Information that investigators will learn, both now and in the future, will help doctors and other health professionals to better treat children with SCID. This natural history study is the largest coordinated prospective study of participants with SCID ever performed. Information is also being gathered on how and when the immune system recovers after bone marrow transplant (BMT), quality of life for long-term survivors, and about whether children develop normally after treatment. The goal of this study is to learn more about: (1) outcomes from the treatment of SCID in the modern era of medicine (2) what factors lead to the best long-term outcomes, such as best donor, conditioning regimen, timing of transplant, etc., and (3) what impact newborn screening and the early diagnosis of SCID has had on the long-term outcomes following BMT or gene therapy. There are no experimental therapies on this study.
Participants are then followed according to a schedule set out by the study protocol after the procedure. This study follows participants with SCID prospectively, meaning the study enrolls participants where there is a plan to receive a blood and marrow transplant, enzyme therapy, or gene therapy in the future. Severe Combined Immunodeficiency (SCID) Leaky SCID Omenn Syndrome Reticular Dysgenesis ADA SCID XSCID Stratum C: ADA deficient SCID and XSCID patients receiving alternative therapy including PEG-ADA ERT or gene therapy.Įach Group/Cohort Stratum will be analyzed separately.Stratum B: Atypical SCID (leaky SCID, Omenn syndrome and reticular dysgenesis with limited T cell diversity or number and reduced function), and.Stratum A: Typical SCID with virtual absence of autologous T cells and poor T cell function.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.Ĭhildren will be divided into three strata: 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. Why Should I Register and Submit Results?.
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