Rituximab for minimal change disease in adults: long-term follow-up.

BACKGROUND: Minimal change nephropathy or disease (MCD) accounts for 10-15% of cases of the nephrotic syndrome in adults with frequent relapses occurring in up to 25% of cases. The drug of choice is glucocorticoids (GCs), but GC-dependence is seen in 25-30%. Treatment with rituximab has been found to be effective in relapsing and GC-dependent cases, but little data are available regarding long-term outcome in adults.

PATIENTS: We present nine female and seven male patients, ranging from 19 to 73 years of age with multirelapsing, GC-dependent or GC-resistant disease with a kidney biopsy consistent with MCD. Twelve patients were steroid-dependent with a lowest daily GC dose between 5 and 20 mg/day.

TREATMENT AND OUTCOMES: Rituximab with a total dose 1000-2800 mg divided in two to four doses was given together with GC achieving B-cell depletion before the second dose. No major side-effects occurred. Thirteen of the patients responded with complete remission enabling discontinuation or tapering of GC significantly below levels, where relapses had occurred in the past (P < 0.001). Two patients reached partial remission and one had no response to therapy. Follow-up was 12-70 months (median 44). Eight patients have remained in remission, whereas relapses occurred in seven patients after 9-28 months with repeated rituximab treatment in four of these.

CONCLUSIONS: Our study reinforces the role of rituximab as a GC-sparing agent in the challenging GC-dependent and multirelapsing MCD patients. In this emerging therapeutic field randomized studies with extended follow-up will add important information regarding optimal treatment, relapse and safety.