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Terminal Complement...
Terminal Complement Inhibitor Eculizumab in Atypical Hemolytic-Uremic Syndrome
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Legendre, C. M. (author)
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Licht, C. (author)
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Muus, P. (author)
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Greenbaum, L. A. (author)
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Babu, S. (author)
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Bedrosian, C. (author)
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Bingham, C. (author)
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Cohen, D. J. (author)
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Delmas, Y. (author)
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Douglas, K. (author)
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Eitner, F. (author)
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Feldkamp, T. (author)
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Fouque, D. (author)
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Furman, R. R. (author)
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Gaber, O. (author)
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- Herthelius, M. (author)
- Karolinska Institutet
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Hourmant, M. (author)
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- Karpman, Diana (author)
- Lund University,Lunds universitet,Pediatrik, Lund,Sektion V,Institutionen för kliniska vetenskaper, Lund,Medicinska fakulteten,Pediatrisk nefrologi,Forskargrupper vid Lunds universitet,Paediatrics (Lund),Section V,Department of Clinical Sciences, Lund,Faculty of Medicine,Pediatric Nephrology,Lund University Research Groups
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Lebranchu, Y. (author)
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Mariat, C. (author)
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Menne, J. (author)
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Moulin, B. (author)
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Nuernberger, J. (author)
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Ogawa, M. (author)
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Remuzzi, G. (author)
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Richard, T. (author)
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Sberro-Soussan, R. (author)
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Severino, B. (author)
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Sheerin, N. S. (author)
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Trivelli, A. (author)
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Zimmerhackl, L. B. (author)
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Goodship, T. (author)
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Loirat, C. (author)
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(creator_code:org_t)
- 2013
- 2013
- English.
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In: New England Journal of Medicine. - 0028-4793 .- 1533-4406. ; 368:23, s. 2169-2181
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https://portal.resea... (primary) (free)
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http://dx.doi.org/10...
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https://lup.lub.lu.s...
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https://doi.org/10.1...
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http://kipublication...
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Abstract
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- Background Atypical hemolytic-uremic syndrome is a genetic, life-threatening, chronic disease of complement-mediated thrombotic microangiopathy. Plasma exchange or infusion may transiently maintain normal levels of hematologic measures but does not treat the underlying systemic disease. Methods We conducted two prospective phase 2 trials in which patients with atypical hemolytic-uremic syndrome who were 12 years of age or older received eculizumab for 26 weeks and during long-term extension phases. Patients with low platelet counts and renal damage (in trial 1) and those with renal damage but no decrease in the platelet count of more than 25% for at least 8 weeks during plasma exchange or infusion (in trial 2) were recruited. The primary end points included a change in the platelet count (in trial 1) and thrombotic microangiopathy event-free status (no decrease in the platelet count of >25%, no plasma exchange or infusion, and no initiation of dialysis) (in trial 2). Results A total of 37 patients (17 in trial 1 and 20 in trial 2) received eculizumab for a median of 64 and 62 weeks, respectively. Eculizumab resulted in increases in the platelet count; in trial 1, the mean increase in the count from baseline to week 26 was 73x10(9) per liter (P<0.001). In trial 2, 80% of the patients had thrombotic microangiopathy event-free status. Eculizumab was associated with significant improvement in all secondary end points, with continuous, time-dependent increases in the estimated glomerular filtration rate (GFR). In trial 1, dialysis was discontinued in 4 of 5 patients. Earlier intervention with eculizumab was associated with significantly greater improvement in the estimated GFR. Eculizumab was also associated with improvement in health-related quality of life. No cumulative toxicity of therapy or serious infection-related adverse events, including meningococcal infections, were observed through the extension period. Conclusions Eculizumab inhibited complement-mediated thrombotic microangiopathy and was associated with significant time-dependent improvement in renal function in patients with atypical hemolytic-uremic syndrome.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Pediatrik (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Pediatrics (hsv//eng)
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- By the author/editor
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Legendre, C. M.
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Licht, C.
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Muus, P.
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Greenbaum, L. A.
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Babu, S.
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Bedrosian, C.
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show more...
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Bingham, C.
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Cohen, D. J.
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Delmas, Y.
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Douglas, K.
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Eitner, F.
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Feldkamp, T.
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Fouque, D.
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Furman, R. R.
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Gaber, O.
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Herthelius, M.
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Hourmant, M.
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Karpman, Diana
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Lebranchu, Y.
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Mariat, C.
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Menne, J.
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Moulin, B.
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Nuernberger, J.
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Ogawa, M.
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Remuzzi, G.
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Richard, T.
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Sberro-Soussan, ...
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Severino, B.
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Sheerin, N. S.
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Trivelli, A.
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Zimmerhackl, L. ...
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Goodship, T.
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Loirat, C.
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show less...
- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Pediatrics
- Articles in the publication
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New England Jour ...
- By the university
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Lund University
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Karolinska Institutet