Author + information
- Received November 1, 2018
- Revision received February 16, 2019
- Accepted February 19, 2019
- Published online June 24, 2019.
- aDivision of Cardiology, Cardio-Oncology Center of Excellence, Washington University in St. Louis School of Medicine, Saint Louis, Missouri
- bDivision of Oncology, Washington University in St. Louis School of Medicine, Saint Louis, Missouri
- ↵∗Address for correspondence:
Dr. Kathleen W. Zhang, Division of Cardiology, Washington University in St. Louis School of Medicine, Campus Box 8086, 660 South Euclid Avenue, St. Louis, Missouri 63110-1093.
• Cardiac amyloidosis has high associated morbidity and, until recently, limited treatment options.
• This review discusses the mechanism and clinical trial performance of multiple emerging therapies.
• Additional studies should identify optimal treatment paradigms and biomarker strategies for cardiac response to therapy.
Cardiac amyloidosis is a restrictive cardiomyopathy that results from the deposition of misfolded light chain or transthyretin proteins, most commonly, in cardiac tissue. Traditionally, treatment options for light chain (AL) and transthyretin (ATTR) amyloidosis have been limited. However, there are now multiple novel therapeutics in development and several therapeutics recently approved that promise to revolutionize clinical management of AL and ATTR. Most of these agents disrupt specific stages of amyloidogenesis such as light chain or transthyretin protein production, formation of amyloidogenic intermediates, or amyloid fibril aggregation. Others aim to remove existing amyloid tissue deposits using monoclonal antibody technology. Although these advances represent an important step forward in the care of cardiac amyloidosis patients, additional studies are needed to define the optimal treatment paradigms for AL and ATTR and to validate clinical, imaging, or serum biomarker strategies that may confirm a cardiac response to therapy.
Dr. Stockerl-Goldstein is on the advisory board for Celgene; has stock equity in Abbott and AbbVie; and has received grants from Millenium Pharmaceuticals, Janssen Pharmaceuticals, BioLineRx Ltd., Pfizer Inc., and GlaxoSmithKline. Dr. Lenihan has received personal fees from Pfizer, Prothena, Akcea, and Takeda. Dr. Zhang has reported that she has no relationships relevant to the contents of this paper to disclose.
All authors attest they are in compliance with human studies committees and animal welfare regulations of the authors’ institutions and U.S. Food and Drug Administration guidelines, including patient consent where appropriate. For more information, visit the JACC: Basic to Translational Science author instructions page.
- Received November 1, 2018.
- Revision received February 16, 2019.
- Accepted February 19, 2019.
- 2019 The Authors