Pirfenidone Therapy for Familial Pulmonary Fibrosis: A Real-Life Study
Familial pulmonary fibrosis (FPF) is defined as an idiopathic diffuse parenchymal lung disease affecting two or more members of the same primary biological family. The aim of this study was to compare disease progression and tolerance to pirfenidone in a population of FPF patients who presented with radiological and/or histological evidence of UIP, and a group of idiopathic pulmonary fibrosis (IPF) patients.
Seventy-three patients (19 with FPF and 54 with IPF) were enrolled and data were collected retrospectively at 6, 12 and 24 months follow-up.
FPF patients were statistically younger and more frequently females. A significantly greater decline in FVC and DLCO was recorded in FPF than in IPF patients at 24 months follow-up. At the 6-min walking test, walked distance declined significantly in FPF patients than IPF at 24 months. No statistically significant differences in drug tolerance or side effects were recorded between groups.
Different rate of progression was observed in patients with IPF and FPF on therapy with pirfenidone; our findings may not be due to lack of effectiveness of therapy, but to the different natural history and evolution of these two conditions. Pirfenidone was well tolerated by FPF and IPF patients. Specific unbiased randomized clinical trials on larger populations to validate our preliminary exploratory results are needed.
KeywordsPirfenidone Familial pulmonary fibrosis Idiopathic pulmonary fibrosis Therapy
Compliance with Ethical Standards
Conflict of interest
Dr. Bennett reports personal fees from Boehringer Ingelheim, personal fees from Roche, personal fees from Biotest, outside the submitted work. Prof. Rottoli reports personal fees and other from Roche, personal fees from Boehringer ingelheim, grants and personal fees from Novartis, personal fees from TEVA, other from Menarini, outside the submitted work. Dr. Rosa Metella Refini, Dr. Maria Lucia Valentini, Dr. Annalisa Fui, Dr. Antonella Fossi, Dr. Maria Pieroni and Prof. Maria Antonietta Mazzei and have nothing to disclose.
- 1.Travis WD, Costabel U, Hansell DM, King TE Jr, Lynch DA, Nicholson AG, Ryerson CJ, Ryu JH, Selman M, Wells AU, Behr J, Bouros D, Brown KK, Colby TV, Collard HR, Cordeiro CR, Cottin V, Crestani B, Drent M, Dudden RF, Egan J, Flaherty K, Hogaboam C, Inoue Y, Johkoh T, Kim DS, Kitaichi M, Loyd J, Martinez FJ, Myers J, Protzko S, Raghu G, Richeldi L, Sverzellati N, Swigris J, Valeyre D, ATS/ERS Committee on Idiopathic Interstitial Pneumonias (2013) An official American Thoracic Society/European Respiratory Society statement: update of the international multidisciplinary classification of the idiopathic interstitial pneumonias. Am J Respir Crit Care Med 188(6):733–748CrossRefPubMedCentralGoogle Scholar
- 3.Ravaglia C, Tomassetti S, Gurioli C, Piciucchi S, Dubini A, Gurioli C, Casoni GL, Romagnoli M, Carloni A, Tantalocco P, Buccioli M, Chilosi M, Poletti V (2014) Features and outcome of familial idiopathic pulmonary fibrosis. Sarcoidosis Vasc Diffuse Lung Dis 31(1):28–36Google Scholar
- 9.King TE Jr, Bradford WZ, Castro-Bernardini S, Fagan EA, Glaspole I, Glassberg MK, Gorina E, Hopkins PM, Kardatzke D, Lancaster L, Lederer DJ, Nathan SD, Pereira CA, Sahn SA, Sussman R, Swigris JJ, Noble PW, ASCEND Study Group (2014) A phase 3 trial of pirfenidone in patients with idiopathic pulmonary fibrosis. N Engl J Med 370:2083–2092CrossRefGoogle Scholar
- 10.Noble PW, Albera C, Bradford WZ, Costabel U, Glassberg MK, Kardatzke D, King TE Jr, Lancaster L, Sahn SA, Szwarcberg J, Valeyre D, du Bois RM, CAPACITY Study Group (2011) Pirfenidone in patients with idiopathic pulmonary fibrosis (CAPACITY): two randomized trials. Lancet 377:1760–1769CrossRefGoogle Scholar
- 11.Richeldi L, du Bois RM, Raghu G, Azuma A, Brown KK, Costabel U, Cottin V, Flaherty KR, Hansell DM, Inoue Y, Kim DS, Kolb M, Nicholson AG, Noble PW, Selman M, Taniguchi H, Brun M, Le Maulf F, Girard M, Stowasser S, Schlenker-Herceg R, Disse B, Collard HR, INPULSIS Trial Investigators (2014) Efficacy and safety of nintedanib in idiopathic pulmonary fibrosis. N Engl J Med 370(22):2071–2082CrossRefGoogle Scholar
- 12.Raghu G, Remy-Jardin M, Myers JL, Richeldi L, Ryerson CJ, Lederer DJ, Behr J, Cottin V, Danoff SK, Morell F, Flaherty KR, Wells A, Martinez FJ, Azuma A, Bice TJ, Bouros D, Brown KK, Collard HR, Duggal A, Galvin L, Inoue Y, Jenkins RG, Johkoh T, Kazerooni EA, Kitaichi M, Knight SL, Mansour G, Nicholson AG, Pipavath SNJ, Buendía-Roldán I, Selman M, Travis WD, Walsh S, Wilson KC, American Thoracic Society, European Respiratory Society, Japanese Respiratory Society, and Latin American Thoracic Society (2018) Diagnosis of idiopathic pulmonary fibrosis. An official ATS/ERS/JRS/ALAT clinical practice guideline. Am J Respir Crit Care Med 198(5):e44–e68CrossRefGoogle Scholar
- 13.Meyer KC, Raghu G, Baughman RP, Brown KK, Costabel U, du Bois RM, Drent M, Haslam PL, Kim DS, Nagai S, Rottoli P, Saltini C, Selman M, Strange C, Wood B, American Thoracic Society Committee on BAL in Interstitial Lung Disease (2012) An official American Thoracic Society clinical practice guideline: the clinical utility of bronchoalveolar lavage cellular analysis in interstitial lung disease. Am J Respir Crit Care Med 185(9):1004–1014. https://doi.org/10.1164/rccm.201202-0320ST CrossRefGoogle Scholar
- 14.Harari S, Caminati A, Albera C, Vancheri C, Poletti V, Pesci A, Luppi F, Saltini C, Agostini C, Bargagli E, Sebastiani A, Sanduzzi A, Giunta V, Della Porta R, Bandelli GP, Puglisi S, Tomassetti S, Biffi A, Cerri S, Mari A, Cinetto F, Tirelli F, Farinelli G, Bocchino M, Specchia C, Confalonieri M (2015) Efficacy of pirfenidone for idiopathic pulmonary fibrosis: an Italian real life study. Respir Med 109(7):904–913CrossRefGoogle Scholar