Approaching treatment of transplant‐associated thrombotic Microangiopathy from two directions with Eculizumab and transitioning from Tacrolimus to Sirolimus. Issue 11 (6th October 2019)

• Record Type: Journal Article
• Authors: Jan, Anna S.; Hosing, Chitra; Aung, Fleur; Yeh, Jason
• Is Part Of: Transfusion. Volume 59:Issue 11(2019)
• Page Start: 3519
• Page End: 3524
• Publication Date: 2019-10-06
• Subjects: Hematology -- Periodicals; Blood -- Transfusion -- Periodicals; Blood Group Antigens -- Periodicals; Blood Preservation -- Periodicals; Blood Transfusion -- Periodicals; 615
• DOI: 10.1111/trf.15534
• Languages: English
• ISSNs: 0041-1132

Abstract

Abstract : BACKGROUND: In the past, conventional treatment strategies for transplant‐associated thrombotic microangiopathy (TA‐TMA) have not proven to be very effective. Recently, eculizumab which is a humanized monoclonal antibody that works as a terminal complement inhibitor has demonstrated promise in the treatment landscape of TA‐TMA. METHODS AND MATERIALS: This was a single‐center retrospective analysis of 20 consecutive adult patients with TA‐TMA: 10 patients who received conventional therapy and 10 patients who received eculizumab‐based therapy. These patients had undergone allogeneic HSCT at MD Anderson Cancer Center between August 2011 and September 2016. RESULTS: When comparing the treatment outcomes in the two cohorts, none of the patients in the conventional therapy group obtained a hematologic or complete response according to our response criteria whereas seven patients in the eculizumab group achieved a hematologic response with one patient achieving a complete response with organ recovery. In addition, overall survival at the end of assessment was 60% in the eculizumab cohort and 30% in the conventional cohort. One major difference in practice at our institution versus previously published studies is the management of immunosuppression. In a majority of patients, tacrolimus was continued or transitioned to sirolimus for GVHD prophylaxis. CONCLUSION: Response rates and survival were improved for patients who were transitioned to sirolimus, so a two‐pronged approach of inhibiting complement along with providing an alternative effective immunosuppressive agent may be beneficial in the treatment of early onset TA‐TMA.