Haddad M, Kale A, Butani L, et al. - Findings demonstrate that North-American children with minimal change disease (MCD) who have difficult to treat steroid resistant nephrotic syndrome (SRNS-DTT) can achieve long term remission with intravenous (IV) cyclophosphamide (CTX) treatment.

• In this retrospective review, data from children (n=8) with SRNS-DTT treated with IV CTX [monthly doses (median 6; range 4–6)], were analyzed to assess the outcome of IV CTX treatment in North American children with SRNS-DTT.

• Complete remission (CR) and partial remission were achieved in four (50%) patients and in one patient, respectively, no response was seen in 3, and MCD was present in three of the 4 responders.

• Post-completion of IV CTX infusions, the median time to CR was reported to be 6.5 (range 0.5–8) months, excluding the 1 child who responded following the 4th infusion.

• At a median of 8.5 years post-completion of CTX, three continued to be in CR; relapse and steroid dependency occurred in one child.

• No infections or life-threatening complications associated with IV CTX were noted.