RT Journal Article
T1 Terminal Complement Inhibitor Eculizumab in Adult Patients With Atypical Hemolytic Uremic Syndrome: A Single-Arm, Open-Label Trial.
A1 Fakhouri, Fadi
A1 Hourmant, Maryvonne
A1 Campistol, Josep M
A1 Cataland, Spero R
A1 Espinosa, Mario
A1 Gaber, A Osama
A1 Menne, Jan
A1 Minetti, Enrico E
A1 Provôt, François
A1 Rondeau, Eric
A1 Ruggenenti, Piero
A1 Weekers, Laurent E
A1 Ogawa, Masayo
A1 Bedrosian, Camille L
A1 Legendre, Christophe M
K1 Eculizumab
K1 Soliris
K1 TMA response
K1 adults
K1 atypical hemolytic uremic syndrome (aHUS)
K1 clinical trial
K1 hematologic normalization
K1 hemoglobin
K1 kidney disease
K1 lactate dehydrogenase (LDH)
K1 platelet count
K1 renal function
K1 terminal complement inhibitor
K1 thrombotic microangiopathy (TMA)
AB 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.
YR 2016
FD 2016-03-21
LK http://hdl.handle.net/10668/9943
UL http://hdl.handle.net/10668/9943
LA en
DS RISalud
RD Apr 7, 2025