Immunodeficiencies Clinical Trial
Official title:
Phase I/II Study of Reduced Intensity Conditioning for Patients With Non-Malignant Diseases Using CD3+/CD19+ Depleted Unrelated Donor or Partially Matched Related Donor Peripheral Stem Cells
This is a Phase II trial to determine the ability of a reduced intensity conditioning regimen to allow successful engraftment with CD3+ /CD19+ depleted peripheral stem cell grafts from mismatched donors. There are two conditioning regimens depending upon patient diagnosis and age.
This study will allow transplantation using a reduced intensity conditioning regimen for
children with non-malignant diseases who lack a matched related or unrelated donor. Donors
will be unrelated or partially matched related, depending upon urgency and availability. If
each parent is haploidentical, the mother will be preferred, as there is evidence of reduced
transplant related mortality and superior survival with a maternal donor. The risks of severe
graft vs host disease (GVHD) and Epstein-Barr lymphoproliferative disorder will be reduced or
eliminated by T and B cell depletion using the Miltenyi Clinimacs device. Patients with bone
marrow failure syndromes, who are at high risk for rejection, will undergo pre-conditioning
immune suppression with Thymoglobulin. It is recommended that patients with
immunedysregulation syndromes receive pre-RIC alemtuzumab as this may reduce the risk on
non-engraftment and hyperinflammatory states.
Post-transplant immune suppression will be used to prevent GVHD, as CD3 depletion does not
deplete as completely as CD34+ selection. It will be rapidly weaned if no GVHD by day 100 to
allow immune reconstitution.
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