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 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 ◽  
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 ◽  
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 OP0159 EFFICACY OF COMPREHENSIVE TECHNOLOGY-ASSISTED HOME-BASED EXERCISE IN ANKYLOSING SPONDYLITIS: A RANDOMIZED, CONTROLLED TRIAL

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 100.3-100
Author(s):  
Y. Wang ◽  
X. Liu ◽  
Y. Shi ◽  
X. Ji ◽  
W. Wang ◽  
...  
Keyword(s):  
Exercise Intervention ◽  
Medical Center ◽  
Controlled Trial ◽  
Intervention Group ◽  
Exercise Program ◽  
Group Differences ◽  
Control Group ◽  
Home Based ◽  
The Mean

Background:Clinical practice guidelines recommend that exercise is an essential component in the self-management of Ankylosing Spondylitis (AS). Attending supervised interventions requiring periodic medical center visits can be burdensome and patients may decline participation, whereas, effective home-based exercise interventions that do not need regular medical center visits are likely to be more accessible and acceptable for patients with AS. Recently, increasing evidences have been accumulated that the wearable devices could facilitate patients with inflammatory arthritis by giving exercise instructions and improving self-efficacy. Therefore, patients with AS may benefit from an effective technology-assisted home-based exercise intervention.Objectives:To investigate the efficacy of a comprehensive technology-assisted home-based exercise intervention on disease activity in patients with AS.Methods:This study was a 16-week assessor-blinded, randomized, waiting-list controlled trial (ChiCTR1900024244). Patients with AS were randomly allocated to the home-based exercise intervention group and the waiting-list control group. A 16-week comprehensive exercise program consisting of a moderate intensity (64%-76% HRmax) aerobic training for 30min on 5 days/week and a functional training for 60min on 3 days/week was given to patients in the intervention group immediately after randomization, with 1.5h training sessions for two consecutive days by a study physical therapist at baseline and Week 8. The aerobic exercise intensity was controlled by a Mio FUSE Wristband with a smartphone application. The functional training consisted of the posture training, range of motion exercises, strength training, stability training and stretching exercises. Patients in control group received standard care during the 16-week follow-up and started to receive the exercise program at Week 16. The primary outcome was ASDAS at Week 16. The secondary outcomes were BASDAI, BASFI, BASMI, ASAS HI, peak oxygen uptake, body composition and muscle endurance tests. The mean difference between groups in change from baseline was analyzed with the analysis of covariance.Results:A total of 54 patients with AS were enrolled (26 in intervention group and 28 in control group) and 46 (85.2%) patients completed the 16-week follow-up. The mean difference of ASDAS between groups in change from baseline to 16-week follow-up was −0.2 (95% CI, −0.4 to 0.003, P = 0.032), and the mean change from baseline was -0.4 (95% CI, -0.5 to -0.2) in the intervention group vs -0.1 (95% CI, -0.3 to 0.01) in the control group, respectively. Significant between-group differences were found between groups for BASDAI (−0.5 [95% CI, −0.9 to −0.2], P = 0.004), BASMI (−0.7 [95% CI, −1.1 to −0.4], P <0.001), BASFI (−0.3 [95% CI, −0.6 to 0.01], P=0.035), peak oxygen uptake (2.7 [95% CI, 0.02 to 5.3] ml/kg/min, P=0.048) and extensor endurance test (17.8 [95% CI, 0.5 to 35.2]s, P=0.044) at Week 16. Between-group differences were detected in ASAS HI (−0.9 [95% CI, −1.7 to −0.1], P=0.030), body fat percentage (−1.0 [95% CI, −2.0 to −0.01] %, P=0.048) and visceral adipose tissue (−4.9 [95% CI, −8.5 to −1.4] cm2, P=0.008) at Week 8, but not at Week 16. No significant between-group differences were detected in the total lean mass, time up and go test and the flexor endurance test during the follow-up.Conclusion:Comprehensive technology-assisted home-based exercise has been shown to have beneficial effects on disease activity, physical function, spinal mobility, aerobic capacity, and body composition as well as in improving fatigue and morning stiffness of patients with AS.References:[1]van der Heijde D, Ramiro S, Landewé R, et al. Ann Rheum Dis 2017;76:978–991.Disclosure of Interests:None declared

DigiKnowIt News: Educating youth about pediatric clinical trials using an interactive, multimedia educational website

2021 ◽  
pp. 136749352110037
Author(s):  
Alison E. Parker ◽  
Tracy M. Scull ◽  
Abigail M. Morrison
Keyword(s):  
Clinical Trials ◽  
Interactive Multimedia ◽  
Controlled Trial ◽  
Intervention Group ◽  
Control Group ◽  
Wait List ◽  
Wait List Control Group ◽  
Wait List Control ◽  
Satisfaction Questionnaire ◽  
Study Participants

