Real-world Comparative Studies Research Articles

Choosing the optimal therapy for metastatic renal cell carcinoma: Insights from a real-world comparative study.

The evolution of combination therapies integrating immune checkpoint inhibitors has revolutionized the first-line treatment of metastatic renal cell carcinoma (mRCC). Although these therapies are clinically approved, direct comparisons between dual immune checkpoint inhibitors (IOIO) and immune checkpoint inhibitors combined with tyrosine kinase inhibitors (IOTKI) in clinical trials are lacking. This gap creates uncertainties in selecting the most appropriate treatment based on patient-specific factors. This study employed the inverse probability of treatment weighting (IPTW) method to analyze progression-free survival (PFS) and overall survival (OS) for patients with mRCC receiving IOIO or IOTKI treatment regimens. A total of 171 patients were analyzed after applying inclusion criteria and propensity scoring. The study found no significant differences in PFS and OS between the two treatment modalities in the IPTW cohort. However, subgroup analyses revealed that IOTKI therapy was associated with better PFS and OS for patients without bone metastases and better OS for patients with a body mass index (BMI) over 25. IOIO therapy showed better OS for patients with a BMI below 18.5. Both IOIO and IOTKI therapies were effective. Therapy selection could be better tailored to patient characteristics by including factors such as the presence of bone metastases and BMI. This study enhances understanding of how patient-specific factors interact with different treatment modalities, potentially guiding more personalized treatment decisions in clinical practice for mRCC.

Cost analysis of patiromer vs sodium zirconium cyclosilicate for the treatment of hyperkalemia in Spain and the UK

Background Hyperkalemia is an electrolyte abnormality with potentially life-threatening consequences. Published data have shown that potassium-binding polymer patiromer (Veltassa) is associated with reduced rates of severe edema and hospitalization for heart failure compared with sodium zirconium cyclosilicate (SZC, Lokelma) when treating hyperkalemia. The aim of this study was to evaluate the possible costs associated with these interventions in the Spanish and UK settings. Methods A cost-analysis model was developed in Microsoft Excel to compare the costs associated with patiromer and SZC for the management of hyperkalemia. Clinical event rates were taken from a published real-world comparative study, with the base case capturing the statistically significant reduction in severe edema with patiromer vs SZC and a sensitivity analysis also including the non-statistically significant reduction in hospitalization for heart failure. Country-specific costs, expressed in 2022 Euros (EUR) and British pounds sterling (GBP), were evaluated from a healthcare payer perspective and included pharmacy costs and costs of clinical events. Results Patiromer may be associated with cost savings of EUR 107 and GBP 630 per patient-year of treatment vs SZC in Spain and the UK, respectively. The majority of cost savings were due to the possible lower daily cost of patiromer compared with SZC. Including the difference in heart failure hospitalization rates in a sensitivity analysis led to greater cost savings with patiromer over SZC, increasing to EUR 460 and GBP 902 in Spain and the UK, respectively. Extrapolation of patient-level economic outcomes to a population level found that patiromer was associated with annual cost savings of EUR 30.6 million in Spain, and GBP 801.7 million in the UK vs SZC. Conclusions Patiromer has the potential to be cost saving vs SZC for the treatment of hyperkalemia in Spain and the UK based on the results of a real-world evidence analysis.

Adverse events following immunization of co- and separate administration of DTaP-IPV/Hib vaccines: A real-world comparative study

ABSTRACT To fully understand the safety of DTaP-IPV/Hib vaccination, we evaluated the differences between DTaP-IPV/Hib co-administration and separate administration of the DTaP, IPV and Hib vaccines (DTaP+IPV+Hib) based on adverse events following immunization (AEFI). All AEFI reported in Hebei Province, China, between 2020 and 2022 were included in this study. The risk difference (RD%), relative risk (RR), and Chi-square value were used to compare the differences in reported rates of AEFI between the DTaP-IPV/Hib and DTaP+IPV+Hib groups. From 2020 to 2022, 130 AEFI cases were reported in Hebei Province after DTaP-IPV/Hib vaccination, corresponding to an AEFI reported rate of 66.9/million doses, which was significantly lower than that for DTaP+IPV+Hib (9836 AEFI with a reported rate of 637.8/million doses). The overall reported rate of non-severe AEFI for DTaP+IPV+Hib vaccines was 9.5 times that of DTaP-IPV/Hib vaccination [95% confidence interval (CI): 8.0, 11.3]. Meanwhile, the reported rate of AEFI among infants aged 0–1 y was 9.8 times higher for DTaP+IPV+Hib than for DTaP-IPV/Hib (95% CI: 8.2, 11.7). DTaP+IPV+Hib vaccination also resulted in higher risks of high fever, localized redness and swelling, localized induration, and allergic rash compared with DTaP-IPV/Hib vaccination. The risk of AEFI, which were mostly mild reaction, was higher after vaccination with DTaP+IPV+Hib than after DTaP-IPV/Hib vaccination.

