Stratification of patients by tumor type using molecular profiling in real-world data.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e19262-e19262
Author(s):  
Alyssa Antonopoulos ◽  
Elizabeth Eldridge ◽  
George Managadze ◽  
Elia Stupka ◽  
Hakim Lakhani ◽  
...  
Keyword(s):  
Real World ◽  
Clinical Decision Making ◽  
Single Gene ◽  
Data Access ◽  
Clinical Decision ◽  
Molecular Data ◽  
Tumor Type ◽  
Real World Data ◽  
Number Of Patients ◽  
Biomarker Data

e19262 Background: While Next-Generation Sequencing (NGS) tests become increasingly more common for diagnosis, molecular characterization, and treatment, a significant amount of molecular data derives from single-gene or analyte tests. Single gene test information is stored in disparate sources including electronic medical record (EMR) and data access for clinical use remains a challenge. A solution that harmonizes biomarker data beyond standard NGS-centric data and linked to rich clinical data is required for the complete patient picture. Methods: Health Catalyst’s extended real-world database, Touchstone includes a molecular data mart that integrates data from provider and life sciences proprietary NGS panels, Laboratory Information Systems, and other repositories. A portion of the data is derived from single-gene tests documented in the EMR. Biomarker data from EMRs was extracted from six health systems via a proprietary pipeline for extracting biomarker data. The algorithm relies on a curated ontology for molecular terms and publicly available terminologies for human genetics. Minor transformations extract pertinent variant information where available to harmonize with NGS-level data. Results: Over 44 thousand molecular labs from over 24 thousand patients were identified with this method. The oncology classes for which molecular data was identified in the greatest number of patients include skin, hematological, breast, digestive, and lung cancers (Table). PRTN3, EGFR, BRAF, JAK2, ERBB2, and KRAS are among the most commonly tested genes. Conclusions: Integrated real-world clinical and biomarker data from single gene tests can inform clinical decision-making and support clinical trial recruitment across a broader set of patient population. [Table: see text]

scholarly journals Ondansetron use is associated with lower COVID-19 mortality in a Real-World Data network-based analysis

2021 ◽  
Author(s):  
Gregory M Miller ◽  
Austin J Ellis ◽  
Rangaprasad Sarangarajan ◽  
Amay Parikh ◽  
Leonardo O Rodrigues ◽  
...  
Keyword(s):  
Artificial Intelligence ◽  
Real World ◽  
Clinical Decision Making ◽  
Clinical Decision ◽  
Long Term Effects ◽  
Real World Data ◽  
World Data ◽  
Antiemetic Agent ◽  
Future Loss ◽  
Selection Operator

Objective: The COVID-19 pandemic generated a massive amount of clinical data, which potentially holds yet undiscovered answers related to COVID-19 morbidity, mortality, long term effects, and therapeutic solutions. The objective of this study was to generate insights on COVID-19 mortality-associated factors and identify potential new therapeutic options for COVID-19 patients by employing artificial intelligence analytics on real-world data. Materials and Methods: A Bayesian statistics-based artificial intelligence data analytics tool (bAIcis®) within Interrogative Biology® platform was used for network learning, inference causality and hypothesis generation to analyze 16,277 PCR positive patients from a database of 279,281 inpatients and outpatients tested for SARS-CoV-2 infection by antigen, antibody, or PCR methods during the first pandemic year in Central Florida. This approach generated causal networks that enabled unbiased identification of significant predictors of mortality for specific COVID-19 patient populations. These findings were validated by logistic regression, regression by least absolute shrinkage and selection operator, and bootstrapping. Results: We found that in the SARS-CoV-2 PCR positive patient cohort, early use of the antiemetic agent ondansetron was associated with increased survival in mechanically ventilated patients. Conclusions: The results demonstrate how real world COVID-19 focused data analysis using artificial intelligence can generate valid insights that could possibly support clinical decision-making and minimize the future loss of lives and resources.

