scholarly journals Does evidence support the high expectations placed in precision medicine? A bibliographic review

F1000Research ◽  
2018 ◽  
Vol 7 ◽  
pp. 30 ◽  
Author(s):  
Jordi Cortés ◽  
José Antonio González ◽  
María Nuncia Medina ◽  
Markus Vogler ◽  
Marta Vilaró ◽  
...  
Keyword(s):  
Precision Medicine ◽  
Intervention Group ◽  
Individual Characteristics ◽  
Outcome Variable ◽  
Point Estimate ◽  
Control Group ◽  
Implicit Assumption ◽  
Variance Ratio ◽  
Eligibility Criteria ◽  
High Expectations

Background: Precision medicine is the Holy Grail of interventions that are tailored to a patient’s individual characteristics. However, conventional clinical trials are designed to find differences with the implicit assumption that the effect is the same in all patients within the eligibility criteria. If this were the case, then there would be no grounds for treating different patients differently. One consequence of the assumption is that the variance in the outcome variable would be the same in treated and control arms. We reviewed the literature to see if this were the case and so to see how often precision medicine would not be useful. Methods: We reviewed parallel trials with quantitative outcomes published in 2004, 2007, 2010 and 2013. We collected baseline and final standard deviations of the main outcome. We assessed homoscedasticity by comparing the variance of the primary endpoint between arms through the outcome variance ratio (treated to control group). Results: The review provided 208 articles with enough information to conduct the analysis. One out of seven studies (n = 30, 14.4%) had statistically different variances between groups, leading a non-constant-effect. The adjusted point estimate of the mean outcome variance ratio (treated to control group) is 0.89 (95% CI 0.81 to 0.97). Conclusions: We found that the outcome variance was more often smaller in the intervention group, suggesting that treated patients may end up pertaining more often to reference or “normality” values and thus would not require further precision medicine. However, this result may also be compatible with a reduced effect in some patients, which would require studying whether the effect merits enduring the side effects as well as the economic costs. We have shown that the comparison of variances is a useful but not definitive tool to asses if the design assumption of a constant effect holds.

scholarly journals Does evidence support the high expectations placed in precision medicine? A bibliographic review

F1000Research ◽  
2018 ◽  
Vol 7 ◽  
pp. 30 ◽  
Author(s):  
Jordi Cortés ◽  
José Antonio González ◽  
María Nuncia Medina ◽  
Markus Vogler ◽  
Marta Vilaró ◽  
...  
Keyword(s):  
Precision Medicine ◽  
Intervention Group ◽  
Individual Characteristics ◽  
Outcome Variable ◽  
Point Estimate ◽  
Control Group ◽  
Implicit Assumption ◽  
Variance Ratio ◽  
Eligibility Criteria ◽  
High Expectations

Background: Precision medicine is the Holy Grail of interventions that are tailored to a patient’s individual characteristics. However, conventional clinical trials are designed to find differences with the implicit assumption that the effect is the same in all patients within the eligibility criteria. If this were the case, then there would be no grounds for treating different patients differently. One consequence of the assumption is that the variance in the outcome variable would be the same in treated and control arms. We reviewed the literature to see if this were the case and so to see how often precision medicine would not be useful. Methods: We reviewed parallel trials with quantitative outcomes published in 2004, 2007, 2010 and 2013. We collected baseline and final standard deviations of the main outcome. We assessed homoscedasticity by comparing the variance of the primary endpoint between arms through the outcome variance ratio (treated to control group). Results: The review provided 208 articles with enough information to conduct the analysis. One out of seven studies (n = 30, 14.4%) had statistically different variances between groups, leading a non-constant-effect. The adjusted point estimate of the mean outcome variance ratio (treated to control group) is 0.89 (95% CI 0.81 to 0.97). Conclusions: We found that the outcome variance was more often smaller in the intervention group, suggesting that treated patients may end up pertaining more often to reference or “normality” values and thus would not require further precision medicine. However, this result may also be compatible with a reduced effect in some patients, which would require studying whether the effect merits enduring the side effects as well as the economic costs. We have shown that the comparison of variances is a useful but not definitive tool to asses if the design assumption of a constant effect holds.

scholarly journals Does evidence support the high expectations placed in precision medicine? A bibliographic review

