Eculizumab as first-line therapy for atypical hemolytic uremic syndrome.
Atypical hemolytic uremic syndrome (aHUS) is a genetic, life-threatening, chronic disease that can affect patients of all ages. aHUS is caused by uncontrolled complement activation due to genetic defects of complement regulation. Plasma exchange or infusion has been used to manage aHUS and may transiently maintain hematologic variables in some patients, but as the underlying complement dysregulation persists, end-stage renal disease or death occurs in 33% to 40% of patients during the first clinical manifestation. Here we present a pediatric case showing that first-line eculizumab treatment successfully blocked the progression of thrombotic microangiopathy in aHUS.
Klinik für Kinder- und Jugendmedizin, Pädiatrische Intensivstation, Goethe-Universität, Frankfurt/Main, Germany; martin.christmann@kispi.uzh.ch.
Martin
M
Christmann
Clementine Kinderkrankenhaus, Pädiatrische Nephrologie, Frankfurt/Main, Germany; and.
Matthias
M
Hansen
Bioscientia Institut für Medizinische Diagnostik GmbH, Humangenetik, Ingelheim, Germany.
Carsten
C
Bergmann
Klinik für Kinder- und Jugendmedizin, Pädiatrische Intensivstation, Goethe-Universität, Frankfurt/Main, Germany;
Dirk
D
Schwabe
Klinik für Kinder- und Jugendmedizin, Pädiatrische Intensivstation, Goethe-Universität, Frankfurt/Main, Germany;
Jörg
J
Brand
Klinik für Kinder- und Jugendmedizin, Pädiatrische Intensivstation, Goethe-Universität, Frankfurt/Main, Germany;
Wilfried
W
Schneider
D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005260:Female; D006463:Hemolytic-Uremic Syndrome / Q000175:diagnosis / Q000188:drug therapy* / Q000276:immunology*; D006801:Humans; D008297:Male; D006435:Renal Dialysis
D061067:Antibodies, Monoclonal, Humanized; C481642:eculizumab
atypical hemolytic uremic syndrome; complement; eculizumab; infant