PMID- 29643170 OWN - NLM STAT- MEDLINE DCOM- 20190227 LR - 20220410 IS - 2044-6055 (Electronic) IS - 2044-6055 (Linking) VI - 8 IP - 4 DP - 2018 Apr 10 TI - Treatment options in idiopathic subglottic stenosis: protocol for a prospective international multicentre pragmatic trial. PG - e022243 LID - 10.1136/bmjopen-2018-022243 [doi] LID - e022243 AB - INTRODUCTION: 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. METHODS AND ANALYSIS: 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. ETHICS AND DISSEMINATION: 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. TRIAL REGISTRATION NUMBER: NCT02481817. CI - (c) Article author(s) (or their employer(s) unless otherwise stated in the text of the article) 2018. All rights reserved. No commercial use is permitted unless otherwise expressly granted. FAU - Gelbard, Alexander AU - Gelbard A AUID- ORCID: 0000-0003-0078-1305 AD - Department of Otolaryngology, Vanderbilt University, Nashville, Tennessee, USA. FAU - Shyr, Yu AU - Shyr Y AD - Department of Biostatistics, Vanderbilt University, Nashville, Tennessee, USA. FAU - Berry, Lynne AU - Berry L AD - Department of Biostatistics, Vanderbilt University, Nashville, Tennessee, USA. FAU - Hillel, Alexander T AU - Hillel AT AD - Department of Otolaryngology, Johns Hopkins University, Baltimore, Maryland, USA. FAU - Ekbom, Dale C AU - Ekbom DC AD - Department of Otolaryngology, Mayo Clinic, Rochester, Minnesota, USA. FAU - Edell, Eric S AU - Edell ES AD - Department of Pulmonology, Mayo Clinic, Rochester, Minnesota, USA. FAU - Kasperbauer, Jan L AU - Kasperbauer JL AD - Department of Otolaryngology, Mayo Clinic, Rochester, Minnesota, USA. FAU - Lott, David G AU - Lott DG AD - Department of Otorhinolaryngology, Mayo Clinic Scottsdale, Scottsdale, Arizona, USA. FAU - Donovan, Donald T AU - Donovan DT AD - Department Otolaryngology, Baylor College of Medicine, Houston, Texas, USA. FAU - Garrett, C Gaelyn AU - Garrett CG AD - Department of Otolaryngology, Vanderbilt University, Nashville, Tennessee, USA. FAU - Sandhu, Guri AU - Sandhu G AD - Department of Otolaryngology, Imperial College Healthcare NHS, London, UK. FAU - Daniero, James J AU - Daniero JJ AD - Department Otolaryngology, University of Virginia Health System, Charlottesville, Virginia, USA. FAU - Netterville, James L AU - Netterville JL AD - Department of Otolaryngology, Vanderbilt University, Nashville, Tennessee, USA. FAU - Schindler, Josh S AU - Schindler JS AD - Department of Otolaryngology, Oregon Health and Science University, Portland, Oregon, USA. FAU - Smith, Marshall E AU - Smith ME AD - Department of Otolaryngology, University of Utah, Salt Lake City, Utah, USA. FAU - Bryson, Paul C AU - Bryson PC AD - Department of Otolaryngology, The Cleveland Clinic, Cleveland, Ohio, USA. FAU - Lorenz, Robert R AU - Lorenz RR AD - Department of Otolaryngology, The Cleveland Clinic, Cleveland, Ohio, USA. FAU - Francis, David O AU - Francis DO AD - Department of Surgery, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin, USA. LA - eng SI - ClinicalTrials.gov/NCT02481817 PT - Clinical Trial Protocol PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20180410 PL - England TA - BMJ Open JT - BMJ open JID - 101552874 SB - IM MH - Adolescent MH - Adult MH - Constriction, Pathologic MH - Female MH - Humans MH - *Laryngostenosis/therapy MH - Larynx MH - Multicenter Studies as Topic MH - Pragmatic Clinical Trials as Topic MH - Prospective Studies MH - *Quality of Life MH - Research Design MH - Treatment Outcome PMC - PMC5898326 OTO - NOTNLM OT - NoAAC OT - PR02 OT - iSGS OT - pragmatic trial OT - subglottic stenosis COIS- Competing interests: None declared. EDAT- 2018/04/13 06:00 MHDA- 2019/02/28 06:00 PMCR- 2018/04/10 CRDT- 2018/04/13 06:00 PHST- 2018/04/13 06:00 [entrez] PHST- 2018/04/13 06:00 [pubmed] PHST- 2019/02/28 06:00 [medline] PHST- 2018/04/10 00:00 [pmc-release] AID - bmjopen-2018-022243 [pii] AID - 10.1136/bmjopen-2018-022243 [doi] PST - epublish SO - BMJ Open. 2018 Apr 10;8(4):e022243. doi: 10.1136/bmjopen-2018-022243.