A randomised trial of lung sealant versus medical therapy for advanced emphysema

Carolyn E. Come, Mordechai R. Kramer, Mark T. Dransfield, Muhanned Abu-Hijleh, David Berkowitz, Michela Bezzi, Surya P. Bhatt, Michael B. Boyd, Enrique Cases, Alexander C. Chen, Christopher B. Cooper, Javier Flandes, Thomas Gildea, Mark Gotfried, D. Kyle Hogarth, Kumaran Kolandaivelu, William Leeds, Timothy Liesching, Nathaniel Marchetti, Charles Marquette & 17 others Richard A. Mularski, Victor M. Pinto-Plata, Michael A. Pritchett, Samaan Rafeq, Edmundo R. Rubio, Dirk Jan Slebos, Grigoris Stratakos, Alexander Sy, Larry W. Tsai, Momen Wahidi, John Walsh, J. Michael Wells, Patrick E. Whitten, Roger Yusen, Javier J. Zulueta, Gerard J. Criner, George R. Washko

Research output: Contribution to journalArticle

65 Citations (Scopus)

Abstract

Uncontrolled pilot studies demonstrated promising results of endoscopic lung volume reduction using emphysematous lung sealant (ELS) in patients with advanced, upper lobe predominant emphysema. We aimed to evaluate the safety and efficacy of ELS in a randomised controlled setting. Patients were randomised to ELS plus medical treatment or medical treatment alone. Despite early termination for business reasons and inability to assess the primary 12-month end-point, 95 out of 300 patients were successfully randomised, providing sufficient data for 3- and 6-month analysis. 57 patients (34 treatment and 23 control) had efficacy results at 3 months; 34 (21 treatment and 13 control) at 6 months. In the treatment group, 3-month lung function, dyspnoea, and quality of life improved significantly from baseline when compared to control. Improvements persisted at 6 months with >50% of treated patients experiencing clinically important improvements, including some whose lung function improved by >100%. 44% of treated patients experienced adverse events requiring hospitalisation (2.5-fold more than control, p=0.01), with two deaths in the treated cohort. Treatment responders tended to be those experiencing respiratory adverse events. Despite early termination, results show that minimally invasive ELS may be efficacious, yet significant risks (probably inflammatory) limit its current utility.

Original languageEnglish (US)
Pages (from-to)651-662
Number of pages12
JournalEuropean Respiratory Journal
Volume46
Issue number3
DOIs
StatePublished - Sep 1 2015

Fingerprint

Emphysema
Lung
Therapeutics
Pneumonectomy
Dyspnea
Hospitalization
Quality of Life
Safety

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine

Cite this

Come, C. E., Kramer, M. R., Dransfield, M. T., Abu-Hijleh, M., Berkowitz, D., Bezzi, M., ... Washko, G. R. (2015). A randomised trial of lung sealant versus medical therapy for advanced emphysema. European Respiratory Journal, 46(3), 651-662. https://doi.org/10.1183/09031936.00205614

A randomised trial of lung sealant versus medical therapy for advanced emphysema. / Come, Carolyn E.; Kramer, Mordechai R.; Dransfield, Mark T.; Abu-Hijleh, Muhanned; Berkowitz, David; Bezzi, Michela; Bhatt, Surya P.; Boyd, Michael B.; Cases, Enrique; Chen, Alexander C.; Cooper, Christopher B.; Flandes, Javier; Gildea, Thomas; Gotfried, Mark; Hogarth, D. Kyle; Kolandaivelu, Kumaran; Leeds, William; Liesching, Timothy; Marchetti, Nathaniel; Marquette, Charles; Mularski, Richard A.; Pinto-Plata, Victor M.; Pritchett, Michael A.; Rafeq, Samaan; Rubio, Edmundo R.; Slebos, Dirk Jan; Stratakos, Grigoris; Sy, Alexander; Tsai, Larry W.; Wahidi, Momen; Walsh, John; Wells, J. Michael; Whitten, Patrick E.; Yusen, Roger; Zulueta, Javier J.; Criner, Gerard J.; Washko, George R.

In: European Respiratory Journal, Vol. 46, No. 3, 01.09.2015, p. 651-662.

