Background/Purpose: Behçet´s disease (BD) is characterized 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. 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 14 BD patients treated with apremilast at standard dose of 30 mg twice daily. The main outcome was resolution of oral and/or genital ulcers.

Results: We included 14 patients (10 women/ 4 men), with a mean age of 44.3±14.4 years. Before apremilast, all patients had also received several systemic conventional drugs: oral corticosteroids (n=13), colchicine (n=14), NSAIDS (n=7), methotrexate (n=7), azathioprine (n=5), cyclosporine (n=4), infliximab (n= 3), adalimumab (n=3), dapsone (n=3), mycophenolate mofetil (n=1), tocilizumab (n= 1), etanercept (n=1), secukinumab (n=1). The main clinical symptoms for starting apremilast were oral aphthous ulcers (n=14) and genital ulcers (n=10). Other manifestations present at apremilast onset were arthralgia/arthritis (n=4), folliculitis (n=4), furunculosis (n=1), erythematosus and scaly skin lesions (n=1), psoriasis (n=1), erythema nodosum (n=1), deep venous thrombosis (n=1), ileitis (n=1), asthenia (n=3). Ten patients experienced improvement in the first two weeks after starting apremilast. TABLE shows the evolution of the patients. After a median follow-up of 3.5 [interquartile range, 3-9] months, most of the patients experienced a total or partial clinical improvement. In this period of time, 7 patients developed side-effects: dyspepsia (n=4), nausea (n=3), diarrhea (n=3), headache (n=1), abdominal pain (n=1), anorexia (n=1), and halitosis (n=1). However, only 2 of 6 patients had to reduce the dose of apremilast to 30 mg/day. In 3 patients (21%) apremilast was withdrawn after 3 months of treatment due to unsatisfactory response.

Conclusion: 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.

Table.

The number of patients with available data is shown in parentheses.

ACR Meeting Abstracts - https://acrabstracts.org/abstract/apremilast-for-refractory-mucocutaneous-ulcers-of-behcets-disease-national-multicenter-study-of-14-cases/