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Advances in the Diagnosis and Management of Transthyretin Amyloid Cardiomyopathy

  • Cardio-Oncology (M Fradley, Section Editor)
  • Published:
Current Treatment Options in Cardiovascular Medicine Aims and scope Submit manuscript

Abstract

Purpose of review

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a disease with high morbidity and mortality. This disease is significantly underdiagnosed and is more common than previously appreciated, particularly among older adults and people of African descent. This review discusses recent advances in the diagnosis and treatment for ATTR-CM.

Recent findings

Historically, ATTR-CM was diagnosed via endomyocardial biopsy, a resource-intensive and invasive approach. However, in most cases, ATTR-CM can now be diagnosed non-invasively using bone tracer cardiac scintigraphy, which may facilitate earlier diagnosis. In recent clinical trials, a transthyretin stabilizer (tafamidis) and transthyretin gene silencers (patisiran and inotersen) have emerged as effective ATTR amyloidosis therapies and have been approved for use in the USA and many other countries.

Summary

ATTR-CM is now recognized as an important cause of heart failure. Approaches to the diagnosis and treatment of ATTR-CM are rapidly evolving. Now, more than ever, there are opportunities to improve clinical care of patients with this challenging disease.

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References and Recommended Reading

Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

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Authors and Affiliations

  1. Department of Medicine, Stanford University School of Medicine, Stanford, CA, USA

    Gabriela Spencer-Bonilla MD, MSc

  2. Stanford Amyloid Center, Stanford University School of Medicine, 300 Pasteur Drive, Lane #158, Stanford, CA, 94305, USA

    Gabriela Spencer-Bonilla MD, MSc & Ronald M. Witteles MD

  3. Department of Medicine, Division of Cardiovascular Medicine, Stanford University School of Medicine, Stanford, CA, USA

    Kevin M. Alexander MD & Ronald M. Witteles MD

  4. Stanford Cardiovascular Institute, Stanford University School of Medicine, Stanford, CA, USA

    Kevin M. Alexander MD & Ronald M. Witteles MD

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  1. Gabriela Spencer-Bonilla MD, MSc
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Correspondence to Ronald M. Witteles MD.

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Dr. Spencer-Bonilla does not have any conflict of interest to disclose. Dr. Alexander has received an investigator-initiated research grant from Pfizer and has received consulting fees (modest) from Alnylam, Eidos, and Pfizer. Dr. Witteles has received consulting fees (modest) from Pfizer, Eidos, and Alnylam.

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Spencer-Bonilla, G., Alexander, K.M. & Witteles, R.M. Advances in the Diagnosis and Management of Transthyretin Amyloid Cardiomyopathy. Curr Treat Options Cardio Med 22, 45 (2020). https://doi.org/10.1007/s11936-020-00844-8

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