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December 19, 2023
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Using mycophenolate mofetil in steroid-sensitive idiopathic nephrotic syndrome in children

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Key takeaways:

  • Overall, 30% of patients on mycophenolate mofetil experienced a relapse during induction.
  • In the control group, 3% had a relapse in the same period.

Mycophenolate mofetil may be a viable alternative to steroids for induction therapy in children with steroid-sensitive idiopathic nephrotic syndrome, researchers found in a study.

Investigators led by Alexandra Mazo MD, PhD, of the Maria Fareri Children's Hospital, Westchester Medical Center, Boston Children's Health Physicians and New York Medical College, compared the effectiveness of mycophenolate mofetil (MMF) vs. prednisolone for induction therapy in steroid-sensitive idiopathic nephrotic syndrome in children.

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Overall, 30% of patients on mycophenolate mofetil experienced a relapse during induction. Image: Adobe Stock.

“High-dose steroids are recommended for induction of idiopathic nephrotic syndrome,” Mazo and colleagues wrote Kidney Medicine. “We hypothesized that MMF can be noninferior to steroids in maintaining steroid-induced remission. [Our] second aim was to mitigate side effects typically associated with prolonged steroid usage.”

The researchers conducted an observational study that included patients aged 2 to 11 years who had a first episode of nephrotic syndrome and entered remission within 2 weeks of standard steroid treatment. Of 41 eligible patients, 10 were in the MMF group and eight completed the study. The control group consisted of 31 patients, with 23 completing the follow-up. The experimental group underwent a 12-week induction with MMF, whereas the control group continued with the standard 12-week steroid protocol.

Primary outcomes were relapse rate and relapse-free interval during a 52-week follow-up period.

According to the findings, 30% of patients in the MMF group and 3% in the control group developed a relapse (P = .04) during the induction phase. Moreover, 70% of patients in the MMF group and 61% in the control group experienced a relapse during follow-up (P = .72), the researchers found. Median relapse-free interval was 11 weeks for MMF and 19 weeks for controls (P = .60), and no serious adverse events were reported in either group.

“Our small cohort of patients treated with MMF had a higher relapse rate during the induction phase. However, by 12 months of follow-up, relapse rate and relapse-free interval were similar between groups,” Mazo and colleagues wrote. “All patients tolerated MMF without significant side effects, and those who relapsed remained steroid sensitive. Monitoring MPA-[area under the curve] AUC and higher doses of MMF should be considered.”