F1000Research ◽  
2018 ◽  
Vol 7 ◽  
pp. 30 ◽  
Author(s):  
Jordi Cortés ◽  
José Antonio González ◽  
María Nuncia Medina ◽  
Markus Vogler ◽  
Marta Vilaró ◽  
...  
Keyword(s):  
Precision Medicine ◽  
Randomized Trials ◽  
Intervention Group ◽  
Individual Characteristics ◽  
Point Estimate ◽  
Control Group ◽  
Eligibility Criteria ◽  
High Expectations ◽  
The Mean ◽  
And Control

Background: Precision medicine is the Holy Grail of interventions that are tailored to a patient’s individual characteristics. However, the conventional design of randomized trials assumes that each individual benefits by the same amount. Methods: We reviewed parallel trials with quantitative outcomes published in 2004, 2007, 2010 and 2013. We collected baseline and final standard deviations of the main outcome. We assessed homoscedasticity by comparing the outcome variability between treated and control arms. Results: The review provided 208 articles with enough information to conduct the analysis. At the end of the study, 113 (54%, 95% CI 47 to 61%) papers find less variability in the treated arm. The adjusted point estimate of the mean ratio (treated to control group) of the outcome variances is 0.89 (95% CI 0.81 to 0.97). Conclusions: Some variance inflation was observed in just 1 out of 6 interventions, suggesting the need for further eligibility criteria to tailor precision medicine. Surprisingly, the variance was more often smaller in the intervention group, suggesting, if anything, a reduced role for precision medicine.  Homoscedasticity is a useful tool for assessing whether or not the premise of constant effect is reasonable.

scholarly journals Does evidence support the high expectations placed in precision medicine? A bibliographic review

F1000Research ◽  
2019 ◽  
Vol 7 ◽  
pp. 30 ◽  
Author(s):  
Jordi Cortés ◽  
José Antonio González ◽  
María Nuncia Medina ◽  
Markus Vogler ◽  
Marta Vilaró ◽  
...  
Keyword(s):  
Precision Medicine ◽  
Intervention Group ◽  
Individual Characteristics ◽  
Point Estimate ◽  
Control Group ◽  
Variance Ratio ◽  
Economic Costs ◽  
High Expectations ◽  
The Mean ◽  
And Control

Background: Precision medicine is the Holy Grail of interventions that are tailored to a patient’s individual characteristics. However, conventional clinical trials are designed to find differences in averages, and interpreting these differences depends on untestable assumptions. Although ideal, a constant effect would facilitate individual management. Another consequence of a constant effect is that the outcome variance would be the same in treated and control arms. We reviewed the literature to explore the similarity of these variances as a foundation for examining whether and how often precision medicine is definitively needed. Methods: We reviewed parallel trials with quantitative outcomes published in 2004, 2007, 2010 and 2013. We collected baseline and final standard deviations of the main outcome. We assessed homoscedasticity by comparing the variance of the primary endpoint between arms through the outcome variance ratio (treated to control group). Results: The review provided 208 articles with enough information to conduct the analysis. One out of seven studies (n = 30, 14.4%) had statistically different variances between groups, leading a non-constant-effect. The adjusted point estimate of the mean outcome variance ratio (treated to control group) is 0.89 (95% CI 0.81 to 0.97). Conclusions: We found that the outcome variance was more often smaller in the intervention group, suggesting that treated patients may end up pertaining more often to reference values. This observed reduction in variance might also imply that there could be a subgroup of less ill patients who derive no benefit, which would require studying whether the effect merits enduring the side effects as well as the economic costs. We have shown that the comparison of variances is a useful but not definitive tool for assessing whether or not the assumption of a constant effect holds.

scholarly journals Does evidence support the high expectations placed in precision medicine? A bibliographic review

F1000Research ◽  
2019 ◽  
Vol 7 ◽  
pp. 30 ◽  
Author(s):  
Jordi Cortés ◽  
José Antonio González ◽  
María Nuncia Medina ◽  
Markus Vogler ◽  
Marta Vilaró ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Precision Medicine ◽  
Outcome Measure ◽  
Intervention Group ◽  
Direct Consequence ◽  
Individual Characteristics ◽  
Point Estimate ◽  
Control Group ◽  
Variance Ratio ◽  
The Mean

