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Mortality significantly decreased in patients with Skelleftezjukan who received the drug drug tafamidis. Hospital facilities also declined and patients gained mobility and quality of life. It shows an international study in which patients from Västerbotten participated.
"There are significant differences that give hope to many with Skelleftezjukan," said Kurt Boman, senior professor at the University of Umeå and cardiologist at Skellefteå lasarett and one of those who led the Swedish section of the study.
The drug drug tafamidis has been used in Sweden for some years in some patients with nerve side effects. The new study means that it may be safer to say how effective the treatment is in patients with heart failure and amyloidosis, ie storage. of protein cells in the tissue, frequent complications at Skelleftesjukan. The drug does not cure the disease itself, but reduces the progression of damage to the heart.
In the study, 441 people from 13 countries participated. The study group treated with tafamidis had a mortality significantly lower than that of a group receiving effective preparations, called placebo. The absolute risk reduction was greater than 13%, which represents a significant reduction in these contexts. Hospitalization for cardiovascular disease has also significantly decreased.
After a 30-month check, the group that received the drug passed the test better than the control group. This shows that bodily functions have deteriorated more slowly than without this treatment. The results also showed that the treatment appeared to be equally effective in patients lacking, respectively, a particular genetic mutation associated with Skelleftesjukan. The side effects of the treatments were low, often less than in the placebo group.
"For this reason, we plan to continue studies on more patients with heart failure and amyloidosis," said Kurt Boman.
Skeletal disease, familial amyloidosis with polyneuropathy, is an inherited disease in which genetic engineering determines how the liver forms the mutated trasteidine protein, so that the protein exhibits an abnormal structure and particularly attacks the heart muscle and nerves. The disease can cause many life-threatening complications, but only 10% of carriers of the hereditary mutation develop the disease.
Earlier this year, another study showed that other drugs affect patients for whom Skelleftea disease attacks the nervous system, while the current study focuses more on patients for whom the disease has attacked the nervous system. heart.
In Sweden, about 350 people are affected by Skellefteå disease, mainly in the areas around Piteå and Skellefteå. The disease is present in several parts of the world, including Brazil and Japan. The drug tafamidis is sold in Sweden under the name Vyndagel.
The study is published in the scientific journal New England Journal of Medicine.
Towards the scientific article "Treatment of Tafamidis for patients with amyloid transthyretin cardiomyopathy"
For more information, please contact
Kurt Boman
Senior Professor
Department of Public Health and Clinical Medicine
University of Umeå
Phone: 0910-77 11 13
Mobile: 070 582 18 17
Email: [email protected]
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