Transplant-associated thrombotic microangiopathy: theoretical considerations and a practical approach to an unrefined diagnosis

Bone Marrow Transplant. 2021 Aug;56(8):1805-1817. doi: 10.1038/s41409-021-01283-0. Epub 2021 Apr 19.


Transplant-associated thrombotic microangiopathy (TA-TMA) is an increasingly recognized complication of hematopoietic stem cell transplant (HSCT) with high morbidity and mortality. The triad of endothelial cell activation, complement dysregulation, and microvascular hemolytic anemia has the potential to cause end organ dysfunction, multiple organ dysfunction syndrome and death, but clinical features mimic other disorders following HSCT, delaying diagnosis. Recent advances have implicated complement as a major contributor and the therapeutic potential of complement inhibition has been explored. Eculizumab has emerged as an effective therapy and narsoplimab (OMS721) has been granted priority review by the FDA. Large studies performed mostly in pediatric patients suggest that earlier recognition and treatment may lead to improved outcomes. Here we present a clinically focused summary of recently published literature and propose a diagnostic and treatment algorithm.

Publication types

  • Review

MeSH terms

  • Child
  • Complement System Proteins
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Humans
  • Multiple Organ Failure
  • Thrombotic Microangiopathies* / diagnosis
  • Thrombotic Microangiopathies* / etiology
  • Thrombotic Microangiopathies* / therapy
  • Transplants*


  • Complement System Proteins