Real-world Evidence Analysis Research Articles

Twelve-month analysis of real-world evidence from a telehealth obesity-treatment provider using antiobesity medications.

The objective of this study was to describe weight changes in members of a large-scale telehealth obesity-treatment provider who were using antiobesity medications (AOMs). This retrospective observational study analyzed real-world data from adults who initiated AOM treatment with the WW International, Inc. (formerly Weight Watchers) Clinic telehealth program between January 2022 and July 2023 (n = 53,590). The main outcomes were changes in body weight over 12 months, side effects over follow-up, and medication usage patterns. The 53,590 patients who initiated treatment were predominantly female (88.6%), with a mean BMI of 36.9 kg/m2. Mean (SD) treatment duration was 5.3 (4.3) months. Program retention rates based on the number of patients whose time from enrollment was at least 3, 6, 9, and 12 months were 78% (n = 39,907/51,247), 63% (n = 25,515/40,203), 58% (n = 15,472/26,794), and 77% (n = 6459/8394), respectively. Average weight loss was 8.9% at 3 months (n = 37,565), 14.1% at 6 months (n = 24,140), 17.7% at 9 months (n = 15,169), and 19.4% at 12 months (n = 6089). Glucagon-like peptide-1 receptor agonist (GLP-1-RA)-based treatments were predominant. Side effects were consistent with the classes of medications used, and frequency declined over time. This real-world analysis of a telehealth-delivered obesity-treatment program demonstrated outcomes consistent with recent phase 3 clinical trials of AOMs, suggesting generalizability beyond clinical trial and in-person settings.

A Review and Comparative Case Study Analysis of Real-World Evidence in European Regulatory and Health Technology Assessment Decision Making for Oncology Medicines

ObjectivesReal-world evidence (RWE) is valuable in supporting regulatory and health technology assessment (HTA) decisions; however, the actual contribution to approvals remains elusive. This study aimed to review RWE approaches and use in oncology medicine approvals in Europe and understand cohesion and discrepancy in the acceptance of the RWE by the European Medicines Agency (EMA) and European HTA bodies. MethodsThis scoping review involved a search of the EMA database, National Institute for Health and Care Excellence (NICE), Gemeinsamer Bundesausschuss (G-BA), and Haute Autorité de Santé (HAS) websites to identify final reports and appraisals for oncology medicines with references to RWE. The selection was guided by research terms associated with RWE study designs, data sources, and outcomes. Qualitative analysis was used to systemize the data. Case studies assessed by more than one agency were selected for comparative assessment of RWE approach, use, and acceptability. ResultsRWE was mainly leveraged as an external control for indirect treatment comparisons or contextualization to support clinical trial results by the EMA, NICE, G-BA, and HAS. However, this approach was mostly rejected due to methodology biases. Comparative assessment of RWE acceptability for the same oncology medicines across agencies suggests discrepancies between EMA and European HTA bodies and among NICE, G-BA, and HAS. ConclusionsThere is diverging acceptance of RWE in EMA and European HTA bodies with no clear consensus on the most effective way to leverage RWE in approvals. With the introduction of the joint European Union Joint Clinical Assessment in 2025, it is crucial for European HTA bodies and EMA to develop synergetic standards for the use of RWE to ensure equitable and timely access to medicines.

53950 Risk of thromboembolic events for JAK inhibitors in dermatology: pharmacovigilance analysis of real-world evidence in the Food and Drug Administration Adverse Event Reporting System (FAERS) from the RADAR (Research on Adverse Drug events And Reports) Prog

53950 Risk of thromboembolic events for JAK inhibitors in dermatology: pharmacovigilance analysis of real-world evidence in the Food and Drug Administration Adverse Event Reporting System (FAERS) from the RADAR (Research on Adverse Drug events And Reports) Prog

Use of Artificial Intelligence in Medical Devices for Post-Market Surveillance

Artificial intelligence (AI) has emerged as a transformative tool in post-market surveillance (PMS) for monitoring the safety and performance of medical products. This article explores the role of AI in optimizing PMS practices, focusing on its applications in signal detection, risk assessment, and regulatory compliance. By harnessing machine learning algorithms and big data analytics, AI facilitates the automated analysis of real-world evidence, including patient outcomes data, adverse event reports, and electronic health records. Through pattern recognition and anomaly detection, AI algorithms enable the early identification of potential safety issues and facilitate timely interventions to mitigate risks. Moreover, AI-driven PMS systems enhance regulatory oversight by providing regulators with comprehensive and actionable insights into product safety profiles and emerging trends. However, concerns regarding data privacy, algorithm bias, and interpretability underscore the need for transparent and ethically responsible AI deployment in PMS frameworks.

