Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant
Adenosine Deaminase Deficiency, Autosomal Recessive Disorder, Immune System Disorder
About this trial
This is an interventional treatment trial for Adenosine Deaminase Deficiency
Eligibility Criteria
Inclusion Criteria: Patients with severe combined immunodeficiency syndrome: SCID with presence of B lymphocytes X-linked SCID (presence of B lymphocytes) Autosomal recessive SCID Patients with severe combined immunodeficiency syndrome: SCID with absence of T and B lymphocytes Patients with severe combined immunodeficiency syndrome: Purine metabolite deficiencies, deficiencies of the purine metabolites Adenosine deaminase (ADA) deficiency Purine nucleoside phosphorylase (PNP) deficiency DONOR: Related donor who is human leukocyte antigen (HLA) genotypically identical at least at one haplotype and may be genotypically or phenotypically identical for serological typing for HLA-A, B, C, and at the allele level for DRB1 and DQB1; related donors other than siblings must be matched at HLA-A, B, and C (at highest resolution available at the time of donor selection) and at DRB1 and DQB1 by deoxyribonucleic acid (DNA) typing; if more than one HLA-identical sibling is available, priority will be given to the oldest normal donor DONOR: Unrelated donors who are prospectively matched for HLA-A, B, C, DRB1 and DQB1 by DNA typing at the highest resolution routinely available at the time of donor selection; only a single allele disparity will be allowed for HLA-A, B, or C as defined by high resolution typing Exclusion Criteria: Patients with viral associated T cell immunodeficiency disorders, such as human immunodeficiency virus (HIV) Patients with other disease or organ dysfunction that would limit survival to less than 30 days DONOR: Identical twin DONOR: Pregnancy DONOR: HIV seropositive DONOR: A positive anti-donor cytotoxic cross match is absolute donor exclusion DONOR: Patient and donor pairs homozygous at a mismatched allele in the graft rejection vector are considered a two-HLA allele mismatch, i.e., the patient is A*0201, and this type of mismatch is not allowed DONOR: < 6 months old, > 75 years old
Sites / Locations
- Fred Hutch/University of Washington Cancer Consortium
Arms of the Study
Arm 1
Experimental
Treatment (cyclosporine, mycophenolate mofetil, transplant)
Patients receive cyclosporine PO or IV on days -3 to 100 followed by a taper until day 180 and mycophenolate mofetil PO or IV on days 0-40 with a taper until day 96 in the absence of unacceptable toxicity. Unrelated donor recipients also undergo TBI on day 0. Patients undergo bone marrow transplant on day 0.