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Hematopoietic Stem Cell Transplantation-Northwell Health-sickle cell disease-beta-thalassemia major
- Development stage
- Unknown
- Lead developer
- Northwell Health
- Modality
- Stem Cell Therapies → Cell Therapies, Small Molecules, Monoclonal Antibodies → Antibody-Based Therapeutics
- Administration
- Intravenous
01
Overview
This therapeutic protocol involves allogeneic hematopoietic stem cell transplantation (HSCT) preceded by a reduced intensity conditioning (RIC) regimen. Developed by Northwell Health, the regimen specifically utilizes alemtuzumab, fludarabine, and melphalan to achieve lymphodepletion and myelosuppression with lower toxicity than traditional myeloablative conditioning. It is designed for pediatric and young adult patients with high-risk hemoglobinopathies, including sickle cell disease and beta-thalassemia major. The goal is to allow for donor stem cell engraftment while minimizing transplant-related mortality and morbidity. Post-transplant immunosuppression typically includes cyclosporine, mycophenolate mofetil, or tacrolimus to prevent graft-versus-host disease (GVHD).
- Other names
- Reduced Intensity Conditioning Allogeneic Hematopoietic Stem Cell Transplantation (Northwell Health)RIC-HSCT for HemoglobinopathiesAlemtuzumab + Fludarabine + Melphalan conditioning for HSCT
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