Rivaroxaban real-world evidence: Validating safety and effectiveness in clinical practice

2016 ◽  
Vol 116 (S 02) ◽  
pp. S13-S23 ◽  
Author(s):  
A. Camm ◽  
Craig Coleman ◽  
CAPT Tamayo ◽  
Jan Beyer-Westendorf
Keyword(s):  
Clinical Practice ◽  
Real World ◽  
Clinical Decision Making ◽  
Current Knowledge ◽  
Oral Anticoagulants ◽  
Clinical Decision ◽  
Routine Clinical Practice ◽  
Real World Data ◽  
Real World Evidence ◽  
Randomised Controlled

SummaryRandomised controlled trials (RCTs) are considered the gold standard of clinical research as they use rigorous methodologies, detailed protocols, pre-specified statistical analyses and well-defined patient cohorts. However, RCTs do not take into account the complexity of real-world clinical decision-making. To tackle this, real-world data are being increasingly used to evaluate the long-term safety and effectiveness of a given therapy in routine clinical practice and in patients who may not be represented in RCTs, addressing key clinical questions that may remain. Real-world evidence plays a substantial role in supporting the use of non-vitamin K antagonist (VKA) oral anticoagulants (NOACs) in clinical practice. By providing data on patient profiles and the use of anticoagulation therapies in routine clinical practice, real-world evidence expands the current awareness of NOACs, helping to ensure that clinicians are well-informed on their use to implement patient-tailored clinical decisions. There are various issues with current anticoagulation strategies, including under- or overtreatment and frequent monitoring with VKAs. Real-world studies have demonstrated that NOAC use is increasing (Dresden NOAC registry and Global Anticoagulant Registry in the FIELD-AF [GARFIELD-AF]), as well as reaffirming the safety and effectiveness of rivaroxaban previously observed in RCTs (XArelto on preveNtion of sTroke and non-central nervoUS system systemic embolism in patients with non-valvular atrial fibrillation [XANTUS] and IMS Disease Analyzer). This article will describe the latest updates in real-world evidence across a variety of methodologies, such as non-interventional studies (NIS), registries and database analyses studies. It is anticipated that these studies will provide valuable clinical insights into the management of thromboembolism, and enhance the current knowledge on anticoagulant use and outcomes for patients.

scholarly journals Use of real-world evidence for oncology clinical decision making in emerging economies

2021 ◽  
Author(s):  
Fernando Petracci ◽  
Chirag Ghai ◽  
Andrew Pangilinan ◽  
Luis Alberto Suarez ◽  
Roberto Uehara ◽  
...  
Keyword(s):  
Emerging Economies ◽  
Real World ◽  
Clinical Decision Making ◽  
Clinical Decision ◽  
Data Availability ◽  
Full Potential ◽  
Structural Barriers ◽  
Real World Data ◽  
Real World Evidence ◽  
Dosing Strategies

Real-world evidence (RWE) can provide insights into patient profiles, disease detection, treatment choice, dosing strategies, treatment sequencing, adverse event management and financial toxicity associated with oncology treatment. However, the full potential of RWE is untapped in emerging economies due to structural and behavioral factors. Structural barriers include lack of regulatory engagement, real-world data availability, quality and integrity. Behavioral barriers include entrenched healthcare professional behaviors that impede rapid RWE understanding and adoption. These barriers can be addressed with close collaboration of healthcare stakeholders; of whom, regulators need to be at the forefront given their ability to facilitate use of RWE in healthcare policy and legislation.