737-P: Acute Healthcare Utilization, Mortality, and Cardiovascular Events following GLP-1-RA Initiation by Patients with Advanced Chronic Kidney Disease—A Real-World Comparative Study

737-P: Acute Healthcare Utilization, Mortality, and Cardiovascular Events following GLP-1-RA Initiation by Patients with Advanced Chronic Kidney Disease—A Real-World Comparative Study

Displacement of Submacular Hemorrhage Using Subretinal Cocktail Injection versus Pneumatic Displacement: A Real-World Comparative Study

Introduction: The objective of this study was to compare the outcome of submacular hemorrhage (SMH) displacement using pneumatic displacement with intravitreal expansile gas versus pars plana vitrectomy (PPV) with subretinal injection of tissue plasminogen activator (tPA), anti-vascular endothelial growth factor (VEGF) agent, and air as primary surgery. Methods: Retrospective interventional case series of 63 patients who underwent surgical displacement of SMH secondary to neovascular age-related macular degeneration (nAMD) or polypoidal choroidal vasculopathy (PCV) from May 1, 2015, to October 31, 2022. Medical records were reviewed for diagnosis, logMAR visual acuity (VA), central subfield thickness (CST), and postoperative displacement rates and complications up to 12 months after operation. Results: The diagnosis was nAMD in 24 (38.1%) and PCV in 39 (61.9%) eyes. There were 40 (63.5%) eyes in the pneumatic displacement group (38 received C3F8, 2 received SF6) and 23 (36.5%) eyes in the subretinal cocktail injection. Mean baseline VA was 1.46 and 1.62, respectively (p = 0.404). The subretinal injection group had more extensive SMH (p = 0.005), thicker CST (1,006.6 μm vs. 780.2 μm, p = 0.012), and longer interval between symptom and operation (10.65 vs. 5.53 days, p < 0.001). The mean postoperative VA at 6 months was 0.67 and 0.91 (p = 0.180) for pneumatic displacement and subretinal injection groups, respectively, though VA was significantly better in the pneumatic group at 12-month visit (0.64 vs. 1.03, p = 0.040). At least 10 mean change in VA were >10 letters gain in both groups up to 12 months. Postoperative CST reduction was greater (625.1 μm vs. 326.5 μm, p = 0.008) and complete foveal displacement (87.0% vs. 37.5%), p < 0.001, odds ratio [OR] = 11.1) and displacement to arcade or beyond (52.5% vs. 17.5%, p = 0.009, OR = 5.15) were more frequent in the subretinal injection group. Two patients with failed pneumatic displacement were successfully treated with subretinal cocktail injection as a second operation. Conclusion: Surgical displacement of SMH leads to clinically meaningful improvement in VA. PPV with subretinal cocktail injection is more effective than pneumatic displacement in displacing SMH with similar safety profile despite longer interval before operation, higher CST, and more extensive SMH at baseline. Retinal surgeons could consider this novel technique in cases with thick and extensive SMH or as a rescue secondary operation in selected cases.

Real-world data on the effectiveness of TYRX and TauroPace for preventing CIED infections.

The implantation of cardiac implantable electronic devices (CIEDs) carries a known risk of infection. Two devices (TYRX and TauroPace) have been proposed to reduce this risk. The aim of our study was to compare the effectiveness of TauroPace and TYRX. Real-world comparative studies were included. Data analysis was based on reconstruction of individual patient data from Kaplan-Meier curves using an artificial intelligence algorithm. The endpoint was CIED infection or systemic infection. Statistical tests included heterogeneity assessment, superiority testing, and non-inferiority testing. The primary outcome measure was the hazard ratio (HR) with confidence interval (CI). Our literature search identified two real-world studies suitable for our analysis. Follow-up was 12 months for TauroPace (654 patients) and 60 months for TYRX (872 patients), with a total of 2,083 controls. There was no heterogeneity among controls. Compared to the pooled control group, patients treated with TYRX or TauroPace had fewer CIED infections (HR, 0.3892; 95% CI, 0.2042-0.7419; P=0.00414; HR, 0.3313; 95% CI, 0.1005-1.0925; P=0.06958, respectively). When testing for non-inferiority of TauroPace vs. TYRX, the comparison yielded a HR of 0.8494 (in favor of TYRX) with a 90% CI of 0.27-2.63; this CI of TauroPace did not meet the non-inferiority criterion set at HR>0.75 (i.e., relative difference ≤25%). Both treatments had some important drawbacks. Regarding TYRX, more selective use in higher-risk patients should be advocated to improve its cost-effectiveness, but robust evidence is still lacking. Regarding TauroPace, our analysis testing for a non-inferiority margin of ≤25% did not meet this demonstration.

