Eculizumab in Transplant-Associated Thrombotic Microangiopathy

Prajwal Dhakal, Smith Giri, Ranjan Pathak, Vijaya Raj Bhatt

Research output: Contribution to journalArticlepeer-review

31 Scopus citations


Introduction: Transplant-associated thrombotic microangiopathy (TA-TMA) is a rare entity with no standard of care and high mortality, despite the use of plasma exchange. Methods: Using specific search terms, all cases having TA-TMA treated with eculizumab and indexed in MEDLINE (English language only) by November 2014 were reviewed. Results: A total of 26 cases, 53% men, had a median age of 33 years (range 2-61). Transplant-associated thrombotic microangiopathy occurred after stem-cell transplant (35%) or solid-organ transplant (65%), frequently associated with the use of cyclosporine or tacrolimus (96%). A disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS 13) level was always >10%. After TA-TMA diagnosis, the following drug adjustments were made: discontinuation of cyclosporine or tacrolimus in 45%, dose reduction in another 27%, continuation of the drugs in 23%, and switch from cyclosporine to tacrolimus in remaining 5%. Plasma exchange was performed in 1/443%. The median interval between transplant and initiation of eculizumab was 63 days (range 11-512). A median of 5.5 doses (range 2-21) of eculizumab was utilized with 92% response occurring after a median of 2 doses (range 1-18). At a median follow-up of 52 weeks (range 3-113), the survivors (92%) were doing well. Conclusion: Within the limits of this retrospective analysis, our study demonstrates that eculizumab use may result in high response rate and 1-year survival in patients with TA-TMA refractory to discontinuation of calcineurin inhibitor and plasma exchange.

Original languageEnglish (US)
Pages (from-to)175-180
Number of pages6
JournalClinical and Applied Thrombosis/Hemostasis
Issue number2
StatePublished - Mar 1 2017


  • calcineurin inhibitors
  • eculizumab
  • solid-organ transplant
  • stem cell transplant
  • thrombotic microangiopathy

ASJC Scopus subject areas

  • Hematology


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