Systematic review of drug effects in humans and models with surfactant-processing disease

European Respiratory Review : an Official Journal of the European Respiratory Society
Dymph KlayColine H M van Moorsel

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 mos...Continue Reading

References

Jan 1, 1984·Virchows Archiv. A, Pathological Anatomy and Histopathology·A Takahashi, T Yokoyama
Mar 17, 2000·Immunological Reviews·P R Lawson, K B Reid
Feb 22, 2001·Annual Review of Physiology·T E Weaver, J J Conkright
Feb 24, 2001·The New England Journal of Medicine·L M NogeeJ A Whitsett
Jul 20, 2002·Molecular Genetics and Metabolism·William A GahlBernadette Gochuico
Mar 29, 2003·Human Genetics·Paul D AndersonWilliam A Gahl
Mar 25, 2004·American Journal of Medical Genetics. Part a·Mohammed TredanoMichel Bahuau
Apr 27, 2004·American Journal of Medical Genetics. Part a·Esther B BachliWilliam A Gahl
May 11, 2004·Modern Pathology : an Official Journal of the United States and Canadian Academy of Pathology, Inc·Rajni ChibbarEdmond G Lemire
Aug 6, 2004·The European Respiratory Journal·F BraschM F Beers
Dec 2, 2004·The European Respiratory Journal·S PercopoF Santamaria
Jan 14, 2005·The New England Journal of Medicine·Dennis M Rosen, David A Waltz
Nov 11, 2006·The Journal of Pediatrics·Adrienne PrestridgeLawrence Nogee
Jun 26, 2007·American Journal of Physiology. Lung Cellular and Molecular Physiology·Surafel MulugetaMichael F Beers
Jul 10, 2007·European Journal of Pediatrics·Takuya YokotaNobuya Inagaki
Apr 9, 2008·American Journal of Physiology. Lung Cellular and Molecular Physiology·William E LawsonTimothy S Blackwell
Jun 12, 2008·Annual Review of Genomics and Human Genetics·Marjan HuizingWilliam A Gahl
Jul 8, 2008·Experimental and Molecular Pathology·D Radford ShanklinHeather S Baldwin
Aug 5, 2008·American Journal of Physiology. Lung Cellular and Molecular Physiology·Yoshihiro MatsumuraNobuya Inagaki
Dec 23, 2008·American Journal of Human Genetics·Yongyu WangChristine Kim Garcia
Jan 17, 2009·Pediatric Pulmonology·Rola Abou TaamJacques de Blic
Feb 18, 2009·Pediatric and Developmental Pathology : the Official Journal of the Society for Pediatric Pathology and the Paediatric Pathology Society·Susan E WertLawrence M Nogee
Sep 5, 2009·American Journal of Respiratory and Critical Care Medicine·Farshid N RouhaniBernadette R Gochuico
Dec 17, 2009·Orphanet Journal of Rare Diseases·Matthias GrieseDominik Hartl
Apr 21, 2010·Archives of Disease in Childhood·Guillaume ThouveninRalph Epaud
May 14, 2010·American Journal of Respiratory Cell and Molecular Biology·Jean Ann MaguireMichael F Beers
May 15, 2010·The Journal of Biological Chemistry·Meenakshi MaitraChristine Kim Garcia
Jul 27, 2010·American Journal of Respiratory and Critical Care Medicine·Coline H M van MoorselJan C Grutters
Aug 24, 2010·Orphanet Journal of Rare Diseases·Annick ClementHarriet Corvol
Jan 11, 2011·Respiratory Research·Nina WeichertMatthias Griese
Mar 23, 2011·Molecular Genetics and Metabolism·Kevin O'BrienWilliam A Gahl
Apr 19, 2011·American Journal of Respiratory and Critical Care Medicine·Vincent Cottin, Jean-François Cordier
May 28, 2011·American Journal of Respiratory and Critical Care Medicine·Elena N Atochina-VassermanSusan Guttentag
Jun 11, 2011·Pulmonary Medicine·Satish K MadalaStephan W Glasser
Jul 16, 2011·The Journal of Biological Chemistry·Harikrishna TanjoreTimothy S Blackwell
Sep 16, 2011·Respiratory Medicine·Cecilia García-SanchoMoisés Selman
Oct 22, 2011·The International Journal of Biochemistry & Cell Biology·Jean Ann MaguireMichael F Beers

❮ Previous
Next ❯

Citations

Jul 22, 2020·American Journal of Respiratory Cell and Molecular Biology·Jennifer A WambachF Sessions Cole
Aug 29, 2019·Journal of Clinical Medicine·Nadia NathanAnnick Clement
Apr 3, 2020·Current Opinion in Pulmonary Medicine·Dymph KlayColine H M van Moorsel
Feb 19, 2021·European Respiratory Review : an Official Journal of the European Respiratory Society·Coline H M van MoorselJan C Grutters

❮ Previous
Next ❯

Related Concepts

Related Feeds

Antimalarial Agents (ASM)

Antimalarial agents, also known as antimalarials, are designed to prevent or cure malaria. Discover the latest research on antimalarial agents here.

Apoptosis

Apoptosis is a specific process that leads to programmed cell death through the activation of an evolutionary conserved intracellular pathway leading to pathognomic cellular changes distinct from cellular necrosis

Antimalarial Agents

Antimalarial agents, also known as antimalarials, are designed to prevent or cure malaria. Discover the latest research on antimalarial agents here.