利妥昔单抗治疗原发性干燥综合征（pss）疗效有限
发表者：作者:Devauchelle-Pensec V 翻译:赵金霞
日期：2015-02-27
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摘要: 背景：原发性干燥综合征（pSS）是一种以眼干和口干或系统损害为主要特征的自身免疫性疾病。
目的：评价利妥昔单抗治疗新发或有系统损害的pSS患者的疗效和安全性。
试验设计：2008年3月至2011年1月进行的一项随机安慰剂平行对照试验。对研究人员（除药剂师），研究者，和患者均设盲。（ClinicalTrials.gov: NCT00740948） 实施：法国的14个大学医院。 患者：直观模拟量表（VAS）4项中（总体病情，疼痛，疲乏和干燥）至少有2项的评分大于等于50mm的初发（＜10年）活动的或有系统损害的pSS患者共120例。 干预：随机（1:1）分为利妥昔单抗组（0和2周各1g）或安慰剂组。 评价指标：主要终点是24周时4项VAS中有2项改善至少在30mm以上。
结果：两组的主要终点无明显差异(差异： 1.0% [95% CI, -16.7%至18.7%])。第6周时，利妥昔单抗组中4项VAS至少2项改善达30mm的患者比例高于安慰剂组（22.4% vs. 9.1%; P = 0.036）。利妥昔单抗组中在治疗6周(P < 0.001)和16周(P = 0.012)时疲乏的VAS评分改善达30mm以上者高于安慰剂组，从基线到24周时疲乏的改善程度在利妥昔单抗组更显著。除利妥昔单抗组输液反应高于安慰剂组，两组的不良反应相似。 局限性：基线时疾病活动度低，主要终点可能对于判断临床表现的主要变化并不敏感。
结论：利妥昔单抗治疗pSS可以在治疗早期缓解一些临床症状，但是在24周时并未减轻症状或降低疾病活动度。
附原文:Astract BACKGROUND:Primary Sjögren syndrome (pSS) is an autoimmune disorder characterized by ocular and oral dryness or systemic manifestations.OBJECTIVE:To evaluate efficacy and harms of rituximab in adults with recent-onset or systemic pSS. DESIGN: Randomized, placebo-controlled, parallel-group trial conducted between March 2008 and January 2011. Study personnel (except pharmacists), investigators, and patients were blinded to treatment group. (ClinicalTrials.gov: NCT00740948). SETTING: 14 university hospitals in France.PATIENTS: 120 patients with scores of 50 mm or greater on at least 2 of 4 visual analogue scales (VASs) (global disease, pain, fatigue, and dryness) and recent-onset (< 10 years) biologically active or systemic pSS. INTERVENTION: Randomization (1:1 ratio) to rituximab (1 g at weeks 0 and 2) or placebo. MEASUREMENTS: Primary end point was improvement of at least 30 mm in 2 of 4 VASs by week 24. RESULTS: No significant difference between groups in the primary end point was found (difference, 1.0% [95% CI, -16.7% to 18.7%]). The proportion of patients with at least 30-mm decreases in at least two of the four VAS scores was higher in the rituximab group at week 6 (22.4% vs. 9.1%; P = 0.036). An improvement of at least 30 mm in VAS fatigue score was more common with rituximab at weeks 6 (P < 0.001) and 16 (P = 0.012), and improvement in fatigue from baseline to week 24 was greater with rituximab. Adverse events were similar between groups except for a higher rate of infusion reactions with rituximab. LIMITATION:Low disease activity at baseline and a primary outcome that may have been insensitive to detect clinically important changes. CONCLUSION:Rituximab did not alleviate symptoms or disease activity in patients with pSS at week 24, although it alleviated some symptoms at earlier time points.