Efficacy of Supera Stent in the Treatment of Complex Femoropopliteal Artery Lesions: A Short-Term Result Analysis of Real-World Evidence

When dealing with complex femoropopliteal lesions, there is a growing preference for the utilization of drug-coated balloons (DCB) or stents. However, in real-world scenarios, a greater number of elderly patients with longer lesion lengths are encountered. The purpose of our study was to compare the efficacy and safety of different interventional strategies, including the utilization of Supera stent and DCB, in a real-world setting. This was a retrospective study that collected treatment and follow-up data of patients with complex femoropopliteal artery lesions treated between January 2019 and March 2022. All patients were categorized into three groups: "non-Supera stent group,""Supera stent group," and "Supera stent + DCB group." The primary effectiveness outcome was primary patency at 12 months, defined as duplex ultrasound peak systolic velocity ratio<2.5 at the 12-month visit. Other outcomes included improvements in Rutherford categories and safety. A total of 162 patients were enrolled in this study. Compared to non-Supera group (47.6%) in the primary patency rate at 12 months, Supera group (58.7%) or Supera + DCB group (60.0%) showed no significant difference. There were no significant differences in all-cause death, major amputation of the target limb, and target lesion revascularization (TLR) between the groups. Supera group showed a significant improvement in Rutherford category without TLR at 12 months compared to non-Supera group (73.3% vs. 54.8%, P=0.041). In 12-month follow-up, over 65% of patients maintained a Rutherford category of 3 or below, particularly in Supera group where 74.7% of patients had mild symptoms. The Supera stent has demonstrated its efficacy and safety in treating complex peripheral artery disease. However, combining Supera stent with DCB did not provide a significant advantage. Further research is necessary to validate these findings in a larger patient population.

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.

Investigating the Variability among Indicators for Quantifying Antimicrobial Use in the Intensive Care Units: Analysis of Real-world Evidence.

Deshwal PR, Tiwari P. Investigating the Variability among Indicators for Quantifying Antimicrobial Use in the Intensive Care Units: Analysis of Real-world Evidence. Indian J Crit Care Med 2024;28(7):662-676.

Post-Market Surveillance of Medical Devices Using AI

Artificial intelligence (AI) has emerged as a transformative tool in post-market surveillance (PMS) for monitoring the safety and performance of medical products. This article explores the role of AI in optimizing PMS practices, focusing on its applications in signal detection, risk assessment, and regulatory compliance. By harnessing machine learning algorithms and big data analytics, AI facilitates the automated analysis of real-world evidence, including patient outcomes data, adverse event reports, and electronic health records. Through pattern recognition and anomaly detection, AI algorithms enable the early identification of potential safety issues and facilitate timely interventions to mitigate risks. Moreover, AI-driven PMS systems enhance regulatory oversight by providing regulators with comprehensive and actionable insights into product safety profiles and emerging trends. However, concerns regarding data privacy, algorithm bias, and interpretability underscore the need for transparent and ethically responsible AI deployment in PMS frameworks.

Efficacy and safety of regorafenib combined with PD-1 inhibitors in patients with advanced hepatocellular beyond atezolizumab plus bevacizumab: A retrospective analysis of real-world evidence.

