RX Review: Understanding ATTR-CM's New Era of Management - Episode null
RX Review: Understanding ATTR-CM's New Era of Management
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In this video, part 1 of a 4-part series, panelists discuss recent advancements in ATTR-CM management.
Transthyretin amyloid cardiomyopathy (ATTR-CM), once considered a rare disease, is now recognized as a more prevalent and underdiagnosed cause of cardiomyopathy, heart failure, and increased hospitalization. Advances in diagnostic strategies, including non-biopsy methods, as well as pharmacological breakthroughs, have contributed to earlier detection and a shift in disease management.
In the first of this 4-part RX Review series celebrating advancement in ATTR-CM, our panel of experts introduces the topic and dives into how these advances have influenced the unmet need within the disease state. After opening the conversation, Mazen Hanna, MD, director of the Heart Failure Intensive Care Unit and codirector of the Amyloid Program at Cleveland Clinic, turns to Marian Vandyck-Acquah, MD, director of Education and Quality-Noninvasive Cardiology at Hackensack Meridian Health Heart and Vascular Hospital, who notes that ATTR-CM is no longer “hidden in plain sight” but is increasingly identified due to heightened clinical awareness and the availability of targeted therapies.
Later, Jesus Almendral, MD, director of the Advanced Heart Failure Center at the Jersey Shore University Medical Center, highlights the growing recognition of ATTR-CM in clinical practice, with seemingly unrelated symptoms—such as carpal tunnel syndrome, spinal stenosis, and polyneuropathy—now understood as potential indicators of systemic disease. He attributes this shift to increased awareness from professional organizations, updated screening guidelines, and direct-to-consumer campaigns that prompt patients to seek evaluation. Hanna emphasizes the ongoing need for improved detection, citing data indicating that approximately 10% of older patients with heart failure with preserved ejection fraction and increased wall thickness may have ATTR-CM. Additionally, a notable proportion of elderly patients undergoing transcatheter aortic valve replacement for severe aortic stenosis have been diagnosed with ATTR-CM, further underscoring the importance of early identification.
Our Panelists:
Mazen Hanna, MD, is a cardiologist at the Cleveland Clinic's Vascular & Thoracic Institute. Hanna is also director of the Heart Failure Intensive Care Unit and codirector of the Amyloid Program at Cleveland Clinic and serves as the moderator for this panel discussion.
Marian Vandyck-Acquah, MD, is an assistant professor of medicine in the cardiology department at the Hackensack Meridian School of Medicine. Vandyck-Acquah is also the director of Education and Quality-Noninvasive Cardiology at Hackensack Meridian Health Heart and Vascular Hospital.
Jesus Almendral, MD, is a cardiovascular disease specialist board-certified in advanced heart failure and heart transplant, cardiovascular disease and internal medicine at the Jersey Shore University Medical Center. He is the director of the Advanced Heart Failure Center at the center and serves as the third panelist for our discussion.
Relevant disclosures for Hanna include Pfizer, Alnylam, Ionis, and Eidos. Almendral and Vandyck-Acquah have no relevant disclosures to report.
References:
Office of the Commissioner. FDA approves new treatments for heart disease caused by a serious rare disease, transthyretin mediated amyloidosis. FDA. Published May 6, 2019. Accessed March 28, 2025. https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatments-heart-disease-caused-serious-rare-disease-transthyretin-mediated.
BridgeBio. AttrubyTM (acoramidis), a Near Complete TTR Stabilizer (≥90%), approved by FDA to Reduce Cardiovascular Death and Cardiovascular-related Hospitalization in ATTR-CM Patients - BridgeBio. BridgeBio. Published November 22, 2024. Accessed March 28, 2025. https://bridgebio.com/news/attruby-acoramidis-a-near-complete-ttr-stabilizer-%E2%89%A590-approved-by-fda-to-reduce-cardiovascular-death-and-cardiovascular-related-hospitalization-in-attr-cm-patients/.
Alnylam. Alnylam Announces FDA Approval of AMVUTTRA® (vutrisiran), the First RNAi Therapeutic to Reduce Cardiovascular. Investor Relations | Alnylam Pharmaceuticals, Inc. Published March 20, 2025. Accessed March 28, 2025. https://investors.alnylam.com/press-release?id=28831.
