Efficacy of tafamidis in transthyretin amyloid cardiomyopathy: A systematic review

Hua Ling; David Ombengi; Courtney Campbell

doi:10.1002/jac5.1204

Efficacy of tafamidis in transthyretin amyloid cardiomyopathy: A systematic review

Hua Ling, David Ombengi, Courtney Campbell

Department of Pharmacy Practice

Research output: Contribution to conference › Presentation

Abstract

<div class="line" id="line-13"> <b> Background </b> : Transthyretin cardiomyopathy (ATTR&hyphen;CM) is a rare, progressively fatal disease caused by the deposition of transthyretin amyloid fibrils in the myocardium. It can be hereditary because of mutations in the transthyretin gene or acquired with aging. Tafamidis is a selective transthyretin stabilizer, preventing tetramer dissociation and amyloidogenesis. The study aims to examine the efficacy of tafamidis compared with standard of care (SOC) on mortality and cardiovascular hospitalizations in patients with ATTR&hyphen;CM.</div><div class="line" id="line-16"> <br/></div><div class="line" id="line-18"> <b> Methods </b> : A systematic literature search was performed through May 20 <span style="font-size: 12px;"> th </span> , 2019 using PubMed, EMBASE and  <a href="http://clinicaltrials.gov/"> <span style="color: rgb(0, 82, 116); background-color: transparent; font-size: inherit;"> clinicaltrials.gov </span> </a>  with the following key terms: “tafamidis,” “cardiomyopathy,” and “transthyretin amyloidosis.” The review was restricted to controlled trials published in English in ATTR&hyphen;CM patients with mortality or cardiovascular hospitalizations reported. Studies on amyloid neuropathy were excluded. The Cochrane Risk of Bias Tool was used to assess bias risk.</div><div class="line" id="line-25"> <br/></div><div class="line" id="line-27"> <b> Results </b> : Two trials with a total of 505 patients were included. Tafamidis was significantly associated with lower mortality than SOC (odds ratio 0.53, 95% CI 0.36&hyphen;0.78, I <span style="font-size: 12px;"> 2 </span>  = 0%). However, there was no difference in the percentage of cardiovascular hospitalized patients between tafamidis and SOC (odds ratio 0.71, 95% CI 0.49&hyphen;1.02, I <span style="font-size: 12px;"> 2 </span>  = 0%). Of note, the study conducted by Falk  <i> et al  </i> was an open&hyphen;label nonrandomized study, therefore carrying higher risk of biases.</div><div class="line" id="line-36"> <br/></div><div class="line" id="line-38"> <b> Discussion </b> : Despite the small number of trials and participants included in the study, our results showed that tafamidis was significantly associated with reductions in mortality. No difference was identified between tafamidis and SOC for the percentage of cardiovascular hospitalized patients, however, it should be noted that, in the ATTR&hyphen;ACT study, a significant reduction in the rate of cardiovascular hospitalizations was reported to be associated with tafamidis, indicating a benefit of tafamidis in reducing cardiovascular hospitalizations compared with SOC. In conclusion, the findings of our study support tafamidis as an effective therapy for ATTR&hyphen;CM.</div>

Original language	American English
DOIs	https://doi.org/10.1002/jac5.1204
State	Published - Oct 2019
Event	2019 ACCP Annual Meeting - Duration: Oct 1 2019 → …

Conference

Conference	2019 ACCP Annual Meeting
Period	10/1/19 → …

Disciplines

Medicine and Health Sciences

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10.1002/jac5.1204License: Unspecified

Cite this

@conference{4f9cbb658d454aa9ad8b12b8ec363b4c,

title = "Efficacy of tafamidis in transthyretin amyloid cardiomyopathy: A systematic review",

abstract = " Background : Transthyretin cardiomyopathy (ATTR&hyphen;CM) is a rare, progressively fatal disease caused by the deposition of transthyretin amyloid fibrils in the myocardium. It can be hereditary because of mutations in the transthyretin gene or acquired with aging. Tafamidis is a selective transthyretin stabilizer, preventing tetramer dissociation and amyloidogenesis. The study aims to examine the efficacy of tafamidis compared with standard of care (SOC) on mortality and cardiovascular hospitalizations in patients with ATTR&hyphen;CM. Methods : A systematic literature search was performed through May 20 th , 2019 using PubMed, EMBASE and  clinicaltrials.gov  with the following key terms: “tafamidis,” “cardiomyopathy,” and “transthyretin amyloidosis.” The review was restricted to controlled trials published in English in ATTR&hyphen;CM patients with mortality or cardiovascular hospitalizations reported. Studies on amyloid neuropathy were excluded. The Cochrane Risk of Bias Tool was used to assess bias risk. Results : Two trials with a total of 505 patients were included. Tafamidis was significantly associated with lower mortality than SOC (odds ratio 0.53, 95% CI 0.36&hyphen;0.78, I 2  = 0%). However, there was no difference in the percentage of cardiovascular hospitalized patients between tafamidis and SOC (odds ratio 0.71, 95% CI 0.49&hyphen;1.02, I 2  = 0%). Of note, the study conducted by Falk  et al  was an open&hyphen;label nonrandomized study, therefore carrying higher risk of biases. Discussion : Despite the small number of trials and participants included in the study, our results showed that tafamidis was significantly associated with reductions in mortality. No difference was identified between tafamidis and SOC for the percentage of cardiovascular hospitalized patients, however, it should be noted that, in the ATTR&hyphen;ACT study, a significant reduction in the rate of cardiovascular hospitalizations was reported to be associated with tafamidis, indicating a benefit of tafamidis in reducing cardiovascular hospitalizations compared with SOC. In conclusion, the findings of our study support tafamidis as an effective therapy for ATTR&hyphen;CM.",

