scholarly journals Estimating and evaluating personalized treatment recommendations from randomized trials with ptr

2021 ◽  
Vol 21 (2) ◽  
pp. 348-359
Author(s):  
Matthias Pierce ◽  
Richard Emsley
Keyword(s):  
Randomized Trials ◽  
Patient Characteristics ◽  
Treatment Recommendations ◽  
Trial Data ◽  
Personalized Treatment ◽  
Treatment Recommendation ◽  
Bootstrap Resampling ◽  
Recommendation Algorithm ◽  
Continuous Outcome ◽  
Alternative Regime

One of the targets of personalized medicine is to provide treatment recommendations using patient characteristics. We present the command ptr, which both predicts a personalized treatment recommendation algorithm and evaluates its effectiveness versus an alternative regime, using randomized trial data. The command allows for multiple (continuous or categorical) biomarkers and a binary or continuous outcome. Confidence intervals for the evaluation parameter are provided using bootstrap resampling.

scholarly journals Predicting Therapy Success and Costs for Personalized Treatment Recommendations Using Baseline Characteristics: Data-Driven Analysis (Preprint)

2018 ◽  
Author(s):  
Vincent Bremer ◽  
Dennis Becker ◽  
Spyros Kolovos ◽  
Burkhardt Funk ◽  
Ward van Breda ◽  
...  
Keyword(s):  
Decision Making ◽  
Cost Effectiveness ◽  
Incremental Cost ◽  
Treatment Recommendations ◽  
Prediction Performance ◽  
Personalized Treatment ◽  
Machine Learning Techniques ◽  
Treatment Recommendation ◽  
Individual Treatment ◽  
Treatment Type

BACKGROUND Different treatment alternatives exist for psychological disorders. Both clinical and cost effectiveness of treatment are crucial aspects for policy makers, therapists, and patients and thus play major roles for healthcare decision-making. At the start of an intervention, it is often not clear which specific individuals benefit most from a particular intervention alternative or how costs will be distributed on an individual patient level. OBJECTIVE This study aimed at predicting the individual outcome and costs for patients before the start of an internet-based intervention. Based on these predictions, individualized treatment recommendations can be provided. Thus, we expand the discussion of personalized treatment recommendation. METHODS Outcomes and costs were predicted based on baseline data of 350 patients from a two-arm randomized controlled trial that compared treatment as usual and blended therapy for depressive disorders. For this purpose, we evaluated various machine learning techniques, compared the predictive accuracy of these techniques, and revealed features that contributed most to the prediction performance. We then combined these predictions and utilized an incremental cost-effectiveness ratio in order to derive individual treatment recommendations before the start of treatment. RESULTS Predicting clinical outcomes and costs is a challenging task that comes with high uncertainty when only utilizing baseline information. However, we were able to generate predictions that were more accurate than a predefined reference measure in the shape of mean outcome and cost values. Questionnaires that include anxiety or depression items and questions regarding the mobility of individuals and their energy levels contributed to the prediction performance. We then described how patients can be individually allocated to the most appropriate treatment type. For an incremental cost-effectiveness threshold of 25,000 €/quality-adjusted life year, we demonstrated that our recommendations would have led to slightly worse outcomes (1.98%), but with decreased cost (5.42%). CONCLUSIONS Our results indicate that it was feasible to provide personalized treatment recommendations at baseline and thus allocate patients to the most beneficial treatment type. This could potentially lead to improved decision-making, better outcomes for individuals, and reduced health care costs.

scholarly journals Education, Implementation, and Teams: 2020 International Consensus on Cardiopulmonary Resuscitation and Emergency Cardiovascular Care Science With Treatment Recommendations

Circulation ◽  
2020 ◽  
Vol 142 (16_suppl_1) ◽  
Author(s):  
Robert Greif ◽  
Farhan Bhanji ◽  
Blair L. Bigham ◽  
Janet Bray ◽  
Jan Breckwoldt ◽  
...  
Keyword(s):  
Cardiopulmonary Resuscitation ◽  
Performance Feedback ◽  
Life Support ◽  
Task Force ◽  
Treatment Recommendations ◽  
Treatment Recommendation ◽  
Opioid Overdose ◽  
International Consensus ◽  
Scoping Reviews ◽  
Cardiovascular Care

