The discovery that a monthly injection substantially reduces bleeding in hemophilia patients has the potential to transform their lives.
Approximately two-thirds (66%) of patients with hemophilia A or B did not hemorrhage during clinical trials of the fitusiran treatment.
According to experts, preventative treatment could soon improve patients’ daily routines by reducing hospital visits.
Haemophilia is a frequently inherited disorder that impairs the blood’s ability to coagulate, putting patients at risk for excessive bleeding.
It affects approximately 9,000 individuals, predominantly men, in the United Kingdom. Patients are frequently treated with clotting factor medicines, to replace what is missing, or with bleeding-control medications.
However, haemophilia A and B require preventative medicines every other day and two to three times per week, respectively.
Inhibitors, immune system antibodies, reduce the efficacy of blood clotting factor medicines in some people.
Small interfering RNA (siRNA) is a novel treatment that functions by interfering with the production of specified proteins.
Fitusiran is the first siRNA developed for hemophilia. It targets antithrombin, a protein that inhibits blood coagulation, to improve clotting ability.
In the first study, 25 out of 38 (66%) participants with inhibitors who received fitusiran injections had no hemorrhage after nine months, compared to only one out of 19 (5%) who were given an on-demand bypassing agent.
According to the findings published in The Lancet Haematology, 51% of the 79 patients without inhibitors who received the monthly injections did not experience any bleeding, compared to 2% of the 40 patients in the control group.
However, potential adverse effects, such as blood clotting and liver injury, require further study, according to the researchers.
Professor Guy Young of the University of Southern California, who led the study, stated: ‘The data is encouraging and suggests it may be the first prophylactic treatment – meaning it can be administered to prevent bleeds as opposed to treating them after they have already occurred – that is effective for both hemophilia A and B patients with inhibitors.
Current treatment options for hemophilia B patients are limited to on-demand treatments, which treat bleeding after it has occurred.
Professor Alok Srivastava, of the Christian Medical College in Vellore, India, and co-author of the study, remarked, “By administering this medication once a month or even less frequently, the treatment burden is significantly reduced.”
Hemophilia patients will have fewer hospital trips, which can be stressful and disruptive to their daily lives. This would result in an improvement in life quality.
