AL type cardiac amyloidosis: a devastating fatal disease

Nasrullah, Adeel; Javed, Anam; Jayakrishnan, Thejus; Brumbaugh, Aaron D.; Sandhu, Ariel; Hardman, Brent

doi:10.1080/20009666.2021.1915547

Cited by 2 publications

(3 citation statements)

References 16 publications

(24 reference statements)

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“…The heart is affected in 75% of confirmed cases [15]. The most common presenting symptoms are fatigue, weakness, and decreased exercise tolerance [16]. During clinical examination, there are usually signs and symptoms of diastolic heart failure because of restrictive cardiomyopathy.…”

Section: Q6: What Is the Most Commonly Affected Organ In Al Amyloidosis?mentioning

confidence: 99%

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Light-Chain Amyloidosis: The Great Impostor

Stefani,

Kouvata,

Vassilopoulos

2023

Life

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Light-chain amyloidosis (AL) is a disease of protean manifestations due to a wide spectrum of organs that can be affected. The disorder is caused by the deposition of an extracellular amorphous material, the amyloid, which is produced by malignant plasma cells. The latter usually reside in the bone marrow; plasma cell infiltration is often low, in sharp contrast to what we observe in multiple myeloma. The disease may run below the physician’s radar for a while before clinical suspicion is raised and targeted tests are performed. In this short review, we try to answer most of the questions that a practicing physician may ask in a relative clinical setting. The text is formed as a series of reader-friendly questions that cover the subject of AL amyloidosis from history to current therapy.

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Section: Q6: What Is the Most Commonly Affected Organ In Al Amyloidosis?mentioning

confidence: 99%

“…• Myocardial PET/CT shows enhancement of the radiotracer (technetium) due to the amyloid binding; currently, it is only used as a research tool [16].…”

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confidence: 99%

Light-Chain Amyloidosis: The Great Impostor

Stefani,

Kouvata,

Vassilopoulos

2023

Life

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“…CA is subdivided based on the types of proteins being deposited within the myocardium. Though over 30 proteins have been identified to cause amyloidosis, the two most common subtypes are immunoglobulin light chain (AL) (Falk et al, 2016;Joshi et al, 2020;Palladini et al, 2020;Nasrullah et al, 2021) and transthyretin (TTR) (Lane et al, 2019;Hanna et al, 2020;Kittleson et al, 2020). Here, we focus on the latter, detailing the underlying pathogenesis of the disease, current therapeutic regimens, novel research efforts, and potential new strategies for the management of amyloid transthyretin cardiomyopathy (ATTR-CM).…”

Section: Introductionmentioning

confidence: 99%

Current and potential therapeutic strategies for transthyretin cardiac amyloidosis

Williams

Shankar

Vaishnav

et al. 2022

Front. Drug. Discov.

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Cardiac amyloidosis is a progressive disorder caused by the deposition of amyloid, abnormal proteins that aggregate to form insoluble plaques in the myocardium resulting in restrictive cardiomyopathy. The two most common subtypes of cardiac amyloidosis are immunoglobulin light chain (AL) and transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM). ATTR-CM can further be subdivided into two main categories, wild-type or hereditary TTR. TTR is a homotetrameric protein complex that is synthesized in the liver and is secreted into the circulation for retinol and vitamin A transfer. Genetic mutations in the TTR gene can disrupt the thermodynamic stability of the homotetrameric complex causing dissociation into monomers that, when taken up by the myocardium, will aggregate to form insoluble fibers. Though the mechanism of wild-type TTR is not fully elucidated, it is thought to be an age-related process. Myocardial uptake and aggregation of TTR monomeric subunits result in cytotoxicity, impaired cardiac function, and eventually heart failure. Historically, ATTR-CM had a poor prognosis, with no therapeutics available to specifically target ATTR-CM and treatment focused on managing symptoms and disease-related complications. In 2019, the FDA approved the first-in-class TTR stabilizer for ATTR-CM, which has led to improved outcomes. In recent years, several promising novel therapies have emerged which aim to target various points of the ATTR-CM amyloidogenic cascade. In this review, we discuss the mechanistic underpinnings of ATTR-CM, review current FDA-approved strategies for treatment, and highlight ongoing research efforts as potential therapeutic options in the future.

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AL type cardiac amyloidosis: a devastating fatal disease

Cited by 2 publications

References 16 publications

Light-Chain Amyloidosis: The Great Impostor

Light-Chain Amyloidosis: The Great Impostor

Current and potential therapeutic strategies for transthyretin cardiac amyloidosis

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