Background Behçet’s disease (BD) is characterised by recurrent oral and/or genital ulcers accompanied by ocular, cutaneous, articular, gastrointestinal, and/or neurologic manifestations. Oral and/or genital aphthous ulcers are often refractory to conventional treatment. Apremilast is an orally-active small molecule which inhibits phosphodiesterase-4 (PDE-4) that modulates some inflammatory pathways.

Objectives Our aim was to assess the efficacy of apremilast in BD patients with oral and/or genital ulcers refractory to conventional treatment.

Methods Retrospective national multicenter open-label study on 19 BD patients treated with apremilast at standard dose of 30 mg twice daily. The main outcome was achievement of oral ulcers remission.

Results We included 19 patients (14 women and 5 men) with a mean age of 43.6±14.8 years. Before apremilast, all patients had also received several systemic conventional drugs: oral corticosteroids (n=18), colchicine (n=19), NSAIDs (n=10), methotrexate (n=10), azathioprine (n=10), cyclosporine (n=6), infliximab (n=3), adalimumab (n=5), dapsone (n=3), etanercept (n=1), mycophenolate mofetil (n=1), tocilizumab (n=1). The main clinical symptoms for starting apremilast were oral aphthous ulcers (n=19) and genital ulcers (n=14). Other manifestations present at apremilast onset were arthralgia/arthritis (n=6), folliculitis/pseudofolliculitis (n=6), asthenia (n=5), furunculosis (n=1), erythema nodosum (n=1), erythematosus and scaly skin lesions (n=1), psoriasis (n=1), deep venous thrombosis (n=2) and ileitis (n=1). Table 1 shows the evolution of the patients. After a median follow-up of 6 [interquartile range, 5–10] months, most of the patients experienced clinical improvement. In this period of time, 11 patients developed any side-effect: dyspepsia (n=5), nauseas (n=4), diarrhoea (n=4), abdominal pain (n=4), headache (n=3), loss of appetite (n=3), weight loss (n=1) and halitosis (n=1). Three patients had to reduce the dose to 30 mg/day. Apremilast was discontinued in 4 patients: because of not obtaining the expected improvement (n=2), due to desire of pregnancy (n=1) and due to development of neurological involvement (n=1).

Conclusions Apremilast leads to a rapid and maintained improvement in many patients with refractory mucocutaneous ulcers of BD. Even in patients refractory to several systemic drugs including biologic therapy. However, the development of adverse digestive effects is frequent.

Disclosure of Interest None declared