title: Insights from the German Compassionate Use Program of Nintedanib for the Treatment of Idiopathic Pulmonary Fibrosis.
abstract: BACKGROUND
  Nintedanib is approved for the treatment of idiopathic pulmonary fibrosis (IPF) and has been shown to slow disease progression by reducing annual lung function decline.
  
  
  OBJECTIVE
  To evaluate the results of a large cohort of IPF patients treated with nintedanib within a compassionate use program (CUP) in Germany (9 centers).
  
  
  METHODS
  Patients (≥40 years) were required to have a confirmed diagnosis of IPF, a forced vital capacity (FVC) ≥50% predicted (pred.) and a carbon monoxide diffusing capacity (DLCO) 30-79% pred. and not to be eligible for pirfenidone treatment. Clinical data, pulmonary function tests and adverse events were recorded up to July 2015.
  
  
  RESULTS
  Sixty-two patients (48 male/14 female) with moderate IPF (FVC 64 ± 17% pred. and DLCO 40 ± 10% pred.) were treated with nintedanib. 77% of patients switched from pirfenidone (mean treatment duration 14 ± 2 months) mostly due to disease progression (mean decline in FVC 7.4 ± 3% pred. in the 6 months prior to nintedanib intake). Initiation of nintedanib treatment occurred 69 ± 29 months after IPF diagnosis, and mean treatment duration was 8 ± 4 months. Most patients (63%) stabilized 6 months after treatment start (mean FVC decline 3 ± 1 vs. -17 ± 2% in patients with disease progression; p < 0.01). The most common adverse events were diarrhea (63%) and weight loss (50%). Dose reduction occurred in 34% of cases and treatment discontinuation in 10%.
  
  
  CONCLUSIONS
  Nintedanib treatment was generally well tolerated and was associated with FVC stabilization in the majority of IPF patients in this CUP setting where most patients were not treatment naïve. Our data are in agreement with the previously published data.
authors.affiliation: Ruhrlandklinik, University of Duisburg-Essen, Essen, Germany.
authors.forename: Francesco
authors.initials: F
authors.lastname: Bonella
authors.forename: Michael
authors.initials: M
authors.lastname: Kreuter
authors.forename: Lars
authors.initials: L
authors.lastname: Hagmeyer
authors.forename: Claus
authors.initials: C
authors.lastname: Neurohr
authors.forename: Claus
authors.initials: C
authors.lastname: Keller
authors.forename: Martin J
authors.initials: MJ
authors.lastname: Kohlhaeufl
authors.forename: Joachim
authors.initials: J
authors.lastname: Müller-Quernheim
authors.forename: Katrin
authors.initials: K
authors.lastname: Milger
authors.forename: Antje
authors.initials: A
authors.lastname: Prasse
mesh_terms: D000368:Aged; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D057176:Compassionate Use Trials; D005260:Female; D005858:Germany; D006801:Humans; D054990:Idiopathic Pulmonary Fibrosis / Q000188:drug therapy*; D007211:Indoles / Q000627:therapeutic use*; D008297:Male; D055118:Medication Adherence; D012189:Retrospective Studies; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D007211:Indoles; C530716:nintedanib