For this 2020 International Consensus on Cardiopulmonary Resuscitation and Emergency Cardiovascular Care Science With Treatment Recommendations , the Education, Implementation, and Teams Task Force applied the population, intervention, comparator, outcome, study design, time frame format and performed 15 systematic reviews, applying the Grading of Recommendations, Assessment, Development, and Evaluation guidance. Furthermore, 4 scoping reviews and 7 evidence updates assessed any new evidence to determine if a change in any existing treatment recommendation was required. The topics covered included training for the treatment of opioid overdose; basic life support, including automated external defibrillator training; measuring implementation and performance in communities, and cardiac arrest centers; advanced life support training, including team and leadership training and rapid response teams; measuring cardiopulmonary resuscitation performance, feedback devices, and debriefing; and the use of social media to improve cardiopulmonary resuscitation application.

Application of RSClin to guide treatment recommendations for premenopausal patients with early-stage hormone-positive breast cancer and intermediate risk oncotype recurrence scores.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e12511-e12511
Author(s):  
Brittney Shulman Zimmerman ◽  
Shana Berwick ◽  
Alaina J Kessler ◽  
Danielle Seidman ◽  
Sara Malin Hovstadius ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Early Stage ◽  
Medical Center ◽  
Academic Medical Center ◽  
Recurrence Score ◽  
Treatment Recommendations ◽  
Definitive Treatment ◽  
Treatment Recommendation ◽  
Intermediate Risk ◽  
Premenopausal Patients

e12511 Background: The RSClin model, which incorporates the Oncotype Recurrence Score (RS) and clinicopathologic features, was recently developed to further tailor prognosis and prediction of chemotherapy benefit for patients with early-stage hormone positive (HR+) breast cancer (BC) (Sparano et al, 2020). The RSClin calculator is available online to assist treatment planning for situations where chemotherapy benefit is uncertain. Covariates include Oncotype RS, tumor grade, tumor size and patient age. The risk calculator generates a 10-year distant recurrence risk and absolute chemotherapy benefit. This tool may be especially helpful to determine treatment management for premenopausal patients with early-stage HR+ BC with intermediate risk (IR) Oncotype RS (16-25). We retrospectively applied RSClin to this patient population to determine if it would have changed treatment recommendations. Methods: We identified premenopausal women with node-negative early-stage BC with IR RS (16-25) within our large Oncotype database. Using the RSClin model, we selected >5% absolute chemotherapy benefit as a reasonable cutoff to recommend chemotherapy. We compared the treatment recommendation based on RSClin with the treatment previously recommended by breast oncologists at our large academic medical center in New York City. Results: There were 86 patients who met criteria with a median age of 46 years. Of these, 26 patients (30%) were recommended chemotherapy plus endocrine therapy (ET) and 60 (70%) were recommended ET alone. After applying the RSClin model (data available for 83/86 patients), 19 (23%) would have resulted in a change in treatment recommendation and 64 (77%) would have remained unchanged. Overall, 8 (10%) would have withheld chemotherapy when it was previously offered and 11 (13%) would have recommended chemotherapy when it was previously excluded. There were 8 (9%) secondary invasive breast events in this population, with 2 (2%) being ipsilateral, 3 (3%) being contralateral and 3 (3%) metastatic at a median follow up of 46.9 months. Conclusions: The RSClin model would have changed management of premenopausal patients with IR RS in 23% of patients. This model, although not yet prospectively validated, may help individualize therapy for patients with less definitive treatment plans. Using RSClin, we can aim to minimize recurrence rates and avoid unnecessary chemotherapy in selected patients. This model is easy to apply and will have important clinical utility moving forward.

