A 5-year-old boy presents with periorbital edema, massive proteinuria (urine protein:creatinine ratio 9), hypoalbuminemia (serum albumin 1.8 g/dL), and hypercholesterolemia. He responds completely to prednisolone. Two years later he relapses for the sixth time in 12 months while being tapered. Which agent is MOST appropriate for steroid-sparing in frequently relapsing nephrotic syndrome?
- A Rituximab 375 mg/m² IV one dose
- B Tacrolimus 0.1 mg/kg/day
- C Mycophenolate mofetil 600 mg/m² twice daily
- D Cyclophosphamide 2 mg/kg/day for 8 weeks ✓
Explanation
In frequently relapsing or steroid-dependent minimal change nephrotic syndrome (MCNS) in children, cyclophosphamide (CPM) 2–3 mg/kg/day orally for 8–12 weeks is the first-choice steroid-sparing agent; it induces sustained remission in ~40–60% of cases and is a one-time course. Alternative agents include levamisole, mycophenolate mofetil, and calcineurin inhibitors. Rituximab (anti-CD20 monoclonal antibody) is increasingly used for steroid-dependent/frequently relapsing nephrotic syndrome but is reserved for patients who fail or are intolerant of cyclophosphamide or calcineurin inhibitors — it is not first-line steroid-sparing. Tacrolimus and MMF are alternatives for steroid-dependent disease but are used when cyclophosphamide fails or is contraindicated.
Reference: Ghai Essential Pediatrics, 10th ed.
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