWCc5q2jAOCjQXhld7uscdUQLr_IAOao5NU2GjOeiKtZoZwHuoNbsqrk9zj8H_mCAmOdiowTk1gp-ipFXDCG55QzP65hHa-ymM-bpMcSFaQWueqfpE6eBjDGDkMdhBhVkSLBdHZS_PjsrF0aWcHc261-fp036A7o_qt4UZeHcCIH_Hg4Ugo87GaOhsAJ1k5-1_VvwCLv-3bQ</recordid><startdate>20210215</startdate><enddate>20210215</enddate><creator>Hanna, Mazen</creator><creator>Damy, Thibaud</creator><creator>Grogan, Martha</creator><creator>Stewart, Michelle</creator><creator>Gundapaneni, Balarama</creator><creator>Patterson, Terrell A.</creator><creator>Schwartz, Jeffrey H.</creator><creator>Sultan, Marla B.</creator><creator>Maurer, Mathew S.</creator><general>Elsevier Inc</general><general>Elsevier Limited</general><scope>6I.</scope><scope>AAFTH</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>3V.</scope><scope>7RV</scope><scope>7TS</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8FD</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>8G5</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AZQEC</scope><scope>BENPR</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FR3</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>GUQSH</scope><scope>K9.</scope><scope>KB0</scope><scope>M0S</scope><scope>M1P</scope><scope>M2O</scope><scope>M7Z</scope><scope>MBDVC</scope><scope>NAPCQ</scope><scope>P64</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>Q9U</scope><scope>7X8</scope></search><sort><creationdate>20210215</creationdate><title>Impact of Tafamidis on Health-Related Quality of Life in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial)</title><author>Hanna, Mazen ; Damy, Thibaud ; Grogan, Martha ; Stewart, Michelle ; Gundapaneni, Balarama ; Patterson, Terrell A. ; Schwartz, Jeffrey H. ; Sultan, Marla B. ; Maurer, Mathew S.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c440t-e98268a97352637eaec79f74b2b0d1a90c2b0ede570f640fc7ec438c3fe0be5c3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2021</creationdate><topic>Activities of Daily Living</topic><topic>Aged</topic><topic>Aged, 80 and over</topic><topic>Amyloid</topic><topic>Amyloid Neuropathies, Familial - drug therapy</topic><topic>Amyloid Neuropathies, Familial - physiopathology</topic><topic>Benzoxazoles - therapeutic use</topic><topic>Cardiomyopathies - drug therapy</topic><topic>Cardiomyopathies - physiopathology</topic><topic>Cardiomyopathy</topic><topic>Cardiovascular diseases</topic><topic>Clinical trials</topic><topic>Confidence intervals</topic><topic>Cost of Illness</topic><topic>Domains</topic><topic>Female</topic><topic>Heart failure</topic><topic>Humans</topic><topic>Male</topic><topic>Patient Reported Outcome Measures</topic><topic>Patients</topic><topic>Placebos</topic><topic>Quality of Life</topic><topic>Questionnaires</topic><topic>Self Efficacy</topic><topic>Social Participation</topic><topic>Tariffs</topic><topic>Transthyretin</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Hanna, Mazen</creatorcontrib><creatorcontrib>Damy, Thibaud</creatorcontrib><creatorcontrib>Grogan, Martha</creatorcontrib><creatorcontrib>Stewart, Michelle</creatorcontrib><creatorcontrib>Gundapaneni, Balarama</creatorcontrib><creatorcontrib>Patterson, Terrell A.</creatorcontrib><creatorcontrib>Schwartz, Jeffrey H.</creatorcontrib><creatorcontrib>Sultan, Marla B.</creatorcontrib><creatorcontrib>Maurer, Mathew S.</creatorcontrib><collection>ScienceDirect Open Access Titles</collection><collection>Elsevier:ScienceDirect:Open Access</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Central (Corporate)</collection><collection>Nursing & Allied Health Database</collection><collection>Physical Education Index</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>Medical Database (Alumni Edition)</collection><collection>Technology Research Database</collection><collection>Hospital Premium Collection</collection><collection>Hospital Premium Collection (Alumni Edition)</collection><collection>ProQuest Central (Alumni) (purchase pre-March 2016)</collection><collection>Research Library (Alumni Edition)</collection><collection>ProQuest Central (Alumni Edition)</collection><collection>ProQuest Central UK/Ireland</collection><collection>ProQuest Central Essentials</collection><collection>ProQuest