e16172 Background: In patients with advanced hepatocellular carcinoma (HCC) progressing after atezolizumab and bevacizumab, the optimal therapy scheme is debatable and the efficacy of regorafenib combined with PD-1 inhibitors is still unclear. Methods: In this retrospective study, advanced HCC patients who received regorafenib combined with PD-1 inhibitors progressing after atezolizumab plus bevacizumab were consecutively enrolled from February 2022 to January 2024. Main efficacy endpoint was overall survival (OS), following progression free survival (PFS), evaluated in accordance with RECIST v1.1. Adverse events (AEs) were recorded according to CTCAE v5.0. Results: A total of 11 eligible patients were enrolled, with 8 in Child-Pugh A and 3 in Child-Pugh B7. All patients had hepatitis B infection by with tumor embolus. All patients were classified as Barcelona Clinic Liver Cancer stage C. The AFP level is above 400ng/ml in all patients. At data cut-off at February 2024, the median OS was 16.9 months and the median PFS was 3.7 months. The 3-month and 6-month PFS rates were 70% and 35% , respectively. The 12-month OS rate was 71.4%. Grade≥3 TRAEs occurred in 9.1% (1/11) of the patients. Conclusions: Though application of regorafenib combined with PD-1 inhibitors progressing after atezolizumab plus bevacizumab present safe and effective in our cohort, these data need to be confirmed in prospective comparative studies. [Table: see text]

Abstract 6464: Real-world evidence (RWE) presented at AACR annual meetings: A 10-year trend analysis

Abstract Introduction: RWE is increasingly used in the oncology community, particularly following the 2016 21st Century Cures Act. A recent analysis of RWE presented at ASCO reported a significant increase in relative frequency of RWE abstracts from 2011-2021[1]. We conducted a similar trend analysis of RWE presented at AACR and predictive factors associated with publication. Methods: EMBASE was queried for abstracts presented at AACR 2013-2022, using terms related to study designs and methodologies that employ use of RWE. Details on study design, location, data source, patient number, cancer site, analytical approach, endpoints, and presentation type (oral vs. poster) were extracted and analyzed. Searches in Google Scholar identified published manuscripts associated with each abstract. The Cochran-Armitage trend test and Monte-Carlo estimation of the exact test were used to detect trends over time; p-values are provided for descriptive purposes. Relative risk ratios were calculated to determine statistically significant predictors of publication. Results: Of 56,920 total abstracts presented at AACR from 2013-2022, 611 (1.1%) RWE abstracts were identified. The proportion of presented abstracts with RWE increased from 0.6% in 2013 to 2.0% in 2022 (p&amp;lt;0.001). Among the RWE abstracts, use of electronic medical record (2013: 0% vs. 2022: 9.3%; p=0.002) and genomics databases (i.e., GENIE) (2013: 0% vs. 2022: 8.3%; p=0.006) increased over time. While majority of RWE abstracts were presented as posters (95.9%), the proportion of oral presentations increased in the last decade (2013: 0.0% vs. 2022: 3.7%; p=0.005). Abstracts with sample sizes &amp;lt;500 decreased significantly over time (p=0.002); those with sample sizes 5,000-&amp;lt;50,000 increased significantly over time (p=0.001). The proportion of abstracts including multiple cancer sites (vs. single-site) increased from 5.1% in 2013 to 20.4% in 2022 (p&amp;lt;0.001). The most common cancer sites were lung, breast, and colorectal. Statistically significant factors associated with publishing an abstract into a manuscript included study sample size 500-&amp;lt;5,000 or ≥100,000, inferential statistics (vs. descriptive), propensity score matching, and effectiveness or disease burden endpoints.Conclusion: RWE studies presented at AACR increased in the recent decade, with more robust study designs, incorporation of novel clinico-genomic databases such as GENIE, and advanced analytical approaches. These findings suggest the value of RWE has been increasingly recognized by the oncology research community and that RWE is being used to provide critical insights and inform patient care. Given FDA’s recent guidance on RWE to support regulatory decision-making, this trend will likely continue. [1] Azad Fet al. Longitudinal trends of real-world evidence (RWE) reporting in oncology conferences: An 11-year ASCO Annual Meeting analysis. Citation Format: Jihong Zong, Lauren C. Bylsma, Orsolya Lunacsek, Jon Fryzek. Real-world evidence (RWE) presented at AACR annual meetings: A 10-year trend analysis [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 1 (Regular Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(6_Suppl):Abstract nr 6464.

