Steroid-dependent nephrotic syndrome (SDNS) carries a high risk of toxicity from steroids or steroid-sparing agents. This open-label, noninferiority, randomized controlled trial at four sites in Italy tested whether rituximab is noninferior to steroids in maintaining remission in juvenile SDNS. We enrolled children age 1-16 years who had developed SDNS in the previous 6-12 months and were maintained in remission with high prednisone doses (≥0.7 mg/kg per day). We randomly assigned participants to continue prednisone alone for 1 month (control) or to add a single intravenous infusion of rituximab (375 mg/m(2); intervention). Prednisone was tapered in both groups after 1 month. For noninferiority, rituximab had to permit steroid withdrawal and maintain 3-month proteinuria (mg/m(2) per day) within a prespecified noninferiority margin of three times the levels among controls (primary outcome). We followed participants for ≥1 year to compare risk of relapse (secondary outcome). Fifteen children per group (21 boys; mean age, 7 years [range, 2.6-13.5 years]) were enrolled and followed for ≤60 months (median, 22 months). Three-month proteinuria was 42% lower in the rituximab group (geometric mean ratio, 0.58; 95% confidence interval, 0.18 to 1.95 [i.e., within the noninferiority margin of three times the levels in controls]). All but one child in the control group relapsed within 6 months; median time to relapse in the rituximab group was 18 months (95% confidence interval, 9 to 32 months). In the rituximab group, nausea and skin rash during infusion were common; transient acute arthritis occurred in one child. In conclusion, rituximab was noninferior to steroids for the treatment of juvenile SDNS.
Rituximab in children with steroid-dependent nephrotic syndrome : a multicenter, open-label, noninferiority, randomized controlled trial / P. Ravani, R. Rossi, A. Bonanni, R. Quinn, F. Sica, M. Bodria, A. Pasini, G. Montini, A. Edefonti, M. Belingheri, D. De Giovanni, G. Barbano, L. Degl'Innocenti, F. Scolari, L. Murer, J. Reiser, A. Fornoni, G. Ghiggeri. - In: JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY. - ISSN 1046-6673. - 26:9(2015 Sep), pp. 2259-2266.
|Titolo:||Rituximab in children with steroid-dependent nephrotic syndrome : a multicenter, open-label, noninferiority, randomized controlled trial|
|Parole Chiave:||nephrotic syndrome; primary glomerulonephritis; randomized controlled trials|
|Settore Scientifico Disciplinare:||Settore MED/38 - Pediatria Generale e Specialistica|
|Data di pubblicazione:||set-2015|
|Digital Object Identifier (DOI):||http://dx.doi.org/10.1681/ASN.2014080799|
|Appare nelle tipologie:||01 - Articolo su periodico|