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N Engl J Med. 2014 May 29;370(22):2083-92. doi: 10.1056/NEJMoa1402582. Epub 2014 May 18.

A phase 3 trial of pirfenidone in patients with idiopathic pulmonary fibrosis.

Collaborators (146)

Allen JN Jr, Alves RL, Antin-Ozerkis D, Bajwa AA, Bascom R, Baughman R, Beckert LE, Belperio J, Ben-Dov I, Breuer R, Butler JA, de Carvalho CR, Chan AL, Chang J, Chavarría Martínez U, Clifford DP, Cordova F, Corte T, Covelli HD, Davies H, Davis GS, De Andrade JM, Díaz Castañón JJ, Dilling DF, Eaton S, Enelow R, Ettinger N, Evans T, Feldman JP, Fiss E, Flaherty KR, Floreani A, Foti PR, Frost AE, Ganesh S, Garay S, Gerke A, Gibson K, Ginns LE, Girgis R, Girod C, Golden J, Gotfried MH, Grinnan D, Guerreros Benavides AG, Haller HD Jr, Hamblin MJ, Holmes M, Horiuchi TK, Horton M, Huggins JT, Iberico Barrera CA, Ilowite JS, Kallay MC, Kim HJ, Kinasewitz G, Kramer MR, LaCamera P, Landis J, Lasky JA, Lee AS, Lipchik R, Lorch DG Jr, Low SY, Malouf M, Matsuno Fuchigami A, Meyer KC, Migliore C, Morrison LD, Morrow L, Musk M, Nambiar A, de Oliveira ME, Padilla M, Panos R, Pantano J, Parambil J, Patel NM, Perea Sánchez RA, Perez RL, Peroš-Golubiĉić T, Piñeiro García Calderón A, Posadas Valay R, Pun Leon LE, Rai NS, Ramaswamy M, Rose CE, Rosen GD, Rubin AS, Russell T, Russell DW, Ryu JH, Salazar Oré DJ, Sanchez J, Schaumberg T, Scholand MB, Schwarz YA, Selman Lama ME, Serisier D, Shen W, Shlobin O, Simonelli P, Sinkowitz DM, Strek M, Thien F, Thompson AB, Tillis WP, Tino G, Torres Sales JW, Tudoric N, Veale A, Veitch E, Wencel ML, Wesselius LJ, Wilsher M, Yagan M, Yigla M, Yoder MA, Zawadski DK, King TE Jr, Noble PW, Bradford WZ, Fagan EA, Glaspole I, Glassberg MK, Lancaster L, Lederer DJ, Nathan SD, Pereira CA, Sahn SA, Swigris JJ, Davis CS, Grippi M, Wise R, Zisman DA, Danoff SK, Kaner RJ, Whelan T, Godwin J, Kanne JP, Lynch DA, Webb WR, Colby TV, Katzenstein AL, Leslie KO, Tazelaar HD.

Erratum in

  • N Engl J Med. 2014 Sep 18;371(12):1172.



In two of three phase 3 trials, pirfenidone, an oral antifibrotic therapy, reduced disease progression, as measured by the decline in forced vital capacity (FVC) or vital capacity, in patients with idiopathic pulmonary fibrosis; in the third trial, this end point was not achieved. We sought to confirm the beneficial effect of pirfenidone on disease progression in such patients.


In this phase 3 study, we randomly assigned 555 patients with idiopathic pulmonary fibrosis to receive either oral pirfenidone (2403 mg per day) or placebo for 52 weeks. The primary end point was the change in FVC or death at week 52. Secondary end points were the 6-minute walk distance, progression-free survival, dyspnea, and death from any cause or from idiopathic pulmonary fibrosis.


In the pirfenidone group, as compared with the placebo group, there was a relative reduction of 47.9% in the proportion of patients who had an absolute decline of 10 percentage points or more in the percentage of the predicted FVC or who died; there was also a relative increase of 132.5% in the proportion of patients with no decline in FVC (P<0.001). Pirfenidone reduced the decline in the 6-minute walk distance (P=0.04) and improved progression-free survival (P<0.001). There was no significant between-group difference in dyspnea scores (P=0.16) or in rates of death from any cause (P=0.10) or from idiopathic pulmonary fibrosis (P=0.23). However, in a prespecified pooled analysis incorporating results from two previous phase 3 trials, the between-group difference favoring pirfenidone was significant for death from any cause (P=0.01) and from idiopathic pulmonary fibrosis (P=0.006). Gastrointestinal and skin-related adverse events were more common in the pirfenidone group than in the placebo group but rarely led to treatment discontinuation.


Pirfenidone, as compared with placebo, reduced disease progression, as reflected by lung function, exercise tolerance, and progression-free survival, in patients with idiopathic pulmonary fibrosis. Treatment was associated with an acceptable side-effect profile and fewer deaths. (Funded by InterMune; ASCEND number, NCT01366209.).

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