Treatment of amyloidosis: present and future
Maria Teresa Mallus1, Vittoria Rizzello1
1Cardiology Unit, Cardiovascular Department, San Giovanni Addolorata Hospital, Rome.
Insights
Cardiac amyloidosis (CA) is an underdiagnosed infiltrative heart disease. Recent advances in understanding its pathophysiology have led to effective therapies for light chain (AL) and transthyretin (ATTR) amyloidosis.
Area of Science:
- Cardiology
- Internal Medicine
- Genetics
Background:
- Cardiac amyloidosis (CA) is an infiltrative myocardial disease caused by amyloid fibril deposition.
- The primary forms include light chain (AL) and transthyretin amyloidosis (ATTR), with ATTR further classified into familial and wild-type/senile subtypes.
- CA is often underdiagnosed, leading to delayed treatment and poorer prognosis.
Purpose of the Study:
- To review the pathophysiology of cardiac amyloidosis.
- To discuss current and emerging therapeutic strategies for AL and ATTR amyloidosis.
- To highlight the importance of timely diagnosis and comprehensive supportive care for CA patients.
Main Methods:
- Literature review of recent advancements in CA diagnosis and treatment.
- Analysis of therapeutic approaches targeting amyloid fibril formation and stabilization.
- Overview of supportive care measures for managing CA complications.
Main Results:
- Effective therapies for AL amyloidosis involve proteasome inhibitors and monoclonal antibodies.
- ATTR amyloidosis treatments focus on transthyretin (TTR) gene silencers and TTR tetramer stabilizers.
- Understanding CA pathophysiology has enabled targeted therapeutic development.
Conclusions:
- Early diagnosis and initiation of specific therapies significantly improve outcomes for CA patients.
- Novel treatments targeting amyloidogenesis offer new hope for managing AL and ATTR amyloidosis.
- Comprehensive management of CA requires addressing amyloid deposition and associated cardiac complications.
Abstract:
Cardiac amyloidosis (CA) is an infiltrative heart disease resulting from the deposition of amyloid fibrils in the interstitial spaces of the myocardium. The two main forms of CA are represented by light chain amyloidosis (AL) and transthyretin amyloidosis (ATTR) in the two forms familial or variant or wild-type or senile. Although considered a rare disease, CA is an underdiagnosed disease. Delay in diagnosis has a negative impact on the prognosis, delaying the initiation of specific therapy. The treatment of both forms of CA is based on: (i) prevention and slowing of the generation and deposition of amyloid fibrils and (ii) supportive care of complications. The main success of recent years has been the development of effective therapies that have been possible thanks to the understanding of the pathophysiology of amyloidosis. For the AL form, new therapeutic combinations between a proteasome inhibitor and a monoclonal antibody have been developed. For ATTR forms, the main strategies are transthyretin (TTR) production 'silencers' and TTR tetramer stabilizers. Supportive care of patients with CA involves various clinical aspects including treatment of heart failure, arrhythmias, conduction disturbances, thrombo-embolism, and the concomitant presence of aortic stenosis.
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