scholarly journals Assessing prognosis and prediction of treatment response in early rheumatoid arthritis: systematic reviews

2018 ◽  
Vol 22 (66) ◽  
pp. 1-294 ◽  
Author(s):  
Rachel Archer ◽  
Emma Hock ◽  
Jean Hamilton ◽  
John Stevens ◽  
Munira Essat ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Prediction Models ◽  
Meta Analysis ◽  
External Validation ◽  
Model Development ◽  
Predictive Performance ◽  
Assessment Tools ◽  
Clinical Prediction ◽  
Clinical Prediction Models ◽  
Early Ra

Background Rheumatoid arthritis (RA) is a chronic, debilitating disease associated with reduced quality of life and substantial costs. It is unclear which tests and assessment tools allow the best assessment of prognosis in people with early RA and whether or not variables predict the response of patients to different drug treatments. Objective To systematically review evidence on the use of selected tests and assessment tools in patients with early RA (1) in the evaluation of a prognosis (review 1) and (2) as predictive markers of treatment response (review 2). Data sources Electronic databases (e.g. MEDLINE, EMBASE, The Cochrane Library, Web of Science Conference Proceedings; searched to September 2016), registers, key websites, hand-searching of reference lists of included studies and key systematic reviews and contact with experts. Study selection Review 1 – primary studies on the development, external validation and impact of clinical prediction models for selected outcomes in adult early RA patients. Review 2 – primary studies on the interaction between selected baseline covariates and treatment (conventional and biological disease-modifying antirheumatic drugs) on salient outcomes in adult early RA patients. Results Review 1 – 22 model development studies and one combined model development/external validation study reporting 39 clinical prediction models were included. Five external validation studies evaluating eight clinical prediction models for radiographic joint damage were also included. c-statistics from internal validation ranged from 0.63 to 0.87 for radiographic progression (different definitions, six studies) and 0.78 to 0.82 for the Health Assessment Questionnaire (HAQ). Predictive performance in external validations varied considerably. Three models [(1) Active controlled Study of Patients receiving Infliximab for the treatment of Rheumatoid arthritis of Early onset (ASPIRE) C-reactive protein (ASPIRE CRP), (2) ASPIRE erythrocyte sedimentation rate (ASPIRE ESR) and (3) Behandelings Strategie (BeSt)] were externally validated using the same outcome definition in more than one population. Results of the random-effects meta-analysis suggested substantial uncertainty in the expected predictive performance of models in a new sample of patients. Review 2 – 12 studies were identified. Covariates examined included anti-citrullinated protein/peptide anti-body (ACPA) status, smoking status, erosions, rheumatoid factor status, C-reactive protein level, erythrocyte sedimentation rate, swollen joint count (SJC), body mass index and vascularity of synovium on power Doppler ultrasound (PDUS). Outcomes examined included erosions/radiographic progression, disease activity, physical function and Disease Activity Score-28 remission. There was statistical evidence to suggest that ACPA status, SJC and PDUS status at baseline may be treatment effect modifiers, but not necessarily that they are prognostic of response for all treatments. Most of the results were subject to considerable uncertainty and were not statistically significant. Limitations The meta-analysis in review 1 was limited by the availability of only a small number of external validation studies. Studies rarely investigated the interaction between predictors and treatment. Suggested research priorities Collaborative research (including the use of individual participant data) is needed to further develop and externally validate the clinical prediction models. The clinical prediction models should be validated with respect to individual treatments. Future assessments of treatment by covariate interactions should follow good statistical practice. Conclusions Review 1 – uncertainty remains over the optimal prediction model(s) for use in clinical practice. Review 2 – in general, there was insufficient evidence that the effect of treatment depended on baseline characteristics. Study registration This study is registered as PROSPERO CRD42016042402. Funding The National Institute for Health Research Health Technology Assessment programme.

Statistical Development and Validation of Clinical Prediction Models

2021 ◽  
Author(s):  
Steven J. Staffa ◽  
David Zurakowski
Keyword(s):  
Prediction Model ◽  
Prediction Models ◽  
External Validation ◽  
Model Development ◽  
Predictive Ability ◽  
Binary Outcome ◽  
Clinical Prediction ◽  
Clinical Prediction Models ◽  
Surgery Research ◽  
Development And Validation

