A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome
Conditions
- Immunodeficiency
- Hematopoietic Stem Cell Transplantation
Interventions
- PROCEDURE: Allogeneic HSCT
- DRUG: Busulfan test dose
- DRUG: Mycophenolate mofetil (MMF)
- DRUG: Tacrolimus
- DRUG: Busulfan
- RADIATION: Total Body Irradiation (TBI)
- DRUG: Fludarabine
- DRUG: Cyclophosphamide (CY)
- DRUG: Post-Transplant Cyclophosphamide (PTCY)
Sponsor
National Cancer Institute (NCI)