SAN DIEGO — Apremilast quickly relieves painful oral and genital ulcers in patients with Behçet's disease and improves quality of life, compared with placebo, results of a phase 2 study suggest.
"The improvement in disease activity was statistically significant and clinically meaningful for patients," said lead investigator Gulen Hatemi, MD, from Cerrahpasa Medical School in Istanbul, Turkey. "The drug was safe and improved patient-reported outcomes."
Behçet's is a rare chronic inflammatory disease characterized by painful, recurrent oral and genital ulcers and other skin lesions. Its prevalence is highest in the Middle East, Asia, and Japan; about 10,000 Americans suffer from the disease.
"Behçet's disease can have a severe negative impact on patients' quality of life, and there are limited options available," Dr. Hatemi explained, "so there is a clear need for a new therapy to help this patient population."
She presented the results of the trial studying this first-in-class oral, targeted phosphodiesterase 4 inhibitor during a plenary session here at the American College of Rheumatology (ACR) 2013 Annual Meeting.
The phase 2 multicenter, randomized, placebo-controlled, double-blind, parallel-group study known as BCT-001 includes more than 100 patients with active oral ulcers receiving treatment with apremilast 30 mg twice daily or placebo.
At the end of 12 weeks, all patients were allowed to cross over to apremilast and were observed for an additional 4 weeks. Ninety-five patients completed 12 weeks of the study, and 91 completed 24 weeks. The mean age was 34.5 years, 69% were women, and mean disease duration was about 5 years.
Significantly more patients receiving apremilast achieved a complete response and were ulcer-free at week 12 compared with patients receiving placebo (70.9% vs 28.6%, P<.0001).
Among the 10 patients with genital ulcers at baseline in the apremilast group, 100% had a complete response; 6 patients in the placebo group had genital ulcers at baseline, and 3 (50%) had a complete response at week 12 (P = .036).
When the drug was stopped, the ulcers recurred, Dr. Hatemi noted.
Disease activity, as measured by the Behçet's Disease Current Activity Form and Behçet's Disease Activity Scale, was significantly improved with apremilast vs placebo. "These responses were clinically meaningful," she said.
Pain associated with oral ulcers was improved in the apremilast group on the visual analog scale (-44.7 with apremilast vs -16 with placebo, P < .0001). Patient-reported quality of life was also significantly improved on the Behçet's disease questionnaire (P = .039), Dr. Hatemi reported.
The frequency of adverse events was similar between groups; 89.3% of the apremilast group vs 89.1% of the placebo group reported 1 or more adverse events. Headache was the most common effect in both groups, whereas nausea, vomiting, and diarrhea were seen more commonly in patients treated with apremilast. Dr. Hatemi said the nausea was not severe enough to require prophylaxis.
ACR president Audrey Uknis, MD, said, "Behçet's is a painful and difficult-to-manage syndrome that negatively affects quality of life, and the state-of-the art treatment is inadequate." She noted, "This study suggests that providing therapy that works via a different mechanism improves the ability to manage this disease."
This study was funded by Celgene. Dr. Hatemi reports no relevant financial relationships. Dr. Uknis reports receiving research grants and consulting fees from ABIM, Biogen-Idec, and Roche.
ACR 2013 Annual Meeting: Abstract 761. Presented October 27, 2013.
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Cite this: Apremilast Potential Treatment for Behcet's - Medscape - Oct 28, 2013.