Summary Clinical prediction models in anesthesia and surgery research have many clinical applications including preoperative risk stratification with implications for clinical utility in decision-making, resource utilization, and costs. It is imperative that predictive algorithms and multivariable models are validated in a suitable and comprehensive way in order to establish the robustness of the model in terms of accuracy, predictive ability, reliability, and generalizability. The purpose of this article is to educate anesthesia researchers at an introductory level on important statistical concepts involved with development and validation of multivariable prediction models for a binary outcome. Methods covered include assessments of discrimination and calibration through internal and external validation. An anesthesia research publication is examined to illustrate the process and presentation of multivariable prediction model development and validation for a binary outcome. Properly assessing the statistical and clinical validity of a multivariable prediction model is essential for reassuring the generalizability and reproducibility of the published tool.

scholarly journals External validation of clinical prediction models using big datasets from e-health records or IPD meta-analysis: opportunities and challenges

BMJ ◽  
2016 ◽  
pp. i3140 ◽  
Author(s):  
Richard D Riley ◽  
Joie Ensor ◽  
Kym I E Snell ◽  
Thomas P A Debray ◽  
Doug G Altman ◽  
...  
Keyword(s):  
Prediction Models ◽  
Meta Analysis ◽  
External Validation ◽  
Clinical Prediction ◽  
Clinical Prediction Models ◽  
Health Records

scholarly journals Developing clinical prediction models when adhering to minimum sample size recommendations: The importance of quantifying bootstrap variability in tuning parameters and predictive performance

2021 ◽  
pp. 096228022110463
Author(s):  
Glen P Martin ◽  
Richard D Riley ◽  
Gary S Collins ◽  
Matthew Sperrin
Keyword(s):  
Sample Size ◽  
Prediction Models ◽  
Model Development ◽  
Likelihood Estimation ◽  
Predictive Performance ◽  
Minimum Sample Size ◽  
Clinical Prediction ◽  
Clinical Prediction Models ◽  
Tuning Parameters ◽  
Minimum Sample

Recent minimum sample size formula (Riley et al.) for developing clinical prediction models help ensure that development datasets are of sufficient size to minimise overfitting. While these criteria are known to avoid excessive overfitting on average, the extent of variability in overfitting at recommended sample sizes is unknown. We investigated this through a simulation study and empirical example to develop logistic regression clinical prediction models using unpenalised maximum likelihood estimation, and various post-estimation shrinkage or penalisation methods. While the mean calibration slope was close to the ideal value of one for all methods, penalisation further reduced the level of overfitting, on average, compared to unpenalised methods. This came at the cost of higher variability in predictive performance for penalisation methods in external data. We recommend that penalisation methods are used in data that meet, or surpass, minimum sample size requirements to further mitigate overfitting, and that the variability in predictive performance and any tuning parameters should always be examined as part of the model development process, since this provides additional information over average (optimism-adjusted) performance alone. Lower variability would give reassurance that the developed clinical prediction model will perform well in new individuals from the same population as was used for model development.

P–419 On prognosis after unexplained recurrent pregnancy losses (RPL); a systematic review and external validation of clinical prediction models

2021 ◽  
Vol 36 (Supplement_1) ◽  
Author(s):  
A Youssef
Keyword(s):  
Systematic Review ◽  
Supportive Care ◽  
Prediction Model ◽  
Pregnancy Outcome ◽  
Prediction Models ◽  
External Validation ◽  
Model Development ◽  
Predictive Performance ◽  
Pregnancy Rates ◽  
Cochrane Library

Abstract Study question Which models that predict pregnancy outcome in couples with unexplained RPL exist and what is the performance of the most used model? Summary answer We identified seven prediction models; none followed the recommended prediction model development steps. Moreover, the most used model showed poor predictive performance. What is known already RPL remains unexplained in 50–75% of couples For these couples, there is no effective treatment option and clinical management rests on supportive care. Essential part of supportive care consists of counselling on the prognosis of subsequent pregnancies. Indeed, multiple prediction models exist, however the quality and validity of these models varies. In addition, the prediction model developed by Brigham et al is the most widely used model, but has never been externally validated. Study design, size, duration We performed a systematic review to identify prediction models for pregnancy outcome after unexplained RPL. In addition we performed an external validation of the Brigham model in a retrospective cohort, consisting of 668 couples with unexplained RPL that visited our RPL clinic between 2004 and 2019. Participants/materials, setting, methods A systematic search was performed in December 2020 in Pubmed, Embase, Web of Science and Cochrane library to identify relevant studies. Eligible studies were selected and assessed according to the TRIPOD) guidelines, covering topics on model performance and validation statement. The performance of predicting live birth in the Brigham model was evaluated through calibration and discrimination, in which the observed pregnancy rates were compared to the predicted pregnancy rates. Main results and the role of chance Seven models were compared and assessed according to the TRIPOD statement. This resulted in two studies of low, three of moderate and two of above average reporting quality. These studies did not follow the recommended steps for model development and did not calculate a sample size. Furthermore, the predictive performance of neither of these models was internally- or externally validated. We performed an external validation of Brigham model. Calibration showed overestimation of the model and too extreme predictions, with a negative calibration intercept of –0.52 (CI 95% –0.68 – –0.36), with a calibration slope of 0.39 (CI 95% 0.07 – 0.71). The discriminative ability of the model was very low with a concordance statistic of 0.55 (CI 95% 0.50 – 0.59). Limitations, reasons for caution None of the studies are specifically named prediction models, therefore models may have been missed in the selection process. The external validation cohort used a retrospective design, in which only the first pregnancy after intake was registered. Follow-up time was not limited, which is important in counselling unexplained RPL couples. Wider implications of the findings: Currently, there are no suitable models that predict on pregnancy outcome after RPL. Moreover, we are in need of a model with several variables such that prognosis is individualized, and factors from both the female as the male to enable a couple specific prognosis. Trial registration number Not applicable

