Five Years of Real World Pirfenidone Experience Gareth Hughes1, 2, Colm Leonard1, 2, Nazia Chaudhuri1, 2 1 North West Interstitial Lung Disease Unit, University Hospital of South Manchester, Wythenshawe Hospital, Southmoor Road, Wythenshawe, Manchester M29 9LT, UK 2 Manchester Academic Health Science Centre, The University of Manchester, Oxford Road, Manchester M13 9PL, UK INTRODUCTION RESULTS Pirfenidone is an effective treatment in Idiopathic Pulmonary Fibrosis which has both anti-inflammatory and anti-fibrotic effects. This agent was first approved for use by the National Institute for Health and Care Excellence (NICE) in 2013. We set out our five year real world experience1 with this agent which is compelling given as our population is older, has more severe disease and significantly more comorbidities compared to those recruited to the clinical trials. 1. Cohort Demographics Cohort of 351 patients Average age of 70 years (range 47–91) 81% male Pre-treatment average FVC of 69% and DLCO 42%. 16% (n = 56) with emphysema on CT. 26% (n = 90) on either ambulatory or long-term oxygen therapy. 3. Frequency of Adverse Events 5. Reduction in decline of mean FVC before and after pirfenidone treatment. 2. Real-world adverse events compared with clinical trials. 4. Impact of Adverse Event on Treatment Adverse Event (AE) Percentage of Total Events Percentage of Patients experiencing AE from CAPACITY2 Percentage of Patients experiencing AE from ASCEND3 Appetite Loss 17% 11% 15.8% Nausea & Vomiting 15% 36% Nausea 14% Vomiting 12.9% Vomiting Lethargy 12% 7% 20.9% Gastro-Oesophageal Reflux (GOR) 19% dyspepsia 10% abdominal distenson 11.9% GOR 14.7% dyspepsia Weight Loss 5% 8% 12.6% Diarrhoea 3% - 22.3% Constipation 1% 11.5% Rash 32% 28.1% Dizziness 2% 18% 17.6% Insomnia 10% 11.2% Headache 25.9% Death 6% 4% METHODS SUMMARY Single centre retrospective observational study Data analysis of information captured at patients routine clinic visits including pulmonary function tests, routine bloods and adverse event data. Subsequent analysis using GraphPad Prism v5 with statistical testing with a Oneway ANOVA and Dunnett’s post test or unpaired t –test where indicated. Our retrospective observational data demonstrates favourable preservation of lung function, and similar safety profile and discontinuation rates to previous published data. Patients who stopped treatment due to AEs persevered with treatment for a mean of 204 days, had more severe disease (p<0.05) as depicted by baseline DLCO, but no difference in age, body mass index (BMI) or baseline FVC. REFERENCES 1. Hughes G, Toellner H, Morris H, Leonard C, Chaudhuri N. Real World Experiences: Pirfenidone and Nintedanib are Effective and Well Tolerated Treatments for Idiopathic Pulmonary Fibrosis. Journal of Clinical Medicine 2016; 5(9): E78. DOI:10.3390/jcm5090078 2. Noble PW, Albera C, Bradford WZ et al. Pirfenidone in patients with idiopathic pulmonary fibrosis (CAPACITY): two randomised trials. Lancet 2011; 377(9779):1760-1769. DOI: 10.1016/S0140-6736(11)60405-4 3. King TE, Bradford WZ, Castro-Bernardini S. A Phase 3 Trial of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis. New England Journal of Medicine 2014; 370:2083-2092