Novel Potential Treatment for Hemophilia A

Highlights:

Treatments for hemophilia A have advanced more than ever in the recent decade
A new efanesoctocog alpha injection works by detaching factor VIII from its related protein carrier, known as the von Willebrand Factor, and therefore altering its 'half-life'. This means that people with hemophilia A only need injections once a week rather than three times a week
Patients with hemophilia A can now live the lives they want because they feel safe and protected, and many of them tell me they no longer have the everyday joint discomfort they used to have. They may not be cured, but this treatment is a close second

A potential new medication that could be a game changer for those suffering from hemophilia A, a potentially fatal genetic blood illness, was tested by researchers from McMaster University.

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‘Once-a-week injections of efanesoctocog alfa can reduce bleeding and promote the near-normal activity of the clotting factor VIII, both of which are critical for ending the chronic bleeding caused by hemophilia A. Individuals who used efanesoctocog alfa also had a greater joint function, overall wellness, and reduced pain.’

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Effect of Efanesoctocog Alfa Injections in Hemophilia

Once-weekly injections of efanesoctocog alfa, according to co-principal investigator Davide Matino, can reduce bleeding and encourage near-normal activity by the clotting agent factor VIII, which is critical for preventing the persistent bleeding caused by hemophilia A. Individuals who used efanesoctocog alfa also had a greater joint function, overall wellness, and reduced pain.

Matino explained that efanesoctocog alpha operates by detaching factor VIII from its accompanying protein carrier, known as the von Willebrand Factor, hence overriding its 'half-life'. This means that people with hemophilia A only need injections once a week rather than three times a week.

The findings were published in The New England Journal of Medicine.

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Health Benefits of Efanesoctocog Alfa Injections

“With this treatment, patients with hemophilia A are free to live the lives that they want as they feel safe and protected and many of them tell me they don’t feel the daily joint pain they used to have. They may not be cured, but this treatment is the next best thing,” said Matino, an assistant professor of the Department of Medicine.

“With only one injection for an entire week, people with hemophilia A will enjoy normal or near-normal levels of factor VIII, which is unheard of. Previously, patients had to inject every other day, which was a huge burden for them, yet obtaining a lower level of protection” he said.

“The treatments for hemophilia A have improved more in the last decade than ever before. Now we have gene therapies and editing in the pipeline, monoclonal antibody treatments and cell-based therapies.”

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Efanesoctocog Alfa Injections Improve Quality of Life of Hemophilia Patients

The experiment, which finished in 2021, discovered that weekly treatment for 133 hemophilia patients for a year improved their results and quality of life compared to their previous treatment.

According to Matino and fellow McMaster study author Alfonso Iorio, McMaster is the only clinic in Canada enrolling adult patients.

“The research infrastructure and leadership we built at McMaster as a leader for the Canadian hemophilia data collection network allowed us to attract international clinical studies on innovative treatments, such as the efanesoctocog alfa trial and gene therapy,” said Alfonso Iorio, professor and director of McMaster’s hemophilia clinic.

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What is Hemophilia?

Hemophilia A is a rare bleeding disorder caused by a mutation in the gene that codes for factor VIII, which essentially prevents clotting activity.

Beginning in early childhood, the disorder causes repeated bleeding into joints and can be fatal. When a person's factor VIII level is less than 1% of normal, they have severe hemophilia A.

“The patients we enrolled in this study have been using efanesoctocog alfa for several years and we are delighted that McMaster managed to offer them this treatment and thus the opportunity to enjoy a vastly better quality of life,” said Iorio.

Source-Medindia