• Je něco špatně v tomto záznamu ?

Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST)

EF. McKone, D. Borowitz, P. Drevinek, M. Griese, MW. Konstan, C. Wainwright, F. Ratjen, I. Sermet-Gaudelus, B. Plant, A. Munck, Y. Jiang, G. Gilmartin, JC. Davies, . ,

. 2014 ; 2 (11) : 902-10. (Respiratory medicine) [pub] 20141009

Jazyk angličtina Země Anglie, Velká Británie

Typ dokumentu klinické zkoušky, fáze III, časopisecké články, práce podpořená grantem

Perzistentní odkaz   https://www.medvik.cz/link/bmc16000586

BACKGROUND: Ivacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, is approved for the treatment of patients with cystic fibrosis aged 6 years or older with Gly551Asp-CFTR. We assessed the safety and efficacy of ivacaftor during 96 weeks of PERSIST in patients with cystic fibrosis who completed a previous 48-week, placebo-controlled trial of the drug (STRIVE or ENVISION). METHODS: In this phase 3, open-label extension study, patients received ivacaftor 150 mg every 12 h in addition to their prescribed cystic fibrosis therapies. Patients who received placebo in their previous study initiated ivacaftor in this extension study. Patients were eligible if they had a Gly551Asp-CFTR mutation on at least one allele. The primary objective was to assess the long-term safety profile of ivacaftor as assessed by adverse events, clinical laboratory assessments, electrocardiograms, vital signs, and physical examination; secondary measures included change in forced expiratory volume in one second (FEV1), weight, and pulmonary exacerbations. This study is registered with ClinicalTrials.gov, number NCT01117012 and EudraCT, number 2009-012997-11. FINDINGS: Between July 8, 2010, and April 8, 2013, 144 adolescents/adults (≥12 years) from STRIVE and 48 children (6-11 years) from ENVISION were enrolled. Across both trials, 38 (20%) patients had a serious adverse event during the first 48 weeks and 44 (23%) during the subsequent 48 weeks. Two adults (1%) and one child (<1%) discontinued because of adverse events. The most common adverse events were pulmonary exacerbation, cough, and upper respiratory tract infection. Patients previously treated with ivacaftor had sustained improvements in FEV1, weight, and rate of pulmonary exacerbations for up to 144 weeks of treatment. Among adolescents/adults and children who previously received ivacaftor, absolute change in FEV1 at week 96 (144 weeks ivacaftor) was 9·4 and 10·3 % points and absolute increase in weight was 4·1 kg and 14·8 kg, respectively. For adolescents/adults only, the pulmonary exacerbation rate remained suppressed compared with that of patients who received placebo in the placebo-controlled study. INTERPRETATION: At 144 weeks of treatment, ivacaftor was well tolerated, with no new safety concerns. Ivacaftor also provided durable effects for 144 weeks in patients who had received active treatment in the placebo-controlled study. Those patients who previously received placebo had improvements comparable to those of patients treated with ivacaftor in the placebo-controlled study. FUNDING: Vertex Pharmaceuticals Inc.

