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<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with a <italic>G551D</italic> Mutation</title><author><name sortKey="Davies, Jane C" sort="Davies, Jane C" uniqKey="Davies J" first="Jane C." last="Davies">Jane C. Davies</name><affiliation><nlm:aff id="aff1"></nlm:aff></affiliation><affiliation><nlm:aff id="aff2"></nlm:aff></affiliation></author><author><name sortKey="Wainwright, Claire E" sort="Wainwright, Claire E" uniqKey="Wainwright C" first="Claire E." last="Wainwright">Claire E. Wainwright</name><affiliation><nlm:aff id="aff3"></nlm:aff></affiliation></author><author><name sortKey="Canny, Gerard J" sort="Canny, Gerard J" uniqKey="Canny G" first="Gerard J." last="Canny">Gerard J. Canny</name><affiliation><nlm:aff id="aff4"></nlm:aff></affiliation></author><author><name sortKey="Chilvers, Mark A" sort="Chilvers, Mark A" uniqKey="Chilvers M" first="Mark A." last="Chilvers">Mark A. Chilvers</name><affiliation><nlm:aff id="aff5"></nlm:aff></affiliation></author><author><name sortKey="Howenstine, Michelle S" sort="Howenstine, Michelle S" uniqKey="Howenstine M" first="Michelle S." last="Howenstine">Michelle S. Howenstine</name><affiliation><nlm:aff id="aff6"></nlm:aff></affiliation></author><author><name sortKey="Munck, Anne" sort="Munck, Anne" uniqKey="Munck A" first="Anne" last="Munck">Anne Munck</name><affiliation><nlm:aff id="aff7"></nlm:aff></affiliation></author><author><name sortKey="Mainz, Jochen G" sort="Mainz, Jochen G" uniqKey="Mainz J" first="Jochen G." last="Mainz">Jochen G. Mainz</name><affiliation><nlm:aff id="aff8"></nlm:aff></affiliation></author><author><name sortKey="Rodriguez, Sally" sort="Rodriguez, Sally" uniqKey="Rodriguez S" first="Sally" last="Rodriguez">Sally Rodriguez</name><affiliation><nlm:aff id="aff9"></nlm:aff></affiliation></author><author><name sortKey="Li, Haihong" sort="Li, Haihong" uniqKey="Li H" first="Haihong" last="Li">Haihong Li</name><affiliation><nlm:aff id="aff9"></nlm:aff></affiliation></author><author><name sortKey="Yen, Karl" sort="Yen, Karl" uniqKey="Yen K" first="Karl" last="Yen">Karl Yen</name><affiliation><nlm:aff id="aff9"></nlm:aff></affiliation></author><author><name sortKey="Ordo Ez, Claudia L" sort="Ordo Ez, Claudia L" uniqKey="Ordo Ez C" first="Claudia L." last="Ordo Ez">Claudia L. Ordo Ez</name><affiliation><nlm:aff id="aff9"></nlm:aff></affiliation></author><author><name sortKey="Ahrens, Richard" sort="Ahrens, Richard" uniqKey="Ahrens R" first="Richard" last="Ahrens">Richard Ahrens</name><affiliation><nlm:aff id="aff10"></nlm:aff></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PMC</idno><idno type="pmid">23590265</idno><idno type="pmc">3734608</idno><idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3734608</idno><idno type="RBID">PMC:3734608</idno><idno type="doi">10.1164/rccm.201301-0153OC</idno><date when="2013">2013</date><idno type="wicri:Area/Pmc/Corpus">001709</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Corpus" wicri:corpus="PMC">001709</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a" type="main">Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with a <italic>G551D</italic> Mutation</title><author><name sortKey="Davies, Jane C" sort="Davies, Jane C" uniqKey="Davies J" first="Jane C." last="Davies">Jane C. Davies</name><affiliation><nlm:aff id="aff1"></nlm:aff></affiliation><affiliation><nlm:aff id="aff2"></nlm:aff></affiliation></author><author><name sortKey="Wainwright, Claire E" sort="Wainwright, Claire E" uniqKey="Wainwright C" first="Claire E." last="Wainwright">Claire E. Wainwright</name><affiliation><nlm:aff id="aff3"></nlm:aff></affiliation></author><author><name sortKey="Canny, Gerard J" sort="Canny, Gerard J" uniqKey="Canny G" first="Gerard J." last="Canny">Gerard J. Canny</name><affiliation><nlm:aff id="aff4"></nlm:aff></affiliation></author><author><name sortKey="Chilvers, Mark A" sort="Chilvers, Mark A" uniqKey="Chilvers M" first="Mark A." last="Chilvers">Mark A. Chilvers</name><affiliation><nlm:aff id="aff5"></nlm:aff></affiliation></author><author><name sortKey="Howenstine, Michelle