Systematic review of drug effects in humans and models with surfactant-processing disease.
Identifieur interne : 000C13 ( Main/Exploration ); précédent : 000C12; suivant : 000C14Systematic review of drug effects in humans and models with surfactant-processing disease.
Auteurs : Dymph Klay [Pays-Bas] ; Thijs W. Hoffman [Pays-Bas] ; Ankie M. Harmsze [Pays-Bas] ; Jan C. Grutters [Pays-Bas] ; Coline H M. Van Moorsel [Pays-Bas]Source :
- European respiratory review : an official journal of the European Respiratory Society [ 1600-0617 ] ; 2018.
Descripteurs français
- KwdFr :
- Agents de l'appareil respiratoire (usage thérapeutique), Animaux, Humains, Modèles animaux de maladie humaine, Mutation, Phénotype, Pneumopathies interstitielles idiopathiques (diagnostic), Pneumopathies interstitielles idiopathiques (génétique), Pneumopathies interstitielles idiopathiques (métabolisme), Pneumopathies interstitielles idiopathiques (traitement médicamenteux), Poumon (), Poumon (anatomopathologie), Poumon (métabolisme), Protéines associées au surfactant pulmonaire (génétique), Protéines associées au surfactant pulmonaire (métabolisme), Prédisposition génétique à une maladie, Résultat thérapeutique, Souris.
- MESH :
- anatomopathologie : Poumon.
- diagnostic : Pneumopathies interstitielles idiopathiques.
- génétique : Pneumopathies interstitielles idiopathiques, Protéines associées au surfactant pulmonaire.
- métabolisme : Pneumopathies interstitielles idiopathiques, Poumon, Protéines associées au surfactant pulmonaire.
- traitement médicamenteux : Pneumopathies interstitielles idiopathiques.
- usage thérapeutique : Agents de l'appareil respiratoire.
- Animaux, Humains, Modèles animaux de maladie humaine, Mutation, Phénotype, Poumon, Prédisposition génétique à une maladie, Résultat thérapeutique, Souris.
English descriptors
- KwdEn :
- Animals, Disease Models, Animal, Genetic Predisposition to Disease, Humans, Idiopathic Interstitial Pneumonias (diagnosis), Idiopathic Interstitial Pneumonias (drug therapy), Idiopathic Interstitial Pneumonias (genetics), Idiopathic Interstitial Pneumonias (metabolism), Lung (drug effects), Lung (metabolism), Lung (pathology), Mice, Mutation, Phenotype, Pulmonary Surfactant-Associated Proteins (genetics), Pulmonary Surfactant-Associated Proteins (metabolism), Respiratory System Agents (therapeutic use), Treatment Outcome.
- MESH :
- chemical , genetics : Pulmonary Surfactant-Associated Proteins.
- diagnosis : Idiopathic Interstitial Pneumonias.
- drug effects : Lung.
- drug therapy : Idiopathic Interstitial Pneumonias.
- genetics : Idiopathic Interstitial Pneumonias.
- metabolism : Idiopathic Interstitial Pneumonias, Lung, Pulmonary Surfactant-Associated Proteins.
- pathology : Lung.
- chemical , therapeutic use : Respiratory System Agents.
- Animals, Disease Models, Animal, Genetic Predisposition to Disease, Humans, Mice, Mutation, Phenotype, Treatment Outcome.
Abstract
Fibrotic interstitial pneumonias are a group of rare diseases characterised by distortion of lung interstitium. Patients with mutations in surfactant-processing genes, such as surfactant protein C (SFTPC), surfactant protein A1 and A2 (SFTPA1 and A2), ATP binding cassette A3 (ABCA3) and Hermansky-Pudlak syndrome (HPS1, 2 and 4), develop progressive pulmonary fibrosis, often culminating in fatal respiratory insufficiency. Although many mutations have been described, little is known about the optimal treatment strategy for fibrotic interstitial pneumonia patients with surfactant-processing mutations.We performed a systematic literature review of studies that described a drug effect in patients, cell or mouse models with a surfactant-processing mutation. In total, 73 articles were selected, consisting of 55 interstitial lung disease case reports/series, two clinical trials and 16 cell or mouse studies. Clinical effect parameters included lung function, radiological characteristics and clinical symptoms, while experimental outcome parameters included chemokine/cytokine expression, surfactant trafficking, necrosis and apoptosis. SP600125, a c-jun N-terminal kinase (JNK) inhibitor, hydroxychloroquine and 4-phenylbutyric acid were most frequently studied in disease models and lead to variable outcomes, suggesting that outcome is mutation dependent.This systematic review summarises effect parameters for future studies on surfactant-processing disorders in disease models and provides directions for future trials in affected patients.
