Apremilast in Refractory Behçet’s Syndrome: A Multicenter Observational Study

• Published in: Frontiers in immunology Vol. 11; p. 626792
• Main Authors: Vieira, Matheus, Buffier, Solène, Vautier, Mathieu, Le Joncour, Alexandre, Jamilloux, Yvan, Gerfaud-Valentin, Mathieu, Bouillet, Laurence, Lazaro, Estibaliz, Barete, Stéphane, Misery, Laurent, Gobert, Delphine, Goulenok, Tiphaine, Fain, Olivier, Sacre, Karim, Sève, Pascal, Cacoub, Patrice, Comarmond, Cloé, Saadoun, David
• Format: Journal Article
• Language: English
• Published: Switzerland Frontiers 04.02.2021; Frontiers Media S.A
• Subjects: apremilast; Behçet; efficacy; Human health and pathology; Immunology; Immunotherapy; joint; Life Sciences; Rhumatology and musculoskeletal system; safety; skin; Behçet; joint; safety; skin; cohort; apremilast; efficacy
• ISSN: 1664-3224; 1664-3224
• DOI: 10.3389/fimmu.2020.626792

Mucocutaneous and joint disorders are the most common manifestations in Behçet's syndrome (BS) and are frequently clustered in the so-called minor forms of BS. There remains a need for safe and effective treatment for joint lesions in BS. We report the long-term safety and effectiveness of apremilast in refractory joint and mucocutaneous manifestations of BS. French nationwide multicenter study including 50 BS patients with either active joint and/or mucocutaneous manifestations resistant to colchicine and/or DMARDs. Patients received apremilast 30 mg twice a day. Primary effectiveness endpoint was the proportion of patients with complete response (CR) of articular symptoms at month 6 (M6), defined as resolution of inflammatory arthralgia and arthritis, with joint count equal to zero. At inclusion, the median tender and swollen joint count was of 4 [2-6] and 2 [1-2], respectively. The proportion of CR in joint disease at M6 was 65% (n = 15/23), and 17% (n = 4/23) were partial responders. CR of oral and genital ulcers, and pseudofolliculitis at M6 was 73% (n = 24/33), 94% (n = 16/17) and 71% (n = 10/14), respectively. The overall response at M6 was 74% for the entire cohort and 70% for the mucocutaneous-articular cluster (n = 27). The median Behçet's syndrome activity score significantly decreased during study period [50 (40-60) 20 (0-40); 0.0001]. After a median follow-up of 11 [6-13] months, 27 (54%) patients were still on apremilast. Reasons for apremilast withdrawal included adverse events (n = 15, 30%) and treatment failure (n = 8, 16%). Thirty-three (66%) patients experienced adverse events, mostly diarrhea (n = 19, 38%), nausea (n = 17, 34%) and headache (n = 16, 32%). Apremilast seems effective in BS-related articular disease refractory to colchicine and DMARDs. Discontinuation rates were significantly higher than that reported in clinical trials.