FDA OKs Two Tafamidis Formulations for Rare Cardiomyopathy FDA OKs Two Tafamidis Formulations for Rare Cardiomyopathy
These are the first FDA-approved treatments for transthyretin-mediated amyloidosis cardiomyopathy, a rare and often fatal disease caused by deposition of amyloid fibrils in the myocardium.FDA Approvals
ConclusionsRisk stratification for cardiac amyloidosis with the newly developed “HeiRisk” score may be superior to other staging systems for patients with advanced heart failure due to amyloid cardiomyopathy.
Conclusions In clinical practice, pimobendan seems to have beneficial effects in heart failure management for improving physical activities and the quality of life in patients with transthyretin cardiac amyloidosis. PMID: 31588087 [PubMed - as supplied by publisher]
Authors: Milani P, Mussinelli R, Perlini S, Palladini G, Obici L Abstract Introduction: Hereditary transthyretin-mediated amyloidosis (ATTRv; v for variant) is a rare, progressive, fatal multi-systemic disease, autosomal dominantly inherited with heterogeneous clinical phenotype caused by mutations in the TTR gene. Mutations promoting proteolytic remodeling and tetramer dissociation result in fragmented and full-length TTR monomers that misfold, aggregate and deposit at multiple sites (mainly nerves and heart) causing peripheral neuropathy and/or cardiomyopathy. Areas covered: The authors discuss patisiran, the fir...
Cardiac involvement is associated with poor prognosis in immunoglobulin light-chain amyloidosis (AL amyloidosis), with a median survival of 6 months without heart transplantation (HT) from the onset of symptomatic heart failure. Although HT is a promising treatment option for patients with amyloid cardiomyopathy (ACM), consensus on its indication is not fully established because of limited experience. Method: We reviewed our prospective AL amyloidosis database to identify all patients who received HT for their ACM.
Condition: Transthyretin (TTR) Amyloid Cardiomyopathy Intervention: Drug: Treatment for TTR amyloidosis Sponsor: Pfizer Not yet recruiting
AbstractTransthyretin cardiac amyloidosis (ATTR ‐CA) demonstrates progressive, potentially fatal, and infiltrative cardiomyopathy caused by extracellular deposition of transthyretin‐derived insoluble amyloid fibrils in the myocardium. Two distinct types of transthyretin (wild type or variant) become unstable, and misfolding forms aggregate, r esulting in amyloid fibrils. ATTR‐CA, which has previously been underrecognized and considered to be rare, has been increasingly recognized as a cause of heart failure with preserved ejection fraction among elderly persons. With the advanced technology, the diagnostic tools have...
ConclusionOur findings reveal that ATTR p.A97S is a cardiomyopathy as well as a polyneuropathic syndrome. Routine use of more contemporary echocardiographic techniques are recommended to identify cardiac amyloidosis and provide prognostic information.
CONCLUSION: Our findings reveal that ATTR p.A97S is a cardiomyopathy as well as a polyneuropathic syndrome. Routine use of more contemporary echocardiographic techniques are recommended to identify cardiac amyloidosis and provide prognostic information. PMID: 31521469 [PubMed - as supplied by publisher]