Survey of rituximab treatment for childhood-onset refractory nephrotic syndrome.
BACKGROUND
Rituximab (RTX) is a promising option for treating childhood-onset steroid-dependent (SDNS), frequently relapsing (FRNS), and steroid-resistant (SRNS) nephrotic syndrome.


METHODS
We retrospectively surveyed RTX treatment for these conditions to evaluate its indications, efficacy and adverse events. Questionnaires were sent to 141 hospitals in Japan.


RESULTS
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.


CONCLUSIONS
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.
Division of Pediatric Nephrology and Rheumatology, National Center for Child Health and Development, Tokyo, Japan. ito-shu@ncchd.go.jp
Shuichi
S
Ito
Koichi
K
Kamei
Masao
M
Ogura
Tomohiro
T
Udagawa
Shuichiro
S
Fujinaga
Mari
M
Saito
Mayumi
M
Sako
Kazumoto
K
Iijima
D000293:Adolescent; D058846:Antibodies, Monoclonal, Murine-Derived / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use; D018572:Disease-Free Survival; D004351:Drug Resistance; D005260:Female; D006801:Humans; D007155:Immunologic Factors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007223:Infant; D007564:Japan; D053208:Kaplan-Meier Estimate; D008297:Male; D009404:Nephrotic Syndrome / Q000145:classification / Q000188:drug therapy*; D017060:Patient Satisfaction; D011239:Prednisolone / Q000627:therapeutic use; D012008:Recurrence; D012189:Retrospective Studies; D000069283:Rituximab; D011795:Surveys and Questionnaires
D058846:Antibodies, Monoclonal, Murine-Derived; D007155:Immunologic Factors; D007166:Immunosuppressive Agents; D000069283:Rituximab; D016572:Cyclosporine; D011239:Prednisolone