Hypertonic saline in patients with primary ciliary dyskinesia: on the road to evidence-based treatment for a rare lung disease

The study of Paff et al. [1], published in this issue of the European Respiratory Journal on Rare Disease Day 2017, describes the first randomised clinical trial of inhaled hypertonic saline on quality of life and other outcomes in patients with primary ciliary dyskinesia (PCD). Although the results were negative, the study is a major step towards evidence-based treatment of PCD. Participation in clinical studies should be an essential part of routine care for patients with rare lung diseases <http://ow.ly/WfAm307Tqsg> C.E. Kuehni is a participant in the European Union's Seventh Framework Programme under EG-GA No. 35404 BESTCILIA: Better Experimental Screening and Treatment for Primary Ciliary Dyskinesia. All authors participate in the network of COST Action BEAT-PCD: Better Experimental Approaches to Treat PCD (BM 1407); C.E. Kuehni and M. Goutaki were members of the ERS PCD Taskforce for PCD Diagnostics (ERS TF-2014-04). C.E. Kuehni receives funding from the Swiss National Science Foundation (32003B_162820). The views expressed in this manuscript do not reflect official views of these collaborations. We thank Christopher Ritter (Institute of Social and Preventive Medicine, University of Bern, Switzerland) for proofreading and editorial contributions.