Analysis of randomized comparative clinical trial data for personalized treatment selections.
Ontology highlight
ABSTRACT:
SUBMITTER: Cai T
PROVIDER: S-EPMC3062150 | biostudies-literature | 2011 Apr
REPOSITORIES: biostudies-literature
Publications
Analysis of randomized comparative clinical trial data for personalized treatment selections.
Cai Tianxi T Tian Lu L Wong Peggy H PH Wei L J LJ
Biostatistics (Oxford, England) 20100928 2
Suppose that under the conventional randomized clinical trial setting, a new therapy is compared with a standard treatment. In this article, we propose a systematic, 2-stage estimation procedure for the subject-level treatment differences for future patient's disease management and treatment selections. To construct this procedure, we first utilize a parametric or semiparametric method to estimate individual-level treatment differences, and use these estimates to create an index scoring system f ...[more]
Similar Datasets
Project description:In a typical randomized clinical study to compare a new treatment with a control, oftentimes each study subject may experience any of several distinct outcomes during the study period, which collectively define the "risk-benefit" profile. To assess the effect of treatment, it is desirable to utilize the entirety of such outcome information. The times to these events, however, may not be observed completely due to, for example, competing risks or administrative censoring. The standard analyses based on the time to the first event, or individual component analyses with respect to each event time, are not ideal. In this paper, we classify each patient's risk-benefit profile, by considering all event times during follow-up, into several clinically meaningful ordinal categories. We first show how to make inferences for the treatment difference in a two-sample setting where categorical data are incomplete due to censoring. We then present a systematic procedure to identify patients who would benefit from a specific treatment using baseline covariate information. To obtain a valid and efficient system for personalized medicine, we utilize a cross-validation method for model building and evaluation and then make inferences using the final selected prediction procedure with an independent data set. The proposal is illustrated with the data from a clinical trial to evaluate a beta-blocker for treating chronic heart failure patients.
| S-EPMC4263228 | biostudies-literature
Project description:A genome-wide RNA expression study based on a Phase II randomized placebo-controlled clinical trial of topiramate (TPM) treatment of methamphetamine (METH) dependence. Gene expression data were analyzed based on TPM responses at Weeks 8 and 12, to identify differentially expressed genes and related pathways.
2017-11-17 | GSE107015 | GEO
Project description:The aim of this study was to describe the design, methods, and baseline characteristics of children enrolled in the Personalized Addition lenses Clinical Trial (PACT). PACT aims to test the myopia control efficacy of progressive addition lenses (PALs) with personalized addition values compared with standard (+2.00 D) addition PALs and single vision lenses (SVLs).PACT is a randomized, controlled, double-masked clinical trial. Two hundred eleven myopic Chinese children (7-12 years) were enrolled and randomized into 1 of the 3 following groups: personalized addition PALs; +2.00 addition PALs; and SVLs. Personalized addition values were determined based on the highest addition that satisfied Sheard criterion. Axial length and other biometric data were also recorded.At baseline, no differences were found between the right and left eyes for any of the main parameters. The enrolled children were 9.7?±?1.1 years' old with cycloplegic autorefraction (right eye [OD]: -2.36?±?0.64 D), near phoria (1.0?±?5.0 prism diopter esophoria), lag of accommodation (1.40?±?0.50 D) and axial length (OD: 24.58?±?0.74?mm). The personalized addition values ranged from +0.75 to +3.00 (average?±?SD: 2.19?±?0.73 D).PACT is a clinical trial evaluating whether myopia progression in children can be slowed by wearing personalized addition PALs compared with fixed addition PALs and SVLs as measured by cycloplegic autorefraction and axial length. Baseline data were comparable with those of previous myopia control studies in children. Subjects will be followed up every 6 months for 2 years.