scholarly journals A systematic review of the quality of clinical prediction models in in vitro fertilisation

2020 ◽  
Vol 35 (1) ◽  
pp. 100-116 ◽  
Author(s):  
M B Ratna ◽  
S Bhattacharya ◽  
B Abdulrahim ◽  
D J McLernon
Keyword(s):  
Systematic Review ◽  
Live Birth ◽  
Prediction Models ◽  
Conflicts Of Interest ◽  
External Validation ◽  
Post Treatment ◽  
Treatment Model ◽  
Clinical Prediction ◽  
Clinical Prediction Models

Abstract STUDY QUESTION What are the best-quality clinical prediction models in IVF (including ICSI) treatment to inform clinicians and their patients of their chance of success? SUMMARY ANSWER The review recommends the McLernon post-treatment model for predicting the cumulative chance of live birth over and up to six complete cycles of IVF. WHAT IS KNOWN ALREADY Prediction models in IVF have not found widespread use in routine clinical practice. This could be due to their limited predictive accuracy and clinical utility. A previous systematic review of IVF prediction models, published a decade ago and which has never been updated, did not assess the methodological quality of existing models nor provided recommendations for the best-quality models for use in clinical practice. STUDY DESIGN, SIZE, DURATION The electronic databases OVID MEDLINE, OVID EMBASE and Cochrane library were searched systematically for primary articles published from 1978 to January 2019 using search terms on the development and/or validation (internal and external) of models in predicting pregnancy or live birth. No language or any other restrictions were applied. PARTICIPANTS/MATERIALS, SETTING, METHODS The PRISMA flowchart was used for the inclusion of studies after screening. All studies reporting on the development and/or validation of IVF prediction models were included. Articles reporting on women who had any treatment elements involving donor eggs or sperm and surrogacy were excluded. The CHARMS checklist was used to extract and critically appraise the methodological quality of the included articles. We evaluated models’ performance by assessing their c-statistics and plots of calibration in studies and assessed correct reporting by calculating the percentage of the TRIPOD 22 checklist items met in each study. MAIN RESULTS AND THE ROLE OF CHANCE We identified 33 publications reporting on 35 prediction models. Seventeen articles had been published since the last systematic review. The quality of models has improved over time with regard to clinical relevance, methodological rigour and utility. The percentage of TRIPOD score for all included studies ranged from 29 to 95%, and the c-statistics of all externally validated studies ranged between 0.55 and 0.77. Most of the models predicted the chance of pregnancy/live birth for a single fresh cycle. Six models aimed to predict the chance of pregnancy/live birth per individual treatment cycle, and three predicted more clinically relevant outcomes such as cumulative pregnancy/live birth. The McLernon (pre- and post-treatment) models predict the cumulative chance of live birth over multiple complete cycles of IVF per woman where a complete cycle includes all fresh and frozen embryo transfers from the same episode of ovarian stimulation. McLernon models were developed using national UK data and had the highest TRIPOD score, and the post-treatment model performed best on external validation. LIMITATIONS, REASONS FOR CAUTION To assess the reporting quality of all included studies, we used the TRIPOD checklist, but many of the earlier IVF prediction models were developed and validated before the formal TRIPOD reporting was published in 2015. It should also be noted that two of the authors of this systematic review are authors of the McLernon model article. However, we feel we have conducted our review and made our recommendations using a fair and transparent systematic approach. WIDER IMPLICATIONS OF THE FINDINGS This study provides a comprehensive picture of the evolving quality of IVF prediction models. Clinicians should use the most appropriate model to suit their patients’ needs. We recommend the McLernon post-treatment model as a counselling tool to inform couples of their predicted chance of success over and up to six complete cycles. However, it requires further external validation to assess applicability in countries with different IVF practices and policies. STUDY FUNDING/COMPETING INTEREST(S) The study was funded by the Elphinstone Scholarship Scheme and the Assisted Reproduction Unit, University of Aberdeen. Both D.J.M. and S.B. are authors of the McLernon model article and S.B. is Editor in Chief of Human Reproduction Open. They have completed and submitted the ICMJE forms for Disclosure of potential Conflicts of Interest. The other co-authors have no conflicts of interest to declare. REGISTRATION NUMBER N/A

scholarly journals Fundamentals of Clinical Prediction Modeling for the Neurosurgeon

