ObjectiveTo systematically review the difference in 30-day readmission rates among acute heart failure patients between real-world studies vs. randomized controlled trials (RCTs). MethodsPubMed, EMbase, The Cochrane Library, CNKI, CBM, VIP and WanFang Data databases were electronically searched to collect clinical studies on 30-day readmission rates in patients with acute heart failure from inception to April 12th, 2021. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies. Meta-analysis was then performed using Stata 16.0 software. ResultsA total of 33 real-world studies and 11 RCTs involving 106 722 subjects were included. The results of meta-analysis showed that the 30-day heart failure-related readmission rates in the real-world studies and RCTs were 10.8% (95%CI 9.3% to 12.3%) and 6.9% (95%CI 5.3% to 8.4%), respectively. The 30-day all-cause readmission rates in real-world studies and randomized controlled studies were 18.6% (95%CI 15.7% to 21.4%) and 14.2% (95%CI 12.0% to 16.3%), respectively. There were statistically significant differences between two kinds of studies (P<0.05). ConclusionsCurrent evidence suggests that the 30-day heart failure-related and all-cause readmission rates in patients of acute heart failure in real-world studies are significantly higher than those in patients of RCTs. Due to the limited quality and quantity of included studies, more high-quality studies are required to verify the above conclusions.
ObjectiveTo study the clinical characteristics of patients with partial and transitional atrioventricular septal defects (P/TAVSDs) in our hospital, and to evaluate the early follow-up outcomes from a real-world research perspective.MethodsThe clinical data of all patients diagnosed with P/TAVSDs from January 1, 2018 to July 12, 2020, in our hospital were collected, and all patients' examination results were used as the real-world follow-up data, univariable Cox risk proportional model was used to analyze the outcomes. A total of 93 patients were finally included in the analysis, 72 with partial and 21 with transitional AVSD. There were 38 males and 55 females at age of 182.0 months (20.0 d to 779.5 months).ResultsUnivariable Cox proportional risk model suggested that at least one cardiac malformation (HR=15.00, 95%CI 3.00 to 75.00, P=0.001), preoperative moderate or greater mitral regurgitation (HR=6.60, 95%CI 1.70 to 26.00, P=0.007), and preoperative moderate or greater tricuspid regurgitation (HR=13.00, 95%CI 3.10 to 51.00, P<0.0001) were risk factors for moderate or greater postoperative atrioventricular valve regurgitation.ConclusionChildren with coarctation of the aorta or partial pulmonary vein connection, moderate or greater preoperative mitral regurgitation, and moderate or greater preoperative tricuspid regurgitation need to be alerted to the risk of moderate or greater postoperative atrioventricular valve regurgitation. Real-world data, with relaxed statistical P values and combined expertise, can suggest clinical conclusions that are close to those of high-quality retrospective studies.
ObjectiveTo compare the effectiveness of haemocoagulase agkistrodon and tranexamic acid and sodium chloride in the prevention and treatment of perioperative bleeding in a real world setting. MethodsA research database was constructed based on the records of inpatient visits using haemocoagulase agkistrodon and tranexamic acid and sodium chloride according to the SuValue® database from January 1, 2016 to December 31, 2020. The patients were divided into two groups according to the different interventions. After matching with a 1∶1 propensity score, the effectiveness of two groups was compared. ResultsA total of 858 patients were included in each of the two groups, and there was no statistically significant difference in baseline characteristics between the two groups (P>0.05). Research results showed that patients using haemocoagulase agkistrodon had significantly reduced length of hospital stay, decrease in hematocrit, average estimated surgical bleeding, and decrease in hemoglobin (P<0.01). ConclusionHaemocoagulase agkistrodon has better effectiveness than tranexamic acid and sodium chloride for reducing perioperative blood loss based on current real world evidence.
Randomized double-blind controlled trials (RCTs) conduct researches in carefully selected populations to ensure results of RCTs are unaffected by external disturbances and provide evidence of safety and efficacy. Real-world researches further help to understand the real world effects of new technologies in different medical environments after-market authorization. RCTs are the evidence foundation of real-world researches, and real-world researches provide valuable complement to RCTs. Medical insurance database is one of the most important database in real-world researches. Now, China's national medical insurance is entering a new era and transits from passive payment and compensation into a value-based strategic purchase mechanism for its insured population to buy the most cost-effective services. It is necessary to establish a mature, well-organized and value-based mechanism. The core of such mechanism is values, which is the price/performance ratio of innovative medicines and technologies rather than looking at the price solely. Demonstrating innovative drug value is an essential part of health care assessment. The authors argue that the assessment of the overall value of innovative technologies or medicines should include and based on the following four dimensions: clinical value, economic value, patient value and society value.