Real-world comparative study of drug retention of Janus kinase inhibitors in patients with rheumatoid arthritis.

Janus kinase (JAK) inhibitors (JAKis) are effective therapeutic agents against rheumatoid arthritis (RA). However, patients having RA with particular risk factors may have a higher incidence of adverse effects (AEs), including major cardiovascular events (MACE) and infections. In this multicenter cohort study, we aimed to clarify the risk factors affecting the drug retention of JAKis in patients with RA. We retrospectively evaluated patients with RA who received their first JAKi (tofacitinib, baricitinib, upadacitinib, or filgotinib) at our institute. The clinical outcomes, including AEs, were recorded, particularly MACE and serious infections. The drug retention rates were analyzed using the Kaplan-Meier method, and risk factors affecting drug retention rates were determined using a multivariable Cox regression hazards model. Overall 184 patients with RA receiving their first use of baricitinib (57.6%), tofacitinib (23.9%), upadacitinib (12.0%), or filgotinib (6.5%) were included in this study. Fifty-six (30.4%) patients discontinued JAKi treatment owing to ineffectiveness (9.2%) or AEs, including infections (21.2%). The overall drug retention rates were significantly lower in patients treated with pan-JAKi than in those treated with JAK1 inhibitors (p = 0.03). In the Cox regression model, the presence of baseline high RA disease activity, use of glucocorticoid and treatments with pan-JAKis were associated with reduced drug retention rates of JAKis (p < 0.001, p = 0.01 and 0.04, respectively). Pan-JAKi treated patients with high disease activity had significantly lower drug retention rates (p < 0.001). In a real-world setting, the drug retention rates of JAKis were reduced mainly by treatment discontinuation owing to AEs. Treatment with pan-JAKis and high baseline RA disease activity were identified as predictive factors for the discontinuation of JAKis. Lower drug retention rates were found in patients receiving pan-JAKis with high disease activity than in those without high disease activity.

Real-world comparative study of the efficacy of Janus kinase inhibitors in patients with rheumatoid arthritis: the ANSWER cohort study.

This multicentre, retrospective study compared the efficacy and safety of tofacitinib, baricitinib, peficitinib and upadacitinib in real-world clinical settings after minimizing selection bias and adjusting the confounding patient characteristics. The 622 patients were selected from the ANSWER cohort database and treated with tofacitinib (TOF), baricitinib (BAR), peficitinib (PEF) or upadacitinib (UPA). The patient's background was matched using propensity score-based inverse probability of treatment weighting (IPTW) among four treatment groups. The values of Clinical Disease Activity Index (CDAI), C-reactive protein (CRP), and modified Health Assessment Questionnaire (mHAQ) after drug initiation and the remission or low disease activity (LDA) rates of CDAI at 6 months after drug initiation were compared among the four groups. Further, the predictive factor for TOF and BAR efficacy was analysed. The retention and discontinuation rates until 6 months after drug initiations were not significantly different among the four JAK inhibitors treatment groups. Mean CDAI value, CDAI remission rate, and CDAI-LDA rate at 6 months after drug initiation were not significantly different among treatment groups. Baseline CDAI (TOFA: OR 1.09, P < 0.001; BARI: OR 1.07, P < 0.001), baseline CRP (TOFA: OR 1.32, P = 0.049), baseline glucocorticoid dose (BARI: OR 1.18, 95% CI 1.01-1.38, P = 0.035), a number of previous biological or targeted synthetic disease-modifying antirheumatic drugs (biological/targeted synthetic DMARDs) (BARI: OR 1.36, P = 0.004) were predictive factors for resistance to CDAI-LDA achievement to JAK inhibitor treatment. The efficacy and safety of TOF, BAR, PEF and UPA were not significantly different for the treatment of patients with rheumatoid arthritis.

