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Precision medicine statistics

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https://www.readbyqxmd.com/read/29451516/-evidence-based-medicine-vs-personalized-medicine
#1
Lucio Capurso
Evidence-based medicine (EBM) and personalized medicine (PM) are driven by two diverse modes of reasoning about «evidence making». EBM has been criticized since his quality mark has been misappropriated by vested interests, the benefits statistically significant may be marginal in clinical practice, rigid rules and technology may produce care that is management driven rather than patient centered. On the contrary PM (or "precision medicine") refers to the tailoring of medical treatment to the specific characteristics of each patient involving the ability to classify individuals into subpopulations that are uniquely susceptible to a specific treatment, sparing expense and side effects and is derived from doubts on the results of subgroup analyses and on non responders in clinical trials typical of EBM...
January 2018: Recenti Progressi in Medicina
https://www.readbyqxmd.com/read/29446868/the-amyloid-polymorphism-in-the-protein-folding-and-aggregation-energy-landscape
#2
Raffaele Mezzenga, Jozef Adamcik
Protein folding (and misfolding) is of paramount importance to an extraordinary vast class of fields, spanning from biochemistry to biology, medicine, biophysics and statistical thermodynamics. The folding process involves a large number of steps and conformations in which the folding protein samples different thermodynamic states characterized by local minima and activation energies in between. The kinetically trapped on- or off-pathway intermediates identifying the relative minima are metastable folding intermediates towards the lowest absolute energy minima, which have been postulated to be the natively folded state for the regime in which the intramolecular interactions dominate, and the amyloid status for the regime where intermolecular interactions rule...
February 15, 2018: Angewandte Chemie
https://www.readbyqxmd.com/read/29438006/statistics-refresher-for-molecular-imaging-technologists-part-2-accuracy-of-interpretation-significance-and-variance
#3
Mary Beth Farrell
This article is the second part of continuing education series reviewing basic statistics that nuclear medicine and molecular imaging technologists should understand. In this article, the statistics for evaluating interpretation accuracy, significance and variance are discussed. Throughout the article, actual statistics are pulled from published literature. Part two begins by explaining two methods for quantifying interpretive accuracy: inter-reader and intra-reader reliability. Agreement among readers can simply be expressed by percentage...
February 2, 2018: Journal of Nuclear Medicine Technology
https://www.readbyqxmd.com/read/29417022/a-study-of-knowledge-attitude-and-practice-regarding-administration-of-pediatric-dosage-forms-and-allied-health-literacy-of-caregivers-for-children
#4
Amrita Sil, Chaitali Sengupta, Alak Kumar Das, Puspita Das Sil, Supratim Datta, Avijit Hazra
Context: Caregivers of sick children have to be careful with medicine dosing and giving medicines to a reluctant child can be challenging. Aim: To assess the knowledge, attitude, and practices of caregivers regarding pediatric medicine administration and health literacy allied to this task. Settings and Design: This cross-sectional study was carried out on outpatient and inpatient basis in the pediatrics department of a teaching hospital over 6 months...
July 2017: Journal of Family Medicine and Primary Care
https://www.readbyqxmd.com/read/29409561/validation-of-the-register-based-lifetime-antimicrobial-usage-measurement-for-finisher-batches-based-on-comparison-with-recorded-antimicrobial-usage-at-farm-level
#5
V D Andersen, P Munk, L V de Knegt, M S Jensen, F M Aarestrup, H Vigre
Assessing the relationship between antimicrobial usage (AMU) and antimicrobial resistance (AMR) requires the accurate and precise utilisation of register data. Therefore, validation of register-based data is essential for evaluating the quality and, subsequently, the internal validity of studies based on the data. In this study, different smoothing methods for Veterinary Medicine Statistic Program database (VetStat)-records were validated by comparing these with farm-records. Comparison between measurements included accuracy as; completeness and correctness, and precision as; a relative difference of the error, correlation with Fisher's z transformation and reliability coefficient...