Background: Precision medicine is the Holy Grail of interventions that are tailored to a patient’s individual characteristics. However, conventional clinical trials are designed to find differences in averages, and interpreting these differences depends on untestable assumptions. Although only an ideal, a constant effect of treatment would facilitate individual management. A direct consequence of a constant effect is that the variance of the outcome measure would be the same in the treated and control arms. We reviewed the literature to explore the similarity of these variances as a foundation for examining whether and how often precision medicine is definitively required. Methods: We reviewed parallel clinical trials with numerical primary endpoints published in 2004, 2007, 2010 and 2013. We collected the baseline and final standard deviations of the main outcome measure. We assessed homoscedasticity by comparing the variance of the primary endpoint between arms through the outcome variance ratio (treated to control group). Results: The review provided 208 articles with enough information to conduct the analysis. One out of five studies (n = 40, 19.2%) had statistically different variances between groups, implying a non-constant-effect. The adjusted point estimate of the mean outcome variance ratio (treated to control group) is 0.89 (95% CI 0.81 to 0.97). Conclusions: The mean variance ratio is significantly lower than 1 and the lower variance was found more often in the intervention group than in the control group, suggesting it is more usual for treated patients to be stable. This observed reduction in variance might also imply that there could be a subgroup of less ill patients who derive no benefit from treatment. This would require further study as to whether the treatment effect outweighs the side effects as well as the economic costs. We have shown that there are ways to analyze the apparently unobservable constant effect.

scholarly journals Are patients willing to remove, and capable of removing, their own nonabsorbable sutures?

CJEM ◽  
2012 ◽  
Vol 14 (04) ◽  
pp. 221-227 ◽  
Author(s):  
Peter Macdonald ◽  
Nadia Primiani ◽  
Adam Lund
Keyword(s):  
Wound Care ◽  
Intervention Group ◽  
Comfort Level ◽  
Patient Comfort ◽  
Control Group ◽  
Complication Rates ◽  
Eligibility Criteria ◽  
Telephone Contact ◽  
Physician Visits

ABSTRACTObjectives:Providing patients with instructions and equipment regarding self-removal of nonabsorbable sutures could represent a new efficiency in emergency department (ED) practice. The primary outcome was to compare the proportion of patients successfully removing their own sutures when provided with suture removal instructions and equipment versus the standard advice and follow-up care. Secondary outcomes included complication rates, number of physician visits, and patient comfort level.Methods:This prospective, controlled, single-blinded, pseudorandomized trial enrolled consecutive ED patients who met the eligibility criteria (age > 19 years, simple lacerations, nonabsorbable sutures, immunocompetent). The study group was provided with wound care instructions, a suture removal kit, and instructions regarding suture self-removal. The control group received wound care instructions alone. Outcomes were assessed by telephone contact at least 14 days after suturing using a standardized questionnaire.Results:Overall, 183 patients were enrolled (93 in the intervention group; 90 in the control group). Significantly more patients performed suture self-removal in the intervention group (91.5%; 95% CI 85.4–97.5) compared to the control group (62.8%; 95% CI 52.1–73.6) (p< 0.001). Patients visited their physician less often in the intervention group (9.8%; 95% CI 3.3–16.2) compared to the control group (34.6%; 95% CI 24.1–45.2%) (p< 0.001). Complication rates were similar in both groups.Conclusion:Most patients are willing to remove, and capable of removing, their own sutures. Providing appropriate suture removal instructions and equipment to patients with simple lacerations in the ED appears to be both safe and acceptable.

scholarly journals The Use of Telehealth Technology in Assessing the Accuracy of Self-Reported Weight and the Impact of a Daily Immediate-Feedback Intervention among Obese Employees

2011 ◽  
Vol 2011 ◽  
pp. 1-6 ◽  
Author(s):  
Nicolaas P. Pronk ◽  
A. Lauren Crain ◽  
Jeffrey J. VanWormer ◽  
Brian C. Martinson ◽  
Jackie L. Boucher ◽  
...  
Keyword(s):  
Body Weight ◽  
Mixed Model ◽  
Linear Mixed Model ◽  
Controlled Trial ◽  
Intervention Group ◽  
Study Groups ◽  
Outcome Variable ◽  
Control Group ◽  
Immediate Feedback ◽  
The Impact

Objective.To determine the accuracy of self-reported body weight prior to and following a weight loss intervention including daily self-weighing among obese employees.Methods.As part of a 6-month randomized controlled trial including a no-treatment control group, an intervention group received a series of coaching calls, daily self-weighing, and interactive telemonitoring. The primary outcome variable was the absolute discrepancy between self-reported and measured body weight at baseline and at 6 months. We used general linear mixed model regression to estimate changes and differences between study groups over time.Results.At baseline, study participants underreported their weight by an average of 2.06 (se=0.33) lbs. The intervention group self-reported a smaller absolute body weight discrepancy at followup than the control group.Conclusions.The discrepancy between self-reported and measured body weight appears to be relatively small, may be improved through daily self-monitoring using immediate-feedback telehealth technology, and negligibly impacts change in body weight.

