scholarly journals Effects of low-dose pirfenidone on survival and lung function decline in patients with idiopathic pulmonary fibrosis (IPF): Results from a real-world study

PLoS ONE ◽  
2021 ◽  
Vol 16 (12) ◽  
pp. e0261684
Author(s):  
Eung Gu Lee ◽  
Tae-Hee Lee ◽  
Yujin Hong ◽  
Jiwon Ryoo ◽  
Jung Won Heo ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Pulmonary Function ◽  
Real World ◽  
Low Dose ◽  
Randomized Clinical Trials ◽  
Laboratory Data ◽  
Unknown Etiology ◽  
The Real

Background Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive fibrosing interstitial pneumonia of unknown etiology. In several randomized clinical trials, and in the clinical practice, pirfenidone is used to effectively and safely treat IPF. However, sometimes it is difficult to use the dose of pirfenidone used in clinical trials. This study evaluated the effects of low-dose pirfenidone on IPF disease progression and patient survival in the real-world. Methods This retrospective, observational study enrolled IPF patients seen at the time of diagnosis at a single center from 2008 to 2018. Longitudinal clinical and laboratory data were prospectively collected. We compared the clinical characteristics, survival, and pulmonary function decline between patients treated and untreated with various dose of pirfenidone. Results Of 295 IPF patients, 100 (33.9%) received pirfenidone and 195 (66.1%) received no antifibrotic agent. Of the 100 patients who received pirfenidone, 24 (24%), 50 (50%), and 26 (26%), respectively, were given 600, 1200, and 1800 mg pirfenidone daily. The mean survival time was 57.03 ± 3.90 months in the no-antifibrotic drug group and 73.26 ± 7.87 months in the pirfenidone-treated group (p = 0.027). In the unadjusted analysis, the survival of the patients given pirfenidone was significantly better (hazard ratio [HR] = 0.69, 95% confidence interval [CI]: 0.48–0.99, p = 0.04). After adjusting for age, gender, body mass index, and the GAP score [based on gender (G), age (A), and two physiological lung parameters (P)], survival remained better in the patients given pirfenidone (HR = 0.56, 95% CI: 0.37–0.85, p = 0.006). In terms of pulmonary function, the decreases in forced vital capacity (%), forced expiratory volume in 1 s (%) and the diffusing capacity of lung for carbon monoxide (%) were significantly smaller (p = 0.000, p = 0.001, and p = 0.007, respectively) in patients given pirfenidone. Conclusions Low-dose pirfenidone provided beneficial effects on survival and pulmonary function decline in the real-world practice.

How Do Patients With Idiopathic Pulmonary Fibrosis in the Real World Compare With Eligibility Criteria for Clinical Trials?

CHEST Journal ◽  
2017 ◽  
Vol 152 (4) ◽  
pp. A451
Author(s):  
Victoria Gamerman ◽  
Margaret Salisbury ◽  
Daniel Culver ◽  
Thomas Leonard ◽  
Megan Neely ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Real World ◽  
Eligibility Criteria ◽  
The Real

scholarly journals A retrospective study of the tolerability of nintedanib for severe idiopathic pulmonary fibrosis in the real world

2019 ◽  
Vol 7 (12) ◽  
pp. 262-262 ◽  
Author(s):  
Masayuki Nakamura ◽  
Masaki Okamoto ◽  
Kiminori Fujimoto ◽  
Tomohiro Ebata ◽  
Masaki Tominaga ◽  
...  
Keyword(s):  
Retrospective Study ◽  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Real World ◽  
The Real

scholarly journals Longitudinal clinical outcomes in a real-world population of patients with idiopathic pulmonary fibrosis: the PROOF registry

2019 ◽  
Vol 20 (1) ◽  
Author(s):  
Wim A. Wuyts ◽  
Caroline Dahlqvist ◽  
Hans Slabbynck ◽  
Marc Schlesser ◽  
Natacha Gusbin ◽  
...  
Keyword(s):  
Pulmonary Hypertension ◽  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Pulmonary Function ◽  
Clinical Outcomes ◽  
Real World ◽  
Lung Transplant ◽  
World Population ◽  
Duration Of Treatment

