Evidence map›Paper›PMID 25651368›Full record

Trial reportKidney international2015

Efficacy and safety of eculizumab in atypical hemolytic uremic syndrome from 2-year extensions of phase 2 studies.

Christoph Licht, Larry A Greenbaum, Petra Muus, Sunil Babu, Camille L Bedrosian, David J Cohen, Yahsou Delmas, Kenneth Douglas, Richard R Furman, Osama A Gaber and 8 more

Abstract readClinical Trial, Phase II
In one paragraph

Trial report in Kidney international, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 207 papers, 5 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
207citing papers in PubMed, 5 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

207 citing papers in PubMed, 5 syntheses or guidelines pooled it.

  1. Pooled it
  2. Interventions for atypical haemolytic uraemic syndrome.The Cochrane database of systematic reviews · 2021
    Pooled it
  3. Pooled it
  4. Pooled it
  5. Clinical guides for atypical hemolytic uremic syndrome in Japan.Clinical and experimental nephrology · 2016
    Guideline
  6. Trial
  7. Eculizumab monotherapy for NMOSD: Data from PREVENT and its open-label extension.Multiple sclerosis (Houndmills, Basingstoke, England) · 2022
    Trial
  8. Trial
  9. Trial
  10. Trial
  11. Trial
  12. Trial
  13. Safety and efficacy of eculizumab in the prevention of antibody-mediated rejection in living-donor kidney transplant recipients requiring desensitization therapy: A randomized trial.American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons · 2019
    Trial
  14. Safety and efficacy of eculizumab for the prevention of antibody-mediated rejection after deceased-donor kidney transplantation in patients with preformed donor-specific antibodies.American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons · 2019
    Trial
  15. Trial
  16. Article
  17. Article
  18. Article
  19. Article
  20. Updates in atypical hemolytic syndrome.Current opinion in pediatrics · 2026
    Review

147 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

18 authors.

Christoph LichtDivision of Nephrology and Program in Cell Biology, The Hospital for Sick Children, Toronto, Ontario, Canada.
Larry A GreenbaumEmory University and Children's Healthcare of Atlanta, Atlanta, Georgia, USA.
Petra MuusDepartment of Hematology, Radboud University Nijmegen Medical Centre, Nijmegen, The Netherlands.
Sunil BabuFort Wayne Medical Oncology and Hematology, Fort Wayne, Indiana, USA.
Camille L BedrosianAlexion Pharmaceuticals, Inc., Cheshire, Connecticut, USA.
David J CohenDepartment of Kidney and Pancreas Transplantation, Columbia University Medical Center, New York, New York, USA.
Yahsou DelmasDepartment of Nephrology, Centre Hospitalier Universitaire de Bordeaux, Bordeaux, France.
Kenneth DouglasBeatson West Scotland Cancer Centre, Glasgow, UK.
Richard R FurmanDivision of Hematology/Oncology, Weill Cornell Medical College, New York, New York, USA.
Osama A GaberDepartment of Surgery/Transplant Center, Houston Methodist Hospital, Houston, Texas, USA.
Timothy GoodshipDepartment of Renal Medicine, Newcastle University, Newcastle upon Tyne, UK.
Maria HertheliusDepartment of Clinical Science, Intervention and Technology (CLINTEC), H9, Karolinska University Hospital, Stockholm, Sweden.
Maryvonne HourmantDepartment of Nephrology, CHU Hotel Dieu-Nantes, Nantes, France.
Christophe M LegendreAdult Kidney Transplant Unit, Université Paris Descartes & Hôpital Necker, Paris, France.
Giuseppe RemuzziIstituto di Ricerche Farmacologiche Mario Negri, Bergamo, Italy.
Neil SheerinDepartment of Nephrology, Newcastle University, Newcastle upon Tyne, UK.
Antonella TrivelliDepartment of Nephrology, Istituto Giannina Gaslini, Genova, Italy.
Chantal LoiratDepartment of Pediatric Nephrology, Assistance Publique-Hôpitaux de Paris, Hôpital Robert-Debré, Paris, France.

Funding

Atlanta Clinical and Translational Science Institute (ACTSI) RenewalUL1TR000454 · NCATS · EMORY UNIVERSITY · PI STEPHENS, DAVID S · 2012 to 2016
$25.8M
NCATS NIH HHS UL1 TR000454NCATS NIH HHS UL1TR000454
6 · The paper itself

Abstract

Atypical hemolytic uremic syndrome (aHUS) is a rare, possibly life-threatening disease characterized by platelet activation, hemolysis and thrombotic microangiopathy (TMA) leading to renal and other end-organ damage. We originally conducted two phase 2 studies (26 weeks and 1 year) evaluating eculizumab, a terminal complement inhibitor, in patients with progressing TMA (trial 1) and those with long duration of aHUS and chronic kidney disease (trial 2). The current analysis assessed outcomes after 2 years (median eculizumab exposure 100 and 114 weeks, respectively). At all scheduled time points, eculizumab inhibited terminal complement activity. In trial 1 with 17 patients, the platelet count was significantly improved from baseline, and hematologic normalization was achieved in 13 patients at week 26, and in 15 patients at both 1 and 2 years. The estimated glomerular filtration rate (eGFR) was significantly improved compared with baseline and year 1. In trial 2 with 20 patients, TMA event-free status was achieved by 16 patients at week 26, 17 patients at year 1, and 19 patients at year 2. Criteria for hematologic normalization were met by 18 patients at each time point. Improvement of 15 ml/min per 1.73 m(2) or more in eGFR was achieved by 1 patient at week 26, 3 patients at 1 year, and 8 patients at 2 years. The mean change in eGFR was not significant compared with baseline, week 26, or year 1. Eculizumab was well tolerated, with no new safety concerns or meningococcal infections. Thus, a 2-year analysis found that the earlier clinical benefits achieved by eculizumab treatment of aHUS were maintained at 2 years of follow-up.

Indexed as

AdolescentAdultAgedAntibodies, Monoclonal, HumanizedAtypical Hemolytic Uremic SyndromeFemaleGlomerular Filtration RateHumansMaleMiddle AgedQuality of LifeTreatment OutcomeYoung AdultAntibodies, Monoclonal, Humanizedeculizumab

Identifiers

PMID25651368
PMCPMC4424817

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.