Pediatric clinical trials allow for the testing of appropriate and effective treatments for children. However, some challenges exist with recruitment. This study examined the effectiveness of DigiKnowIt News, an interactive, multimedia website (which includes activities, videos, and comic books) designed to educate children about clinical trials. A randomized controlled trial was conducted in 2018 with 91 participants ( M age = 10.92 years; SD = 2.06). Participants were randomly assigned to intervention or wait-list control groups and completed questionnaires at pretest and posttest (1 week later) about their knowledge, attitudes, beliefs about clinical trials, and self-efficacy for participating in clinical trials. Participants in the intervention group received access to DigiKnowIt News between pretest and posttest and completed a satisfaction questionnaire at posttest. At the end of the study, participants in the wait-list control group were offered the option to use the website and complete a satisfaction questionnaire. At posttest, participants in the intervention group, compared to participants in the wait-list control group, had more knowledge about clinical trials and more reported confidence for participating in clinical trials. Participants reported high levels of satisfaction with DigiKnowIt News. The findings suggest that an educational website can improve factors related to increasing rates of participation in clinical trials.

The effectiveness of emotional freedom techniques (EFT) on depression of postmenopausal women: a randomized controlled trial

2021 ◽  
Vol 0 (0) ◽  
Author(s):  
Asieh Mehdipour ◽  
Parvin Abedi ◽  
Somayeh Ansari ◽  
Maryam Dastoorpoor
Keyword(s):  
Randomized Controlled Trial ◽  
Postmenopausal Women ◽  
Controlled Trial ◽  
Intervention Group ◽  
Control Group ◽  
Randomized Controlled ◽  
Moderate Depression ◽  
Before And After ◽  
The Mean ◽  
And Control

Abstract Objectives Postmenopausal women are at greater risk of depression. Depression may negatively affect the quality of life of women. An emotional freedom technique (EFT) is an evidence-based therapy combining cognitive and exposure components with acupressure. This study aimed to evaluate the effect of EFT on depression in postmenopausal women. Methods This was a randomized controlled trial in which 88 women with mild to moderate depression recruited from a menopausal clinic in Ahvaz, Iran, and randomly assigned into two groups of EFT (n=44) and control for sham therapy (n=44). Women in the EFT group received two sessions of training and asked to continue EFT for 8 weeks, one time per day. The Beck Depression Inventory (BDI2) completed by women before and after the intervention. The control group received training on sham acupressure points similar to the intervention group. Data collected using a demographic and BDI2. Women requested to complete the BDI2 before and after the intervention. The independent t-test, chi-square, and ANCOVA were used to analyze data. Results The mean depression score in the intervention group reduced from 20.93 ± 4.6 to 10.96 ± 4.38 in comparison to the control group that reduced from 19.18 ± 2.79 to 17.01 ± 6.05 after intervention (p=0.001). After the 8 week intervention, the frequency of moderate depression decreased from 56.8 to 9.35% in the intervention and from 50 to 29.5% in the control group. In total, 63.4 and 34.15% in the intervention and control groups were free of depression respectively after the intervention (p<0.001). Conclusions The results of this study showed that using EFT for 8 weeks could significantly reduce the mean score of depression in postmenopausal women. Using this method in public health centers for postmenopausal women is recommended.

Effects of an Off-Site Walking Program on Energy Expenditure, Serum Lipids, and Glucose Metabolism in Middle-Aged Women

2003 ◽  
Vol 4 (3) ◽  
pp. 181-192 ◽  
Author(s):  
Fumiko Furukawa ◽  
Keiko Kazuma ◽  
Masako Kawa ◽  
Mitsunori Miyashita ◽  
Kyohko Niiro ◽  
...  
Keyword(s):  
Energy Expenditure ◽  
Nurse Managers ◽  
Intervention Group ◽  
Density Lipoprotein ◽  
Control Group ◽  
Middle Aged ◽  
Walking Program ◽  
Before And After ◽  
The Mean ◽  
Blood Profiles

The present study aims to identify the effects of systematic walking on exercise energy expenditure (EEE) and blood profiles in middle-aged women. Fifty-two female nurse managers, aged 32 to 57 years (42.0 ± 6.2), were randomly assigned to an intervention group (IG) and a control group (CG) for a 12-week study of the walking program. EEE was measured using a microelectronic device. Blood profiles were assessed before and after the walking program. The mean EEE (kcal/kg/d) in the IG and CG was 4.73 ± 1.02 and 3.88 ± 0.81 ( P = 0.01), indicating an increase of 1.17 ± 0.98 and 0.46 ± 0.68 from baseline ( P = 0.01), respectively. The mean change in high-density lipoprotein cholesterol in the IG and CG was 1.8 ± 8.3 mg/dL and −2.9 ± 7.0 mg/dL ( P = 0.051); that in insulin was −4.5 ± 7.5 μU/dL and −0.6 ± 4.3 μU/dL ( P = 0.046), respectively. These results show that systematic walking increases EEE and improves blood profiles.

