Survey of rituximab treatment for childhood-onset refractory nephrotic syndrome
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Rituximab (RTX) is a promising option for treating childhood-onset steroid-dependent (SDNS), frequently relapsing (FRNS), and steroid-resistant (SRNS) nephrotic syndrome.
We retrospectively surveyed RTX treatment for these conditions to evaluate its indications, efficacy and adverse events. Questionnaires were sent to 141 hospitals in Japan.
Seventy-four patients (52 SDNS; 3 FRNS; 19 SRNS) were treated with RTX because of resistance to various immunosuppressive agents. Most patients received a single administration of RTX (85%). Forty-one of 53 SDNS/FRNS (77%) and 5 of 17 SRNS (29%) patients successfully discontinued prednisolone (16 SDNS/FRNS and 6 SRNS achieved their first discontinuation since onset), and 17 out of 53 SDNS/FRNS patients (31%) discontinued cyclosporine. However, 28 of the 53 patients (51%) relapsed. Although immunosuppressive agents did not extend B cell depletion, relapses were significantly less if immunosuppressive agents were continued after RTX (P = 0.006; hazard ratio = 0.2). Among the SRNS patients, complete (n = 6) and partial remission (n = 6) were achieved. No life-threatening adverse events were experienced.
Although this was a multi-center survey where treatment of nephrotic syndrome varied between centers, the steroid-sparing effect of RTX in SDNS/FRNS was excellent. If single administration of RTX is chosen, continuation of immunosuppressive agents is recommended for prevention of relapse.
KeywordsRituximab Nephrotic syndrome Children Steroid Steroid-dependent nephrotic syndrome Steroid-resistant nephrotic syndrome Immunosuppressive agents
This work had no financial support. The authors appreciate the doctors who collaborated on our questionnaire: Daishi Hirano, Saitama Children’s Medical Center, Saitama, Japan; Hiroshi Kaito, Kobe University, Kobe, Japan; Tomonori Harada, Yokohama City University Medical Center, Yokohama, Japan; Hiroshi Tanaka, Hirosaki University, Hirosaki, Japan; Toshio Watanabe, Gunma University, Maebashi, Japan; Masaki Shimizu, Kanazawa University, Kanazawa, Japan; Naohiro Wada, Shizouka Children’s Hospital, Shizuoka, Japan; Osamu Uemura, Aichi Children’s Health and Medical Center, Ohbu, Japan; Masashi Nishida, Kyoto Prefectural University of Medicine, Kyoto, Japan; Kenichi Satomura, Osaka Medical Center and Research Institute for Maternal and Child Health, Osaka, Japan; Rika Fujimaru, Osaka City General Hospital, Osaka Japan; Ryojiro Tanaka, Hyogo Prefectural Kobe Children’s Hospital, Kobe, Japan; and Kohei Maekawa, Hyogo College of Medicine, Nishinomiya, Japan.
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