An innovative treatment against hemophilia, developed by the French giant Sanofi, shows high effectiveness against the symptoms of this disease, according to a study published in the scientific journal The Lancet, even if its use still raises questions in view of the risk of severe side effects, AFP reports on Thursday, quoted by Agerpres.ro.
“This therapy could have the potential to improve the monitoring of hemophilia patients,” summarized the authors of the study, which evaluates the therapeutic interest of Fitusiran.
This molecule, for which Sanofi has held the rights since 2018, is one of the main innovative ways to treat hemophilia, a genetic disease that mainly affects men and results in excessive bleeding caused by a clotting problem.
To prevent bleeding, hemophiliacs are generally treated prophylactically by directly administering the clotting proteins they lack due to a genetic defect.
However, a significant number of patients develop “inhibitory” antibodies against these proteins, because of which they cannot be given preventive treatments. Therefore, for them there is no other option than to be treated punctually, during the bleeding episodes.
For several years, research has focused on another therapeutic path: preventive treatments that, instead of directly replacing the clotting proteins, aim to achieve the same result by reducing the anticoagulant action of certain cells.
This is the case of Sanofi’s Fitusiran as well as another molecule, developed by Roche, Emicizumab, already approved in the United States and the European Union (EU) against the main form of hemophilia, known as type A.
Another form of hemophilia, type B, however, does not yet have a preventive treatment if patients have developed inhibitory antibodies.
According to the study published in the Lancet, performed on forty patients, compared to twenty others who did not receive any preventive treatment, Fitusiran drastically reduced bleeding episodes in both types of hemophilia.
Therefore, this treatment has the potential to become a revolutionary solution, but it still has “many unknowns”, the independent researchers who led the study, among them the hematology specialist Flora Peyvandi, emphasized in the same issue of the Lancet.
These “unknowns” mainly concern the side effects of Fitusiran. Of the forty patients treated, approximately ten had an abnormal concentration of an enzyme characteristic of serious liver problems.
Also, two patients had thromboembolic episodes, that is, the formation of clots that prevent blood circulation. “The potential risk of thrombosis (…) needs to be clarified”, warned Peyvandi and his colleagues.
source: Agerpres.ro
