Participant Retention in Randomized, Controlled Trials: The Value of Relational Engagement

Although participant retention is critically important for the conduct of randomized controlled trials, relatively little is known about factors that lead to attrition. I argue that commonly used retention strategies are not cost effective in large trials because most participants do not value them. Attrition in experimental research occurs because we rely on conventional retention strategies and we neglect the unspoken values and beliefs of our participants. The most important of these is the value of relational engagement. Relational engagement does pose a potential threat to internal validity in trials with small sample sizes. This threat is discussed. In conclusion, I suggest that large trials should consider supporting relational engagement rather than spending money on conventional retention strategies.

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Enhancing Patient Centricity and Advancing Innovation in Clinical Research with Virtual Randomized Clinical Trials (vRCTs)

Diagnostics ◽

10.3390/diagnostics11020151 ◽

2021 ◽

Vol 11 (2) ◽

pp. 151

Author(s):

Raphael A. Yaakov ◽

Özgür Güler ◽

Tim Mayhugh ◽

Thomas E. Serena

Keyword(s):

Clinical Trials ◽

Randomized Controlled Trials ◽

Randomized Clinical Trials ◽

Regulatory Compliance ◽

Small Sample ◽

Controlled Trials ◽

Holistic View ◽

Health Crisis ◽

Randomized Controlled ◽

Healthcare Needs

The current public health crisis has highlighted the need to accelerate healthcare innovation. Despite unwavering levels of cooperation among academia, industry, and policy makers, it can still take years to bring a life-saving product to market. There are some obvious limitations, including lack of blinding or masking and small sample size, which render the results less applicable to the real world. Traditional randomized controlled trials (RCTs) are lengthy, expensive, and have a low success rate. There is a growing acknowledgement that the current process no longer fully meets the growing healthcare needs. Advances in technology coupled with proliferation of telehealth modalities, sensors, wearable and connected devices have paved the way for a new paradigm. Virtual randomized controlled trials (vRCTs) have the potential to drastically shorten the clinical trial cycle while maximizing patient-centricity, compliance, and recruitment. This new approach can inform clinical trials in real time and with a holistic view of a patient’s health. This paper provides an overview of virtual clinical trials, addressing critical issues, including regulatory compliance, data security, privacy, and ownership.

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MANAGEMENT OF ENDOCRINE DISEASE: Effects of telecare intervention on glycemic control in type 2 diabetes: a systematic review and meta-analysis of randomized controlled trials

Acta Endocrinologica ◽

10.1530/eje-14-0441 ◽

2015 ◽

Vol 172 (3) ◽

pp. R93-R101 ◽

Cited By ~ 42

Author(s):

Zhenru Huang ◽

Hong Tao ◽

Qingdong Meng ◽

Long Jing

Keyword(s):

Type 2 Diabetes ◽

Glycemic Control ◽

Randomized Controlled Trials ◽

Sample Size ◽

Plasma Glucose ◽

Small Sample ◽

Cochrane Library ◽

Controlled Trials ◽

Randomized Controlled

ObjectiveTo review the published literature on the effects of telecare intervention in patients with type 2 diabetes and inadequate glycemic control.Design and methodsA review of randomized controlled trials on telecare intervention in patients with type 2 diabetes, and a search of electronic databases such as The Cochrane Library, PubMed, EBSCO, CINAHL, Science Direct, Journal of Telemedicine and Telecare, and China National Knowledge Infrastructure (CNKI), were conducted from December 8 to 16, 2013. Two evaluators independently selected and reviewed the eligible studies. Changes in HbA1c, fasting plasma glucose (FPG), post-prandial plasma glucose (PPG), BMI, and body weight were analyzed.ResultsAn analysis of 18 studies with 3798 subjects revealed that telecare significantly improved the management of diabetes. Mean HbA1c values were reduced by −0.54 (95% CI, −0.75 to −0.34; P<0.05), mean FPG levels by −9.00 mg/dl (95% CI, −17.36 to −0.64; P=0.03), and mean PPG levels reduced by −52.86 mg/dl (95% CI, −77.13 to −28.58; P<0.05) when compared with the group receiving standard care. Meta-regression and subgroup analyses indicated that study location, sample size, and treatment-monitoring techniques were the sources of heterogeneity.ConclusionsPatients monitored by telecare showed significant improvement in glycemic control in type 2 diabetes when compared with those monitored by routine follow-up. Significant reduction in HbA1c levels was associated with Asian populations, small sample size, and telecare, and with those patients with baseline HbA1c greater than 8.0%.

