Thymus Transplant

Innovative Procedure for DiGeorge Syndrome

Duke Children’s Hospital is the only U.S. medical center offering thymus transplantation to infants born with complete DiGeorge syndrome. This rare immune disorder is characterized by the lack of a thymus, the small gland near the heart that generates infection-fighting T cells. We strive to give your child the best chance to develop a strong immune system for a healthy life.

What is the Thymus?

The thymus is like a school house attended by stem cells, immature white blood cells from bone marrow, that develop into functioning T cells. In about six months, a small percentage of the developing T cells leave the thymus to become normal T cells that protect the infant by fighting off infection and not attacking the infant’s organs. 

When the thymus is present at birth, it is located on top of the heart. If an infant with a thymus requires heart surgery, the surgeon may remove a portion of the thymus to operate on the part of the heart that is underneath the thymus. This discarded thymus tissue may be used for thymus transplantation after permission is obtained from the infant’s parent(s). 

What is Thymus Transplantation?

Thymus transplantation is an investigational procedure that uses thin slices of thymus tissue to build the immune system of an infant born without a thymus. This can occur when a child is born with complete DiGeorge syndrome, a condition that can also cause other medical concerns, including a heart defect and/or calcium problems. Some infants with DiGeorge may have a genetic abnormality, and problems with vision and hearing, blockage of one or both nasal passages, and abnormal ears.

The donor thymus used for thymus transplantation is cut into thin slices and kept in the laboratory while testing is done on blood from the infant’s mother and from the infant.  The testing ensures the thymus donor does not have an infection that could harm the infant with complete DiGeorge syndrome.  The thin slices of thymus tissue are then implanted into the thigh muscle of an infant born without a thymus who has complete DiGeorge syndrome. Once in place, tiny blood vessels called capillaries grow from the muscle into the thymus tissue providing oxygen and nutrients.  If the procedure is successful, the infant develops T cells that can fight infections within about six to nine months. 

Unparalleled Expertise in Thymus Transplantation

  • Since developing thymus transplantation in 1992, Duke pediatric immunologists have performed more than 90 thymus transplants, primarily in infants with complete DiGeorge Syndrome. The overall survival rate is 71 percent.
  • Our pediatric immunology team trained specialists in the United Kingdom, which is now the only other medical center in the world performing thymus transplants.
  • The investigational biologic therapy used in the transplant process, RVT-802 (licensed to Enzyvant Therapeutics GmbH), recently received Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy designation from the FDA, conferring “special access to the Office of Tissues and Advanced Therapies for development guidance as well as expedited review pathways.”
  • Our thymus transplant team includes many specialists who provide compassionate care to your child and works closely with your child’s referring doctors to ensure individualized care. We guide you through the process and support your child’s recovery.
  • Our pediatric immunologists are dedicated researchers who are constantly striving to better understand disorders such as complete DiGeorge syndrome, and the long term outcomes for children who have undergone thymus transplantation. This work helps guide our approach to thymus transplantation.

THYMUS TRANSPLANTATION
The Process

THYMUS TRANSPLANTATION
Once Approved for Thymus Transplant

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