Efficacy and safety of belimumab in primary Sjogren's syndrome: results of the BELISS open-label phase II study
Efficacy and safety of belimumab in primary Sjogren's syndrome: results of the BELISS open-label phase II study
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DOI：
10.1136/annrheumdis-2013-203991
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发表时间：
2015-03-01
影响因子：
27.4
通讯作者：
De Vita, Salvatore
中科院分区：
文献类型：
--
作者：
Mariette, Xavier;Seror, Raphaele;De Vita, Salvatore
背景
B细胞激活因子（BAFF或B淋巴细胞刺激因子）表达增加可能解释原发性干燥综合征（pSS）的B细胞激活特征。
目的
评估针对BAFF的贝利木单抗在pSS患者中的疗效和安全性。
方法
如果患者符合美国 - 欧洲共识小组标准、抗干燥综合征A抗体阳性且有当前的全身并发症或唾液腺肿大，或者是早期疾病（视觉模拟量表（VAS）干燥评分≥30%、疲劳VAS评分≥30%、VAS疼痛评分≥30%、医生评估的全身活动VAS≥30%和/或任何B细胞激活生物标志物值改善>25%），则被纳入这项为期1年的双中心前瞻性开放标签试验。
结果
在纳入的30例患者中，18例（60%）达到主要终点。欧洲抗风湿病联盟（EULAR）干燥综合征疾病活动指数的平均值（标准差）从8.8（7.4）降至6.3（6.6）（p = 0.0015），EULAR干燥综合征患者报告指数从6.4（1.1）降至5.6（2.0）（p = 0.0174）。平均干燥、疲劳和疼痛VAS分别从7.8（1.8）变为6.2（2.9）（p = 0.0021）、6.9（1.8）变为6.0（2.2）（p = 0.0606）和4.6（2.6）变为4.7（2.4）（p = 0.89）。唾液流量和希尔默试验无变化。
结论
这些令人鼓舞的结果证明未来在最有可能从治疗中获益的选定pSS患者目标人群中进行贝利木单抗随机对照试验是合理的。
Background Increased expression of B cell activating factor (BAFF or B lymphocyte stimulator) may explain the B cell activation characteristic of primary Sjogren's syndrome (pSS).Objectives To evaluate the efficacy and safety of belimumab, targeting BAFF, in patients with pSS.Methods Patients were included in this bi-centric prospective 1-year open-label trial if they fulfilled American European Consensus group criteria, were anti-Sjogren's syndrome A-positive and had current systemic complications or salivary gland enlargement, or early disease (= 30% in dryness score on a visual analogue scale (VAS), >= 30% in fatigue VAS score, >= 30% in VAS pain score, >= 30% in systemic activity VAS assessed by the physician and/or >25% improvement in any B cell activation biomarker values.Results Among 30 patients included, the primary end-point was achieved in 18 (60%). The mean (SD) European League Against Rheumatism (EULAR) Sjogren's Syndrome Disease Activity Index decreased from 8.8 (7.4) to 6.3 (6.6) (p=0.0015) and EULAR) Sjogren's Syndrome Patient Reported Index from 6.4 (1.1) to 5.6 (2.0) (p=0.0174). The mean dryness, fatigue and pain VAS varied from 7.8 (1.8) to 6.2 (2.9) (p=0.0021), 6.9 (1.8) to 6.0 (2.2) (p=0.0606) and 4.6 (2.6) to 4.7 (2.4) (p=0.89), respectively. Salivary flow and Schirmer's test did not change.Conclusions These encouraging results justify future randomised controlled trials of belimumab in a selected target population of pSS patients most likely to benefit from treatment.