kid sitting on the shoulders of parent

Hemophilia

Overview

The UK HealthCare Hemophilia Treatment Center (UKHTC) at Kentucky Children’s Hospital offers comprehensive care for adults and children with hemophilia and other bleeding disorders. Pediatric hemophilia is a complex and rare condition, and it requires specialized, multidisciplinary care. The providers at UKHTC work together with patients and families to prevent bleeding and other related complications while also helping children with rare bleeding disorders lead healthy, normal lives.

What is Hemophilia?

Hemophilia is a bleeding disorder caused by a lack of clotting factor, a protein that naturally occurs in blood to help stop bleeding. Without clotting factor, a child with hemophilia may bleed without accident or injury. Left untreated, it can lead to serious complications, including bleeding in the brain.
 

Types of Hemophilia Treatment

Hemophilia treatments help replace the missing clotting factor in a child’s blood, preventing bleeding and slowing or stopping further complications. This is often done with medications, such as:

  • Plasma-derived factor concentrates, which use plasma from healthy individuals without bleeding disorders to create a product with clotting factors. Plasma-derived factor concentrates may be given through blood transfusions.
  • Recombinant factor concentrates, which are made without human plasma and instead use genetically engineered DNA. This is sometimes called factor VIII concentrate.

Several medications rely on factor VIII concentrate for their effectiveness as hemophilia treatments, including:

  • Desmopressin acetate, which are medications that are similar to hormones a person without bleeding disorders naturally produce. These medications release factor VIII that is stored in the body’s tissues and increase the patient’s own factor VIII. This is often an effective treatment for patients with mild to moderate hemophilia.
  • Emicizumab, which replaces the function of factor VIII entirely, can reduce or prevent bleeding in patients with a specific type of hemophilia called hemophilia A.