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Case Report

Eculizumab Treatment in a Patient with Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy and Steroid-Refractory Acute Graft Versus Host Disease

by
Cristina Fernández
,
Ana Lario
,
Rafael Forés Cachón
* and
Rafael Cabrera
Servicio de Hematología y Hemoterapia, Hospital Puerta de Hierro, calle Joaquín Rodrigo 2, Majadahonda, 28222 Madrid, Spain
*
Author to whom correspondence should be addressed.
Hematol. Rep. 2015, 7(4), 6107; https://doi.org/10.4081/hr.2015.6107
Submission received: 9 July 2015 / Revised: 16 October 2015 / Accepted: 20 October 2015 / Published: 9 December 2015

Abstract

A 30-year-old man with acquired aplastic anemia underwent an HLA-identical bone marrow transplant. He developed a grade III acute graft versus host disease (GVHD) refractory to various lines of treatment. On post-transplant day 196, he was diagnosed with stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA) and he received treatment with eculizumab 900 mg iv weekly for 4 doses followed by a single dose of 1200 mg 2 weeks later. After the first dose of eculizumab, the patient ceased to require transfusions and a progressive improvement in analytical parameters for microangiopathy was observed until their complete normalization. Coinciding with the improved of HSCT-TMA, the patient presented a clear response to his acute GVHD with disappearance of the diarrhea and bilirubin normalization. He was discharged eight weeks after the start of treatment. Unfortunately, one month later, the patient was readmitted for a GVHD relapse and he died two weeks later by an acute respiratory distress syndrome. In our case, the rapid clinical and analytical response to early treatment with eculizumab supports the implication of the complement in HSCT-TMA and suggests that the drug has a beneficial effect when used as coadjuvant therapy in acute GVHD.
Keywords: eculizumab; thrombotic microangiopathy; acute graft versus host disease eculizumab; thrombotic microangiopathy; acute graft versus host disease

Share and Cite

MDPI and ACS Style

Fernández, C.; Lario, A.; Forés Cachón, R.; Cabrera, R. Eculizumab Treatment in a Patient with Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy and Steroid-Refractory Acute Graft Versus Host Disease. Hematol. Rep. 2015, 7, 6107. https://doi.org/10.4081/hr.2015.6107

AMA Style

Fernández C, Lario A, Forés Cachón R, Cabrera R. Eculizumab Treatment in a Patient with Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy and Steroid-Refractory Acute Graft Versus Host Disease. Hematology Reports. 2015; 7(4):6107. https://doi.org/10.4081/hr.2015.6107

Chicago/Turabian Style

Fernández, Cristina, Ana Lario, Rafael Forés Cachón, and Rafael Cabrera. 2015. "Eculizumab Treatment in a Patient with Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy and Steroid-Refractory Acute Graft Versus Host Disease" Hematology Reports 7, no. 4: 6107. https://doi.org/10.4081/hr.2015.6107

APA Style

Fernández, C., Lario, A., Forés Cachón, R., & Cabrera, R. (2015). Eculizumab Treatment in a Patient with Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy and Steroid-Refractory Acute Graft Versus Host Disease. Hematology Reports, 7(4), 6107. https://doi.org/10.4081/hr.2015.6107

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