scholarly journals Hereditary Transthyretin Amyloid Cardiomyopathy

2021 ◽  
Author(s):  
Quentin R. Youmans ◽  
Sanjiv J. Shah ◽  
Sadiya S. Khan
Keyword(s):  
Amyloid Cardiomyopathy

scholarly journals US CARDIOLOGIST AND PATIENT PERSPECTIVES ON COVID-19 TELEHEALTH PRACTICES FOR PATIENTS WITH TRANSTHYRETIN AMYLOID CARDIOMYOPATHY (ATTR-CM)

2021 ◽  
Vol 77 (18) ◽  
pp. 690
Author(s):  
Sumeet Mitter ◽  
Keyur Shah ◽  
Alexandra Haddad-Angulo ◽  
Adam Castano ◽  
Marianna Bruno
Keyword(s):  
Patient Perspectives ◽  
Amyloid Cardiomyopathy

scholarly journals CARDIOVASCULAR SYMPTOM BURDEN PRIOR TO DIAGNOSIS OF TRANSTHYRETIN AMYLOID CARDIOMYOPATHY AMONG MEDICARE BENEFICIARIES

2021 ◽  
Vol 77 (18) ◽  
pp. 746
Author(s):  
Duncan Brown ◽  
Montserrat Vera-Llonch ◽  
Jose Tomas Ortiz Perez ◽  
Sheila R. Reddy ◽  
Eunice Chang ◽  
...  
Keyword(s):  
Symptom Burden ◽  
Medicare Beneficiaries ◽  
Amyloid Cardiomyopathy

Benefits of tafamidis in patients with advanced transthyretin amyloid cardiomyopathy

2020 ◽  
Vol 41 (Supplement_2) ◽  
Author(s):  
C Rapezzi ◽  
A.V Kristen ◽  
B Gundapaneni ◽  
M.B Sultan ◽  
M Hanna
Keyword(s):  
Disease Severity ◽  
Nyha Class ◽  
Funding Source ◽  
Class Iii ◽  
Private Company ◽  
Risk Of Death ◽  
6Mwt Distance ◽  
All Cause Mortality ◽  
Amyloid Cardiomyopathy

Abstract Background In the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT), tafamidis was shown to be an effective treatment for patients with transthyretin amyloid cardiomyopathy (ATTR-CM). Further assessment of the efficacy of tafamidis in patients with more advanced ATTR-CM would aid treatment decisions. Purpose To characterize the benefits of tafamidis in patients with advanced ATTR-CM. Methods In ATTR-ACT, ATTR-CM patients were randomized to tafamidis (n=264) or placebo (n=177) for 30 months. Efficacy outcomes included all-cause mortality and frequency of cardiovascular (CV)-related hospitalisations. Key secondary endpoints were change from baseline to Month 30 in 6MWT distance and KCCQ-OS score. Efficacy assessments in NYHA Class III patients at baseline (n=141) were a pre-specified analysis. In a post-hoc analysis, mortality and CV-related hospitalizations were assessed in all patients grouped into quartiles of increasing disease severity based on 6MWT distance at baseline. Longer-term all-cause mortality (as of 1 Aug 2019) was assessed in NYHA Class III patients utilizing data from ATTR-ACT patients who enrolled in a long-term, extension study (LTE) and continued treatment with higher dose tafamidis (n=55; median treatment duration 51.6 months); or, if previously treated with placebo, started tafamidis treatment (placebo/tafamidis; n=63 [50.1 months]). Results In advanced ATTR-CM patients (NYHA Class III), tafamidis reduced the risk of death (HR [95% CI] 0.837, [0.541, 1.295], P=0.4253), and the decline in 6MWT distance (LS mean [SE], 31.6 (22.1) m; P=0.1526) and KCCQ-OS score (LS mean [SE], 13.1 (5.0); P=0.0090), vs placebo. Paradoxically, there was a higher frequency of CV-related hospitalizations with tafamidis (RR [95% CI] vs placebo, 1.411 [1.048, 1.900]). In all patients by 6MWT quartile, CV-related hospitalizations/year with tafamidis and placebo increased with disease severity, with the exception that placebo-treated patients in the highest severity quartile had fewer CV-related hospitalisations (0.73) than those in the third quartile (0.92). Mortality with tafamidis and placebo increased, and was greater with placebo, in every quartile (Figure). Survival (NYHA Class III patients in ATTR-ACT and LTE) was improved with high dose tafamidis with longer term follow-up (HR vs placebo/tafamidis [95% CI], 0.6569 [0.4175, 1.0336]; P=0.0692). Conclusions These analyses, including longer-term follow-up, demonstrate that patients with advanced ATTR-CM benefit from tafamidis. The decrease in CV-related hospitalisations in more severe patients treated with placebo suggests that the comparatively greater hospitalisation frequency in NYHA Class III patients treated with tafamidis is a consequence of their lower mortality rate. Figure 1 Funding Acknowledgement Type of funding source: Private company. Main funding source(s): This study was sponsored by Pfizer

scholarly journals Wild-type transthyretin amyloid cardiomyopathy complicated by spinal canal stenosis, carpal tunnel syndrome, and rotator cuff tears: a case report

2020 ◽  
Author(s):  
Seiji Takashio ◽  
Masato Nishi ◽  
Yuichiro Tsuruta ◽  
Kenichi Tsujita
Keyword(s):  
Rotator Cuff ◽  
Spinal Canal ◽  
Spinal Canal Stenosis ◽  
Rotator Cuff Tears ◽  
Lumbar Spinal Canal Stenosis ◽  
Canal Stenosis ◽  
Tunnel Syndrome ◽  
Amyloid Cardiomyopathy ◽  
Lumbar Spinal Canal ◽  
Lumbar Spinal