Multi-expert attention network for long-term dam displacement prediction

Monitoring and predicting the dam displacement of concrete dams has attracted increasing attention for ensuring the long-term health conditions. Most existing models focus on just temporal features and ignore the spatial features relations of monitoring data. To address these problems, a dam displacement prediction model based on multi-expert network is developed. In the proposed model, the long short-term memory network is employed to extract the temporal features of each monitoring sensor. Then the multi-head attention network is employed to obtain the spatial features and the adjacency values. The multi-expert graph is employed to describe the spatial relations between different monitoring sensors. The graph convolutional network is employed to integrate the spatio-temporal features and predict the long-term dam displacement. Through a real-world comparative study against eight prediction models, the proposed model performs better than other models in both cyclical and non-cyclical time series. Therefore, the proposed model is suitable for evaluating the dam health condition.

The value of regional and global CACS combined with SPECT MPI in detecting obstructive CAD: a retrospective real-world comparative study

ObjectivePrevious studies have shown that global coronary artery calcium score (CACS) can improve single photon emission computerized tomography (SPECT) myocardial perfusion imaging (MPI) to detect obstructive coronary artery disease (CAD). Whether regional CACS can improve SPECT MPI to detect obstructive CAD remains unclear. The aim of this study was to verify whether regional CACS has additional diagnostic value for obstructive CAD in suspected patients, compared to SPECT MPI and global CACS.MethodsThe study included 321 suspected CAD patients who underwent one-stop rest-stress SPECT MPI and low-dose computed tomography (CT) scan. All patients underwent coronary angiography within one month after examination. MPI images were visually analyzed by 2 experienced nuclear cardiologists. The regional CACS of left anterior descending coronary artery (LAD), left circumflex coronary artery (LCX), right coronary artery (RCA) and global CACS were calculated. Obstructive CAD was defined as ≥ 70% narrowing of the inner diameter of the LAD, LCX, RCA or their main branches and ≥ 50% narrowing of the left main coronary artery (LM).ResultsAmong the 321 patients, 86 (26.8%, 86/321) had obstructive CAD. With the increased in global and regional CACS, there was an increasing trend of patients with obstructive CAD (P for trend < 0.001). Regional CACS had a better diagnostic performance in RCA territories (AUC 0.856, P < 0.001) compared with LAD, LCX territories (AUC 0.690, 0.674, respectively). The AUC of combined regional CACS and MPI was significantly higher than that of MPI alone (0.735 vs. 0.600, P < 0.001). However, based on MPI, the AUC of combined regional CACS was not significantly higher than that of global CACS (0.735 vs. 0.732, P = 0.898). The sensitivity and specificity of regional CACS combined with MPI for detecting obstructive CAD were 64.0% and 72.8%, respectively.ConclusionsRegional CACS was effective in detecting obstructive CAD in RCA territory. Based on SPECT MPI, regional CACS improved the detection of obstructive CAD, but was not superior to global CACS.

Association between Haploidentical Hematopoietic Stem Cell Transplantation Combined with an Umbilical Cord Blood Unit and Graft-Versus-Host Disease in Pediatric Patients with Acquired Severe Aplastic Anemia

