[en] BACKGROUND: 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. STUDY DESIGN: Open-label single-arm phase 2 trial. SETTING & PARTICIPANTS: Patients 18 years or older with aHUS (platelet count <150 x 10(3)/muL, hemoglobin /=1.5 x upper limit of normal [ULN], and serum creatinine >/= ULN) were included in this multicenter multinational study. INTERVENTION: Intravenous eculizumab (900mg/wk for 4 weeks, 1,200mg at week 5 and then every 2 weeks) for 26 weeks. OUTCOMES & MEASUREMENTS: Primary end point was complete TMA response within 26 weeks, defined as hematologic normalization (platelet count >/=150 x 10(3)/muL, LDH
Disciplines :
Urology & nephrology
Author, co-author :
Fakhouri, Fadi
Hourmant, Maryvonne
Campistol, Josep M.
Cataland, Spero R.
Espinosa, Mario
Gaber, A. Osama
Menne, Jan
Minetti, Enrico E.
Provot, Francois
Rondeau, Eric
Ruggenenti, Piero
WEEKERS, Laurent ; Centre Hospitalier Universitaire de Liège - CHU > Service de néphrologie
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