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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.
- Jodele et al, Diagnostic and risk criteria for HSCT-associated thrombotic microangiopathy: a study in children and young adults. Blood. 2014 Jul 24;124(4):645 53.
- Jodele, et al, Transplantation-Associated Thrombotic Microangiopathy Risk Stratification: Is There a Window of Opportunity to Improve Outcomes? Transplantation and Cellular Therapy, Volume 28, Issue 7. 2022 Jul; 392.e1-392.e9.