MT2013-31:Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis following Conditioning with Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
To evaluate the ability to achieve high-level donor hematopoietic engraftment (defined as neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant) using related and unrelated BM, PBSC, or UCB grafts following a reduced intensity conditioning regimen based on targeted-exposure busulfan, fludarabine +/- serotherapy in patients with inherited metabolic disorders and severe osteopetrosis.
• up to 55 years old
• diagnosis of an Inherited Metabolic Disorders (IMD)
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
• uncontrolled bacterial, fungal or viral infections including HIV
• women who are pregnant
Drug: IMD Preparative Regimen, Drug: Osteopetrosis Haploidentical Only Preparative Regimen, Drug: Osteopetrosis Only Preparative Regimen, Biological: Stem Cell Transplantation, Drug: cALD HR-D (High-Risk, Regimen C), Drug: cALD HR-D (High-Risk, Regimen D), Drug: cALD SR-A (Standard-Risk, Regimen A), Drug: cALD SR-B (Standard-Risk, Regimen B)
Rare Diseases
Allogeneic Hematopoietic Cell Transplantation, Clinics and Surgery Center (CSC), IMD, Inherited metabolic disorders, osteopetrosis