dc.contributor |
Universitat de Barcelona |
dc.contributor.author |
Fakhouri, Fadi |
dc.contributor.author |
Hourmant, Maryvonne |
dc.contributor.author |
Campistol Plana, Josep M. |
dc.contributor.author |
Cataland, Spero R. |
dc.contributor.author |
Espinosa, Mario |
dc.contributor.author |
Gaber, A. Osama |
dc.contributor.author |
Menne, Jan |
dc.contributor.author |
Minetti, Enrico E. |
dc.contributor.author |
Provôt, François |
dc.contributor.author |
Rondeau, Eric |
dc.contributor.author |
Ruggenenti, Piero |
dc.contributor.author |
Weekers, Laurent E. |
dc.contributor.author |
Ogawa, Masayo |
dc.contributor.author |
Bedrosian, Camille L. |
dc.contributor.author |
Legendre, Christophe M. |
dc.date |
2017-06-12T11:10:19Z |
dc.date |
2017-06-12T11:10:19Z |
dc.date |
2016-03-21 |
dc.date |
2017-06-12T11:10:19Z |
dc.identifier.citation |
0272-6386 |
dc.identifier.citation |
663270 |
dc.identifier.uri |
http://hdl.handle.net/2445/112242 |
dc.format |
10 p. |
dc.format |
application/pdf |
dc.language.iso |
eng |
dc.publisher |
Elsevier |
dc.relation |
Reproducció del document publicat a: https://doi.org/10.1053/j.ajkd.2015.12.034 |
dc.relation |
American Journal of Kidney Diseases, 2016, vol. 68, num. 1, p. 84-93 |
dc.relation |
https://doi.org/10.1053/j.ajkd.2015.12.034 |
dc.rights |
cc-by-nc-nd (c) Fakhouri et al., 2016 |
dc.rights |
info:eu-repo/semantics/openAccess |
dc.rights |
http://creativecommons.org/licenses/by-nc-nd/3.0/es |
dc.subject |
Assaigs clínics |
dc.subject |
Malalties del ronyó |
dc.subject |
Hematologia |
dc.subject |
Diàlisi |
dc.subject |
Clinical trials |
dc.subject |
Kidney diseases |
dc.subject |
Hematology |
dc.subject |
Dialysis |
dc.title |
Terminal Complement Inhibitor Eculizumab in Adult Patients With Atypical Hemolytic Uremic Syndrome: A Single-Arm, Open-Label Trial |
dc.type |
info:eu-repo/semantics/article |
dc.type |
info:eu-repo/semantics/publishedVersion |
dc.description.abstract |
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 × 10(3)/μL, hemoglobin ≤ lower limit of normal, lactate dehydrogenase ≥1.5 × 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 × 10(3)/μL, LDH ≤ ULN), and preservation of kidney function (<25% serum creatinine increase from baseline), confirmed by 2 or more consecutive measurements obtained 4 or more weeks apart. RESULTS: 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<0.001). All 35 patients on baseline plasma exchange/plasma infusion discontinued by week 26. Of 24 patients requiring baseline dialysis, 5 recovered kidney function before eculizumab initiation and 15 of the remaining 19 (79%) discontinued dialysis during eculizumab treatment. No patients lost existing transplants. Quality-of-life measures were significantly improved. Two patients developed meningococcal infections; both recovered, and 1 remained on eculizumab treatment. LIMITATIONS: Single-arm open-label design. CONCLUSIONS: 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. |