The diagnosis of idiopathic pulmonary fibrosis (IPF) is currently one of the most common
diagnoses for patients under evaluation for lung transplantation. In recent years, an
absolute increase in prevalence/ incidence of IPF has been observed. There is evidence that
patients with IPF on waiting list for lung transplantation might benefit from pirfenidone
treatment. Until now, no data are published regarding this important issue in lung
transplantation.
Primary objective is to determine whether there is a difference in the duration time of
mechanical ventilation (weaning) directly after lung transplantation between patients treated
with pirfenidone and patients without pirfenidone treatment. The Secondary objectives are to
determine whether there are differences between the pirfenidone treatment group and the
control group regarding survival after LUTX, the score on the Saint Georges Respiratory
Questionnaire and the decline in forced vital capacity (FVC%) In this Investigator initiated,
non- interventional single center study , patients on the waiting list for transplant
pirfenidone treatment receive oral pirfenidone at the standard dose of 2403 mg per day. The
treatment duration will range from 6 to 12 months. A control group will be used to correlate
the outcome-parameters for a descriptive comparison. The control group includes patients with
IPF on the waiting list who were on another IPF specific (or no) treatment for IPF The Study
Population are Patients aged between 40-70 years who are admitted to the lung transplantation
department and fulfill the international criteria for idiopathic pulmonary fibrosis (
existence of a usual interstitial pneumonia (UIP) pattern in the high-resolution computed
tomography (HRCT) is necessary).
Variables: Duration of mechanical ventilation after LUTX (hours), Forced Vital capacity
relative to reference value at baseline (FVC0%), Forced Vital capacity relative to reference
value after 6 months (FVC6%),Forced Vital capacity relative to reference value after 12
months (FVC12%) Study Size: 30 patients in the Pirfenidone group, 20 patients in the control
group.
For the primary Endpoint, the mean, standard deviation, median, minimum and maximum of the
weaning time of patients who received a pirfenidone treatment, as well as of patients from
the control group will be computed and presented in a table. Additionally, a Kaplan-Meier
curve will be estimated and plotted alongside the respective 95% CI calculated using the
method of Brookmeyer and Crowley. Furthermore, a stepwise linear regression using forward
selection and Age, RBMI, FVC0%, (FVC6%-FVC0%), TLC, FEV1% and ECMO, as well as the
pirfenidone treatment as predictors will be computed. The null hypothesis is that the
pirfenidone treatment has no influence on the weaning time. The according model coefficient
estimate and standard error will be used to test the null hypothesis using a t-test at
significance level α=0.05.
For the secondary endpoints, the mean, standard deviation, median, minimum and maximum of
patients who received a pirfenidone treatment, as well as of patients from the control group
will be computed and presented in a tableStepwise Cox Regression using forward selection and
Age, RBMI, FVC0%, (FVC6%-FVC0%) and ECMO, as well as the pirfenidone treatment as predictors
will be computed in order to compare the treatment and the control group a . If p-values are
calculated for the secondary endpoint analysis, they serve only descriptive purposes.
Therefore no multiple testing corrections are applied.
