Intravenous cyclophosphamide induces remission in children with difficult to treat steroid resistant nephrotic syndrome from minimal change disease

BMC Nephrology

Lavjay Butani¹  Maha Haddad¹  Arundhati Kale¹ 

[1] Section of Pediatric Nephrology, University of California Davis, 2516 Stockton Blvd, 95817, Sacramento, CA, USA;

关键词: Steroid resistant nephrotic syndrome;    Cyclophosphamide;    Pediatric;    Remission;   

DOI  :  10.1186/s12882-021-02605-6

来源: Springer

【 摘 要 】

BackgroundSteroid resistant nephrotic syndrome (SRNS), while uncommon in children, is associated with significant morbidity. Calcineurin inhibitors (CNIs) remain the first line recommended therapy for children with non-genetic forms of SRNS, but some children fail to respond to them. Intravenous (IV) cyclophosphamide (CTX) has been shown to be effective in Asian-Indian children with difficult to treat SRNS (SRNS-DTT). Our study evaluated the outcome of IV CTX treatment in North American children with SRNS-DTT.MethodsRetrospective review of the medical records of children with SRNS-DTT treated with IV CTX from January 2000 to July 2019 at our center. Data abstracted included demographics, histopathology on renal biopsy, prior and concomitant use of other immunosuppressive agents and serial clinical/laboratory data. Primary outcome measure was attainment of complete remission (CR).ResultsEight children with SRNS-DTT received monthly doses (median 6; range 4–6) of IV CTX. Four (50%) went into CR, 1 achieved partial remission and 3 did not respond. Three of the 4 responders had minimal change disease (MCD). Excluding the 1 child who responded after the 4th infusion, the median time to CR was 6.5 (range 0.5–8) months after completion of IV CTX infusions. Three remain in CR at a median of 8.5 years (range: 3.7–10.5 years) after completion of CTX; one child relapsed and became steroid-dependent. No infections or life-threatening complications related to IV CTX were observed.ConclusionsIV CXT can induce long term remission in North-American children with MCD who have SRNS-DTT.

【 授权许可】

CC BY