Allogeneic hematopoietic stem cell transplantation (Northwell Health)

Allogeneic hematopoietic stem cell transplantation (HSCT) at Northwell Health for high-risk hemoglobinopathies involves the use of a reduced intensity conditioning (RIC) regimen followed by the infusion of healthy donor stem cells. The RIC protocol specifically employs alemtuzumab (a monoclonal antibody targeting CD52 for lymphodepletion), fludarabine (a purine analog), and melphalan (an alkylating agent) to suppress the recipient's immune system and bone marrow, allowing for the engraftment of donor cells with reduced toxicity compared to traditional myeloablative approaches. This therapy aims to establish stable donor chimerism, thereby providing a source of functional hemoglobin and curing sickle cell disease or beta-thalassemia major. The protocol is primarily evaluated in pediatric and young adult patients to assess its efficacy and safety in achieving long-term disease correction.

Other names

Reduced Intensity HSCT for Sickle Cell and ThalassemiaAllogeneic HSCT (Northwell Health)