Pirfenidone: an update on clinical trial data and insights from everyday practice

Kreuter, Michael

doi:10.1183/09059180.00008513

Cited by 34 publications

(30 citation statements)

References 15 publications

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“…Pirfenidone is a novel antifibrotic agent that has recently been licensed by the European authorities for the treatment of mild-to-moderate IPF. It is the first substance with proven clinical efficacy in the treatment of IPF [6]. In vitro and in vivo studies have shown that pirfenidone exhibits anti-inflammatory and antifibrotic properties by inhibiting fibroblast proliferation, inflammatory cell accumulation, and profibrotic and proinflammatory cytokine production, and by reducing extracellular matrix deposition [7,8].…”

Section: Introductionmentioning

confidence: 99%

Pirfenidone in Idiopathic Pulmonary Fibrosis: Real-Life Experience from a German Tertiary Referral Center for Interstitial Lung Diseases

Oltmanns

Kahn²,

Palmowski³

et al. 2014

Respiration

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Background: Pirfenidone is a novel antifibrotic drug for the treatment of mild-to-moderate idiopathic pulmonary fibrosis (IPF). However, adverse events may offset treatment benefits and compliance. Objectives: To assess recent course of disease, adverse events and compliance in patients who started pirfenidone. Methods: In an observational cohort study, 63 patients with mild-to-moderate IPF who started pirfenidone between May 2011 and June 2013 were reviewed. Pulmonary function, adverse events and treatment compliance were recorded at each clinic visit. Disease progression was defined as a reduction of vital capacity ≥10% and/or diffusion capacity (DLCO) ≥15%. Results: Follow-up time on pirfenidone treatment was 11 (±7) months. Sixty-six percent of the patients continued with pirfenidone monotherapy and 34% of the patients received pirfenidone combined with corticosteroids (CCS) and/or N-acetylcysteine (NAC). There was a nonsignificant reduction in mean decline of percent predicted forced vital capacity after treatment start (0.7 ± 10.9%) compared to the pretreatment period (6.6 ± 6.7%, p = 0.098). Sixty-two percent of the patients had stable disease on pirfenidone treatment. Adverse events affected 85% of the patients, leading to discontinuation of pirfenidone in 20%. Adverse events and treatment discontinuation were seen more frequently in patients with concomitant CCS and/or NAC treatment. Conclusions: Adverse events affect the majority of patients treated with pirfenidone, but are mostly manageable with supportive measures. In this heterogeneous patient group, a nonsignificant effect of pirfenidone treatment on pulmonary function was seen, underlining the need for more data on patient selection criteria and efficacy of pirfenidone, particularly in patients with coexistent emphysema and concomitant NAC/CCS treatment.

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Section: Introductionmentioning

confidence: 99%

Pirfenidone in Idiopathic Pulmonary Fibrosis: Real-Life Experience from a German Tertiary Referral Center for Interstitial Lung Diseases

Oltmanns

Kahn²,

Palmowski³

et al. 2014

Respiration

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“…In contrast to many orphan drug approvals, the FDA's approval of pirfenidone (Esbriet) came three-and-a-half years after it had been approved by the EMA for the treatment of idiopathic pulmonary fibrosis [49]. InterMune had originally filed a NDA for pirfenidone in November 2009 but in May 2010 the FDA had requested an additional Phase III study be performed prior to approval.…”

Section: Other Orphan Approvalsmentioning

confidence: 99%

Orphan drug approvals of 2014: Europe and the United States

Norman¹

2015

Expert Opinion on Orphan Drugs

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Introduction: The development of orphan drugs for the treatment of rare diseases has attracted increasing interest from the pharmaceutical industry. The year 2014 saw an increased level of orphan drug approvals, possibly reflecting the increased attention given to rare diseases. Areas covered: The review discusses all the orphan drug approvals of 2014. It first considers the 12 European approvals, which includes eight novel chemical entities (NCEs). It then considers the 44 US approvals, which includes 17 NCEs. The discrepancies in approval timings between these markets are then discussed. Expert opinion: The increase in the number of orphan drug approvals appears attributable both to an increased awareness of rare diseases and to the FDA's efforts to expedite the approval of drugs to treat rare diseases and serious conditions. Europe's failure to follow suit appears to be one factor in the delays between US and European approvals of orphan drugs.

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“…It is almost a real-life study but a major bias is that patients included in this study come from clinical trials, with all the aforementioned limitations of this population. Data from an interim analysis performed in April 2012 have been recently published in the European Respiratory Review (ERR) [28]. 603 patients (mean age 68.3 years, 72% male, mean 2.6 years since IPF diagnosis) were enrolled in the RECAP study.…”

Section: International Collaborative Experiencesmentioning

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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Pirfenidone: an update on clinical trial data and insights from everyday practice

Cited by 34 publications

References 15 publications

Pirfenidone in Idiopathic Pulmonary Fibrosis: Real-Life Experience from a German Tertiary Referral Center for Interstitial Lung Diseases

Pirfenidone in Idiopathic Pulmonary Fibrosis: Real-Life Experience from a German Tertiary Referral Center for Interstitial Lung Diseases

Orphan drug approvals of 2014: Europe and the United States

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

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