Corticosteroid as an Adjunct in the Treatment of Endobronchial Tuberculosis: A Systematic Review & Meta-analysis

2020 ◽  
Vol 16 (1) ◽  
pp. 53-60
Author(s):  
Nihar R. Mishra ◽  
Manoj K. Panigrahi ◽  
Girish C. Bhatt ◽  
Rashmi R. Das
Keyword(s):  
Adverse Events ◽  
Meta Analysis ◽  
Intervention Group ◽  
Oral Prednisolone ◽  
Oral Steroid ◽  
Control Group ◽  
Eligibility Criteria ◽  
Steroid Group ◽  
Significant Difference ◽  
Endobronchial Tuberculosis

Background: Corticosteroid exerts anti-inflammatory action and can prevent tissue damage resulting from various causes. Studies have shown that corticosteroids may prevent the damaging effect of tuberculosis (TB) in various organs, but there is no published meta-analysis specifically looking for the effect of corticosteroid in endobronchial TB. Objective: To synthesize the evidence regarding the usefulness of corticosteroid in endo-bronchial TB. Methods: A comprehensive search was performed of the major electronic databases till 30th November 2018. Randomized trials comparing treatment with corticosteroid as an adjunct to antitubercular drugs (ATT) versus placebo/no treatment in endobronchial TB were included. Three authors independently applied eligibility criteria, assessed the studies for methodological quality, and extracted data. The review is registered at PROSPERO database [CRD42016047063]. Results: Out of 525 search results, 4 trials including 205 patients (151 children) were eligible for inclusion. Oral prednisolone was used in various dose schedules. Rifampicin containing ATT regimen was used in 3 trials. The bronchoscopy findings showed no significant improvement at 1 month (effect size could not be calculated due to 0 event in the intervention group, p = 0.05), 2 months (RR 1.26, 95% CI 0.89 to 1.8), and at completion of ATT (RR 0.63, 95% CI 0.1 to 4.14) in steroid-treated group compared to the control group. The need for repeat bronchoscopy was significantly decreased in the steroid group (RR 0.13, 95% CI 0.02 to 0.9). Among the adverse events, the infection rate was significantly lesser in the steroid group (RR 0.53, 95% CI 0.29 to 0.97); but other adverse events (mortality, hypertension, and abdominal distension) showed no significant difference between the two groups. The GRADE evidence generated was of very low quality. Conclusions: The present meta-analysis showed that oral steroid does not help patients with endobronchial tuberculosis. However, the quality of evidence was very low. Future trials with robust design and a larger sample size would be required to provide any firm recommendation regarding the use of oral prednisolone in endobronchial tuberculosis.

scholarly journals Can the Onset of Type 2 Diabetes Be Delayed by a Group-Based Lifestyle Intervention in Women with Prediabetes following Gestational Diabetes Mellitus (GDM)? Findings from a Randomized Control Mixed Methods Trial

2015 ◽  
Vol 2015 ◽  
pp. 1-11 ◽  
Author(s):  
Angela O’Dea ◽  
Marie Tierney ◽  
Brian E. McGuire ◽  
John Newell ◽  
Liam G. Glynn ◽  
...  
Keyword(s):  
Type 2 Diabetes ◽  
Mixed Methods ◽  
Gestational Diabetes ◽  
Lifestyle Intervention ◽  
Intervention Group ◽  
Outcome Variable ◽  
Control Group ◽  
One Year

Objective. To evaluate a 12-week group-based lifestyle intervention programme for women with prediabetes following gestational diabetes (GDM).Design. A two-group, mixed methods randomized controlled trial in which 50 women with a history of GDM and abnormal glucose tolerance postpartum were randomly assigned to intervention (n=24) or wait control (n=26) and postintervention qualitative interviews with participants.Main Outcome Measures. Modifiable biochemical, anthropometric, behavioural, and psychosocial risk factors associated with the development of type 2 diabetes. The primary outcome variable was the change in fasting plasma glucose (FPG) from study entry to one-year follow-up.Results. At one-year follow-up, the intervention group showed significant improvements over the wait control group on stress, diet self-efficacy, and quality of life. There was no evidence of an effect of the intervention on measures of biochemistry or anthropometry; the effect on one health behaviour, diet adherence, was close to significance.Conclusions. Prevention programmes must tackle the barriers to participation faced by this population; home-based interventions should be investigated. Strategies for promoting long-term health self-management need to be developed and tested.