Research output: Contribution to journalArticle

Come, CE, Kramer, MR, Dransfield, MT, Abu-Hijleh, M, Berkowitz, D, Bezzi, M, Bhatt, SP, Boyd, MB, Cases, E, Chen, AC, Cooper, CB, Flandes, J, Gildea, T, Gotfried, M, Hogarth, DK, Kolandaivelu, K, Leeds, W, Liesching, T, Marchetti, N, Marquette, C, Mularski, RA, Pinto-Plata, VM, Pritchett, MA, Rafeq, S, Rubio, ER, Slebos, DJ, Stratakos, G, Sy, A, Tsai, LW, Wahidi, M, Walsh, J, Wells, JM, Whitten, PE, Yusen, R, Zulueta, JJ, Criner, GJ & Washko, GR 2015, 'A randomised trial of lung sealant versus medical therapy for advanced emphysema', European Respiratory Journal, vol. 46, no. 3, pp. 651-662. https://doi.org/10.1183/09031936.00205614
Come, Carolyn E. ; Kramer, Mordechai R. ; Dransfield, Mark T. ; Abu-Hijleh, Muhanned ; Berkowitz, David ; Bezzi, Michela ; Bhatt, Surya P. ; Boyd, Michael B. ; Cases, Enrique ; Chen, Alexander C. ; Cooper, Christopher B. ; Flandes, Javier ; Gildea, Thomas ; Gotfried, Mark ; Hogarth, D. Kyle ; Kolandaivelu, Kumaran ; Leeds, William ; Liesching, Timothy ; Marchetti, Nathaniel ; Marquette, Charles ; Mularski, Richard A. ; Pinto-Plata, Victor M. ; Pritchett, Michael A. ; Rafeq, Samaan ; Rubio, Edmundo R. ; Slebos, Dirk Jan ; Stratakos, Grigoris ; Sy, Alexander ; Tsai, Larry W. ; Wahidi, Momen ; Walsh, John ; Wells, J. Michael ; Whitten, Patrick E. ; Yusen, Roger ; Zulueta, Javier J. ; Criner, Gerard J. ; Washko, George R. / A randomised trial of lung sealant versus medical therapy for advanced emphysema. In: European Respiratory Journal. 2015 ; Vol. 46, No. 3. pp. 651-662.
@article{d4690ab7dcba4cb68be27772b8c83da1,
title = "A randomised trial of lung sealant versus medical therapy for advanced emphysema",
abstract = "Uncontrolled pilot studies demonstrated promising results of endoscopic lung volume reduction using emphysematous lung sealant (ELS) in patients with advanced, upper lobe predominant emphysema. We aimed to evaluate the safety and efficacy of ELS in a randomised controlled setting. Patients were randomised to ELS plus medical treatment or medical treatment alone. Despite early termination for business reasons and inability to assess the primary 12-month end-point, 95 out of 300 patients were successfully randomised, providing sufficient data for 3- and 6-month analysis. 57 patients (34 treatment and 23 control) had efficacy results at 3 months; 34 (21 treatment and 13 control) at 6 months. In the treatment group, 3-month lung function, dyspnoea, and quality of life improved significantly from baseline when compared to control. Improvements persisted at 6 months with >50{\%} of treated patients experiencing clinically important improvements, including some whose lung function improved by >100{\%}. 44{\%} of treated patients experienced adverse events requiring hospitalisation (2.5-fold more than control, p=0.01), with two deaths in the treated cohort. Treatment responders tended to be those experiencing respiratory adverse events. Despite early termination, results show that minimally invasive ELS may be efficacious, yet significant risks (probably inflammatory) limit its current utility.",
author = "Come, {Carolyn E.} and Kramer, {Mordechai R.} and Dransfield, {Mark T.} and Muhanned Abu-Hijleh and David Berkowitz and Michela Bezzi and Bhatt, {Surya P.} and Boyd, {Michael B.} and Enrique Cases and Chen, {Alexander C.} and Cooper, {Christopher B.} and Javier Flandes and Thomas Gildea and Mark Gotfried and Hogarth, {D. Kyle} and Kumaran Kolandaivelu and William Leeds and Timothy Liesching and Nathaniel Marchetti and Charles Marquette and Mularski, {Richard A.} and Pinto-Plata, {Victor M.} and Pritchett, {Michael A.} and Samaan Rafeq and Rubio, {Edmundo R.} and Slebos, {Dirk Jan} and Grigoris Stratakos and Alexander Sy and Tsai, {Larry W.} and Momen Wahidi and John Walsh and Wells, {J. Michael} and Whitten, {Patrick E.} and Roger Yusen and Zulueta, {Javier J.} and Criner, {Gerard J.} and Washko, {George R.}",
year = "2015",
month = "9",
day = "1",
doi = "10.1183/09031936.00205614",
language = "English (US)",
volume = "46",
pages = "651--662",
journal = "European Respiratory Journal",
issn = "0903-1936",
publisher = "European Respiratory Society",
number = "3",

}

TY - JOUR

T1 - A randomised trial of lung sealant versus medical therapy for advanced emphysema

AU - Come, Carolyn E.

