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New statistical methods to compare the effectiveness of adaptive treatment plans
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Year: 2020 Publisher: Washington, D.C. : Patient-Centered Outcomes Research Institute (PCORI),

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BACKGROUND: During routine clinical care, treatments are adaptive to patients' responses to previous treatment assignments. However, methods for comparative effectiveness research (CER) are predominately designed for nonadaptive treatments. This project aimed to evaluate the comparative effectiveness of patient-centered adaptive treatment strategies (PCATS) at the initiation of treatment and over the course of the disease progression. As a case in point, despite many medication options, polyarticular-course juvenile idiopathic arthritis (pcJIA) is often refractory and requires better adaptive treatment strategies (ATS). OBJECTIVES: Aim 1. To develop, refine, and disseminate bayesian causal inference methods for evaluating clinical effectiveness and for informing better PCATS. Aim 2. To evaluate the clinical effectiveness of the recommended ATS for patients with pcJIA using real-world data. METHODS: We propose the GPMatch method, a nonparametric full bayesian doubly robust causal inference method that uses Gaussian process (GP) prior as a matching tool. We performed simulation studies to evaluate its performance compared with that of some widely used causal inference methods: propensity score subclassification, augmented inverse treatment probability weighting, regression adjustment, and bayesian additive regression trees (BART), under dual-misspecification settings. We extended both GPMatch and BART methods for ATS and applied them to electronic medical record (EMR) data to compare 2 consensus treatment plans (CTPs) that began with a disease-modifying antirheumatic drug (DMARD) at different times in treating children with pcJIA: the early-combination plan uses biologic and nonbiologic DMARDs (b+nbDMARD) soon after diagnosis, while the step-up plan starts with an nbDMARD first and then introduces bDMARDs later. The primary end points were Clinical Juvenile Arthritis Disease Activity Score (cJADAS10, with a cutoff at 10 for active joint count) results at 6 and 12 months, and the secondary end point was the Pediatric Quality of Life Inventory (PedsQL) score at 12 months. RESULTS: Simulation studies demonstrated that GPMatch, followed by BART, performed as well as or better than some commonly used non-bayesian causal inference methods for comparing both nonadaptive treatment strategies and ATS, as measured by the root mean square error (RMSE) and median absolute error (MAE). The pcJIA CER suggests that by 6 months, the early-combination plan reduced disease activity on average by 2.0 points (95% CI, 0.4-3.6 points) more than the step-up plan as measured by the cJADAS10. By 12 months, the early-combination plan remained more effective than the step-up plan: The average improvement in cJADAS10 was 2.6 points (95% CI, 0.6-4.6 points) if the first-line treatment was continued or reduced and 2.2 points (95% CI, 0.3-4.14 points) if the treatment was escalated. Both CTPs were effective in improving the PedsQL score by 12 months, reporting improvements of 74.8 (+/-) 2.0 and 80.4 (+/-) 3.7 points for the step-up and early-combination CTPs, respectively. If treated on the early-combination plan, patients were expected to achieve an average of 5.61 (95% CI, −3.89 to 15.12) more points on the PedsQL than were patients treated on the step-up plan. CONCLUSIONS: The GPMatch method accomplishes matching and flexible modeling in the same step and has well-calibrated frequentist properties. It is doubly robust in the sense that the average treatment effects are correctly estimated when either of the following conditions is satisfied: (1) The GP mean function correctly specifies the potential outcome model; and (2) the covariance function correctly specifies the matching structure. The pcJIA CER study suggests that the early-combination plan is more effective in reducing disease activity 1 year later. We developed a user-friendly graphic interface online R Shiny application, "PCATS," which is easy to use, making GPMatch and BART methods accessible to general CER investigators. LIMITATIONS: The GPMatch method is computationally intensive and not yet extended to nonnormally distributed outcomes. The PCATS online app assumes no missing data and single time-dependent confounding. Missing data are an inherent feature of EMR data, and our CER study addressed missingness at the design, data management, and data analysis steps. Nevertheless, the study results may be limited by the missing data handling procedures. We assume that the EMR captures important treatment considerations from the clinician's perspective, but not from the patient's perspective. Sensitivity analyses were performed to account for missing potential confounders from the patient's perspective. Finally, the CER study only analyzed 2 of the 3 CTPs.


