Hemophilia Treatment Options

People with hemophilia need treatment to enhance their body’s ability to form blood clots and stop uncontrolled bleeding from occurring. In hemophilia, the body is missing one or more clotting factors, or proteins in the blood that help it clot. Treatment needs vary, depending on the severity of disease (mild, moderate, or severe) and the type of hemophilia. We also treat Von Willebrand disease, other factor deficiencies, and inherited platelet disorders.

Our team has decades of experience treating children using blood factor and other standard treatments, and we are also pioneering the use of promising new treatments like gene therapy. Current treatments include the following:

Factor product

Your child’s need for clotting protein replacement depends on the severity (mild, moderate, or severe) and type of bleeding disorder. As the first academic research center in the nation to isolate the use of blood factors for hemophilia, we are experts in determining the right type and amount your child needs. Most people receive regular infusions to promote clotting and prevent bleeds.

Antibody product

For patients with hemophilia A who have inhibitors against blood factor (preventing clotting), we offer antibodies to boost clotting, such as emicizumab, which can promote clotting with a subcutaneous (under the skin) shot and eliminate the need for frequent IV or port infusions. Emicizumab is easier to use and often provides better control than factor replacement for those with inhibitors.

Emicizumab is also available to patients with hemophilia A without inhibitors.

Gene therapy for hemophilia

Our Hemophilia Treatment Center was one of the first centers to conduct gene therapy trials in the early 1990s. We are committed to supporting innovative therapies and are currently conducting gene therapy clinical trials for adults with hemophilia. In gene therapy, a working copy of the gene is introduced to correct the missing factor. Gene therapy is a promising future treatment for hemophilia.

Genetic testing for hemophilia

Hemophilia is often inherited, but not always, and it occurs in approximately 1 in 5,000 males. If your family has a history of hemophilia, we provide genetic testing and genetic counseling to determine if you or your child is a carrier.

Other hemophilia treatments

Depending on your child’s situation, we might offer other therapies to promote the release of blood clotting factors in the body, prevent blood clots from breaking down, and control bleeding from nosebleeds or heavy menstrual periods.