Treatment with tafamidis over nine years demonstrated sustained benefits in slowing the progression of transthyretin amyloid cardiomyopathy (ATTR-CM) in two older patients, according to a study published recently in JACC: Case Reports.
Both cases showed that long-term treatment could prevent hospitalization for heart failure while maintaining cardiac function and stabilizing biomarkers, highlighting the drug’s potential to manage the challenging disease.
What is ATTR-CM?
Transthyretin amyloidosis cardiomyopathy (ATTR-CM) is a rare progressive disease of the heart muscle that leads to congestive heart failure. It occurs when the transthyretin protein produced by the liver is unstable. Symptoms include fatigue; shortness of breath; irregular heart rate or palpitations; swelling of the legs, ankles and stomach; brain fog; wheezing; and dizziness. It often goes underdiagnosed because of a lack of awareness and knowledge of the disease. There is currently no cure for ATTR-CM.
Despite the progressive nature of ATTR-CM, treatment with tafamidis was associated with minimal adverse effects and no cardiac-related deaths during the observation period, the study said.
Read more about ATTR-CM therapies
“Tafamidis treatment was continued for 9 years in both of the cases presented here of wild-type ATTR-CM,” the study authors said. “Long-term tafamidis treatment is safe and helps prevent disease progression in patients with wild-type ATTR-CM.”
The first patient, an 82-year-old man diagnosed with wild-type ATTR-CM, began taking 80 mg of tafamidis after participating in the ATTR-ACT study. Over nine years, he experienced no hospitalizations due to heart failure, even after receiving a pacemaker for sick sinus syndrome.
Some cardiac function measures, such as left ventricular ejection fraction, gradually declined, but biomarkers such as high-sensitivity troponin T and brain natriuretic peptide remained stable. Echocardiography revealed no significant worsening of left ventricular hypertrophy or systolic function, suggesting that tafamidis effectively slowed disease progression.
Similarly, an 80-year-old male patient started tafamidis therapy after a diagnosis of wild-type ATTR-CM. Despite initial symptoms of heart failure and a history of bronchial asthma, he also avoided hospitalizations due to heart failure during the nine-year treatment period.
The study observed mild increases in left ventricular wall thickness and declines in strain measures, but the patient’s brain natriuretic peptide and high-sensitivity troponin T levels did not worsen significantly. Cardiac imaging confirmed minimal changes in left ventricular function over time, further supporting the drug’s stabilizing effects.
Both cases revealed that tafamidis could be administered safely for nearly a decade with no severe side effects reported. The result aligns with prior research demonstrating tafamidis’s efficacy in reducing cardiac amyloid deposition. Importantly, neither patient required intensive diuretic therapy, emphasizing the drug’s role in maintaining a stable clinical status.
The results are promising but are limited by the small sample size and lack of a control group. Larger studies are needed to confirm the broader applicability of the results. But the cases underscored tafamidis’s potential value in delaying the progression of ATTR-CM and improving long-term outcomes for patients.
