Treatment options in idiopathic subglottic stenosis: protocol for a prospective international multicentre pragmatic trial.
Treatment options in idiopathic subglottic stenosis: protocol for a prospective international multicentre pragmatic trial.
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DOI：
10.1136/bmjopen-2018-022243
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发表时间：
2018-04-10
期刊：
影响因子：
2.9
通讯作者：
Francis DO
中科院分区：
文献类型：
--
作者：
Gelbard A;Shyr Y;Berry L;Hillel AT;Ekbom DC;Edell ES;Kasperbauer JL;Lott DG;Donovan DT;Garrett CG;Sandhu G;Daniero JJ;Netterville JL;Schindler JS;Smith ME;Bryson PC;Lorenz RR;Francis DO
特发性声门下狭窄（iSGS）是一种原因不明的进行性上气道阻塞，几乎仅发生在成年白人女性中。这种疾病的特征是粘膜炎症和局部纤维化，导致危及生命的上呼吸道堵塞。由于高复发率，iSGS患者在初次诊断后经常需要多次手术。这种疾病及其治疗方法都深刻地影响着患者的呼吸、交流和吞咽能力。已经有多种治疗方法来控制这种情况。然而，关于独特方法的有效性和副作用的比较数据从未被系统地评估过。本研究将创建一个国际、多机构的iSGS患者前瞻性队列。它将比较三种手术方法，以确定iSGS“工作”中最常用的治疗方法以及与每种方法相关的生活质量（QOL）权衡。一项前瞻性实用性试验，比较了多个国际机构的iSGS“标准治疗”。诊断为iSGS且无血管炎临床或实验室证据或症状发作前2年有气管插管史的患者将入选本研究。将对需要手术干预的疾病复发、经验证的患者报告结局（PRO）指标以及患者生成的健康数据（移动的峰值流量记录和每日步数）进行前瞻性评价，纵向跟踪36个月。主要终点是治疗有效性，定义为至复发手术的时间。次要终点与治疗副作用有关，包括声音、吞咽、呼吸和总体QOL方面的PRO指标以及患者生成的健康数据。本方案于2015年7月获得范德比尔特大学医学中心当地IRB委员会的批准。该试验的结果将通过同行评审期刊、国家和国际会议报告传播，并通过基于社交媒体的支持团体直接向iSGS患者传播。 NCT02481817。
Idiopathic subglottic stenosis (iSGS) is an unexplained progressive obstruction of the upper airway that occurs almost exclusively in adult, Caucasian women. The disease is characterised by mucosal inflammation and localised fibrosis resulting in life-threatening blockage of the upper airway. Because of high recurrence rates, patients with iSGS will frequently require multiple procedures following their initial diagnosis. Both the disease and its therapies profoundly affect patients’ ability to breathe, communicate and swallow. A variety of treatments have been advanced to manage this condition. However, comparative data on effectiveness and side effects of the unique approaches have never been systematically evaluated. This study will create an international, multi-institutional prospective cohort of patients with iSGS. It will compare three surgical approaches to determine how well the most commonly used treatments in iSGS ‘work’ and what quality of life (QOL) trade-offs are associated with each approach. A prospective pragmatic trial comparing the ‘Standard of Care’ for iSGS at multiple international institutions. Patients with a diagnosis of iSGS without clinical or laboratory evidence of vasculitis or a history of endotracheal intubation 2 years prior to symptom onset will be included in the study. Prospective evaluation of disease recurrence requiring operative intervention, validated patient-reported outcome (PRO) measures as well as patient-generated health data (mobile peak flow recordings and daily steps taken) will be longitudinally tracked for 36 months. The primary endpoint is treatment effectiveness defined as time to recurrent operative procedure. Secondary endpoints relate to treatment side effects and include PRO measures in voice, swallowing, breathing and global QOL as well as patient-generated health data. This protocol was approved by the local IRB Committee of the Vanderbilt University Medical Center in July 2015. The findings of the trial will be disseminated through peer-reviewed journals, national and international conference presentations and directly to patient with iSGS via social media-based support groups. NCT02481817.
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DOI：
10.1002/lary.25708
发表时间：
2016-06
期刊：
The Laryngoscope
影响因子：
--
作者：
Gelbard A;Donovan DT;Ongkasuwan J;Nouraei SA;Sandhu G;Benninger MS;Bryson PC;Lorenz RR;Tierney WS;Hillel AT;Gadkaree SK;Lott DG;Edell ES;Ekbom DC;Kasperbauer JL;Maldonado F;Schindler JS;Smith ME;Daniero JJ;Garrett CG;Netterville JL;Rickman OB;Sinard RJ;Wootten CT;Francis DO
通讯作者：
Francis DO
影响因子：
7.2
作者：
Rassen, Jeremy A.;Brookhart, M. Alan;Glynn, Robert J.;Mittleman, Murray A.;Schneeweiss, Sebastian
通讯作者：
Schneeweiss, Sebastian
影响因子：
1.4
作者：
Miller, Claire Kane;Kelchner, Lisa N.;Willging, J. Paul
通讯作者：
Willging, J. Paul
影响因子：
2.6
作者：
Nouraei, S. A. R.;Sandhu, G. S.
通讯作者：
Sandhu, G. S.
影响因子：
3.7
作者：
Angrist, JD;Imbens, GW;Rubin, DB
通讯作者：
Rubin, DB