Hope for thousands with hemophilia as new injection may drastically reduce risk of dangerous bleeding
A monthly injection could be life-changing for hemophilia sufferers after it was found to significantly reduce bleeding.
Studies of a treatment called Fitusiran found it stopped bleeding in up to two-thirds (66 percent) of people with hemophilia A or B.
Experts say the preventative treatment could soon improve patients’ daily lives by reducing the number of hospital stays.
Hemophilia is a commonly inherited condition that affects the blood’s ability to clot and puts patients at risk of serious bleeding.
It affects around 9,000 people, mostly men, in the UK, with patients often being treated with clotting factor medicines to replicate what they are missing, or anti-reaction medicines if they bleed.
A monthly injection could change the lives of hemophilia sufferers after it was found to significantly reduce bleeding (stock image)
But the preventive drugs need to be injected regularly, usually every other day for hemophilia A and 2-3 times a week for hemophilia B.
They can also become less effective over time because some people taking blood clotting factor medications develop antibodies in their immune system called inhibitors.
It uses a new type of treatment called small interfering RNA (siRNA) that disrupts the production of certain proteins.
Fitusiran is the first siRNA developed for hemophilia, targeting a protein that reduces blood clotting called antithrombin to increase its ability to clot.
During the first study, 25 of 38 (66 percent) participants with inhibitors who received Fitusiran injections had no bleeding after nine months, compared with one of 19 (5 percent) who received an as-needed bypass agent.
Research on patients without inhibitors found that 40 out of 79 (51 percent) of those given the monthly vaccinations had no bleeding, compared with 2 out of 40 (5 percent) in the other group, according to results published in The Lancet Hematology .
However, possible side effects such as blood clotting and liver damage require further investigation, the researchers said.
Professor Guy Young of the University of Southern California, who led the study, said: “The data are encouraging and suggest this could be the first prophylactic treatment – meaning it can be given to prevent bleeding, rather than treating them after they’ve already happened – which works in both hemophilia A and hemophilia B patients with inhibitors.
Treatment options for hemophilia B patients are currently limited to on-demand treatments that treat bleeding after it occurs.’
Professor Alok Srivastava, Christian Medical College, Vellore, India, and study co-author, said: “If this drug is administered once a month or even less frequently, the treatment burden is significantly reduced.
“This means patients with hemophilia can manage their condition with fewer hospital stays, which can lead to worry and disruption to daily life. This would lead to an improved quality of life.”
WHAT IS HEMOPHILIA?
Hemophilia is a rare condition that affects the blood’s ability to clot. It is usually inherited, and most people who have it are male.
Normally, when you cut yourself, substances in the blood known as clotting factors combine with blood cells called platelets to make the blood sticky. This eventually stops the bleeding.
People with hemophilia don’t have as many clotting factors in their blood as they should. This means they bleed longer than usual.
There are different types of hemophilia. The following two are the most common:
- Hemophilia A (classic hemophilia) caused by a deficiency or decrease in coagulation factor VIII.
- Hemophilia B (Christmas sickness) caused by a deficiency or decrease in clotting factor IX.
There is no cure for hemophilia, but treatment usually allows a person with the condition to enjoy a good quality of life.
Genetically engineered clotting factor drugs are used to prevent and treat prolonged bleeding. These medicines are given as an injection.
Hemophilia occurs in about one in every 5,000 male births, according to the CDC. About 20,000 people in the US and 400,000 worldwide are currently living with this disorder.
In the UK, figures suggest there are 2,000 affected.
https://www.dailymail.co.uk/health/article-11919195/Hope-thousands-haemophilia-form-new-injection.html?ns_mchannel=rss&ns_campaign=1490&ito=1490 Hope for thousands with hemophilia in the form of a new injection