Hope for thousands of people with hemophilia because the new injection can significantly reduce the risk of serious bleeding
The monthly injection could change the lives of hemophilia patients after it was shown to significantly reduce bleeding.
Trials of the treatment, called Phytoceran, found that it stopped bleeding in up to two-thirds (66 percent) of patients with hemophilia A or B.
Experts say preventative treatment could soon improve patients’ daily lives by reducing the number of hospital trips.
Hemophilia is an often inherited condition that affects the blood’s ability to clot, putting patients at risk for severe bleeding.
It affects around 9,000 people, mostly men, in the UK, where patients are often treated with clotting factor drugs, to replenish what they have lost, or with retrograde drugs when they bleed.
A monthly injection could change the lives of hemophilia patients after it was found to significantly reduce bleeding (stock image)
But preventive medications must be injected regularly, usually every day for hemophilia A and 2-3 times a week for hemophilia B.
They can also become less effective over time with some people taking blood-clotting factor medication developing antibodies in their immune system, called inhibitors.
This uses a new type of treatment, called small interfering RNA (siRNA), which works by interfering with the production of certain proteins.
Phytoceran is the first siRNA compound developed for hemophilia and targets a protein that reduces blood clotting, called antithrombin, to increase clotting ability.
During the first trial, 25 out of 38 (66 percent) of the inhibitor-treated participants who received phytoceran injections had zero bleeding after nine months, compared to 1 in 19 (5 percent) who were given an on-demand bypass agent.
Research in patients without inhibitors found that 40 of 79 (51 percent) of those who received monthly vaccinations experienced no bleeding, compared with 2 of 40 (5 percent) in the placebo group, according to findings published in The Lancet Hematology.
However, the researchers said that potential side effects such as blood clotting and liver damage require further investigation.
Professor Jae Young, from the University of Southern California, who led the study, said: ‘The data are encouraging and suggest it may be the first-line preventive treatment – meaning it can be given to prevent bleeding rather than treat it after it has already occurred. – Works with hemophilia A and B patients with inhibitors.
Treatment options for patients with hemophilia B are currently limited to on-demand therapies, which treat bleeding after it has occurred.
Professor Alok Srivastava, Christian Medical College, Vellore, India, and co-author of the study, said: ‘With this drug given only once a month or even less, there is a significant reduction in the treatment burden.
This means hemophilia patients can manage their condition with fewer trips to the hospital, which can cause anxiety and get in the way of daily life. This will improve the 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 affected 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 makes the bleeding eventually stop.
People with hemophilia do not have as many clotting factors as they should in their blood. This means that they bleed longer than usual.
There are several different types of hemophilia. The following two types are the most common:
- Hemophilia A (classical hemophilia), caused by deficiency or deficiency of clotting factor VIII.
- Hemophilia B (Christmas disease), caused by a lack or deficiency of 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 modified clotting factor drugs are used to prevent and treat prolonged bleeding. These medicines are given by injection.
Hemophilia occurs in about 1 in 5,000 male births, according to the CDC. Currently, about 20,000 people in the United States and 400,000 worldwide live with this disorder.
In the UK, figures show that there are 2,000 affected by the condition.