Abstract

Rituximab (RTX), a specific antibody to human CD20, has been successfully used to treat intractable nephrotic syndrome (NS). Recent studies have suggested a direct effect of RTX on podocytes by targeting sphingomyelinase phosphodiesterase acid-like 3b (SMPDL-3b). Thus, we examined the urinary excretion of SMPDL-3b as well as its immunoreactivity in biopsy specimens from children with intractable NS. Urine samples from six patients (five with minimal-change NS and one with focal segmental glomerulosclerosis) and from four healthy adults were examined. Glomerular immunoreactivity and urinary excretion of SMPDL3b in proteinuric NS patients decreased compared with controls. Interestingly, urine samples obtained from the same patients at the remission stage after RTX treatment showed an increase in urinary SMPDL-3b excretion compared with the proteinuric stage. Urinary excretion level of SMPDL-3b could thus be used to predict the clinical efficacy of RTX treatment in NS patients.

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