Efficacy of Pirfenidone vs. Placebo in Unclassifiable Interstitial Lung Disease, by Surgical Lung Biopsy Status: Data From a post-hoc Analysis
Approximately 12–13% of patients with interstitial lung disease (ILD) are diagnosed with unclassifiable ILD (uILD), often despite thorough evaluation. A recent Phase 2 study (NCT03099187) described a significant effect of pirfenidone vs. placebo on forced vital capacity (FVC) measured by site spirom...
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Frontiers Media S.A.
2022-06-01
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fmed.2022.897102/full |
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| author | Maria Molina-Molina Maria Molina-Molina Michael Kreuter Michael Kreuter Vincent Cottin Vincent Cottin Tamera J. Corte Tamera J. Corte Frank Gilberg Klaus-Uwe Kirchgaessler Judit Axmann Toby M. Maher Toby M. Maher Toby M. Maher |
| author_facet | Maria Molina-Molina Maria Molina-Molina Michael Kreuter Michael Kreuter Vincent Cottin Vincent Cottin Tamera J. Corte Tamera J. Corte Frank Gilberg Klaus-Uwe Kirchgaessler Judit Axmann Toby M. Maher Toby M. Maher Toby M. Maher |
| author_sort | Maria Molina-Molina |
| collection | DOAJ |
| description | Approximately 12–13% of patients with interstitial lung disease (ILD) are diagnosed with unclassifiable ILD (uILD), often despite thorough evaluation. A recent Phase 2 study (NCT03099187) described a significant effect of pirfenidone vs. placebo on forced vital capacity (FVC) measured by site spirometry in patients with progressive fibrosing uILD (hereafter referred to as the pirfenidone in uILD study). Here, we present the results from a post-hoc analysis of this study to assess patient baseline characteristics and the efficacy of pirfenidone vs. placebo analyzed by surgical lung biopsy (SLB) status. Mean FVC (mL) change over 24 weeks was included as a post-hoc efficacy outcome. Of 253 randomized patients, 88 (34.8%) had a SLB and 165 (65.2%) did not. Baseline characteristics were generally similar between SLB subgroups; however, patients who had a SLB were slightly younger and had a higher 6-min walk distance than those without a SLB. Mean FVC change over 24 weeks for pirfenidone vs. placebo was −90.9 vs. −146.3 mL, respectively, in patients who had a SLB, and 8.2 vs. −85.3 mL, respectively, in patients without a SLB. Overall, the results from the post-hoc analysis identified that pirfenidone may be an effective treatment in progressive fibrosing uILD over 24 weeks, irrespective of SLB status; however, caution should be taken when interpreting these data due to several limitations. There are differences in the treatment effect of pirfenidone between the subgroups that require further pathological and radiological investigation. In this manuscript, we also descriptively compared baseline characteristics from the overall pirfenidone in uILD study population with other uILD populations reported in the literature, with the aim of understanding if there are any similarities or differences within these cohorts. Most baseline characteristics for patients in the pirfenidone in uILD study were within the ranges reported in the literature; however, ranges were wide, highlighting the heterogeneity of uILD populations.Clinical Trial RegistrationClinicalTrials.gov, identifier: NCT03099187. |
| first_indexed | 2024-04-13T17:58:27Z |
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| language | English |
| last_indexed | 2024-04-13T17:58:27Z |
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| spelling | doaj.art-ee6f556226ca452ba20b217abf867f7c2022-12-22T02:36:22ZengFrontiers Media S.A.Frontiers in Medicine2296-858X2022-06-01910.3389/fmed.2022.897102897102Efficacy of Pirfenidone vs. Placebo in Unclassifiable Interstitial Lung Disease, by Surgical Lung Biopsy Status: Data From a post-hoc AnalysisMaria Molina-Molina0Maria Molina-Molina1Michael Kreuter2Michael Kreuter3Vincent Cottin4Vincent Cottin5Tamera J. Corte6Tamera J. Corte7Frank Gilberg8Klaus-Uwe Kirchgaessler9Judit Axmann10Toby M. Maher11Toby M. Maher12Toby M. Maher13ILD Unit, Respiratory Department, University Hospital of Bellvitge, Institut d'Investigació Biomèdica de Bellvitge (IDIBELL), L'Hospitalet de Llobregat, Barcelona, SpainCentro de Investigación Biomédica en Red Enfermedades Respiratorias (CIBERES), Madrid, SpainCenter for Interstitial and Rare Lung Diseases, Dep-Pneumonology, Thoraxklinik, University of Heidelberg, Heidelberg, GermanyGerman Center for Lung Research, Heidelberg, GermanyNational Reference Coordinating Center for Rare Pulmonary Diseases, Louis Pradel Hospital, Hospices Civils de Lyon, Member of ERN-LUNG, Lyon, FranceIVPC, Université