Book
New statistical methods to compare the effectiveness of adaptive treatment plans
Author:
Year: 2020 Publisher: Washington, D.C. : Patient-Centered Outcomes Research Institute (PCORI),

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Abstract

BACKGROUND: During routine clinical care, treatments are adaptive to patients' responses to previous treatment assignments. However, methods for comparative effectiveness research (CER) are predominately designed for nonadaptive treatments. This project aimed to evaluate the comparative effectiveness of patient-centered adaptive treatment strategies (PCATS) at the initiation of treatment and over the course of the disease progression. As a case in point, despite many medication options, polyarticular-course juvenile idiopathic arthritis (pcJIA) is often refractory and requires better adaptive treatment strategies (ATS). OBJECTIVES: Aim 1. To develop, refine, and disseminate bayesian causal inference methods for evaluating clinical effectiveness and for informing better PCATS. Aim 2. To evaluate the clinical effectiveness of the recommended ATS for patients with pcJIA using real-world data. METHODS: We propose the GPMatch method, a nonparametric full bayesian doubly robust causal inference method that uses Gaussian process (GP) prior as a matching tool. We performed simulation studies to evaluate its performance compared with that of some widely used causal inference methods: propensity score subclassification, augmented inverse treatment probability weighting, regression adjustment, and bayesian additive regression trees (BART), under dual-misspecification settings. We extended both GPMatch and BART methods for ATS and applied them to electronic medical record (EMR) data to compare 2 consensus treatment plans (CTPs) that began with a disease-modifying antirheumatic drug (DMARD) at different times in treating children with pcJIA: the early-combination plan uses biologic and nonbiologic DMARDs (b+nbDMARD) soon after diagnosis, while the step-up plan starts with an nbDMARD first and then introduces bDMARDs later. The primary end points were Clinical Juvenile Arthritis Disease Activity Score (cJADAS10, with a cutoff at 10 for active joint count) results at 6 and 12 months, and the secondary end point was the Pediatric Quality of Life Inventory (PedsQL) score at 12 months. RESULTS: Simulation studies demonstrated that GPMatch, followed by BART, performed as well as or better than some commonly used non-bayesian causal inference methods for comparing both nonadaptive treatment strategies and ATS, as measured by the root mean square error (RMSE) and median absolute error (MAE). The pcJIA CER suggests that by 6 months, the early-combination plan reduced disease activity on average by 2.0 points (95% CI, 0.4-3.6 points) more than the step-up plan as measured by the cJADAS10. By 12 months, the early-combination plan remained more effective than the step-up plan: The average improvement in cJADAS10 was 2.6 points (95% CI, 0.6-4.6 points) if the first-line treatment was continued or reduced and 2.2 points (95% CI, 0.3-4.14 points) if the treatment was escalated. Both CTPs were effective in improving the PedsQL score by 12 months, reporting improvements of 74.8 (+/-) 2.0 and 80.4 (+/-) 3.7 points for the step-up and early-combination CTPs, respectively. If treated on the early-combination plan, patients were expected to achieve an average of 5.61 (95% CI, −3.89 to 15.12) more points on the PedsQL than were patients treated on the step-up plan. CONCLUSIONS: The GPMatch method accomplishes matching and flexible modeling in the same step and has well-calibrated frequentist properties. It is doubly robust in the sense that the average treatment effects are correctly estimated when either of the following conditions is satisfied: (1) The GP mean function correctly specifies the potential outcome model; and (2) the covariance function correctly specifies the matching structure. The pcJIA CER study suggests that the early-combination plan is more effective in reducing disease activity 1 year later. We developed a user-friendly graphic interface online R Shiny application, "PCATS," which is easy to use, making GPMatch and BART methods accessible to general CER investigators. LIMITATIONS: The GPMatch method is computationally intensive and not yet extended to nonnormally distributed outcomes. The PCATS online app assumes no missing data and single time-dependent confounding. Missing data are an inherent feature of EMR data, and our CER study addressed missingness at the design, data management, and data analysis steps. Nevertheless, the study results may be limited by the missing data handling procedures. We assume that the EMR captures important treatment considerations from the clinician's perspective, but not from the patient's perspective. Sensitivity analyses were performed to account for missing potential confounders from the patient's perspective. Finally, the CER study only analyzed 2 of the 3 CTPs.