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Treating COVID-19 With Hydroxychloroquine (TEACH): A Multicenter, Double-Blind Randomized Controlled Trial in Hospitalized Patients

Open Forum Infectious Diseases ◽

10.1093/ofid/ofaa446 ◽

2020 ◽

Vol 7 (10) ◽

Cited By ~ 5

Author(s):

Robert J Ulrich ◽

Andrea B Troxel ◽

Ellie Carmody ◽

Jaishvi Eapen ◽

Martin Bäcker ◽

...

Keyword(s):

Randomized Controlled Trials ◽

Disease Progression ◽

Severe Disease ◽

Small Sample ◽

Hospitalized Patients ◽

Controlled Trials ◽

Double Blind ◽

Randomized Controlled ◽

End Point ◽

Clinical Scores

Abstract Background Effective therapies to combat coronavirus 2019 (COVID-19) are urgently needed. Hydroxychloroquine (HCQ) has in vitro antiviral activity against severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), but the clinical benefit of HCQ in treating COVID-19 is unclear. Randomized controlled trials are needed to determine the safety and efficacy of HCQ for the treatment of hospitalized patients with COVID-19. Methods We conducted a multicenter, double-blind randomized clinical trial of HCQ among patients hospitalized with laboratory-confirmed COVID-19. Subjects were randomized in a 1:1 ratio to HCQ or placebo for 5 days and followed for 30 days. The primary efficacy outcome was a severe disease progression composite end point (death, intensive care unit admission, mechanical ventilation, extracorporeal membrane oxygenation, and/or vasopressor use) at day 14. Results A total of 128 patients were included in the intention-to-treat analysis. Baseline demographic, clinical, and laboratory characteristics were similar between the HCQ (n = 67) and placebo (n = 61) arms. At day 14, 11 (16.4%) subjects assigned to HCQ and 6 (9.8%) subjects assigned to placebo met the severe disease progression end point, but this did not achieve statistical significance (P = .350). There were no significant differences in COVID-19 clinical scores, number of oxygen-free days, SARS-CoV-2 clearance, or adverse events between HCQ and placebo. HCQ was associated with a slight increase in mean corrected QT interval, an increased D-dimer, and a trend toward an increased length of stay. Conclusions In hospitalized patients with COVID-19, our data suggest that HCQ does not prevent severe outcomes or improve clinical scores. However, our conclusions are limited by a relatively small sample size, and larger randomized controlled trials or pooled analyses are needed.

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Mind-Body Interventions, Psychological Stressors, and Quality of Life in Stroke Survivors

Stroke ◽

10.1161/strokeaha.118.021150 ◽

2019 ◽

Vol 50 (2) ◽

pp. 434-440 ◽

Cited By ~ 17

Author(s):

Mary F. Love ◽

Anjail Sharrief ◽

Alejandro Chaoul ◽

Sean Savitz ◽

Jennifer E. Sanner Beauchamp

Keyword(s):

