ATLANTA, Ga. (Ivanhoe Newswire)— 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, Atlanta Protocol.
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.
Contributors to this news report include: Cyndy McGrath, Executive Producer; Milvionne Chery, Field Producer; Roque Correa, Videographer & Editor.
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MEDICAL BREAKTHROUGHS
RESEARCH SUMMARY
TOPIC: ATLANTA PROTOCOL STOPS THE BLEED FOR HEMOPHILIA A
REPORT: MB #4764
BACKGROUND: Hemophilia A, also called factor VIII (FVIII) deficiency or classic hemophilia, is a genetic bleeding and clotting disorder caused by missing or defective factor VIII, a clotting protein. Although it’s passed down from parents to children, about one-third of cases are caused by a spontaneous mutation, a change in a gene. According to the U.S. Centers for Disease Control and Prevention, hemophilia occurs in approximately 1 in 5,000 live births. There are about 20,000 people with hemophilia in the U.S. with all races and ethnic groups affected. Hemophilia A is four times as common as hemophilia B and more than half of patients have a severe form of hemophilia.
(Source: https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A)
DIAGNOSING: It is vital to have an accurate diagnosis of hemophilia so it’s important to determine if other relatives have been diagnosed with a bleeding disorder or have experienced symptoms. Three ways to determine if you are a hemophilia carrier are checking your family tree, getting a clotting factor test, and a DNA Test. A clotting factor test, called an assay, determines the type of hemophilia and its severity. If your level is below 50 percent, you have mild hemophilia. If the level is above 50 percent, you still might have hemophilia; that’s why the three tests are important.
(Sources: https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A, https://www.ucsfhealth.org/conditions/hemophilia/diagnosis)
NEW TECHNOLOGY: Glaivy Batsuli, MD, the team at Children’s Healthcare of Atlanta and researchers from Emory University School of Medicine developed the “Atlanta Protocol” which involves treating patients with high dose factor VIII concentrates along with Emicizumab/Hemlibra. Over the course of a year, doctors collected data on seven patients ranging in age from 21 months to 12 years old. Patients were started on the combination of immune tolerance induction (ITI – can effectively remove an inhibitor in about 70% of patients with hemophilia A), FVIII infusions three times a week and Emicizumab/Hemlibra. They were followed for roughly 35 weeks. The findings demonstrated that three of the seven patients rid their bodies of the inhibitor or reduced the inhibitor to an unmeasurable level. There was also minimal bleeding events and no adverse effects such as blood clots. Six patients underwent surgery during this time and hospital stays decreased from the average stay of three to seven days down to one to two days.
(Sources: https://news.emory.edu/stories/2019/08/hematology_atlanta_protocol/index.html, https://www.hemophilia.org/Bleeding-Disorders/Inhibitors-Other-Complications/Inhibitors-for-Consumers/Immune-Tolerance)
FOR MORE INFORMATION ON THIS REPORT, PLEASE CONTACT:
ALLYSON WRIGHT
SENIOR PUBLIC RELATIONS COORDINATOR
CHILDREN’S HEALTHCARE OF ATLANTA
If this story or any other Ivanhoe story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Marjorie Bekaert Thomas at mthomas@ivanhoe.com
