Ravulizumab for treating thrombotic microangiopathies following haematopoietic stem cell transplantation in children

Ravulizumab is in development for the treatment of thrombotic microangiopathies following haematopoietic stem cell transplantation (HSCT-TMA) in children, adolescents, and adults. Thrombotic microangiopathies (TMA) are clinical syndromes defined by the destruction of red blood cells, low platelets, and organ damage due to the formation of microscopic blood clots in capillaries and small arteries. Haematopoietic stem cell transplantation (HSCT) associated thrombotic microangiopathy, can occur after a HSCT and can often be fatal. Currently there are no approved therapies for the treatment of HSCT-TMA and intervention usually involves withdrawal of triggering agent (e.g., immunosuppressive therapies) or management of triggering conditions.