February 7, 2018: Epidemiology and Infection
https://www.readbyqxmd.com/read/29373992/beyond-genomics-understanding-exposotypes-through-metabolomics
#6
REVIEW
Nicholas J W Rattray, Nicole C Deziel, Joshua D Wallach, Sajid A Khan, Vasilis Vasiliou, John P A Ioannidis, Caroline H Johnson
BACKGROUND: Over the past 20 years, advances in genomic technology have enabled unparalleled access to the information contained within the human genome. However, the multiple genetic variants associated with various diseases typically account for only a small fraction of the disease risk. This may be due to the multifactorial nature of disease mechanisms, the strong impact of the environment, and the complexity of gene-environment interactions. Metabolomics is the quantification of small molecules produced by metabolic processes within a biological sample...
January 26, 2018: Human Genomics
https://www.readbyqxmd.com/read/29359319/subtype-classification-and-heterogeneous-prognosis-model-construction-in-precision-medicine
#7
Na You, Shun He, Xueqin Wang, Junxian Zhu, Heping Zhang
Common diseases including cancer are heterogeneous. It is important to discover disease subtypes and identify both shared and unique risk factors for different disease subtypes. The advent of high-throughput technologies enriches the data to achieve this goal, if necessary statistical methods are developed. Existing methods can accommodate both heterogeneity identification and variable selection under parametric models, but for survival analysis, the commonly used Cox model is semiparametric. Although finite-mixture Cox model has been proposed to address heterogeneity in survival analysis, variable selection has not been incorporated into such semiparametric models...
January 22, 2018: Biometrics
https://www.readbyqxmd.com/read/29352395/association-between-crp-genetic-diversity-and-bipolar-disorder-comorbid-complications
#8
Wahid Boukouaci, José Oliveira, Bruno Etain, Meriem Bennabi, Christina Mariaselvam, Nora Hamdani, Céline Manier, Djaouida Bengoufa, Frank Bellivier, Chantal Henry, Jean-Pierre Kahn, Dominique Charron, Rajagopal Krishnamoorthy, Marion Leboyer, Ryad Tamouza
BACKGROUND: Chronic low-grade inflammation is believed to contribute, at least in a subset of patients, to the development of bipolar disorder (BD). In this context, the most investigated biological marker is the acute phase response molecule, C-reactive protein (CRP). While the genetic diversity of CRP was amply studied in various pathological settings, little is known in BD. METHODS: 568 BD patients along with 163 healthy controls (HC) were genotyped for the following single-nucleotide polymorphisms (SNPs) on the CRP gene: intron rs1417938 (+ 29) T/A, 3'-UTR rs1130864 (+ 1444) G/A, and downstream rs1205 (+ 1846) (C/T)...
January 20, 2018: International Journal of Bipolar Disorders
https://www.readbyqxmd.com/read/29352257/robust-phenotype-prediction-from-gene-expression-data-using-differential-shrinkage-of-co-regulated-genes
#9
Kourosh Zarringhalam, David Degras, Christoph Brockel, Daniel Ziemek
Discovery of robust diagnostic or prognostic biomarkers is a key to optimizing therapeutic benefit for select patient cohorts - an idea commonly referred to as precision medicine. Most discovery studies to derive such markers from high-dimensional transcriptomics datasets are weakly powered with sample sizes in the tens of patients. Therefore, highly regularized statistical approaches are essential to making generalizable predictions. At the same time, prior knowledge-driven approaches have been successfully applied to the manual interpretation of high-dimensional transcriptomics datasets...
January 19, 2018: Scientific Reports
https://www.readbyqxmd.com/read/29352006/machine-learning-in-cardiovascular-medicine-are-we-there-yet
#10
REVIEW
Khader Shameer, Kipp W Johnson, Benjamin S Glicksberg, Joel T Dudley, Partho P Sengupta
Artificial intelligence (AI) broadly refers to analytical algorithms that iteratively learn from data, allowing computers to find hidden insights without being explicitly programmed where to look. These include a family of operations encompassing several terms like machine learning, cognitive learning, deep learning and reinforcement learning-based methods that can be used to integrate and interpret complex biomedical and healthcare data in scenarios where traditional statistical methods may not be able to perform...
