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N Engl J Med. 2014 May 29;370(22):2093-101. doi: 10.1056/NEJMoa1401739. Epub 2014 May 18.

Randomized trial of acetylcysteine in idiopathic pulmonary fibrosis.

Collaborators (158)

Martinez FJ, Raghu G, Schwarz M, Toews GB, Hunninghake G, Zibrak J, Demersky A, Vey M, Rosas IO, Debrosse P, Culver DA, Chapman J, Olman M, Parambil JG, Wehrmann R, Yadav R, Morrison LD, Steele MP, Haram T, Kidd R, Kallay M, Lyda E, Ryu JH, Utz JP, Limper AH, Daniels CE, Meiras K, Walsh S, Sahn S, O'Banner N, Stokes F, Brown KK, Bair C, Kervitsky D, Ettinger NA, Merli S, Criner G, Swift IQ, Satti A, Cordova F, Patel N, West K, Jones G, Lasky JA, Ditta S, de Andrade J, Thannickal VJ, Stewart M, Belperio J, Lynch JP 3rd, Calahan E, Lopez P, King TE Jr, Collard HR, Golden J, Lee J, Wolters P, Eller A, Noth I, White S, Hogarth DK, Brown C, Demchuk C, Holland S, Holly M, Rahimova G, Sandbo N, Sardin L, Strek ME, Roman J, Perez R, Perez T, Glassberg MK, Simonet E, Martinez FJ, Baumann K, Chan K, Chughtai A, Gross B, Flaherty KR, Han ML, Hyzy R, Kazerooni E, Moore B, Myers J, Toews GB, White E, Dahlgren D, Majors C, Rossman M, Kreider M, Le K, Fitzgerald J, Glazer C, Scholand MB, Brewster L, Johnson A, Raghu G, Berry-Bell P, Snydsman A, Loyd JE, Lancaster L, Lawson W, Greer R, Kinser K, Richardson R, Mason W, Kaner RJ, Bandong K, Valerio R, Tobias J, Antin-Ozerkis D, Holm C, Estrom J, Lynch DA, Colby T, Anstrom KJ, Burnett B, Eisenstein EL, Sundy JS, Davidson-Ray L, Dignacco P, Edwards R, Anderson R, Beci R, Calvert S, Gentry-Bumpass T, Hill D, Hwang K, Kaur J, Matti C, Meredith A, Pesarchick J, Ramey S, Roberts RS, Sharlow A, Winsor J, Yang Q, Yow E, Kiley J, Weinmann GG, Reynolds H, Peavy H, Schmetter B, Tian X, Davis GS, Levine R, Nathan SD, Rounds S, Thompson BT, Thompson B, Bitterman PB, Franks TJ, Idell S, Piantadosi S, Rom WN, Selman M, Wilkes DS.



Acetylcysteine has been suggested as a beneficial treatment for idiopathic pulmonary fibrosis, although data from placebo-controlled studies are lacking.


In our initial double-blind, placebo-controlled trial, we randomly assigned patients who had idiopathic pulmonary fibrosis with mild-to-moderate impairment in pulmonary function to receive a three-drug regimen of prednisone, azathioprine, and acetylcysteine; acetylcysteine alone; or placebo. The study was interrupted owing to safety concerns associated with the three-drug regimen. The trial continued as a two-group study (acetylcysteine vs. placebo) without other changes; 133 and 131 patients were enrolled in the acetylcysteine and placebo groups, respectively. The primary outcome was the change in forced vital capacity (FVC) over a 60-week period.


At 60 weeks, there was no significant difference in the change in FVC between the acetylcysteine group and the placebo group (-0.18 liters and -0.19 liters, respectively; P=0.77). In addition, there were no significant differences between the acetylcysteine group and the placebo group in the rates of death (4.9% vs. 2.5%, P=0.30 by the log-rank test) or acute exacerbation (2.3% in each group, P>0.99).


As compared with placebo, acetylcysteine offered no significant benefit with respect to the preservation of FVC in patients with idiopathic pulmonary fibrosis with mild-to-moderate impairment in lung function. (Funded by the National Heart, Lung, and Blood Institute and others; number, NCT00650091.).

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