scholarly journals Effect of Hope-oriented group counseling on mental health of infertile women with failed IVF cycles: a randomized controlled trial

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Roya Rahimi ◽  
Shirin Hasanpour ◽  
Mojgan. Mirghafourvand ◽  
Khalil Esmaeilpour
Keyword(s):  
Mental Health ◽  
Confidence Interval ◽  
Group Counseling ◽  
Controlled Trial ◽  
Intervention Group ◽  
Mean Difference ◽  
Control Group ◽  
The Mean ◽  
And Control

Abstract Background Considering the prevalence of infertility in the community and the consequences of failure of infertility treatments on women’s mental health, interventions that can control stress, anxiety and depression in infertile women with a history of IVF failure will be very helpful. This study aimed to determine the effects of hope-oriented group counseling on mental health (primary outcome) and quality of life (QoL) (secondary outcome) of women with failed IVF cycles. Method This randomized controlled trial was conducted on 60 women with failed IVF cycles visiting Infertility Clinic at Al-Zahra Teaching Hospital of Tabriz- Iran. Participants were allocated to the intervention group (n = 30) and control group (n = 30) based on a randomized block design. Hope-oriented group counseling was provided to the intervention group in six 45–60 min sessions (once a week). The control group only received routine care to undergo another IVF cycle. The Depression Anxiety Stress Scale-21 (DASS-21) and the SF-12 Quality of Life Scale were filled out by interviewing the participants before the intervention and one week and one month after the intervention. After intervention 26 participants in each group were included in the analysis. Results There was no significant difference between the intervention and control groups in the socio-demographic profile of participants (P > 0.05). The post-intervention mean score of stress (adjusted mean difference = − 1.7, 95% confidence interval: − 3.2 to − 0.3, P = 0.018) and depression (adjusted mean difference = − 1.3, 95% confidence interval: − 4.7 to − 1.5, P < 0.001) was significantly lower in the intervention group compared to the control. Although the mean anxiety score was lower in the intervention group compared to the control, the difference between them was not statistically significant (adjusted mean difference = − 1.1, 95% confidence interval: − 2.6 to 0.4, P = 0.153). The mean score of QoL was significantly higher in the intervention group than that of the control group (adjusted mean difference = 6.9, 95% confidence interval: 5.1 to 8.8, P < 0.001). Conclusion Hope-oriented group counseling was effective in reducing stress and depression and improving QoL in women with failed IVF cycles. It is recommended to use this counseling approach, along with other methods, to improve the mental health of women with failed IVF cycles. Trial registration TCT Registration Number: TCTR 20191017003, registered on October 17, 2019.

THE EFFECT OF DHIKR ON ANXIETY AND DEPRESSION LEVEL IN HEMODIALYSIS PATIENTS AT PURWOKERTO ISLAMIC HOSPITAL

2021 ◽  
Vol 2 (1) ◽  
pp. 15-25
Author(s):  
Muhammad Anggun ◽  
Titik Kusumawinakhyu ◽  
Irma Finurina Mustikawati ◽  
Wiharto Wiharto
Keyword(s):  
Intervention Group ◽  
Depression Scale ◽  
Sampling Technique ◽  
Hemodialysis Patients ◽  
Anxiety And Depression ◽  
Control Group ◽  
Control Group Design ◽  
Hemodialysis Unit ◽  
Before And After ◽  
The Mean

Hemodialysis is a routinely performed therapy on chronic kidney patients, leading to psychological problems among subjects who undergo hemodialysis, such as anxiety and depression. One of the efforts to overcome anxiety and depression is with the dhikr intervention. Dhikr presents hearts to remember and be obedient to Allah followed by the words and actions in various conditions.  Discover dhikr's influence on the level of anxiety and depression in patients on hemodialysis in  Purwokerto Islamic Hospital. It was a quantitative study using quasi experiment with a non-quivalent control group design. The number of samples was 12 subjects consisting of 6 subjects in the control group and six subjects in the intervention group with the purposive sampling technique. Instruments were used to measure anxiety and depression are the HADS ( Hospital Anxiety and Depression Scale ) before and after dhikr intervention as much as 12 times. Research is carried out in Hemodialysis Unit of  Purwokerto Islamic Hospital. Test statistics on research are used paired t-test and independent t-tests. This study showed a decrease in the mean level of anxiety from 5, 83 to 1.67 in the experimental group ( p = 0.003). The mean level of depression experienced a decline of 9, 67 becomes 4.67 on a group experiment ( p = 0.003). Dhikr reduces the level of anxiety and depression in hemodialysis patients at the Purwokerto Islamic Hospital.

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