author = "Hua Ling and David Ombengi and Courtney Campbell",

year = "2019",

month = oct,

doi = "10.1002/jac5.1204",

language = "American English",

note = "2019 ACCP Annual Meeting ; Conference date: 01-10-2019",

}

TY - CONF

T1 - Efficacy of tafamidis in transthyretin amyloid cardiomyopathy: A systematic review

AU - Ling, Hua

AU - Ombengi, David

AU - Campbell, Courtney

PY - 2019/10

Y1 - 2019/10

N2 - Background : Transthyretin cardiomyopathy (ATTR&hyphen;CM) is a rare, progressively fatal disease caused by the deposition of transthyretin amyloid fibrils in the myocardium. It can be hereditary because of mutations in the transthyretin gene or acquired with aging. Tafamidis is a selective transthyretin stabilizer, preventing tetramer dissociation and amyloidogenesis. The study aims to examine the efficacy of tafamidis compared with standard of care (SOC) on mortality and cardiovascular hospitalizations in patients with ATTR&hyphen;CM. Methods : A systematic literature search was performed through May 20 th , 2019 using PubMed, EMBASE and  clinicaltrials.gov  with the following key terms: “tafamidis,” “cardiomyopathy,” and “transthyretin amyloidosis.” The review was restricted to controlled trials published in English in ATTR&hyphen;CM patients with mortality or cardiovascular hospitalizations reported. Studies on amyloid neuropathy were excluded. The Cochrane Risk of Bias Tool was used to assess bias risk. Results : Two trials with a total of 505 patients were included. Tafamidis was significantly associated with lower mortality than SOC (odds ratio 0.53, 95% CI 0.36&hyphen;0.78, I 2  = 0%). However, there was no difference in the percentage of cardiovascular hospitalized patients between tafamidis and SOC (odds ratio 0.71, 95% CI 0.49&hyphen;1.02, I 2  = 0%). Of note, the study conducted by Falk  et al  was an open&hyphen;label nonrandomized study, therefore carrying higher risk of biases. Discussion : Despite the small number of trials and participants included in the study, our results showed that tafamidis was significantly associated with reductions in mortality. No difference was identified between tafamidis and SOC for the percentage of cardiovascular hospitalized patients, however, it should be noted that, in the ATTR&hyphen;ACT study, a significant reduction in the rate of cardiovascular hospitalizations was reported to be associated with tafamidis, indicating a benefit of tafamidis in reducing cardiovascular hospitalizations compared with SOC. In conclusion, the findings of our study support tafamidis as an effective therapy for ATTR&hyphen;CM.

AB - Background : Transthyretin cardiomyopathy (ATTR&hyphen;CM) is a rare, progressively fatal disease caused by the deposition of transthyretin amyloid fibrils in the myocardium. It can be hereditary because of mutations in the transthyretin gene or acquired with aging. Tafamidis is a selective transthyretin stabilizer, preventing tetramer dissociation and amyloidogenesis. The study aims to examine the efficacy of tafamidis compared with standard of care (SOC) on mortality and cardiovascular hospitalizations in patients with ATTR&hyphen;CM. Methods : A systematic literature search was performed through May 20 th , 2019 using PubMed, EMBASE and  clinicaltrials.gov  with the following key terms: “tafamidis,” “cardiomyopathy,” and “transthyretin amyloidosis.” The review was restricted to controlled trials published in English in ATTR&hyphen;CM patients with mortality or cardiovascular hospitalizations reported. Studies on amyloid neuropathy were excluded. The Cochrane Risk of Bias Tool was used to assess bias risk. Results : Two trials with a total of 505 patients were included. Tafamidis was significantly associated with lower mortality than SOC (odds ratio 0.53, 95% CI 0.36&hyphen;0.78, I 2  = 0%). However, there was no difference in the percentage of cardiovascular hospitalized patients between tafamidis and SOC (odds ratio 0.71, 95% CI 0.49&hyphen;1.02, I 2  = 0%). Of note, the study conducted by Falk  et al  was an open&hyphen;label nonrandomized study, therefore carrying higher risk of biases. Discussion : Despite the small number of trials and participants included in the study, our results showed that tafamidis was significantly associated with reductions in mortality. No difference was identified between tafamidis and SOC for the percentage of cardiovascular hospitalized patients, however, it should be noted that, in the ATTR&hyphen;ACT study, a significant reduction in the rate of cardiovascular hospitalizations was reported to be associated with tafamidis, indicating a benefit of tafamidis in reducing cardiovascular hospitalizations compared with SOC. In conclusion, the findings of our study support tafamidis as an effective therapy for ATTR&hyphen;CM.

U2 - 10.1002/jac5.1204

DO - 10.1002/jac5.1204

M3 - Presentation

T2 - 2019 ACCP Annual Meeting

Y2 - 1 October 2019

ER -