An interview with Prof. Jack Grzybowski, head of the Cardiomyopathy Unit at the National Institute of Cardiology.
Prof. Jacek Grzybowski: Newly diagnosed patients with transthyretin amyloidosis await drug program
Published March 11, 2024 12:55
What is the situation now for patients with transthyretin amyloidosis?
Patients with transthyretin amyloidosis of the heart have recently had the potential for effective treatment, but also the possibility of effective, non-invasive diagnostic imaging. The problems now are two. First: awareness of the disease is low, because the disease is also very rare. It is important to spread awareness of it. This diagnosis is relatively easy to make in reference centers.
What happens if cardiomyopathy is not treated or not treated properly?
A huge breakthrough in this disease is the possibility of specific treatment. With causal treatment, we can stop the progression of the disease and the development of heart failure. If patients are not treated with specific treatment, heart failure progresses quite rapidly and roughly 50 percent of patients die within the first two to three years after diagnosis. The causal treatment is a new drug, registered in the European Union in 2020. Tafamidis is a transthyretin stabilizer. It inhibits its breakdown and amyloid formation. An alternative for untreated patients is simultaneous liver and heart transplantation. This is a huge procedure and not all patients can be qualified for it.
How is access to causal treatment in Poland?
Treatment with tafamidis is currently available in various reimbursement models in most European countries. In Poland, we are waiting for reimbursement. For the past five years, until last fall, patients were treated under a charity program funded by the manufacturer. Those patients who were treated continue to receive the drug. New patients are waiting for the drug program.
What is the experience of treating patients?
It is very important that we have our own experience of treating almost a hundred patients. They are very good. The treatment slows down the course of the disease, slows down the development of heart failure, prevents numerous exacerbation-related hospitalizations, and is important for patients because it saves their lives. It also allows to generate system savings.