Initiating anticoagulant therapy after ICH is associated with patient characteristics and treatment recommendations

2018 ◽  
Vol 265 (10) ◽  
pp. 2404-2414 ◽  
Author(s):  
Jochen A. Sembill ◽  
Claudia Y. Wieser ◽  
Maximilian I. Sprügel ◽  
Stefan T. Gerner ◽  
Antje Giede-Jeppe ◽  
...  
Keyword(s):  
Anticoagulant Therapy ◽  
Patient Characteristics ◽  
Treatment Recommendations

Abstract P214: Patient and Physician Factors Influence Decision-Making in Hypercholesterolemia

Circulation ◽  
2015 ◽  
Vol 131 (suppl_1) ◽  
Author(s):  
Michel Krempf ◽  
Ross J Simpson ◽  
Dena R Ramey ◽  
Philippe Brudi ◽  
Hilde Giezek ◽  
...  
Keyword(s):  
Decision Making ◽  
Goal Attainment ◽  
Clinical Decision Making ◽  
Treatment Strategies ◽  
Treatment Decision ◽  
Clinical Decision ◽  
Treatment Recommendations ◽  
Treatment Choice ◽  
Treatment Recommendation ◽  
Double Blind

Objectives: Little is known about how patient factors influence physicians’ treatment decision-making in hypercholesterolemia. We surveyed physicians’ treatment recommendations in high-risk patients with LDL-C not controlled on statin monotherapy. Methods: Physicians completed a questionnaire pre-randomization for each patient in a double-blind trial (NCT01154036) assessing LDL-C goal attainment rates with different treatment strategies. Patients had LDL-C ≥100 mg/dL after 5 weeks’ atorvastatin 10 mg/day and before randomization. Physicians were asked about treatment recommendations for three scenarios: (1) LDL-C near goal (100-105 mg/dL), (2) LDL-C far from goal (120 mg/dL), then (3) known baseline LDL-C of enrolled patients on atorvastatin 10 mg/day. Factors considered in their choice were specified. Physicians had been informed of projected LDL-C reductions for each treatment strategy in the trial. Regression analysis identified prognostic factors associated with each scenario, and projected LDL-C values for physicians’ treatment choices were compared to actual LDL-C values achieved in the trial. Results: Physicians at 296 sites completed questionnaires for 1535 patients. The most common treatment strategies for all three scenarios were: 1) not to change therapy, 2) double atorvastatin dose, 3) add ezetimibe, 4) double atorvastatin dose and add ezetimibe. Doubling atorvastatin dose was the most common treatment recommendation in all scenarios (43-52% of patients). ‘No change in therapy’ was recommended in 6.5% of patients when LDL-C was assumed far from goal. Treatment recommendations were more aggressive if actual LDL-C was known or considered far from goal. When compared with the ‘no change in therapy’ recommendation, CV risk factors and desire to achieve a more aggressive LDL-C goal were generally considered in decision-making for each treatment choice, regardless of LDL-C scenario. Patients randomized to a more aggressive regimen than recommended by physicians had larger reductions in LDL-C: the actual reduction in LDL-C in patients randomized to ‘add ezetimibe’ was -20.8% versus a projected reduction of -10.0% when physicians recommended ‘doubling atorvastatin dose’. Conclusions: This study provides insight into physicians’ perspectives on clinical management of hypercholesterolemia and highlights a gap in knowledge translation from guidelines to clinical practice. Targeting lower LDL-C and CV risk were key drivers in clinical decision-making but, generally, physicians were more conservative in their treatment choice than guidelines recommend, which may result in poorer LDL-C reduction. When compared with actual outcomes, projected LDL-C control was better if physicians used more comprehensive strategies rather than simply doubling the statin dose.

Abstract 2399: Low Hospital Mortality Of Acute Myocardial Infarction In Cslinical Practice Due To Optimized Guideline Oriented Treatment: Results Of The Optami-registry

Circulation ◽  
2007 ◽  
Vol 116 (suppl_16) ◽  
Author(s):  
Anselm K Gitt ◽  
Harm Wienbergen ◽  
Uwe Zeymer ◽  
Frank Towae ◽  
Martin G Gottwik ◽  
...  
Keyword(s):  
Myocardial Infarction ◽  
Acute Myocardial Infarction ◽  
Clinical Practice ◽  
Hospital Mortality ◽  
Randomized Trials ◽  
Patient Characteristics ◽  
High Rate ◽  
Internal Quality ◽  
Acute Therapy ◽  
Early Invasive Strategy

