RX Review: Understanding ATTR-CM's New Era of Management - Episode null
RX Review: Future Directions, Changing Landscape of ATTR-CM
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In this video, the last in a 4-part series, panelists discuss how ongoing research and efforts could shape the future of ATTR-CM management.
With such a rapid rate of advancement for diagnostic and treatment capabilities within the management of transthyretin amyloid cardiomyopathy (ATTR-CM), many are playing catch-up with the sheer amount of information. However, many are also already looking to the future for what’s next for the field.
In the fourth and final part of our RX Review on updates and unmet needs in ATTR-CM, Mazen Hanna, MD, director of the Heart Failure Intensive Care Unit and codirector of the Amyloid Program at Cleveland Clinic, leads a discussion with Marian Vandyck-Acquah, MD, director of Education and Quality-Noninvasive Cardiology at Hackensack Meridian Health Heart and Vascular Hospital, and Jesus Almendral, MD, director of the Advanced Heart Failure Center at the Jersey Shore University Medical Center, on what the ever-changing landscape could hold in the near future.
AI and Machine Learning in Diagnosis
Vandyck-Acquah highlights AI's potential in detecting ATTR cardiomyopathy (ATTR-CM) by analyzing EMRs for early signs like spinal stenosis and carpal tunnel syndrome. AI-driven imaging may also improve echocardiographic differentiation of hypertrophic cardiomyopathy (HCM) and amyloidosis. However, careful data interpretation is crucial.
Challenges in Managing Comorbid Patients
Almendral discusses treatment complexities in elderly patients with comorbidities like renal disease and diabetes. Since ATTR-CM therapies take months to show benefits, determining when to start treatment is challenging, with high costs and insurance coverage further impacting treatment choices.
Future Therapies: Gene Editing & Antibodies
Hanna and Vandyck-Acquah explore upcoming therapies, including gene editing (CRISPR-Cas9) and amyloid-targeting antibodies. These may enable earlier intervention, with treatments tailored to disease stage:
- Early-stage patients: Stabilizers or silencers.
- Mid-stage patients: Possible combination therapy.
- Advanced-stage patients: Antibody therapies for amyloid clearance.
Outlook and Key Considerations
The panelists conclude the series with a discussion on what unanswered questions remain about monitoring treatment response, genetic screening, and long-term follow-up for asymptomatic carriers. Cost remains a major barrier, and insurance coverage often dictates treatment choices. However, growing treatment options provide hope for earlier diagnosis and improved patient outcomes.
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.
