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Hemophilia Treatment

Also called: Classic hemophilia, Factor VIII deficiency, Christmas disease, Factor IX deficiency

Hemophilia is a rare bleeding disorder in which the blood does not clot properly. A person with hemophilia may bleed longer after an injury or may bleed internally. The disorder can be mild, moderate, or severe. In some cases, it may be life-threatening.

Hemophilia is caused by low or missing clotting factor. Clotting factors are proteins in the blood needed for normal blood clotting. The type of hemophilia a person has depends on which clotting factor is affected.

Types of hemophilia include:

  • Hemophilia A: lack of clotting factor VIII (8).
  • Hemophilia B: lack of clotting factor IX (9).
  • Hemophilia C: lack of clotting factor XI (11).

Hemophilia usually occurs due to a gene change (mutation) that is inherited (passed down in families). Sometimes the mutation is a new gene change. The disorder most often affects males.

Hemophilia is a lifelong disorder. There is no cure. But most people with hemophilia have a normal life expectancy. With the right treatment and self-care, a person with hemophilia can live a healthy, active life.

Find out more about hemophilia on the Together by St. Jude™ online resource.

Hemophilia treatment

Treatment for hemophilia depends on the severity of the disorder and the patient’s medical needs. Current therapies include:

  • Clotting factor replacement therapy may be given to prevent bleeding episodes (prophlyaxis or prophy) or as needed for treatment of bleeding episodes.
  • Other medicines such as desmopressin acetate (DDVAP) and antifibrinolytic medicines may be used to help the blood to clot or help prevent the breakdown of blood clots.
  • Scientists are also studying new gene therapy treatments for hemophilia. Gene therapies work by changing or repairing genes to correct the defective genes that cause hemophilia. Researchers continue to test gene therapy in clinical trials.

Hemophilia clinical trials

St. Jude offers clinical trials and research studies for children, teens, and young adults with hemophilia. Learn more about clinical research at St. Jude.

GO-8: Gene Therapy for Haemophilia A Using a Novel Serotype 8 Capsid Pseudotyped Adeno-associated Viral Vector Encoding Factor VIII-V3

Study goal:

The main goal of this study is to test the safety and effectiveness of the study drug at different doses to find out what effects, if any, it has on people.


Males at least 18 years old

HEMOCOG: Neurocognitive Functions, Health Literacy, and Transition Readiness in Pediatric Hemophiliacs

Study goal:

This study will help researchers understand how hemophilia affects how patients think, feel, and experience school.


6 to 18 years old

NCBP01: Safety Study of Unlicensed, Investigational Cord Blood Units Manufactured by the NCBP for Unrelated Transplantation

Study goal:

The primary purpose of this study is to examine the safety of administration of the unlicensed investigational NCBP HPC-CORD BLOOD products in a multi-institution setting.

Hemophilia care at St. Jude

  • A multidisciplinary team of staff at St. Jude provides the expert care needed to manage hemophilia and prevent long-term health problems.
  • St. Jude helps each patient and family cope with the physical, emotional, psychological, educational, and social challenges related to having hemophilia.
  • The St. Jude Bleeding Disorders Program is an important resource for patients and families affected by the disorder. The program has been named a Hemophilia Treatment Center by the federal government.
A statue of children running and holding hands

Seeking treatment at St. Jude

Patients accepted to St. Jude must have a disease we treat and must be referred by a physician or other qualified medical professional. We accept most patients based on their ability to enroll in an open clinical trial.

How to seek treatment

Contact the Physician / Patient Referral Office

Call: 1-888-226-4343 (toll-free) or 901-595-4055 (local)  | Fax: 901-595-4011 | Email: | 24-hour pager: 1-800-349-4334


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