Background: Few studies evaluated the role of mycophenolate mofetil (MMF) in inflammatory bowel disease (IBD), and none of them had specifically focused on patients with previous multiple intolerances and/or nonresponses to conventional immunosuppressants and TNF-alpha inhibitors. The aim of this study was to evaluate efficacy and tolerability profile of MMF in patients with IBD and limited medical treatment options.
Methods: All consecutive patients with previous multiple intolerances and/or nonresponses to conventional immunosuppressants and TNF-alpha inhibitors who started an off-label treatment with MMF from January 2014 to February 2016 were entered in a prospectively maintained database. The steroid-free remission and the clinical response, this latter defined as a clear clinical improvement with a concomitant reduction of steroid dosage compared with baseline or discontinuation, were set as clinical end points.
Results: Baseline features of the study population (n=24) are summarized in Table 1.
All patients had at least one previous nonresponse to IM or biologics. In particular, 15 (62.5%) were non responders to at least one IM, and 22 (91.7%) to at least one biologic agent; 12 (50.0%) were not responder to at least one IM plus at least one biologic. In addition, 20 (83.3%) had a previous intolerance to at least one IM, and 13 (54.2%) to at least one biologic. The median duration of total follow-up was 32 weeks (range 12–124). Four weeks after initiation of MMF therapy, a steroid-free remission was achieved in 4 patients (16.7%), while a clinical response in 13 (54.1%). At the end of follow- up, 12 patients (50.0%) remained on MMF. Six achieved and maintained steroid-free remission throughout the study period (25.0% of total), and a further 6 patients (25.0%) achieved a clinical response with complete discontinuation of steroids. Twelve patients (50.0%) were considered as treatment failure, and five of them underwent surgery. There was a trend towards a higher efficacy in patients with ulcerative colitis compared with Crohn's disease (63.6% vs. 38.5%, Figure 1), but this was not significant (p=0.20).
Conclusions: This is the first experience reporting a good efficacy and tolerability of MMF in patients with IBD and multiple previous failures to conventional immunosuppressants and/or TNF-alpha inhibitors.