S" sort="Howenstine, Michelle S" uniqKey="Howenstine M" first="Michelle S." last="Howenstine">Michelle S. Howenstine</name><affiliation><nlm:aff id="aff6"></nlm:aff></affiliation></author><author><name sortKey="Munck, Anne" sort="Munck, Anne" uniqKey="Munck A" first="Anne" last="Munck">Anne Munck</name><affiliation><nlm:aff id="aff7"></nlm:aff></affiliation></author><author><name sortKey="Mainz, Jochen G" sort="Mainz, Jochen G" uniqKey="Mainz J" first="Jochen G." last="Mainz">Jochen G. Mainz</name><affiliation><nlm:aff id="aff8"></nlm:aff></affiliation></author><author><name sortKey="Rodriguez, Sally" sort="Rodriguez, Sally" uniqKey="Rodriguez S" first="Sally" last="Rodriguez">Sally Rodriguez</name><affiliation><nlm:aff id="aff9"></nlm:aff></affiliation></author><author><name sortKey="Li, Haihong" sort="Li, Haihong" uniqKey="Li H" first="Haihong" last="Li">Haihong Li</name><affiliation><nlm:aff id="aff9"></nlm:aff></affiliation></author><author><name sortKey="Yen, Karl" sort="Yen, Karl" uniqKey="Yen K" first="Karl" last="Yen">Karl Yen</name><affiliation><nlm:aff id="aff9"></nlm:aff></affiliation></author><author><name sortKey="Ordo Ez, Claudia L" sort="Ordo Ez, Claudia L" uniqKey="Ordo Ez C" first="Claudia L." last="Ordo Ez">Claudia L. Ordo Ez</name><affiliation><nlm:aff id="aff9"></nlm:aff></affiliation></author><author><name sortKey="Ahrens, Richard" sort="Ahrens, Richard" uniqKey="Ahrens R" first="Richard" last="Ahrens">Richard Ahrens</name><affiliation><nlm:aff id="aff10"></nlm:aff></affiliation></author></analytic><series><title level="j">American Journal of Respiratory and Critical Care Medicine</title><idno type="ISSN">1073-449X</idno><idno type="eISSN">1535-4970</idno><imprint><date when="2013">2013</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><p><italic>Rationale</italic>: Ivacaftor (VX-770), a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, has been shown to improve lung function, pulmonary exacerbation rate, respiratory symptoms, and weight gain compared with placebo in patients with cystic fibrosis aged 12 years or older with a <italic>G551D-CFTR</italic> mutation.</p><p><italic>Objectives</italic>: This randomized, double-blind, placebo-controlled trial evaluated ivacaftor in patients with cystic fibrosis aged 6–11 years with a <italic>G551D-CFTR</italic> mutation on at least one allele.</p><p><italic>Methods</italic>: Patients were randomly assigned to receive ivacaftor administered orally at 150 mg (n = 26) or placebo (n = 26) every 12 hours for 48 weeks in addition to existing prescribed cystic fibrosis therapies.</p><p><italic>Measurements and Main Results</italic>: Despite near-normal mean baseline values in FEV<sub>1</sub>, patients receiving ivacaftor had a significant increase in percent predicted FEV<sub>1</sub> from baseline through Week 24 versus placebo group (treatment effect, 12.5 percentage points; <italic>P</italic> < 0.001). Effects on pulmonary function were evident by 2 weeks, and a significant treatment effect was maintained through Week 48. Patients treated with ivacaftor gained, on average, 2.8 kg more than those receiving placebo at Week 48 (<italic>P</italic> < 0.001). The change from baseline through Week 48 in the concentration of sweat chloride, a measure of CFTR activity, with ivacaftor was −53.5 mmol/L (<italic>P</italic> < 0.001) versus placebo. The incidence of adverse events was similar in the two groups.</p><p><italic>Conclusions</italic>: In patients who are younger and healthier than those in previously studied populations, ivacaftor demonstrated a significant improvement in pulmonary function, weight, and CFTR activity compared with placebo. Clinical trial registered with <ext-link ext-link-type="uri" xlink:href="www.clinicaltrials.gov">www.clinicaltrials.gov</ext-link> (NCT00909727).</p></div></front></TEI><pmc article-type="research-article"><pmc-comment>The publisher of this article does not allow downloading of the full text in XML form.</pmc-comment>
