Risk-stratified treatment for drug-susceptible pulmonary tuberculosis

Abstract The Phase 3 randomized controlled trial, TBTC Study 31/ACTG A5349 (NCT02410772) demonstrated that a 4-month rifapentine-moxifloxacin regimen for drug-susceptible pulmonary tuberculosis was safe and effective. The primary efficacy outcome was 12-month tuberculosis disease free survival, whil...

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Main Authors: Vincent K. Chang, Marjorie Z. Imperial, Patrick P. J. Phillips, Gustavo E. Velásquez, Payam Nahid, Andrew Vernon, Ekaterina V. Kurbatova, Susan Swindells, Richard E. Chaisson, Susan E. Dorman, John L. Johnson, Marc Weiner, Amina Jindani, Thomas Harrison, Erin E. Sizemore, William Whitworth, Wendy Carr, Kia E. Bryant, Deron Burton, Kelly E. Dooley, Melissa Engle, Pheona Nsubuga, Andreas H. Diacon, Nguyen Viet Nhung, Rodney Dawson, Radojka M. Savic, AIDS Clinical Trial Group, Tuberculosis Trials Consortium
Format: Article
Language:English
Published: Nature Portfolio 2024-10-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-024-53273-7
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author Vincent K. Chang
Marjorie Z. Imperial
Patrick P. J. Phillips
Gustavo E. Velásquez
Payam Nahid
Andrew Vernon
Ekaterina V. Kurbatova
Susan Swindells
Richard E. Chaisson
Susan E. Dorman
John L. Johnson
Marc Weiner
Amina Jindani
Thomas Harrison
Erin E. Sizemore
William Whitworth
Wendy Carr
Kia E. Bryant
Deron Burton
Kelly E. Dooley
Melissa Engle
Pheona Nsubuga
Andreas H. Diacon
Nguyen Viet Nhung
Rodney Dawson
Radojka M. Savic
AIDS Clinical Trial Group
Tuberculosis Trials Consortium
author_facet Vincent K. Chang
Marjorie Z. Imperial
Patrick P. J. Phillips
Gustavo E. Velásquez
Payam Nahid
Andrew Vernon
Ekaterina V. Kurbatova
Susan Swindells
Richard E. Chaisson
Susan E. Dorman
John L. Johnson
Marc Weiner
Amina Jindani
Thomas Harrison
Erin E. Sizemore
William Whitworth
Wendy Carr
Kia E. Bryant
Deron Burton
Kelly E. Dooley
Melissa Engle
Pheona Nsubuga
Andreas H. Diacon
Nguyen Viet Nhung
Rodney Dawson
Radojka M. Savic
AIDS Clinical Trial Group
Tuberculosis Trials Consortium
author_sort Vincent K. Chang
collection DOAJ
description Abstract The Phase 3 randomized controlled trial, TBTC Study 31/ACTG A5349 (NCT02410772) demonstrated that a 4-month rifapentine-moxifloxacin regimen for drug-susceptible pulmonary tuberculosis was safe and effective. The primary efficacy outcome was 12-month tuberculosis disease free survival, while the primary safety outcome was the proportion of grade 3 or higher adverse events during the treatment period. We conducted an analysis of demographic, clinical, microbiologic, radiographic, and pharmacokinetic data and identified risk factors for unfavorable outcomes and adverse events. Among participants receiving the rifapentine-moxifloxacin regimen, low rifapentine exposure is the strongest driver of tuberculosis-related unfavorable outcomes (HR 0.65 for every 100 µg∙h/mL increase, 95%CI 0.54–0.77). The only other risk factors identified are markers of higher baseline disease severity, namely Xpert MTB/RIF cycle threshold and extent of disease on baseline chest radiography (Xpert: HR 1.43 for every 3-cycle-threshold decrease, 95%CI 1.07–1.91; extensive disease: HR 2.02, 95%CI 1.07–3.82). From these risk factors, we developed a simple risk stratification to classify disease phenotypes as easier-, moderately-harder, or harder-to-treat TB. Notably, high rifapentine exposures are not associated with any predefined adverse safety outcomes. Our results suggest that the easier-to-treat subgroup may be eligible for further treatment shortening while the harder-to-treat subgroup may need higher doses or longer treatment.
