A randomized trial of low-dose thrombolysis, ultrasound-assisted thrombolysis, or heparin for intermediate-high risk pulmonary embolism—the STRATIFY trial: design and statistical analysis plan

Abstract Background Intermediate-high risk pulmonary embolism (PE) carries a significant risk of hemodynamic deterioration or death. Treatment should balance efficacy in reducing clot burden with the risk of complications, particularly bleeding. Previous studies on high-dose, short-term thrombolysis...

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Main Authors: Jesper Kjærgaard, Jørn Carlsen, Emilie Sonne-Holm, Sebastian Wiberg, Lene Holmvang, Jens Flensted Lassen, Rikke Sørensen, Dan Høfsten, Peter Sommer Ulriksen, Samir Jawad, Pernille Palm, Jens Jakob Thune, Christian Hassager, Ole P. Kristiansen, Kristian Eskesen, Søren Fanø, Lia E. Bang
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Language:English
Published: BMC 2024-12-01
Series:Trials
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Online Access:https://doi.org/10.1186/s13063-024-08688-4
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author Jesper Kjærgaard
Jørn Carlsen
Emilie Sonne-Holm
Sebastian Wiberg
Lene Holmvang
Jens Flensted Lassen
Rikke Sørensen
Dan Høfsten
Peter Sommer Ulriksen
Samir Jawad
Pernille Palm
Jens Jakob Thune
Christian Hassager
Ole P. Kristiansen
Kristian Eskesen
Søren Fanø
Lia E. Bang
author_facet Jesper Kjærgaard
Jørn Carlsen
Emilie Sonne-Holm
Sebastian Wiberg
Lene Holmvang
Jens Flensted Lassen
Rikke Sørensen
Dan Høfsten
Peter Sommer Ulriksen
Samir Jawad
Pernille Palm
Jens Jakob Thune
Christian Hassager
Ole P. Kristiansen
Kristian Eskesen
Søren Fanø
Lia E. Bang
author_sort Jesper Kjærgaard
collection DOAJ
description Abstract Background Intermediate-high risk pulmonary embolism (PE) carries a significant risk of hemodynamic deterioration or death. Treatment should balance efficacy in reducing clot burden with the risk of complications, particularly bleeding. Previous studies on high-dose, short-term thrombolysis with alteplase (rtPA) showed a reduced risk of hemodynamic deterioration but no change in mortality and increased bleeding complications. Catheter-based techniques, including ultrasound-assisted thrombolysis (USAT), and low-dose thrombolysis may offer reasonable efficacy with lower risk. However, studies comparing these methods have been few. This trial aims to address this gap by randomizing patients to three treatment modalities. Methods Multicenter, randomized trial with 1:1:1 allocation of 210 patients with acute intermediate-high risk PE, excluding those with absolute contraindications to thrombolysis. Patients are eligible for inclusion if they are > 18 years of age, have had symptoms < 14 days, and are able to give informed consent. Patients are allocated 1:1:1 into three treatment strategies: (1) unfractionated heparin (UFH)/low molecular weight heparin (LMWH), (2) UFH/LMWH + 20 mg rtPA/6 h intravenously (IV), or (3) UFH + 20 mg rtPA/6 h via USAT. Co-primary outcomes include reduction in clot burden as assessed by refined Miller score from pre-treatment to follow-up (48–96 h) computed tomography pulmonary angiogram (CTPA) comparing low-dose rtPA (± USAT) groups to UFH/LMWH group (p < 0.01, N = 210) and reduction in refined Miller score on follow-up CT angiography comparing low-dose rtPA by USAT to intravenous rtPA, p < 0.04, N = 140). Secondary outcomes comprise bleeding complications, duration of index admission, FiO2, blood pressure, respiratory and heart rate at the time of follow-up CT angiography, mortality in the three groups, incidence of tricuspid regurgitation pressure gradient < 40 mmHg at 3 months follow-up echocardiography, 6-min walk test at 3 months comparing the three groups, and health-related quality of life at 3 months follow-up comparing the three groups. Discussion We hypothesize that in patients with intermediate-high risk PE (1) administration of 20 mg rtPA leads to a greater reduction in clot burden compared to heparins and (2) administration of 20 mg rtPA via USAT results in a greater reduction in clot burden compared to 20 mg rtPA intravenous. Trial registration ClinicalTrials.gov NCT04088292. Registered in September 2019 (retrospectively registered).