Introduction: Haploidentical hematopoietic stem cell transplantation (haplo HSCT) based on granulocyte colony-stimulating factor plus anti-thymocyte regimens ("Beijing Protocol") provides a salvage treatment for patients of acquired severe aplastic anemia (SAA) in China. However, graft-versus-host disease (GVHD) is a major impediment of haplo HSCT due to HLA disparity. Human umbilical cord blood (UCB) is characterized by lower numbers of mature or primed T-cells. Additionally, UCB has been reported to be a rich and rapidly accessible source of regulatory T cells (Tregs) and mesenchymal stromal cells (MSCs). These characteristics of UCB make it a potential prophylactic method for GVHD. Recently, haplo HSCT combined with umbilical cord blood (UCB) (haplo-cord HSCT) is performed in clinical trials to potentially reduce the risk of severe GVHD. Nevertheless, studies comparing GVHD in pediatric patients receiving haplo and haplo-cord HSCT for SAA are limited. Herein, we aim to investigate the impact of UCB co-infusion on GVHD in pediatric patients receiving haplo HSCT for SAA. Methods: Data from 91 consecutive children with SAA treated with "Beijing Protocol"-based haploidentical transplantation with or without co-infusion of UCB between April 2015 and July 2021 in the Children's Hospital of Soochow University were retrospectively analyzed. The cohort included 35 patients in the haplo group and 56 patients in the haplo-cord group. All patients received uniform non-myeloablative conditioning, GVHD prophylaxis. We compared baseline characteristics and transplant outcomes between the haplo and haplo-cord recipients. Log-rank test and Cox proportional regression model were used for univariate and multivariate analysis, respectively. A significance level of 0.05 was used for all analyses. Results: No significant differences in the baseline information were observed between haplo and haplo-cord groups. All 91 patients achieved hematopoietic recovery from haploidentical donors, with a higher 30-day cumulative incidence of platelet engraftment observed with haplo group as compared to haplo-cord group (98.2% versus 97.1%, p = 0.028). Cox regression analysis revealed that the haplo or haplo-cord transplant type was not associated with the time to platelet engraftment, while a low CD34+ cell dose in haploidentical grafts was correlated with delayed platelet recovery (hazard ratio (HR), 2.495; 95% confidence interval (CI), 1.570-3.964; p < 0.001). The haplo-cord group showed a higher incidence of peri-engraftment syndrome compared to the haplo group (75.0% versus 48.6%, p = 0.029), and Cox regression analysis identified haplo-cord HSCT as the only significant factor associated with a higher incidence of peri-ES (HR, 1.767; 95% CI, 1.002-3.115; p = 0.049). Of note, the haplo-cord group showed a lower incidence of II-IV acute GVHD (aGVHD) than the haplo group (16.1% versus 42.9%, p = 0.002). Observed incidences of chronic GVHD (cGVHD) and moderate to severe cGVHD in the haplo-cord group were also lower than that in the haplo group (25.6% versus 51.3%, p = 0.019; 16.2% versus 41.3%, p = 0.016, respectively). Haplo-cord HSCT was identified as the only factor associated with a lower incidence of II-IV aGVHD (HR, 0.335; 95% CI, 0.143-0.784; p = 0.012) and cGVHD (HR, 0.429; 95% CI, 0.203-0.907; p = 0.027) in Cox regression analysis. No differences were observed between the two groups in CMV, EBV infection, overall survival, failure-free survival and GVHD, failure-free survival. Conclusions: Our study provided compelling results of descriptive analysis suggesting that co-infusion of a third-party UCB unit with haplo HSCT might be associated with a lower risk of II-IV aGVHD and cGVHD. Although this co-infusion protocol may come with a higher risk of peri-ES, the survival outcomes may not be negatively affected. Despite the limited sample size, this study is the first real-world comparative study to investigate the impact of haplo-cord HSCT on GVHD in SAA children, which may stimulate further research of haplo-cord HSCT in pediatric patients with SAA and hematologic malignancies, as well as the potential association with GVHD. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

The efficacy and safety of conventional transcatheter arterial chemoembolization combined with PD-1 inhibitor and anti-angiogenesis tyrosine kinase inhibitor treatment for patients with unresectable hepatocellular carcinoma: a real-world comparative study.

AimWe sought to evaluate the efficacy and safety of conventional transcatheter arterial chemoembolization (cTACE) sequentially combined with systemic treatment by programmed cell death protein 1 (PD-1) inhibitor and anti-angiogenesis tyrosine kinase inhibitor (Anti-angiogenesis TKI) in patients with unresectable hepatocellular carcinoma (HCC).Materials and methodsOne hundred and forty-seven advanced HCC patients who received PD-1 inhibitors and TKIs as first-line systemic treatment between August 2019 and April 2021 were collected retrospectively. Fifty-four patients were finally included and divided into cTACE and no-cTACE groups, according to whether cTACE treatment was performed within 8 weeks before systemic treatment. The tumor objective response ratio (ORR), progression-free survival (PFS), overall survival (OS), and adverse events (AEs) were compared between the groups. Significant factors affecting PFS and OS were determined by Cox regression.ResultsThirty-one patients received cTACE followed by systemic treatment and 23 patients received systemic treatment only. The ORRs of the cTACE group were 48.4% (after two cycles of systemic treatment) and 51.6% (after four cycles of systemic treatment), while those of the no-cTACE group were only 17.4% and 21.7%. cTACE patients also had a longer median PFS (11.70 vs. 4.00 months, P = 0.031) and median OS (19.80 vs. 11.6 months, P = 0.006) than no-cTACE patients. Regression analyses indicated that cTACE therapy and Eastern Cooperative Oncology Group performance status were independent risk factors for PFS and OS. AEs by type were similar between the cTACE and no-cTACE groups, except for liver function injury, which was more common among cTACE patients. Fourteen patients suffered with grade 1-2 of rash in 21 patients with objective response, while only 10 patients suffered with rash in 33 patients without objective response, the adjusted hazard ratio (HR) was 4.382 (1.297–14.803).ConclusionsThe combination of cTACE and PD-1 inhibitors and anti-angiogenesis TKIs as therapy significantly improved markers of treatment efficacy, including ORR, PFS, and OS, in unresectable HCC patients, while no more serious AEs recorded in this population compared to those receiving systemic treatment alone. Skin rash might be a predict factor to the efficacy of PD-1 inhibitors and TKI treatment.

