scholarly journals Long-Term Survival With Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy

2021 ◽  
Author(s):  
Perry Elliott ◽  
Brian M. Drachman ◽  
Stephen S. Gottlieb ◽  
James E. Hoffman ◽  
Scott L. Hummel ◽  
...  
Keyword(s):  
New York ◽  
Free Acid ◽  
Heart Association ◽  
Class Iii ◽  
Unique Identifier ◽  
Transthyretin Amyloidosis ◽  
Term Survival ◽  
New York Heart Association ◽  
Amyloid Cardiomyopathy

Background: Tafamidis is approved in many countries for the treatment of transthyretin amyloid cardiomyopathy. This study reports data on the long-term efficacy of tafamidis from an ongoing long-term extension (LTE) to the pivotal ATTR-ACT (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial). Methods: Patients with transthyretin amyloid cardiomyopathy who completed ATTR-ACT could enroll in an LTE, continuing with the same tafamidis dose or, if previously treated with placebo, randomized (2:1) to tafamidis meglumine 80 or 20 mg. All patients in the LTE transitioned to tafamidis free acid 61 mg (bioequivalent to tafamidis meglumine 80 mg) following a protocol amendment. In this interim analysis, all-cause mortality was assessed in patients treated with tafamidis meglumine 80 mg in ATTR-ACT continuing in the LTE, compared with those receiving placebo in ATTR-ACT transitioning to tafamidis in the LTE. Results: Median follow-up was 58.5 months in the continuous tafamidis group (n=176) and 57.1 months in the placebo to tafamidis group (n=177). There were 79 (44.9%) deaths with continuous tafamidis and 111 (62.7%) with placebo to tafamidis (hazard ratio, 0.59 [95% CI, 0.44–0.79]; P <0.001). Mortality was also reduced in the continuous tafamidis (versus placebo to tafamidis) subgroups of: variant transthyretin amyloidosis (0.57 [0.33–0.99]; P =0.05) and wild-type transthyretin amyloidosis (0.61 [0.43–0.87]; P =0.006); and baseline New York Heart Association class I and II (0.56 [0.38–0.82]; P =0.003) and class III (0.65 [0.41–1.01]; P =0.06). Conclusions: In the LTE, patients initially treated with tafamidis in ATTR-ACT had substantially better survival than those first treated with placebo, highlighting the importance of early diagnosis and treatment in transthyretin amyloid cardiomyopathy. REGISTRATION: URL: https://www.clinicaltrials.gov ; Unique identifier: NCT01994889 and NCT02791230.

Left atrial high-grade rhabdomyosarcoma; an unusual location

2020 ◽  
pp. 021849232098348
Author(s):  
Hemant Chaturvedi ◽  
Ravindra Singh Rao ◽  
Navneet Mehta ◽  
Ajeet Bana
Keyword(s):  
New York ◽  
Heart Association ◽  
Surgical Excision ◽  
Interatrial Septum ◽  
Large Mass ◽  
Surgical Removal ◽  
High Grade ◽  
Class Iii ◽  
Term Survival ◽  
New York Heart Association

A 63-year-old diabetic and hypertensive lady presented in New York Heart Association class III–IV dyspnea on exertion. Echocardiography showed a large mass attached to the anterior mitral leaflet and the base of the interatrial septum. After removal of the mass and excision of the anterior and posterior mitral leaflets, a bioprosthetic valve was deployed. The postoperative course was uneventful. Histopathology showed that the tumor was a high-grade rhabdomyosarcoma. Although it is a highly lethal tumor, surgical removal was indicated to relieve dyspnea, clarify the diagnosis, and improve short-term survival. Our patient survived for 8 months after surgical excision.

scholarly journals Hemodynamic Profiles and Their Prognostic Relevance in Cardiac Amyloidosis