DOI: 10.1183/16000617.0135-2017
PubMed: 29997245
Affiliations:
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(diagnosis)</term><term>Idiopathic Interstitial Pneumonias (drug therapy)</term><term>Idiopathic Interstitial Pneumonias (genetics)</term><term>Idiopathic Interstitial Pneumonias (metabolism)</term><term>Lung (drug effects)</term><term>Lung (metabolism)</term><term>Lung (pathology)</term><term>Mice</term><term>Mutation</term><term>Phenotype</term><term>Pulmonary Surfactant-Associated Proteins (genetics)</term><term>Pulmonary Surfactant-Associated Proteins (metabolism)</term><term>Respiratory System Agents (therapeutic use)</term><term>Treatment Outcome</term></keywords><keywords scheme="KwdFr" xml:lang="fr"><term>Agents de l'appareil respiratoire (usage thérapeutique)</term><term>Animaux</term><term>Humains</term><term>Modèles animaux de maladie humaine</term><term>Mutation</term><term>Phénotype</term><term>Pneumopathies interstitielles idiopathiques (diagnostic)</term><term>Pneumopathies interstitielles idiopathiques (génétique)</term><term>Pneumopathies interstitielles idiopathiques (métabolisme)</term><term>Pneumopathies interstitielles idiopathiques (traitement médicamenteux)</term><term>Poumon ()</term><term>Poumon (anatomopathologie)</term><term>Poumon (métabolisme)</term><term>Protéines associées au surfactant pulmonaire (génétique)</term><term>Protéines associées au surfactant pulmonaire (métabolisme)</term><term>Prédisposition génétique à une maladie</term><term>Résultat thérapeutique</term><term>Souris</term></keywords><keywords scheme="MESH" type="chemical" qualifier="genetics" xml:lang="en"><term>Pulmonary Surfactant-Associated Proteins</term></keywords><keywords scheme="MESH" qualifier="anatomopathologie" xml:lang="fr"><term>Poumon</term></keywords><keywords scheme="MESH" qualifier="diagnosis" xml:lang="en"><term>Idiopathic Interstitial Pneumonias</term></keywords><keywords scheme="MESH" qualifier="diagnostic" xml:lang="fr"><term>Pneumopathies interstitielles idiopathiques</term></keywords><keywords scheme="MESH" qualifier="drug effects" xml:lang="en"><term>Lung</term></keywords><keywords scheme="MESH" qualifier="drug therapy" xml:lang="en"><term>Idiopathic Interstitial Pneumonias</term></keywords><keywords scheme="MESH" qualifier="genetics" xml:lang="en"><term>Idiopathic Interstitial Pneumonias</term></keywords><keywords scheme="MESH" qualifier="génétique" xml:lang="fr"><term>Pneumopathies interstitielles idiopathiques</term><term>Protéines associées au surfactant pulmonaire</term></keywords><keywords scheme="MESH" qualifier="metabolism" xml:lang="en"><term>Idiopathic Interstitial Pneumonias</term><term>Lung</term><term>Pulmonary Surfactant-Associated Proteins</term></keywords><keywords scheme="MESH" qualifier="métabolisme" xml:lang="fr"><term>Pneumopathies interstitielles idiopathiques</term><term>Poumon</term><term>Protéines associées au surfactant pulmonaire</term></keywords><keywords scheme="MESH" qualifier="pathology" xml:lang="en"><term>Lung</term></keywords><keywords scheme="MESH" type="chemical" qualifier="therapeutic use" xml:lang="en"><term>Respiratory System Agents</term></keywords><keywords scheme="MESH" qualifier="traitement médicamenteux" xml:lang="fr"><term>Pneumopathies interstitielles idiopathiques</term></keywords><keywords scheme="MESH" qualifier="usage thérapeutique" xml:lang="fr"><term>Agents de l'appareil respiratoire</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Animals</term><term>Disease Models, Animal</term><term>Genetic Predisposition to Disease</term><term>Humans</term><term>Mice</term><term>Mutation</term><term>Phenotype</term><term>Treatment Outcome</term></keywords><keywords scheme="MESH" xml:lang="fr"><term>Animaux</term><term>Humains</term><term>Modèles animaux de maladie humaine</term><term>Mutation</term><term>Phénotype</term><term>Poumon</term><term>Prédisposition génétique à une maladie</term><term>Résultat thérapeutique</term><term>Souris</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Fibrotic interstitial pneumonias are a group of rare diseases characterised by distortion of lung interstitium. Patients with mutations in surfactant-processing genes, such as surfactant protein C (<i>SFTPC</i>), surfactant protein A1 and A2 (<i>SFTPA1</i> and <i>A2</i>), ATP binding cassette A3 (<i>ABCA3</i>) and Hermansky-Pudlak syndrome (<i>HPS1</i>, <i>2</i> and <i>4</i>), develop progressive pulmonary fibrosis, often culminating in fatal respiratory insufficiency. Although many mutations have been described, little is known about the optimal treatment strategy for fibrotic interstitial pneumonia patients with surfactant-processing mutations.We performed a systematic literature review of studies that described a drug effect in patients, cell or mouse models with a surfactant-processing mutation. In total, 73 articles were selected, consisting of 55 interstitial lung disease case reports/series, two clinical trials and 16 cell or mouse studies. Clinical effect parameters included lung function, radiological characteristics and clinical symptoms, while experimental outcome parameters included chemokine/cytokine expression, surfactant trafficking, necrosis and apoptosis. SP600125, a c-jun N-terminal kinase (JNK) inhibitor, hydroxychloroquine and 4-phenylbutyric acid were most frequently studied in disease models and lead to variable outcomes, suggesting that outcome is mutation dependent.This systematic review summarises effect parameters for future studies on surfactant-processing disorders in disease models and provides directions for future trials in affected patients.</div></front></TEI><affiliations><list><country><li>Pays-Bas</li></country></list><tree><country name="Pays-Bas"><noRegion><name sortKey="Klay, Dymph" sort="Klay, Dymph" uniqKey="Klay D" first="Dymph" last="Klay">Dymph Klay</name></noRegion><name sortKey="Grutters, Jan C" sort="Grutters, Jan C" uniqKey="Grutters J" first="Jan C" last="Grutters">Jan C. Grutters</name><name sortKey="Harmsze, Ankie M" sort="Harmsze, Ankie M" uniqKey="Harmsze A" first="Ankie M" last="Harmsze">Ankie M. Harmsze</name><name sortKey="Hoffman, Thijs W" sort="Hoffman, Thijs W" uniqKey="Hoffman T" first="Thijs W" last="Hoffman">Thijs W. Hoffman</name><name sortKey="Van Moorsel, Coline H M" sort="Van Moorsel, Coline H M" uniqKey="Van Moorsel C" first="Coline H M" last="Van Moorsel">Coline H M. Van Moorsel</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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