Hypertonic Saline for Cystic Fibrosis

高渗盐水治疗囊性纤维化

• 批准号: 8199638
• 负责人: Benjamin Yerxa
• 金额: $ 24.48万
• 依托单位: PARION SCIENCES, INC.
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-09-01 至 2013-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8199638
• 关键词: Adhesions; Aerosols; Affect; Breathing; CLCA2 gene; Cannulas; Caring; Chloride Channels; Cilia; Clinical; Clinical Research; Clinical Trials; Clinical Trials Design; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Data; Dehydration; Deposition; Devices; Disease; Dose; Ensure; Epithelial; Epithelium; Excision; Exhibits; Goals; Hereditary Disease; Hour; Hydration status; Individual; Infection; Infectious Agent; Ion Channel; Left; Lubricants; Lung; Maintenance; Measures; Mucociliary Clearance; Mucous body substance; Nebulizer; Nose; Oxygen Therapy Care; Patients; Pattern; Performance; Physical therapy; Property; Publishing; Pulmonary Cystic Fibrosis; Quality of life; Regimen; Rehydrations; Relative (related person); Respiratory physiology; Safety; Saline; Science; Sleep; Sodium Channel; Sodium Chloride; Sputum; Surface; System; Testing; Therapeutic; Time; Treatment Protocols; Water; absorption; adverse outcome; aerosolized; airway inflammation; airway surface liquid; apical membrane; clinical efficacy; compliance behavior; cystic fibrosis airway; cystic fibrosis patients; design; effective therapy; epithelial Na+ channel; improved; irritation; meetings; mortality; novel; programs; prototype; pulmonary function; radiotracer; response; standard of care; therapy development; water flow

DESCRIPTION (provided by applicant): Cystic fibrosis (CF) is an autosomal lethal disease characterized that affects ~30,000 individuals in the US. CF lung disease, the major cause of mortality in CF, is caused by dehydration of airway secretions that leads to mucous adhesion, infection and airways inflammation. A simple means to restore hydration and mucous clearance in CF airways is the inhalation of hypertonic saline (HS), which osmotically draws water onto the airway surface. Rehydration of the lubricant layer of the airway surface liquid facilitates mucociliary clearance (MCC) and therefore the removal of inhaled infectious agents. Previously published evidence has shown that short-term administration of HS improves the rheologic properties and transportability of sputum, hydrates the airway surface, and improves MCC and lung function in patients with CF. Recent clinical studies support the efficacy of daily HS treatments for CF demonstrating improvements in lung function, a reduction in pulmonary exacerbations, and improvements in quality of life. As a result, inhaled HS is becoming a standard of care for CF and is now used by ~60% of patients nationwide. One consequence of the addition of HS to CF treatment regimens is the time required by CF patients for its administration. Daily HS treatments require ~1 hour of time commitment, which is superimposed on the ~2 hours of care typically asked of these patients for other aerosols and physiotherapy. Thus, there is a great need to reduce the treatment burden (time commitment) of this therapy to improve the quality of life of CF patients. Parion Science's goal is to develop therapies that provide major improvements in efficacy over current treatments for CF. Parion is developing a specialized device of overnight administration of HS that will reduce daytime treatment requirements for CF patients. Parion has completed a device prototype which has been designed to administer a therapeutic dose of HS overnight that is comfortable, quiet, and compatible with sleep. Parion will test the tolerability and efficacy of this novel HS regimen in clinical trials. If successful, we believe that this approach will improve the quality of life of CF patients by decreasing the treatment burden associated with daily HS treatments and improve compliance of this therapy. PUBLIC HEALTH RELEVANCE: Cystic Fibrosis (CF) is one of the most common lethal genetic disorders in the US. Inhaled hypertonic saline is a simple, safe, and effective therapy for CF, but it also adds a significant time burden to patients, requiring an additional hour per day on top of the ~2 hours spent taking other treatments. Parion Sciences is developing a device to administer hypertonic saline overnight to patients in order to reduce the treatment burden and improve compliance with this therapy.

描述（由申请人提供）：囊性纤维化 (CF) 是一种常染色体致死性疾病，影响美国约 30,000 人。 CF 肺部疾病是 CF 死亡的主要原因，是由气道分泌物脱水导致粘液粘连、感染和气道炎症引起的。恢复 CF 气道水合作用和粘液清除的一个简单方法是吸入高渗盐水 (HS)，它通过渗透作用将水吸到气道表面。气道表面液体润滑层的再水化有利于粘膜纤毛清除（MCC），从而清除吸入的传染源。 先前发表的证据表明，短期给予 HS 可改善痰液的流变特性和输送性，使气道表面水合，并改善 CF 患者的 MCC 和肺功能。最近的临床研究支持每日 HS 治疗 CF 的疗效，证明其可改善肺功能、减少肺部病情加重并改善生活质量。因此，吸入 HS 正在成为 CF 的护理标准，目前全国约有 60% 的患者使用。 CF 治疗方案中添加 HS 的后果之一是 CF 患者给药所需的时间。每日 HS 治疗需要约 1 小时的时间投入，这与这些患者通常要求的约 2 小时的其他气雾剂和物理治疗护理时间相叠加。因此，非常需要减轻这种疗法的治疗负担（时间投入）以提高CF患者的生活质量。 Parion Science 的目标是开发一种疗法，与目前的囊性纤维化治疗方法相比，在疗效上有重大改进。 Parion 正在开发一种夜间给予 HS 的专用设备，这将减少 CF 患者的日间治疗需求。 Parion 已经完成了一个设备原型，该原型设计用于在夜间施用治疗剂量的 HS，该设备舒适、安静且与睡眠相容。 Parion 将在临床试验中测试这种新型 HS 方案的耐受性和有效性。如果成功，我们相信这种方法将通过减少与日常 HS 治疗相关的治疗负担来改善 CF 患者的生活质量，并提高这种治疗的依从性。 公共卫生相关性：囊性纤维化 (CF) 是美国最常见的致命遗传性疾病之一。吸入高渗盐水是一种简单、安全、有效的 CF 治疗方法，但它也给患者增加了显着的时间负担，除了接受其他治疗约 2 小时外，每天还需要额外的时间。 Parion Sciences 正在开发一种设备，可在夜间向患者注射高渗盐水，以减轻治疗负担并提高对该疗法的依从性。