Hope for thousands with haemophilia in form of new injection

A monthly injection could change the lives of hemophiliacs after it was found to significantly reduce bleeding.

Trials of the treatment, called fitusiran, showed it stopped bleeding in up to two-thirds (66 percent) of patients with hemophilia A or B.

Experts say the preventive treatment can quickly improve patients’ daily lives by reducing hospital visits.

Hemophilia is an often inherited condition that affects the blood’s ability to clot, putting patients at risk of heavy bleeding.

It affects around 9,000 people, mostly men, in the UK, with patients often treated with clotting factor drugs, to replicate what they’re missing, or with reactionary drugs if they’re bleeding.

But the preventive drugs must 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, with some people taking blood clotting factor drugs developing antibodies in their immune system called inhibitors.

This uses a new type of treatment called small interfering RNA (siRNA) that works by disrupting the production of specific proteins.

Fitusiran is the first siRNA developed for hemophilia and targets a protein that reduces blood clotting called antithrombin to increase clotting ability.

In the first trial, 25 of 38 (66 percent) inhibitor participants who received fitusiran injections had zero bleeding after nine months, compared to one in 19 (5 percent) who received an on-demand bypassing agent.

A study of patients without inhibitors found that 40 of 79 (51 percent) of those who received the monthly shots experienced no bleeding, compared to 2 of 40 (5 percent) in the other group, according to findings 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 from the University of Southern California, who led the study, said: ‘The data are encouraging and suggest that this could be the first prophylactic treatment – meaning it could be given to prevent bleeding rather than stop it. treat after they have already occurred. – that works for both hemophilia A and B patients with inhibitors.

“Treatment options for hemophilia B patients are currently limited to on-demand treatments, which treat bleeding after it occurs.”

Professor Alok Srivastava, Christian Medical College, Vellore, India, and co-author of the study, said: ‘Because this drug is administered only once a month or even less often, there is a marked reduction in the treatment burden.

‘This means that patients with haemophilia can manage their condition with fewer hospital visits, which can be a concern and disrupt daily life. That would lead to a better quality of life.’