引自: Devauchelle-Pensec V, Mariette X, Jousse-Joulin S,et al.Treatment of primary Sjögren syndrome with rituximab: a randomized trial. Ann Intern Med. 2014 Feb 18;160(4):233-42. （注：干燥综合症病人或亲属可加QQ群交流，群号： 118194945 ，本网站站长私人微信号： ssgzz88 ）
目的：评价利妥昔单抗治疗新发或有系统损害的pSS患者的疗效和安全性。
试验设计：2008年3月至2011年1月进行的一项随机安慰剂平行对照试验。对研究人员（除药剂师），研究者，和患者均设盲。（ClinicalTrials.gov: NCT00740948） 实施：法国的14个大学医院。 患者：直观模拟量表（VAS）4项中（总体病情，疼痛，疲乏和干燥）至少有2项的评分大于等于50mm的初发（＜10年）活动的或有系统损害的pSS患者共120例。 干预：随机（1:1）分为利妥昔单抗组（0和2周各1g）或安慰剂组。 评价指标：主要终点是24周时4项VAS中有2项改善至少在30mm以上。
结果：两组的主要终点无明显差异(差异： 1.0% [95% CI, -16.7%至18.7%])。第6周时，利妥昔单抗组中4项VAS至少2项改善达30mm的患者比例高于安慰剂组（22.4% vs. 9.1%; P = 0.036）。利妥昔单抗组中在治疗6周(P < 0.001)和16周(P = 0.012)时疲乏的VAS评分改善达30mm以上者高于安慰剂组，从基线到24周时疲乏的改善程度在利妥昔单抗组更显著。除利妥昔单抗组输液反应高于安慰剂组，两组的不良反应相似。 局限性：基线时疾病活动度低，主要终点可能对于判断临床表现的主要变化并不敏感。
结论：利妥昔单抗治疗pSS可以在治疗早期缓解一些临床症状，但是在24周时并未减轻症状或降低疾病活动度。
附原文:Astract BACKGROUND:Primary Sjögren syndrome (pSS) is an autoimmune disorder characterized by ocular and oral dryness or systemic manifestations.OBJECTIVE:To evaluate efficacy and harms of rituximab in adults with recent-onset or systemic pSS. DESIGN: Randomized, placebo-controlled, parallel-group trial conducted between March 2008 and January 2011. Study personnel (except pharmacists), investigators, and patients were blinded to treatment group. (ClinicalTrials.gov: NCT00740948). SETTING: 14 university hospitals in France.PATIENTS: 120 patients with scores of 50 mm or greater on at least 2 of 4 visual analogue scales (VASs) (global disease, pain, fatigue, and dryness) and recent-onset (< 10 years) biologically active or systemic pSS. INTERVENTION: Randomization (1:1 ratio) to rituximab (1 g at weeks 0 and 2) or placebo. MEASUREMENTS: Primary end point was improvement of at least 30 mm in 2 of 4 VASs by week 24. RESULTS: No significant difference between groups in the primary end point was found (difference, 1.0% [95% CI, -16.7% to 18.7%]). The proportion of patients with at least 30-mm decreases in at least two of the four VAS scores was higher in the rituximab group at week 6 (22.4% vs. 9.1%; P = 0.036). An improvement of at least 30 mm in VAS fatigue score was more common with rituximab at weeks 6 (P < 0.001) and 16 (P = 0.012), and improvement in fatigue from baseline to week 24 was greater with rituximab. Adverse events were similar between groups except for a higher rate of infusion reactions with rituximab. LIMITATION:Low disease activity at baseline and a primary outcome that may have been insensitive to detect clinically important changes. CONCLUSION:Rituximab did not alleviate symptoms or disease activity in patients with pSS at week 24, although it alleviated some symptoms at earlier time points.
引自: Devauchelle-Pensec V, Mariette X, Jousse-Joulin S,et al.Treatment of primary Sjögren syndrome with rituximab: a randomized trial. Ann Intern Med. 2014 Feb 18;160(4):233-42. （注：干燥综合症病人或亲属可加QQ群交流，群号： 118194945 ，本网站站长私人微信号： ssgzz88 ）