New Hospital Protocol for Treatment of Infants with SCID

Care for Severe Combined Immunodeficiency Video length: 16:34

Patients with SCID can only be cured by a successful transplantation of hematopoietic stem cells from a healthy donor.  For SCID infants who have a brother or sister who is a "match", the transplant process is relatively easy, with a high rate of success.  However, most infants with SCID do not have a matched family donor and therefore need a hematopoietic stem cell transplant from another donor. 

Many HSCT centers have used bone marrow from parents to transplant infants with SCID. Because parents are usually only a "half-match" with their children, it is necessary to treat the parent’s bone marrow to remove the T cells that could react against the child’s body.  When bone marrow that has been depleted of T cells is given, it may be rejected by the patient, unless he or she is first given chemotherapy to "make space" for the bone marrow.  While some transplant centers give T cell-depleted parent’s marrow without first giving chemotherapy, these patients often have no engraftment or incomplete recovery of their immune system.  However, while chemotherapy can increase the chances for full recovery of the immune system, it can have significant side effects, such as lowering blood cell counts and causing mouth sores for the first few weeks afterward.

Kenneth Weinberg, MD, a physician-scientist at Childrens Hospital and SCID specialist, has recently developed a new protocol to treat SCID infants.  In this protocol, one of the chemotherapy drugs that is usually used for transplants (cytoxan) has been eliminated and replaced with a new antibody medicine (Campath H-1).  Campath H-1 may be able to help the T cell-depleted bone marrow grow in the patient without the side-effects of chemotherapy. 

This new treatment protocol was started in January, 2003 for patients treated at Childrens Hospital.  Initial experience has shown that this new protocol does, indeed, have the predicted improvement in side-effects, while leading to restoration of the immune system.