Risultati per: Gestione della stenosi mitralica reumatica (MVA <= 1,5 cm)

Araldi, ‘con MedMal Report identifichiamo aree più a rischio’

Circulation, Ahead of Print. Background: In the phase 3 randomized controlled study, ATTRibute-CM, acoramidis, a transthyretin (TTR) stabilizer, demonstrated significant efficacy on the primary endpoint. Participants with transthyretin amyloid cardiomyopathy (ATTR-CM) who completed ATTRibute-CM were invited to enroll in an open-label extension study (OLE). We report efficacy and safety data of acoramidis in participants who completed ATTRibute-CM and enrolled in the ongoing OLE.Methods: Participants who previously received acoramidis through Month 30 (M30) in ATTRibute-CM continued to receive it (continuous acoramidis), and those who received placebo through M30 were switched to acoramidis (placebo to acoramidis). Participants who received concomitant tafamidis in ATTRibute-CM were required to discontinue it to be eligible to enroll in the OLE. Clinical efficacy outcomes analyzed through Month 42 (M42) included time to event for all-cause mortality (ACM) or first cardiovascular-related hospitalization (CVH), ACM alone, first CVH alone, ACM or recurrent CVH, change from baseline in N-terminal pro-B-type natriuretic peptide (NT-proBNP), 6-minute walk distance (6MWD), serum TTR, and the Kansas City Cardiomyopathy Questionnaire Overall Summary score (KCCQ-OS). Safety outcomes were analyzed through M42.Results: Overall, 438 of 632 participants in ATTRibute-CM completed treatment and 389 enrolled in the ongoing OLE (263 continuous acoramidis, 126 placebo to acoramidis). The hazard ratio (HR) (95% CI) for ACM or first CVH was 0.57 (0.46, 0.72) at M42 based on a stratified Cox proportional hazards model (P-value < 0.0001) favoring continuous acoramidis. Similar analyses were performed on ACM alone and first CVH alone, with HRs (95% CI) of 0.64 (0.47, 0.88) and 0.53 (0.41, 0.69), respectively, at M42. Treatment effects for NT-proBNP and 6MWD also favored continuous acoramidis. Upon initiation of open-label acoramidis in the placebo-to-acoramidis arm there was a prompt increase in serum TTR. Quality of life assessed by KCCQ-OS was well preserved in continuous acoramidis participants compared with the placebo to acoramidis participants. No new clinically important safety issues were identified in this long-term evaluation.Conclusions: Early initiation and continuous use of acoramidis in the ATTRibute-CM study through M42 of the ongoing OLE study was associated with sustained clinical benefits in a contemporary ATTR-CM cohort, with no clinically important safety issues newly identified.

I moderni dispositivi permettono di adattare la terapia insulinica alle esigenze individuali di ciascun paziente, migliorando l’efficacia del trattamento

Circulation, Volume 150, Issue Suppl_1, Page A4138981-A4138981, November 12, 2024. Background:Conduction system disease, atrial and ventricular arrhythmias may require pacemaker or ICD implantation in transthyretin amyloid cardiomyopathy (ATTR-CM). The optimal pacing mode in ATTR-CM patients remains unknown.Objective:To assess how availability of cardiac resynchronization and conduction system pacing influenced pacemaker indications and to compare early electrical performance of conventional, resynchronisation and conduction system pacing in ATTR-CM.Methods and Results:67 of 250 (26.8%) patients presenting with ATTR-CM between June 2019 and February 2023 received a pacemaker [HA1] and were included in this retrospective analysis. Pacemaker implantation occurred in 25 patients (37.3%) prior to, and in 42 (62.7%) after ATTR-CM diagnosis. Implantation of conventional single- (VVI) or dual-chamber (DDD) pacemakers was more common (n=17/25, 68%) in undiagnosed ATTR-CM, while physiological pacing systems (CRT/ CSP) were preferably implanted in patients (n=24/42; 57.1%) after diagnosis. Sick sinus syndrome (11/35; 31.4% vs. 6/32; 18.8%) and higher degree AV-block (20/35; 57.1% vs. 9/32; 28.1%) were more common indications for VVI/DDD compared to CRT/CSP pacemakers, with pursuit of a pace/ablate strategy (12/32; 37.5%) and heart failure (5/32; 15.6%) contributing significantly to implantation of CRT/CSP. QRS width was significantly lower with CSP [122ms (IQR: 120-139)], compared to CRT [155ms (IQR: 141-160); p=0.005] or VVI/DDD [160ms (IQR: 144-180)], with the latter resulting in a significant increase in QRS width compared to intrinsic QRS [from 138 (IQR: 123-150) (p0.05).Conclusions:Pacing indications are changing with earlier diagnosis of ATTR-CM. CSP may offer improved electrical performance and resynchronization, the effect of conduction system pacing on clinical outcomes should be further explored.

Circulation, Volume 150, Issue Suppl_1, Page A4143171-A4143171, November 12, 2024. Background:Consensus on a definition of disease progression in transthyretin amyloid cardiomyopathy (ATTR-CM) is lacking. Recent data showed that among a large cohort 50% of patients progressed as defined by either N-terminal pro-B type natriuretic peptide (Nt-proBNP) increase of 700 pg/ml AND 30% or increase of oral diuretics (ODI) after 1 year. However, most of the patients were not on disease modifying treatment (DMT).Aim:To describe the pattern of progression at 1 year in patients with ATTR-CM on tafamidis, the only approved drug for ATTR-CM.Methods:Patients diagnosed with ATTR-CM at Columbia University and treated with tafamidis after 2018 were enrolled in the study assuming they survived a year to measure disease progression and had data available at follow-up. A total of 16 patients died before the 1-year follow-up and were excluded from the study. Disease progression was defined as: increase in Nt-proBNP >700 pg/mL and >30% from baseline, ODI or increases in both compared to baseline. Survival analysis was done using Kaplan Meier curves and Cox regression adjusted for age and disease severity by National Amyloidosis Center stage.Results:A total of 157 patients were enrolled, with 62 (39.5%) having disease progression at 1-year. Of these 62 patients, 51 met one progression criterion (34 patients had ODI and 17 had NtproBNP increase), with 11 having both criteria of disease progression at 1 year. Survival analysis revealed a significantly increased risk for all-cause mortality during follow-up in patients with disease progression at 1-year (HR=2.13, 95%CI:1.04-4.35, p=0.03, Figure).Conclusion:The recently proposed criteria for disease progression performed well in a cohort of patients on DMT, which may reduce the number of patients progressing at 1 year.