| S-EPMC5369877 | biostudies-other
Project description:BackgroundLateral epicondylitis (LE) is a challenging condition for clinicians, and research has yet not proven the superiority of one specific treatment approach. However, manual therapy (elbow mobilization) in addition to eccentric exercise has been found to be superior to exercise alone. As well, acupuncture is effective in short-term pain relief when compared with sham treatment, but there is little knowledge on the comparative effectiveness of manual therapy and acupuncture treatment of LE in terms of pain relief. The primary objective of this pilot trial was to assess the feasibility (retention and adherence rates) of performing a randomized controlled trial (RCT) to explore the clinical effectiveness of acupuncture and manual therapy treatment of LE.MethodsThis pilot trial took place in an outpatient interdisciplinary institute of sports medicine and rehabilitation in Oslo, Norway. Thirty-six adults with clinically diagnosed LE were randomly allocated into one of three groups: eccentric exercise alone, eccentric exercise plus acupuncture, or eccentric exercise plus manual therapy for a 12-week treatment period. Primary outcomes were patient retention and adherence rates. Secondary outcomes included patient-reported pain (NRS), level of disability (Quick-DASH), and participant's satisfaction with treatment and global perceived effect.ResultsNine (69%) patients in the acupuncture group completed the 1-year follow-up, compared to eight (67%) in the manual therapy group and five (45%) in exercise alone. Our goal was to demonstrate a retention rate above 80% to avoid serious threats to validity, but the result was lower than expected. The majority of participants (64%) in both treatment groups received only three-treatment sessions; the reasons included non-attendance or recovery from pain. Secondary outcomes support the rationale for conduction of an RCT. There were no adverse advents related to study participation.ConclusionsBased on differences in pain relief between groups, patient retention, and adherence rates, an RCT seems to be feasible to assess treatment effectiveness more precisely. In a future definitive trial, greater dropout may be reduced by maintaining contact with the participants in the exercise alone group throughout the intervention, and objective assessments might be considered.Trial registrationClinicalTrials.gov, NCT02321696.
| S-EPMC6731611 | biostudies-literature
Project description:In comparative studies, treatment effect is often assessed using a binary outcome that indicates response to the therapy. Commonly used summary measures for response include the cumulative and current response rates at a specific time point. The current response rate is sometimes called the probability of being in response (PBIR), which regards a patient as a responder only if they have achieved and remain in response at present. The methods used in practice for estimating these rates, however, may not be appropriate. Moreover, whereas an effective treatment is expected to achieve a rapid and sustained response, the response at a fixed time point does not provide information about the duration of response (DOR). As an alternative, a curve constructed from the current response rates over the entire study period may be considered, which can be used for visualizing how rapidly patients responded to therapy and how long responses were sustained. The area under the PBIR curve is the mean DOR. This connection between response and DOR makes this curve attractive for assessing the treatment effect. In contrast to the conventional method for analyzing the DOR data, which uses responders only, the above procedure includes all patients in the study. Although discussed extensively in the statistical literature, estimation of the current response rate curve has garnered little attention in the medical literature. This article illustrates how to construct and analyze such a curve using data from a recent study for treating renal cell carcinoma. Clinical trialists are encouraged to consider this robust and clinically interpretable procedure as an additional tool for evaluating treatment effects in clinical studies.
| S-EPMC7773521 | biostudies-literature
Project description:ImportanceLumbar spinal stenosis (LSS) is the most common reason for spine surgery in older US adults. There is an evidence gap about nonsurgical LSS treatment options.ObjectiveTo explore the comparative clinical effectiveness of 3 nonsurgical interventions for patients with LSS.Design, setting, and participantsThree-arm randomized clinical trial of 3 years' duration (November 2013 to June 2016). Analysis began in August 2016. All interventions were delivered during 6 weeks with follow-up at 2 months and 6 months at an outpatient research clinic. Patients older than 60 years with LSS were recruited from the general public. Eligibility required anatomical evidence of central canal and/or lateral recess stenosis (magnetic resonance imaging/computed tomography) and clinical symptoms associated with LSS (neurogenic claudication; less symptoms with flexion). Analysis was intention to treat.InterventionsMedical care, group exercise, and manual therapy/individualized exercise. Medical care consisted of medications and/or epidural injections provided by a physiatrist. Group exercise classes were supervised by fitness instructors in senior community centers. Manual therapy/individualized exercise consisted of spinal mobilization, stretches, and strength training provided by chiropractors and physical therapists.Main outcomes and measuresPrimary outcomes were between-group differences at 2 months in self-reported symptoms and physical function measured by the Swiss Spinal Stenosis questionnaire (score range, 12-55) and a measure of walking capacity using the self-paced walking test (meters walked for 0 to 30 minutes).ResultsA total of 259 participants (mean [SD] age, 72.4 [7.8] years; 137 women [52.9%]) were allocated to medical care (88 [34.0%]), group exercise (84 [32.4%]), or manual therapy/individualized exercise (87 [33.6%]). Adjusted between-group analyses at 2 months showed manual therapy/individualized exercise had greater improvement of symptoms and physical function compared with medical care (-2.0; 95% CI, -3.6 to -0.4) or group exercise (-2.4; 95% CI, -4.1 to -0.8). Manual therapy/individualized exercise had a greater proportion of responders (≥30% improvement) in symptoms and physical function (20%) and walking capacity (65.3%) at 2 months compared with medical care (7.6% and 48.7%, respectively) or group exercise (3.0% and 46.2%, respectively). At 6 months, there were no between-group differences in mean outcome scores or responder rates.Conclusions and relevanceA combination of manual therapy/individualized exercise provides greater short-term improvement in symptoms and physical function and walking capacity than medical care or group exercises, although all 3 interventions were associated with improvements in long-term walking capacity.Trial registrationClinicalTrials.gov Identifier: NCT01943435.
