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About VYNDAQEL

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Click here for VYNDAQEL®▼ (tafamidis) Prescribing Information. 

Real-world survival data for patients with ATTR-CM from the THAOS registry1Who was included in this post-hoc analysis?1

A post-hoc analysis of the THAOS registry (Transthyretin Amyloidosis Outcomes survey) was conducted with data from 1,441 patients who had ATTR-CM and a predominantly cardiac phenotype. Patients were categorised as tafamidis-treated if they received tafamidis while enrolled in THAOS or tafamidis-untreated if they have never received tafamidis while enrolled in THAOS. The analysis included all patients in the registry with a predominately cardiac phenotype at enrolment, defined as patients with ATTR-CM without signs and symptoms suggestive of associated ATTR amyloidosis- related neuropathy.

Most patients in this THAOS analysis were on tafamidis meglumine 80mg daily/ tafamidis 61mg daily1 

The post-hoc analysis included patients who received any dosage of tafamidis, not just the licensed tafamidis meglumine 80 mg daily/tafamidis 61 mg daily, to provide real-world representation beyond the scope of randomised clinical trials.1

The results presented below on this page will only focus on patients who received tafamidis meglumine 80 mg daily/tafamidis 61 mg daily

What were the key survival data? 

Adapted from: Garcia-Pavia P et al. J Card Fail 2024; S1071-9164(24)00222-7

What did the safety profile look like? What are the key takeaways?1

In this real-world study of patients with ATTR-CM, observed survival rates were higher among patients who received the approved tafamidis dose versus untreated patients. These observed survival rates
are also supported by further analysis of a contemporary patient population (enrolled 2019–2023).

The higher rate of survival observed across both tafamidis meglumine 80 mg daily/tafamidis 61 mg daily and placebo arms enrolled in 2019 or later, compared with the same cohorts enrolled during any year of THAOS, may be the result of advances in cardiac imaging and increased awareness amongst physicians that facilitate earlier detection of ATTR-CM. This suggests the importance of early diagnosis and treatment of ATTR-CM.

There were no new safety signals reported in patients treated with tafamidis meglumine 80mg/tafamidis 61mg in this THAOS analysis, supporting the long-term safety and tolerability profile of tafamidis in the real-world settings.

What are the limitations of this post hoc analysis?1
  • This study was an observational registry in which data are not always complete, and baseline data were not available for all patients; most of the data came from US study sites from patients with a predominantly cardiac phenotype
  • Propensity-matched analyses were not possible, given the amount of incomplete data
  • The observational nature of this study had the potential to introduce selection bias, ascertainment bias, or temporal‡ bias
  • Median follow-up time was 2 years in both groups, limiting the reliability of the survival analyses in later months
  • The small number of patients with variant TTR genotypes prevents robust conclusions regarding survival in these subgroups
  • Data on the use of SPECT imaging used in patients diagnosed with scintigraphy are not available, so misdiagnosis is possible as cardiac scintigraphy in the absence of SPECT can lead to false positives
‡Treated patients had less severe disease than untreated patients as indicated by a lower median NT-proBNP value and were more likely enrolled in THAOS in 2019 or later.2
Explore more We have a team ready to support you with any queries.Get in touchLoadingAcronyms 
AE, adverse event; ATTR-ACT, Transthyretin Amyloidosis Cardiomyopathy Clinical Trial; ATTR-CM, transthyretin amyloid cardiomyopathy;
ATTRv, variant transthyretin amyloidosis; ATTRwt, wild-type transthyretin amyloidosis; NT-proBNP, N-terminal prohormone of brain natriuretic peptide;
OS, overall survival; SPECT, Speckle-tracking echocardiography; TEAE, treatment-emergent adverse event; THAOS, Transthyretin Amyloidosis
Outcomes Survey; TTR, transthyretin.


References:

Garcia Pavia P, et al J Card Fail 2024; S1071-9164(24)00222-7ClinicalTrials.gov. NCT00628745 https://clinicaltrials.gov/study/NCT00628745 (Accessed October 2024)VYNDAQEL 61 mg (tafamidis) Summary of Product Characteristics.
PP-VYN-GBR-1712. October 2024
Download THAOS summaryLoading

Remind yourself of the key ATTR-ACT results

ATTR-ACT results Loading

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Adverse events should also be reported to Pfizer Medical Information on 01304 616161

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