Eculizumab in anti-factor h antibodies associated with atypical hemolytic uremic syndrome.
Atypical hemolytic uremic syndrome (aHUS) is a life-threatening multisystemic condition often leading to end-stage renal failure. It results from an increased activation of the alternative pathway of the complement system due to mutations of genes coding for inhibitors of this pathway or from autoantibodies directed against them. Eculizumab is a monoclonal antibody directed against complement component C5 and inhibiting the activation of the effector limb of the complement system. Its efficacy has already been demonstrated in aHUS. The present article reports for the first time the use of eculizumab in a patient presenting with aHUS associated with circulating anti-complement Factor H autoantibodies and complicated by cardiac and neurologic symptoms. Our observation highlights the efficacy of eculizumab in this form of aHUS not only on renal symptoms but also on the extrarenal symptoms. It also suggests that eculizumab should be used very promptly after aHUS presentation to prevent life-threatening complications and to reduce the risk of chronic disabilities. To obtain a complete inhibition of the effector limb activation, the advised dosage must be respected. After this initial therapy in the autoimmune aHUS form, a long-term immunosuppressive treatment should be considered, to prevent relapses by reducing anti-complement Factor H autoantibody plasma levels.
Departments of Pediatric Nephrology, and.
Benedetta
B
Diamante Chiodini
Departments of Pediatric Nephrology, andDepartment of Pediatric Nephrology, Emma Children's Hospital-Academic Medical Centre, University of Amsterdam, Amsterdam, the Netherlands;
Jean-Claude
JC
Davin
Department of Immunology, CHU Brugmann Hospital (ULB), Brussels, Belgium; and.
Francis
F
Corazza
Pediatric Cardiology, Hôpital Universitaire des Enfants-Reine Fabiola, Université Libre de Bruxelles (ULB), Brussels, Belgium;
Karim
K
Khaldi
Center for Human Genetics, Université Catholique de Louvain, Brussels, Belgium.
Karin
K
Dahan
Departments of Pediatric Nephrology, and.
Khalid
K
Ismaili
Departments of Pediatric Nephrology, and brigitte.adams@huderf.be.
Brigitte
B
Adams
D061067:Antibodies, Monoclonal, Humanized / Q000009:adverse effects / Q000627:therapeutic use*; D001323:Autoantibodies / Q000097:blood*; D001921:Brain / Q000473:pathology; D002545:Brain Ischemia / Q000175:diagnosis / Q000188:drug therapy / Q000276:immunology; D002648:Child; D017242:Complement Factor H / Q000276:immunology*; D003404:Creatinine / Q000097:blood; D004305:Dose-Response Relationship, Drug; D061345:Early Medical Intervention; D004562:Electrocardiography; D005500:Follow-Up Studies; D006452:Hemoglobinometry; D006463:Hemolytic-Uremic Syndrome / Q000175:diagnosis / Q000188:drug therapy* / Q000276:immunology*; D006801:Humans; D007676:Kidney Failure, Chronic / Q000175:diagnosis / Q000188:drug therapy / Q000276:immunology; D019344:Lactic Acid / Q000097:blood; D008134:Long-Term Care; D008279:Magnetic Resonance Imaging; D008297:Male; D017202:Myocardial Ischemia / Q000175:diagnosis / Q000188:drug therapy / Q000276:immunology; D006435:Renal Dialysis; D055502:Secondary Prevention
D061067:Antibodies, Monoclonal, Humanized; D001323:Autoantibodies; D019344:Lactic Acid; D017242:Complement Factor H; C481642:eculizumab; D003404:Creatinine
anti–Factor H autoantibodies; atypical hemolytic uremic syndrome; cardiomyopathy; eculizumab; multisystemic aHUS; neurologic involvement; thrombotic microangiopathy