title: A new paradigm: Diagnosis and management of HSCT-associated thrombotic microangiopathy as multi-system endothelial injury.
abstract: Hematopoietic stem cell transplantation (HSCT)-associated thrombotic microangiopathy (TA-TMA) is now a well-recognized and potentially severe complication of HSCT that carries a high risk of death. In those who survive, TA-TMA may be associated with long-term morbidity and chronic organ injury. Recently, there have been new insights into the incidence, pathophysiology, and management of TA-TMA. Specifically, TA-TMA can manifest as a multi-system disease occurring after various triggers of small vessel endothelial injury, leading to subsequent tissue damage in different organs. While the kidney is most commonly affected, TA-TMA involving organs such as the lung, bowel, heart, and brain is now known to have specific clinical presentations. We now review the most up-to-date research on TA-TMA, focusing on the pathogenesis of endothelial injury, the diagnosis of TA-TMA affecting the kidney and other organs, and new clinical approaches to the management of this complication after HSCT.
authors.affiliation: Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, USA. Electronic address: sonata.jodele@cchmc.org.
authors.forename: Sonata
authors.initials: S
authors.lastname: Jodele
authors.affiliation: Division of Nephrology, The Children's Hospital of Philadelphia, USA.
authors.forename: Benjamin L
authors.initials: BL
authors.lastname: Laskin
authors.affiliation: Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, USA.
authors.forename: Christopher E
authors.initials: CE
authors.lastname: Dandoy
authors.affiliation: Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, USA.
authors.forename: Kasiani C
authors.initials: KC
authors.lastname: Myers
authors.affiliation: Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, USA.
authors.forename: Javier
authors.initials: J
authors.lastname: El-Bietar
authors.affiliation: Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, USA.
authors.forename: Stella M
authors.initials: SM
authors.lastname: Davies
authors.affiliation: Division of Nephrology and Hypertension, Cincinnati Children's Hospital Medical Center, USA.
authors.forename: Jens
authors.initials: J
authors.lastname: Goebel
authors.affiliation: Division of Nephrology and Hypertension, Cincinnati Children's Hospital Medical Center, USA.
authors.forename: Bradley P
authors.initials: BP
authors.lastname: Dixon
mesh_terms: D003165:Complement System Proteins / Q000276:immunology; D019468:Disease Management; D004730:Endothelium, Vascular / Q000276:immunology / Q000378:metabolism / Q000473:pathology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D015994:Incidence; D012307:Risk Factors; D057049:Thrombotic Microangiopathies / Q000175:diagnosis* / Q000453:epidemiology / Q000209:etiology* / Q000628:therapy
chemical_list: D003165:Complement System Proteins
keywords: Complement activation; Eculizumab; Hematopoietic cell transplant; Kidney disease; TA-TMA; Thrombotic microangiopathy