fludarabine-based conditioning and allogeneic hematopoietic stem cell transplant (Masonic Cancer Center)

This therapeutic regimen consists of a fludarabine-based preparative conditioning regimen followed by an allogeneic hematopoietic stem cell transplant (HSCT). Developed by the Masonic Cancer Center at the University of Minnesota, the protocol is specifically tailored for patients with bone marrow failure syndromes, including dyskeratosis congenita (DC) and severe aplastic anemia (SAA). The conditioning phase utilizes a combination of lymphodepleting and myelosuppressive agents—such as fludarabine, cyclophosphamide, alemtuzumab, or anti-thymocyte globulin (ATG)—often supplemented with low-dose total body irradiation (TBI) to facilitate donor engraftment. By using a reduced-intensity or modified conditioning approach, the regimen aims to provide a curative stem cell replacement while minimizing the high risk of regimen-related toxicity and organ failure typically seen in DC and SAA patients undergoing standard transplantation.

Other names

Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic AnemiaReduced-intensity conditioning HSCT for DC and SAA