Imaging data show approved ATTR-CM therapy improves heart structure
New analysis reviewed 13 studies involving over 1K participants
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Treatment with Vyndamax (tafamidis) improves certain features of the heart’s structure and function in adults with transthyretin amyloid cardiomyopathy (ATTR-CM), according to an analysis of 13 imaging studies that involved more than 1,000 participants.
The Australian research team, which noted that the drug’s “clinical efficacy is established,” examined these studies to investigate whether Vyndamax use affects heart function or structure. The scientists concluded that several measures “are consistently responsive to treatment.”
Specifically, in their analysis, the researchers found that use of the approved ATTR-CM therapy was linked to certain “significant” improvements in the structure of the heart’s upper left chamber and performance in its lower right chamber. Meanwhile, traditional markers of the lower left heart chamber’s structure showed little change with Vydamax treatment, the team noted.
“Imaging parameters … may serve as sensitive [measures] of therapeutic response in future clinical trials, offering mechanistic insights beyond conventional [lower left chamber] metrics,” the researchers wrote, noting that “this analysis is, to our knowledge, the first to comprehensively synthesise multimodal imaging data.”
The findings were described in the study “Differential Effects of Tafamidis on Cardiac Remodelling in Transthyretin Amyloid Cardiomyopathy: Evidence From Contemporary Imaging Data,” which was published in the European Journal of Clinical Investigation. Vyndamax is sold in the European Union under the brand name Vyndaqel.
ATTR-CM occurs when clumps of misfolded transthyretin protein, called amyloid deposits, build up in the heart muscle. The condition can be hereditary, caused by TTR gene mutations, or wild-type, which is thought to occur due to aging-related processes. Without treatment, the disease tends to progress steadily, eventually leading to heart failure and other related complications.
Reviewed covered wide range of data spanning 25 years
Vyndamax, taken as a once-daily oral capsule, is approved to reduce hospitalization and death related to cardiovascular disease in adults with either type of ATTR-CM. It works by stabilizing transthyretin, which helps prevent the formation of new amyloid deposits. While clinical trials have already shown that the therapy reduces deaths and hospitalizations related to heart failure, its specific effects on the heart’s structure and function have not been fully described.
To help fill this knowledge gap, the team systematically reviewed studies published from 2000 to 2025 that reported cardiac (heart) imaging data from ATTR-CM patients treated or not with Vyndamax. The control group included patients who received a placebo or no treatment, as well as those given standard of care without Vyndamax.
Altogether, 13 studies covering 1,014 adults with ATTR-CM were used in the meta-analysis, which combines and pools data from several independent studies to provide a single summary estimate of an effect or outcome. About half (53%) of the studies involved both hereditary and wild-type ATTR-CM patients.
Data from three noninvasive cardiac imaging methods were available. Transthoracic echocardiography is a noninvasive ultrasound test that creates moving pictures of the heart’s structures and allows the assessment of its function.
Cardiac MRI also evaluates the heart’s structure and function, allowing the indirect assessment of amyloid deposits in the heart. Bone-tracer cardiac scintigraphy detects the buildup of amyloid deposits in the heart.
Pooled results showed that over a mean follow-up of 16 months, or nearly 1.5 years, Vyndamax treatment was associated with a significant improvement in left atrial reservoir strain (LASr), a measure of how well the heart’s left upper chamber (atrium) stretches as it fills with blood.
A related measure, left atrial volume index (LAVi), which assesses the size of the left atrium adjusted for body size, showed a modest but statistically significant improvement in Vyndamax-treated patients relative to controls. Both LASr and LAVi were assessed with transthoracic echocardiography.
On cardiac MRI, right ventricular ejection fraction (RVEF), which reflects the pumping strength of the heart’s right lower chamber (ventricle), also improved significantly in patients treated with Vyndamax.
The researchers noted that the right ventricle’s thinner wall and greater sensitivity to changes in physical pressure may allow it to show functional benefit earlier than other heart structures once disease progression slows.
Vyndamax found to have ‘selective and measurable effects’
In contrast, the analysis showed no significant effects for several standard measures of the heart’s left ventricle. These included the amount of blood pumped with each heartbeat, how much the heart muscle shortens lengthwise when it squeezes, and wall thickness.
The researchers suggested this was not a surprise given the study’s length: “Compared to the thin-walled [right ventricle], the [left ventricle] is a thicker, more heavily infiltrated chamber in many patients with ATTR-CM, such that true regression of amyloid burden and wall thickness is biologically less likely over short to intermediate time frames,” the team wrote.
Available data from two bone-tracer cardiac scintigraphy studies showed that Vyndamax-treated patients had fewer amyloid deposits in the heart muscle relative to controls, but this difference did not reach statistical significance, meaning it could be due to chance.
In exploratory analyses, the team found no significant effect of strain-measurement software, ATTR–CM type, follow-up duration, or heart failure medications, on the observed imaging changes.
Vyndamax “was associated with selective and measurable effects on cardiac structure and function in patients with [ATTR-CM], with the most consistent improvements observed in markers of left atrial function and right ventricular performance,” the researchers wrote.
The team suggested that LASr, LAVi, and RVEF may serve as useful markers for tracking early treatment response in future research, while noting that further long-term imaging studies are needed to confirm these patterns and their relationship to clinical outcomes.
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