First experience in Switzerland in Phe508del homozygous cystic fibrosis patients with end-stage pulmonary disease enrolled in a lumacaftor-ivacaftor therapy trial – preliminary results | Zendy

Christian Murer | Zendy; Lars Huber | Zendy; Thomas Kurowski | Zendy; A. Hirt | Zendy; C. Robinson | Zendy; Urs Bürgi | Zendy; Christian Benden | Zendy

Open Access

First experience in Switzerland in Phe508del homozygous cystic fibrosis patients with end-stage pulmonary disease enrolled in a lumacaftor-ivacaftor therapy trial – preliminary results

Author(s) -

Christian Murer,

Lars Huber,

Thomas Kurowski,

A. Hirt,

C. Robinson,

Urs Bürgi,

Christian Benden

Publication year - 2018

Publication title -

schweizerische medizinische wochenschrift

Language(s) - English

Resource type - Journals

ISSN - 0036-7672

DOI - 10.4414/smw.2018.14593

Subject(s) - medicine , ivacaftor , cystic fibrosis , vital capacity , cystic fibrosis transmembrane conductance regulator , adverse effect , body mass index , pulmonary function testing , surgery , lung , diffusing capacity , lung function

Phe508del homozygous cystic fibrosis patients with end-stage pulmonary disease tolerated LUM/IVA, although RAEs occurred early and were severe. This positive finding was probably due to the stepwise dose increases. There was clinical benefit mainly from reduction in AER and stabilisation of lung function. We propose that all suitable Phe508del homozygous cystic fibrosis patients with end-stage pulmonary disease should have a trial of LUM/IVA treatment in experienced centres.

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