title: Refractory atypical hemolytic uremic syndrome with monoclonal gammopathy responsive to bortezomib-based therapy.
abstract: Atypical hemolytic uremic syndrome (aHUS) is a relatively rare disorder described by the triad of hemolytic anemia, thrombocytopenia, and renal failure. Atypical HUS could be genetic, acquired, or idiopathic (without known genetic changes or environmental triggers). Monoclonal protein has uncommonly been reported as a cause of microangiopathic hemolytic anemia (MAHA). We report a 59-year-old white man who presented with acute kidney injury (AKI) with MAHA and was given a diagnosis of aHUS with monoclonal gammopathy. His kidney function and proteinuria worsened with persistent hemolysis despite eculizumab and later cyclophosphamide and prednisone treatment. He responded well to VRD (bortezomib, lenalidomide, and dexamethasone) regimen. Renal function, proteinuria, and hemolysis all improved, and he was been in remission for more than 15 months. To our knowledge, this is the first report of successful treatment with bortezomib-based regimen for a patient with aHUS and monoclonal protein refractory to eculizumab therapy.
authors.forename: Wisit
authors.initials: W
authors.lastname: Cheungpasitporn
authors.forename: Nelson
authors.initials: N
authors.lastname: Leung
authors.forename: Sanjeev
authors.initials: S
authors.lastname: Sethi
authors.forename: Morie A
authors.initials: MA
authors.lastname: Gertz
authors.forename: Fernando C
authors.initials: FC
authors.lastname: Fervenza
mesh_terms: D058186:Acute Kidney Injury / Q000188:drug therapy; D061067:Antibodies, Monoclonal, Humanized; D065766:Atypical Hemolytic Uremic Syndrome / Q000188:drug therapy*; D001897:Boronic Acids / Q000627:therapeutic use*; D000069286:Bortezomib; D006801:Humans; D008297:Male; D008875:Middle Aged; D010265:Paraproteinemias / Q000188:drug therapy*; D011719:Pyrazines / Q000627:therapeutic use*
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D001897:Boronic Acids; D011719:Pyrazines; D000069286:Bortezomib; C481642:eculizumab