Stopping the bleed for hemophilia a
Every year about 400 babies are born with hemophilia A, a rare bleeding disorder in which they are missing a critical clotting protein, called factor VIII. Without the protein, their blood is unable to clot when they get a cut and some patients may develop antibodies that fight against the medicine that is treating their condition. The details on a new approach that is showing success.
Fourteen-year-old Jaylynn Smith-Brown always knew that he had to keep his distance from others.
“I couldn’t run around with all the other kids, play tag or nothing,” recalled Smith-Brown.
Jaylynn was born with a bleeding disorder called hemophilia A, or factor VIII deficiency. If he got a cut, it could cause severe bleeding.
“It felt like I was stuck in a bubble,” Smith-Brown shared.
Even worse, his body was attacking the factor VIII infusions that were meant to treat his condition.
“Because their body doesn’t recognize it, because their body doesn’t make it, their bodies can actually attack the medicine that’s there to keep them safe,” explained Glaivy Batsuli, MD, an assistant professor at Emory University School of Medicine department of pediatrics and pediatric hematologist and oncologist at the Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta.
So, to treat it …
“We give the factor medicine very often, usually every day at very high doses to try to trick your body into thinking that the medicine should be there,” elaborated Dr. Batsuli.
“That’s pretty difficult on a child. They become very emotionally exhausted,” added Robert F. Sidonio Jr., MD, MSC, associate director of the hemostasis and thrombosis program and director of clinical operations and clinical research of the Hemostasis and Thrombosis Program at Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta and associate professor at Emory University School of Medicine.
Doctors at Children’s Healthcare of Atlanta devised a first-of-its-kind approach called the Atlanta Protocol. By treating patients with a high dose of factor VIII along with the drug emicizumab, doctors can get rid of the antibodies that fight against factor VIII and prevent patients from bleeding. Jaylynn was one of the first patients in the world to try this new approach in 2019.
“He’s one of the first two patients in the world to be tolerized using this regimen,” illustrated Dr. Sidonio.
For Jaylynn, that means he does not have to live his life in a bubble anymore.
“It feels like I’m a regular boy now,” Smith-Brown exclaimed.
With the Atlanta Protocol, patients only have to do the factor VIII infusions three times a week versus every day with the current line of treatment. It is called the Atlanta Protocol as a nod to the city in which it was developed.
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