期刊
TRANSFUSION AND APHERESIS SCIENCE
卷 54, 期 2, 页码 181-190出版社
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.transci.2016.04.007
关键词
Thrombotic microangiopathy; TA-TMA; Complement; Eculizumab; Hematopoietic stem cell transplant
类别
资金
- NCATS NIH HHS [UL1 TR001425] Funding Source: Medline
- NICHD NIH HHS [K12 HD028827] Funding Source: Medline
Hematopoietic stem cell transplantation (HSCT)-associated thrombotic microangiopathy (TA-TMA) is an understudied complication of HSCT that significantly affects transplant related morbidity and mortality. Over the past several decades, the cause of TA-TMA has remained unknown, limiting treatment options to non-specific therapies adapted from other diseases. Recent prospective studies dedicated to the study of TA-TMA have provided new insights into the pathogenesis of, and genetic susceptibility to TA-TMA, raising awareness of this important transplant complication and allowing for the identification of potentially novel therapeutic targets. Specifically, many patients with TA-TMA develop multi organ tissue injury through endothelial damage mediated by the activation of the complement pathway, leading to rational therapeutic strategies including complement blockade. This new knowledge has the potential to favorably influence clinical practice and change the standard of care for how patients with TA-TMA are managed. In this review, we summarize novel approaches to the recognition and management of TA-TIVIA, using case examples to illustrate key clinical points that hopefully lead to improved short and long-term outcomes for these complex HSCT patients, who remain at significant risk for treatment related morbidity and mortality. (C) 2016 Elsevier Ltd. All rights reserved.
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