<front><journal-meta><journal-id journal-id-type="nlm-ta">Am J Respir Crit Care Med</journal-id><journal-id journal-id-type="iso-abbrev">Am. J. Respir. Crit. Care Med</journal-id><journal-id journal-id-type="publisher-id">ajrccm</journal-id><journal-title-group><journal-title>American Journal of Respiratory and Critical Care Medicine</journal-title></journal-title-group><issn pub-type="ppub">1073-449X</issn><issn pub-type="epub">1535-4970</issn><publisher><publisher-name>American Thoracic Society</publisher-name></publisher></journal-meta><article-meta><article-id pub-id-type="pmid">23590265</article-id><article-id pub-id-type="pmc">3734608</article-id><article-id pub-id-type="publisher-manuscript">201301-0153OC</article-id><article-id pub-id-type="doi">10.1164/rccm.201301-0153OC</article-id><article-categories><subj-group subj-group-type="heading"><subject>Articles</subject><subj-group><subject>D. Cystic Fibrosis</subject></subj-group></subj-group></article-categories><title-group><article-title>Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with a <italic>G551D</italic> Mutation</article-title></title-group><contrib-group><contrib contrib-type="author" corresp="yes"><name><surname>Davies</surname><given-names>Jane C.</given-names></name><xref ref-type="aff" rid="aff1"><sup>1</sup></xref><xref ref-type="aff" rid="aff2"><sup>2</sup></xref></contrib><contrib contrib-type="author"><name><surname>Wainwright</surname><given-names>Claire E.</given-names></name><xref ref-type="aff" rid="aff3"><sup>3</sup></xref></contrib><contrib contrib-type="author"><name><surname>Canny</surname><given-names>Gerard J.</given-names></name><xref ref-type="aff" rid="aff4"><sup>4</sup></xref></contrib><contrib contrib-type="author"><name><surname>Chilvers</surname><given-names>Mark A.</given-names></name><xref ref-type="aff" rid="aff5"><sup>5</sup></xref></contrib><contrib contrib-type="author"><name><surname>Howenstine</surname><given-names>Michelle S.</given-names></name><xref ref-type="aff" rid="aff6"><sup>6</sup></xref></contrib><contrib contrib-type="author"><name><surname>Munck</surname><given-names>Anne</given-names></name><xref ref-type="aff" rid="aff7"><sup>7</sup></xref></contrib><contrib contrib-type="author"><name><surname>Mainz</surname><given-names>Jochen G.</given-names></name><xref ref-type="aff" rid="aff8"><sup>8</sup></xref></contrib><contrib contrib-type="author"><name><surname>Rodriguez</surname><given-names>Sally</given-names></name><xref ref-type="aff" rid="aff9"><sup>9</sup></xref></contrib><contrib contrib-type="author"><name><surname>Li</surname><given-names>Haihong</given-names></name><xref ref-type="aff" rid="aff9"><sup>9</sup></xref></contrib><contrib contrib-type="author"><name><surname>Yen</surname><given-names>Karl</given-names></name><xref ref-type="aff" rid="aff9"><sup>9</sup></xref></contrib><contrib contrib-type="author"><name><surname>Ordoñez</surname><given-names>Claudia L.</given-names></name><xref ref-type="aff" rid="aff9"><sup>9</sup></xref></contrib><contrib contrib-type="author"><name><surname>Ahrens</surname><given-names>Richard</given-names></name><xref ref-type="aff" rid="aff10"><sup>10</sup></xref><on-behalf-of>on behalf of the VX08-770-103 (ENVISION) Study Group</on-behalf-of></contrib><aff id="aff1"><label><sup>1</sup></label>Paediatric Respiratory Medicine and Respiratory Biomedical Research Unit, Royal Brompton and Harefield NHS Foundation Trust, London, United Kingdom</aff><aff id="aff2"><label><sup>2</sup></label>Imperial College London, London, United Kingdom</aff><aff id="aff3"><label><sup>3</sup></label>Queensland Children's Medical Research Institute, University of Queensland, Brisbane, Australia</aff><aff id="aff4"><label><sup>4</sup></label>Our Lady’s Children’s Hospital, Dublin, Ireland</aff><aff id="aff5"><label><sup>5</sup></label>British Columbia Children’s Hospital, Vancouver, British Columbia, Canada</aff><aff id="aff6"><label><sup>6</sup></label>Riley Hospital for Children, Indianapolis, Indiana</aff><aff id="aff7"><label><sup>7</sup></label>Hôpital Robert Debré, Paris, France</aff><aff id="aff8"><label><sup>8</sup></label>Jena University Hospital, Department of Pediatrics, Cystic Fibrosis Center, Jena, Germany</aff><aff