| S-EPMC6324321 | biostudies-literature
Project description:BackgroundA mobile phone-based application can be useful for patients with type 1 diabetes in managing their disease. This results in large datasets accumulated on the patient's devices, which can be used for individualized feedback. The effect of such feedback is investigated in this article.Materials and methodsWe developed an application that included a data-driven feedback module known as Diastat for patients on self-measured blood glucose regimens. Using a stepped-wedge design, both groups initially received an application without Diastat. Group 1 activated Diastat after 4 weeks, whereas Group 2 activated Diastat 12 weeks after startup (T1). End points were glycated hemoglobin (HbA1c) level and number of out-of-range (OOR) measurements (i.e., outside the range 72-270 mg/dL).ResultsThirty patients were recruited to the study, and 15 were assigned to each group after the initial meeting. There were no significant differences between groups at T1 in HbA1c or OOR events. Overall, all patients had a decrease of 0.6 percentage points in mean HbA1c (P < 0.001) and 14.5 in median OOR events over 2 weeks (P < 0.001).ConclusionsThe study does not provide evidence that data-driven feedback improves glycemic control. The decrease in HbA1c was sizeable and significant, even though the study was not powered to detect this. The overall improvement in glycemic control suggests that, in general, mobile phone-based interventions can be useful in diabetes self-management.
| S-EPMC4504254 | biostudies-literature
Project description:ImportancePolicymakers and researchers have proposed a variety of interpretative front-of-package food labeling systems, but it remains unclear which is most effective at encouraging people to choose healthier foods and beverages, including among people with less education.ObjectiveTo test the effects of 4 interpretative front-of-package food labeling systems on the healthfulness of beverage and snack selections, overall and by education level.Design, setting, and participantsThis randomized clinical trial of a national sample of US adults 18 years and older was conducted online from November 16 to December 3, 2022.InterventionParticipants were randomized to view products with 1 of 5 food labeling systems, including control (calorie labels only) or 1 of 4 interpretative labeling systems: green ("choose often") labels added to healthy foods; single traffic light labels added to healthy, moderately healthy, and unhealthy foods; physical activity calorie equivalent labels added to all products; and nutrient warning labels added to products high in calories, sugar, saturated fat, or sodium. All conditions had calorie labels on all products.Main outcomes and measuresParticipants selected 1 of 16 beverages and 1 of 16 snacks that they wanted to hypothetically purchase. The primary outcomes were calories selected from beverages and from snacks. Secondary outcomes included label reactions and perceptions.ResultsA total of 7945 participants completed the experiment and were included in analyses (4078 [51%] female, 3779 [48%] male, and 88 [1%] nonbinary or another gender; mean [SD] age, 47.5 [17.9 years]). Compared with the control arm, exposure to the green (average differential effect [ADE], -34.2; 95% CI, -42.2 to -26.1), traffic light (ADE, -31.5; 95% CI, -39.5 to -23.4), physical activity (ADE, -39.0; 95% CI, -47.0 to -31.1), or nutrient warning labels (ADE, -28.2; 95% CI, -36.2 to -20.2) led participants to select fewer calories from beverages (all P < .001). Similarly, compared with the control label, exposure to the green (ADE, -12.7; 95% CI, -17.3 to -8.2), traffic light (ADE, -13.7; 95% CI, -18.2 to -9.1), physical activity (ADE, -18.5; 95% CI, -23.1 to -13.9), or nutrient warning labels (ADE, -14.2; 95% CI, -18.8 to -9.6) led participants to select fewer calories from snacks (all P < .001). These effects did not differ by education level. The green labels were rated as less stigmatizing than the other interpretative systems but otherwise generally received the least favorable label reactions and perceptions (eg, elicited less attention, were perceived as less trustworthy), while the nutrient warnings and physical activity labels received the most favorable ratings.Conclusions and relevanceIn this randomized clinical trial of front-of-package food labeling systems, all 4 interpretative labeling systems reduced calories selected from beverages and from snacks compared with calorie labels, with no differences by education level.Trial registrationClinicalTrials.gov Identifier: NCT05432271.
