High-dose cyclophosphamide for aplastic anemia and autoimmunity.
High-dose cyclophosphamide was developed as a conditioning regimen for allogeneic bone marrow transplantation. Later, it was discovered that high-dose cyclophosphamide spares early hematopoietic stem cells because of their relatively high levels of the enzyme aldehyde dehydrogenase; thus, high-dose cyclophosphamide is a potent immunosuppressive agent, but nonmyeloablative. Recent reports demonstrate that high-dose cyclophosphamide without bone marrow transplantation induces durable treatment-free remissions in severe aplastic anemia and a variety of other autoimmune disorders; however, there is lingering concern about the safety of this approach.
Brodsky
Robert A
RA
Johns Hopkins Oncology Center, Baltimore, Maryland 21231-1000, USA. rbrodsky@jhmi.edu
D000741:Anemia, Aplastic / Q000188:drug therapy*; D001327:Autoimmune Diseases / Q000188:drug therapy*; D015551:Autoimmunity; D016026:Bone Marrow Transplantation; D003520:Cyclophosphamide / Q000008:administration & dosage*; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage*
D007166:Immunosuppressive Agents; D003520:Cyclophosphamide