Neurosurgery ◽  
2019 ◽  
Vol 85 (3) ◽  
pp. 302-311 ◽  
Author(s):  
Hendrik-Jan Mijderwijk ◽  
Ewout W Steyerberg ◽  
Hans-Jakob Steiger ◽  
Igor Fischer ◽  
Marcel A Kamp
Keyword(s):  
Prediction Models ◽  
Model Development ◽  
Scientific Work ◽  
Evidence Based ◽  
Clinical Prediction ◽  
Prediction Modeling ◽  
Clinical Prediction Models ◽  
Prognostic Variables ◽  
Basic Understanding ◽  
Statistical Background

AbstractClinical prediction models in neurosurgery are increasingly reported. These models aim to provide an evidence-based approach to the estimation of the probability of a neurosurgical outcome by combining 2 or more prognostic variables. Model development and model reporting are often suboptimal. A basic understanding of the methodology of clinical prediction modeling is needed when interpreting these models. We address basic statistical background, 7 modeling steps, and requirements of these models such that they may fulfill their potential for major impact for our daily clinical practice and for future scientific work.

A framework for developing, implementing, and evaluating clinical prediction models in an individual participant data meta-analysis

2013 ◽  
Vol 32 (18) ◽  
pp. 3158-3180 ◽  
Author(s):  
Thomas P.A. Debray ◽  
Karel G.M. Moons ◽  
Ikhlaaq Ahmed ◽  
Hendrik Koffijberg ◽  
Richard David Riley
Keyword(s):  
Prediction Models ◽  
Meta Analysis ◽  
Individual Participant Data ◽  
Clinical Prediction ◽  
Clinical Prediction Models ◽  
Individual Participant

scholarly journals Machine learning-based clinical outcome prediction in surgery for acromegaly

Endocrine ◽  
2021 ◽  
Author(s):  
Olivier Zanier ◽  
Matteo Zoli ◽  
Victor E. Staartjes ◽  
Federica Guaraldi ◽  
Sofia Asioli ◽  
...  
Keyword(s):  
Machine Learning ◽  
Transsphenoidal Surgery ◽  
Prediction Models ◽  
External Validation ◽  
Clinical Prediction ◽  
Clinical Prediction Models ◽  
Total Resection ◽  
Prior Surgery ◽  
Using Data ◽  
Csf Leaks

Abstract Purpose Biochemical remission (BR), gross total resection (GTR), and intraoperative cerebrospinal fluid (CSF) leaks are important metrics in transsphenoidal surgery for acromegaly, and prediction of their likelihood using machine learning would be clinically advantageous. We aim to develop and externally validate clinical prediction models for outcomes after transsphenoidal surgery for acromegaly. Methods Using data from two registries, we develop and externally validate machine learning models for GTR, BR, and CSF leaks after endoscopic transsphenoidal surgery in acromegalic patients. For the model development a registry from Bologna, Italy was used. External validation was then performed using data from Zurich, Switzerland. Gender, age, prior surgery, as well as Hardy and Knosp classification were used as input features. Discrimination and calibration metrics were assessed. Results The derivation cohort consisted of 307 patients (43.3% male; mean [SD] age, 47.2 [12.7] years). GTR was achieved in 226 (73.6%) and BR in 245 (79.8%) patients. In the external validation cohort with 46 patients, 31 (75.6%) achieved GTR and 31 (77.5%) achieved BR. Area under the curve (AUC) at external validation was 0.75 (95% confidence interval: 0.59–0.88) for GTR, 0.63 (0.40–0.82) for BR, as well as 0.77 (0.62–0.91) for intraoperative CSF leaks. While prior surgery was the most important variable for prediction of GTR, age, and Hardy grading contributed most to the predictions of BR and CSF leaks, respectively. Conclusions Gross total resection, biochemical remission, and CSF leaks remain hard to predict, but machine learning offers potential in helping to tailor surgical therapy. We demonstrate the feasibility of developing and externally validating clinical prediction models for these outcomes after surgery for acromegaly and lay the groundwork for development of a multicenter model with more robust generalization.

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