allogeneic hematopoietic stem cells (Masonic Cancer Center)

Allogeneic hematopoietic stem cell transplantation (HSCT) is an investigational cellular therapy program developed and studied by the Masonic Cancer Center at the University of Minnesota. This therapeutic approach involves the infusion of healthy, donor-derived (allogeneic) hematopoietic stem cells into patients with high-risk, non-malignant hematologic disorders. The primary goal is to replace the patient's diseased or dysfunctional hematopoietic system with healthy donor cells capable of reconstituting normal blood cell production and immune function. The program, specifically evaluated in the MT2014-10C clinical trial, targets a variety of rare conditions including sickle cell disease, thalassemia, Diamond-Blackfan anemia, paroxysmal nocturnal hemoglobinuria, Glanzmann's thrombasthenia, severe congenital neutropenia, and Shwachman-Diamond syndrome. The treatment protocol utilizes various conditioning regimens—such as reduced toxicity ablative, reduced intensity, and myeloablative regimens—incorporating agents like alemtuzumab, fludarabine, busulfan, and cyclophosphamide to facilitate successful donor engraftment.

Other names

allogeneic hematopoietic stem cell transplantationHSCT for High Risk HemoglobinopathiesMT2014-10CMT-2014-10CMT 2014-10C