| S-EPMC10500374 | biostudies-literature
Project description:OBJECTIVE:The present study aimed to evaluate the efficacy and safety of adjunctive aripiprazole treatment in schizophrenia patients with risperidone-induced hyperprolactinemia. METHODS:One hundred and thirteen patients who were receiving a stable dose of risperidone were randomly assigned to either adjunctive aripiprazole treatment (10 mg/day) (aripiprazole group) or no additional treatment (control group) at a 1:1 ratio for 8 weeks. Schizophrenia symptoms were measured using the Positive and Negative Syndrome Scale (PANSS). Rating scales and safety assessments (RSESE, BARS, UKU) were performed at baseline and at weeks 4 and 8. Serum levels of prolactin were determined at baseline and at weeks 2, 4, 6 and 8. Metabolic parameters were determined at baseline and again at weeks 4 and 8. RESULTS:One hundred and thirteen patients were enrolled in this study, and 107 patients completed the study (54 in the aripiprazole group, and 53 in the control group). PANSS-total scores in the aripiprazole group decreased significantly at week 4 (P = 0.003) and week 8 (P = 0.007) compared with the control group. PANSS-negative scores in the aripiprazole group also decreased significantly at week 4 (P = 0.005) and week 8 (P< 0.001) compared with the control group. Serum levels of prolactin in the aripiprazole group decreased significantly at week 2 (P< 0.001), week 4 (P< 0.001), week 6 (P< 0.001) and week 8 (P< 0.001) compared with the control group. There were no significant differences in changes of Fasting Plasma Glucose, Total cholesterol, Triglycerides and High Density Lipoprotein within each group at week 4 and 8 execpt low density lipoproteins. There was no significant difference in the incidence of adverse reactions between the two groups. CONCLUSIONS:Adjunctive aripiprazole treatment may be beneficial in reducing serum levels of prolactin and improving negative symptoms in schizophrenia patients with risperidone-induced hyperprolactinemia. TRIAL REGISTRATION:chictr.org ChiCTR-IOR-15006278.
| S-EPMC4598102 | biostudies-literature
Project description:ObjectiveRumination heightens risk for depression and anxiety, which increase substantially during adolescence. Smartphone apps offer a convenient and cost-effective means for adolescents to access mindfulness training, which may reduce rumination. Despite their increasing popularity, it is unclear which adolescents benefit from mindfulness apps.MethodAdolescents (n = 152) with elevated trait rumination were randomly assigned to 3 weeks of app-based mindfulness training or a mood-monitoring control. Multilevel models tested group differences in state rumination change, assessed via ecological momentary assessment. Baseline adolescent characteristics were submitted to elastic net regularization models to develop a "Personalized Advantage Index" indicating an individual's expected outcome from the mindfulness app relative to the mood-monitoring control. Finally, we translated a predictive model (developed in an external sample) for personalized recommendations of expected benefit from the mindfulness app.ResultsAdolescents in the mindfulness app condition reported significantly greater reductions in rumination than adolescents in the control condition. Individuals predicted to have better outcomes from the mindfulness app relative to mood monitoring had significantly greater reductions in rumination if randomly assigned to the mindfulness condition. In contrast, between-condition differences in outcome were not significant for adolescents predicted to have better outcomes in the mood-monitoring condition.ConclusionsFindings support the efficacy of a mindfulness app to reduce state rumination in adolescents, particularly among adolescents high in trait rumination. A predictive model is put forth, which could be used to objectively communicate expected mindfulness app outcomes to adolescents prior to engagement in app-based mindfulness training. (PsycInfo Database Record (c) 2022 APA, all rights reserved).
| S-EPMC9886135 | biostudies-literature