Hemophilia: New Treatments for an Old Disease
Jan. 12, 2016 -- Although it’s been nearly a century since hemophilia was considered an early death sentence, treating it can be time-consuming and troublesome. Many people with the blood disease give themselves frequent drug infusions to keep their condition under control, sometimes as often as several times a week.
Several newly approved medications, while far from a cure, aim to lift some of that burden. Some will allow patients to go longer between treatments. Another drug in development aims to overcome a problem with current treatments, which can sometimes stop working for people whose bodies become resistant to them.
“There’s never been a time when the pipeline is as full as it is now,” says Nigel Key, MB, ChB, a hematologist and director of the Hemophilia and Thrombosis Center at the University of North Carolina. “I think it’s a reflection of the ability of biotechnology to bring these things forward.”
Two Types of Hemophilia
About 400,000 people worldwide -- including about 20,000 Americans -- have hemophilia, a bleeding disorder that slows the blood-clotting process.
People who have the condition are missing or low in one of two proteins, also called clotting factors, that work to make blood clot. A person who has the condition bleeds more than someone without it. The disease is either hemophilia A or B, depending on which clotting factors you're missing.
Last month, the FDA gave the green light to Adynovate, a new drug for people with type A. This comes on the heels of two other hemophilia drugs approved in 2015 - Nuwiq for hemophilia A and Ixinity for B. At least four other meds -- three for A and one for B -- are expected to follow in 2016.
Current Treatment and Challenges
Hemophilia can cause excessive bleeding after minor injuries. It can also lead to spontaneous internal bleeding into the joints, muscles, or organs, which can damage joints and cause other potentially life-threatening problems.
Those with the disease keep it under control by giving themselves shots of the appropriate protein. Depending on the severity of their condition, they may need injections only when bleeding happens. This type of treatment is called “on demand.” Or they may give themselves regular infusions to prevent bleeding. “The goal is to get the bleeds down to two or less a year,” Key says. “It’s basically eliminating bleeds.”