Circulation, Volume 150, Issue Suppl_1, Page A4139239-A4139239, November 12, 2024. Objective:Identification of individuals with reduced or preserved ejection fraction heart failure (HFrEF/HFpEF) within claims data is typically based on ICD-10-CM diagnosis codes that use systolic and diastolic HF (SHF/DHF) nomenclature. The objective of this study was to assess the performance characteristics of using ICD-10-CM diagnostic codes from claims data for HFrEF and HFpEF classification relative to a reference standard using EF results or clinician documentation within an integrated claims/EMR database.Methods:EMR data from the Healthcare Integrated Research Database (HIRD®) were searched to identify patients with EF assessment between 01/01/2016 and 01/31/2023. HFrEF was defined as EF ≤ 40% or documented reduced EF, while HFpEF was defined as EF ≥ 50% or documented preserved/normal EF. The most recent EF assessment date or EMR entry date (if EF assessment date not available) was set as the index date. Claims submitted from 7 days to 6 months post index date were then reviewed to identify SHF and DHF diagnoses as well as comorbid conditions. Analyses were performed to determine sensitivity, specificity, and positive/negative predictive values (PPV/NPV), accuracy and F1 scores of the claims-based algorithm, with a sensitivity analysis performed using the subset of patients with a known EF assessment date available.Results:A total of 45,272 patients had EF assessment in the EMR data with either a SHF or DHF diagnoses in the claims data. Mean (SD) age was 71.7 (12.7) years, 51.2% were male. The most common comorbidities of interest included hypertension (89.5%), dyslipidemia (71.9%), atrial fibrillation (45.9%), type 2 diabetes (43.7%), and chronic kidney disease (39.6%). Counts by heart failure classification and algorithm performance characteristics are in Table 1. Sensitivity analyses for those with known EF assessment dates showed similar results.Conclusions:Overall performance of the claims-based algorithm was good to very good, although EF data integrated with claims data can improve HF classification. Future claims-based algorithm development could also incorporate treatments and comorbidities to improve performance.

Circulation, Volume 150, Issue Suppl_1, Page A4124506-A4124506, November 12, 2024. Background:ATTR-CM has an age dependent prevalence and is a disorder that almost exclusively affects older adults. Objective evaluations of function are critical to assessing and managing ATTR-CM in older adults. The short physical performance battery (SPPB) is a valid measure of functional capacity that predicts morbidity and mortality in older adults but its utility in ATTR-CM remains unknown.Aims/hypothesis:To establish SPPB as a useful marker of disease severity and predictor of outcomes in ATTR-CM. We hypothesized that SPPB scores would correlate with validated markers of ATTR-CM severity and improve clinical prediction.Methods:This is a retrospective analysis of patients referred to the Columbia University Cardiac Amyloid Program. Patients were stratified into low (SPPB 0-6), moderate (7-9), and high (10-12) cohorts based on initial SPPB score and baseline characteristics were compared between groups. Cox proportional hazard models and Kaplan Meier (KM) curves were generated to assess associations with mortality as well as a composite of death and cardiovascular (CV) hospitalization in follow-up.Results:A total of 263 patients, age 78 years (IQR 73, 84), 86% male, 22% with ATTRv (variant) and 78% with ATTRwt (wild type) were studied. SPPB showed no limitation in 59%, mild limitation in 33%, and severe limitation in 8%. Lower SPPB was associated (p

Circulation, Volume 150, Issue Suppl_1, Page A4147141-A4147141, November 12, 2024. Background:Acoramidis is a novel, potent, investigational, transthyretin (TTR) stabilizer under development for the treatment of TTR amyloidosis that results in near-complete (≥90%) TTR stabilization. In a phase 3 study, ATTRibute-CM, acoramidis demonstrated improved clinical outcomes in participants with transthyretin amyloid cardiomyopathy (ATTR-CM), including a 50% reduction in the risk of CVH compared to placebo over 30 months. The study also demonstrated that cardiovascular hospitalization (CVH) during the study predicted a higher subsequent mortality in participants with ATTR-CM.Hypothesis:CVH portends a higher risk of mortality in participants with ATTR-CM. Since acoramidis reduces CVH, it can improve the prognosis of participants with ATTR-CM.Aim:To evaluate the relationship between CVH and survival in the acoramidis group within ATTRibute-CM.Methods:In this post-hoc analysis, the relationship between those with or without CVH and survival was analyzed within the acoramidis treatment group using the Kaplan-Meier (KM) estimator method.Results:Demographics and baseline disease characteristics were mostly comparable between acoramidis-treated participants with and without CVH, although participants with CVH had a higher baseline NT-proBNP and lower eGFR. At Month 30, in acoramidis-treated participants, those without any CVH (n=300) had a higher survival [86.8% (95% CI = 82.2, 90.3)] versus 62.4% (95% CI = 52.6, 70.7) in those who had any CVH (n=109); p