March 2012. Volume 8. Number 1

In patients with cystic fibrosis with the mutation G551D, ivacaftor can improve lung function and other clinical parameters

 
 
 
 
 
 
 
 
 
 
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AVC | Critically appraised articles

Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevínek P et al; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365:1663-72.
Reviewers: Buñuel Álvarez JC1, Lojo Pons P2, Castan Campanera A3.
1Àrea Bàsica de Salut Girona-4. Institut Català de la Salut. Girona. España.
2Hospital Universitario de Girona Josep Trueta. Girona. España.
3Hospital Universitario de Girona Josep Trueta. Girona. Girona. España.
Correspondence: José Cristóbal Buñuel Álvarez. Email: jcbunuel@gmail.com
Reception date: 27/01/2012
Acceptance date: 03/02/2012
Publication date: 08/02/2012

Abstract

Authors' conclusions: ivacaftor administration was associated with a significant and sustained improvement in lung function between the second and the 48 week of follow up in patients with cystic fibrosis (G551D mutation). A decrease in pulmonary exacerbations was also found, as well as improvement in patient-perceived symptoms and weight gain in patients who received ivacaftor.

Reviewers' commentary: the results of this study are encouraging but they should be confirmed by others that solved its methodological limitations (lack of randomisation sequence concealment and doubts about the proper masking). Variables such as safety or prolonged beneficial effects of ivacaftor beyond the week 48 should be assessed in well designed studies with larger sample sizes. It would be recommendable to use the pulmonary events rate or the number of hospital admissions as first outcomes.

How to cite this article

Buñuel Álvarez JC, Lojo Pons P, Castan Campanera A. En pacientes con fibrosis quística con la mutación G551D el ivacaftor puede mejorar la función pulmonar y otros parámetros clínicos. Evid Pediatr. 2012;8:14.

AVC | Critically appraised articles

Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevínek P et al; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365:1663-72.
Reviewers: Buñuel Álvarez JC1, Lojo Pons P2, Castan Campanera A3.
1Àrea Bàsica de Salut Girona-4. Institut Català de la Salut. Girona. España.
2Hospital Universitario de Girona Josep Trueta. Girona. España.
3Hospital Universitario de Girona Josep Trueta. Girona. Girona. España.
Correspondence: José Cristóbal Buñuel Álvarez. Email: jcbunuel@gmail.com
Reception date: 27/01/2012
Acceptance date: 03/02/2012
Publication date: 08/02/2012

How to cite this article

Buñuel Álvarez JC, Lojo Pons P, Castan Campanera A. En pacientes con fibrosis quística con la mutación G551D el ivacaftor puede mejorar la función pulmonar y otros parámetros clínicos. Evid Pediatr. 2012;8:14.

References

  1. Patient registry: 2008 annual data report to the Center directors. Bethesda, MD: Cystic Fibrosis Foundation; 2009.
  2. ClinicalTrials.gov. Study of VX-770 in Cystic Fibrosis Subjects Age 6 to 11 With the G551D Mutation [en línea][fecha de consulta: 2-II-2012]. Disponible en: http://clinicaltrials.gov/ct2/show/NCT00909727?term=NCT00909727&rank=1
  3. Davis PB. Therapy for cystic fibrosis—the end of the beginning? N Engl J Med. 2011;365:1734-5.
08/02/2012

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