Hope for thousands with haemophilia as new injection can drastically slash risk of dangerous bleeds
A monthly injection could transform the life of haemophilia patients after it was found to significantly reduce bleeds.
Trials of the treatment, called fitusiran, found it stopped bleeds in up to two-thirds (66 per cent) of patients with haemophillia A or B.
Experts say the preventative treatment could soon improve patients’ daily lives by slashing the number of hospital trips.
Haemophilia is an often-inherited condition which affects the blood’s ability to clot, leaving patients at risk of heavy bleeding.
It affects around 9,000 people, mainly men, in the UK, with patients often treated with clotting factor medicines, to replicate what they are missing, or with reactionary medicines when they bleed.
A monthly injection could transform the life of haemophilia patients after it was found to significantly reduce bleeds (stock image)
But the preventative medicines need to be injected regularly, typically every other day for haemophilia A and 2-3 times a week for haemophilia B.
They can also become less effective over time with some people who take blood clotting factor medicine develop 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 specific proteins.
Fitusiran is the first siRNA developed for haemophilia and targets a protein that reduces blood clotting, called antithrombin, to increase clotting ability.
During the first trial, 25 out of 38 (66 per cent) participants with inhibitors who received fitusiran injections had zero bleeds after nine months, compared to one out of 19 (5 per cent) who were given an on-demand bypassing agent.
Research on patients without inhibitors found 40 out of 79 (51 per cent) of those given the monthly jabs experienced no bleeds, compared to 2 out of 40 (5 per cent) in the other group, according to the findings published in The Lancet Haematology.
However, potential 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 is encouraging and suggests it may be the first prophylactic treatment – meaning it can be given to prevent bleeds rather than to treat them after they have already occurred – that works for both haemophilia A and B patients with inhibitors.
‘Haemophilia B patients’ treatment options are currently limited to on-demand treatments, which treat bleeds after they have occurred.’
Professor Alok Srivastava, Christian Medical College, Vellore, India, and co-author of the study, said: ‘With this drug being administered just once a month or even less frequently, there is marked reduction of treatment burden.
‘This means patients with haemophilia could manage their condition with fewer trips to hospital, which can cause worry and be and disruptive to daily life. This would lead to an improved quality of life.’
WHAT IS HAEMOPHILIA?
Haemophilia is a rare condition that affects the blood’s ability to clot. It’s 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 makes the bleeding stop eventually.
People with haemophilia don’t have as many clotting factors as there should be in the blood. This means they bleed for longer than usual.
There are several different types of haemophilia. The following two are the most common:
- Haemophilia A (Classic Haemophilia), caused by a lack or decrease of clotting factor VIII.
- Haemophilia B (Christmas Disease), caused by a lack or decrease of clotting factor IX.
There’s no cure for haemophilia, but treatment usually allows a person with the condition to enjoy a good quality of life.
Genetically engineered clotting factor medicines are used to prevent and treat prolonged bleeding. These medicines are given as an injection.
Haemophilia occurs in about one of every 5,000 male births, according to the CDC. Currently, about 20,000 people in the US and 400,000 worldwide are living with the disorder.
In the UK, figures suggest there are 2,000 sufferers of the condition.