2020 ◽  
Vol 9 (4) ◽  
pp. 1093 ◽  
Author(s):  
Franz Duca ◽  
Amir Snidat ◽  
Christina Binder ◽  
René Rettl ◽  
Theresa-Marie Dachs ◽  
...  
Keyword(s):  
New York ◽  
Cardiac Amyloidosis ◽  
Heart Association ◽  
Prognostic Impact ◽  
Hemodynamic Parameters ◽  
Class Iii ◽  
Laboratory Assessment ◽  
Transthyretin Amyloidosis ◽  
Prognostic Relevance ◽  
New York Heart Association

This study sought to characterize cardiac amyloidosis (CA) patients with respect to hemodynamic parameters and asses their prognostic impact in different CA cohorts. Intracardiac and pulmonary arterial pressures (PAPs) are among the strongest predictors of outcomes in patients with heart failure (HF). Despite that, the hemodynamic profiles of patients with CA and their relation to prognosis have rarely been investigated. Invasive hemodynamic, clinical, and laboratory assessment, as well as cardiac magnetic resonance imaging were performed in our CA cohort. A total of 61 patients, 35 (57.4%) with wild-type transthyretin amyloidosis (ATTRwt) and 26 (42.6%) with light-chain amyloidosis (AL) were enrolled. ATTRwt patients had lower N-terminal prohormone of brain natriuretic peptide values and were less frequently in New York Heart Association class ≥ III. Intracardiac and PAPs were elevated, but hemodynamic parameters did not differ between CA groups. Whereas in ATTRwt, the median mean PAP (hazard ratio (HR): 1.130, p = 0.040) and pulmonary vascular resistance (HR: 1.010, p = 0.046) were independent predictors of outcome, no hemodynamic parameter was associated with outcome in the AL group. Cardiac ATTRwt and AL patients feature elevated intracardiac and PAPs and show similar hemodynamic profiles. However, hemodynamic parameters are of greater prognostic relevance in ATTRwt, potentially providing a new therapeutic target.

Tafamidis: A First-in-Class Transthyretin Stabilizer for Transthyretin Amyloid Cardiomyopathy

2019 ◽  
Vol 54 (5) ◽  
pp. 470-477 ◽  
Author(s):  
Jonathan Park ◽  
Ugochukwu Egolum ◽  
Shanea Parker ◽  
Ebony Andrews ◽  
David Ombengi ◽  
...  
Keyword(s):  
New York ◽  
English Language ◽  
Data Extraction ◽  
Functional Decline ◽  
Heart Association ◽  
Underlying Disease ◽  
Class Iii ◽  
Association Class ◽  
New York Heart Association ◽  
Amyloid Cardiomyopathy

Objective: To review the pharmacology, efficacy, and safety of the selective transthyretin inhibitor tafamidis for transthyretin amyloid cardiomyopathy (ATTR-CM). Data Sources: A PubMed (1966 to October 2019) and ClinicalTrials. gov search was conducted using the keywords tafamidis, Fx-1006A, Vyndaqel, and Vyndamax. Additional articles were identified from references. Study Selection and Data Extraction: We included English-language clinical studies evaluating the pharmacology, efficacy, or safety of tafamidis in humans for ATTR-CM. Data Synthesis: Tafamidis binds to the thyroxine-binding sites of the transthyretin tetramer and inhibits its dissociation into monomers, which is the rate-limiting step in the amyloidogenic process. Treatment with tafamidis was significantly associated with a significant reduction in mortality, lowered cardiovascular-related hospitalizations, less functional decline, and improved transthyretin stabilization compared with placebo. Additionally, tafamidis was found to have fewer adverse events, with no difference found compared with placebo. Relevance to Patient Care and Clinical Practice: Historically, symptomatic management for ATTR-CM was the only option, and the treatment of the underlying disease was limited to liver or heart transplantation. Tafamidis is the first medication approved for the treatment of ATTR-CM and the only medication that showed a reduction in all-cause mortality and cardiovascular-related hospitalizations in patients with amyloidosis. However, the role of tafamidis in patients with the New York Heart Association class III/IV heart failure or mutated transthyretin remains unclear. Conclusion: Tafamidis is an effective and safe oral medication for the treatment of the cardiomyopathy of transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization.

