Minimum clinically important difference in Quantitative Lung Fibrosis score associated with all-cause mortality in idiopathic pulmonary fibrosis: subanalysis from two phase II trials of pamrevlumab

Objectives Idiopathic pulmonary fibrosis (IPF) is a progressive interstitial lung disease. Chest high-resolution CT (HRCT) is instrumental in IPF management, and the Quantitative Lung Fibrosis (QLF) score is a computer-assisted metric for quantifying lung disease using HRCT. This study aimed to asse...

Full description

Saved in:
Bibliographic Details
Main Authors: Matthew S Brown, Jonathan Goldin, Grace Hyun Kim, Xueping Zhang, Lona Poole
Format: Article
Language:English
Published: BMJ Publishing Group 2025-05-01
Series:BMJ Open
Online Access:https://bmjopen.bmj.com/content/15/5/e094559.full
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1850190254199275520
author Matthew S Brown
Jonathan Goldin
Grace Hyun Kim
Xueping Zhang
Lona Poole
author_facet Matthew S Brown
Jonathan Goldin
Grace Hyun Kim
Xueping Zhang
Lona Poole
author_sort Matthew S Brown
collection DOAJ
description Objectives Idiopathic pulmonary fibrosis (IPF) is a progressive interstitial lung disease. Chest high-resolution CT (HRCT) is instrumental in IPF management, and the Quantitative Lung Fibrosis (QLF) score is a computer-assisted metric for quantifying lung disease using HRCT. This study aimed to assess the change in QLF score associated with a minimum clinically important difference (MCID) of IPF symptoms and physiological lung function, and also determine the MCID of QLF change associated with all-cause mortality to serve as an imaging biomarker to confirm disease progression and response to therapy.Design and study setting We conducted post hoc analyses of prospective data from two IPF phase II studies of pamrevlumab, a fully human monoclonal antibody that binds to and inhibits connective tissue growth factor activity.Participants Overall, 152 patients with follow-up visits after week 24.Methods We used the anchor-based Jaeschke’s method to estimate the MCID of the QLF score that corresponded with the already established MCID of St. George’s Respiratory Questionnaire (SGRQ) and percent-predicted forced vital capacity (ppFVC). We also conducted a Cox regression analysis to establish a sensitive and robust MCID of the QLF score in predicting all-cause mortality.Results QLF changes of 4.4% and 3.6% corresponded to the established MCID of a 5-point increase in SGRQ and a 3.4% reduction in ppFVC, respectively. QLF changes of 1% (HR=4.98, p=0.05), 2% (HR=4.04, p=0.041), 20 mL (HR=6.37, p=0.024) and 22 mL (HR=6.38, p=0.024) predicted mortality.Conclusion A conservative metric of 2% can be used as the MCID of QLF for predicting all-cause mortality. This may be considered in IPF trials in which the degree of structural fibrosis assessed via HRCT is an endpoint. The MCID of SGRQ and FVC corresponds with a greater amount of QLF and may reflect that a greater amount of change in fibrosis is required before there is functional change.Trial registration number NCT01262001, NCT01890265.
format Article
id doaj-art-a7d782a22d7449c4b27672ee1c037ea4
institution OA Journals
issn 2044-6055
language English
publishDate 2025-05-01
publisher BMJ Publishing Group
record_format Article
series BMJ Open
spelling doaj-art-a7d782a22d7449c4b27672ee1c037ea42025-08-20T02:15:20ZengBMJ Publishing GroupBMJ Open2044-60552025-05-0115510.1136/bmjopen-2024-094559Minimum clinically important difference in Quantitative Lung Fibrosis score associated with all-cause mortality in idiopathic pulmonary fibrosis: subanalysis from two phase II trials of pamrevlumabMatthew S Brown0Jonathan Goldin1Grace Hyun Kim2Xueping Zhang3Lona Poole4University of California Los Angeles David Geffen School of Medicine, Los Angeles, California, USAUniversity of California Los Angeles David Geffen School of Medicine, Los Angeles, California, USAUniversity of California Los Angeles David Geffen School of Medicine, Los Angeles, California, USAFibroGen Inc, San Francisco, California, USAFibroGen Inc, San Francisco, California, USAObjectives Idiopathic pulmonary fibrosis (IPF) is a progressive interstitial lung disease. Chest high-resolution CT (HRCT) is instrumental in IPF management, and the Quantitative Lung Fibrosis (QLF) score is a computer-assisted metric for quantifying lung