Eligibility criteria from pharmaceutical randomised controlled trials of idiopathic pulmonary fibrosis: a registry-based study.

BACKGROUND

Little is known about generalisability of randomised controlled trials (RCTs) for idiopathic pulmonary fibrosis (IPF). We evaluated eligibility criteria for phase III IPF RCTs to determine their representativeness in clinical registries, and calculated forced vital capacity (FVC) changes according to eligibility criteria.

METHODS

Common eligibility criteria used in >60% of IPF RCTs were identified from a literature search and applied to patients with IPF from prospective Australian and Canadian registries. Additional pre-specified criteria of 6-min walk distance (6MWD) and different measures of preceding disease progression were also evaluated. Joint longitudinal-survival modelling was used to compare FVC decline according to eligibility for individual and composite criteria.

RESULTS

Out of 990 patients with IPF, 527 (53%) met all common RCT eligibility criteria at the first clinic visit, including 343 with definite IPF and 184 with radiological probable usual interstitial pneumonia pattern without histological confirmation (

CONCLUSIONS

Eligibility criteria of previous IPF RCTs have limited generalisability to clinical IPF populations, with FVC decline differing between eligible and ineligible populations.

Informations de copyright

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Déclaration de conflit d'intérêts

Conflict of interest: Y.H. Khor reports fellowship support from NHMRC Investigator Grant, and support for the CARE-PF registry (with no influence on this study) from Boehringer Ingelheim, during the conduct of the study. K.A. Johannson reports personal fees, non-financial support and other from Boehringer Ingelheim, personal fees from Hoffman-La Roche Ltd, Pliant Therapeutics and Thyron, grants from University Hospital Foundation, Pulmonary Fibrosis Society of Calgary and University of Calgary Cumming School of Medicine, and personal fees and non-financial support from Three Lakes Foundation, outside the submitted work. V. Marcoux reports grants from Boehringer Ingelheim, during the conduct of the study; grants from University of British Columbia and Boehringer Ingelheim, outside the submitted work. J.H. Fisher reports grants from Boehringer Ingelheim, during the conduct of the study; grants from Canadian Pulmonary Fibrosis Foundation and University of Toronto, and personal fees from Boehringer Ingelheim and AstraZeneca, outside the submitted work. D. Assayag reports grants from Boehringer Ingelheim Canada, and lecture honoraria from Boehringer Ingelheim and Hoffman La Roche, outside the submitted work. H. Manganas reports support for the present manuscript from University of British Columbia; grants from Boehringer Ingelheim Canada, Hoffmann La Roche, Galapagos and BMS, honoraria for educational events from Boehringer Ingelheim Canada, and advisory board membership for Boehringer Ingelheim Canada, outside the submitted work. M. Kolb reports grants from Boehringer Ingelheim, Pieris and Roche, consulting fees from Boehringer Ingelheim, Roche, Horizon, Cipla, Abbvie, Bellerophon, Algernon, CSL Behring, United Therapeutics and LabCorp, lecture honoraria from Roche, Novartis and Boehringer Ingelheim, payment for expert testimony from Roche, advisory board participation with United Therapeutics and LabCorp, and reports an editorial allowance from ERJ, outside the submitted work. C.J. Ryerson reports grants from Boehringer Ingelheim, during the conduct of the study; grants and personal fees from Boehringer Ingelheim and Hoffmann-La Roche, and personal fees from Veracyte, Pliant Therapeutics, AstraZeneca and Cipla Ltd, outside the submitted work. All other authors have nothing to disclose.