Impact of genotype and phenotype on cardiac biomarkers in patients with transthyretin amyloidosis – Report from the Transthyretin Amyloidosis Outcome Survey (THAOS)
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Key result
Lower baseline BNP/NT-proBNP is linked to ~49% lower mortality in transthyretin amyloidosis.
- HR 0.508
- 95% CI 0.278-0.928
- P<0.001
- n=1,617
Why the study?
Data on cardiac biomarkers in transthyretin amyloidosis (ATTR) are lacking despite their established role in light-chain amyloidosis risk stratification.
Population
1617 patients with transthyretin amyloidosis with baseline cardiac biomarkers from THAOS
Comparison
Differences in cardiac biomarkers between genotypes and phenotypes of ATTR
Design
Observational cohort study from the Transthyretin Amyloidosis Outcomes Survey (THAOS)
Follow-up
Median 1.2 years
Authors
Discussion
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Implication
Registry data fill a key ATTR evidence gap; leaves open whether biomarkers add prognostic value beyond genotype and phenotype.
Study Design
Type
Observational (n=1,617)
Multicenter
Yes
Structured PICO
P
Population
1,617 adult patients with transthyretin amyloidosis (ATTR) and available baseline cardiac biomarkers, followed for a median of 1.2 years.
O
Outcome
Survival (mortality) and differences in cardiac biomarkers between genotypes and phenotypeshard clinical
Main Result
Hazard Ratio: 0.508 (95% CI 0.278–0.928)
Absolute Event Rate: 95.8% vs 65.2%
p-value: p=<0.001
Cardiac biomarkers (BNP/NT-proBNP and troponins) are frequently abnormal in ATTR amyloidosis and serve as independent predictors of survival.
Limitations
- Variations in the type of clinical investigations conducted and in the completeness of data due to the observational nature of the registry.
- Potential selection bias by attending physicians, as biomarker measures were not obligatory and not available for all patients.
- Low numbers of patients with troponin T and I measurements.
- Limited availability of confounding factors, such as cardiovascular risk factors or pharmacotherapy.
Cite This Study
Kristen et al. (2017) conducted an observational in Transthyretin amyloidosis (ATTR) (n=1,617). BNP/NT-proBNP Q1-Q3 pooled vs. BNP/NT-proBNP Q4 was evaluated on Overall survival (3-year estimate) (HR 0.508, 95% CI 0.278-0.928, p=<0.001). Lower baseline BNP/NT-proBNP levels (Q1-Q3 pooled) were independently associated with improved survival compared to the highest quartile (Q4) in patients with transthyretin amyloidosis (HR 0.508).
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