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大学・研究所にある論文を検索できる 「Rituximab in combination with cyclosporine and steroid pulse therapy for childhood-onset multidrug-resistant nephrotic syndrome: a multicenter single-arm clinical trial (JSKDC11 trial)」の論文概要。リケラボ論文検索は、全国の大学リポジトリにある学位論文・教授論文を一括検索できる論文検索サービスです。

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Rituximab in combination with cyclosporine and steroid pulse therapy for childhood-onset multidrug-resistant nephrotic syndrome: a multicenter single-arm clinical trial (JSKDC11 trial)

Nozu, Kandai Sako, Mayumi Tanaka, Seiji Kano, Yuji Ohwada, Yoko Morohashi, Tamaki Hamada, Riku Ohtsuka, Yasufumi Oka, Masafumi Kamei, Koichi Inaba, Aya Ito, Shuichi Sakai, Tomoyuki Kaito, Hiroshi Shima, Yuko Ishikura, Kenji Nakamura, Hidefumi Nakanishi, Koichi Horinouchi, Tomoko Konishi, Akihide Omori, Takashi Iijima, Kazumoto 神戸大学

2023.11.27

概要

Background: Only 80% of children with idiopathic nephrotic syndrome respond well to glucocorticoid therapy. Multidrug-resistant nephrotic syndrome (MRNS) is associated with a poor kidney prognosis. Several retrospective studies have identified rituximab as an effective treatment for MRNS; however, prospective studies are required to assess its efficacy and safety. Methods: We conducted a multicenter, non-blinded, single-arm trial to investigate the efficacy and safety of rituximab in patients with childhood-onset MRNS who were resistant to cyclosporine and more than three courses of steroid pulse therapy. The enrolled patients received four 375 mg/m² doses of rituximab in combination with baseline cyclosporine and steroid pulse therapy. The primary endpoint was a > 50% reduction in the urinary protein/creatinine ratio from baseline on day 169. Complete and partial remissions were also evaluated. Results: Six patients with childhood-onset MRNS were enrolled. All patients were negative for pathogenic variants of podocyte-related genes. On day 169, five patients (83.3%) showed a > 50% reduction in the urinary protein/creatinine ratio, two patients showed partial remission, and two patients showed complete remission. No deaths occurred and severe adverse events occurred in two patients (infection in one patient and acute kidney injury in one patient). Three patients needed treatment for moderate-to-severe infection. Conclusions: The study treatment effectively reduced the urinary protein/creatinine ratio in patients with childhood-onset MRNS. The adverse events in this study were within the expected range; however, attention should be paid to the occurrence of infections.

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Publisher's Note Springer Nature remains neutral with regard to

jurisdictional claims in published maps and institutional affiliations.

Authors and Affiliations

Kandai Nozu1 · Mayumi Sako2 · Seiji Tanaka3 · Yuji Kano4 · Yoko Ohwada4 · Tamaki Morohashi5 · Riku Hamada6 ·

Yasufumi Ohtsuka7 · Masafumi Oka7 · Koichi Kamei8 · Aya Inaba9 · Shuichi Ito9 · Tomoyuki Sakai10 · Hiroshi Kaito11 ·

Yuko Shima12 · Kenji Ishikura13 · Hidefumi Nakamura14 · Koichi Nakanishi15 · Tomoko Horinouchi1 ·

Akihide Konishi16 · Takashi Omori16 · Kazumoto Iijima1

* Kandai Nozu

nozu@med.kobe-u.ac.jp

Department of Pediatrics, Kobe University Graduate

School of Medicine, 7‑5‑1 Kusunoki‑Cho, Chuo‑Ku,

Kobe 650‑0017, Japan

Division for Clinical Trials, Department of Clinical Research

Promotion, Clinical Research Center, National Center

for Child Health and Development, Tokyo, Japan

Department of Pediatrics, Kurume University School

of Medicine, Kurume, Japan

13

Clinical and Experimental Nephrology

Department of Pediatrics, Dokkyo Medical University School

of Medicine, Tochigi, Japan

11

Department of Pediatrics, Nihon University School

of Medicine, Tokyo, Japan

12

Department of Nephrology, Tokyo Metropolitan Children’s

Medical Center, Tokyo, Japan

13

Department of Pediatrics, Saga University School

of Medicine, Saga, Japan

14

Division of Nephrology and Rheumatology, National Center

for Child Health and Development, Tokyo, Japan

15

Department of Pediatrics, Graduate School of Medicine,

Yokohama City University, Yokohama, Japan

10

Department of Pediatrics, Shiga University of Medical

Science, Shiga, Japan

13

Department of Nephrology, Hyogo Prefectural Kobe

Children’s Hospital, Kobe, Japan

Department of Pediatrics, Wakayama Medical University,

Wakayama, Japan

Department of Pediatrics, Kitasato University School

of Medicine, Kanagawa, Japan

Clinical Research Center, National Center for Child Health

and Development, Tokyo, Japan

Department of Child Health and Welfare (Pediatrics),

Graduate School of Medicine, University of the Ryukyus,

Okinawa, Japan

16

Clinical and Translational Research Center, Kobe University

Hospital, Kobe, Japan

...

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