Rituximab for very low dose steroid-dependent nephrotic syndrome in children: a randomized controlled study.
Pediatr Nephrol
; 35(8): 1437-1444, 2020 08.
Article
em En
| MEDLINE
| ID: mdl-32232637
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 (EudraCT2008-004486-26).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.Palavras-chave
Texto completo:
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Base de dados:
MEDLINE
Assunto principal:
Rituximab
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Anticorpos Monoclonais
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Síndrome Nefrótica
Tipo de estudo:
Clinical_trials
Limite:
Child
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Child, preschool
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Female
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Humans
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Male
Idioma:
En
Ano de publicação:
2020
Tipo de documento:
Article