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Complement functional tests for monitoring eculizumab treatment in patients with atypical hemolytic uremic syndrome: an update

Authors
  • Ardissino, Gianluigi1
  • Tel, Francesca1
  • Sgarbanti, Martina2
  • Cresseri, Donata3
  • Giussani, Antenore4
  • Griffini, Samantha5
  • Grovetto, Elena5
  • Possenti, Ilaria1
  • Perrone, Michela1
  • Testa, Sara2
  • Paglialonga, Fabio2
  • Messa, Piergiorgio3
  • Cugno, Massimo5
  • 1 Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Center for HUS Prevention, Control and Management at the Pediatric and Dialysis Unit, v. Commenda, 9, Milan, 20122, Italy , Milan (Italy)
  • 2 Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Center for HUS Prevention, Control and Management at the Molecular Biology Laboratory, Milan, Italy , Milan (Italy)
  • 3 Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Center for HUS Prevention, Control and Management at the Nephrology Unit, Milan, Italy , Milan (Italy)
  • 4 Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Center for HUS Prevention, Control and Management at the Kidney Transplant Unit, Milan, Italy , Milan (Italy)
  • 5 Università degli Studi di Milano, Center for HUS Prevention, Control, and Management at Medicina Interna, Dipartimento di Fisiopatologia Medico-Chirurgica e dei Trapianti, Milan, Italy , Milan (Italy)
Type
Published Article
Journal
Pediatric Nephrology
Publisher
Springer-Verlag
Publication Date
Oct 18, 2017
Volume
33
Issue
3
Pages
457–461
Identifiers
DOI: 10.1007/s00467-017-3813-2
Source
Springer Nature
Keywords
License
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Abstract

BackgroundAtypical hemolytic uremic syndrome (aHUS) is a thrombotic microangiopathy (TMA) characterized by platelet consumption, hemolysis, and organ damage. Eculizumab (ECU), a humanized antibody that blocks complement activity, has been successfully used in aHUS, but the best treatment schedule is not yet clear.MethodsHere, we report our experience with ECU maintenance treatment and the interval between subsequent doses being extended based on global classical complement pathway (CCP) activity aimed at <30% for maintaining aHUS into remission.ResultsWe report on 38 patients with aHUS, 13 children, 21 female, with a median age of 25.0 years (range 0.5–60) at disease onset treated with ECU standard schedule for a median of 2.6 months (range 0.4–24.6). Once stable TMA remission was obtained, the interval between ECU doses was extended based on complement function, with a target CCP activity of <30%. With this approach, 22 patients regularly receive ECU infusion every 28 days and 16 every 21. During a median observation period on ECU, an extended interval of 26.9 months (range 0.8–80.9), with a cumulative observation period of 1,208 months, none of the patients relapsed.ConclusionMonitoring complement activity allows a safe reduction in the frequency of ECU administration in aHUS while keeping the disease in remission.

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