Abstract Background The PROOF registry is an observational study initiated in October 2013 with the aim to monitor disease progression in a real-world population of patients with idiopathic pulmonary fibrosis (IPF). Here, we present longitudinal clinical outcomes from the PROOF registry. Methods Patients with IPF were enrolled across eight centers in Belgium and Luxembourg. For all patients, clinical outcomes data were collected, including mortality, lung transplant, acute exacerbations, and pulmonary hypertension. For patients treated with pirfenidone at any time during follow-up (2013–2017), for any duration of treatment (the pirfenidone-treated population): pirfenidone treatment patterns were collected; changes in pulmonary function (forced vital capacity [FVC] and carbon monoxide diffusing capacity [DLco]) were reviewed up to 24 months post-inclusion; and time-to-event analyses from the time of registry inclusion were performed. Results The PROOF registry enrolled a total of 277 patients. During follow-up, 23.1% of patients died, 5.1% received a lung transplant, 5.4% experienced an acute exacerbation, and 6.1% had comorbid pulmonary hypertension. In the pirfenidone-treated population (N = 233, 84.1%), 12.9% of patients had a temporary dose discontinuation and 31.8% had a temporary dose reduction; 4.3% of patients permanently discontinued pirfenidone due to an adverse drug reaction. Mean percent predicted FVC was 81.2% (standard deviation [SD] 19.0) at Month 0 and 78.3% (SD 25.0) at Month 24, and mean percent predicted DLco was 47.0% (SD 13.2) and 45.0% (SD 16.5), respectively. Rates of ≥ 10% absolute decline in percent predicted FVC and ≥ 15% absolute decline in percent predicted DLco over 24 months were 31.0% and 23.2%, respectively. Mean times from registry inclusion to categorical absolute decline in percent predicted FVC and percent predicted DLco were 20.1 (standard error [SE] 0.6) months and 23.4 (SE 0.5) months, respectively; mean time from registry inclusion to death was 31.0 (SE 0.9) months. Conclusions The PROOF registry is a source of European data characterizing longitudinal clinical outcomes of patients with IPF. Over 12 months of follow-up, pulmonary function remained largely stable in patients with IPF who received pirfenidone for any duration of treatment. Pulmonary function remained similar at 24 months of follow-up, although patient numbers were lower. Trial registration PROOF is registered with the relevant authorities in Belgium and Luxembourg, with registration to Comité National d’Éthique et de Recherche (CNER) N201309/03–12 September 2013 and a notification to Comité National de Protection des Données (CNDP) for Luxembourg.

Survival among metastatic colorectal patients in clinical trials compared to the real world.

2015 ◽  
Vol 33 (3_suppl) ◽  
pp. 673-673
Author(s):  
Ziwei Wang ◽  
Lindsay Hwang ◽  
James Don Murphy
Keyword(s):  
Colorectal Cancer ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Metastatic Colorectal Cancer ◽  
Median Survival ◽  
Real World ◽  
Randomized Clinical Trials ◽  
Phase Iii ◽  
The Real ◽  
Clinical Trial Results

673 Background: Randomized clinical trials play a central role in clinical research though only a small fraction of patients partake in clinical studies. Questions thus arise regarding the generalizability of clinical trial results to the remainder of the population. This study evaluated whether patient survival from randomized clinical trials in metastatic colorectal cancer reflects real world outcomes. Methods: A Pubmed search was used to identify randomized phase III clinical trials of first-line treatment for metastatic colorectal cancer published between 2005 and 2010. We excluded secondary or pooled analyses, second-line treatments, non-metastatic patients, non-English language, and non-randomized studies. Thirty-one clinical trials met these criteria, comprised of 79 distinct clinical trial arms. Overall survival among clinical trial patients was compared to metastatic colorectal cancer patients within the Surveillance, Epidemiology, and End Results (SEER) program. Within SEER, we restricted the analysis time-period and age of patients to match the enrollment period and age of patients within each individual clinical trial. Results: The clinical trials enrolled a total of 16,614 patients. Among all clinical trial arms the median survival ranged from 6.7-62 months, 1-year survival ranged from 30-97%, and 2-year survival ranged from 6-88%. Compared to SEER, the median survival was higher in 95% of the individual clinical trial arms by an average of 5.4 months (p<0.0001). The 1-year survival was higher in 94% of the clinical trial arms by an average of 16.7% (p<0.0001). The 2-year survival was higher in 71% of the clinical trial arms by an average of 7.2% (p<0.0001). Conclusions: This study found substantially improved survival among clinical trial participants compared to patients in the SEER database suggesting that survival estimates from clinical trials may not generalize to the “real world.” Potential patient factors such as differences in underlying comorbidity, performance status, disease burden, as well as variation in treatment could not be addressed in this study, though these factors likely explain some of the observed survival differences.

scholarly journals The real-world outcomes of multiple myeloma patients treated with daratumumab

PLoS ONE ◽  
2021 ◽  
Vol 16 (10) ◽  
pp. e0258487
Author(s):  
Agoston Gyula Szabo ◽  
Tobias Wirenfeldt Klausen ◽  
Mette Bøegh Levring ◽  
Birgitte Preiss ◽  
Carsten Helleberg ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Clinical Trials ◽  
Real World ◽  
Randomized Clinical Trials ◽  
Proteasome Inhibitors ◽  
Median Number ◽  
The Real ◽  
Treatment Timing ◽  
Risk Patients ◽  
Standard Risk

