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Hematopoietic stem cell transplant–associated thrombotic microangiopathy: review of pharmacologic treatment options

2014; Wiley; Volume: 55; Issue: 2 Linguagem: Inglês

10.1111/trf.12859

1537-2995

Sara Kim, Monank Patel, Kendra Yum, Alla Keyzner,

Renal Transplantation Outcomes and Treatments

Background Transplant‐associated thrombotic microangiopathy ( TA ‐ TMA ) is a multifactorial disorder, which occurs as a result of treatment‐related endothelial injury and underlying disease process after hematopoietic stem cell transplantation ( HSCT ). The reported incidence of TA ‐ TMA after HSCT is 0% to 74% and has shown to be associated with mortality rate of up to 100%. TA ‐ TMA is often diagnosed late in the disease progression, and therapeutic plasma exchange ( TPE ) has not been shown to produce a high response rate. Study Design and Methods All E nglish‐language articles describing pharmacologic treatments for TA ‐ TMA were identified using O vid in the M edline database (1966‐ M ay 2014). Search was limited to the HSCT population. Results Approximately 50% to 63% of patients with TA ‐ TMA respond to withdrawal of the offending agent (calcineurin inhibitors) and TPE , and many will require additional treatment to better control the disease. Unfortunately, there is no established treatment strategy for TA ‐ TMA . A number of pharmacologic agents that have been explored for the treatment of TA ‐ TMA include rituximab, vincristine, defibrotide, pravastatin, and eculizumab. The overall response rates of these agents were similar (69%‐80%); however, the differences in the treatment costs vary significantly between these agents. Defibrotide is an investigational agent in the U nited S tates; therefore, it is not readily available for use. Conclusion Larger studies are warranted to validate the role of these pharmacologic agents in TA ‐ TMA as upfront therapy and in TPE ‐refractory patients. Recently suggested predictive biomarkers for TA ‐ TMA , such as neutrophil extracellular traps and circulating endothelial cells, deserve more attention in future studies.