A new paradigm: Diagnosis and management of HSCT-associated thrombotic microangiopathy as multi-system endothelial injury

Blood Rev. 2015 May;29(3):191-204. doi: 10.1016/j.blre.2014.11.001. Epub 2014 Nov 28.

Abstract

Hematopoietic stem cell transplantation (HSCT)-associated thrombotic microangiopathy (TA-TMA) is now a well-recognized and potentially severe complication of HSCT that carries a high risk of death. In those who survive, TA-TMA may be associated with long-term morbidity and chronic organ injury. Recently, there have been new insights into the incidence, pathophysiology, and management of TA-TMA. Specifically, TA-TMA can manifest as a multi-system disease occurring after various triggers of small vessel endothelial injury, leading to subsequent tissue damage in different organs. While the kidney is most commonly affected, TA-TMA involving organs such as the lung, bowel, heart, and brain is now known to have specific clinical presentations. We now review the most up-to-date research on TA-TMA, focusing on the pathogenesis of endothelial injury, the diagnosis of TA-TMA affecting the kidney and other organs, and new clinical approaches to the management of this complication after HSCT.

Keywords: Complement activation; Eculizumab; Hematopoietic cell transplant; Kidney disease; TA-TMA; Thrombotic microangiopathy.

Publication types

  • Review

MeSH terms

  • Complement System Proteins / immunology
  • Disease Management
  • Endothelium, Vascular / immunology
  • Endothelium, Vascular / metabolism
  • Endothelium, Vascular / pathology
  • Hematopoietic Stem Cell Transplantation / adverse effects*
  • Humans
  • Incidence
  • Risk Factors
  • Thrombotic Microangiopathies / diagnosis*
  • Thrombotic Microangiopathies / epidemiology
  • Thrombotic Microangiopathies / etiology*
  • Thrombotic Microangiopathies / therapy

Substances

  • Complement System Proteins