Extended Hemophilia Treatment Safe and Effective in Children

Combination of recombinant factor VIII with a fusion protein allows for less frequent treatment.

A long lasting novel anti-hemophilia therapy was found to be safe and effective for use in children in a recent study.

During treatment for hemophilia A, children receive 3 to 4 infusions per week to prevent bleeding episodes, chronic pain, and joint damage. The frequent injections can affect quality of life due to discomfort from the infusions.

As a result of the diminished quality of life, children are commonly under-dosed, which increases the risk for bleeding episodes and death. The study, published in the Journal of Thrombosis and Haemostasis, evaluated the extended half-life factor VIII, which allows patients to receive 1 or 2 infusions per week without an increased bleeding risk.

Hemophilia patients are typically treated with replacement therapy and receive infusions of the clotting factor that is deficient in their blood. These infusions keep the factor at a protective blood level that allows for clotting when an injury occurs.

Current treatment with factor VIII persists in the blood for a relatively short duration, which subsequently requires infusions every other day.

The extended therapy combines recombinant factor VIII with a fusion protein to allow the molecule to remain in circulation longer, which leads to less frequent treatment. The researchers found that twice-weekly infusions of the novel therapy were well tolerated and caused low incidence of bleeding events in children with severe hemophilia A.

It was also noted that no patients developed inhibitor antibodies against the factor.

"Through clinical trials, we are able to offer our patients the very latest treatments, often before they are available anywhere else,” said study lead Guy Young, MD, in a press release. “In this trial, we demonstrated the safety and efficacy of a medication that will allow children with severe hemophilia A to be treated with a safe and effective medicine but with a reduced burden of the treatment."