口服埃弗罗利斯在异常性亚结节狭窄症扩张后:一期非随机临床试验
Raymond J So1, Samuel L Collins1, Sarah Collins2
1Department of Otolaryngology-Head and Neck Surgery, Johns Hopkins University School of Medicine, Baltimore, Maryland.
JAMA otolaryngology-- head & neck surgery
|September 26, 2024
概括
作为一种mTOR抑制剂的Everolimus通过改善空气流量和耐受性,在一期治疗异常性小结肠狭窄症 (iSGS) 的试验中表现有前途. 建议进一步进行2期试验,以确认iSGS患者的疗效和安全性.
科学领域:
- 肺部病理学 肺部病理学
- 药理学 药理学是指药理学的学科.
- 临床试验 临床试验
背景情况:
- 目前针对异常性小结节狭窄症 (iSGS) 的治疗方法不足以防止痕组织的形成和进展.
- 作为mTOR抑制剂的Everolimus在各种纤维增殖条件和临床前iSGS模型中显示出抗纤维作用.
研究的目的:
- 评估口服埃弗罗利斯的安全性和初步疗效,以减少ISGS患者手术后狭窄症复发的可能性.
主要方法:
- 一个开放标签,单臂,第一阶段,非随机临床试验,涉及7名参与者与iSGS.
- 参与者在扩张手术后42天内每天接受1.5毫克口服剂量的恒利木斯.
- 通过不良事件评估安全性,二级结局包括高峰呼气流量,CT测量亮面积和180天的生命质量得分.
主要成果:
- 总体而言,Everolimus耐受性良好,报告了轻微的不良事件 (口腔,尿道感染,每个1名参与者的皮肤感染).
- 参与者在手术后的13周内保持了改善的峰值呼气流.
- 从第14天到第180天,CT测量的光线面积略有减少,这表明狭窄症可能复发.
结论:
- 辅助性口服Everolimus似乎是iSGS患者手术后的安全和耐受的治疗选择.
- 观察到改善空气流量的维持支持进一步调查everolimus的疗效.
- 结果要求进行第二阶段试验,以全面评估everolimus在iSGS中的有效性和安全性.
相关概念视频
Clinical Trials: Overview
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists
Endothelins (ETs) are potent vasoactive peptides critical in the human body's various physiological and pathological processes. One of the most promising therapeutic strategies for treating pulmonary arterial hypertension (PAH) involves counteracting the effects of these endothelins using a class of drugs known as endothelin receptor antagonists.
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme (ECE). Of...
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme (ECE). Of...
Endoscopic Studies I: Bronchoscopy and Thoracoscopy
Endoscopy is a non-surgical medical technique used to examine a person's internal organs and vessels. This lesson will focus on two types of endoscopic studies: bronchoscopy and thoracoscopy.
Bronchoscopy
Description
Bronchoscopy is a procedure that involves direct visualization of the larynx, trachea, and bronchi for diagnostic and therapeutic purposes. A flexible fiber optic or rigid bronchoscope is used to carry out the procedure. The fiber-optic bronchoscope is more frequently used due to...
Bronchoscopy
Description
Bronchoscopy is a procedure that involves direct visualization of the larynx, trachea, and bronchi for diagnostic and therapeutic purposes. A flexible fiber optic or rigid bronchoscope is used to carry out the procedure. The fiber-optic bronchoscope is more frequently used due to...
Esophageal Strictures-I: Introduction
Esophageal strictures involve abnormal narrowing or tightening of the esophagus. They vary in length and severity, ranging from mild constriction to complete obstruction, and are classified as benign (noncancerous) or malignant (cancerous).
Etiology
The primary cause of esophageal strictures is long-standing gastroesophageal reflux disease (GERD), accounting for about 70 to 80% of adult cases. Chronic acid reflux can lead to injury and scarring of the esophageal lining, culminating in...
Etiology
The primary cause of esophageal strictures is long-standing gastroesophageal reflux disease (GERD), accounting for about 70 to 80% of adult cases. Chronic acid reflux can lead to injury and scarring of the esophageal lining, culminating in...
Esophageal Strictures-II: Clinical Features and Management
Patients with esophageal strictures often experience a range of symptoms. Initially, they may have difficulty swallowing solid foods, which can progress to include liquids. Additional symptoms may involve chest pain or discomfort, regurgitating food and fluids, heartburn, unintentional weight loss, coughing or choking during meals, and hoarseness.
Healthcare providers should gather a comprehensive medical history and conduct a physical examination for diagnosis. If esophageal stricture is...
Healthcare providers should gather a comprehensive medical history and conduct a physical examination for diagnosis. If esophageal stricture is...
Mitral Stenosis III: Medical Management
Mitral stenosis, a condition marked by the narrowing of the mitral valve, necessitates an integrated approach for effective management. This approach includes preventative measures, medical therapy, and surgical interventions to reduce symptoms and prevent complications.PreventionPrevention of mitral stenosis primarily focuses on reducing the incidence of bacterial infections, particularly streptococcal infections, which can lead to rheumatic fever and subsequent valvular damage. Timely...