Quality criteria for Real-World Data in pharmaceutical research and healthcare decision making. An Austrian Expert Consensus (Preprint)

2021 ◽  
Author(s):  
Peter Klimek ◽  
Dejan Baltic ◽  
Martin Brunner ◽  
Alexander Degelsegger-Marquez ◽  
Gerhard Garhöfer ◽  
...  
Keyword(s):  
Decision Making ◽  
Medical Research ◽  
Real World ◽  
Pharmaceutical Medicine ◽  
Pharmaceutical Research ◽  
Quality Criteria ◽  
Clinical Decision ◽  
European Medicines Agency ◽  
Real World Data ◽  
World Data

UNSTRUCTURED Real-world data (RWD) collected in routine healthcare processes and transformed to real-world evidence (RWE) has become increasingly interesting within research and medical communities to enhance medical research and support regulatory decision making. Despite numerous European initiatives, there is still no cross-border consensus or guideline determining which quality RWD must meet in order to be acceptable for decision making within regulatory or routine clinical decision support. An Austrian expert group led by GPMed (Gesellschaft für Pharmazeutische Medizin, Austrian Society for Pharmaceutical Medicine) reviewed drafted guidelines, published recommendations or viewpoints to derive a consensus statement on quality criteria for RWD to be used more effectively for medical research purposes beyond registry-based studies discussed in the European Medicines Agency (EMA) guideline for registry-based studies

PATH-39. INTEGRATED MOLECULAR-MORPHOLOGICAL MENINGIOMA CLASSIFICATION: A MULTICENTER RETROSPECTIVE ANALYSIS, RETRO- AND PROSPECTIVELY VALIDATED

2021 ◽  
Vol 23 (Supplement_6) ◽  
pp. vi123-vi124
Author(s):  
Sybren Maas ◽  
Damian Stichel ◽  
Thomas Hielscher ◽  
Philipp Sievers ◽  
Anna Berghoff ◽  
...  
Keyword(s):  
Dna Methylation ◽  
Copy Number ◽  
Retrospective Cohort ◽  
Clinical Decision Making ◽  
Clinical Decision ◽  
Molecular Data ◽  
Copy Number Variations ◽  
Who Grade ◽  
Risk Of Progression ◽  
The Individual

Abstract PURPOSE Meningiomas are the most frequent primary intracranial tumors. Patient outcome varies widely from cases with benign to highly aggressive, ultimately fatal courses. Reliable identification of risk of progression for the individual patient is of pivotal importance in clinical management. However, only biomarkers for highly aggressive tumors are established at present (CDKN2A/B and TERT), while no molecularly-based stratification exists for the broad spectrum of low- and intermediate-risk meningioma patients. PATIENTS AND METHODS DNA methylation data and copy-number information were generated for 3,031 meningiomas of 2,868 individual patients, with mutation data for 858 samples. DNA methylation subgroups, copy-number variations (CNV), mutations and WHO grading were comparatively analyzed. Prediction power for outcome of these parameters was assessed in an initial retrospective cohort of 514 patients, and validated on a retrospective cohort of 184, and on a prospective cohort of 287 multi-center cases, respectively. RESULTS Both CNV and methylation family- (MF)-based subgrouping independently resulted in an increase in prediction accuracy of risk of recurrence compared to the WHO classification (c-indexes WHO 2016, CNV, and MF 0.699, 0.706 and 0.721, respectively). Merging all independently powerful risk stratification approaches into an integrated molecular-morphological score resulted in a further, substantial increase in accuracy (c-index 0.744). This integrated score consistently provided superior accuracy in all three cohorts, significantly outperforming WHO grading (c-index difference p=0.005). Besides the overall stratification advantage, the integrated score separates more precisely for risk of progression at the diagnostically challenging interface of WHO grade 1 and grade 2 tumors (HR 4.56 [2.97;7.00], 4.34 [2.48;7.57] and 3.34 [1.28; 8.72] for discovery, retrospective, and prospective validation cohort, respectively). CONCLUSIONS Merging these layers of histological and molecular data into an integrated, three-tiered score significantly improves the precision in meningioma stratification. Implementation into diagnostic routine informs clinical decision-making for meningioma patients on the basis of robust outcome prediction.

scholarly journals Awareness, Knowledge, and Utility of RCT Data vs RWE: Results From a Survey of US Cardiologists: Real-world Evidence in Clinical Decision Making

