Apremilast in refractory orogenital ulcers and other manifestations of Behçet's disease. National multicenter study of 51 cases in clinical practice.

Abstract Background Oral and/or genital aphthous ulcers are the most common symptoms of Behçet´s disease (BD), and are often refractory to conventional treatment. The inhibitor of phosphodiesterase-4 apremilast (APR) has demonstrated efficacy in the treatment of this manifestations. The objective of the present study was to assess the efficacy of APR in the management of refractory oral and/or genital ulcers in patients with BD. Methods National multicenter open-label observational study on BD patients with recurrent oral and/or genital ulcers. In all cases orogenital ulcers were refractory to conventional therapy. APR was given and maintained at standard dose of 30 mg twice daily. The main outcome was the achievement of oral and/or genital ulcers remission. Efficacy of APR for other clinical manifestations was also evaluated. Results We included 51 patients (35 women/16 men; mean age 44.7 ± 13.2 years). Before APR, all patients had received several systemic conventional and/or biologic drugs. APR was initiated because of refractory oral (n = 19) or genital (n = 2) aphthous ulcers or both (n = 30). Other manifestations found at APR onset were arthralgia/arthritis (n = 16), folliculitis/pseudofolliculitis (n = 14), erythema nodosum (n = 3), furunculosis (n = 2), paradoxical psoriasis induced by TNFα-inhibitors (n = 2), ileitis (n = 2), deep venous thrombosis (n = 2), leg ulcers (n = 1), erythematosus and scaly skin lesions (n = 1), fever (n = 1), unilateral anterior uveitis (n = 1) and neurobehçet (n = 1). After a mean follow-up of 8.5 ± 6.9 months, most patients had experienced improvement of orogenital ulcers and prednisone dose had been successfully reduced or discontinued. APR also yielded improvement of some non-aphthous manifestations such as the cutaneous follicular and intestinal manifestations. However, the effect on musculoskeletal manifestations was variable. Conclusion APR yielded a rapid and maintained improvement of refractory mucocutaneous ulcers of BD, even in patients refractory to several systemic drugs including biologic therapy.