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Efficacy and safety of rituximab in childhood-onset, difficult-to-treat nephrotic syndrome A multicenter open-label trial in Korea
- Title
- Efficacy and safety of rituximab in childhood-onset, difficult-to-treat nephrotic syndrome A multicenter open-label trial in Korea
- Authors
- Ahn, Yo Han; Kim, Seong Heon; Han, Kyoung Hee; Choi, Hyun Jin; Cho, Heeyeon; Lee, Jung Won; Shin, Jae Il; Cho, Min Hyun; Lee, Joo Hoon; Park, Young Seo; Ha, Il-Soo; Cheong, Hae Il; Kim, Su Young; Lee, Seung Joo; Kang, Hee Gyung
- Ewha Authors
- 이승주; 이정원
- SCOPUS Author ID
- 이승주; 이정원
- Issue Date
- 2018
- Journal Title
- MEDICINE
- ISSN
- 0025-7974
1536-5964
- Citation
- MEDICINE vol. 97, no. 46
- Keywords
- nephrotic syndrome; rituximab; children
- Publisher
- LIPPINCOTT WILLIAMS &
WILKINS
- Indexed
- SCIE; SCOPUS
- Document Type
- Article
- Abstract
- 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/m(2) 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.
- DOI
- 10.1097/MD.0000000000013157
- Appears in Collections:
- 의과대학 > 의학과 > Journal papers
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