Rituximab for very low dose steroid-dependent nephrotic syndrome in children: a randomized controlled study
- 30 March 2020
- journal article
- research article
- Published by Springer Science and Business Media LLC in Pediatric Nephrology
- Vol. 35 (8), 1437-1444
- https://doi.org/10.1007/s00467-020-04540-4
Abstract
Background Steroid-dependent nephrotic syndrome (SDNS) carries a high risk of toxicity from steroids or steroid-sparing agents. This open-label, randomized controlled trial was designed to test whether the monoclonal antibody rituximab is non-inferior to steroids in maintaining remission in juvenile forms of SDNS and how long remission may last (EudraCT:2008-004486-26). Methods We enrolled 30 children 4–15 years who had developed SDNS 6–12 months before and were maintained in remission with low prednisone doses (0.1–0.4 mg/Kg/day). Participants were randomized following a non-inferiority design to continue prednisone alone (n 15, controls) or to add a single intravenous infusion of rituximab (375 mg/m2, n 15 intervention). Prednisone was tapered in both arms after 1 month. Children assigned to the control arm were allowed to receive rituximab to treat disease relapse. Results Proteinuria increased at 3 months in the prednisone group (from 0.14 to 1.5 g/day) (p < 0.001) and remained unchanged in the rituximab group (0.14 g/day). Fourteen children in the control arm relapsed within 6 months. Thirteen children assigned to rituximab (87%) were still in remission at 1 year and 8 (53%) at 4 years. Responses were similar in children of the control group who received rituximab to treat disease relapse. We did not record significant adverse events. Conclusions Rituximab was non-inferior to steroids for the treatment of juvenile SDNS. One in two children remains in remission at 4 years following a single infusion of rituximab, without significant adverse events. Further studies are needed to clarify the superiority of rituximab over low-dose corticosteroid as a treatment of SDNS.Keywords
Funding Information
- Ministero della Salute (RC2019, PE-2016-02361576)
- Compagnia di San Paolo (ROL98-49)
This publication has 16 references indexed in Scilit:
- Efficacy and safety of mycophenolate mofetil versus levamisole in frequently relapsing nephrotic syndrome: an open-label randomized controlled trialKidney International, 2018
- Efficacy of Rituximab vs Tacrolimus in Pediatric Corticosteroid-Dependent Nephrotic SyndromeJAMA Pediatrics, 2018
- Adverse events linked with the use of chimeric and humanized anti‐CD20 antibodies in children with idiopathic nephrotic syndromeBritish Journal of Clinical Pharmacology, 2018
- A randomized clinical trial indicates that levamisole increases the time to relapse in children with steroid-sensitive idiopathic nephrotic syndromeKidney International, 2017
- Anti-CD20 Antibodies for Idiopathic Nephrotic Syndrome in ChildrenClinical Journal of the American Society of Nephrology, 2016
- Rituximab in Children with Steroid-Dependent Nephrotic SyndromeJournal of the American Society of Nephrology, 2015
- The KDIGO practice guideline on glomerulonephritis: reading between the (guide)lines—application to the individual patientKidney International, 2012
- Corticosteroid therapy for nephrotic syndrome in childrenPublished by Wiley ,2005
- Cyclosporine in patients with steroid-resistant nephrotic syndrome: an open-label, nonrandomized, retrospective studyClinical Therapeutics, 2004
- Treatment of nephrotic syndrome in children and controlled trials.Nephrology Dialysis Transplantation, 2003