A large-scale real-world comparative study using pre-COVID lockdown and post-COVID lockdown data on predicting shipment times of therapeutics in e-pharmacy supply chains

PurposeThe purpose of this study is to present a large-scale real-world comparative study using pre-COVID lockdown data versus post-COVID lockdown data on predicting shipment times of therapeutic supplies in e-pharmacy supply chains and show that our proposed methodology is robust to lockdown effects.Design/methodology/approachThe researchers used organic data of over 5.9 million records of therapeutic shipments, with 2.87 million records collected pre-COVID lockdown and 3.03 million records collected post-COVID lockdown. The researchers built various Machine Learning (ML) classifier models on the two datasets, namely, Random Forest (RF), Extra Trees (XRT), Decision Tree (DT), Multi-Layer Perceptron (MLP), XGBoost (XGB), CatBoost (CB), Linear Stochastic Gradient Descent (SGD) and the Linear Naïve Bayes (NB). Then, the researchers stacked these base models and built meta models on top of them. Further, the researchers performed a detailed comparison of the performances of ML models on pre-COVID lockdown and post-COVID lockdown datasets.FindingsThe proposed approach attains performance of 93.5% on real-world post-COVID lockdown data and 91.35% on real-world pre-COVID lockdown data. In contrast, the turn-around times (TAT) provided by therapeutic supply logistics providers are 62.91% accurate compared to reality in post-COVID lockdown times and 73.68% accurate compared to reality pre-COVID lockdown times. Hence, it is clear that while the TAT provided by logistics providers has deteriorated in the post-pandemic business climate, the proposed method is robust to handle pandemic lockdown effects on e-pharmacy supply chains.Research limitations/implicationsThe implication of the study provides a novel ML-based framework for predicting the shipment times of therapeutics, diagnostics and vaccines, and it is robust to COVID-19 lockdown effects.Practical implicationsE-pharmacy companies can readily adopt the proposed approach to enhance their supply chain management (SCM) capabilities and build resilience during COVID lockdown times.Originality/valueThe present study is one of the first to perform a large-scale real-world comparative analysis on predicting therapeutic supply shipment times in the e-pharmacy supply chain with novel ML ensemble stacking, obtaining robust results in these COVID lockdown times.

A Real-World Comparative Study of Microwave and Radiofrequency Ablation in Treatment-Naïve and Recurrent Hepatocellular Carcinoma.

The efficacy and safety of microwave ablation (MWA) compared to radiofrequency ablation (RFA) for patients with treatment-naïve and recurrent hepatocellular carcinoma (HCC) has not been clarified in Korea. There were 150 HCC patients (100 in the RFA group and 50 in the MWA group) enrolled in our study. The primary outcome was one- and two-year disease-free survival (DFS). Secondary outcomes were complete response (CR) rate, two-year survival rate, risk factors for DFS and complication rate. Treatment outcomes were also assessed using propensity-score matching (PSM). The MWA group had better one- and two-year DFS than the RFA group (p = 0.035 and p = 0.032, respectively), whereas the CR rate, two-year survival rate, and complication rate were similar between the two groups with fewer major complications in the MWA group (p = 0.043). Patients with perivascular tumors, high risk of recurrence, and small tumor size (≤3 cm) were more suitable for MWA than RFA. MWA was also an independent factor for favorable one- and two-year DFS. Finally, the MWA group still showed better one- and two-year DFS than the RFA group after PSM. In conclusion, MWA could be an alternative treatment to RFA especially in patients with a high risk of recurrence, perivascular tumors, and small tumor size.

