title: Efficacy of inhaled N-acetylcysteine monotherapy in patients with early stage idiopathic pulmonary fibrosis.
abstract: OBJECTIVE
  Idiopathic pulmonary fibrosis (IPF) is a fatal disorder for which there are currently no specific or effective medical treatments. A multicentre, prospective, randomized, controlled clinical trial was conducted to assess the efficacy of inhaled N-acetylcysteine (NAC) monotherapy in Japanese patients with early stage IPF.
  
  
  METHODS
  Eligible patients had well-defined IPF of mild-to-moderate severity, with no desaturation on exercise. Of 100 patients screened, 76 were randomly assigned to an NAC treatment group (group A; n = 38) that received 352.4 mg of NAC by inhalation twice daily or to a control group (group B; n = 38) that received no therapy. The primary endpoint was the change from baseline in forced vital capacity (FVC) at 48 weeks.
  
  
  RESULTS
  There were no significant overall differences in the change in FVC between groups A and B. Post hoc exploratory analyses showed that NAC therapy was associated with stability of FVC in (i) a subset of patients with initial FVC <95% of predicted (n = 49; difference in FVC decline 0.12 L; P = 0.02) and (ii) in patients with initial diffusing capacity of carbon monoxide <55% of predicted (n = 21; difference in FVC decline 0.17 L; P = 0.009).
  
  
  CONCLUSIONS
  These findings indicate that NAC monotherapy may have some beneficial effect in patients with early stage IPF. Further trials in more select IPF populations with progressive disease are required to prove the efficacy of inhaled NAC.
authors.affiliation: Department of Respiratory Medicine, Toho University Omori Medical Center, Tokyo, Japan.
authors.forename: Sakae
authors.initials: S
authors.lastname: Homma
authors.forename: Arata
authors.initials: A
authors.lastname: Azuma
authors.forename: Hiroyuki
authors.initials: H
authors.lastname: Taniguchi
authors.forename: Takashi
authors.initials: T
authors.lastname: Ogura
authors.forename: Yoshiro
authors.initials: Y
authors.lastname: Mochiduki
authors.forename: Yukihiko
authors.initials: Y
authors.lastname: Sugiyama
authors.forename: Koichiro
authors.initials: K
authors.lastname: Nakata
authors.forename: Kunihiko
authors.initials: K
authors.lastname: Yoshimura
authors.forename: Masahiro
authors.initials: M
authors.lastname: Takeuchi
authors.forename: Shoji
authors.initials: S
authors.lastname: Kudoh
mesh_terms: D000111:Acetylcysteine / Q000627:therapeutic use*; D000280:Administration, Inhalation; D000368:Aged; D044466:Asian People / Q000706:statistics & numerical data; D018450:Disease Progression; D005260:Female; D006801:Humans; D054990:Idiopathic Pulmonary Fibrosis / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D012720:Severity of Illness Index; D016896:Treatment Outcome; D014797:Vital Capacity / Q000187:drug effects
chemical_list: D000111:Acetylcysteine