scholarly journals 55 Lumacaftor/ivacaftor (LUM/IVA) in patients (pts) with cystic fibrosis (CF) and advanced lung disease homozygous for F508del-CFTR: a 24-week open-label study

2017 ◽  
Vol 16 ◽  
pp. S78 ◽  
Author(s):  
J. Taylor-Cousar ◽  
M. Jain ◽  
T.L. Barto ◽  
T. Haddad ◽  
J. Atkinson ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Lung Disease ◽  
Open Label ◽  
Open Label Study ◽  
Label Study

Multicenter, open-label study of recombinant human DNase in cystic fibrosis patients with moderate lung disease

1998 ◽  
Vol 26 (3) ◽  
pp. 155-161 ◽  
Author(s):  
H. Karsten Harms ◽  
Elias Matouk ◽  
Guy Tournier ◽  
Horst von der Hardt ◽  
Peter H. Weller ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Lung Disease ◽  
Open Label ◽  
Open Label Study ◽  
Label Study

Effect of Bosentan on Systemic Sclerosis-associated Interstitial Lung Disease Ineligible for Cyclophosphamide Therapy: A Prospective Open-label Study

2011 ◽  
Vol 38 (10) ◽  
pp. 2186-2192 ◽  
Author(s):  
YOSHIAKI FURUYA ◽  
MASATAKA KUWANA
Keyword(s):  
Systemic Sclerosis ◽  
Interstitial Lung Disease ◽  
Pulmonary Function ◽  
Lung Disease ◽  
Pulmonary Function Tests ◽  
High Dose ◽  
Open Label ◽  
High Resolution Computed Tomography ◽  
Open Label Study ◽  
Label Study

Objective.To evaluate the clinical benefits of the endothelin receptor antagonist bosentan on interstitial lung disease (ILD) in patients with systemic sclerosis (SSc) who are ineligible for cyclophosphamide (CYC) therapy.Methods.In this prospective open-label study, 9 patients with SSc and ILD received bosentan for 24 months. The main reasons for avoiding CYC included severely impaired lung function, long disease duration, and relapse after CYC treatment. Pulmonary function tests and Doppler echocardiograms were evaluated every 6 months, and high-resolution computed tomography (HRCT) was performed every 12 months. For an extended survival analysis, 17 historical controls who met the inclusion criteria at referral and had not used any immunosuppressive or antifibrotic agents thereafter were selected from the SSc database.Results.Two patients did not finish the study; one developed vasculitis requiring high-dose corticosteroids and another died of bacterial pneumonia. The remaining 7 patients tolerated bosentan and completed the study period. There were trends toward mildly reduced forced vital capacity, total lung capacity, and diffusing capacity for carbon monoxide over time. Two patients developed pulmonary hypertension during the 24-month period. HRCT scores for ground-glass opacity, pulmonary fibrosis, and honeycomb cysts gradually increased. In the extended study, there was no difference in cumulative survival rate between the bosentan-treated and historical control groups.Conclusion.The gradual worsening of pulmonary function and HRCT findings in patients treated with bosentan was consistent with the natural course of SSc-associated ILD. This study does not support the use of bosentan for SSc-associated ILD even when CYC treatment is inadvisable.

scholarly journals WS7.5 Interim results of the phase 3 open-label study of ataluren in nonsense mutation cystic fibrosis (nmCF)

2013 ◽  
Vol 12 ◽  
pp. S15 ◽  
Author(s):  
E. Kerem ◽  
M. Wilschanski ◽  
I. Sermet-Gaudelus ◽  
K. De Boeck ◽  
F.J. Accurso ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Nonsense Mutation ◽  
Open Label ◽  
Phase 3 ◽  
Open Label Study ◽  
Label Study

scholarly journals Open label study of inhaled aztreonam for Pseudomonas eradication in children with cystic fibrosis: The ALPINE study

2015 ◽  
Vol 14 (1) ◽  
pp. 111-119 ◽  
Author(s):  
H.A.W.M. Tiddens ◽  
K. De Boeck ◽  
J.P. Clancy ◽  
M. Fayon ◽  
Arets H.G.M. ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Open Label ◽  
Open Label Study ◽  
Label Study
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