SCID PILOT STUDY

Some background on SCID

SCID, or Severe Combined Immunodeficiency, is a disorder that severely affects the immune system. Unless treated, babies with this disorder will die at a few months of age because they cannot fight off the usual infections that all babies get. With treatment, most babies live.

Treatment for a SCID baby includes a bone-marrow transplant. This allows the baby to live because it can make T cells that untreated SCID babies cannot make.

Purpose

The purpose of the SCID pilot is to determine the best way to find SCID babies.

We believe that a molecular test will help us to know which babies are making T cells and which babies are not. We may try other tests to see if they can help us to predict which babies have SCID and which do not. The molecular test means that we will be looking for a piece of DNA that is present in most babies. If we cannot find the piece of DNA in a baby, then we may ask for another sample to be sure of the result or we may recommend that the baby be seen by an expert in immunology to have some additional testing done. If the baby has SCID, then the immunologist will work with specialists in bone marrow transplantation for the best plan for the baby.

We expect that once we start testing to find SCID babies, we may find babies with other immune diseases. If we do find such babies, they would also be seen by an immunologist.