Changes in benign prostatic hyperplasia management in Valencia: a real-world evidence analysis

Introduction and objectivesA training program was developed to increase general practitioners' engagement in the optimal management of Benign Prostatic Hyperplasia (BPH). The goal of this study was to evaluate changes in BPH management after the implementation of a training program. Material and MethodsThis observational retrospective cohort study was conducted between 2019 and 2020. Aggregated data were analyzed in three evaluation periods (2010, 2012 and 2015), addressing quality indicators for diagnosis, treatment, and treatment outcomes. ResultsOverall, 118 795 patients who presented any data points were included. All quality indicators (number of IPSS and PSA determinations) increased between the first period and the last. Combination (α-blocker + 5-ARI) therapy was increasingly prescribed during the study periods whereas the proportion of prescriptions for single-agent α-blocker showed no significant differences among the periods analyzed. However, the total number of patients eligible for combination therapy who actually received this treatment was low in all periods (7.5%, 17.9%, and 20.1%, in 2010, 2012, and 2015, respectively). The outcome indicators revealed a decrease in referrals to the urology unit mostly among newly diagnosed patients. Even though the proportion of patients who underwent BPH-related surgeries increased significantly from the first to the second period, the number of surgeries remained stable between the second and third periods. ConclusionsThe training program had a generally positive impact on the management of BPH patients in PC, but the overall study period may be insufficient to show an effect on some outcome indicators such as the number of surgeries.

P712 Effectiveness of switching from intravenous to subcutaneous infliximab in Inflammatory Bowel Disease patients: A combined analysis of real-world evidence

Abstract Background Since approval, numerous inflammatory bowel disease (IBD) patients have been treated with subcutaneous (SC) infliximab (IFX), but real-life data based on a large multi-national population of patients switching from intravenous (IV) to SC IFX is lacking. Methods The ASSEMBLE project is an initiative to combine multi-national real-world cohort datasets and analyse the effectiveness and safety of SC IFX therapy. In the ASSEMBLE-1 analysis, three studies from France and the United Kingdom1-3 were integrated to assess the clinical outcomes up to 6 months (6M) after switching from IV to SC IFX. Clinical remission was defined as Harvey-Bradshaw Index (HBI) or modified HBI (mHBI) &amp;lt;5 for Crohn’s disease (CD) and Simple Clinical Colitis Activity Index (SCCAI) or partial Mayo score (PMS) &amp;lt;3 for ulcerative colitis (UC). Treatment persistence was assessed by Kaplan-Meier survival analysis. Results The data of 428 patients were pooled from the three datasets (70.6% CD, 29.4% UC). 85.4% of patients were in clinical remission before switching with median HBI or mHBI of 1 (IQR 0,2) for CD and median SCCAI of 2 (IQR 1,3) or PMS of 0 (IQR 0,0) for UC. 59.6% of patients were treated with the standard dose of IV IFX (5 mg/kg Q8W) before switching and 54.0% of patients were on concomitant immunomodulator therapy. Patients were switched to SC IFX 120 mg Q2W (94.4%) or 120 mg QW (5.6%) regimen. After switching, the overall clinical remission rates were 83.4% and 84.7% at 3M and 6M, respectively. CD patients were associated with higher remission rate (89.8%) than UC patients (71.9%) at 6M (p&amp;lt;0.001). The disease activity scores and the levels of fecal calprotectin and C-reactive protein of the overall population were maintained stable after switching (Table 1). High persistence was observed in both CD (97.3% at 3M, 94.8% at 6M) and UC patients (96% at 3M, 94.1% at 6M), with no significant difference between indications (p=0.47 at 3M and p=0.78 at 6M). Perianal CD patients (n=89) did not have a significantly worse treatment persistence rate than non-perianal CD patients (p=0.30). The most common reasons leading to drug discontinuation were lost to follow-up, consent withdrawal, worsening of disease activity, and worsening of perianal disease (3 cases for each). Two patients discontinued due to injection-site pain. Conclusion This pooled analysis of ASSEMBLE-1 confirms that switching from IV to SC IFX is effective and safe for CD and UC patients in clinical practice and that the clinical remission is well maintained up to 6M after switching, with no new concerns in safety profiles. [1] Smith et al. J Crohns Colitis 2022 [2] Buisson et al. Clin Gastroenterol Hepatol 2023 [3] Rahmany et al. Gastroenterology 2023.

