Scopus
🔓 Açık Erişim
Apremilast for Behçet's syndrome - A phase 2, placebo-controlled study
New England Journal of Medicine · Nisan 2015
Makale Bilgileri
DergiNew England Journal of Medicine
Yayın TarihiNisan 2015
Cilt / Sayfa372 · 1510-1518
Scopus ID2-s2.0-84928019857
Erişim🔓 Açık Erişim
Özet
BACKGROUND: Oral ulcers, the hallmark of Behçet'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 Behçet'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 (0.51.0 vs. 2.12.6) (P<0.001). The mean decline in pain from oral ulcers from baseline to week 12 was greater with apremilast than with placebo (-44.724.3 mm vs. -16.032.5 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 Behçet's syndrome. This preliminary study was neither large enough nor long enough to assess long-term efficacy, the effect on other manifestations of Behçet's syndrome, or the risk of uncommon serious adverse events. (Funded by Celgene; ClinicalTrials.gov number, NCT00866359.)
Yazarlar (13)
1
Gulen Hatemi
2
Melike Melikoglu
3
Recep Tunc
4
Cengiz Korkmaz
5
Banu Turgut Ozturk
ORCID: 0000-0003-0702-6951
6
Cem Mat
7
Peter A. Merkel
8
Kenneth T. Calamia
9
Ziqi Liu
10
Lilia Pineda
11
Randall M. Stevens
12
Yusuf Yazici
13
Hasan Yazici
Kurumlar
Celgene Corporation
Summit United States
Eskişehir Osmangazi Üniversitesi
Eskisehir Turkey
İstanbul University-Cerrahpaşa Cerrahpaşa Faculty of Medicine
Istanbul Turkey
Mayo Clinic in Jacksonville, Florida
Jacksonville United States
NYU Grossman School of Medicine
New York United States
Selçuk Üniversitesi
Selçuklu Turkey
University of Pennsylvania
Philadelphia United States
Metrikler
187
Atıf
13
Yazar