2020 ◽  
Vol 14 ◽  
pp. 117954682095341 ◽  
Author(s):  
Todd C Villines ◽  
Mark J Cziraky ◽  
Alpesh N Amin
Keyword(s):  
Decision Making ◽  
Clinical Practice ◽  
Real World ◽  
Randomized Clinical Trials ◽  
Clinical Decision Making ◽  
Clinical Decision ◽  
The Body ◽  
Representative Sampling ◽  
Real World Evidence ◽  
Wide Range

Real-world evidence (RWE) provides a potential rich source of additional information to the body of data available from randomized clinical trials (RCTs), but there is a need to understand the strengths and limitations of RWE before it can be applied to clinical practice. To gain insight into current thinking in clinical decision making and utility of different data sources, a representative sampling of US cardiologists selected from the current, active Fellows of the American College of Cardiology (ACC) were surveyed to evaluate their perceptions of findings from RCTs and RWE studies and their application in clinical practice. The survey was conducted online via the ACC web portal between 12 July and 11 August 2017. Of the 548 active ACC Fellows invited as panel members, 173 completed the survey (32% response), most of whom were board certified in general cardiology (n = 119, 69%) or interventional cardiology (n = 40, 23%). The survey results indicated a wide range of familiarity with and utilization of RWE amongst cardiologists. Most cardiologists were familiar with RWE and considered RWE in clinical practice at least some of the time. However, a significant minority of survey respondents had rarely or never applied RWE learnings in their clinical practice, and many did not feel confident in the results of RWE other than registry data. These survey findings suggest that additional education on how to assess and interpret RWE could help physicians to integrate data and learnings from RCTs and RWE to best guide clinical decision making.

scholarly journals In-depth phenotyping for clinical stratification of Gaucher disease

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Simona D’Amore ◽  
Kathleen Page ◽  
Aimée Donald ◽  
Khadijeh Taiyari ◽  
Brian Tom ◽  
...  
Keyword(s):  
Real World ◽  
Gaucher Disease ◽  
Orthopaedic Surgery ◽  
Clinical Decision Making ◽  
Burden Of Illness ◽  
Fragility Fractures ◽  
Neurological Manifestations ◽  
Real World Data ◽  
World Data ◽  
Advanced Therapies

Abstract Background The Gaucher Investigative Therapy Evaluation is a national clinical cohort of 250 patients aged 5–87 years with Gaucher disease in the United Kingdom—an ultra-rare genetic disorder. To inform clinical decision-making and improve pathophysiological understanding, we characterized the course of Gaucher disease and explored the influence of costly innovative medication and other interventions. Retrospective and prospective clinical, laboratory and radiological information including molecular analysis of the GBA1 gene and comprising > 2500 variables were collected systematically into a relational database with banking of collated biological samples in a central bioresource. Data for deep phenotyping and life-quality evaluation, including skeletal, visceral, haematological and neurological manifestations were recorded for a median of 17.3 years; the skeletal and neurological manifestations are the main focus of this study. Results At baseline, 223 of the 250 patients were classified as type 1 Gaucher disease. Skeletal manifestations occurred in most patients in the cohort (131 of 201 specifically reported bone pain). Symptomatic osteonecrosis and fragility fractures occurred respectively in 76 and 37 of all 250 patients and the first osseous events occurred significantly earlier in those with neuronopathic disease. Intensive phenotyping in a subgroup of 40 patients originally considered to have only systemic features, revealed neurological involvement in 18: two had Parkinson disease and 16 had clinical signs compatible with neuronopathic Gaucher disease—indicating a greater than expected prevalence of neurological features. Analysis of longitudinal real-world data enabled Gaucher disease to be stratified with respect to advanced therapies and splenectomy. Splenectomy was associated with an increased hazard of fragility fractures, in addition to osteonecrosis and orthopaedic surgery; there were marked gender differences in fracture risk over time since splenectomy. Skeletal disease was a heavy burden of illness, especially where access to specific therapy was delayed and in patients requiring orthopaedic surgery. Conclusion Gaucher disease has been explored using real-world data obtained in an era of therapeutic transformation. Introduction of advanced therapies and repeated longitudinal measures enabled this heterogeneous condition to be stratified into obvious clinical endotypes. The study reveals diverse and changing phenotypic manifestations with systemic, skeletal and neurological disease as inter-related sources of disability.

