Hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA)

HSCT-TMA is an acute, rare, and potentially life-threatening complication that can arise in children and adults who undergo a hematopoietic stem cell transplant or HSCT.

Thrombotic microangiopathy or TMA is a disease where microscopic blood clots form in small blood vessels that can lead to organ damage.¹ The complement system, which is part of the body’s immune system, can become overactivated in patients with TMA, causing the disease to progress.²

TMA can be difficult to recognize since patients can present varied symptoms, and many of these signs and symptoms are similar to other HSCT-related complications.¹

Up to 40% of transplants result in TMA, and about 40% of those cases can be fatal.¹  With no medicines currently approved for HSCT-TMA, there is significant unmet need for treatment.

Pegcetacoplan in HSCT-TMA

Pegcetacoplan is a targeted C3 therapy under investigation for HSCT-TMA. We believe pegcetacoplan may have the potential to control the symptoms of HSCT-TMA, with the goal of lowering mortality and helping to prevent long-term organ damage.