Antibacterial agents and the fight against antibiotic resistance: A real-world evidence analysis of consumption and spending by an Italian healthcare company

The escalating bacterial resistance stands as an increasingly pertinent concern, particularly in the post-pandemic era where the use of antibiotics appears to be relentlessly surging, giving rise to profound apprehensions. The substantial utilization of last-generation penicillins and cephalosporins is anticipated to imminently result in the emergence of superbugs for which therapeutic solutions will be scarce. An analysis of antibiotic consumption in the hospital setting has been conducted in an Italian healthcare organization. Querying the internal management system facilitated the calculation of indicators and assessment of prescription trends. A comparison has been made between the first half of 2023 and the first half of 2022, to highlight the exponential growth in the consumption of beta-lactam antibiotics, with consumption doubling compared to the previous year's semester. Overall, considering the prescription averages, there is a prescribing growth of +29% concerning hospitalization and +28% concerning hospital discharge. However, it should be noted that the consumption of certain antibiotics such as sulphonamides and trimethoprim (-103.00%), tetracyclines (-54.00%), macrolides, lincosamides and streptogramins (-50.00%) and colistin (-13.00%) decreased. This real-world evidence analysis aimed to support the justified and comprehensible global concerns regarding bacterial resistance. The extensive consumption of antibiotics will inevitably lead to the development of increasingly drug-resistant bacteria for which no antibiotic may be efficacious. National programs addressing antibiotic resistance and the awareness of all healthcare personnel must be accorded the utmost priority to enhance consumption data and, consequently, safeguard future human survival.

CO132 A Real-World Evidence Analysis of the Impact of Patient-Physician Sex Concordance on Cancer Treatment Practices and Outcomes

CO132 A Real-World Evidence Analysis of the Impact of Patient-Physician Sex Concordance on Cancer Treatment Practices and Outcomes

Optimizing large real-world data analysis with parquet files in R: A step-by-step tutorial.

The use of open-source programming languages can facilitate open science practices in real-world evidence (RWE) studies. Real-world studies often rely on using big data, which makes using such languages complicated. We demonstrate an efficient approach that enables RWE researchers to use R to undertake RWE analysis tasks from cohort building to reporting. Using the Merative Marketscan data (2017-2019), we developed an R function to transform the data into parquet format to be used in R. Then, we compared the differences in data size before and after transformation. We compared the performance of the transformed data in R to the original data in terms of numerical consistency and running times required to complete simple exploratory tasks. To show how the transformed databases can be used in practice, we conducted a simplified replication of an active comparator new user study from the literature. All codes are available on GitHub. Our approach exhibited high efficiency in data storage, as evidenced by the converted data size, which ranged from 10% to 43% of that of the original data files. The runtime of the exploratory tasks in R generally outperformed that of the original data with SAS. We showed, through example, how the converted data can be efficiently used to implement an RWE study. We demonstrate a free and efficient solution to facilitate the use of open-source programming languages with big real-world databases, which can facilitate the adoption of open science practices.

Immune tolerance via FCR001 cell therapy compared with maintenance immunosuppression for kidney transplantation: Real-world evidence analysis of safety and efficacy.

While kidney transplantation (KTx) has traditionally required lifelong immunosuppression, an investigational stem cell therapy, FCR001, has been demonstrated to induce tolerance and eliminate the need for immunosuppression through the establishment of persistent mixed chimerism in a phase 2 clinical study. Real-world evidence (RWE) methods were employed to compare the safety and efficacy of non-myeloablative conditioning with FCR001 with standard of care [SOC] immunosuppression in a retrospective single-center analysis of outcomes among propensity score matched living-donor KTx receiving SOC (n=144) or FCR001 (n=36). Among the FCR001 recipients, 26 (72%) developed persistent chimerism allowing durable elimination of all immunosuppression. There was no significant difference in the composite primary endpoint (biopsy-proven acute rejection [BPAR], graft loss, or death) at 60 months (FCR001 27.8%, n=10 and SOC 28.5%, n=41; p=.9). FCR001 recipients demonstrated superior kidney function at 5 years (estimated glomerular filtration rate [eGFR] [mean±standard deviation]: 64.1±15.3) compared to SOC (51.7±18.8; p=.02). At 5 years, FCR001 recipients experienced fewer complications including new-onset diabetes post-transplant, although two patients developed graft versus host disease. In conclusion, RWE demonstrated that KTx combined with non-myeloablative conditioning and FCR001 resulting in superior kidney function without increasing the risk of rejection, graft loss, or death among patients off immunosuppression.

