July 10 (UPI) -- Researchers at Children's Hospital Los Angeles developed a new therapy called emicizumab that decreases bleeding episodes by nearly 90 percent.
People with hemophilia A have excessive bleeding and need prophylactic treatment with intravenously administered clotting factor about three times a week. This weekly administration of clotting factor can cause the development of antibodies known as inhibitors that destroy the clotting factor and make treatment more challenging.
Emicizumab, a new monoclonal antibody, decreased the incidence of bleeding episodes in patients with hemophilia A with inhibitors and can be given subcutaneously once a week.
"This is a breakthrough for individuals with hemophilia A who no longer respond to conventional clotting therapies," Dr. Alan S. Wayne, director of the Children's Center for Cancer and Blood Diseases, said in a press release.
"The standard treatment for patients with hemophilia A and inhibitors has been to use medications called 'by-passing agents.' This new therapy is dramatically more effective at preventing bleeding. Additionally, in comparison to by-passing agents, emicizumab is easier to administer, requires less frequent dosing, and based on this study, appears to have an improved safety profile."
The study, which was published Monday in the New England Journal of Medicine, consisted of 109 males over the age of 12 with hemophilia A with inhibitors.
Participants who received emicizumab had 87 percent fewer bleeding episodes compared to untreated patients, and 79 percent fewer bleeding episodes compared to patients treated with bypassing agents.
"This is the most significant advancement I have seen during my 20 years working in the field of hemophilia," said Dr. Guy Young, who is also a professor of pediatrics at the Keck School of Medicine of University of Southern California and a hematologist at Children's Hospital Los Angeles. "We've had families flying in from all over the country to get access to this medication."