id="aff9"><label><sup>9</sup></label>Vertex Pharmaceuticals Incorporated, Cambridge, Massachusetts; and</aff><aff id="aff10"><label><sup>10</sup></label>Department of Pediatrics, University of Iowa, Iowa City, Iowa</aff></contrib-group><author-notes><corresp>Correspondence and requests for reprints should be addressed to Jane C. Davies, M.D., M.B.Ch.B., Department of Gene Therapy, Imperial College London, Emmanuel Kaye Building, Manresa Road, London SW3 6LR, UK. E-mail: <email xlink:href="j.c.davies@imperial.ac.uk">j.c.davies@imperial.ac.uk</email></corresp></author-notes><pub-date pub-type="epub-ppub"><day>01</day><month>6</month><year>2013</year><pmc-comment>string-date: June 1, 2013</pmc-comment>
</pub-date><pmc-comment>Fake epub and ppub dates generated by PMC from publisher
pub-date/@pub-type='epub-ppub' </pmc-comment>
<pub-date pub-type="epub"><day>01</day><month>6</month><year>2013</year><pmc-comment>string-date: June 1, 2013</pmc-comment>
</pub-date><pub-date pub-type="ppub"><day>01</day><month>6</month><year>2013</year><pmc-comment>string-date: June 1, 2013</pmc-comment>
</pub-date><volume>187</volume><issue>11</issue><fpage>1219</fpage><lpage>1225</lpage><history><date date-type="received"><day>24</day><month>1</month><year>2013</year></date><date date-type="accepted"><day>09</day><month>3</month><year>2013</year></date></history><permissions><copyright-statement>Copyright © 2013 by the American Thoracic Society</copyright-statement><copyright-year>2013</copyright-year></permissions><self-uri content-type="pdf" xlink:href="rccm.201301-0153OC.pdf"></self-uri><abstract><p><italic>Rationale</italic>: Ivacaftor (VX-770), a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, has been shown to improve lung function, pulmonary exacerbation rate, respiratory symptoms, and weight gain compared with placebo in patients with cystic fibrosis aged 12 years or older with a <italic>G551D-CFTR</italic> mutation.</p><p><italic>Objectives</italic>: This randomized, double-blind, placebo-controlled trial evaluated ivacaftor in patients with cystic fibrosis aged 6–11 years with a <italic>G551D-CFTR</italic> mutation on at least one allele.</p><p><italic>Methods</italic>: Patients were randomly assigned to receive ivacaftor administered orally at 150 mg (n = 26) or placebo (n = 26) every 12 hours for 48 weeks in addition to existing prescribed cystic fibrosis therapies.</p><p><italic>Measurements and Main Results</italic>: Despite near-normal mean baseline values in FEV<sub>1</sub>, patients receiving ivacaftor had a significant increase in percent predicted FEV<sub>1</sub> from baseline through Week 24 versus placebo group (treatment effect, 12.5 percentage points; <italic>P</italic> < 0.001). Effects on pulmonary function were evident by 2 weeks, and a significant treatment effect was maintained through Week 48. Patients treated with ivacaftor gained, on average, 2.8 kg more than those receiving placebo at Week 48 (<italic>P</italic> < 0.001). The change from baseline through Week 48 in the concentration of sweat chloride, a measure of CFTR activity, with ivacaftor was −53.5 mmol/L (<italic>P</italic> < 0.001) versus placebo. The incidence of adverse events was similar in the two groups.</p><p><italic>Conclusions</italic>: In patients who are younger and healthier than those in previously studied populations, ivacaftor demonstrated a significant improvement in pulmonary function, weight, and CFTR activity compared with placebo. Clinical trial registered with <ext-link ext-link-type="uri" xlink:href="www.clinicaltrials.gov">www.clinicaltrials.gov</ext-link> (NCT00909727).</p></abstract><kwd-group><title>Keywords</title><kwd>cystic fibrosis</kwd><kwd>cystic fibrosis transmembrane conductance regulator protein</kwd><kwd>cystic fibrosis, pulmonary</kwd><kwd><italic>G551D-CFTR</italic> mutation</kwd><kwd>sweat test</kwd></kwd-group><counts><fig-count count="3"></fig-count><table-count count="2"></table-count><page-count count="7"></page-count></counts></article-meta></front></pmc></record>Pour manipuler ce document sous Unix (Dilib)
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