Transplant-associated thrombotic microangiopathy: opening Pandora's box

Bone Marrow Transplant. 2017 Oct;52(10):1355-1360. doi: 10.1038/bmt.2017.39. Epub 2017 Mar 13.

Abstract

Transplant-associated thrombotic microangiopathy (TA-TMA) is an early complication of hematopoietic cell transplantation (HCT). A high mortality rate is documented in patients who are refractory to calcineurin inhibitor cessation. Estimates of TA-TMA prevalence vary significantly and are higher in allogeneic compared with autologous HCT. Furthermore, our understanding of the pathophysiology that is strongly related to diagnosis and treatment options is limited. Recent evidence has linked TA-TMA with atypical hemolytic uremic syndrome, a disease of excessive activation of the alternative pathway of complement, opening the Pandora's box in treatment options. As conventional treatment management is highly inefficient, detection of complement activation may allow for early recognition of patients who will benefit from complement inhibition. Preliminary clinical results showing successful eculizumab administration in children and adults with TA-TMA need to be carefully evaluated. Therefore, realizing the unmet needs of better understanding TA-TMA in this complex setting, we aimed to summarize current knowledge focusing on (1) critical evaluation of diagnostic criteria, (2) epidemiology and prognosis, (3) recent evidence of complement activation and endothelial damage and (4) treatment options.

Publication types

  • Review

MeSH terms

  • Allografts
  • Atypical Hemolytic Uremic Syndrome* / etiology
  • Atypical Hemolytic Uremic Syndrome* / metabolism
  • Atypical Hemolytic Uremic Syndrome* / mortality
  • Atypical Hemolytic Uremic Syndrome* / physiopathology
  • Autografts
  • Complement Activation*
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Thrombotic Microangiopathies* / etiology
  • Thrombotic Microangiopathies* / metabolism
  • Thrombotic Microangiopathies* / mortality
  • Thrombotic Microangiopathies* / physiopathology