The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
A 4/6, 5/6 or 6/6 HLA matched related or unrelated UCB unit available that will deliver a pre-cryopreservation total nucleated cell dose of ≥ 3 x 10e7 cells/kg, or double unit grafts, each cord blood unit delivering at least 2 x 10e7 cells/kg OR an 8 of 8 or 7 of 8 HLA allele level matched unrelated donor bone marrow or peripheral blood progenitor graft.
Creatinine ≤ 2.0 mg/dL and creatinine clearance ≥ 50 mL/min/1.73 m2.
Hepatic transaminases (ALT/AST) ≤ 4 x upper limit of normal (ULN).
Adequate cardiac function by echocardiogram or radionuclide scan (shortening fraction > 26% or ejection fraction > 40% or > 80% of normal value for age).
None
Parallel
Hydroxyurea
DrugOral administration
Alemtuzumab
DrugIntravenous (IV) administration.
Fludarabine
DrugIV administration
Melphalan
DrugIV administration
Thiotepa
DrugIV administration
Post-transplant treatment-related mortality (TRM)
The number of deaths related to the research intervention at day 100, 6 months, and 1 year post-transplant.
Neurodevelopmental milestones
Evaluation of the pace of attaining neurodevelopmental milestones after reduced-intensity conditioning as compared to myeloablative conditioning historical controls from the target population(s).
Immune Reconstitution
Evaluation of the pace of immune reconstitution.
Severe opportunistic infections
Evaluation of the incidence of severe opportunistic infections.
Donor cell engraftment
Determination of the feasibility of attaining robust donor cell engraftment (\>50% donor chimerism at 6 months) following reduced-intensity conditioning (RIC) regimens prior to HSCT in the target population(s).
Normal enzyme level
Determination of the feasibility of attaining and sustaining normal enzyme levels in the target population(s).
Neutrophil recovery
Determination of the pace of neutrophil recovery.
Platelet recovery
Determination of the pace of platelet recovery.
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Paul Szabolcs
Lead sponsor
University of Pittsburgh
Sponsor institution