DRLSTM: A dual-stage deep learning approach driven by raw monitoring data for dam displacement prediction

Dam displacement is an important indicator of the overall dam health status. Numerical prediction of such displacement based on real-world monitoring data is a common practice for dam safety assessment. However, the existing methods are mainly based on statistical models or shallow machine learning models. Although they can capture the timing of the dam displacement sequence, it is difficult to characterize the complex coupling relationship between displacement and multiple influencing factors (e.g., water level, temperature, and time). In addition, input factors of most dam displacement prediction models are artificially constructed based on modelers’ personal experience, which lead to a loss of valuable information, thus prediction power, provided by the full set of raw monitoring data. To address these problems, this paper proposes a novel dual-stage deep learning approach based on one-Dimensional Residual network and Long Short-Term Memory (LSTM) unit, referred to herein as the DRLSTM model. In the first stage, the raw monitoring sequence is processed and spliced with convolution to form a combined sequence. After the timing information is extracted, the convolution direction is switched to learn the complex relationship between displacement and its influencing factors. LSTM is used to extract this relationship to obtain Stage I prediction. The second stage takes the difference between the actual measurement and the Stage I prediction as inputs, and LSTM extracts the stochastic features of the monitoring system to obtain Stage II prediction. The sum of two stage predictions forms the final prediction. The DRLSTM model only requires raw monitoring data of water level and temperature to accurately predict displacement. Through a real-world comparative study against four commonly used shallow learning models and three deep learning models, the root mean square error and mean absolute error of our proposed method are the smallest, being 0.198 mm and 0.149 mm respectively, while the correlation coefficient is the largest at 0.962. It is concluded that the DRLSTM model performance well for evaluating dam health status.

Identification of cancer patients using claims data from health insurance systems: A real-world comparative study.

ObjectiveTo evaluate the accuracy of identifying cancer patients by use of medical claims data in a health insurance system in China, and provide the basis for establishing the claims-based cancer surveillance system in China.MethodsWe chose Hua County, Henan Province as the study site, and randomly selected 300 and 1,200 qualified inpatient electronic medical records (EMRs) as well as the New Rural Cooperative Medical Scheme (NCMS) claims records for cancer patients in Hua County People’s Hospital (HCPH) and Anyang Cancer Hospital (ACH) in 2017. Diagnostic information for NCMS claims was evaluated on an individual level, and sensitivity and positive predictive value (PPV) were calculated taking the EMRs as the gold standard.ResultsThe sensitivity of NCMS was 95.2% (93.8%−96.3%) and 92.0% (88.3%−94.8%) in ACH and HCPH, respectively. The PPV of the NCMS was 97.8% (96.7%−98.5%) in ACH and 89.0% (84.9%−92.3%) in HCPH. Overall, the weighted and combined sensitivity and PPV of NCMS in Hua County was 93.1% and 92.1%, respectively. Significantly higher sensitivity and PPV in identifying patients with common cancers than non-common cancers were detected in HCPH and ACH separately (P<0.01).ConclusionsIdentification of cancer patients by use of the NCMS is accurate on individual level, and it is therefore feasible to conduct claims-based cancer surveillance in areas not covered by cancer registries in China.

First-line afatinib for the treatment of EGFR mutation-positive non-small-cell lung cancer in the 'real-world' clinical setting.

Afatinib is an ErbB family blocker that is approved for the treatment of epidermal growth factor receptor (EGFR) mutation-positive non-small-cell lung cancer (NSCLC). Pivotal randomized clinical studies demonstrated that afatinib significantly prolonged progression-free survival compared with platinum-based chemotherapy (LUX-Lung 3, LUX-Lung 6), and with gefitinib (LUX-Lung 7), with manageable side effects. However, these results were derived from controlled studies conducted in selected patients and are not necessarily representative of real-world use of afatinib. To gain a broader understanding of the effectiveness and safety of first-line afatinib, we have undertaken a literature review of real-world studies that have assessed its use in a variety of patient populations. We focused on patients with uncommon EGFR mutations, brain metastases, or those of advanced age, as these patients are often excluded from clinical studies but are regularly seen in routine clinical practice. The available real-world studies suggest that afatinib has clinical activity, and is tolerable, in diverse patient populations in an everyday clinical practice setting. Moreover, consistent with LUX-Lung 7, several real-world comparative studies indicate that afatinib might confer better efficacy than first-generation EGFR tyrosine kinase inhibitors. Tolerability-guided dose adjustment, undertaken in 21–68% of patients in clinical practice, did not appear to reduce the efficacy of afatinib. Taken together, these findings provide further support for the use of afatinib as a treatment option in patients with EGFR mutation-positive NSCLC.

