Abstract
This study is a prospective evaluation of children with Severe Combined Immune Deficiency
(SCID) who are treated under a variety of protocols used by participating institutions. In
order to determine the patient, recipient and transplant-related variables that are most
important in determining outcome, study investigators will uniformly collect pre-, post- and
peri-transplant (or other treatment) information on all children enrolled into this study.
Children will be divided into three strata:
- Stratum A: Typical SCID with virtual absence of autologous T cells and poor T cell
function
- Stratum B: Atypical SCID (leaky SCID, Omenn syndrome and reticular dysgenesis with
limited T cell diversity or number and reduced function), and
- Stratum C: ADA deficient SCID and XSCID patients receiving alternative therapy including
PEG-ADA ERT or gene therapy.
Each Group/Cohort Stratum will be analyzed separately.