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spelling doaj-art-de940f5d4bbc4e00aaa1dfc2d65e721a2025-08-20T02:25:12ZengNature PortfolioNature Communications2041-17232024-10-0115111210.1038/s41467-024-53273-7Risk-stratified treatment for drug-susceptible pulmonary tuberculosisVincent K. Chang0Marjorie Z. Imperial1Patrick P. J. Phillips2Gustavo E. Velásquez3Payam Nahid4Andrew Vernon5Ekaterina V. Kurbatova6Susan Swindells7Richard E. Chaisson8Susan E. Dorman9John L. Johnson10Marc Weiner11Amina Jindani12Thomas Harrison13Erin E. Sizemore14William Whitworth15Wendy Carr16Kia E. Bryant17Deron Burton18Kelly E. Dooley19Melissa Engle20Pheona Nsubuga21Andreas H. Diacon22Nguyen Viet Nhung23Rodney Dawson24Radojka M. Savic25AIDS Clinical Trial GroupTuberculosis Trials ConsortiumDepartment of Bioengineering and Therapeutic Sciences, University of California San FranciscoDepartment of Bioengineering and Therapeutic Sciences, University of California San FranciscoUCSF Center for Tuberculosis, University of California San FranciscoUCSF Center for Tuberculosis, University of California San FranciscoUCSF Center for Tuberculosis, University of California San FranciscoCenters for Disease Control and PreventionCenters for Disease Control and PreventionUniversity of Nebraska Medical CenterCenter for Tuberculosis Research, Johns Hopkins University School of MedicineMedical University of South CarolinaCase Western Reserve University, University Hospitals Cleveland Medical CenterUniversity of Texas Health Science Center at San Antonio and the South Texas Veterans Health Care SystemSt. George’s, University of LondonSt. George’s, University of LondonCenters for Disease Control and PreventionCenters for Disease Control and PreventionCenters for Disease Control and PreventionCenters for Disease Control and PreventionCenters for Disease Control and PreventionDivision of Infectious Diseases, Vanderbilt UniversityUniversity of Texas Health Science Center at San Antonio and the South Texas Veterans Health Care SystemUganda–Case Western Reserve University Research CollaborationTASK, Cape Town, Western CapeVietnam National Tuberculosis Program–University of California, San Francisco Research Collaboration UnitLung Institute and Division of Pulmonology, Department of Medicine, University of Cape TownDepartment of Bioengineering and Therapeutic Sciences, University of California San FranciscoAbstract The Phase 3 randomized controlled trial, TBTC Study 31/ACTG A5349 (NCT02410772) demonstrated that a 4-month rifapentine-moxifloxacin regimen for drug-susceptible pulmonary tuberculosis was safe and effective. The primary efficacy outcome was 12-month tuberculosis disease free survival, while the primary safety outcome was the proportion of grade 3 or higher adverse events during the treatment period. We conducted an analysis of demographic, clinical, microbiologic, radiographic, and pharmacokinetic data and identified risk factors for unfavorable outcomes and adverse events. Among participants receiving the rifapentine-moxifloxacin regimen, low rifapentine exposure is the strongest driver of tuberculosis-related unfavorable outcomes (HR 0.65 for every 100 µg∙h/mL increase, 95%CI 0.54–0.77). The only other risk factors identified are markers of higher baseline disease severity, namely Xpert MTB/RIF cycle threshold and extent of disease on baseline chest radiography (Xpert: HR 1.43 for every 3-cycle-threshold decrease, 95%CI 1.07–1.91; extensive disease: HR 2.02, 95%CI 1.07–3.82). From these risk factors, we developed a simple risk stratification to classify disease phenotypes as easier-, moderately-harder, or harder-to-treat TB. Notably, high rifapentine exposures are not associated with any predefined adverse safety outcomes. Our results suggest that the easier-to-treat subgroup may be eligible for further treatment shortening while the harder-to-treat subgroup may need higher doses or longer treatment.https://doi.org/10.1038/s41467-024-53273-7
spellingShingle Vincent K. Chang
Marjorie Z. Imperial
Patrick P. J. Phillips
Gustavo E. Velásquez
Payam Nahid
Andrew Vernon
Ekaterina V. Kurbatova
Susan Swindells
Richard E. Chaisson
Susan E. Dorman
John L. Johnson
Marc Weiner
Amina Jindani
Thomas Harrison
Erin E. Sizemore
William Whitworth
Wendy Carr
Kia E. Bryant
Deron Burton
Kelly E. Dooley
Melissa Engle
Pheona Nsubuga
Andreas H. Diacon
Nguyen Viet Nhung
Rodney Dawson
Radojka M. Savic
AIDS Clinical Trial Group
Tuberculosis Trials Consortium
Risk-stratified treatment for drug-susceptible pulmonary tuberculosis
Nature Communications
title Risk-stratified treatment for drug-susceptible pulmonary tuberculosis
title_full Risk-stratified treatment for drug-susceptible pulmonary tuberculosis
title_fullStr Risk-stratified treatment for drug-susceptible pulmonary tuberculosis
title_full_unstemmed Risk-stratified treatment for drug-susceptible pulmonary tuberculosis
title_short Risk-stratified treatment for drug-susceptible pulmonary tuberculosis
title_sort risk stratified treatment for drug susceptible pulmonary tuberculosis
url https://doi.org/10.1038/s41467-024-53273-7
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