Central</collection><collection>ProQuest One Community College</collection><collection>ProQuest Central Korea</collection><collection>Engineering Research Database</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>ProQuest Central Student</collection><collection>Research Library Prep</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Nursing & Allied Health Database (Alumni Edition)</collection><collection>Health & Medical Collection (Alumni Edition)</collection><collection>Medical Database</collection><collection>Research Library</collection><collection>Biochemistry Abstracts 1</collection><collection>Research Library (Corporate)</collection><collection>Nursing & Allied Health Premium</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>ProQuest Central China</collection><collection>ProQuest Central Basic</collection><collection>MEDLINE - Academic</collection><jtitle>The American journal of cardiology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Hanna, Mazen</au><au>Damy, Thibaud</au><au>Grogan, Martha</au><au>Stewart, Michelle</au><au>Gundapaneni, Balarama</au><au>Patterson, Terrell A.</au><au>Schwartz, Jeffrey H.</au><au>Sultan, Marla B.</au><au>Maurer, Mathew S.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Impact of Tafamidis on Health-Related Quality of Life in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial)</atitle><jtitle>The American journal of cardiology</jtitle><addtitle>Am J Cardiol</addtitle><date>2021-02-15</date><risdate>2021</risdate><volume>141</volume><spage>98</spage><epage>105</epage><pages>98-105</pages><issn>0002-9149</issn><eissn>1879-1913</eissn><abstract>In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial, tafamidis significantly reduced all-cause mortality and cardiovascular-related hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is associated with a significant burden of disease; further analysis of patient-reported quality of life will provide additional data on the efficacy of tafamidis. In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial, 441 adult patients with ATTR-CM were randomized (2:1:2) to tafamidis 80 mg, tafamidis 20 mg, or placebo for 30 months, with pooled tafamidis (80 mg and 20 mg) compared with placebo. Change in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) domain scores, EQ-5D-3L scores, and patient global assessment, were prespecified exploratory end points. A greater proportion of patients improved KCCQ-OS score at month 30 with tafamidis (41.8%) versus placebo (21.4%). Tafamidis significantly reduced the decline in all 4 KCCQ-OS domains (p <0.0001 for all), and in EQ-5D-3L utility (0.09 [confidence interval 0.05 to 0.12]; p <0.0001) and EQ visual analog scale (9.11 [confidence interval 5.39 to 12.83]; p <0.0001) scores at month 30 versus placebo. A larger proportion of tafamidis-treated patients reported their patient global assessment improved at month 30 (42.3% vs 23.8% with placebo). In conclusion, tafamidis effectively reduced the decline in patient-reported outcomes, providing further insight into its efficacy in health-related quality of life in patients with ATTR-CM.</abstract><cop>United States</cop><pub>Elsevier Inc</pub><pmid>33220323</pmid><doi>10.1016/j.amjcard.2020.10.066</doi><tpages>8</tpages><oa>free_for_read</oa></addata></record> |
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subjects | Activities of Daily Living Aged Aged, 80 and over Amyloid Amyloid Neuropathies, Familial - drug therapy Amyloid Neuropathies, Familial - physiopathology Benzoxazoles - therapeutic use Cardiomyopathies - drug therapy Cardiomyopathies - physiopathology Cardiomyopathy Cardiovascular diseases Clinical trials Confidence intervals Cost of Illness Domains Female Heart failure Humans Male Patient Reported Outcome Measures Patients Placebos Quality of Life Questionnaires Self Efficacy Social Participation Tariffs Transthyretin |
title | Impact of Tafamidis on Health-Related Quality of Life in Patients With Transthyretin Amyloid Cardiomyopathy (from the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial) |
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