Treatment options in idiopathic subglottic stenosis: Protocol for a prospective international multicentre pragmatic trial

Alexander Gelbard, Yu Shyr, Lynne Berry, Alexander T. Hillel, Dale C. Ekbom, Eric S. Edell, Jan L. Kasperbauer, David G. Lott, Donald T. Donovan, C. Gaelyn Garrett, Guri Sandhu, James J. Daniero, James L. Netterville, Joshua Schindler, Marshall E. Smith, Paul C. Bryson, Robert R. Lorenz, David O. Francis

Research output: Contribution to journalArticle

3 Citations (Scopus)

Abstract

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.

Original languageEnglish (US)
Article numbere022243
JournalBMJ Open
Volume8
Issue number4
DOIs
StatePublished - Apr 1 2018

Fingerprint

Pragmatic Clinical Trials
Multicenter Studies
Pathologic Constriction
Deglutition
Therapeutics
Quality of Life
Social Media
Recurrence
Intratracheal Intubation
Telemedicine
Self-Help Groups
Research Ethics Committees
Operative Surgical Procedures
Airway Obstruction
Standard of Care
Vasculitis
Ethics
Respiration
Fibrosis
Inflammation

Keywords

  • iSGS
  • NoAAC
  • PR02
  • pragmatic trial
  • subglottic stenosis

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Gelbard, A., Shyr, Y., Berry, L., Hillel, A. T., Ekbom, D. C., Edell, E. S., ... Francis, D. O. (2018). Treatment options in idiopathic subglottic stenosis: Protocol for a prospective international multicentre pragmatic trial. BMJ Open, 8(4), [e022243]. https://doi.org/10.1136/bmjopen-2018-022243

Treatment options in idiopathic subglottic stenosis : Protocol for a prospective international multicentre pragmatic trial. / Gelbard, Alexander; Shyr, Yu; Berry, Lynne; Hillel, Alexander T.; Ekbom, Dale C.; Edell, Eric S.; Kasperbauer, Jan L.; Lott, David G.; Donovan, Donald T.; Garrett, C. Gaelyn; Sandhu, Guri; Daniero, James J.; Netterville, James L.; Schindler, Joshua; Smith, Marshall E.; Bryson, Paul C.; Lorenz, Robert R.; Francis, David O.

In: BMJ Open, Vol. 8, No. 4, e022243, 01.04.2018.

Research output: Contribution to journalArticle

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, J, Smith, ME, Bryson, PC, Lorenz, RR & Francis, DO 2018, 'Treatment options in idiopathic subglottic stenosis: Protocol for a prospective international multicentre pragmatic trial', BMJ Open, vol. 8, no. 4, e022243. https://doi.org/10.1136/bmjopen-2018-022243
Gelbard, Alexander ; Shyr, Yu ; Berry, Lynne ; Hillel, Alexander T. ; Ekbom, Dale C. ; Edell, Eric S. ; Kasperbauer, Jan L. ; Lott, David G. ; Donovan, Donald T. ; Garrett, C. Gaelyn ; Sandhu, Guri ; Daniero, James J. ; Netterville, James L. ; Schindler, Joshua ; Smith, Marshall E. ; Bryson, Paul C. ; Lorenz, Robert R. ; Francis, David O. / Treatment options in idiopathic subglottic stenosis : Protocol for a prospective international multicentre pragmatic trial. In: BMJ Open. 2018 ; Vol. 8, No. 4.
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N2 - 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.

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