Cystic fibrosis papers of the year 2010-2011

Abstract

This review is based upon a Medline search of the literature on cystic fibrosis for the latter half of 2010 onwards. The Medline search was carried out in September 2011 and also includes some references published after September 2011. This search revealed articles relating to approximately 482 paediatric studies and 419 adult studies with much overlap between the two groups. Taking into account the multidisciplinary nature of the audience for this symposium and also taking into account the topics in the rest of the programme for the day, a shortlist of 65 papers was then derived. A diversity of topics was clearly necessary and also a mixture of paediatric and adult publications, hence the following papers were chosen.

An Update on In Utero Gene Therapy for Cystic Fibrosis

“…17 After 28 days, researchers discovered that the group of patients who received 150 mg of VX-770 had a median change in nasal potential difference of −3.5 mV (P = .13 vs placebo), a median change of the level of sweat chloride of −59.5 mmol per liter (P = .02 vs placebo), and a median change in the percentage of predicted forced expiratory volume in 1 second of 8.7% (P = .56 vs placebo). 17,18 Following the positive results published by NEJM and multiple other studies that yielded similar findings in support of the effectiveness of VX-770 among patients with the G551D gating mutation, the drug became accepted as an established, reliable postnatal treatment option for patients with CF and the most common gating mutation.…”