Abstract
Objective: To evaluate response rates and survival in adults with transplant-associated thrombotic microangiopathy (TA-TMA) after allogeneic hematopoietic stem cell transplantation (HSCT) who were treated with eculizumab (ECU). Methods: Patients were identified retrospectively and data collected through HSCT and pharmacy databases. Results: Ten patients with TA-TMA after allogeneic HSCT were treated with ECU between 2013 and 2016. TA-TMA was diagnosed at a median of 93 days post-HSCT. Organ-specific injury was documented in all ten patients at time of TA-TMA diagnosis, the most common being renal dysfunction (90%). Acute GVHD (70%) and active infection (80%) were common at time of diagnosis. The median time to ECU initiation from TA-TMA diagnosis was 4 days. Seven patients received ECU as first-line therapy in combination with other treatment modalities, while three patients were treated with ECU as second-line therapy. ECU was well tolerated with the exception of one case of severe skin rash leading to discontinuation. ECU achieved an overall hematologic response rate of 70% and an overall survival rate of 60%. One patient achieved a complete response with corresponding organ recovery. Conclusion: Early initiation of ECU may not alter the disease process enough to restore organ function, but it may prolong survival.
Original language | English (US) |
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Pages (from-to) | 389-398 |
Number of pages | 10 |
Journal | European Journal of Haematology |
Volume | 101 |
Issue number | 3 |
DOIs | |
State | Published - Sep 2018 |
Keywords
- bone marrow transplantation
- eculizumab
- transplant-associated thrombotic microangiopathy
ASJC Scopus subject areas
- Hematology