In the last decade, better awareness among healthcare providers, improved diagnostic tools and new treatments have helped doctors diagnose transthyretin amyloid cardiomyopathy (ATTR-CM) earlier and start treatment sooner, according to a review published recently in Circulation: Heart Failure.
Due to earlier detection, patients now often have milder disease at diagnosis, which can mean slower disease progression and improved overall health upon treatment.
Patients participating in clinical trials also have milder disease compared to the past. This makes it harder for clinicians to measure the difference between those getting treatment and those on placebo, since patients in the placebo group are healthier and experience slower disease progression.
“This evolution in the patient population poses a new challenge for clinicians when assessing treatment responses because the differences observed between placebo and active treatment will be smaller, given the improved prognosis of the untreated patients,” the authors said.
Read more about treatment and care for ATTR-CM
A closer look at four clinical trials for ATTR-CM conducted five to six years apart proves that patients are being diagnosed and treated earlier. In the 2018 ATTR-ACT study, patients tended to have more advanced disease and their condition worsened faster compared to those in more recent trials like the 2023 APOLLO-B study, the 2024 ATTRibute-CM study and the 2024 HELIOS-B study.
While treatments have advanced, the authors said there is still room for improvement. New drugs that act in different ways and treat the disease from different angles are needed.
Doctors need a better understanding of how to use different treatments together, how to choose the best one for each person and when it’s time to try something else if the first treatment doesn’t work as well as hoped. Currently, doctors don’t have a clear-cut way of assessing whether a treatment is working or not, and more research is needed to guide these decisions.
Another unmet need in ATTR-CM management is the lack of information from the patients’ perspective. “Understanding what matters most to patients in terms of their disease symptomatology and management will also help to inform clinical decision-making,” the authors said.
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