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Transplant-associated thrombotic microangiopathy: opening Pandora’s box

Bone Marrow Transplantation volume 52, pages 1355–1360 (2017)Cite this article

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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.

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Acknowledgements

This work was supported by a grant from the Aplastic Anemia and MDS International Foundation and R01HL133113 (to RAB). EG is supported by the European Hematology Association Clinical Research Grant.

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  1. Hematology Department–Bone Marrow Transplantation Unit, G. Papanicolaou Hospital, Thessaloniki, Greece

    E Gavriilaki, I Sakellari & A Anagnostopoulos

  2. Division of Hematology, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA

    E Gavriilaki & R A Brodsky

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RAB is a member of the Scientific Advisory Board of Achillion Pharmaceuticals, Alexion Pharmaceuticals and Apellis Pharmaceuticals. The remaining authors declare no conflict of interest.

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Gavriilaki, E., Sakellari, I., Anagnostopoulos, A. et al. Transplant-associated thrombotic microangiopathy: opening Pandora’s box. Bone Marrow Transplant 52, 1355–1360 (2017). https://doi.org/10.1038/bmt.2017.39

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