Clinical scenario: Nephrotic syndrome | Steroid dependent | Alternate therapy
A 9-year-old girl has steroid-dependent nephrotic syndrome. She has frequent relapses whenever prednisolone is tapered below 10 mg/day. She previously received cyclophosphamide but it was discontinued due to severe neutropenia. Her renal function is normal, and she has no hypertension.
Which is the most appropriate steroid-sparing therapy?
A. Mycophenolate mofetil
B. High-dose prednisolone
C. Long-term cyclophosphamide
D. Plasma exchange
E. Angiotensin-converting enzyme inhibitor
Correct answer & Explanation:
Correct answer: A. Mycophenolate mofetil
Explanation:
This child has steroid-dependent nephrotic syndrome (SDNS) with toxicity from cyclophosphamide. The aim is to maintain remission while reducing steroid exposure.
Mycophenolate mofetil (MMF) is an accepted steroid-sparing agent in SDNS, especially when:
- Steroid toxicity is significant
- Alkylating agents (cyclophosphamide) cannot be continued due to adverse effects
Why other options are incorrect:
- High-dose prednisolone → increases steroid toxicity.
- Long-term cyclophosphamide → risk of bone marrow suppression, infertility, and malignancy.
- Plasma exchange → used in selected antibody-mediated diseases, not SDNS.
- ACE inhibitor → reduces proteinuria but is not a steroid-sparing treatment.
Key point:
Steroid-dependent nephrotic syndrome + cyclophosphamide toxicity → consider MMF or calcineurin inhibitors (tacrolimus/cyclosporine).
