BACKGROUND: Atypical 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. METHODS: Here, 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. RESULTS: We 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. CONCLUSION: Monitoring complement activity allows a safe reduction in the frequency of ECU administration in aHUS while keeping the disease in remission.
Complement functional tests for monitoring eculizumab treatment in patients with atypical hemolytic uremic syndrome: an update / G. Ardissino, F. Tel, M. Sgarbanti, D. Cresseri, A. Giussani, S. Griffini, E. Grovetto, I. Possenti, M. Perrone, S. Testa, F. Paglialonga, P. Messa, M. Cugno. - In: PEDIATRIC NEPHROLOGY. - ISSN 0931-041X. - 33:3(2018 Mar), pp. 457-461.
|Titolo:||Complement functional tests for monitoring eculizumab treatment in patients with atypical hemolytic uremic syndrome: an update|
|Parole Chiave:||aHUS; Complement activity; Eculizumab; Maintenance; Remission; Nephrology; Pediatrics, Perinatology and Child Health|
|Settore Scientifico Disciplinare:||Settore MED/09 - Medicina Interna|
Settore MED/14 - Nefrologia
|Data di pubblicazione:||mar-2018|
|Data ahead of print / Data di stampa:||18-ott-2017|
|Digital Object Identifier (DOI):||http://dx.doi.org/10.1007/s00467-017-3813-2|
|Appare nelle tipologie:||01 - Articolo su periodico|