Quality Of Life ◽

Randomized Controlled Trials ◽

Small Sample ◽

Controlled Trials ◽

Stroke Survivors ◽

Poststroke Depression ◽

Randomized Controlled ◽

Psychological Stressors ◽

Biological Outcomes

Background and Purpose—Psychological stressors, including poststroke depression, poststroke anxiety, and posttraumatic stress disorder, are highly prevalent in stroke survivors. These symptoms exact a significant toll on stroke survivors. Clinical and research efforts in stroke recovery focus on motor disability, speech and language deficits, and cognitive dysfunction while largely neglecting psychological stressors. Evidence suggests mind-body interventions in other chronic illness populations decrease symptoms of depression, regulate immune responses, and promote resilience, yet similar studies are lacking in stroke populations. This review aims to synthesize evidence of the effects of mind-body interventions on psychological stressors, quality of life, and biological outcomes for stroke survivors.Methods—A systematic search of PubMed, PsycINFO, and CINAHL was conducted from database inception to November 2017.Results—Eight studies were included in the review, with a total of 292 participants. Mind-body interventions included yoga or tai chi. Of the 5 included randomized controlled trials, most were pilot or feasibility studies with small sample sizes. Psychological stressors, including poststroke depression and anxiety, along with the quality of life, improved over time, but statistically significant between-group differences were largely absent. The 3 included studies with a qualitative design reported themes reflecting improvement in psychological stressors and quality of life. No included studies reported biological outcomes.Conclusions—Studies of mind-body interventions suggest a possible benefit on psychological stressors and quality of life; however, rigorously designed, sufficiently powered randomized controlled trials with mixed-methods design are warranted to delineate specific treatment effects of these interventions. Studies with both biological and psychological stressors as outcomes would provide evidence about interaction effects of these factors on stroke-survivor responses to mind-body interventions.

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Trigger Point Injections for Chronic Non-Malignant Musculoskeletal Pain: A Systematic Review

Pain Medicine ◽

10.1111/j.1526-4637.2008.00526.x ◽

2009 ◽

Vol 10 (1) ◽

pp. 54-69 ◽

Cited By ~ 92

Author(s):

N. Ann Scott ◽

Bing Guo ◽

Pamela M. Barton ◽

Robert D. Gerwin

Keyword(s):

Systematic Review ◽

Randomized Controlled Trials ◽

Musculoskeletal Pain ◽

Systematic Reviews ◽

Trigger Point ◽

Small Sample ◽

Controlled Trials ◽

Safe Procedure ◽

Randomized Controlled ◽

Published Evidence

ABSTRACT Objective. This systematic review assessed the available published evidence on the efficacy and safety of using trigger point injection (TPI) to treat patients with chronic non-malignant musculoskeletal pain that had persisted for at least 3 months. Methods. All published systematic reviews or randomized controlled trials detailing the use of TPI in patients with chronic, non-malignant musculoskeletal pain (persisting for >3 months) were identified by systematically searching literature databases and the Websites of various health technology assessment agencies, research registers, and guidelines sites up to July 2006. Results. Although no systematic reviews were identified, 15 peer-reviewed randomized controlled trials met the inclusion criteria. However, deficiencies in reporting, small sample sizes, and marked inter-study heterogeneity precluded a definitive synthesis of the data. TPI is a safe procedure when used by clinicians with appropriate expertise and training. It relieved symptoms when used as a sole treatment for patients with chronic head, neck, shoulder, and back pain or whiplash syndrome, regardless of the injectant used, and may be a useful adjunct to intra-articular injection in the treatment of osteoarthritis pain. Although the addition of TPI to stretching exercises augments treatment outcomes, this was also true of other therapies such as ultrasound and laser. Conclusion. The efficacy of TPI is no more certain than it was a decade ago as, overall, there is no clear evidence of either benefit or ineffectiveness. The only advantage of injecting anesthetic into trigger points may be to reduce the pain of the needling process, which may not be an insignificant benefit.

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Characteristic of Clinical Studies on Baduanjin during 2000–2019: A Comprehensive Review

Evidence-based Complementary and Alternative Medicine ◽

10.1155/2020/4783915 ◽

2020 ◽

Vol 2020 ◽

pp. 1-9

Author(s):

Jing Zhou ◽

Yunyang Yu ◽

Biwei Cao ◽

Xiaoya Li ◽

Miao Wu ◽

...