Abstract Background Wild-type transthyretin amyloid cardiomyopathy (ATTRwt-CM) is receiving increasing attention due to the availability of novel treatment options. Carpal tunnel syndrome (CTS) and lumbar spinal canal stenosis are known early symptoms of transthyretin (TTR) amyloidosis preceding the cardiac involvement and are considered as ‘Red Flags’ for transthyretin amyloid cardiomyopathy (ATTR-CM). Case summary A 67-year-old man with a history of lumbar spinal canal stenosis for the last 10 years, right rotator cuff tears for the last 4 years, and bilateral CTS for the last 1 year was scheduled for orthopaedic surgery for lumbar spinal canal stenosis. Investigations revealed severe left ventricular hypertrophy and hypertroponinaemia, which were suggestive of cardiac amyloidosis. Cardiac magnetic resonance imaging and 99mTc-labelled pyrophosphate scintigraphy demonstrated positive findings for ATTR-CM. Transthyretin deposition was found in both the myocardium and the yellow ligamentum excised during surgery. There was no transthyretin mutation on genetic testing. The final diagnosis was ATTRwt-CM. Discussion Transthyretin deposition in the ligaments or tendons has been observed in a number of patients with CTS, spinal canal stenosis, and rotator cuff tears. These orthopaedic diseases are predictive for the future occurrence of ATTR-CM. In addition, the coexistence of these multiple diseases might strongly predict ATTR-CM. This knowledge needs to be shared with orthopaedicians and cardiologists for the early diagnosis of ATTR-CM.

scholarly journals TEMPORAL TRENDS IN DIAGNOSTIC TESTING PATTERNS FOR WILD-TYPE TRANSTHYRETIN AMYLOID CARDIOMYOPATHY IN THE MEDICARE FEE-FOR-SERVICE POPULATION

2021 ◽  
Vol 77 (18) ◽  
pp. 528
Author(s):  
Jamieson M Bourque ◽  
Alexander Schepart ◽  
Rahul Bhambri ◽  
Adam Castaño ◽  
Alex O’Brien ◽  
...  
Keyword(s):  
Diagnostic Testing ◽  
Temporal Trends ◽  
Fee For Service ◽  
Wild Type ◽  
Amyloid Cardiomyopathy ◽  
Testing Patterns

Transthyretin Amyloid Cardiomyopathy—Current and Future Therapies

2021 ◽  
pp. 106002802110003
Author(s):  
Jankhna D. Yadav ◽  
Harjot Othee ◽  
Kelly A. Chan ◽  
Damen C. Man ◽  
Paul P. Belliveau ◽  
...  
Keyword(s):  
Cardiac Amyloidosis ◽  
Complex Disease ◽  
Clinical Presentation ◽  
Target Therapy ◽  
Data Extraction ◽  
Organ Transplant ◽  
Standard Of Care ◽  
Transthyretin Amyloidosis ◽  
Treatment Benefits ◽  
Amyloid Cardiomyopathy

Objective: To describe the clinical presentation of transthyretin amyloid cardiomyopathy (ATTR-CM) and discuss current treatments and investigational products and their effect on patient outcomes. Data Sources: A literature search was performed in PubMed (September 2018 to December 2020) using the following keywords: transthyretin amyloidosis, cardiomyopathy, polyneuropathy and transthyretin amyloid cardiomyopathy, monoclonal light-chain, tafamidis, cardiac amyloidosis, ATTR cardiomyopathy, green tea and inhibition of cardiac amyloidosis, AG10, tolcapone, tolcapone and leptomeningeal ATTR, PRX004, NI006, patisiran, inotersen, vutrisiran, AKCEA-TTR-LRx, and NTLA-2001. Study Selection and Data Extraction: Clinical trials were evaluated for evidence supporting pharmacology, safety, efficacy, and measured outcomes. Data Synthesis: Until 2019, there were no approved treatments for ATTR-CM. Treatment consisted of symptom management and organ transplant. Nonpharmacological and pharmacological treatments focused on the symptoms of heart failure (HF) associated with ATTR-CM. However, there are several emerging therapies recently approved or in development to address the underlying pathophysiology. Treatment classes for ATTR-CM include transthyretin stabilizers, human monoclonal antibodies, gene silencers, and CRISPR/Cas9 gene editing. Relevance to Patient Care and Clinical Practice: ATTR-CM is a complex disease in which amyloidosis causes cardiomyopathy. Underdiagnosis is attributed to the clinical presentation being heterogeneous, indistinguishable from HF caused by other etiologies, and the need for invasive testing modalities, including endomyocardial biopsy. Improved diagnostic approaches along with targeted therapies can slow disease progression and enhance patient quality of life. Conclusion: Diagnostic modalities along with biomarker and genetic testing could detect disease earlier and target therapy more accurately. Novel therapies demonstrate potential treatment benefits and can help shape the standard of care for these patients.

Molecular diagnosis of the transthyretin (TTR) Met111 mutation in familial amyloid cardiomyopathy of Danish origin

1992 ◽  
Vol 89 (4) ◽  
Author(s):  
Bj�rn-Yngvar Nordv�g ◽  
Gunnar Husby ◽  
Ida Ranl�v ◽  
M.Raafat El-Gewely
Keyword(s):  
Molecular Diagnosis ◽  
Amyloid Cardiomyopathy
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