Abstract 131: Ranolazine Improves Cardiovascular Outcomes and Healthcare Utilization: A Comparative Effective Analysis with Traditional Antianginal Medications

Introduction: Real-world comparative studies evaluating traditional with newer antianginal medications in chronic stable angina (CSA) on cardiovascular (CV) outcomes and healthcare utilization are limited. Methods: Medical and pharmacy claims from 2008-2012 were analyzed using a large commercial database. Patients with a CSA diagnosis receiving ranolazine, beta blocker (BB), calcium channel blocker (CCB), or long-acting nitrates (nitrates) were identified and followed for 12 months after a change in antianginal therapy. Patients on traditional antianginal medication: BB, CCB, and nitrates were required to have concurrent sublingual nitroglycerin. Therapy change was defined as adding or switching to another traditional antianginal or ranolazine to identify patients who had failed prior therapy. Four groups were identified (BB, CCB, nitrates, or ranolazine users) and matched for age,gender, baseline Charlson Comorbidity Index (CCI), acute coronary syndrome,hypertension, diabetes, heart failure, hyperlipidemia, diabetes related complications, and cardiovascular healthcare costs. Rates for percutaneous intervention (PCI) and coronary bypass graft (CABG) at 30, 60, 90, 180 and 360 days, as well as, annual number of CV-related outpatient visits, emergency room (ER) visits, and inpatient admissions post therapy change were compared between groups. Results: A total of 8008 patients were identified with 2002 patients in each antianginal matched group. The majority (63-65%) were male with a mean age of 66 years and a CCI of 2.89-3.01. The Table summarizes the annual rate per 1000 for CV outcomes. Compared to other antianginal therapies, ranolazine consistently exhibited lower PCI and CABG rates at all time periods following therapy change. Compared to BB, CCB,and nitrates,ranolazine had a 21% (p=0.004), 4% (p=0.578), and 23% (p&lt;0.0001) lower mean number of annual CV inpatient admissions; and 15% (p&lt;0.0001), 4% (p=0.224), and 13% (p&lt;0.0001) lower mean number of annual CV outpatient visits,respectively.Ranolazine had a 17-21% lower mean annual number of CV ER visits compared to the other antianginal groups (p=0.073,p=0.079, p=0.106,respectively). Conclusion: Ranolazine improves CV outcomes and lowers healthcare utilization compared to traditional antianginal therapies.

Varenicline versus transdermal nicotine patch: a 3-year follow-up in a smoking cessation clinic in Taiwan

A network meta-analysis of randomized trials and real-world comparative studies strongly suggest that varenicline is more effective in aiding smoking cessation than single form nicotine replacement therapy (NRT). Modeling the health benefits attributable to this difference relies on extrapolation to lifetime cessation, but to date, follow-up has only extended to 12 months. Longer term follow-up data are helpful in checking these assumptions. This study aimed to compare the sustained abstinence rates of smokers using varenicline versus nicotine patch in their quit attempt up to 36 months. Five hundred eighty-seven smokers were recruited at Kaohsiung Veteran General Hospital between Feb 2006 and Aug 2009. Participants received counseling from a physician and received either varenicline (N=296) or the nicotine patch (N=291) for smoking cessation. Both varenicline and nicotine patch users could receive their medications for a maximum of 8 weeks. Participants were followed up by telephone at 3, 6, 12, and 36 months from the first visit. The primary outcome measure was self-reported sustained abstinence up to 36 months. Measures were also taken of smoking characteristics, cigarette dependence, and sociodemographic characteristics. Multiple logistic regression of 36-month sustained abstinence on to medication adjusting for other baseline variables showed a significant advantage for varenicline, OR=7.94 (95 % CI 1.87-33.74). An 8-week course of varenicline appears to yield higher abstinence rate up to 3 years than a similar length course of nicotine transdermal patch in routine clinical practice where behavioral support is available.

Does device matter and at what cost? Real-world comparative study of insulin glargine treatment using disposable pen vs vial-and-syringe in Medicaid patients with type 2 diabetes mellitus

Does device matter and at what cost? Real-world comparative study of insulin glargine treatment using disposable pen vs vial-and-syringe in Medicaid patients with type 2 diabetes mellitus