Claude Bernard Lyon 1, UMR754, INRAE, Lyon, FranceDepartment of Respiratory Medicine, Royal Prince Alfred Hospital, University of Sydney, Sydney, NSW, AustraliaMedical School, University of Sydney, Camperdown, NSW, AustraliaF. Hoffmann-La Roche, Ltd., Basel, SwitzerlandF. Hoffmann-La Roche, Ltd., Basel, SwitzerlandF. Hoffmann-La Roche, Ltd., Basel, Switzerland0Interstitial Lung Disease Unit, Royal Brompton Hospital, London, United Kingdom1National Heart and Lung Institute, Imperial College London, London, United Kingdom2Hastings Center for Pulmonary Research and Division of Pulmonary, Critical Care and Sleep Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA, United StatesApproximately 12–13% of patients with interstitial lung disease (ILD) are diagnosed with unclassifiable ILD (uILD), often despite thorough evaluation. A recent Phase 2 study (NCT03099187) described a significant effect of pirfenidone vs. placebo on forced vital capacity (FVC) measured by site spirometry in patients with progressive fibrosing uILD (hereafter referred to as the pirfenidone in uILD study). Here, we present the results from a post-hoc analysis of this study to assess patient baseline characteristics and the efficacy of pirfenidone vs. placebo analyzed by surgical lung biopsy (SLB) status. Mean FVC (mL) change over 24 weeks was included as a post-hoc efficacy outcome. Of 253 randomized patients, 88 (34.8%) had a SLB and 165 (65.2%) did not. Baseline characteristics were generally similar between SLB subgroups; however, patients who had a SLB were slightly younger and had a higher 6-min walk distance than those without a SLB. Mean FVC change over 24 weeks for pirfenidone vs. placebo was −90.9 vs. −146.3 mL, respectively, in patients who had a SLB, and 8.2 vs. −85.3 mL, respectively, in patients without a SLB. Overall, the results from the post-hoc analysis identified that pirfenidone may be an effective treatment in progressive fibrosing uILD over 24 weeks, irrespective of SLB status; however, caution should be taken when interpreting these data due to several limitations. There are differences in the treatment effect of pirfenidone between the subgroups that require further pathological and radiological investigation. In this manuscript, we also descriptively compared baseline characteristics from the overall pirfenidone in uILD study population with other uILD populations reported in the literature, with the aim of understanding if there are any similarities or differences within these cohorts. Most baseline characteristics for patients in the pirfenidone in uILD study were within the ranges reported in the literature; however, ranges were wide, highlighting the heterogeneity of uILD populations.Clinical Trial RegistrationClinicalTrials.gov, identifier: NCT03099187.https://www.frontiersin.org/articles/10.3389/fmed.2022.897102/fulllung functionpirfenidonepost-hoc analysissurgical lung biopsyunclassifiable interstitial lung disease |
| spellingShingle | Maria Molina-Molina Maria Molina-Molina Michael Kreuter Michael Kreuter Vincent Cottin Vincent Cottin Tamera J. Corte Tamera J. Corte Frank Gilberg Klaus-Uwe Kirchgaessler Judit Axmann Toby M. Maher Toby M. Maher Toby M. Maher Efficacy of Pirfenidone vs. Placebo in Unclassifiable Interstitial Lung Disease, by Surgical Lung Biopsy Status: Data From a post-hoc Analysis Frontiers in Medicine lung function pirfenidone post-hoc analysis surgical lung biopsy unclassifiable interstitial lung disease |
| title | Efficacy of Pirfenidone vs. Placebo in Unclassifiable Interstitial Lung Disease, by Surgical Lung Biopsy Status: Data From a post-hoc Analysis |
| title_full | Efficacy of Pirfenidone vs. Placebo in Unclassifiable Interstitial Lung Disease, by Surgical Lung Biopsy Status: Data From a post-hoc Analysis |
| title_fullStr | Efficacy of Pirfenidone vs. Placebo in Unclassifiable Interstitial Lung Disease, by Surgical Lung Biopsy Status: Data From a post-hoc Analysis |
| title_full_unstemmed | Efficacy of Pirfenidone vs. Placebo in Unclassifiable Interstitial Lung Disease, by Surgical Lung Biopsy Status: Data From a post-hoc Analysis |
| title_short | Efficacy of Pirfenidone vs. Placebo in Unclassifiable Interstitial Lung Disease, by Surgical Lung Biopsy Status: Data From a post-hoc Analysis |
| title_sort | efficacy of pirfenidone vs placebo in unclassifiable interstitial lung disease by surgical lung biopsy status data from a post hoc analysis |
| topic | lung function pirfenidone post-hoc analysis surgical lung biopsy unclassifiable interstitial lung disease |
| url | https://www.frontiersin.org/articles/10.3389/fmed.2022.897102/full |
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