Efficacy of rituximab therapy in children with refractory nephrotic syndrome: a prospective observational study in Shanghai.
BACKGROUND
Idiopathic nephrotic syndrome is the most common glomerular disease in children. This study was undertaken to observe the efficacy and side-effects of rituximab (RTX) in treating children with different types of refractory primary nephrotic syndrome.


METHODS
Twelve patients with steroid dependent nephrotic syndrome (SDNS), frequently relapsing nephritic syndrome (FRNS), and steroid resistant nephrotic syndrome (SRNS) were enrolled in our study. There were obvious drug side-effects, and proteinuria remained difficult to control. RTX was administered at a dose of 375 mg/m(2) body surface area, once or twice weekly.


RESULTS
The male to female ratio was 3:1, and the onset age was 1.6-8.9 years. There were 9 patients with steroid sensitive nephrotic syndrome (SDNS or FRNS), and 3 patients with SRNS. There were 7 patients with minimal change disease (MCD), 3 patients with focal segmental glomerular sclerosis (FSGS), 1 with focal proliferative glomerulonephritis, and 1 without renal biopsy. The total effective treatment rate of RTX was 91.67%, and for 77.78% of the patients, steroid dosage could be reduced. Six months before and after RTX infusion, the mean steroid dosage was significantly decreased (P=0.014) and the recurrence number was significantly reduced (P<0.001). The results were better in MCD patients than in FSGS patients (P=0.045). There was no significant difference between FRNS/SDNS and SRNS patients (P=0.175). During RTX administration, 3 patients developed skin rashes, 1 developed hypotension, and 1 developed a fever. One patient experienced a persistent decrease in serum immunoglobulin level but without serious infection.


CONCLUSIONS
RTX was effective in the treatment of refractory nephrotic syndrome, and it could significantly reduce the use of steroid and immunosuppressants.
Department of Nephrology and Rheumatology, Children's Hospital of Fudan University, Shanghai, 201102, China.
Li
L
Sun
Hong
H
Xu
Qian
Q
Shen
Qi
Q
Cao
Jia
J
Rao
Hai-Mei
HM
Liu
Xiao-Yan
XY
Fang
Li-Jun
LJ
Zhou
D017668:Age of Onset; D058846:Antibodies, Monoclonal, Murine-Derived / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D005260:Female; D005919:Glomerular Filtration Rate; D006801:Humans; D007223:Infant; D008297:Male; D009404:Nephrotic Syndrome / Q000188:drug therapy* / Q000503:physiopathology; D011446:Prospective Studies; D000069283:Rituximab; D016896:Treatment Outcome; D064804:Urological Agents / Q000627:therapeutic use*
D058846:Antibodies, Monoclonal, Murine-Derived; D064804:Urological Agents; D000069283:Rituximab