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Targeted treatments of AL and ATTR amyloidosis

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Abstract

The therapeutic landscape for cardiac amyloidosis is rapidly evolving. In the last decade, our focus has shifted from dealing with the inevitable complications of continued extracellular infiltration of amyloid fibrils to earlier identification of these patients with prompt initiation of targeted therapy to prevent further deposition. Although much of the focus on novel targeted therapies is within the realm of transthyretin amyloidosis, light chain amyloidosis has benefited due to an overlap particularly in the final common pathway of fibrillogenesis and extraction of amyloid fibrils from the heart. Here, we review the targeted therapeutics for transthyretin and light chain amyloidosis. For transthyretin amyloidosis, the list of current and future therapeutics continues to evolve; and therefore, it is crucial to become familiar with the underlying mechanistic pathways of the disease. Although targeted therapeutic choices in AL amyloidosis are largely driven by the hematology team, the cardiac adverse effect profiles of these therapies, particularly in those with advanced amyloidosis, provide an opportunity for early recognition to prevent decompensation and can help inform recommendations regarding therapy changes when required.

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Authors and Affiliations

  1. Amyloidosis Center, Knight Cardiovascular Institute, Oregon Health & Science University, Portland, OR, USA

    Pranav Chandrashekar

  2. Department of Medicine, Oregon Health & Science University, Portland, OR, USA

    Anish K. Desai

  3. Methodist DeBakey Heart and Vascular Center, Houston, TX, USA

    Barry H. Trachtenberg

  4. Cardio-Oncology and Cardiac Amyloidosis Program, Advanced Heart Failure Fellowship Program, Methodist DeBakey Heart and Vascular Centers, J.C. Walter Transplant Center, Houston, USA

    Barry H. Trachtenberg

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  1. Pranav Chandrashekar
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  3. Barry H. Trachtenberg
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Correspondence to Barry H. Trachtenberg.

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Pranav Chandrashekar is supported by a nationally competitive educational grant from Pfizer paid directly to OHSU. Barry Trachtenberg is a speaker for Pfizer, Akcea, and Alnylam.

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Chandrashekar, P., Desai, A.K. & Trachtenberg, B.H. Targeted treatments of AL and ATTR amyloidosis. Heart Fail Rev 27, 1587–1603 (2022). https://doi.org/10.1007/s10741-021-10180-z

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