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Hypersensitivity pneumonitis: Lessons from a randomized controlled trial in children
Author(s) -
Griese Matthias,
Stehling Florian,
Schwerk Nicolaus,
Rosewich Martin,
Jerkic PeraSilvija,
Rock Hans,
Ruckes Christian,
Kronfeld Kai,
Sebah Daniela,
Wetzke Martin,
Seidl Elias
Publication year - 2021
Publication title -
pediatric pulmonology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.866
H-Index - 106
eISSN - 1099-0496
pISSN - 8755-6863
DOI - 10.1002/ppul.25513
Subject(s) - medicine , prednisolone , placebo , randomized controlled trial , hypersensitivity pneumonitis , pediatrics , intensive care medicine , lung , pathology , alternative medicine
Hypersensitivity pneumonitis (HP) in children is a severe interstitial lung disease and potentially, a chronic condition, if not treated appropriately. No evidence‐based guidelines are available; in particular, the role of systemic glucocorticoid therapy is unclear. Methods The aim of this randomized, double‐blind, placebo‐controlled, parallel‐group, multi‐center, phase II trial in pediatric HP was to assess the outcome of HP in children after 6 months of treatment and to compare 3 months of treatment with oral prednisolone or placebo. Results After 1.5 years and the inclusion of only four children, we terminated the study prematurely. Two of the children randomized to prednisolone did not achieve the predefined response of FVC to normal. One child treated with placebo recovered to normal, similar to another child treated with prednisolone. All children treated with steroids developed drug‐related side effects. Discussion This uncompleted study illustrates the urgent medical need for evidence‐based treatment protocols for this condition. We discuss the hurdles which were specific for completion of this trial in a rare condition. Among other options, we suggest the inclusion of children into an all‐age study of HP, as in adults the same questions are unanswered.