A Phase 3 Trial of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis

King, Talmadge E.; Bradford, Williamson Z.; Castro-Bernardini, Socorro; Fagan, Elizabeth A.; Glaspole, Ian; Glassberg, Marilyn K.; Gorina, Eduard; Hopkins, Peter; Kardatzke, David; Lancaster, Lisa; Lederer, David J.; Nathan, Steven D.; Pereira, Carlos Alberto de Castro; Sahn, Steven A.; Sussman, Robert; Swigris, Jeffrey J.; Noble, Paul W.

doi:10.1056/nejmoa1402582

Cited by 3,149 publications

(2,946 citation statements)

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“…It should be emphasized that these drugs are not curative, but instead appear to slow down the decline of lung fibrosis in IPF. Pirfenidone, a small molecule capable of inhibiting TGF-b, showed success in slowing pulmonary dysfunction in clinical trials with patients with IPF (64,65).…”

Section: Clinical Management Of Pulmonary Fibrosismentioning

confidence: 99%

Pulmonary Fibrosis in Hermansky-Pudlak Syndrome

et al. 2016

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Hermansky-Pudlak syndrome (HPS) is a rare autosomal recessive genetic disorder characterized by oculocutaneous albinism and a bleeding diathesis due to platelet dysfunction. More than 50% of cases worldwide are diagnosed on the Caribbean island of Puerto Rico. Genetic testing plays a growing role in diagnosis; however, not all patients with HPS have identified genetic mutations. In Puerto Rico, patients with HPS are often identified shortly after birth by their albinism, although the degree of hypopigmentation is highly variable. Ten subtypes have been described. Patients with HPS-1, HPS-2, and HPS-4 tend to develop pulmonary fibrosis in Puerto Rico; 100% of patients with HPS-1 develop HPS-PF. HPS-PF and idiopathic pulmonary fibrosis are considered similar entities (albeit with distinct causes) because both can show similar histological disease patterns. However, in contrast to idiopathic pulmonary fibrosis, HPS-PF manifests much earlier, often at 30-40 years of age. The progression of HPS-PF is characterized by the development of dyspnea and increasingly debilitating hypoxemia. No therapeutic interventions are currently approved by the U.S. Food and Drug Administration for the treatment of HPS and HPS-PF. However, the approval of two new antifibrotic drugs, pirfenidone and nintedanib, has prompted new interest in identifying drugs capable of reversing or halting the progression of HPS-PF. Thus, lung transplantation remains the only potentially life-prolonging treatment. At present, two clinical trials are recruiting patients with HPS-PF to identify biomarkers for disease progression. Advances in the diagnosis and management of these patients will require the establishment of multidisciplinary centers of excellence staffed by experts in this disease.

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Section: Clinical Management Of Pulmonary Fibrosismentioning

confidence: 99%

Pulmonary Fibrosis in Hermansky-Pudlak Syndrome

et al. 2016

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“…Pirfenidone is currently approved and used for IPF/UIP not only in Japan, but also in the USA (13,14). Pirfenidone demonstrates pleiotropic pharmacological effects such as anti-fibrotic, anti-inflammatory, and anti-oxidative effects.…”

Section: Discussionmentioning

confidence: 99%

A Patient with Idiopathic Pleuroparenchymal Fibroelastosis Showing a Sustained Pulmonary Function due to Treatment with Pirfenidone

et al. 2016

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The patient was a 68-year-old man presenting with body weight loss and exertional dyspnea. Highresolution computed tomography of the chest showed dense subpleural consolidation with traction bronchiectasis and volume loss predominantly in bilateral apical lesions and upper lobes. A histopathological analysis of a specimen of the right upper lobe showed histological patterns which were consistent with idiopathic pleuroparenchymal fibroelastotis (IPPFE). Treatment with pirfenidone was introduced with the expectation of its potential benefit. The effect of pirfenidone was satisfactory, and a decline in forced vital capacity was inhibited during treatment. This is the first case report suggesting the efficacy of pirfenidone for patients with IPPFE.

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“…Progress in these research efforts was highlighted by the 2014 Federal Drug Administration approval of pirfenidone and nintedanib for idiopathic pulmonary fibrosis (IPF), both of which significantly slow disease progression. 1,2 However, novel therapies to promote the resolution of fibrosis have yet to be identified. Because most patients have a significant fibrotic burden at the time of clinical presentation, understanding the basic mechanisms mediating the lungs' capacity to resolve fibrosis is critically important, and may lead to novel therapeutic strategies to actually reverse established disease.…”

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confidence: 99%