TY - JOUR
T1 - Prospective evaluation of the morbidity and mortality of wild-type and V122I mutant transthyretin amyloid cardiomyopathy
T2 - The Transthyretin Amyloidosis Cardiac Study (TRACS)
AU - Ruberg, Frederick L.
AU - Maurer, Mathew S.
AU - Judge, Daniel P.
AU - Zeldenrust, Steven
AU - Skinner, Martha
AU - Kim, Antony Y.
AU - Falk, Rodney H.
AU - Cheung, Kin N.
AU - Patel, Ayan R.
AU - Pano, Arian
AU - Packman, Jeffrey
AU - Grogan, Donna Roy
PY - 2012/8
Y1 - 2012/8
N2 - Background TRACS sought to describe the clinical outcomes and disease progression of transthyretin (TTR) cardiac amyloidosis (ATTR) in an observational study. Clinical course is largely determined by disease type with ATTR categorized as wild-type (ATTRwt) or genetic-variant protein (ATTRm). Prospective data are lacking in the most common TTR mutation, V122I, present in approximately 3.5% of African Americans. Methods Patients with ATTRwt (n = 18) and V122I ATTRm (n = 11) were longitudinally assessed every 6 months for up to 2 years by functional class assessments, biochemical markers, and echocardiography. Results At baseline, no differences in clinical characteristics, biomarkers, or echocardiographic parameters were noted between patients with ATTRwt and patients with ATTRm. After 15.5 ± 8 months, there were 11 deaths and 1 cardiac transplant, with higher mortality (73% vs 22%, P = .03) and cardiovascular hospitalization (64% vs 28%, P = .02) among patients with ATTRm. The median survival from diagno is was 25.6 months for ATTRm vs 43.0 months for ATTRwt (P = .04). nivariate predictors of mortality included disease duration, heart rate ≤70 beats/min, baseline stroke volume, left ventricular ejection fraction b50%, and ATTRm status. For each 6-month increment, the mean 6-minute walk distance declined by 25.8 m, N-terminal pro b-type natriuretic peptide increased by 1,816 pg/mL, and left ventricular ejection fraction fell by 3.2%, for the entire cohort. Conclusions In this prospective study, disease progression, morbidity, and mortality were observed in ATTR cardiomyopathy, particularly due to V122I, over a short duration. Given the prevalence of this mutation, further study of V122I in at-risk African American patients is warranted. (Am Heart J 2012;164:222-228.e1.)
AB - Background TRACS sought to describe the clinical outcomes and disease progression of transthyretin (TTR) cardiac amyloidosis (ATTR) in an observational study. Clinical course is largely determined by disease type with ATTR categorized as wild-type (ATTRwt) or genetic-variant protein (ATTRm). Prospective data are lacking in the most common TTR mutation, V122I, present in approximately 3.5% of African Americans. Methods Patients with ATTRwt (n = 18) and V122I ATTRm (n = 11) were longitudinally assessed every 6 months for up to 2 years by functional class assessments, biochemical markers, and echocardiography. Results At baseline, no differences in clinical characteristics, biomarkers, or echocardiographic parameters were noted between patients with ATTRwt and patients with ATTRm. After 15.5 ± 8 months, there were 11 deaths and 1 cardiac transplant, with higher mortality (73% vs 22%, P = .03) and cardiovascular hospitalization (64% vs 28%, P = .02) among patients with ATTRm. The median survival from diagno is was 25.6 months for ATTRm vs 43.0 months for ATTRwt (P = .04). nivariate predictors of mortality included disease duration, heart rate ≤70 beats/min, baseline stroke volume, left ventricular ejection fraction b50%, and ATTRm status. For each 6-month increment, the mean 6-minute walk distance declined by 25.8 m, N-terminal pro b-type natriuretic peptide increased by 1,816 pg/mL, and left ventricular ejection fraction fell by 3.2%, for the entire cohort. Conclusions In this prospective study, disease progression, morbidity, and mortality were observed in ATTR cardiomyopathy, particularly due to V122I, over a short duration. Given the prevalence of this mutation, further study of V122I in at-risk African American patients is warranted. (Am Heart J 2012;164:222-228.e1.)
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U2 - 10.1016/j.ahj.2012.04.015
DO - 10.1016/j.ahj.2012.04.015
M3 - Article
C2 - 22877808
AN - SCOPUS:84864674714
SN - 0002-8703
VL - 164
JO - American Heart Journal
JF - American Heart Journal
IS - 2
ER -