Citace poskytuje Crossref.org

000      
00000naa a2200000 a 4500
001      
bmc16000586
003      
CZ-PrNML
005      
20160520110127.0
007      
ta
008      
160108s2014 enk f 000 0|eng||
009      
AR
024    7_
$a 10.1016/S2213-2600(14)70218-8 $2 doi
035    __
$a (PubMed)25311995
040    __
$a ABA008 $b cze $d ABA008 $e AACR2
041    0_
$a eng
044    __
$a enk
100    1_
$a McKone, Edward F $u St Vincent's University Hospital, Dublin, Ireland; University College Dublin School of Medicine, Dublin, Ireland. Electronic address: e.mckone@svuh.ie.
245    10
$a Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST) / $c EF. McKone, D. Borowitz, P. Drevinek, M. Griese, MW. Konstan, C. Wainwright, F. Ratjen, I. Sermet-Gaudelus, B. Plant, A. Munck, Y. Jiang, G. Gilmartin, JC. Davies, . ,
520    9_
$a BACKGROUND: Ivacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, is approved for the treatment of patients with cystic fibrosis aged 6 years or older with Gly551Asp-CFTR. We assessed the safety and efficacy of ivacaftor during 96 weeks of PERSIST in patients with cystic fibrosis who completed a previous 48-week, placebo-controlled trial of the drug (STRIVE or ENVISION). METHODS: In this phase 3, open-label extension study, patients received ivacaftor 150 mg every 12 h in addition to their prescribed cystic fibrosis therapies. Patients who received placebo in their previous study initiated ivacaftor in this extension study. Patients were eligible if they had a Gly551Asp-CFTR mutation on at least one allele. The primary objective was to assess the long-term safety profile of ivacaftor as assessed by adverse events, clinical laboratory assessments, electrocardiograms, vital signs, and physical examination; secondary measures included change in forced expiratory volume in one second (FEV1), weight, and pulmonary exacerbations. This study is registered with ClinicalTrials.gov, number NCT01117012 and EudraCT, number 2009-012997-11. FINDINGS: Between July 8, 2010, and April 8, 2013, 144 adolescents/adults (≥12 years) from STRIVE and 48 children (6-11 years) from ENVISION were enrolled. Across both trials, 38 (20%) patients had a serious adverse event during the first 48 weeks and 44 (23%) during the subsequent 48 weeks. Two adults (1%) and one child (<1%) discontinued because of adverse events. The most common adverse events were pulmonary exacerbation, cough, and upper respiratory tract infection. Patients previously treated with ivacaftor had sustained improvements in FEV1, weight, and rate of pulmonary exacerbations for up to 144 weeks of treatment. Among adolescents/adults and children who previously received ivacaftor, absolute change in FEV1 at week 96 (144 weeks ivacaftor) was 9·4 and 10·3 % points and absolute increase in weight was 4·1 kg and 14·8 kg, respectively. For adolescents/adults only, the pulmonary exacerbation rate remained suppressed compared with that of patients who received placebo in the placebo-controlled study. INTERPRETATION: At 144 weeks of treatment, ivacaftor was well tolerated, with no new safety concerns. Ivacaftor also provided durable effects for 144 weeks in patients who had received active treatment in the placebo-controlled study. Those patients who previously received placebo had improvements comparable to those of patients treated with ivacaftor in the placebo-controlled study. FUNDING: Vertex Pharmaceuticals Inc.
650    _2
$a mladiství $7 D000293
650    _2
$a dospělí $7 D000328
650    _2
$a aminofenoly $x škodlivé účinky $x terapeutické užití $7 D000627
650    _2
$a dítě $7 D002648
650    _2
$a kašel $x chemicky indukované $7 D003371
650    _2
$a cystická fibróza $x farmakoterapie $x genetika $x patofyziologie $7 D003550
650    _2
$a protein CFTR $x genetika $7 D019005
650    _2
$a progrese nemoci $7 D018450
650    _2
$a ženské pohlaví $7 D005260
650    _2
$a usilovný výdechový objem $x účinky léků $7 D005541
650    _2
$a lidé $7 D006801
650    _2
$a mužské pohlaví $7 D008297
650    _2
$a mutace $7 D009154
650    _2
$a chinolony $x škodlivé účinky $x terapeutické užití $7 D015363
650    _2
$a infekce dýchací soustavy $x chemicky indukované $7 D012141
650    _2
$a časové faktory $7 D013997
650    _2
$a hmotnostní přírůstek $x účinky léků $7 D015430
650    _2
$a mladý dospělý $7 D055815
655    _2
$a klinické zkoušky, fáze III $7 D017428
655    _2
$a časopisecké články $7 D016428
655    _2
$a práce podpořená grantem $7 D013485
700    1_
$a Borowitz, Drucy $u State University of New York at Buffalo, Buffalo, NY, USA.
700    1_
$a Drevinek, Pavel $u University Hospital Motol and 2nd Faculty of Medicine, Charles University, Prague, Czech Republic.
700    1_
$a Griese, Matthias $u Hauner Children's Hospital, Munich, Germany; Ludwig Maximillians University, Munich, Germany.
700    1_
$a Konstan, Michael W $u Case Western Reserve University School of Medicine and Rainbow Babies and Children's Hospital, Cleveland, OH, USA.
700    1_
$a Wainwright, Claire $u Royal Children's Hospital and Queensland Children's Medical Research Institute, The University of Queensland, Brisbane, QLD, Australia.
700    1_
$a Ratjen, Felix $u Division of Respiratory Medicine, Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada; University of Toronto, Toronto, ON, Canada.
700    1_
$a Sermet-Gaudelus, Isabelle $u Assistance Publique Hôpitaux de Paris, INSERM U 1151 Université Paris Sorbonne, Paris, France.
700    1_
$a Plant, Barry $u Cork University Hospital, University College Cork, Ireland.
700    1_
$a Munck, Anne $u Assistance Publique-Hôpitaux de Paris, Hôpital Robert Debré, CF Centre, Université Paris 7, Paris, France.
700    1_
$a Jiang, Ying $u Vertex Pharmaceuticals Inc, Boston, MA, USA.
700    1_
$a Gilmartin, Geoffrey $u Vertex Pharmaceuticals Inc, Boston, MA, USA.
700    1_
$a Davies, Jane C $u Royal Brompton Hospital, London, UK.
700    1_
$a ,
773    0_
$w MED00188827 $t The Lancet. Respiratory medicine. Respiratory medicine $x 2213-2619 $g Roč. 2, č. 11 (2014), s. 902-10
856    41
$u https://pubmed.ncbi.nlm.nih.gov/25311995 $y Pubmed
910    __
$a ABA008 $b sig $c sign $y a $z 0
990    __
$a 20160108 $b ABA008
991    __
$a 20160520110240 $b ABA008
999    __
$a ok $b bmc $g 1102867 $s 924792
BAS    __
$a 3
BAS    __
$a PreBMC
BMC    __
$a 2014 $b 2 $c 11 $d 902-10 $e 20141009 $i 2213-2619 $m The Lancet Respiratory medicine $n Lancet Respir Med $x MED00188827 $o Respiratory medicine
LZP    __
$a Pubmed-20160108

Najít záznam

Citační ukazatele

Možnosti archivace