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http://hdl.handle.net/2445/112242
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DC Field | Value | Language |
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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.accessioned | 2017-06-12T11:10:19Z | - |
dc.date.available | 2017-06-12T11:10:19Z | - |
dc.date.issued | 2016-03-21 | - |
dc.identifier.issn | 0272-6386 | - |
dc.identifier.uri | http://hdl.handle.net/2445/112242 | - |
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. | - |
dc.format.extent | 10 p. | - |
dc.format.mimetype | application/pdf | - |
dc.language.iso | eng | - |
dc.publisher | Elsevier | - |
dc.relation.isformatof | Reproducció del document publicat a: https://doi.org/10.1053/j.ajkd.2015.12.034 | - |
dc.relation.ispartof | American Journal of Kidney Diseases, 2016, vol. 68, num. 1, p. 84-93 | - |
dc.relation.uri | https://doi.org/10.1053/j.ajkd.2015.12.034 | - |
dc.rights | cc-by-nc-nd (c) Fakhouri et al., 2016 | - |
dc.rights.uri | http://creativecommons.org/licenses/by-nc-nd/3.0/es | - |
dc.source | Articles publicats en revistes (Medicina) | - |
dc.subject.classification | Assaigs clínics | - |
dc.subject.classification | Malalties del ronyó | - |
dc.subject.classification | Hematologia | - |
dc.subject.classification | Diàlisi | - |
dc.subject.other | Clinical trials | - |
dc.subject.other | Kidney diseases | - |
dc.subject.other | Hematology | - |
dc.subject.other | 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.identifier.idgrec | 663270 | - |
dc.date.updated | 2017-06-12T11:10:19Z | - |
dc.rights.accessRights | info:eu-repo/semantics/openAccess | - |
dc.identifier.pmid | 27012908 | - |
Appears in Collections: | Articles publicats en revistes (Medicina) |
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663270.pdf | 477.49 kB | Adobe PDF | View/Open |
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