Navigating Therapeutic Pathways and Transforming Care in ATTR-CM - Episode 7
Clinical Implications of Rapid Transthyretin Knockdown in ATTR-CM
The panel discusses the concept of achieving a rapid reduction, or “knockdown,” of transthyretin (TTR) production in ATTR-CM. They note that the rationale is intuitive: slowing or halting new amyloid formation as early as possible may help interrupt disease progression. The comparison is made to AL amyloidosis, where shutting down the source of the abnormal protein is central to treatment. However, the panel emphasizes that ATTR-CM develops gradually over many years, and as a result, even when TTR production is reduced, clinical improvement is typically gradual rather than immediate. The experts highlight the importance of setting realistic patient expectations—treatment reduces ongoing injury but does not rapidly remove existing amyloid deposits. They also discuss the need to understand which symptoms are truly amyloid-related versus coincidental, and they stress the long-term, supportive relationships clinicians build with patients and families, particularly when helping individuals adjust goals and maintain quality of life over time.
The panel discusses the concept of achieving a rapid reduction, or “knockdown,” of transthyretin (TTR) production in ATTR-CM. They note that the rationale is intuitive: slowing or halting new amyloid formation as early as possible may help interrupt disease progression. The comparison is made to AL amyloidosis, where shutting down the source of the abnormal protein is central to treatment. However, the panel emphasizes that ATTR-CM develops gradually over many years, and as a result, even when TTR production is reduced, clinical improvement is typically gradual rather than immediate.
The experts highlight the importance of setting realistic patient expectations—treatment reduces ongoing injury but does not rapidly remove existing amyloid deposits. They also discuss the need to understand which symptoms are truly amyloid-related versus coincidental, and they stress the long-term, supportive relationships clinicians build with patients and families, particularly when helping individuals adjust goals and maintain quality of life over time.
