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Title: Terminal Complement Inhibitor Eculizumab in Adult Patients With Atypical Hemolytic Uremic Syndrome: A Single-Arm, Open-Label Trial.
Authors: Fakhouri, Fadi
Hourmant, Maryvonne
Campistol, Josep M
Cataland, Spero R
Espinosa, Mario
Gaber, A Osama
Menne, Jan
Minetti, Enrico E
Provôt, François
Rondeau, Eric
Ruggenenti, Piero
Weekers, Laurent E
Ogawa, Masayo
Bedrosian, Camille L
Legendre, Christophe M
Keywords: Eculizumab;Soliris;TMA response;adults;atypical hemolytic uremic syndrome (aHUS);clinical trial;hematologic normalization;hemoglobin;kidney disease;lactate dehydrogenase (LDH);platelet count;renal function;terminal complement inhibitor;thrombotic microangiopathy (TMA)
metadata.dc.subject.mesh: Adolescent
Aged, 80 and over
Antibodies, Monoclonal, Humanized
Atypical Hemolytic Uremic Syndrome
Middle Aged
Prospective Studies
Remission Induction
Young Adult
Issue Date: 21-Mar-2016
Abstract: Atypical hemolytic uremic syndrome (aHUS) is a rare genetic life-threatening disease of chronic uncontrolled complement activation leading to thrombotic microangiopathy (TMA) and severe end-organ damage. Eculizumab, a terminal complement inhibitor approved for aHUS treatment, was reported to improve hematologic and renal parameters in 2 prior prospective phase 2 studies. This is the largest prospective study of eculizumab in aHUS to date, conducted in an adult population. Open-label single-arm phase 2 trial. Patients 18 years or older with aHUS (platelet count  Intravenous eculizumab (900mg/wk for 4 weeks, 1,200mg at week 5 and then every 2 weeks) for 26 weeks. Primary end point was complete TMA response within 26 weeks, defined as hematologic normalization (platelet count ≥150 × 10(3)/μL, LDH ≤ ULN), and preservation of kidney function ( 41 patients were treated; 38 (93%) completed 26 weeks of treatment. 30 (73%) were included during their first TMA manifestation. 30 (73%) had complete TMA response. Platelet counts and estimated glomerular filtration rates increased from baseline (P Single-arm open-label design. Results highlight the benefits of eculizumab in adult patients with aHUS: improvement in hematologic, renal, and quality-of-life parameters; dialysis discontinuation; and transplant protection.
metadata.dc.identifier.doi: 10.1053/j.ajkd.2015.12.034
Appears in Collections:Producción 2020

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