scholarly journals A Large Gap in Patients’ Characteristics and Outcomes between the Real-World and Clinical Trial Settings in Community-Acquired Pneumonia and Healthcare-Associated Pneumonia

2022 ◽  
Vol 11 (2) ◽  
pp. 297
Author(s):  
Nobuhiro Asai ◽  
Yuichi Shibata ◽  
Daisuke Sakanashi ◽  
Hideo Kato ◽  
Mao Hagihara ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Real World ◽  
Randomized Clinical Trials ◽  
Community Acquired Pneumonia ◽  
Rank Test ◽  
Exclusion Criteria ◽  
The Real ◽  
Log Rank Test ◽  
Healthcare Associated ◽  
Based Medicine

(1) Introduction: Evidence-based medicine (EBM) is necessary to standardize treatments for infections because EBM has been established based on the results of clinical trials. Since entry criteria for clinical trials are very strict, it may cause skepticism or questions on whether the results of clinical trials reflect the real world of medical practice. (2) Methods: To examine how many patients could join any randomized clinical trials for the treatment of community-acquired pneumonia (CAP) and healthcare-associated pneumonia (HCAP). We reviewed all the pneumonia patients in our institute during 2014–2017. The patients were divided into two groups: patients who were eligible for clinical trials (participation-possible group), and those who were not (participation-impossible group). Exclusion criteria for clinical trials were set based on previous clinical trials. (3) Results: A total of 406 patients were enrolled in the present study. Fifty-seven (14%) patients were categorized into the participation-possible group, while 86% of patients belonged to the participation-impossible group. Patients in the participation-possible group had less comorbidities and more favorable outcomes than those with the participation-impossible group. As for the outcomes, there were significant differences in the 30-day and in-hospital mortality rates between the two groups. In addition, the participation-possible group showed a longer overall survival time than the participation-impossible group (p < 0.001 by Log-Rank test). (4) Conclusion: There is a difference in patients’ profile and outcomes between clinical trials and the real world. Though EBM is essential to advance medicine, we should acknowledge the facts and the limits of the clinical trials.

scholarly journals To What Degree Could Clinical Trials in Evidence Based Medicine Reflect The Real World in The Treatment of Pneumonia?

2021 ◽  
Author(s):  
Nobuhiro Asai ◽  
Yuichi Shibata ◽  
Daisuke Sakanashi ◽  
Arufumi Shiota ◽  
Hideo Kato ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Real World ◽  
Randomized Clinical Trials ◽  
Evidence Based ◽  
Exclusion Criteria ◽  
The Real ◽  
Evidenced Based ◽  
Based Medicine ◽  
Community Onset ◽  
Evidenced Based Medicine

Abstract BackgroundEvidenced based medicine (EBM) is necessary to standardize treatments for infections because EBM　has been established based on the results of clinical trials. Since entry criteria for clinical trials are　very strict, it ​may cause skepticism or question whether the results of clinical trials　reflect the real world ​of medical practice.MethodsTo examine how many patients could join any randomized clinical trials for the　treatment of community-onset pneumonia, we reviewed all the pneumonia patients in our institute　during 2014-2017. The patients were divided into two groups: patients who were eligible for clinical　trials (participation possible group), and those who were not (participation impossible group).　Exclusion criteria for clinical trials were set based on previous clinical trials.ResultsA total of 406 patients were enrolled in the present study. Fifty-seven (14%) patients were categorized into the participation possible group, while 86% ​of patients belonged to the participation impossible group. Patients in the participation possible group had less comorbidities and ​more favorable outcomes than those with the participation impossible group. As for the outcomes, there were significant differences in the 30-day and in-hospital mortality rates between the two groups. In addition, the participation possible group showed a longer overall survival time than the participation impossible group (p<0.001 by Log-Rank test).ConclusionThere is a difference in patients’ profile and outcomes between clinical trials and the real world. Though EBM is essential to advance ​medicine, we should acknowledge the fact​s and the limit​s of the clinical trial.

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 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.

Abstract 261: Applying Clinical Trial Data to Real-World: Apixaban, Dabigatran, and Rivaroxaban

2014 ◽  
Vol 7 (suppl_1) ◽  
Author(s):  
Alpesh Amin ◽  
Michael Stokes ◽  
Ning Wu ◽  
Elyse Gatt ◽  
Dinara Makenbaeva ◽  
...  
Keyword(s):  
Atrial Fibrillation ◽  
Clinical Trials ◽  
Relative Risk ◽  
Clinical Outcome ◽  
Intracranial Hemorrhage ◽  
Major Bleeding ◽  
Real World ◽  
Randomized Clinical Trials ◽  
Absolute Risk ◽  
The Real

BACKGROUND: Data from randomized controlled trials and a real-world sample of non-valvular atrial fibrillation patients were combined to estimate the absolute effect of each new oral anticoagulant (NOAC, apixaban, dabigatran, and rivaroxaban) versus warfarin on stroke and major bleeding rates in real-world clinical practice. METHODS: Non-valvular atrial fibrillation patients were selected from Medco healthplans during 2007-2010. Reference rates for stroke and major bleeding excluding intracranial hemorrhage (to avoid double counting) were calculated for real-world Medco patients during warfarin use. Real-world event rates for NOACs were estimated by multiplying the corresponding relative risk from the randomized clinical trials by each reference rate. Absolute risk reductions and numbers needed to treat (NNT) or numbers needed to harm (NNH) for each NOAC vs. warfarin were then estimated. Reduction in net clinical outcome was calculated by summing the absolute risk reductions for stroke and major bleeding excluding intracranial hemorrhage for each NOAC versus warfarin. RESULTS: Each NOAC resulted in a reduction in stroke events compared with warfarin in the real-world (TABLE). Apixaban was the only NOAC to reduce the rate of major bleeding excluding intracranial hemorrhage compared with warfarin. The NNT to avoid one net clinical outcome (stroke plus major bleeding excluding intracranial hemorrhage) per year was 32 and 84 for apixaban and dabigatran, respectively. Rivaroxaban resulted in an increase in net clinical outcome (NNH=166). CONCLUSIONS: If relative risk reductions from randomized clinical trials persist in the real-world, apixaban would result in the greatest clinical benefit versus warfarin of all NOACs in terms of stroke and major bleeding excluding intracranial hemorrhage events avoided.

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