Aug 2026· Current Urology Reports· Vol 27· 0 citations· 80 references
Medicine
TL;DR
This narrative review aims to summarize the contribution of population-based cancer registries, hospital-based clinical registries, and administrative healthcare databases to the understanding and management of prostate, bladder, renal, and other genitourinary malignancies.
An overview of the landscape of major U.S. neuro-oncology data resources is provided and how these datasets are used in contemporary research is evaluated, including population registries, clinical data networks, federal and consortium research cohorts, institutional datasets, specialized resources, and artificial intelligence benchmarking resources.
Anjali Kapoor, A. Alyakin, J. Markert et al.· Journal of Neuro-Oncology· 0 citations
Background Randomized controlled trials (RCTs) remain the gold standard for evidence on treatment efficacy but face limitations: restrictive eligibility criteria exclude real-world populations, head-to-head comparisons between approved regimens are rare, and absolute effectiveness in unselected cohorts often differs from trial results. High-quality cancer registries address these gaps by documenting treatment reality and quantifying effectiveness outside trial settings. We describe how the breadth and depth of prospective, longitudinal oncology registry data enable advanced research complementing RCTs. Materials and methods The iOMEDICO oncologist and hematologist network currently operates nine prospective, multicenter registry platforms across major cancer types in Germany. The registries employ regulatory-grade electronic data capture with audit trails, continuous data management, and consecutive patient enrollment. Beyond demographics and clinical variables, platforms systematically collect comorbidities, comprehensive biomarker data, complete treatment pathways, and patient-reported outcomes (PROs). Results More than 55 000 patients have been documented so far across >400 sites representing diverse care settings. Our registry data enable patient-centered research in diverse applications: (i) characterizations of patient populations, treatment patterns, and outcomes to evaluate the current standards of care, identify unmet needs, and follow developments over time; (ii) predictive and prognostic model developments; (iii) comparative effectiveness research such as target trial emulation for head-to-head treatment comparisons; (iv) translational research into biomarker prevalence; and (v) evaluation of PROs. Conclusion Well-designed prospective, longitudinal registries collecting broad and deep real-world data complement RCTs, inform Health Technology Assessments, and fill knowledge gaps by creating treatment transparency, addressing evidence gaps, and providing realistic outcome expectations for the heterogeneous populations in routine care.
M. Jänicke, S. Dille, M. Frank et al.· ESMO real world data and dig...· 0 citations
OBJECTIVE
Renal cell carcinoma (RCC) is a common malignancy that metastasizes to the spine, leading to complex treatment challenges and reduced survival. The aim of this study was to evaluate the prevalence of oncological biomarkers in patients with RCC spinal metastasis and analyze how these biomarkers, along with clinical factors, impact survival outcomes.
METHODS
This retrospective cohort study included patients with RCC spinal metastasis who were treated surgically at a single academic center between 2013 and 2024. Clinical, surgical, and treatment data were collected. Immunohistochemical analysis of the 10 most prevalent biomarkers was performed on spinal tumor specimens. Predictive modeling of overall mortality was conducted using multivariate logistic regression, decision tree, and random forest algorithms. Model performance was assessed using the area under the curve, and key interactions were identified through interaction depth analysis. Unsupervised clustering was used to stratify patients into biomarker-defined risk groups.
RESULTS
Thirty-six patients (mean age 60 years) were included in the analysis. The overall mortality rate was 61.1%, with a mean follow-up duration after diagnosis of spinal metastasis of 16.1 months. CK7 and AE1/AE3 were the most significant predictors of mortality, with CK7-positive and AE1/AE3-negative patients having an 80% mortality rate versus 25% in AE1/AE3-positive patients (p < 0.01). CAM5.2 and KRAS expression were associated with 100% and 80% mortality, respectively. Patients with EGFR positivity and CK7 negativity had 0% mortality (p = 0.01). Decision tree analysis identified CAM5.2, CK7, and AE1/AE3 as key hierarchical classifiers. In the random forest analysis, CK7 and AE1/AE3 (mean minimal depths of 2.04 and 2.34, respectively) had the highest variable importance scores (Gini p < 0.01) (area under the curve: logistic regression = 0.74; decision tree = 0.72; random forest = 0.81). Unsupervised clustering stratified patients into three molecular subgroups with distinct mortality risks: cluster 1 (61.5%), cluster 2 (50.0%), and cluster 3 (66.7%).
CONCLUSIONS
Immunohistochemical biomarkers, particularly CK7, AE1/AE3, CAM5.2, and EGFR, hold significant prognostic value in RCC spinal metastasis and can help stratify patients into high- and low-risk groups. The integration of these biomarkers into tree-based machine learning models provides interpretable data-driven decision tools to support personalized surgical and systemic treatment planning. These findings warrant validation in larger multi-institutional prospective cohorts and further analysis of biomarker interactions.
