This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).
Immunodeficiencies
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
This is a standard of care treatment guideline for allogeneic hematopoietic stem cell transplant (HSCT) in patients with primary immune deficiencies.
NOTE: many likely fall in the eligibility category of “CID” (see up to date “Combined immunodeficiencies, Table 1)
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
This is a standard of care treatment guideline for allogeneic hematopoietic stem cell transplant (HSCT) in patients with primary immune deficiencies. NOTE: PNP falls under eligibility criteria “CID”
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
This is a standard of care treatment guideline for allogeneic hematopoietic stem cell transplant (HSCT) in patients with primary immune deficiencies.