disease using HRCT. This study aimed to assess the change in QLF score associated with a minimum clinically important difference (MCID) of IPF symptoms and physiological lung function, and also determine the MCID of QLF change associated with all-cause mortality to serve as an imaging biomarker to confirm disease progression and response to therapy.Design and study setting We conducted post hoc analyses of prospective data from two IPF phase II studies of pamrevlumab, a fully human monoclonal antibody that binds to and inhibits connective tissue growth factor activity.Participants Overall, 152 patients with follow-up visits after week 24.Methods We used the anchor-based Jaeschke’s method to estimate the MCID of the QLF score that corresponded with the already established MCID of St. George’s Respiratory Questionnaire (SGRQ) and percent-predicted forced vital capacity (ppFVC). We also conducted a Cox regression analysis to establish a sensitive and robust MCID of the QLF score in predicting all-cause mortality.Results QLF changes of 4.4% and 3.6% corresponded to the established MCID of a 5-point increase in SGRQ and a 3.4% reduction in ppFVC, respectively. QLF changes of 1% (HR=4.98, p=0.05), 2% (HR=4.04, p=0.041), 20 mL (HR=6.37, p=0.024) and 22 mL (HR=6.38, p=0.024) predicted mortality.Conclusion A conservative metric of 2% can be used as the MCID of QLF for predicting all-cause mortality. This may be considered in IPF trials in which the degree of structural fibrosis assessed via HRCT is an endpoint. The MCID of SGRQ and FVC corresponds with a greater amount of QLF and may reflect that a greater amount of change in fibrosis is required before there is functional change.Trial registration number NCT01262001, NCT01890265.https://bmjopen.bmj.com/content/15/5/e094559.full
spellingShingle Matthew S Brown
Jonathan Goldin
Grace Hyun Kim
Xueping Zhang
Lona Poole
Minimum clinically important difference in Quantitative Lung Fibrosis score associated with all-cause mortality in idiopathic pulmonary fibrosis: subanalysis from two phase II trials of pamrevlumab
BMJ Open
title Minimum clinically important difference in Quantitative Lung Fibrosis score associated with all-cause mortality in idiopathic pulmonary fibrosis: subanalysis from two phase II trials of pamrevlumab
title_full Minimum clinically important difference in Quantitative Lung Fibrosis score associated with all-cause mortality in idiopathic pulmonary fibrosis: subanalysis from two phase II trials of pamrevlumab
title_fullStr Minimum clinically important difference in Quantitative Lung Fibrosis score associated with all-cause mortality in idiopathic pulmonary fibrosis: subanalysis from two phase II trials of pamrevlumab
title_full_unstemmed Minimum clinically important difference in Quantitative Lung Fibrosis score associated with all-cause mortality in idiopathic pulmonary fibrosis: subanalysis from two phase II trials of pamrevlumab
title_short Minimum clinically important difference in Quantitative Lung Fibrosis score associated with all-cause mortality in idiopathic pulmonary fibrosis: subanalysis from two phase II trials of pamrevlumab
title_sort minimum clinically important difference in quantitative lung fibrosis score associated with all cause mortality in idiopathic pulmonary fibrosis subanalysis from two phase ii trials of pamrevlumab
url https://bmjopen.bmj.com/content/15/5/e094559.full
work_keys_str_mv AT matthewsbrown minimumclinicallyimportantdifferenceinquantitativelungfibrosisscoreassociatedwithallcausemortalityinidiopathicpulmonaryfibrosissubanalysisfromtwophaseiitrialsofpamrevlumab
AT jonathangoldin minimumclinicallyimportantdifferenceinquantitativelungfibrosisscoreassociatedwithallcausemortalityinidiopathicpulmonaryfibrosissubanalysisfromtwophaseiitrialsofpamrevlumab
AT gracehyunkim minimumclinicallyimportantdifferenceinquantitativelungfibrosisscoreassociatedwithallcausemortalityinidiopathicpulmonaryfibrosissubanalysisfromtwophaseiitrialsofpamrevlumab
AT xuepingzhang minimumclinicallyimportantdifferenceinquantitativelungfibrosisscoreassociatedwithallcausemortalityinidiopathicpulmonaryfibrosissubanalysisfromtwophaseiitrialsofpamrevlumab
AT lonapoole minimumclinicallyimportantdifferenceinquantitativelungfibrosisscoreassociatedwithallcausemortalityinidiopathicpulmonaryfibrosissubanalysisfromtwophaseiitrialsofpamrevlumab