Severe transplant-associated thrombotic microangiopathy in patients with hemoglobinopathies.
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.
Department of Pediatric Bone Marrow Transplant and Communicable Diseases, University of Michigan, Ann Arbor, Michigan.
Ghada A
GA
Abusin
Department of Pediatric Hematology, Oncology and Bone Marrow Transplant, Nationwide Children's Hospital, Columbus, Ohio.|Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio.
Rolla
R
Abu-Arja
Department of Pediatric Hematology, Oncology and Bone Marrow Transplant, Nationwide Children's Hospital, Columbus, Ohio.|Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio.
Rajinder P S
RPS
Bajwa
Department of Pediatric Hematology, Oncology and Bone Marrow Transplant, Nationwide Children's Hospital, Columbus, Ohio.|Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio.
Edwin M
EM
Horwitz
Department of Pediatric Hematology, Oncology and Bone Marrow Transplant, Nationwide Children's Hospital, Columbus, Ohio.|Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio.|Department of Pediatric Infectious Diseases, Nationwide Children's Hospital, Columbus, Ohio.
Jeffery J
JJ
Auletta
Department of Pediatric Hematology, Oncology and Bone Marrow Transplant, Nationwide Children's Hospital, Columbus, Ohio.|Department of Pediatrics, The Ohio State University College of Medicine, Columbus, Ohio.
Hemalatha G
HG
Rangarajan
D000755:Anemia, Sickle Cell / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D008297:Male; D051436:Renal Insufficiency, Chronic / Q000209:etiology; D057049:Thrombotic Microangiopathies / Q000209:etiology*; D017086:beta-Thalassemia / Q000628:therapy
eculizumab; hemoglobinopathies; transplant-associated thrombotic microangiopathy