AU - Kramer, Mordechai R.

AU - Dransfield, Mark T.

AU - Abu-Hijleh, Muhanned

AU - Berkowitz, David

AU - Bezzi, Michela

AU - Bhatt, Surya P.

AU - Boyd, Michael B.

AU - Cases, Enrique

AU - Chen, Alexander C.

AU - Cooper, Christopher B.

AU - Flandes, Javier

AU - Gildea, Thomas

AU - Gotfried, Mark

AU - Hogarth, D. Kyle

AU - Kolandaivelu, Kumaran

AU - Leeds, William

AU - Liesching, Timothy

AU - Marchetti, Nathaniel

AU - Marquette, Charles

AU - Mularski, Richard A.

AU - Pinto-Plata, Victor M.

AU - Pritchett, Michael A.

AU - Rafeq, Samaan

AU - Rubio, Edmundo R.

AU - Slebos, Dirk Jan

AU - Stratakos, Grigoris

AU - Sy, Alexander

AU - Tsai, Larry W.

AU - Wahidi, Momen

AU - Walsh, John

AU - Wells, J. Michael

AU - Whitten, Patrick E.

AU - Yusen, Roger

AU - Zulueta, Javier J.

AU - Criner, Gerard J.

AU - Washko, George R.

PY - 2015/9/1

Y1 - 2015/9/1

N2 - Uncontrolled pilot studies demonstrated promising results of endoscopic lung volume reduction using emphysematous lung sealant (ELS) in patients with advanced, upper lobe predominant emphysema. We aimed to evaluate the safety and efficacy of ELS in a randomised controlled setting. Patients were randomised to ELS plus medical treatment or medical treatment alone. Despite early termination for business reasons and inability to assess the primary 12-month end-point, 95 out of 300 patients were successfully randomised, providing sufficient data for 3- and 6-month analysis. 57 patients (34 treatment and 23 control) had efficacy results at 3 months; 34 (21 treatment and 13 control) at 6 months. In the treatment group, 3-month lung function, dyspnoea, and quality of life improved significantly from baseline when compared to control. Improvements persisted at 6 months with >50% of treated patients experiencing clinically important improvements, including some whose lung function improved by >100%. 44% of treated patients experienced adverse events requiring hospitalisation (2.5-fold more than control, p=0.01), with two deaths in the treated cohort. Treatment responders tended to be those experiencing respiratory adverse events. Despite early termination, results show that minimally invasive ELS may be efficacious, yet significant risks (probably inflammatory) limit its current utility.

AB - Uncontrolled pilot studies demonstrated promising results of endoscopic lung volume reduction using emphysematous lung sealant (ELS) in patients with advanced, upper lobe predominant emphysema. We aimed to evaluate the safety and efficacy of ELS in a randomised controlled setting. Patients were randomised to ELS plus medical treatment or medical treatment alone. Despite early termination for business reasons and inability to assess the primary 12-month end-point, 95 out of 300 patients were successfully randomised, providing sufficient data for 3- and 6-month analysis. 57 patients (34 treatment and 23 control) had efficacy results at 3 months; 34 (21 treatment and 13 control) at 6 months. In the treatment group, 3-month lung function, dyspnoea, and quality of life improved significantly from baseline when compared to control. Improvements persisted at 6 months with >50% of treated patients experiencing clinically important improvements, including some whose lung function improved by >100%. 44% of treated patients experienced adverse events requiring hospitalisation (2.5-fold more than control, p=0.01), with two deaths in the treated cohort. Treatment responders tended to be those experiencing respiratory adverse events. Despite early termination, results show that minimally invasive ELS may be efficacious, yet significant risks (probably inflammatory) limit its current utility.

UR - http://www.scopus.com/inward/record.url?scp=84941769988&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84941769988&partnerID=8YFLogxK

U2 - 10.1183/09031936.00205614

DO - 10.1183/09031936.00205614

M3 - Article

VL - 46

SP - 651

EP - 662

JO - European Respiratory Journal

JF - European Respiratory Journal

SN - 0903-1936

IS - 3

ER -