Rituximab is a safe and effective long-term treatment for children with steroid and calcineurin inhibitor-dependent idiopathic nephrotic syndrome

Pietro Ravani, Alessandro Ponticelli, Chiara Siciliano, Alessia Fornoni, Alberto Magnasco, Felice Sica, Monica Bodria, Gianluca Caridi, Changli Wei, Mirco Belingheri, Luciana Ghio, Sandra Merscher-Gomez, Alberto Edefonti, Andrea Pasini, Giovanni Montini, Corrado Murtas, Xiangyu Wang, Daniel Muruve, Augusto Vaglio, Davide MartoranaAntonello Pani, Francesco Scolari, Jochen Reiser, Gian M. Ghiggeri

Research output: Contribution to journalArticle

71 Scopus citations

Abstract

In children with idiopathic nephrotic syndrome, rituximab can maintain short-term remission with withdrawal of prednisone and calcineurin inhibitors. Long-term effects including the number of repeated infusions to maintain remission are unknown. To test this, we treated 46 consecutive children with idiopathic nephrotic syndrome lasting for at least 1 year (mean 6.3 years), maintained in remission with oral prednisone and calcineurin inhibitors. They received 1-5 rituximab courses during a median follow-up of 3 years. Oral agents were tapered after each infusion, and completely withdrawn within 45 days. Rituximab was well tolerated. Six-month probabilities of remission were 48% after the first infusion and 37% after subsequent infusions. One-and 2-year-remission probabilities were, respectively, 20 and 10%. Median time intervals between complete oral-agent withdrawal and relapse were 5.6 and 8.5 months, respectively, following the first and subsequent courses. The time to reconstitution of CD20 cells correlated with the duration of remission, but was not associated with variation in FcyR, CD20, or SMPDL-3B polymorphisms. Podocyte Src phosphorylation was normal. Thus, rituximab can be safely and repeatedly used as a prednisone and calcineurin inhibitor-sparing therapy in a considerable proportion of children with dependent forms of idiopathic nephrotic syndrome. Further study is needed to identify patients who will benefit most from rituximab therapy.

Original languageEnglish (US)
Pages (from-to)1025-1033
Number of pages9
JournalKidney international
Volume84
Issue number5
DOIs
StatePublished - Nov 2013

Keywords

  • clinical trial
  • nephrotic syndrome
  • pediatrics
  • proteinuria

ASJC Scopus subject areas

  • Nephrology

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    Ravani, P., Ponticelli, A., Siciliano, C., Fornoni, A., Magnasco, A., Sica, F., Bodria, M., Caridi, G., Wei, C., Belingheri, M., Ghio, L., Merscher-Gomez, S., Edefonti, A., Pasini, A., Montini, G., Murtas, C., Wang, X., Muruve, D., Vaglio, A., ... Ghiggeri, G. M. (2013). Rituximab is a safe and effective long-term treatment for children with steroid and calcineurin inhibitor-dependent idiopathic nephrotic syndrome. Kidney international, 84(5), 1025-1033. https://doi.org/10.1038/ki.2013.211