Fakhouri, FadiHourmant, MaryvonneCampistol, Josep MCataland, Spero REspinosa, MarioGaber, A OsamaMenne, JanMinetti, Enrico EProvôt, FrançoisRondeau, EricRuggenenti, PieroWeekers, Laurent EOgawa, MasayoBedrosian, Camille LLegendre, Christophe M2023-01-252023-01-252016-03-21http://hdl.handle.net/10668/9943Atypical 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.enAttribution-NonCommercial-NoDerivatives 4.0 Internationalhttp://creativecommons.org/licenses/by-nc-nd/4.0/EculizumabSolirisTMA responseadultsatypical hemolytic uremic syndrome (aHUS)clinical trialhematologic normalizationhemoglobinkidney diseaselactate dehydrogenase (LDH)platelet countrenal functionterminal complement inhibitorthrombotic microangiopathy (TMA)AdolescentAdultAgedAged, 80 and overAntibodies, Monoclonal, HumanizedAtypical Hemolytic Uremic SyndromeFemaleHumansMaleMiddle AgedProspective StudiesRemission InductionYoung Adultresearch article27012908open access10.1053/j.ajkd.2015.12.0341523-6838http://www.ajkd.org/article/S0272638616001591/pdf