Joseph Rajasekaran, Xinlan Yang, A. Ghaith et al.· Journal of Neurosurgery : Sp...· 0 citations
Background: Population-scale molecular profiling integrated into routine healthcare could accelerate biomarker discovery, validation, and implementation, but the feasibility and sustainability of such an approach have rarely been demonstrated prospectively. The Sweden Cancerome Analysis Network - Breast (SCAN-B) Initiative was established to integrate prospective molecular profiling with population-based breast cancer care and create an infrastructure for translating molecular discoveries into clinical practice (ClinicalTrials.gov identifier NCT02306096). Methods: We evaluated the first 10 full calendar years of SCAN-B, encompassing patients with primary invasive breast cancer enrolled between August 30, 2010 and December 31, 2020. Enrollment and biospecimen collection were compared with all eligible breast cancer diagnoses in participating hospitals to assess population coverage and representativeness. Clinicopathological characteristics, treatments, recurrence-free survival, overall survival, RNA-sequencing-based molecular subtypes and risk-of-recurrence, and somatic mutations were evaluated. We additionally report the translation of SCAN-B molecular profiling from the research setting into routine clinical diagnostics. Results: Among 16,381 estimated eligible breast cancer diagnoses, 13,940 patients (85.1%) were prospectively enrolled across participating Swedish hospitals. Baseline blood samples were obtained from 98.4% of enrolled patients and tumor specimens from 71.1%; 9,323 tumors (94.0% of submitted tumor specimens) underwent RNA-sequencing. The enrolled cohort was broadly representative of the underlying breast cancer population across major clinicopathological characteristics. Integration of longitudinal clinical data with molecular profiling enabled characterization of real-world treatment patterns, long-term outcomes, molecular subtypes, risk-of-recurrence, and the somatic mutational landscape in this population-based cohort. Building on prospective real-time RNA-sequencing and subsequent development and validation of single-sample molecular subtype and risk-of-recurrence predictors, the SCAN-B workflow was transferred into routine clinical molecular diagnostics in Sk[a]ne and Blekinge in 2021. Through January 2026, more than 3,000 patients had received clinical RNA-sequencing-based molecular subtype and risk-of-recurrence reports, while prospective SCAN-B enrollment and transfer of samples and molecular data into the research infrastructure continued. Patient enrollment continues prospectively, with over 23,000 patients accrued as of January 2026. Conclusions: A prospective, population-based molecular profiling program can be integrated into routine breast cancer care at scale while maintaining high population coverage and representativeness. Over more than a decade, SCAN-B progressed from prospective biosampling and molecular profiling through biomarker development and validation to implementation of RNA sequencing-based testing in routine healthcare. This model establishes a continuous framework linking population-based molecular research, biomarker discovery and validation, and clinical implementation, and provides a strategy for integrating precision oncology research with routine cancer care.
L. Saal, H. Dalal, P. Meng et al.· medRxiv· 0 citations
Lymphomas comprise a complex and heterogeneous group of malignancies which pose challenges in understanding their epidemiology, pathobiology, treatment responses and long-term outcomes. Evolving diagnostic classification and fast-paced therapy development compound these challenges. Robust real-world data (RWD) collection and analysis using clinical registries can contribute significantly to address gaps in understanding of practice variation and provide evidence for health technology assessments. However, to maximize the impact of lymphoma registries, and those in other diseases, there is a compelling need for global collaboration, data harmonization and automated integration between registries and other large datasets. Technologies that enable safer data sharing are already available, but historical legal frameworks and evolving privacy concerns are not keeping pace, undermining their intended purpose and limiting the full potential of available high-quality RWD to improve patient care. This White Paper written by the Global Lymphoma Registry Alliance (LyRA) discusses the importance and value of lymphoma registries for different stakeholders as well as benefits of forming a global alliance of the registry network. An alliance such as LyRA serves both academic endeavors and public interest through collaboration between patient and community organizations, policy-makers, regulatory authorities, industry and others seeking to use RWD. Bringing these stakeholders together and raising awareness more broadly will facilitate timely clinical trial result contextualization and innovation in public-private collaborations on novel trial emulations and designs, including external comparator cohorts. The LyRA leadership propose strategies for overcoming barriers to facilitate these key collaborations towards improving patient outcomes on a global scale.
Eliza A. Hawkes, E. Chung, M. Bishton et al.· Haematologica· 0 citations
Spending on cancer medicines has increased rapidly worldwide, driven by the introduction of immunotherapy and targeted therapy. Australia exemplifies this trend, with cancer medicines representing one of the largest and fastest-growing areas of expenditure for Australia’s public medicines funder, the Pharmaceutical Benefits Scheme. While this growth reflects therapeutic innovation and expanded clinical use, it has intensified concerns around the real-world safety, effectiveness, and value of novel therapies once adopted into routine care. Randomised clinical trials remain essential for regulatory approval but often provide limited insight into outcomes in broader, more heterogeneous populations, particularly as many therapies enter practice via accelerated pathways based on surrogate endpoints. Population-based cancer registries offer an important resource for postmarket surveillance when linked with national administrative datasets such as dispensing and hospitalisations records. However, limitations in registries’ timeliness, disease stage ascertainment, biomarker and genomic data capture, and information on recurrence and progression constrain their current utility. This perspective examines the Australian population-based cancer registry landscape, highlighting its strengths, untapped potential, and critical gaps. We outline priority enhancements required to realise a robust, whole-of-population cancer medicine surveillance system that can inform clinical practice, policy, and sustainable health care decision making.
B. Daniels, N. Meagher, J. Ruiz et al.· ESMO real world data and dig...· 0 citations