Identifying treatment responders using counterfactual modeling and potential outcomes

2018 ◽  
Vol 28 (10-11) ◽  
pp. 3346-3362 ◽  
Author(s):  
Raphaël Porcher ◽  
Justine Jacot ◽  
Jay S Wunder ◽  
David J Biau
Keyword(s):  
Estimation Method ◽  
Treatment Strategies ◽  
Study Data ◽  
Potential Outcomes ◽  
Control Treatment ◽  
Individual Treatment ◽  
Right Censored Data ◽  
Monotonicity Assumption ◽  
Selection Of Patients ◽  
Selection Of

Individualizing treatment according to patients' characteristics is central for personalized or precision medicine. There has been considerable recent research in developing statistical methods to determine optimal personalized treatment strategies by modeling the outcome of patients according to relevant covariates under each of the alternative treatments, and then relying on so-called predicted individual treatment effects. In this paper, we use potential outcomes and principal stratification frameworks and develop a multinomial model for left and right-censored data to estimate the probability that a patient is a responder given a set of baseline covariates. The model can apply to RCT or observational study data. This method is based on the monotonicity assumption, which implies that no patients would respond to the control treatment but not to the experimental one. We conduct a simulation study to evaluate the properties of the proposed estimation method. Results showed that the predictions of the probability of being a responder were well calibrated even if we observed variability and a small bias when many parameters were estimated. We finally applied the method to a cohort study on the selection of patients for additional radiotherapy after resection of a soft-tissue sarcoma.

Abstract P542: Management and Outcome of Stroke Patients With Tandem Carotid Occlusion in the ESCAPE NA1-Trial

Stroke ◽  
2021 ◽  
Vol 52 (Suppl_1) ◽  
Author(s):  
Martha Marko ◽  
Petra Cimflova ◽  
Nishita Singh ◽  
Johanna Ospel ◽  
Nima Kashani ◽  
...  
Keyword(s):  
Treatment Strategies ◽  
Carotid Stenting ◽  
Carotid Occlusion ◽  
Catheter Angiography ◽  
Stroke Patients ◽  
Treatment Allocation ◽  
Tandem Occlusion ◽  
Stent Treatment ◽  
Selection Of Patients ◽  
Selection Of

Background: The optimal treatment for stroke patients with tandem cervical carotid occlusion is debated. We analyzed the treatment strategies and outcomes of tandem occlusion patients in the ESCAPE NA1 trial. Methods: ESCAPE NA1 was a multicenter international randomized trial of nerinetide vs. placebo in patients with acute ischemic stroke who underwent EVT. We defined tandem occlusions as complete occlusion of the cervical ICA on catheter angiography. The influence of tandem occlusions on outcome was analyzed using regression modeling with adjustment for age, sex, baseline NIHSS and ASPECTS, occlusion location, thrombolysis and treatment allocation. Results: 115 of 1105 patients (10.4%) had tandem occlusions. 73/115 tandem patients (66.0%) received treatment for the cervical occlusion: 21.9% were stented before thrombectomy, 68.5% were stented after thrombectomy, and 8.2% had angioplasty alone. Successful reperfusion was significantly higher in patients who had thrombectomy first followed by carotid treatment (eTICI 2b-3: 40/40 (100.0%)) or carotid angioplasty before and cervical stent after intracranial thrombectomy (9/10 (90.0%)) compared to carotid intervention before intracranial thrombectomy: (19/23 (82.1%), p=0.016). 90-day mRS 0-2 was achieved in 82/115 patients (71.3%) with tandem occlusions (treated occlusions: 74.0%, untreated: 66.7%) compared to 579/981 (59.5%) patients without tandem occlusions. In adjusted analysis, tandem occlusion was not predictive of outcome. In the subgroup of tandem patients, cervical stent-treatment was nominally associated with better outcomes (OR 2.2, 95% CI 0.5 - 9.2). Conclusion: Cervical carotid stenting may improve outcomes for EVT patients with tandem occlusions, but these results are limited by the sample size and non-randomized selection of patients for stenting.

Obesity - an epidemic of the twenty-first century: an update for psychiatrists

2005 ◽  
Vol 19 (6_suppl) ◽  
pp. 6-15 ◽  
Author(s):  
Richard I. G. Holt
Keyword(s):  
Premature Mortality ◽  
Obese Individual ◽  
First Century ◽  
Individual Treatment ◽  
Obesity Epidemic ◽  
Current Trends ◽  
Prevalence Of Obesity ◽  
Normal Body Weight ◽  
Selection Of Patients ◽  
Selection Of

Obesity is a chronic relapsing condition associated with significant morbidity and premature mortality. The prevalence of obesity has increased dramatically over the last 20 years and continues to do so, primarily as a result of changes in dietary intake and exercise patterns. There are considerable challenges associated with the management of the obesity epidemic involving both public health policies and individual treatment. Management of the obese individual involves lifelong lifestyle change for all, drugs for some, and surgery for a few. Appropriate selection of patients and the setting of realistic goals are crucial to the success of any weight-reducing programme. The aim of obesity management is to reduce associated morbidity and mortality, not necessarily to restore normal body weight. While the current trends in obesity are depressing, a better understanding of the pathophysiology and treatment of the condition should allow the clinician to be more optimistic for the future.

Selection of patients for portal-systemic shunts

JAMA ◽  
1966 ◽  
Vol 196 (12) ◽  
pp. 1039-1044 ◽  
Author(s):  
R. E. Hermann
Keyword(s):  
Selection Of Patients ◽  
Selection Of

Perioperative acute internal carotid thrombosis after general surgery

2020 ◽  
Vol 99 (3) ◽  
pp. 136-140
Keyword(s):  
Cardiac Surgery ◽  
Internal Carotid ◽  
Right Hemicolectomy ◽  
Diagnostic Assessment ◽  
Left Carotid Artery ◽  
Perioperative Stroke ◽  
Internal Carotid Occlusion ◽  
Acute Brain Ischemia ◽  
Selection Of Patients ◽  
Selection Of

Introduction: The average incidence of perioperative stroke during major non-cardiac surgery is less than 1%, suggesting that it is rarely a major problem for the vast majority of patients. Methods: In our paper we present a 46-year-old patient undergoing acute right hemicolectomy who developed right-sided hemiparesis in the perioperative setting. Immediate CTAg examination showed an ischemic stroke in the left hemisphere as a result of left internal carotid thrombosis. A surgical procedure to recanalize the left carotid artery was performed 14 hours from the onset of neurological symptomatology and the neurological deficit gradually recovered fully. Conclusion: Our case report supports studies showing that a thorough diagnostic assessment allows the selection of patients who may benefit from urgent revascularization of acute internal carotid occlusion during the phase of acute brain ischemia.

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