Book
Pharmacotherapy casebook : a patient-focused approach
Authors: --- --- --- ---
ISBN: 9781260116670 1260116670 Year: 2020 Publisher: New York : McGraw-Hill,

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Dissertation
Les maisons médicales et la capitation : une voie pour l'efficience?
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Year: 2011 Publisher: [S.l.]: [chez l'auteur],

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Periodical
Journal of Participatory Medicine
ISSN: 21527202

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Book
Associations en médecine générale : facilitation à l'association et aide à la création d'une maison médicale
Authors: ---
Year: 2010 Publisher: Bruxelles: UCL,

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Ce travail de fin d’études est consacré au développement des associations en médecine générale. Il montre comment les évolutions sociologiques et démographiques de notre société rendent nécessaire l’adaptation de la première ligne de soins. La pratique de groupe est un des éléments de réponse à ces changements. II existe une série d’aides pour soutenir l’émergence des ces nouvelles pratiques. Ce travail se penche plus précisément sur les aides au développement d’une structure de type Maison Médicale. H explore aussi le principe de la facilitation à l’association en médecine générale qui est encore en gestation du côté francophone du pays.


Book
Pharmacotherapy casebook : a patient-focused approach
Authors: --- --- --- ---
ISBN: 9781259640919 1259640914 Year: 2017 Publisher: New York : McGraw-Hill Education,

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Book
The Hologram : feminist, peer-to-peer health for a post-pandemic future
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ISBN: 0745343236 0745343333 9780745343334 9780745343235 9780745343242 0745343244 9780745343327 0745343325 Year: 2020 Publisher: London, England : Pluto Press,

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In an era when capitalism leaves so many to suffer and to die, with neoliberal 'self-care' offering little more than a bandaid, how can we take health and care back into our hands? In The Hologram, Cassie Thornton puts forward a bold vision for revolutionary care: a viral, peer-to-peer feminist health network. The premise is simple: three people - a 'triangle' - meet on a regular basis, digitally or in person, to focus on the physical, mental and social health of a fourth - the 'hologram'. The hologram, in turn, teaches their caregivers how to give and also receive care; each member of their triangle becomes a hologram for another, different triangle, and so the system expands. Drawing on radical models developed in the Greek solidarity clinics during a decade of crisis, and directly engaging with discussions around mutual aid and the coronavirus pandemic, The Hologram develops the skills and relationships we desperately need for the anti-capitalist struggles of the present, and the post-capitalist society of the future. One part art, one part activism, one part science fiction, this book offers the reader a guide to establishing a Hologram network as well as reflections on this cooperative work in progress.


Book
L'éthique en chemin : démarche et créativité pour les soignants
Authors: --- --- ---
ISBN: 2747566315 9782747566315 Year: 2004 Publisher: Paris: L'Harmattan,

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Chaque être humain porte en lui un espace secret, un jardin intérieur, un lieu d'intimité, de dialogue avec soi-même. Ce lieu de profonde solitude est aussi celui d'un subtil partage quand, à travers la rencontre de l'autre, s'éprouve ce qui a pris forme au creux de l'être. C'est un lieu d'errance parfois. Un lieu de reliance aussi. Un lieu de résonance encore. Lieu commun entre tous, lieu sacré sans doute, il laisse émerger l'éthique à la fois comme mise en question et comme source de créativité. Les soignants, comme tous les autres, le soupçonnent, le découvrent, le délaissent, y retournent… A partir de leur expérience de confrontation avec la maladie, la souffrance, la vieillesse, la mort, ils demandent de plus en plus souvent d'être accompagnés pour y accueillir leurs doutes et leurs questions, pour en amplifier l'écho, pour en dessiner le sens. Ils expriment le besoin d'être soutenus dans leur réflexion, leurs prises de décision, leur recherche d'une attitude éthique au quotidien. Ce livre est là pour eux et pour tous ceux qui se sentent une âme de soignant ! Il est simplement né du désir de leur partager quelques repères, de leur proposer l'une ou l'autre balise, de leur donner envie de se mettre en route pour découvrir de nouveaux paysages. Invitation à laisser ouvert l'espace du questionnement, à toujours travailler avec un regard de débutant, à sans cesse cultiver les possibilités de transformation, il veut garder vif le souci éthique de chacun !


Book
WHO recommendations on antenatal care for a positive pregnancy experience
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ISBN: 9241549912 Year: 2016 Publisher: Geneva : World Health Organization,

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Within the continuum of reproductive health care, antenatal care (ANC) provides a platform for important healthcare functions, including health promotion, screening and diagnosis, and disease prevention. It has been established that, by implementing timely and appropriate evidence-based practices, ANC can save lives. Endorsed, by the UN Secretary-General, this is a comprehensive WHO guideline on routine ANC for pregnant women and adolescent girls. It aims to complement existing WHO guidelines on the management of specific pregnancy-related complications. The guidance aims to capture the complex nature of the issues surrounding ANC health care practices and delivery, and to prioritize person-centred health and well-being, not only the prevention of death and morbidity, in accordance with a human rights-based approach.

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