Real-World Evidence of the Top 100 Prescribed Drugs in the USA and Their Potential for Drug Interactions with Nirmatrelvir; Ritonavir

Nirmatrelvir (coadministered with ritonavir as PAXLOVIDTM) reduces the risk of COVID-19-related hospitalizations and all-cause death in individuals with mild-to-moderate COVID-19 at high risk of progression to severe disease. Ritonavir is coadministered as a pharmacokinetic enhancer. However, ritonavir may cause drug-drug interactions (DDIs) due to its interactions with various drug-metabolizing enzymes and transporters, including cytochrome P450 (CYP) 3A, CYP2D6, and P-glycoprotein transporters. To better understand the extent of DDIs (or lack thereof) of nirmatrelvir; ritonavir in a clinical setting, this study used real-world evidence (RWE) from the Optum Clinformatics Data Mart database to identify the top 100 drugs most commonly prescribed to US patients at high risk of progression to severe COVID-19 disease. The top 100 drugs were identified based on total counts associated with drugs prescribed to high-risk patients (i.e., ≥ 1 medical condition associated with an increased risk of severe COVID-19) who were continuously enrolled in the database throughout 2019 and had ≥ 1 prescription claim. Each of the 100 drugs was then assessed for DDI risk based on their metabolism, excretion, and transport pathways identified from available US prescribing and medical literature sources. Seventy drugs identified were not expected to have DDIs with nirmatrelvir; ritonavir, including many cardiovascular agents, anti-infectives, antidiabetic agents, and antidepressants. Conversely, 30 drugs, including corticosteroids, narcotic analgesics, anticoagulants, statins, and sedatives/hypnotics, were expected to cause DDIs with nirmatrelvir; ritonavir. This RWE analysis is complementary to the prescribing information and other DDI management tools for guiding healthcare providers in managing DDIs.Graphical

Efficacy and safety in unresectable hepatocellular carcinoma with VP4 tumor embolus using sinilimab plus bevacizumab: A retrospective analysis of real-world evidence.

e16156 Background: Recently, sinilimab plus bevacizumab has shown promising effects on unresectable hepatocellular carcinoma (uHCC) in ORIENT-32. However, its efficacy and safety in uHCC patients with VP4 cancer embolus remained unclear. Methods: In this retrospective study, uHCC patients with VP4 tumor embolus who received first-line sinilimab plus bevacizumab were consecutively enrolled. We also included similar patients who were treated with first-line sorafenib as comparison. Main efficacy endpoint was overall survival (OS), following progression free survival (PFS), objective response rate (ORR) and disease control rate (DCR) to be secondary endpoints, evaluated in accordance with RECIST v1.1. Adverse events (AEs) were recorded according to CTCAE v5.0. Results: Total 43 eligible patients were enrolled, with 15 in sinilimab plus bevacizumab arm (arm 1) and the other 28 in sorafenib arm (arm 2). No statistical differences were found in etiology, liver function and performance status. At data cut-off at February 2023, median PFS in arm 1 and arm 2 were 3.7 (95%CI: 1.2-6.2) and 3.8 (95%CI:3.1-4.5) months, following ORR and DCR were 6.7% &amp; 46.7% and 7.1% &amp; 35.7%. OS in arm 1 was not matured and in arm 2 was 16.1 (11.1-21.1). Not any difference was detected among all efficacy endpoints. AEs happened among almost all patients. But nobody died of AEs. Conclusions: Efficacy and safety of sinilimab plus bevacizumab in uHCC patients with VP4 tumor embolus was similar with sorafenib, which needs to be further confirmed. [Table: see text]

Efficacy and safety in unresectable hepatocellular carcinoma using atezolizumab plus bevacizumab in later-line: A retrospective analysis of real-world evidence.

