Without treatment, this disease kills. – We can now recognize transtithetin amyloidosis well, there are treatment centers in Poland. There is a drug that stops the progression of the disease and prolongs life. We are waiting for its introduction in Poland. I am an optimist, this treatment must be included in the drug program – says Prof. Przemysław Mitkowski, past president of the Polish Cardiological Society.
In cardiac amyloidosis – as this disease is sometimes called – amyloid fibers are deposited in the heart, the wall of which is overgrown, which prevents it from contracting and relaxing. The result is worsening heart failure.
However, typical drugs used in heart failure do not help and often even worsen the prognosis. – We have already learned to diagnose this disease very well and differentiate it from other cardiomyopathies. These patients have a very poor prognosis – the disease leads to death. It is preceded by a significant decrease in exercise tolerance and symptoms of heart failure. We cannot remove amyloid deposits from the heart. However, we can stop the progression of the disease – that is why early diagnosis and treatment are important – emphasizes Prof. Przemysław Mitkowski.
Treatment prevents amyloid from accumulating in the heart and inhibits the progression of the disease. There is a significant delay in the advanced stage of heart failure.
Experts call for a drug program
In Poland, several dozen patients are currently receiving treatment – as part of charity therapy, after completing a clinical trial. – The program has been operating for several years, but it was of a temporary nature. Of course, until reimbursement occurs, patients covered by this program will continue to receive treatment. However, the current situation of newly diagnosed patients is worrying. The treatment has been registered in Europe for almost four years, it’s high time to start refunding it in Poland. A reimbursement application has been submitted for the third time to create a drug program for this group of patients, notes Prof. Mitkowski.
– I am an optimist, this treatment must be implemented, a drug program must be created. We anticipate that it will cover a maximum of approximately 300 patients. It has been confirmed that the drug is effective and reduces mortality. It’s high time to offer this treatment to Polish patients – emphasizes Prof. Mitkowski.
Patients are waiting for medicines
One of the people receiving charity treatment is Zbigniew Pawłowski, president of the Association of Families with TTR Amyloidosis. Throughout his life, he was a very active person, a swimming coach, and a PE teacher; At some point, he noticed increasing fatigue, deterioration of physical fitness, and problems with walking. – When I was diagnosed with aTTR, I said goodbye to life; I heard that I have at most half a year left. I have been taking the treatment for four years now, I see that it is effective, I can work, I am a school principal. The test results show that the process of cardiac changes in the body has almost stopped, said Zbigniew Pawłowski.
This year, the guidelines of the European Society of Cardiology were published, covering cardiopathies. Recently, the opinion of experts from the Polish Society of Cardiology was published, in which the treatment is clearly recommended. There are centers and guidelines from the Polish Society of Cardiology have been created: there is only one thing missing: treatment. We fight for it because it is a treatment that saves our lives, says Zbigniew Pawłowski.