Key result
Tafamidis increased median 1-year serum TTR by 8.5 mg/dL, with increases ≥8.5 mg/dL associated with baseline carpal tunnel syndrome (OR 5.210; p=0.004) and lower hs-troponin T (p=0.025).
Why the study?
Early increases in circulating transthyretin levels with stabilizer therapy have been linked to better prognoses in wild-type transthyretin cardiomyopathy, yet the optimal TTR concentration and its clinical correlations remain unclear.
Does tafamidis therapy increase serum TTR concentrations at 1 year and correlate with baseline clinical parameters and outcomes in patients with wild-type transthyretin cardiomyopathy (ATTRwt-CM)?
Observational (n=62)
Does tafamidis therapy increase serum TTR concentrations at 1 year and correlate with baseline clinical parameters and outcomes in patients with wild-type transthyretin cardiomyopathy (ATTRwt-CM)?
Greater increases in serum TTR concentration after one year of tafamidis therapy in ATTRwt-CM patients are associated with milder baseline disease characteristics (such as lower BNP, higher eGFR, and NYHA class I) and lead to reduced high-sensitivity troponin T levels, emphasizing the importance of early diagnosis and treatment.
Background Wild-type transthyretin cardiomyopathy (ATTRwt-CM) arises from age-related destabilization of the transthyretin (TTR) tetramer, leading to amyloid protein deposition in various organs. The introduction of TTR stabilizers has improved patient outcomes. Early increases in circulating TTR levels have been linked to better prognoses, yet optimal TTR concentration and clinical correlations remain unclear. Purpose This retrospective study aimed to investigate serum TTR concentration changes following tafamidis therapy initiation in patients with ATTRwt-CM, assessing the relationship between TTR increases and clinical outcomes. Methods We performed an analysis of 108 ATTRwt-CM patients treated with tafamidis from September 2019 to 2024, focusing on those with baseline and 1-year serum TTR measurements. Out of these, 62 patients met the inclusion criteria. We calculated median changes in TTR (ΔTTR) and categorized patients accordingly. Additionally, we examined correlations between TTR levels and clinical parameters. Logistic regression was performed, and assessed laboratory parameters influenced by TTR changes at 6 and 12 months through mixed-effects analysis. Results The ΔTTR after 1 year had a median increase of 8.5 mg/dL [5.7, 10.4], categorizing patients into two groups. No difference was observed in the baseline TTR concentration (Overall: 22.2 [17.7, 25.4] mg/dL;< 8.5mg/dL group: 22.4 [17.6, 25.4] mg/dL; ≧ 8.5 mg/dL group: 21.1 [17.7, 25.7] mg/dL, p=0.894). The correlation between 1-year change in serum TTR concentration and clinical parameters, included diagnosis age (Pearson r=-0.280; 95% CI, -0.458, -0.280; p=0.027), the baseline NYHA class I (Pearson r=0.275; 95% CI, 0.278, 5.429; p=0.030), prevalence of carpal tunnel syndrome (Pearson r=0.386; 95% CI, 1.286, 5.439; p=0.002), the baseline AF (Pearson r=-0.273; 95% CI, -4.902, -0.227; p=0.032), B-type natriuretic peptide (BNP) levels (Spearman r=-0.322; 95% CI, -0.602, -0.085; p=0.012), and estimated glomerular filtration rate (eGFR) (Pearson r=0.280; 95% CI, 0.109, 0.593; p=0.028) were shown. Logistic regression confirmed these associations, highlighting the impact of baseline CTS, BNP, and eGFR on TTR outcomes (CTS; OR 5.210; 95% CI, 1.694, 16.026; p=0.004, BNP baseline per 100 pg/mL increase; OR, 0.643; 95%CI, 0.460, 0.898; p=0.010, eGFR baseline per 10 mL/min/1.73m2increase; OR, 1.552; 95%CI, 1.068, 2.256; p=0.021). Moreover, the ΔTTR≧8.5 mg/dL group showed reduced high sensitivity troponin T levels during 1 year of tafamidis treatment (p=0.025). Conclusions The study indicates that serum TTR concentration increases are associated with relatively mild diagnosed cases, indicating the need for early diagnosis and adequate treatment to optimize the effects of TTR stabilizers.
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Yoshie et al. (2026) conducted an observational in Wild-type transthyretin cardiomyopathy (ATTRwt-CM) (n=62). Tafamidis therapy (1-year serum TTR change ≥8.5 mg/dL) vs. 1-year serum TTR change <8.5 mg/dL was evaluated on 1-year change in serum transthyretin (ΔTTR) ≥8.5 mg/dL and its association with baseline clinical parameters (such as carpal tunnel syndrome) (OR 5.210, 95% CI 1.694-16.026, p=0.004). Tafamidis increased median 1-year serum TTR by 8.5 mg/dL, with increases ≥8.5 mg/dL associated with baseline carpal tunnel syndrome (OR 5.210; p=0.004) and lower hs-troponin T (p=0.025).
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