e16173 Background: As far, no data has been published about later-line application of atezolizumab plus bevacizumab (T+A) in patients with unresectable hepatocellular carcinoma (uHCC). Methods: We retrospectively recruited uHCC patients who treated with later-line T+A. Efficacy endpoints included overall survival (OS) and progression free survival (PFS), in accordance with RECIST v1.1. Safety was evaluated with CTCAE v5.0. We also continually enrolled uHCC patients who received later-line regorafenib plus PD-1 inhibitors (Reg+PD-1) as a comparison. Results: Total 33 patients were continually included, 12 used T+A and 21 chose Reg+PD-1. Baseline performance status, liver function and BCLC tumor stage were similar between two cohorts. At data cut-off at February 2023, following median following-up at 18.9 months, median OS in cohort T+A and Reg+PD-1 were 8.2 (95%CI: 5.8-10.6) vs 15.6 (95%CI: 7.6-23.6) (p = 0.237). Median PFS in cohort T+A and Reg+PD-1were 5.1 (95%CI: 1.1-9.1) vs 3.5 (95%CI: 2.1-4.9) (p = 0.575). OS rate and PFS rate are listed. None of efficacy endpoints reached statistical difference. All patients suffered from AEs. No AEs-related death occurred. Conclusions: Efficacy and safety of later-line T+A in uHCC patients was similar with those who used regorafenib plus PD-1 inhibitors, which needed to be further confirmed. [Table: see text]

Real-world evidence analysis of early-stage prostate cancer using natural language processing.

e17007 Background: Real-world evidence (RWE) on the epidemiology and management of localized/locally advanced prostate cancer (LPC) is lacking. Here we used EHRead, a natural language processing framework based on SNOMED CT terminology, to extract clinical information from electronic health records (EHRs) to perform a comprehensive description of a real-world cohort of LPC patients. Here we present a follow-up of the project already communicated at ESMO 2020. Methods: This is an observational, multicenter, retrospective study based on the secondary analysis of free-text and structured clinical information found in EHRs from all observed adult patients with LPC between January 2014 and December 2018 in 12 Spanish hospitals. A total of 375 variables were extracted using EHRead to assess patient clinical characteristics, treatment patterns and journey across different prostate cancer stages. Event-free survival (EFS) was analyzed in incident LPC patients with presence of Gleason or PSA and follow-up greater than 0 days. A multivariable Cox-proportional hazards model was applied to obtain adjusted hazard ratios (aHR). Results: We observed 19788 patients with LPC during a median of 3.5 (interquartile range 1.4-7.4) years of follow-up. 52% of patients had low- (LR), 21% intermediate- (IR) and 27% high-risk (HR) disease. 3938 (18%) patients transitioned to other disease stages, leading to castration-resistant prostate cancer (mCRPC) in 4%, 7%, 16% of patients with LR, IR, and HR LPC, respectively. 7884 (42%) patients received radiotherapy (RT), 7021 (38%) prostatectomy (PT), 6061 (31%) androgen deprivation therapy (ADT), and 1034 (6%) active surveillance. Local recurrence was observed in 3% and distant metastasis in 11% (57% bone, 37% visceral). Distant metastases were observed in 7%, 12% and 21% of HR, IR and LR; 8% vs. 9% of PT vs. RT; and 16% vs 6% of ADT vs. no ADT, respectively. EFS was assessed in 7465 (38%) patients, with a 4-year EFS rate of 18%. Median EFS was 22, 13, and 8 months for LR, IR, HR; 23 vs. 12 months for PT vs. RT; and 8 vs. 21 months for ADT vs. no ADT, respectively. A multivariable cox proportional hazards model is presented in the following Table. Conclusions: During the study period, 11% of all LPC patients developed metastatic disease, with only 18% remaining event-free at 4 years. Transition to mCRPC was 4- and 2-fold higher in HR vs. IR and LR disease, respectively. Disease risk, treatment with RT and/or addition of ADT were independently associated with shorter EFS. Our findings suggest that, despite adjuvant treatment, LPC local extension and histopathological characteristics at diagnosis remain critical determinants of patient prognosis. A more detailed analysis in patients with available Gleason and PSA is underway. [Table: see text]