Intraindividual Response to Treatment with Pirfenidone in Idiopathic Pulmonary Fibrosis

Loeh, Benjamin; Drakopanagiotakis, Fotios; Bandelli, Gian Piero; Beck, Daniel von der; Tello, Silke; Cordani, Elisa; Rizza, E; Barrocu, Laura; Markart, Philipp; Seeger, Werner; Güenther, Andreas; Albera, Carlo

doi:10.1164/rccm.201406-1106le

Cited by 53 publications

(44 citation statements)

References 14 publications

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“…In clinical trials, pirfenidone has slowed disease progression and decreased mortality compared with placebo, and observations of attenuated decline in lung function (forced vital capacity, FVC) from real-world data are consistent with findings from clinical trials [2,3,4,5,6,7,8]. Pirfenidone slowed disease progression as measured by changes in FVC [9,10] and reduced the risk of death from any cause by 48% at 1 year [3,11].…”

Section: Introductionsupporting

confidence: 63%

An Open-Label Study of the Long-Term Safety of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis (RECAP)

Costabel

Albera²,

Lancaster³

et al. 2017

Respiration

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131

101

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Background: RECAP (NCT00662038) was an open-label extension study in patients with idiopathic pulmonary fibrosis (IPF) who completed either the Assessment of Pirfenidone to Confirm Efficacy and Safety in Idiopathic Pulmonary Fibrosis (ASCEND) 016 phase 3 trial or the Clinical Studies Assessing Pirfenidone in Idiopathic Pulmonary Fibrosis: Research of Efficacy and Safety Outcomes (CAPACITY) 004/006 phase 3 trials. Objective: To obtain long-term safety data for pirfenidone in patients with IPF in RECAP. Methods: Of the 1,334 patients who participated in the phase 3 trials, 1,058 entered RECAP. The final analysis from enrollment (September 2008) to June 2015 is presented. Results: Mean (SD) and median (range) pirfenidone exposures in RECAP were 122 (98) weeks and 88 (>0 to 349) weeks, respectively, with a mean daily dose of 2,091.1 mg. Cumulative total exposure was 2,482 patient exposure years (PEY). The treatment-emergent adverse event (TEAE) rate was 701.9 per 100 PEY. The serious TEAE rate was 53.5 per 100 PEY, with the most common serious TEAE being IPF (11.1 per 100 PEY). Of the 231 deaths (9.3 per 100 PEY), the most common cause was IPF (5.4 per 100 PEY). The treatment discontinuation rate due to a TEAE was 17.9 per 100 PEY; discontinuations were due to IPF (7.2 per 100 PEY), pneumonia, respiratory failure, acute respiratory failure, rash (0.5 per 100 PEY each), and nausea (0.4 per 100 PEY). For patients from CAPACITY 004/006 who entered RECAP, the mean change in percent predicted forced vital capacity from RECAP baseline at 180 weeks was -9.6%. Median on-treatment survival from the first pirfenidone dose in RECAP was 77.2 months. Conclusions: RECAP provides long-term follow-up and safety data for pirfenidone that were consistent with the known profile, with no new safety signals observed.

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Section: Introductionsupporting

confidence: 63%

An Open-Label Study of the Long-Term Safety of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis (RECAP)

Costabel

Albera²,

Lancaster³

et al. 2017

Respiration

Self Cite

131

101

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“…With regard to attenuation of decline in FVC, the results of the analysis according to the GAP index staging system confirmed that, compared with patients with less advanced disease (GAP stage I), those with more advanced disease (GAP stages II and III) could benefit the most from the administration of pirfenidone ( p-value for homogeneity of difference between strata of 0.041). Ultimately, in this trial, as already reported by LOEH et al [23], 16 (12.5%) out of 128 patients improved their FVC by >10% during therapy with pirfenidone [24]. Only 22 (17.1%) patients out of 128 were treated with lower than the standard dose of the drug (2403 mg·day −1 ); however, such dose reduction did not influence the outcomes.…”

supporting

confidence: 71%

“…The more recent study by LOEH et al [23], which was also conducted in a real-life setting, considered in a retrospective analysis 197 IPF patients followed in an Italian centre and a German centre. It confirmed that patients with a clear progression of disease before pirfenidone therapy showed an even more favourable course under pirfenidone treatment, in agreement with the data previously published by OKUDA et al [19].…”

mentioning

confidence: 99%

Idiopathic pulmonary fibrosis: from clinical trials to real-life experiences

Harari

2015

Eur Respir Rev

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Randomised controlled clinical trials are fundamental in medicine to develop new effective drugs and new therapeutic regimens and are the strength of evidence-based medicine. These studies allow us to avoid the repetition of misleading experiences that have been reported in the past, where drugs or associations were utilised without compelling evidence and ultimately proven to be ineffective. In recent years, randomised clinical trials have been conducted and concluded for many rare diseases, including idiopathic pulmonary fibrosis. However, clinical trials do not always reflect the real-life scenario. Patients selected for clinical trials present fewer comorbidities, they fall between certain age limits, and the severity of their disease is defined; therefore, they do not always reflect the whole of the population affected by a specific disease. These are the reasons why we also need data that mirror real-life experience. The limitations that these kind of studies present are always several and the studies should be interpreted with caution, although they can fill the important gap between efficacy and effectiveness. In this article, we will review the existing clinical data on real-life treatment of idiopathic pulmonary fibrosis. @ERSpublications Real-life studies are useful in confirming the experimental data obtained from randomised controlled clinical trials http://ow.ly/PYUCC

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“…The adverse effect profiles show some overlap regarding gastrointestinal side-effects and elevation of liver enzymes. Furthermore, individual response to pirfenidone treatment is variable, with progression of fibrosis despite pirfenidone treatment in some patients, while most patients remain or become stable [6].…”

mentioning

confidence: 99%

“…Rapid progression as an adverse event was defined as an absolute decline in FVC of >10% within 6 months and, in this case, treatment was stopped due to clinical ineffectiveness. Beyond this, in the treatment-response analysis, clinical stability was defined as a relative semi-annual decline of FVC <5% as previously reported [6,9].…”

mentioning

confidence: 99%

Switching to nintedanib after discontinuation of pirfenidone due to adverse events in IPF

et al. 2015

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Intraindividual Response to Treatment with Pirfenidone in Idiopathic Pulmonary Fibrosis

Cited by 53 publications

References 14 publications

An Open-Label Study of the Long-Term Safety of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis (RECAP)

An Open-Label Study of the Long-Term Safety of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis (RECAP)

Idiopathic pulmonary fibrosis: from clinical trials to real-life experiences

Switching to nintedanib after discontinuation of pirfenidone due to adverse events in IPF

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