Keyword(s):

Randomized Controlled Trials ◽

Clinical Studies ◽

Methodological Quality ◽

Case Reports ◽

Case Series ◽

Small Sample ◽

Chronic Obstructive ◽

Controlled Trials ◽

Randomized Controlled

To date, a growing number of clinical studies have demonstrated the safety and health benefits from Baduanjin intervention. Based on this, our objective is to systematically retrieve and summarize the clinical studies on Baduanjin, with a view to providing more evidence-based evidence in support of the application of Baduanjin for healthcare, and to identify the shortcomings of existing research and provide feasibility suggest for further clinical research. Both four English language and four Chinese language electronic databases were used to search articles related to Baduanjin during 2000–2019. SPSS 22.0 software was used to analyze the data, and the risk of bias tool in the RevMan 5.3.5 software was used to evaluate the methodological quality of randomized controlled trials. A total of 810 publications were identified, including 43 (5.3%) systematic reviews, 614 (75.8%) randomized controlled trials, 66 (8.1%) nonrandomized controlled clinical studies, 84 (10.4%) case series, and 3 (0.4%) case reports. The top 10 diseases/conditions included diabetes, chronic obstructive pulmonary disease, hypertension, low back pain, neck pain, stroke, coronary heart disease, cognitive impairment, insomnia, and osteoporosis or osteopenia. The style of State General Administration of Sport of China in 2003 was the most commonly used version of Baduanjin, and Baduanjin was practiced with an average of 35 minutes, 1 or 2 times a day, 3–5 days per week, and a 18-week average duration. It is also worth noting that there were no serious adverse events related to Baduanjin intervention. Most studies were small sample size research, and the methodological quality of randomized controlled trials is generally low. The clinical studies of Baduanjin have a substantial quantity and evidence base. However, there are significant differences among different studies in the specific intervention measures such as style, intensity, duration, learning, and practice methods, which need to be further standardized and unified. Further high-quality designed and reporting studies are recommended to further validate the clinical benefits of Baduanjin.

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Efficiency and safety of TachoSil® in the treatment of postoperative air leakage following pulmonary surgery: a meta-analysis of randomized controlled trials

Japanese Journal of Clinical Oncology ◽

10.1093/jjco/hyz076 ◽

2019 ◽

Vol 49 (9) ◽

pp. 862-869

Author(s):

Jian Zhou ◽

Mengyuan Lyu ◽

Long Pang ◽

Yuanjing Gao ◽

Kang Ning ◽

...

Keyword(s):

Randomized Controlled Trials ◽

Meta Analysis ◽

Cost Effective ◽

Controlled Trials ◽

Air Leakage ◽

Randomized Controlled ◽

Pulmonary Surgery

TachoSil® was cost-effective and safe in preventing postoperative air leakage for the patients undergoing pulmonary surgery.

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Randomized Controlled Trials and Comparative Effectiveness Research

Journal of Clinical Oncology ◽

10.1200/jco.2012.42.2352 ◽

2012 ◽

Vol 30 (34) ◽

pp. 4194-4201 ◽

Cited By ~ 22

Author(s):

Olwen M. Hahn ◽

Richard L. Schilsky

Keyword(s):

Health Care ◽

Randomized Controlled Trials ◽

Comparative Effectiveness Research ◽

Comparative Effectiveness ◽

Cost Effective ◽

Health Care Research ◽

Controlled Trials ◽

Effectiveness Research ◽

Randomized Controlled

Comparative effectiveness research (CER) has been promoted as a way to improve the translation gap between clinical research and everyday clinical practice as well as to deliver more cost-effective health care. CER will account for a significant portion of funding allocated by the US government for health care research. Oncology has a rich history of improving clinical outcomes and advancing research through randomized controlled trials (RCTs). In this article, we review the role of RCTs in achieving the goals of CER, with particular emphasis on the role of publicly funded clinical trials.