ObjectiveTo analyze the neoadjuvant therapy of colorectal cancer in this center in the background of real world data by studying Database from Colorectal Cancer (DACCA) in West China Hospital of Sichuan University.MethodsData was selected from DACCA who was updated on August 15, 2019. After deleting duplicate value, patients whose tumor location and tumor pathologic characteristic showed colon or rectum, as well as adenocarcinoma, mucinous adenocarcinoma, and signet ring cell carcinoma were enrolled.ResultsThere were 2 783, 2 789, 2 790, 2 811, 4 148,3 824, 4 191, 3 676, 4 090, and 499 valid data of T, N, and M stages, clinical stages, tumor site, distance from tumor to anal dentate line, tumor pathologic characteristics, degree of tumor differentiation, neoadjuvant therapy, and compliance, respectively. There were 1 839 lines that " nature of the tumor pathology” was not empty and neoadjuvant scheme for the pure chemotherapy, radiotherapy alone or radiation, and chemotherapy, including 50 lines of signet ring cell carcinoma (2.7%), 299 lines of mucous adenocarcinoma (16.3%), 1 490 lines of adenocarcinoma (81.0%), various kinds of pathology in selection of neoadjuvant therapy difference was statistically significant (χ2=9.138, P=0.041). Except for the data lines with null value in the column of " operation date”, there were 2 234 (82.1%) and 486 (17.9%) effective data lines of " recommended” and " not recommended” for the use of neoadjuvant therapy, respectively. In the years with a large amount of data, among the patients who completed neoadjuvant therapy, the proportion of patients meeting the recommended indications was 27.4%–67.6%, with an average of 47.4%. Patients who did not meet the recommended indications but were recommended (off-label use) accounted for 7.3%–70.0%, with an average of 39.8%. According to regression analysis, the proportion in line with the recommendation (\begin{document}$\hat y $\end{document}=–0.032 5x+66.003 2, P=0.020) varies with the year, and the overall trend shows a gradual decline. The proportion of the use of super indications (\begin{document}$\hat y $\end{document}=–0.054 5x+110.174 6, P=0.002) changed with the year, and the overall trend showed a decline. A total of 1 161 valid data with non-null values of " eoadjuvant therapy regimen” and " recommended or not recommended” showed statistically significant difference in the use rate of neoadjuvant therapy among patients with different recommendation groups (χ2=9.244, P=0.002). " Patient compliance” was shown as " active cooperation” and " passive acceptance”, and " neoadjuvant therapy” was shown as " radiotherapy alone”" chemotherapy alone”, and " chemoradiotherapy” were 470 lines. There was no statistically significant difference in neoadjuvant therapy between patients receiving active and passive treatment (χ2=0.537, P=0.841). The effective data of clinical remission degree meeting the research conditions were 388 lines, including 121 lines of complete response (31.2%), 180 lines of partial response (46.4%), 79 lines of stable disease (20.4%), and 8 lines of progressive disease (2.1%). There was no statistically significant difference in clinical response degree among patients with different neoadjuvant therapy (H=0.435, P=0.783). There were 346 lines with effective data of pathologic tumor regression grade (TRG) meeting the study conditions, including 47 lines with TRG0 (13.6%), 39 lines with TRG1 (11.3%), 180 lines with TRG2 (52.0%), and 80 lines with TRG3 (23.1%). There was no statistical difference in the degree of TRG among patients with different neoadjuvant therapy (H=1.816, P=0.518).ConclusionsThe real world study reflects that in the western regional medical center, the demand for neoadjuvant therapy among the patients with colorectal cancer covered is huge. Although the implementation of neoadjuvant therapy is greatly influenced by the doctor’s recommendation behavior, the selection and recommendation of neoadjuvant therapy according to some specific clinical application guidelines are not fully met. The impact of more behavioral factors requires further in-depth analysis and research.
ObjectiveTo explore the association between frailty and in-hospital mortality in older patients with acute exacerbation of chronic obstructive pulmonary disease (AECOPD). Methods Elderly patients who were hospitalized with AECOPD from June 2022 to December 2022 at a large tertiary hospital were selected. The independent prognostic factors including frailty status were determined by multivariate logistic regression analysis. Mediation effect analysis was used to evaluate the mediating relationships between C-reactive protein (CRP) and albumin and in-hospital death. ResultsThe training set included 1 356 patients (aged 86.7±6.6), 25.0% of whom were diagnosed with frailty. The multiple logistic regression analysis showed that frailty, mean arterial pressure, Charlson comorbidity index, neutrophil–lymphocyte ratio, interleukin-6, CRP, albumin, and troponin T were associated with in-hospital mortality. Furthermore, CRP and albumin mediated the associations between frailty and in-hospital mortality. ConclusionFrailty may be an adverse prognostic factor for older patients admitted with an AECOPD. CRP and albumin may be parts of mechanism between frailty and in-hospital death.
With the gradual standardization and improvement of the real-world study system, real-world evidence, as a supplement to evidence from classical randomized controlled trials, is increasingly used to evaluate the effectiveness and safety of pharmaceuticals and medical devices. High-quality real-world evidence is not only related to the quality of real-world data, but also depends on the type of study design. Therefore, as one of the important designs for pragmatic clinical trials, the Zelen design has received much attention from investigators in recent years. This paper discussed the implementation processes, subtypes of design, advantages, limitations, statistical concerns, and appropriate application scenarios of the Zelen design, on the basis of published papers, in order to clarify its application value, and to provide references for future research.