scholarly journals ACADEMIA’S ROLE TO DRIVE CHANGE IN THE ORTHOTICS AND PROSTHETICS PROFESSION

2021 ◽  
Vol 4 (2) ◽  
Author(s):  
Géza Kogler ◽  
Christopher Hovorka
Keyword(s):  
Technology Use ◽  
Clinical Training ◽  
Clinical Decision Making ◽  
New Technologies ◽  
Clinical Care ◽  
Clinical Decision ◽  
The United States ◽  
Academic Program ◽  
Number Of Patients ◽  
Curricular Emphasis

This position paper outlines the important role of academia in shaping the orthotics and prosthetics (O&P) profession and preparing for its future. In the United States, most healthcare professions including O&P are under intense pressure to provide cost effective treatments and quantifiable health outcomes. Pivotal changes are needed in the way O&P services are provided to remain competitive. This will require the integration of new technologies and data driven processes that have the potential to streamline workflows, reduce errors and inform new methods of clinical care and device manufacturing. Academia can lead this change, starting with a restructuring in academic program curricula that will enable the next generation of professionals to cope with multiple demands such as the provision of services for an increasing number of patients by a relatively small workforce of certified practitioners delivering these services at a reduced cost, with the expectation of significant, meaningful, and measurable value. Key curricular changes will require replacing traditional labor-intensive and inefficient fabrication methods with the integration of newer technologies (i.e., digital shape capture, digital modeling/rectification and additive manufacturing). Improving manufacturing efficiencies will allow greater curricular emphasis on clinical training and education – an area that has traditionally been underemphasized. Providing more curricular emphasis on holistic patient care approaches that utilize systematic and evidence-based methods in patient assessment, treatment planning, dosage of O&P technology use, and measurement of patient outcomes is imminent. Strengthening O&P professionals’ clinical decision-making skills and decreasing labor-intensive technical fabrication aspects of the curriculum will be critical in moving toward a digital and technology-centric practice model that will enable future practitioners to adapt and survive. Article PDF Link: https://jps.library.utoronto.ca/index.php/cpoj/article/view/36673/28349 How To Cite: Kogler GF, Hovorka CF. Academia’s role to drive change in the orthotics and prosthetics profession. Canadian Prosthetics & Orthotics Journal. 2021; Volume 4, Issue 2, No.21. https://doi.org/10.33137/cpoj.v4i2.36673 Corresponding Author: Géza F. KoglerOrthotics and Prosthetics Unit, Kennesaw State University.E-Mail: [email protected] ID: https://orcid.org/0000-0003-0212-5520

scholarly journals Deep Learning: A New Horizon for Personalized Treatment of Depression?

2018 ◽  
Vol 16 (1) ◽  
Author(s):  
David Benrimoh ◽  
Robert Fratila ◽  
Sonia Israel ◽  
Kelly Perlman
Keyword(s):  
Deep Learning ◽  
Decision Aid ◽  
Clinical Decision Making ◽  
Clinical Decision ◽  
Personalized Care ◽  
Treatment Of Depression ◽  
Number Of Patients ◽  
Treatment Plans ◽  
Patient Responses ◽  
Deep Learning Model

Globally, depression affects 300 million people and is projected be the leading cause of disability by 2030. While different patients are known to benefit from different therapies, there is no principled way for clinicians to predict individual patient responses or side effect profiles. A form of machine learning based on artificial neural networks, deep learning, might be useful for generating a predictive model that could aid in clinical decision making. Such a model’s primary outcomes would be to help clinicians select the most effective treatment plans and mitigate adverse side effects, allowing doctors to provide greater personalized care to a larger number of patients. In this commentary, we discuss the need for personalization of depression treatment and how a deep learning model might be used to construct a clinical decision aid.

Sign in / Sign up

Export Citation Format

Share Document