Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant
| Status: | Active, not recruiting |
|---|---|
| Conditions: | Other Indications, Infectious Disease, HIV / AIDS, Lymphoma, Hematology |
| Therapuetic Areas: | Hematology, Immunology / Infectious Diseases, Oncology, Other |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 7/26/2018 |
| Start Date: | August 11, 1997 |
Induction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil
This pilot clinical trial studies total-body irradiation followed by cyclosporine and
mycophenolate mofetil in treating patients with severe combined immunodeficiency (SCID)
undergoing donor bone marrow transplant. Giving total-body irradiation (TBI) before a donor
bone marrow transplant using stem cells that closely match the patient's stem cells, helps
stop the growth of abnormal cells. It may also stop the patient's immune system from
rejecting the donor's stem cells. The donated stem cells may mix with the patient's immune
cells and help destroy any remaining abnormal cells. Sometimes the transplanted cells from a
donor can also make an immune response against the body's normal cells. Giving cyclosporine
and mycophenolate mofetil after the transplant may stop this from happening.
mycophenolate mofetil in treating patients with severe combined immunodeficiency (SCID)
undergoing donor bone marrow transplant. Giving total-body irradiation (TBI) before a donor
bone marrow transplant using stem cells that closely match the patient's stem cells, helps
stop the growth of abnormal cells. It may also stop the patient's immune system from
rejecting the donor's stem cells. The donated stem cells may mix with the patient's immune
cells and help destroy any remaining abnormal cells. Sometimes the transplanted cells from a
donor can also make an immune response against the body's normal cells. Giving cyclosporine
and mycophenolate mofetil after the transplant may stop this from happening.
PRIMARY OBJECTIVES:
I. To safely establish partial lymphoid chimerism (1-95% donor cluster of differentiation
[CD]3+ cells) using a non-lethal conditioning regimen in patients with severe combined
immunodeficiency syndrome.
II. To define the kinetics of immune reconstitution following a non-lethal conditioning
regimen in patients with immunodeficiency diseases.
OUTLINE:
Patients receive cyclosporine orally (PO) or intravenously (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.
After completion of study treatment, patients are followed up at 6 months and then yearly for
5 years.
I. To safely establish partial lymphoid chimerism (1-95% donor cluster of differentiation
[CD]3+ cells) using a non-lethal conditioning regimen in patients with severe combined
immunodeficiency syndrome.
II. To define the kinetics of immune reconstitution following a non-lethal conditioning
regimen in patients with immunodeficiency diseases.
OUTLINE:
Patients receive cyclosporine orally (PO) or intravenously (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.
After completion of study treatment, patients are followed up at 6 months and then yearly for
5 years.
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
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