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Acupuncture for tinnitus: a systematic review and meta-analysis of randomized controlled trials

Acupuncture in Medicine ◽

10.1177/0964528420938380 ◽

2020 ◽

pp. 096452842093838 ◽

Cited By ~ 1

Author(s):

Kaiyu Huang ◽

Shuang Liang ◽

Lei Chen ◽

Antoine Grellet

Keyword(s):

Randomized Controlled Trials ◽

Primary Outcome ◽

Meta Analysis ◽

Small Sample ◽

Controlled Trials ◽

Control Groups ◽

Positive Effects ◽

Randomized Controlled ◽

Vas Score ◽

Secondary Outcomes

Objective: To evaluate the efficacy/effectiveness of acupuncture for the treatment of tinnitus. Methods: Four English and four Chinese databases were searched for randomized controlled trials (RCTs) of acupuncture for tinnitus published before 30 September 2018. RCTs applying acupuncture alone compared with conventional treatments, sham acupuncture, or no treatment, as well as acupuncture plus conventional treatments compared with conventional treatments alone, were included. The primary outcome was the visual analogue scale (VAS). Secondary outcomes included tinnitus handicap inventory (THI) and tinnitus severity index (TSI) scores. Meta-analysis was conducted using RevMan V5.3 software. The protocol was registered in the PROSPERO database (ref. CRD42018108692). Results: Eight studies involving 504 participants were included. Meta-analysis showed no significant differences in the VAS score (mean difference (MD) = −1.81, 95% confidence interval (CI) = −3.69 to 0.07; p = 0.06) between the acupuncture and control groups. However, favorable effects of acupuncture on changes in THI score (MD = −10.11, 95% CI = −12.74 to −7.48; p < 0.001) and TSI score (MD = −8.36, 95% CI = −8.87 to −7.86; p < 0.001) were found. Conclusion: Acupuncture had no significant effect on the primary outcome of VAS score compared with control treatment; however, positive effects on secondary outcomes (THI and TSI score) were observed in acupuncture versus control groups. Due to the low quality and small sample size of the included trials, the level of evidence was insufficient to draw any definitive conclusions. Further rigorous and high-quality studies with larger sample sizes should be conducted to confirm the efficacy/effectiveness of acupuncture for tinnitus.

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Evidence-Based Medicine: Determining Quality of Randomized Controlled Trials

Guides Newsletter ◽

10.1001/amaguidesnewsletters.2014.julaug01 ◽

2014 ◽

Vol 19 (4) ◽

pp. 3-5

Author(s):

Melissa Cheng

Keyword(s):

Randomized Controlled Trials ◽

Evidence Based Medicine ◽

Internal Validity ◽

Cochrane Collaboration ◽

Dropout Rate ◽

Controlled Trials ◽

Evidence Based ◽

Double Blind ◽

Randomized Controlled ◽

Based Medicine

Abstract Evidence-based medicine is based on evidence gathered by randomized controlled trials (RCTs), which are considered the “gold standard” of research studies. Users of the medial literature must be able to read critically and evaluate RCTs. The present article uses the standards of the American College of Occupational and Environmental Medicine (ACOEM) practice guidelines in evaluating RCTs according to 11 criteria; these criteria follow those used by the Cochrane Collaboration in their evidence-based reviews. Well-written articles present their randomization schemes to create comparable groups, and studies must be controlled for co-interventions; in a double-blind trial, the co-interventions would be used equally in both groups, and treatment allocations should be concealed. Readers should ask if the study had acceptable compliance; that is, were patients doing what they were asked, and was the dropout rate acceptable (typically, less than 20%)? RCTs should be analyzed by an intention-to-treat analysis that includes all study subjects who were randomized, not just those who completed the study. Having high internal validity ensures a more accurate study that can be reproduced by others, so readers may ask if results are likely to be affected by observational bias, confounding, or chance variation. Readers can determine external validity by assessing study participants according to inclusion and exclusion criteria and baseline characteristics.

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