Most patients cannot be included in randomized clinical trials. We report real-world outcomes of all Danish patients with multiple myeloma (MM) treated with daratumumab-based regimens until 1 January 2019. Methods Information of 635 patients treated with daratumumab was collected retrospectively and included lines of therapy (LOT), hematologic responses according to the International Myeloma Working Group recommendations, time to next treatment (TNT) and the cause of discontinuation of treatment. Baseline characteristics were acquired from the validated Danish Multiple Myeloma Registry (DMMR). Results Daratumumab was administrated as monotherapy (Da-mono) in 27.7%, in combination with immunomodulatory drugs (Da-IMiD) in 57.3%, in combination with proteasome inhibitors (Da-PI) in 11.2% and in other combinations (Da-other) in 3.8% of patients. The median number of lines of therapy given before daratumumab was 5 for Da-mono, 3 for Da-IMiD, 4 for Da-PI, and 2 for Da-other. In Da-mono, overall response rate (ORR) was 44.9% and median time to next treatment (mTNT) was 4.9 months. In Da-IMiD, ORR was 80.5%, and mTNT was 16.1 months. In Da-PI, OOR was 60.6% and mTNT was 5.3 months. In patients treated with Da-other, OOR was 54,2% and mTNT was 5.6 months. The use of daratumumab in early LOT was associated with longer TNT (p<0.0001). Patients with amplification 1q had outcome comparable to standard risk patients, while patients with t(4;14), t(14;16) or del17p had worse outcome (p = 0.0001). Multivariate analysis indicated that timing of treatment (timing of daratumumab in the sequence of all LOT that the patients received throughout the course of their disease) was the most important factor for outcome (p<0.0001). Conclusion The real-world outcomes of multiple myeloma patients treated with daratumumab are worse than the results of clinical trials. Outcomes achieved with daratumumab were best when daratumumab was used in combination with IMIDs and in early LOT. Patients with high-risk CA had worse outcomes, but patients with amp1q had similar outcomes to standard-risk patients.

scholarly journals Chinese Herbal Medicines Compared with N-Acetylcysteine for the Treatment of Idiopathic Pulmonary Fibrosis: A Systematic Review of Randomized Controlled Trials

2019 ◽  
Vol 2019 ◽  
pp. 1-18
Author(s):  
Jing Guo ◽  
Bin Li ◽  
Wenbin Wu ◽  
Zhichao Wang ◽  
Fei Wang ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Idiopathic Pulmonary Fibrosis ◽  
Pulmonary Fibrosis ◽  
Methodological Quality ◽  
Randomized Clinical Trials ◽  
Conventional Medicine ◽  
Herbal Medicines ◽  
Chinese Herbal

Background. Idiopathic pulmonary fibrosis (IPF) is a major global health problem. The prevalence of the disease appears to be increasing. There is no curative therapy for IPF except lung transplantation. Chinese herbal medicines (CHMs) are showing promise for treatment of IPF. However, their effectiveness and safety are still unclear and deserve further investigation. The aim of this systematic review is to access the efficacy and safety of CHMs in treating IPF. Methods. The protocol of this review is registered at PROSPERO. We searched seven main databases for randomized clinical trials (RCTs) on CHMs for IPF from their inception to June 4, 2018. The methodological quality of RCTs was assessed using the Cochrane risk of bias tool. All trials included were analyzed according to the criteria of the Cochrane Handbook. Review Manager 5.3, R-3.5.2 software, and Grade pro GDT web solution were used for data synthesis and analysis. Results. Thirteen randomized clinical trials enrolling 733 patients were included. All trials included had clear outcome indicators. The methodological quality of included trials was generally “poor.” Few trials reported methods of randomization. One trial on Xuefu-zhuyu capsule assessed rate of acute exacerbation and mortality after treatment for 72 weeks and found no statistically significant difference between two groups. This meta-analysis demonstrated a significant improvement in QOL of IPF patients when CHMs was applied or combined with conventional medicine treatment. 6MWT was significantly improved in IPF patients after using CHMs or combined with conventional medicine treatment. CHMs treatment also had a certain improvement in TLC and DLCO, but the effect on FVC was not significant. Besides, CHMs failed to provide benefits in terms of PaO2. The reported adverse events were not obvious and severe. Conclusions. Some CHMs seem effective and safe as alternative remedies for patients with IPF, suggesting that further study of CHMs in the treatment of IPF is warranted. Although this systematic review suggests that CHMs may have positive effect on quality of life, 6-minute walk test distance, and lung function (TLC, DLOC%) and seem to be relatively safe during the course of treatment, the results must be treated with great caution because of the methodological flaws of the included trials. Long-term and high-quality trials are needed in the future to provide clear evidence for the use of CHMs.

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