allogeneic hematopoietic stem cell transplantation

This intervention is a specialized allogeneic hematopoietic stem cell transplantation (HSCT) protocol developed by the Masonic Cancer Center at the University of Minnesota. It is specifically designed for patients with bone marrow failure syndromes, such as dyskeratosis congenita and severe aplastic anemia, who require red blood cell and platelet transfusions. The protocol (MT2013-34C) utilizes a fludarabine-based reduced-intensity conditioning (RIC) regimen—incorporating agents like alemtuzumab (Campath-1H), cyclophosphamide, and low-dose total body irradiation—to achieve donor cell engraftment while minimizing the high toxicity typically associated with myeloablative conditioning in these sensitive patient populations. The primary therapeutic objective is the restoration of normal hematopoiesis through the infusion of healthy donor-derived hematopoietic stem cells.

Other names

HSC Transplant for Dyskeratosis Congenita and Severe Aplastic AnemiaReduced-intensity conditioning HSCTHematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic Anemia