January 19, 2018: Heart: Official Journal of the British Cardiac Society
https://www.readbyqxmd.com/read/29349792/meta-analysis-of-gaussian-individual-patient-data-two-stage-or-not-two-stage
#11
Tim P Morris, David J Fisher, Michael G Kenward, James R Carpenter
Quantitative evidence synthesis through meta-analysis is central to evidence-based medicine. For well-documented reasons, the meta-analysis of individual patient data is held in higher regard than aggregate data. With access to individual patient data, the analysis is not restricted to a "two-stage" approach (combining estimates and standard errors) but can estimate parameters of interest by fitting a single model to all of the data, a so-called "one-stage" analysis. There has been debate about the merits of one- and two-stage analysis...
January 18, 2018: Statistics in Medicine
https://www.readbyqxmd.com/read/29332819/chemometric-analysis-of-nmr-and-gc-datasets-for-chemotype-characterization-of-essential-oils-from-different-species-of-ocimum
#12
João Vito B Freitas, Elenilson G Alves Filho, Lorena Mara A Silva, Guilherme J Zocolo, Edy S de Brito, Nilce V Gramosa
The genus Ocimum (Labiatae) comprises 30 species found in tropical and subtropical regions of the planet, of which species O. basilicum L. and O. gratissimum are widely used in food and traditional medicine. Phytochemical studies on Ocimum have revealed a number of essential oil chemotypes, for example, eugenol, methyl chavicol, linalool, and methyl cinnamate. Since essential oils are commercially assessed according to their content, the aim of this study was to develop a simple and precise method for their qualitative and quantitative analysis using NMR spectroscopy combined with chemometrics...
April 1, 2018: Talanta
https://www.readbyqxmd.com/read/29311660/defining-phenotypes-in-diabetic-nephropathy-a-novel-approach-using-a-cross-sectional-analysis-of-a-single-centre-cohort
#13
Rosa M Montero, Athula Herath, Ashfaq Qureshi, Ehsanollah Esfandiari, Charles D Pusey, Andrew H Frankel, Frederick W K Tam
The global increase in Diabetes Mellitus (DM) has led to an increase in DM-Chronic Kidney Disease (DM-CKD). In this cross-sectional observational study we aimed to define phenotypes for patients with DM-CKD that in future may be used to individualise treatment We report 4 DM-CKD phenotypes in 220 patients recruited from Imperial College NHS Trust clinics from 2004-2012. A robust principal component analysis (PCA) was used to statistically determine clusters with phenotypically different patients. 163 patients with complete data sets were analysed: 77 with CKD and 86 with DM-CKD...
January 8, 2018: Scientific Reports
https://www.readbyqxmd.com/read/29260236/precision-medicine-for-cardiovascular-disease-learning-lessons-from-cardiomyopathies
#14
REVIEW
F Sedaghat-Hamedani, H A Katus, B Meder
Evidence-based medicine has considerably advanced the treatment of highly prevalent cardiovascular diseases. Its implementation was driven by multicenter interventional trials in treatment and placebo cohorts, propelling numerous biomedical innovations toward standard of care. While a uniform treatment can be effective in such disease cohorts ("one size fits all"), it neglects the genetic and phenotypic individuality of a single patient and his or her disease. Accordingly, a recent observation was made that several newer "mega" trials, demanding considerable resources for their execution, showed statistically significant differences in outcome, however, with small overall efficacies that render implementation in the clinics unlikely...
December 19, 2017: Herz
https://www.readbyqxmd.com/read/29251737/integrated-tumor-identification-and-automated-scoring-minimizes-pathologist-involvement-and-provides-new-insights-to-key-biomarkers-in-breast-cancer
#15
Peter Bankhead, José A Fernández, Darragh G McArt, David P Boyle, Gerald Li, Maurice B Loughrey, Gareth W Irwin, D Paul Harkin, Jacqueline A James, Stephen McQuaid, Manuel Salto-Tellez, Peter W Hamilton
Digital image analysis (DIA) is becoming central to the quantitative evaluation of tissue biomarkers for discovery, diagnosis and therapeutic selection for the delivery of precision medicine. In this study, automated DIA using a new purpose-built software platform (QuPath) is applied to a cohort of 293 breast cancer patients to score five biomarkers in tissue microarrays (TMAs): ER, PR, HER2, Ki67 and p53. This software is able to measure IHC expression following fully automated tumor recognition in the same immunohistochemical (IHC)-stained tissue section, as part of a rapid workflow to ensure objectivity and accelerate biomarker analysis...
