Hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA) is a rare complication of stem cell transplant procedures. A thrombus is a clot that can cause a serious blockage(s) in blood vessels. In thrombotic microangiopathy (TMA), clots develop in small blood vessels and capillaries creating damaging blockages that may lead to multiorgan failure and death.
TMA following a stem cell transplant procedure is a rare but serious complication of HSCT that appears to involve complement activation, inflammation, tissue hypoxia and blood clots, leading to progressive organ damage and death. Mortality in patients who develop severe transplant-related TMA is 80% (across both adults and children). Currently, there are no approved treatment options in the U.S. or Europe.
Reference
Rosenthal J. Hematopoietic cell transplantation-associated thrombotic microangiopathy: a review of pathophysiology, diagnosis, and treatment. J Blood Med. 2016;7:181-186.