title: Low-dose ofatumumab for rituximab-resistant nephrotic syndrome.
abstract: Drug resistant idiopathic nephrotic syndrome (DRNS) remains a therapeutic dilemma. In this pilot study, the efficacy of the new fully humanised, anti-CD20 monoclonal antibody ofatumumab was tested in 4 children with persistence of proteinuria for at least 12 months in spite of a full drug approach (including rituximab). We used a low-dose 2-infusion ofatumumab model (300+700 mg/1.73 m(2) 2 weeks apart) using specified premedication. Transient normalisation of proteinuria (persisting for 2 months) was achieved in 1 child while another presented stable remission after 12 months; both had normal renal function. The outcome was not modified in the remaining 2 children presenting an impaired renal function. These results demonstrate that low-dose ofatumumab may induce remittance of proteinuria in children with a long story of DRNS and normal renal function. Further studies are needed to test whether higher doses of ofatumumab can also modify proteinuria in patients with impaired renal function.
authors.affiliation: Division of Nephrology Dialysis and Transplantation, Istituto G Gaslini, Genoa, Italy.
authors.forename: Alice
authors.initials: A
authors.lastname: Bonanni
authors.affiliation: Division of Nephrology Dialysis and Transplantation, Istituto G Gaslini, Genoa, Italy.
authors.forename: Roberta
authors.initials: R
authors.lastname: Rossi
authors.affiliation: Division of Nephrology and Dialysis, Ospedale San Martino, Oristano, Italy.
authors.forename: Corrado
authors.initials: C
authors.lastname: Murtas
authors.affiliation: Division of Nephrology Dialysis and Transplantation, Istituto G Gaslini, Genoa, Italy.
authors.forename: Gian Marco
authors.initials: GM
authors.lastname: Ghiggeri
mesh_terms: D000293:Adolescent; D000911:Antibodies, Monoclonal / Q000008:administration & dosage*; D061067:Antibodies, Monoclonal, Humanized; D002648:Child; D016572:Cyclosporine / Q000008:administration & dosage; D004351:Drug Resistance; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D005923:Glomerulosclerosis, Focal Segmental / Q000175:diagnosis / Q000188:drug therapy; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage; D007262:Infusions, Intravenous / Q000379:methods; D008297:Male; D009173:Mycophenolic Acid / Q000008:administration & dosage / Q000031:analogs & derivatives; D009404:Nephrotic Syndrome / Q000188:drug therapy*; D010865:Pilot Projects; D000069283:Rituximab / Q000494:pharmacology*; D016559:Tacrolimus / Q000008:administration & dosage; D016896:Treatment Outcome
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D007166:Immunosuppressive Agents; D000069283:Rituximab; D016572:Cyclosporine; D009173:Mycophenolic Acid; C527517:ofatumumab; D016559:Tacrolimus