New ‘cutting edge’ hemophilia treatment is a victory for patients, Tulane doctor says

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A new treatment that helps hemophilia A patients maintain high levels of important blood clotting factors with less treatment is a win for patients, according to a new editorial in . New England Journal of Medicine This week by Dr. Cindy Reissinger, director of the Louisiana Center for Bleeding and Coagulation Disorders at Tulane University School of Medicine.

An estimated 20,000 people in the United States have hemophilia A, including about 300 in Louisiana. People with hemophilia A lack clotting factor VIII in their blood, which can lead to painful, sometimes life- and limb-threatening bleeding.

The Food and Drug Administration is fast-tracking a potential new treatment, efanesoctocog alfa, in 2021, and researchers are journal this week. The study shows that this therapy helps the patient maintain high levels of clotting factor VIII with just her one weekly infusion. Patients currently require two to three injections per week using existing therapies that do not prevent all bleeding.

Efanesoctocog alfa offers better protection against bleeding with a more convenient dosing schedule for patients. Most people with hemophilia self-inject themselves with intravenous clotting factors, so reducing the injections from 3 times a week to once a week can be of great help to the patient. “

Cindy Leissinger, Ph.D., Director, Louisiana Center for Bleeding and Coagulation Disorders, Tulane University School of Medicine

Although hemophilia is a rare disease, Leissinger writes in the journal that the aggressive nature of treatment and its cost impose a disproportionate burden of treatment on patients and society. In a crowded field of innovative therapies that have recently been approved, are under review, or are in late-stage clinical trials, efanesoctocog her alpha stands out as a winner, and Leissinger sees it in her Tulane. It may immediately make life easier for patients with

The Tulane center wasn’t part of the efanesoctacog alfa trial, but Leissinger says other new treatments are here, including gene therapy trials that could cure or “nearly cure” some people with hemophilia. It is said that it is being studied in

“Because hemophilia is a rare disease, this kind of progress is only made because most patients are willing to participate in research and volunteer to test new treatments,” Leisinger said. said.

“The future for people with hemophilia has never looked brighter, thanks to cutting-edge research and patients willing to participate in it.”


Journal reference:

Leissinger, C., (2023) Another victory for hemophiliacs. New England Journal of Medicine.

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