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Severe transplant‐associated thrombotic microangiopathy in patients with hemoglobinopathies
Authors:Rajinder P.S. Bajwa  Edwin M. Horwitz  Jeffery J. Auletta  Hemalatha G. Rangarajan
Affiliation:1. Department of Pediatric Hematology, Oncology and Bone Marrow Transplant, Nationwide Children's Hospital, Columbus, Ohio;2. Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio;3. Department of Pediatric Infectious Diseases, Nationwide Children's Hospital, Columbus, Ohio
Abstract:Incidence and severity of transplant‐associated thrombotic microangiopathy (TA‐TMA) in patients with hemoglobinopathies receiving hematopoietic cell transplant is unknown. We report the outcomes for two patients with TA‐TMA who received eculizumab. A 2.5‐year‐old male with sickle cell disease developed TA‐TMA‐associated pericardial tamponade, severe hypertension, and acute kidney injury 2 months after transplant. A 7‐year‐old female with β‐thalassemia major developed TA‐TMA‐related acute kidney injury, severe hypertension, and seizures at 6 months after transplant. Both patients progressed to chronic kidney disease (CKD). In patients with hemoglobinopathies, preexisting endothelial dysfunction may place them at a greater risk for TA‐TMA and subsequent CKD.
Keywords:eculizumab  hemoglobinopathies  transplant‐associated thrombotic microangiopathy
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