Background: Hospital mortality of STEMI in recent randomized trials as ASSENT IV ranges between 3.5 and 6.0%. Although registry data have shown a constant improvement of myocardial infarction outcome over the past years due to better implementation of guidelines for the management of acute myocardial infarction, hospital mortality in clinical practice still was much higher than in the selected patient population of randomized trials. Can ongoing registries in clinical practice as quality assurance programmes further reduce hospital mortality of acute myocardial infarction? Methods: The OPTAMI Register (Optimized Therapy of Acute Myocardial Infarction) enrols consecutive patients with STEMI or NSTEMI in 33 Centres (27 with cathlab facilities) in Germany to document patient characteristics, acute therapy as well as hospital outcome. All centres are provided benchmark reports for internal quality control. Results: Out of 1139 enrolled patients, 629 (55%) presented with STEMI and 510 (45%) with NSTEMI. Patients with NSTEMI were older, more often female and had a significantly higher prevalence of relevant comorbidities. OPTAMI documented an extraordinary high rate of primary PCI in STEMI as well as a high rate of early invasive strategy with PCI <48h in NSTEMI. In both groups, adherence to guidelines for the acute adjunctive medical treatment including antiplatelet therapy, betablockers, ACE-inhibitors and statins was higher than ever documented in any German MI registry. Hospital mortality was 4.0% in consecutive patients with STEMI and 3.9% in consecutive patients with NSTEMI. Conclusion: Preliminary data of the ongoing OPTAMI Registry demonstrate that in selected cnetres (mainly with cath lab facilities) hospital mortality in clinical practice can be reduced to levels of randomised controlled trials by adherence to practice guidelines for the management of acute myocardial infarctions.

scholarly journals Pediatric Life Support: 2020 International Consensus on Cardiopulmonary Resuscitation and Emergency Cardiovascular Care Science With Treatment Recommendations

Circulation ◽  
2020 ◽  
Vol 142 (16_suppl_1) ◽  
Author(s):  
Ian K. Maconochie ◽  
Richard Aickin ◽  
Mary Fran Hazinski ◽  
Dianne L. Atkins ◽  
Robert Bingham ◽  
...  
Keyword(s):  
Cardiopulmonary Resuscitation ◽  
Systematic Reviews ◽  
Life Support ◽  
Treatment Recommendations ◽  
Infants And Children ◽  
Treatment Recommendation ◽  
International Consensus ◽  
Epinephrine Administration ◽  
Evidence Evaluation ◽  
Cardiovascular Care

This 2020 International Consensus on Cardiopulmonary Resuscitation and Emergency Cardiovascular Care Science With Treatment Recommendations (CoSTR) for pediatric life support is based on the most extensive evidence evaluation ever performed by the Pediatric Life Support Task Force. Three types of evidence evaluation were used in this review: systematic reviews, scoping reviews, and evidence updates. Per agreement with the evidence evaluation recommendations of the International Liaison Committee on Resuscitation, only systematic reviews could result in a new or revised treatment recommendation. Systematic reviews performed for this 2020 CoSTR for pediatric life support included the topics of sequencing of airway-breaths-compressions versus compressions-airway-breaths in the delivery of pediatric basic life support, the initial timing and dose intervals for epinephrine administration during resuscitation, and the targets for oxygen and carbon dioxide levels in pediatric patients after return of spontaneous circulation. The most controversial topics included the initial timing and dose intervals of epinephrine administration (new treatment recommendations were made) and the administration of fluid for infants and children with septic shock (this latter topic was evaluated by evidence update). All evidence reviews identified the paucity of pediatric data and the need for more research involving resuscitation of infants and children.

scholarly journals Today's Adjuvant Therapy in Breast Cancer: Who Should Receive What?

1995 ◽  
Vol 2 (3) ◽  
pp. 107327489500200
Author(s):  
Anne E. Roberge ◽  
John K. Erban
Keyword(s):  
Public Health ◽  
Breast Cancer ◽  
Randomized Trials ◽  
Public Health Problem ◽  
Treatment Recommendations ◽  
Current Therapy ◽  
Major Public Health Problem ◽  
Breast Cancer Patients ◽  
Treatment Plans ◽  
Available Information

Breast cancer remains a major public health problem. Prospective randomized trials comparing therapies are essential to improve current therapy but, for those women unable or unwilling to participate in clinical trials, treatment plans are needed. Treatment recommendations can be based on available information from individual trials and from a large collaborative overview. This information will be reviewed with treatment recommendations presented for subpopulations of breast cancer patients.

Sign in / Sign up

Export Citation Format

Share Document