Adalat S, Taylor J, Reid C, et al.
Efficacy of Rituximab in Steroid Dependent and Steroid Resistant
Nephrotic Syndrome in Children and Young Adults.
ASN Annual Meeting -- San Diego
J Am Soc Nephrol
(Nov) 20:416A 2009

Objectives. To explore the efficacy of rituximab in childhood steroid
dependent and steroid resistant nephrotic syndrome and its relationship with
CD20 B lymphocyte depletion.
Background. Rituximab, a CD20 specific B
lymphocyte depleting antibody may induce remission of minimal change disease
(MCND) and focal segmental glomerulosclerosis (FSGS). We tested the
hypothesis that this indicated a pathogenic role for B lymphocytes by
studying 20 cases of idiopathic NS in children and young adults pre- and post
rituximab for 36 months. Further immunological investigations were performed
to acquire clues as to the mechanism of action.
Methods. 20 cases, 4
female and 16 male were treated with rituximab. 10 were steroid resistant and
10 steroid dependent. Histological diagnoses were MCND 9; FSGS 10, C1q
nephropathy 1. The number of courses of rituximab/patient was 1-4. Median
duration of therapy prior to rituximab was 9 months (range 1-156 months).
Cases were examined for clinical response and immunological
alterations.
Results. 16/20 cases responded to the first course of
rituximab in 2 to 4 weeks. 3 non-responders had advanced disease, 1 did not
deplete B cells. No significant adverse side effects were noted. Nephrotic
remission was directly correlated with CD20 B cell depletion and relapse with
CD20 recovery. 90% of cases required re-treatment after 6
9 months. Re-emergence of steroid and/or
calcineurin inhibitor sensitivity was observed. No reduction in efficacy of
rituximab occurred on repeat dosing or alteration in measured clinical
immunological parameters were detected.
Conclusions. Rituximab is safe
and efficacious in childhood NS. Our data indicates an integral role for B
lymphocytes in this disease, but that the response is likely to be more
complex than simply depletion of peripheral B lymphocyte subsets.

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