Failure or intolerance of treatments by azathioprine (AZA) or methotrexate (MTX) is an important problem in the maintenance treatment of refractory CD. Results of the alternative use of mycophenolate mofetil (MMF) are conflicting.

To assess the efficacy and tolerance of MMF in patients with refractory CD included in a multicentre and retrospective study.

Twenty patients (5 men, 15 women, median age 33 years) have been enrolled. The disease was located in the small intestine (n = 7), colon (n = 5) or in both ileon and colon (n = 8). Five patients had perineal disease. Neighteen patients had an active CD and 13 of them received steroids (30 mg daily). All of them had been treated by AZA (failure, n = 5; intolerance, n = 15) and/or MTX (failure, n = 12; intolerance, n = 6). The dose of MMF was 750 mg to 2 g daily for a median of 4 months (range: 4 days-21 months). Response to MMF was determined according to an Harvey-Bradshaw index < 4 and the possibility to taper steroids.

Four patients (AZA: failure, n = 2; intolerance, n = 2; MTX: failure, n = 2; intolerance, n = 1) achieved remission under MMF. Three of them had an active CD before start of MMF, 1 patient was already in remission. Those 4 patients remained in remission during follow up (10-18 months). One of the 5 patients with perineal involvement achieved a complete closure of perineal fistula. Ten patients had to stop the medication for a median of 4 months (range: 2-12 months) because of inefficiency. Eleven patients had early adverse reactions and 5 of them had to stop the medication within 4 days to 3 months: pancreatitis (n = 2), toxidermia (n = 2), diarrhea and abdominal pain (n = 1).

In this short cohort of 20 patients with refractory CD, either intolerant or non responder to AZA or MTX, treatment with MMF resulted in only 20% of success. Intolerance to MMF was observed in 25% of patients.