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spelling doaj-art-0da6d6b2090e43edb417b2d64acacd2f2024-12-29T12:46:12ZengBMCTrials1745-62152024-12-0125111210.1186/s13063-024-08688-4A randomized trial of low-dose thrombolysis, ultrasound-assisted thrombolysis, or heparin for intermediate-high risk pulmonary embolism—the STRATIFY trial: design and statistical analysis planJesper Kjærgaard0Jørn Carlsen1Emilie Sonne-Holm2Sebastian Wiberg3Lene Holmvang4Jens Flensted Lassen5Rikke Sørensen6Dan Høfsten7Peter Sommer Ulriksen8Samir Jawad9Pernille Palm10Jens Jakob Thune11Christian Hassager12Ole P. Kristiansen13Kristian Eskesen14Søren Fanø15Lia E. Bang16Department of Cardiology, The Heart Centre, Copenhagen University Hospital RigshospitaletDepartment of Cardiology, The Heart Centre, Copenhagen University Hospital RigshospitaletDepartment of Cardiology, The Heart Centre, Copenhagen University Hospital RigshospitaletDepartment of Cardiothoracic Anesthesiology, Copenhagen University Hospital RigshospitaletDepartment of Cardiology, The Heart Centre, Copenhagen University Hospital RigshospitaletDepartment of Cardiology, Odense University HospitalDepartment of Cardiology, The Heart Centre, Copenhagen University Hospital RigshospitaletDepartment of Cardiology, The Heart Centre, Copenhagen University Hospital RigshospitaletDepartpent of Radiology, Copenhagen University Hospital RigshospitaletDepartpent of Radiology, Copenhagen University Hospital RigshospitaletDepartment of Cardiology, The Heart Centre, Copenhagen University Hospital RigshospitaletDepartment of Cardiology, Copenhagen University Hospital Bispebjerg and FrederiksbergDepartment of Cardiology, The Heart Centre, Copenhagen University Hospital RigshospitaletDepartment of Cardiology, Copenhagen University Hospital Bispebjerg and FrederiksbergDepartment of Cardiology, The Heart Centre, Copenhagen University Hospital RigshospitaletDepartment of Cardiology, The Heart Centre, Copenhagen University Hospital RigshospitaletDepartment of Cardiology, The Heart Centre, Copenhagen University Hospital RigshospitaletAbstract Background Intermediate-high risk pulmonary embolism (PE) carries a significant risk of hemodynamic deterioration or death. Treatment should balance efficacy in reducing clot burden with the risk of complications, particularly bleeding. Previous studies on high-dose, short-term thrombolysis with alteplase (rtPA) showed a reduced risk of hemodynamic deterioration but no change in mortality and increased bleeding complications. Catheter-based techniques, including ultrasound-assisted thrombolysis (USAT), and low-dose thrombolysis may offer reasonable efficacy with lower risk. However, studies comparing these methods have been few. This trial aims to address this gap by randomizing patients to three treatment modalities. Methods Multicenter, randomized trial with 1:1:1 allocation of 210 patients with acute intermediate-high risk PE, excluding those with absolute contraindications to thrombolysis. Patients are eligible for inclusion if they are > 18 years of age, have had symptoms < 14 days, and are able to give informed consent. Patients are allocated 1:1:1 into three treatment strategies: (1) unfractionated heparin (UFH)/low molecular weight heparin (LMWH), (2) UFH/LMWH + 20 mg rtPA/6 h intravenously (IV), or (3) UFH + 20 mg rtPA/6 h via USAT. Co-primary outcomes include reduction in clot burden as assessed by refined Miller score from pre-treatment to follow-up (48–96 h) computed tomography pulmonary angiogram (CTPA) comparing low-dose rtPA (± USAT) groups to UFH/LMWH group (p < 0.01, N = 210) and reduction in refined Miller score on follow-up CT angiography comparing low-dose rtPA by USAT to intravenous rtPA, p < 0.04, N = 140). Secondary outcomes comprise bleeding complications, duration of index admission, FiO2, blood pressure, respiratory and heart rate at the time of follow-up CT angiography, mortality in the three groups, incidence of tricuspid regurgitation pressure gradient < 40 mmHg at 3 months follow-up echocardiography, 6-min walk test at 3 months comparing the three groups, and health-related quality of life at 3 months follow-up comparing the three groups. Discussion We hypothesize that in patients with intermediate-high risk PE (1) administration of 20 mg rtPA leads to a greater reduction in clot burden compared to heparins and (2) administration of 20 mg rtPA via USAT results in a greater reduction in clot burden compared to 20 mg rtPA intravenous. Trial registration ClinicalTrials.gov NCT04088292. Registered in September 2019 (retrospectively registered).https://doi.org/10.1186/s13063-024-08688-4Pulmonary embolismIntermediate-high riskLow-dose thrombolysisUltrasound-assisted thrombolysisClot burden
spellingShingle Jesper Kjærgaard
Jørn Carlsen
Emilie Sonne-Holm
Sebastian Wiberg
Lene Holmvang
Jens Flensted Lassen
Rikke Sørensen
Dan Høfsten
Peter Sommer Ulriksen
Samir Jawad
Pernille Palm
Jens Jakob Thune
Christian Hassager
Ole P. Kristiansen
Kristian Eskesen
Søren Fanø
Lia E. Bang
A randomized trial of low-dose thrombolysis, ultrasound-assisted thrombolysis, or heparin for intermediate-high risk pulmonary embolism—the STRATIFY trial: design and statistical analysis plan
Trials
Pulmonary embolism
Intermediate-high risk
Low-dose thrombolysis
Ultrasound-assisted thrombolysis
Clot burden
title A randomized trial of low-dose thrombolysis, ultrasound-assisted thrombolysis, or heparin for intermediate-high risk pulmonary embolism—the STRATIFY trial: design and statistical analysis plan
title_full A randomized trial of low-dose thrombolysis, ultrasound-assisted thrombolysis, or heparin for intermediate-high risk pulmonary embolism—the STRATIFY trial: design and statistical analysis plan
title_fullStr A randomized trial of low-dose thrombolysis, ultrasound-assisted thrombolysis, or heparin for intermediate-high risk pulmonary embolism—the STRATIFY trial: design and statistical analysis plan
title_full_unstemmed A randomized trial of low-dose thrombolysis, ultrasound-assisted thrombolysis, or heparin for intermediate-high risk pulmonary embolism—the STRATIFY trial: design and statistical analysis plan
title_short A randomized trial of low-dose thrombolysis, ultrasound-assisted thrombolysis, or heparin for intermediate-high risk pulmonary embolism—the STRATIFY trial: design and statistical analysis plan
title_sort randomized trial of low dose thrombolysis ultrasound assisted thrombolysis or heparin for intermediate high risk pulmonary embolism the stratify trial design and statistical analysis plan
topic Pulmonary embolism
Intermediate-high risk
Low-dose thrombolysis
Ultrasound-assisted thrombolysis
Clot burden
url https://doi.org/10.1186/s13063-024-08688-4
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