scholarly journals Impact of Cardiorenal Anemia Syndrome on Short- and Long-Term Clinical Outcomes in Patients Hospitalized with Heart Failure

2016 ◽  
Vol 6 (4) ◽  
pp. 269-278
Author(s):  
Chan Joon Kim ◽  
Ik-Jun Choi ◽  
Hun-Jun Park ◽  
Tae Hoon Kim ◽  
Pum-Joon Kim ◽  
...  
Keyword(s):  
Heart Failure ◽  
New York ◽  
Primary Outcome ◽  
Heart Association ◽  
Class Iii ◽  
Increased Risk ◽  
New York Heart Association ◽  
Short And Long Term ◽  
The Impact

Background: Dysfunctional interplay between the heart and kidneys may lead to the development of anemia. The aim of this study was to evaluate the impact of cardiorenal anemia syndrome (CRAS) on short- and long-term outcomes among patients hospitalized with heart failure (HF). Methods: We enrolled 303 patients hospitalized with HF. We divided the patients into two groups: a CRAS group (n = 64) and a non-CRAS group (n = 239). We defined CRAS as HF accompanied by (1) an estimated glomerular filtration rate <60 ml/min/1.73 m2 calculated by the Modification of Diet in Renal Disease at admission and (2) a hemoglobin level <12 g/dl for females and <13 g/dl for males at admission. The primary outcome was a composite of cardiac death, non-fatal myocardial infarction and rehospitalization for HF. Results: During a median follow-up period of 25.6 months (range 0.1-35.3 months), the patients with CRAS had a significantly increased risk for the primary outcome (27.5 vs. 10.7%, p < 0.001) compared with the patients in the non-CRAS group. Using Cox proportional hazard analyses, the hazard ratio (HR) for the presence of CRAS was found to be 1.874 (95% confidence interval [CI] 1.011-3.475, p = 0.046); HRs were also computed for the presence of diabetes mellitus (HR = 2.241, 95% CI 1.221-4.112, p = 0.009), New York Heart Association class III or IV HF (HR = 2.948, 95% CI 1.206-7.205, p = 0.018) and the use of intravenous loop diuretics (HR = 2.286, 95% CI 0.926-5.641, p = 0.073). Conclusions: Renal dysfunction and anemia are a fatal combination and are associated with poor prognosis in patients with HF.

scholarly journals Fifteen-year experience with pericardiectomy at a tertiary referral center

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Zainab Faiza ◽  
Anjali Prakash ◽  
Niharika Namburi ◽  
Bailey Johnson ◽  
Lava Timsina ◽  
...  
Keyword(s):  
New York ◽  
Heart Association ◽  
Underlying Disease ◽  
Term Survival ◽  
Disease Etiology ◽  
Mortality And Morbidity ◽  
Tertiary Referral Center ◽  
New York Heart Association ◽  
Overall Mortality

Abstract Purpose Pericardiectomy has traditionally carried relatively high perioperative mortality and morbidity, with few published reports of intermediate- and long- term outcomes. We investigated our 15-year experience performing pericardiectomy at our institution. Methods Retrospective study of all patients who underwent pericardiectomy at our institution between 2005 and 2019. Baseline demographics, intraoperative details, and postoperative outcomes including long-term survival were analyzed. Results Sixty-three patients were included in the study. 66.7% of subjects underwent isolated pericardiectomy while 33.3% underwent pericardiectomy concomitantly with another cardiac surgical procedure. The most common indications for pericardiectomy were constrictive (79.4%) and hemorrhagic (9.5%) pericarditis. Preoperatively, 76.2% of patients were New York Heart Association class II and III, while postoperatively, 71.4% were class I and II. One-, three-, five-, and ten- year overall mortality was 9.5, 14.3, 20.6, and 25.4%, respectively. Overall pericarditis recurrence rate was 4.8%. Conclusion Pericardiectomy carries relatively high overall mortality rates, which likely reflects underlying disease etiology and comorbidities. Patients with prior cardiac intervention, history of dialysis, and immunocompromised state are associated with worse outcomes.

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