scholarly journals Effectiveness of a clinic-based randomized controlled intervention for type 2 diabetes management: an innovative model of intensified diabetes management in Mainland China (C-IDM study)

2020 ◽  
Vol 8 (1) ◽  
pp. e001030 ◽  
Author(s):  
Qinglin Lou ◽  
Qing Ye ◽  
Haidi Wu ◽  
Zhiyong Wang ◽  
Robert S Ware ◽  
...  
Keyword(s):  
Type 2 Diabetes ◽  
Large Scale ◽  
Diabetes Management ◽  
Controlled Trial ◽  
Intervention Group ◽  
Mainland China ◽  
Outcome Variable ◽  
Control Group ◽  
Randomized Controlled

ObjectivesHighly efficient diabetes management programs are needed for tackling diabetes in China. This study aimed to assess the effectiveness of a clinic-based intensified diabetes management model (C-IDM) in Mainland China.Research design and methodsA 2-year clinic-based randomized controlled trial was conducted among patients with type 2 diabetes in Nanjing, China. The C-IDM intervention components comprised four domains (disease targeting management, express referral channel, expert visit, patients’ self-management) and an integrated running system (disease control centers, general hospitals and local clinics). Control group participants received their usual care, while intervention participants received both the C-IDM package and the usual services. The primary outcome variable was change of hemoglobin A1c (HbA1c). Mixed-effects models were used to compute effect estimates and 95% CI with consideration of both individual and cluster-level confounders.ResultsOverall, 1095 of 1143 participants were assessed at study completion. The mean change in HbA1c was significantly greater in the intervention group than in the control group (mean difference (MD)=−0.57, 95% CI −0.79 to –0.36). Similar results were observed for change in body mass index (MD=−0.29, 95% CI −0.49 to –0.10). Participants in the intervention group were more likely to achieve normal HbA1c and body weight compared with their counterparts in control group after adjusting for potentially confounding variables (adjusted OR=1.94, 95% CI 1.35 to 2.81 and 1.79, 95% CI 1.13 to 2.85, respectively).ConclusionsThe C-IDM model is feasible and effective in large-scale management of patients with type 2 diabetes in China. It has public health implications for tackling the burden of diabetes in China.Trial registration numberChiCTR-IOR-15006019.

scholarly journals Early Nutritional Education in the Prevention of Childhood Obesity

2021 ◽  
Vol 18 (12) ◽  
pp. 6569
Author(s):  
Mario Gato-Moreno ◽  
María F. Martos-Lirio ◽  
Isabel Leiva-Gea ◽  
M. Rosa Bernal-López ◽  
Fernando Vegas-Toro ◽  
...  
Keyword(s):  
Educational Intervention ◽  
Intervention Group ◽  
Overweight And Obesity ◽  
The Body ◽  
Secondary Outcome ◽  
Outcome Variable ◽  
Controlled Study ◽  
Control Group ◽  
Nutritional Education ◽  
Overweight Or Obesity

Early childhood is a critical period for obesity prevention. This randomized controlled study evaluated the effectiveness of an educational intervention preventing obesity in preschool-age children. A nutritional education intervention, with a follow-up session one year later, was conducted with parents of children aged 3 to 4 years of public schools in the province of Málaga. The main outcome variable was the body mass index z-score (zBMI). The prevalence of overweight or obesity was the secondary outcome variable. The sample comprised 261 students (control group = 139). Initial BMI, weight, height-for-age and prevalence of overweight and obesity were similar for both groups. After the first year of the intervention, the zBMI of the intervention group decreased significantly from 0.23 to 0.10 (p = 0.002), and the subgroup of patients with baseline zBMI above the median decreased from 1 to 0.72 (p = 0.001), and in the second year from 1.01 to 0.73 (p = 0.002). The joint prevalence of overweight and obesity increased in the control group (12.2% to 20.1%; p = 0.027), while in the intervention group, there were no significant changes. This preschool educational intervention with parents improved their children’s BMI, especially those with a higher BMI for their age, and favored the prevention of overweight or obesity.

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