Favorable outcome in children with dense deposit disease with the use of long-term mycophenolate mofetil and high-dose alternate-day steroids.

BACKGROUND

This study aimed to evaluate the response to therapy and outcome with long-term daily mycophenolate mofetil (MMF) and high-dose alternate-day steroids (HADS) in children with dense deposit disease (DDD).

METHODS

Children with DDD who received long-term MMF (1200 mg/m/day) and HADS (1.5-2 mg/kg AD) with slow tapering were retrospectively evaluated for their clinico-pathological presentation, response to therapy (complete, partial, no remission) and outcome (patient and renal survival).

RESULTS

Six out of eight children with DDD seen over 10 years (3 boys, 3 girls) aged 6-13 years received the above therapy. Clinical presentation was nephrotic syndrome (1/6), gross hematuria (2/6), and nephritic onset of nephrotic syndrome (3/6). Serum creatinine was elevated at presentation in 3/6; C3 levels were low in all. None had crescentic changes on biopsy. The duration of therapy was 2-3 years. On therapy, haematuria resolved by 3 months, and proteinuria decreased to non-nephrotic range by 12 to 18 months in all six. Serum creatinine normalized, and all showed complete remission during treatment. Three had sustained remission. Two children relapsed while tapering steroids and attained partial remission on increasing the steroid dose. One patient relapsed, 1 year after therapy completion and did not respond to restarting the same treatment. Over a mean follow-up period of 5 years, patient and renal survival was 100%.

CONCLUSIONS

Long-term treatment with MMF and HADS showed a beneficial effect in the resolution of proteinuria with preservation of renal function and survival in the medium term in children with DDD with nephritic/nephrotic presentation.