December 18, 2017: Laboratory Investigation; a Journal of Technical Methods and Pathology
https://www.readbyqxmd.com/read/29250116/chimerism-in-myeloid-malignancies-following-stem-cell-transplantation-using-flubu4-with-and-without-busulfan-pharmacokinetics-versus-bucy
#16
Shatha Farhan, Michael Bazydlo, Klodiana Neme, Nancy Mikulandric, Edward Peres, Nalini Janakiraman
In the era of precision medicine, the impact of personalized dosing of busulfan is not clear. We undertook a retrospective analysis of 78 patients with myeloid malignancies who received fludarabine and busulfan (FluBu4) with or without measuring Bu pharmacokinetics (Bu PK) and those who received busulfan with cyclophosphamide (BuCy). Fifty-five patients received FluBu4, of whom 21 had Bu PK measured, and 23 patients received BuCy. Total donor cell chimerism showed that the percentage of patients maintaining 100% donor chimerism on day 100 was 66...
2017: Advances in Hematology
https://www.readbyqxmd.com/read/29226222/learning-processes-and-learning-problems-in-german-postgraduate-medical-education
#17
Hendrik van den Bussche, Lea Krause-Solberg, Martin Scherer, Stine Ziegler
Objective: In order to evaluate the quality of postgraduate medical education in Germany, we examined how the learning of theoretical and practical competencies is conceptualized and how the learning process takes place in real terms. The training conditions, as perceived by medical residents, are compared with the learning objectives, as stated by the Federal Chamber of Physicians in its regulation on postgraduate education. The analysis is based on the data of the "KarMed" study (abbreviation of "career progression and career breaks among physicians during postgraduate education"), a multicentre cohort study of medical school graduates from seven universities who arre followed until they receive their licenses as specialist physicians...
2017: GMS Journal for Medical Education
https://www.readbyqxmd.com/read/29224672/sle-redefined-on-the-basis-of-molecular-pathways
#18
REVIEW
Guillermo Barturen, Marta E Alarcón-Riquelme
The implementation of precision medicine requires the recruiting of patients in statistically enough numbers, the possibility of obtaining enough materials, and the integration of data from various platforms, which are all real limitations. These types of studies have been performed extensively in cancer but barely on systemic lupus erythematosus (SLE) or other rheumatic diseases. To consider the practical use of the information obtained from such studies, we have to take into account the best biological fluid to use, the ease to perform the analysis in clinical practice, and its relevance to clinical practice...
June 2017: Best Practice & Research. Clinical Rheumatology
https://www.readbyqxmd.com/read/29209617/mirroring-the-multiple-potentials-of-micrornas-in-acute-myocardial-infarction
#19
REVIEW
Solenne Paiva, Onnik Agbulut
At present, cardiovascular diseases are depicted to be the leading cause of death worldwide according to the World Health Organization. In the future, projections predict that ischemic heart disease will persist in the top main causes of illness. Within this alarming context, some tiny master regulators of gene expression programs, namely, microRNAs (miRNAs) carry three promising potentials. In fact, miRNAs can prove to be useful not only in terms of biomarkers allowing heart injury detection but also in terms of therapeutics to overcome limitations of past strategies and treat the lesions...
2017: Frontiers in Cardiovascular Medicine
https://www.readbyqxmd.com/read/29205435/relative-efficiency-of-precision-medicine-designs-for-clinical-trials-with-predictive-biomarkers
#20
Weichung Joe Shih, Yong Lin
Prospective randomized clinical trials addressing biomarkers are time consuming and costly, but are necessary for regulatory agencies to approve new therapies with predictive biomarkers. For this reason, recently, there have been many discussions and proposals of various trial designs and comparisons of their efficiency in the literature. We compare statistical efficiencies between the marker-stratified design and the marker-based precision medicine design regarding testing/estimating 4 hypotheses/parameters of clinical interest, namely, treatment effects in each marker-positive and marker-negative cohorts, marker-by-treatment interaction, and the marker's clinical utility...
December 4, 2017: Statistics in Medicine
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