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期刊来源：JAMA

原文链接：https://doi.org/10.1001/jama.2024.8693

摘要内容如下：

重要性

目前对特发性肺纤维化的治疗减缓了肺功能下降的速度，但可能与影响药物依从性的不良事件有关。在2期临床试验中，Pamrevlumab（一种结合并抑制结缔组织生长因子活性的全人单克隆抗体）可减缓特发性肺纤维化的进展，且无实质性不良事件。

目的

评估Pamrevlumab治疗特发性肺纤维化患者的疗效和安全性。

设计、设置和参与者

3期随机临床试验包括356名年龄在40至85岁之间的特发性肺纤维化患者，这些患者在入组时未接受尼替达尼或吡非尼酮的抗纤维化治疗。在2019年7月18日至2022年7月29日期间，从9个国家的117个地点招募患者。最后一次后续遭遇发生在2023年8月28日。

干预措施

Pamrevlumab（每3周静脉给药30mg/kg；n=181）或安慰剂（n=175）治疗48周。

主要成果和措施

主要结果是从基线到第48周用力肺活量（FVC）的绝对变化。有5个次要结果（包括疾病进展时间，其定义为预计FVC下降≥10%或死亡）。探索性结果包括患者报告的症状。报告了不良事件。

结果

356例患者（平均年龄70.5岁；258人[72.5%]为男性；221名[62.1%]为白人），277名（77.8%）完成了试验。从基线到第48周，FVC的绝对变化没有显著的组间差异（Pamrevlumab组的最小二乘平均值为-260 mL[95%CI为-350至-170 mL]，安慰剂组为-330 mL[95%CI为-430至-230 mL]；平均组间差异，70 mL[95%CI，-60至190 mL]，P=.29）。在任何次要结果或患者报告的结果中，没有显著的组间差异。在Pamrevlumab组中，有160名患者（88.4%）出现治疗相关不良事件，51名患者（28.2%）出现严重不良事件，而安慰剂组分别为151名（86.3%）和60名（34.3%）。在研究期间，每组均有23名患者死亡（Pamrevlumab组为12.7%，安慰剂组为13.1%）。

结论和相关性

在接受Pamrevlumab或安慰剂治疗的特发性肺纤维化患者中，从基线到第48周的FVC绝对变化的主要转归无统计学显著的组间差异。

试用注册

ClinicalTrials.gov标识符：NCT03955146。

英文原文如下：

Abstracts

Importance  Current treatments for idiopathic pulmonary fibrosis slow the rate of lung function decline, but may be associated with adverse events that affect medication adherence. In phase 2 trials, pamrevlumab (a fully human monoclonal antibody that binds to and inhibits connective tissue growth factor activity) attenuated the progression of idiopathic pulmonary fibrosis without substantial adverse events.

Objective  To assess the efficacy and safety of pamrevlumab for patients with idiopathic pulmonary fibrosis.

Design, Setting, and Participants  Phase 3 randomized clinical trial including 356 patients aged 40 to 85 years with idiopathic pulmonary fibrosis who were not receiving antifibrotic treatment with nintedanib or pirfenidone at enrollment. Patients were recruited from 117 sites in 9 countries between July 18, 2019, and July 29, 2022; the last follow-up encounter occurred on August 28, 2023.

Interventions  Pamrevlumab (30 mg/kg administered intravenously every 3 weeks; n = 181) or placebo (n = 175) for 48 weeks.

Main Outcomes and Measures  The primary outcome was absolute change in forced vital capacity (FVC) from baseline to week 48. There were 5 secondary outcomes (including time to disease progression, which was defined as a decline of ≥10% in predicted FVC or death). The exploratory outcomes included patient-reported symptoms. Adverse events were reported.

Results  Among 356 patients (mean age, 70.5 years; 258 [72.5%] were men; 221 [62.1%] were White), 277 (77.8%) completed the trial. There was no significant between-group difference for absolute change in FVC from baseline to week 48 (least-squares mean, -260 mL [95% CI, -350 to -170 mL] in the pamrevlumab group vs -330 mL [95% CI, -430 to -230 mL] in the placebo group; mean between-group difference, 70 mL [95% CI, -60 to 190 mL], P = .29). There were no significant between-group differences in any of the secondary outcomes or in the patient-reported outcomes. In the pamrevlumab group, there were 160 patients (88.4%) with treatment-related adverse events and 51 patients (28.2%) with serious adverse events vs 151 (86.3%) and 60 (34.3%), respectively, in the placebo group. During the study, 23 patients died in each group (12.7% in the pamrevlumab group vs 13.1% in the placebo group).

Conclusions and Relevance  Among patients with idiopathic pulmonary fibrosis treated with pamrevlumab or placebo, there was no statistically significant between-group difference for the primary outcome of absolute change in FVC from baseline to week 48.

Trial Registration  ClinicalTrials.gov Identifier: NCT03955146.

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