New drug treats hemophilia A in children

A medication helps children with blood disorder

Norton Children’s Cancer Institute, affiliated with the UofL School of Medicine, cares for children with hemophilia, blood cancers and many other diseases. Emicizumab is a new drug now being used for children with moderate to severe hemophilia A.

Hemophilia is a bleeding disorder that stops a person’s blood from clotting, so it becomes difficult to stop bleeding once it starts, even from minor cuts or other injuries. There are 30,000 to 33,000 people living with hemophilia in the U.S., according to the National Hemophilia Foundation. The two most common types of hemophilia are:

  • Hemophilia A: This type is the most common, occurring in about 1 in 5,000 male births. About 400 babies are born each year with hemophilia A.
  • Hemophilia B: The second most common type, it occurs in about 1 in 25,000 male births.

Signs and symptoms of hemophilia

Hemophilia is typically diagnosed in a child’s infancy since it is usually passed through families.

“We know to check children who have a family history of the disease,” said Ashok B. Raj, pediatric hematologist/oncologist with Norton Children’s Cancer Institute. There are other signs including:

  • Excessive or uncontrollable bleeding from a heel stick (to collect blood for routine tests) or after circumcision
  • Unexplained irritability, sleepiness or lethargy in infants
  • Repeated vomiting
  • Convulsions or seizures

Treating hemophilia

Until recently, the most common treatment for hemophilia A was to replace the missing element in the blood so it can clot properly. The issue with this treatment was that in a third of children, the body produced inhibitors, which made the treatment less and less effective.

Children with inhibitors require more frequent infusions. They also make frequent hospital or emergency department visits, and many require placement of a central venous access device. They need more intervention and have frequent bleeding complications, including infections.

When patients develop inhibitors, it has a significant impact on their quality of life, and it’s very difficult for families to manage these children at home. Regardless of the presence of these inhibitors, bleeding under the skin or inside the body over time can result in irreversible damage and chronic joint pain.

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Many patients have zero bleeds with Emicizumab

Emicizumab (brand name Hemlibra) is a relatively new drug, but its effects are profound. Emicizumab helps prevent or reduce the frequency of bleeding episodes in patients with moderate or severe hemophilia A, with or without factor VIII inhibitors. Moderate hemophilia is defined as 1% to 5% of factor VIII activity. Severe is less than 1%.

Norton Children’s Cancer Institute has been an early adopter of emicizumab, which was approved by the Food and Drug Administration in 2018.

“We have seen a remarkable reduction in bleeding rates,” Dr. Raj said. “Many patients have had zero bleeds.

“We follow a protocol of four weeks of weekly dosing. After that, there are three options for patients: weekly, every two weeks or every four weeks. It’s much less of a strain than factor VIII replacement, which requires injections two or three times a week.”

Caring for patients taking emicizumab

Norton Children’s Cancer Institute offers patients a multidisciplinary team of professionals to support them and their families, including hematologists, nurse practitioners, clinical nurse specialists, physical therapists, social workers and psychologists.

Patients and their families are excited about this new treatment for hemophilia A.