Efgartigimod in Sjögren's disease: a phase 2, randomised, placebo-controlled, parallel-group, double-blinded, proof-of-concept study (RHO).

Methods

We conducted a randomised, double-blinded, placebo-controlled, phase 2, proof-of-concept multicentre study (hereafter referred to as RHO).

我们进行了一项随机、双盲、安慰剂对照的2期概念验证性多中心研究（以下简称RHO研究）。

Participants were randomised 2:1 to receive efgartigimod IV 10 mg/kg or placebo once weekly for 24 weeks.

参与者按2:1的比例随机接受efgartigimod静脉注射10 mg/kg或安慰剂，每周一次，持续24周。

The primary outcome was the proportion of Composite of Relevant Endpoints for Sjögren's Syndrome (CRESS) responders on ≥3 of 5 items (systemic disease activity, patient-reported symptoms, tear and salivary gland function, and serology) at week 24.

主要结果是24周时，系统性疾病活动、患者报告的症状、泪液和唾液腺功能以及血清学5项指标中至少3项达到干燥综合征相关终点综合（CRESS）反应者所占的比例。

Secondary outcomes included the proportion with candidate Sjögren's Tool for Assessing Response (cSTAR) score ≥5 at week 24 and safety.

次要结果包括第24周时具有候选Sjögren's Tool for Assessing Response (cSTAR)评分≥5的比例以及安全性。

No formal statistical hypothesis was tested.

没有进行正式的统计假设检验。

Results

Thirty-four participants were randomised to efgartigimod (N = 23) or placebo (N = 11); 31 were included in the efficacy analysis.

共有34名参与者被随机分配到efgartigimod组（N=23）或安慰剂组（N=11）；其中31名被纳入疗效分析。

A numerically higher proportion of efgartigimod-treated participants responded to ≥3 items of CRESS at week 24 compared with placebo (45.5% vs 11.1%; treatment difference 34.4%).

在第24周，接受efgartigimod治疗的参与者中，有更高比例的人对CRESS的≥3项指标产生了反应，与安慰剂组相比（45.5%对比11.1%；治疗差异为34.4%）。

More specifically, efgartigimod scored higher on 4 of 5 CRESS items.

更具体地说，efgartigimod在5项CRESS指标中有4项得分更高。

Similarly, a numerically higher proportion of efgartigimod-treated participants scored ≥5 on the cSTAR at week 24 (efgartigimod, 54.5% vs placebo, 33.3%).

同样，在第24周，接受efgartigimod治疗的参与者在cSTAR评分上达到或超过5的比例在数值上更高（efgartigimod，54.5% vs 安慰剂，33.3%）。

Treatment-emergent adverse events were reported in 87.0% of efgartigimod-treated participants compared with 63.6% of those on placebo; all were grade 1 or 2 in severity.

在使用efgartigimod治疗的参与者中，有87.0%报告了治疗后出现的不良事件，而在安慰剂组中这一比例为63.6%；所有不良事件的严重程度均为1级或2级。

Conclusions

Data from the RHO study support proof of concept for efgartigimod in SjD.

RHO研究的数据支持efgartigimod在SjD中的概念验证。

Further evaluation in a phase 3 study of efgartigimod in individuals with SjD is warranted.

在患有SjD的个体中，对efgartigimod进行III期研究的进一步评估是有必要的。

The trial was registered with ClinicalTrials.gov (NCT05817669).

该试验已在ClinicalTrials.gov上注册（NCT05817669）。