Emerging therapeutic and preventive approaches to transplant-associated thrombotic microangiopathy.

ABSTRACT:

Purpose of review

Transplant-associated thrombotic microangiopathy (TA-TMA) is a complication that can occur in both allogeneic and autologous haematopoietic cellular therapy (HCT) recipients and is associated with significant morbidity and mortality. Although TA-TMA is a complex disease, there is emerging evidence that complement activation and endothelial dysfunction play a key role in the pathophysiology of the disease. The use of eculizumab has improved survival in patients with high risk and severe disease, but mortality rates in treated patients still exceed 30%, highlighting the need for novel approaches.

Recent findings

There are multiple ongoing and planned clinical trials investigating novel complement agents in TA-TMA and other TMAs. Drugs vary by targets of the c

SUBMITTER: Schoettler M 

PROVIDER: S-EPMC9908033 | biostudies-literature | 2021 Nov

REPOSITORIES: biostudies-literature