Apremilast for Behcet's Syndrome - A Phase 2, Placebo-Controlled Study
Küçük Resim Yok
Tarih
2015
Dergi Başlığı
Dergi ISSN
Cilt Başlığı
Yayıncı
MASSACHUSETTS MEDICAL SOC
Erişim Hakkı
info:eu-repo/semantics/closedAccess
Özet
BACKGROUND Oral ulcers, the hallmark of Behcet's syndrome, can be resistant to conventional treatment; therefore, alternative agents are needed. Apremilast is an oral phosphodiesterase-4 inhibitor that modulates several inflammatory pathways. METHODS We conducted a phase 2, multicenter, placebo-controlled study in which 111 patients with Behcet's syndrome who had two or more oral ulcers were randomly assigned to receive 30 mg of apremilast twice daily or placebo for 12 weeks. This regimen was followed by a 12-week extension phase in which the placebo group was switched to apremilast and a 28-day post-treatment observational follow-up phase. The patients and clinicians were unaware of the study assignments throughout the trial. The primary end point was the number of oral ulcers at week 12. Secondary outcomes included pain from these ulcers (measured on a 100-mm visual-analogue scale, with higher scores indicating worse pain), the number of genital ulcers, overall disease activity, and quality of life. RESULTS The mean (+/- SD) number of oral ulcers per patient at week 12 was significantly lower in the apremilast group than in the placebo group (0.5 +/- 1.0 vs. 2.1 +/- 2.6) (P<0.001). The mean decline in pain from oral ulcers from baseline to week 12 was greater with apremilast than with placebo (-44.7 +/- 24.3 mm vs. -16.0 +/- 32.5 mm) (P<0.001). Nausea, vomiting, and diarrhea were more common in the apremilast group (with 22, 9, and 12 incidents, respectively, among 55 patients) than in the placebo group (with 10, 1, and 2 incidents, respectively, among 56 patients), findings that were similar to those in previous studies of apremilast. There were two serious adverse events in patients receiving apremilast. CONCLUSIONS Apremilast was effective in treating oral ulcers, which are the cardinal manifestation of Behcet's syndrome. This preliminary study was neither large enough nor long enough to assess long-term efficacy, the effect on other manifestations of Behcet's syndrome, or the risk of uncommon serious adverse events.
Açıklama
Anahtar Kelimeler
Kaynak
NEW ENGLAND JOURNAL OF MEDICINE
WoS Q Değeri
Q1
Scopus Q Değeri
Q1
Cilt
372
Sayı
16
