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peripheral blood stem cells (National Institute of Allergy and Infectious Diseases)
- Development stage
- Unknown
- Lead developer
- National Institute of Allergy and Infectious Diseases
- Modality
- Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
Peripheral blood stem cells (PBSC) are hematopoietic progenitor cells collected from a donor's peripheral blood after mobilization. In the specific therapeutic context developed by the National Institute of Allergy and Infectious Diseases (NIAID), these cells are used for allogeneic transplantation in patients with Chronic Granulomatous Disease (CGD). The NIAID protocol (NCT05064540) utilizes a high-dose PBSC infusion following a conditioning regimen of alemtuzumab, busulfan, and total body irradiation. The primary objective is to replace the patient's defective hematopoietic system with donor cells that possess functional NADPH oxidase activity, thereby restoring the ability of phagocytes to produce reactive oxygen species and kill pathogens. To prevent graft-versus-host disease (GvHD), the protocol incorporates post-transplant cyclophosphamide and sirolimus.
- Other names
- PBSCHematopoietic stem cellsHSCAllogeneic peripheral blood stem cell transplant
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