scholarly journals Nintedanib in the management of idiopathic pulmonary fibrosis: clinical trial evidence and real-world experience

2018 ◽  
Vol 12 ◽  
pp. 175346661880061 ◽  
Author(s):  
Pilar Rivera-Ortega ◽  
Conal Hayton ◽  
John Blaikley ◽  
Colm Leonard ◽  
Nazia Chaudhuri
Keyword(s):  
Clinical Trial ◽  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Lung Transplantation ◽  
Real World ◽  
Kinase Inhibitor ◽  
Scientific Evidence ◽  
Postoperative Mortality ◽  
Clinical Trial Evidence ◽  
Trial Evidence

Idiopathic pulmonary fibrosis (IPF) is a fibrotic interstitial lung disease associated with significant morbidity and mortality. Previously, IPF has been managed using immunosuppressive therapy; however, it has been shown that this is associated with increased mortality. In the last 5 years, two disease-modifying agents have been licensed for use in IPF, namely pirfenidone and nintedanib. Nintedanib is a tyrosine kinase inhibitor with antifibrotic properties that has also been shown to significantly reduce the progression of the disease. The scientific evidence shows that nintedanib is effective and well tolerated for the treatment of IPF in mild, moderate and severe stages of the disease. Real-world experiences also support the findings of previously conducted clinical trials and show that nintedanib is effective for the management of IPF and is associated with reducing disease progression. Gastrointestinal events, mainly diarrhoea, are the main adverse events caused by the treatment. Recent real-word studies also suggest that nintedanib stabilizes lung function till lung transplantation, with no increased surgical complications or postoperative mortality after lung transplantation. In this review, we will discuss the clinical trial evidence and real-world experience for nintedanib in the management of IPF.

scholarly journals Clinical Trial Eligibility Criteria and Recently Approved Cancer Therapies for Patients With Brain Metastases

2022 ◽  
Vol 11 ◽  
Author(s):  
Aaron C. Tan ◽  
Drexell H. Boggs ◽  
Eudocia Q. Lee ◽  
Michelle M. Kim ◽  
Minesh P. Mehta ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Brain Metastases ◽  
Real World ◽  
Treatment Options ◽  
Late Phase ◽  
Leptomeningeal Carcinomatosis ◽  
Precision Oncology ◽  
Eligibility Criteria ◽  
Clinical Trial Evidence ◽  
Trial Evidence

Brain metastases cause significant morbidity and mortality in patients with advanced cancer. In the era of precision oncology and immunotherapy, there are rapidly evolving systemic treatment options. These novel therapies may have variable intracranial efficacy, and patients with brain metastases remain a population of special interest. Typically, only patients with stable, asymptomatic and/or treated brain metastases are enrolled in clinical trials, or may be excluded altogether, particularly in the setting of leptomeningeal carcinomatosis. Consequently, this leads to significant concerns on the external validity of clinical trial evidence to real-world clinical practice. Here we describe the current trends in cancer clinical trial eligibility for patients with brain metastases in both early and late phase trials, with a focus on targeted and immunotherapies. We evaluate recent newly FDA approved therapies and the clinical trial evidence base leading to approval. This includes analysis of inclusion and exclusion criteria, requirements for baseline screening for brain metastases, surveillance cerebral imaging and incorporation of trial endpoints for patients with brain metastases. Finally, the use of alternative sources of data such as real-world evidence with registries and collaborative studies will be discussed.

Clinical Trial Evidence for the Real World

2019 ◽  
Vol 179 (10) ◽  
pp. 1333
Author(s):  
Joseph S. Ross ◽  
Ken Covinsky
Keyword(s):  
Clinical Trial ◽  
Real World ◽  
The Real ◽  
Clinical Trial Evidence ◽  
Trial Evidence

Number needed to treat for net effect of anticoagulation in atrial fibrillation: Real‐World vs . Clinical Trial Evidence

2021 ◽  
Author(s):  
Wern Yew Ding ◽  
José Miguel Rivera‐Caravaca ◽  
Francisco Marín ◽  
Guowei Li ◽  
Vanessa Roldán ◽  
...  
Keyword(s):  
Atrial Fibrillation ◽  
Clinical Trial ◽  
Real World ◽  
Number Needed To Treat ◽  
Clinical Trial Evidence ◽  
Trial Evidence

scholarly journals Feasibility of Using Real-World Data to Replicate Clinical Trial Evidence

2019 ◽  
Vol 2 (10) ◽  
pp. e1912869 ◽  
Author(s):  
Victoria L. Bartlett ◽  
Sanket S. Dhruva ◽  
Nilay D. Shah ◽  
Patrick Ryan ◽  
Joseph S. Ross
Keyword(s):  
Clinical Trial ◽  
Real World ◽  
Real World Data ◽  
World Data ◽  
Clinical Trial Evidence ◽  
Trial Evidence

scholarly journals Efficacy and safety of nintedanib in a Greek multicentre idiopathic pulmonary fibrosis registry: a retrospective, observational, cohort study

2020 ◽  
Vol 6 (1) ◽  
pp. 00172-2019 ◽  
Author(s):  
Katerina Antoniou ◽  
Katerina Markopoulou ◽  
Argyrios Tzouvelekis ◽  
Athina Trachalaki ◽  
Eirini Vasarmidi ◽  
...  
Keyword(s):  
Adverse Event ◽  
Survival Rate ◽  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Real World ◽  
Kinase Inhibitor ◽  
Significant Difference ◽  
Observational Cohort ◽  
Greek Hospitals ◽  
Retrospective Observational Cohort Study

Nintedanib is a tyrosine kinase inhibitor approved for the treatment of idiopathic pulmonary fibrosis (IPF). In a retrospective, real-world study across seven Greek hospitals, we evaluated the effectiveness and safety of nintedanib in routine clinical practice. Patients diagnosed with IPF, as per guideline criteria or multidisciplinary diagnosis, received nintedanib between January 2013 and January 2018.We evaluated 244 patients: mean±sd age 71.8±7.5 years, 79.1% male, 45.1% current smokers and 33.1% ex-smokers at treatment initiation. At baseline, predicted forced vital capacity (FVC) was 73.3±20.7% and predicted diffusing capacity of the lungs for carbon monoxide (DLCO) was 42.6±16.7%. On average, patients spent 23.6±15.0 months on nintedanib. At 3 years, 78 patients had died, equating to a 3-year survival rate of 59.4% (unaffected by treatment discontinuation or dose reduction). FVC% pred and DLCO% pred were largely stable at 3 years, with no significant difference from baseline (FVC 73.3±20.7% pred versus 78±20.1% pred, p=0.074; DLCO 42.6±16.7% pred versus 40.4±18.1% pred, p=0.334). Of the 244 patients, 55.7% reported an adverse event. Gastrointestinal events were the most common (173 (77.2%) out of 224 total events) and 45.0% of patients experienced diarrhoea. Only 32 (13.1%) patients had to permanently discontinue nintedanib due to an adverse event.This real-world study shows a 3-year survival rate of 59.4% and a low discontinuation rate due to adverse events. Our experience is consistent with previous findings in clinical trials of nintedanib in IPF.

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