Tacrolimus or Mycophenolate Mofetil for Frequently Relapsing or Steroid-Dependent Nephrotic Syndrome: A Randomized...

Area of Science:

• Pediatric Nephrology
• Immunosuppressive Therapy

Background:

• Frequently relapsing nephrotic syndrome (FRNS) and steroid-dependent nephrotic syndrome (SDNS) in children often require long-term immunosuppression.
• Tacrolimus (TAC) and mycophenolate mofetil (MMF) are recommended, but direct comparative data from randomized trials are lacking.

Purpose of the Study:

• To compare the effectiveness and safety of TAC versus MMF in pediatric patients with FRNS or SDNS.
• To evaluate relapse-free survival, relapse frequency, and cumulative steroid dosage.

Main Methods:

• A multicenter, open-label randomized clinical trial involving 270 children (aged 2-18 years) with FRNS or SDNS.
• Patients were randomized 1:1 to receive either TAC or MMF orally for 1 year, alongside a steroid tapering regimen.
• Primary endpoint was 1-year relapse-free survival; secondary endpoints included relapse frequency and steroid dosage.

Main Results:

• Tacrolimus (TAC) therapy resulted in a significantly higher 1-year relapse-free survival rate compared to mycophenolate mofetil (MMF) (HR, 2.86; P < .001).
• The mean time to first relapse was longer with TAC (323.99 days) than MMF (263.21 days), with a lower annual relapse rate (17.78% vs 41.48%).
• Patients on TAC required significantly lower cumulative steroid doses (0.22 mg/kg/day vs 0.34 mg/kg/day), and safety profiles were similar between groups.

Conclusions:

• One-year tacrolimus (TAC) therapy significantly improves relapse-free survival in children with FRNS or SDNS compared to mycophenolate mofetil (MMF).
• TAC offers a potential advantage in reducing disease recurrence and steroid dependency in this pediatric population.
• The findings support TAC as a preferred therapeutic option for managing FRNS/SDNS in children, given its efficacy and comparable safety profile.