Efficacy and safety of rituximab in childhoodonset, difficult-to-treat nephrotic syndrome: A multicenter open-label trial in Korea

Yo Han Ahn, Seong Heon Kim, Kyoung Hee Han, Hyun Jin Choi, Heeyeon Cho, Jung Won Lee, Jae Il Shin, Min Hyun Cho, Joo Hoon Lee, Young Seo Park, Il Soo Ha, Hae Il Cheong, Su Young Kim, Seung Joo Lee, Hee Gyung Kang

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36 Scopus citations


Background: The anti-CD20 monoclonal antibody rituximab (RTX) has been proposed as a rescue therapy for difficult-to-treat nephrotic syndrome (NS). We conducted a clinical trial to evaluate the efficacy and safety of RTX in children with difficult-to-treat NS dependent on or resistant to steroids and calcineurin inhibitors (CNIs). Methods: A multicenter open-label trial was performed at 8 major pediatric nephrology centers in Korea. The investigation consisted of a randomized controlled trial for steroid- and CNI-dependent NS (DDNS; randomization into the RTX group and the control group, at a ratio of 2:1) and a single-arm study of steroid and CNI-resistant NS (DRNS). DDNS patients in the RTX group and DRNS patients received a single dose of intravenous RTX (375mg/m2 of body surface area) for B-cell depletion. A second RTX dose was administered at week 2 if the first dose failed to achieve depletion of CD19(+) cells. The primary endpoint was rate of maintaining remission at 6 months after treatment for DDNS and rate of remission achievement for DRNS. Results: Sixty-one children with DDNS were enrolled while in remission and randomized to the control group (21 patients) or the RTX group (40 patients). At 6 months after treatment, the remission rates were 74.3% in the RTX group and 31.3% in the control group (P=.003). The mean duration of remission maintenance was significantly higher in the RTX group than in the control group (9.0 vs 2.9 months, P=.004). Of the 23 patients with DRNS enrolled in the single-arm study and treated with RTX, 9 (39.1%) achieved partial or complete remission within 6 months. Depletion of B cells occurred in all patients with RTX therapy. Thirty patients (50.8% of 59 patients analyzed) experienced mild and transient infusion reaction during RTX administration, and most adverse events were mild. Conclusions: RTX administration was safe and effective in patients with difficult-to-treat NS. One or 2 doses of RTX may be sufficient to deplete B cells and achieve better control of pediatric NS.

Original languageEnglish
Article numbere13157
JournalMedicine (United States)
Issue number46
StatePublished - 1 Nov 2018

Bibliographical note

Publisher Copyright:
© 2018 the Author(s). Published by Wolters Kluwer Health, Inc.


  • Children
  • Nephrotic syndrome
  • Rituximab


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