Objective Risk factors for real-word immune checkpoint inhibitor-related pneumonitis in patients with lung cancer were analyzed by systematic analysis. Methods Computerized retrieval of PubMed, EMbase, Web of Science, the Cochrane Library , WanFang Data, CNKI and VIP databases was carried out. Studies were collected from the database establishment to March 2023. Three researchers independently screened the literature, extracted data, and evaluated the risk of bias in the included studies. Meta-analysis was performed using RevMan5.4.1software. Results A total of 18 studies were included with a total of 4 990 patients. The results of meta-analysis showed that, interstitial pneumonia [odds ratio (OR)=9.32, 95% confidence interval (CI) 4.66 - 18.67, P<0.01], smoking history (OR=2.39, 95%CI 1.29 - 4.45, P<0.01), chronic obstructive pulmonary disease (COPD) (OR=5.54, 95%CI 2.96 - 10.36, P<0.01), chest radiotherapy (OR=2.74, 95%CI 1.80 - 4.19, P<0.01), pulmonary fibrosis (OR=7.46, 95%CI 4.25 - 13.09, P<0.01), high programmed death ligand 1 (PD-L1) expression (OR=2.98, 95%CI 1.71 - 5.22, P<0.01), high absolute eosinophil count (AEC) (OR=3.92, 95%CI 2.17 - 7.08, P<0.01) and pembrolizumab (OR=2.90, 95%CI 1.56 - 5.37, P<0.01) were independent risk factors for immune checkpoint inhibitor-related pneumonitis in lung cancer patients. Conclusions Interstitial pneumonia, smoking history, COPD, Chest radiotherapy, pulmonary fibrosis, high PD-L1expression, high AEC and pembrolizumab are independent risk factors for immune checkpoint inhibitor-related pneumonitis in lung cancer patients. Due to insufficient evidence on the risk factors of low albumin, more studies are needed to further identify it.
ObjectiveTo systematically reviewed the progress of Zelen’s design and its modifications in clinical research and clarified its methodological elements, advantages, and limitations. MethodsA systematic literature search was conducted for Zelen’s design from databases. The data were extracted. ResultsOne hundred and twenty-four trials were included. The dominant disease in this design was mental disorders, followed by osteoarthrosis diseases, cancer, cardiovascular diseases, and others. Regarding types of consent, more than half of the trials used a double-consent (71, 57.26%), and 42 used a single-consent. Eleven trials used a modified Zelen’s design nested within an observational study. This design used a two-stage informed consent. Stage 1, patients were invited to participate in a cohort study; Stage 2, patients randomized in the experimental group were informed of the allocation result and asked whether they would like to follow the treatment. Five trials used the McNulty-Zelen design, which could be applied in cluster randomized controlled trials and overcome the potential bias of the Hawthorne effect. Intention-to-treat analysis was the main population used in Zelen’s design. ConclusionZelen’s design has a broad application in the foreground in clinical trials. It could also be used to adapt to research needs by combining with various observational studies. Zelen’s design offers unique advantages in reducing recruitment difficulty, improving patient compliance, and minimizing bias. Although the randomization of patients without their prior consent raises potential ethical concerns, these can be addressed through methods such as nested observational studies or supplementary informed consent. In real-world applications of Zelen’s design, it is necessary to design a reasonable informed consent strategy and data statistical analysis method according to the research context. Attention must be paid to the impact of sample size, group shifting and selection of dataset on the results, to improve the interpretability and accuracy of the results.
ObjectiveTo compare the 5-year survival rates between two different follow-up patterns of postoperative stage Ⅰ-ⅢA non-small cell lung cancer (NSCLC) patients.MethodsPathological stage Ⅰ-ⅢA NSCLC 11 958 patients who underwent surgical resection and received follow-up within 6 months after initial diagnosis through telephone follow-up system were included in nine hospitals from July 2014 to July 2020. The patients were divided into two groups including a proactive follow-up group (n=3 825) and a passive follow-up group (n=8133) according to the way of following-up. There were 6 939 males and 5 019 females aged 59.8±9.5 years. The Kaplan-Meier and Cox proportional hazards regression model were used.ResultsThe median follow-up frequency was 8.0 times in the proactive follow-up group and 7.0 times in the passive follow-up group. The median call duration was 3.77 minutes in the proactive follow-up group and 3.58 minutes in the passive follow-up group. The 5-year survival rate was 81.8% and 74.2% (HR=0.60, 95CI 0.53-0.67, P<0.001) in the proactive follow-up group and the passive follow-up group, respectively. Multivariate analysis showed that follow-up pattern, age, gender and operation mode were independent prognostic factors, and the results were consistent in all subgroups stratified by clinical stages.ConclusionThe proactive follow-up leads to better overall survival for resected stage Ⅰ-ⅢA NSCLC patients, especially in the stage ⅢA.