{Reference Type}: Journal Article
{Title}: Stratifying Disease Progression in Patients With Cardiac ATTR Amyloidosis.
{Author}: Ioannou A;Cappelli F;Emdin M;Nitsche C;Longhi S;Masri A;Cipriani A;Zampieri M;Colio F;Poledniczek M;Porcari A;Razvi Y;Aimo A;Vergaro G;De Michieli L;Rauf MU;Patel RK;Villanueva E;Lustig Y;Venneri L;Martinez-Naharro A;Lachmann H;Wechalekar A;Whelan C;Petrie A;Hawkins PN;Solomon S;Gillmore JD;Fontana M;
{Journal}: J Am Coll Cardiol
{Volume}: 0
{Issue}: 0
{Year}: 2024 Mar 1
{Factor}: 27.203
{DOI}: 10.1016/j.jacc.2023.12.036
{Abstract}: <B>BACKGROUND: </B>Transthyretin cardiac amyloidosis (ATTR-CA) is a progressive cardiomyopathy. The clinical course varies among individuals and there are no established measures to assess disease progression.<BR><B>OBJECTIVE: </B>The goal of this study was to assess the prognostic importance of an increase in N-terminal pro-B-type natriuretic peptide (NT-proBNP) and outpatient diuretic intensification (ODI) as markers of disease progression in a large cohort of patients with ATTR-CA.<BR><B>METHODS: </B>We evaluated landmark survival analysis based on worsening of NT-proBNP and requirement for ODI between time of diagnosis and a 1-year visit, and subsequent mortality in 2,275 patients with ATTR-CA from 7 specialist centers. The variables were developed in the National Amyloidosis Centre (NAC) cohort (n = 1,598) and validated in the external cohort from the remaining centers (n = 677).<BR><B>RESULTS: </B>Between baseline and 1-year visits, 551 (34.5%) NAC patients and 204 (30.1%) patients in the external validation cohort experienced NT-proBNP progression (NT-proBNP increase >700 ng/L and >30%), which was associated with mortality (NAC cohort: HR: 1.82; 95% CI: 1.57-2.10; P < 0.001; validation cohort: HR: 1.75; 95% CI: 1.32-2.33; P < 0.001). At 1 year, 451 (28.2%) NAC patients and 301 (44.5%) patients in the external validation cohort experienced ODI, which was associated with mortality (NAC cohort: HR: 1.88; 95% CI: 1.62-2.18; P < 0.001; validation cohort: HR: 2.05; 95% CI: 1.53-2.74; P < 0.001). When compared with patients with a stable NT-proBNP and stable diuretic dose, a higher risk of mortality was observed in those experiencing either NT-proBNP progression or ODI (NAC cohort: HR: 1.93; 95% CI: 1.65-2.27; P < 0.001; validation cohort: HR: 1.94; 95% CI: 1.36-2.77; P < 0.001), and those experiencing both NT-proBNP progression and ODI (NAC cohort: HR: 2.98; 95% CI: 2.42-3.67; P < 0.001; validation cohort: HR: 3.23; 95% CI: 2.17-4.79; P < 0.001).<BR><B>CONCLUSIONS: </B>NT-proBNP progression and ODI are frequent and consistently associated with an increased risk of mortality. Combining both variables produces a simple, universally applicable model that detects disease progression in ATTR-CA.