Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome
Status:
Completed
Trial end date:
2007-08-01
Target enrollment:
Participant gender:
Summary
Treatment for severe combined immunodeficiency (SCID) is a medical emergency. A stem cell
transplant (immature blood cells that can make other blood cells) from a (MSD) matched
sibling donor (brother or sister who is a "match" for your child's immune (HLA) type),
usually results in complete correction of immune function. However, most patients lack a
matched sibling donor, requiring the use of an alternate donor source.
Transplantation of cells from haploidentical family donors (typically parents) has resulted
in immune system correction in the majority of SCID individuals. However, only 65-80% of
patients survive greater than one year after this procedure. Failure results from
life-threatening infections, graft versus host disease (GvHD) or post-transplant
treatment-related effects. Also, for patients that survive beyond one year, B-cell (type of
blood cell that fights infection) and natural killer cell function (cell that attacks
infections and cancer cells) frequently fail to work, resulting in the need for long-term
treatment with intravenous gamma-globulin (IVIg).
In this study, in an effort to restore the overall cell function in patients with SCID,
researchers will use a highly purified CD133+ hematopoietic cell graft (stem cell transplant
without many mature donor white cells, called T-cells) obtained via use of the Miltenyi
CliniMACS device, a device not FDA approved.