When is a randomised controlled trial health equity relevant? Development and validation of a conceptual framework

BackgroundRandomised controlled trials can provide evidence relevant to assessing the equity impact of an intervention, but such information is often poorly reported. We describe a conceptual framework to identify health equity-relevant randomised trials with the aim of improving the design and reporting of such trials.MethodsAn interdisciplinary and international research team engaged in an iterative consensus building process to develop and refine the conceptual framework via face-to-face meetings, teleconferences and email correspondence, including findings from a validation exercise whereby two independent reviewers used the emerging framework to classify a sample of randomised trials.ResultsA randomised trial can usefully be classified as ‘health equity relevant’ if it assesses the effects of an intervention on the health or its determinants of either individuals or a population who experience ill health due to disadvantage defined across one or more social determinants of health. Health equity-relevant randomised trials can either exclusively focus on a single population or collect data potentially useful for assessing differential effects of the intervention across multiple populations experiencing different levels or types of social disadvantage. Trials that are not classified as ‘health equity relevant’ may nevertheless provide information that is indirectly relevant to assessing equity impact, including information about individual level variation unrelated to social disadvantage and potentially useful in secondary modelling studies.ConclusionThe conceptual framework may be used to design and report randomised trials. The framework could also be used for other study designs to contribute to the evidence base for improved health equity.

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Patient Preferences in Randomised Trials: Threat or Opportunity?

Journal of Health Services Research & Policy ◽

10.1177/135581969600100403 ◽

1996 ◽

Vol 1 (4) ◽

pp. 194-197 ◽

Cited By ~ 78

Author(s):

David J. Torgerson ◽

Jennifer Klaber-Moffett ◽

Ian T. Russell

Keyword(s):

Back Pain ◽

External Validity ◽

Controlled Trial ◽

Randomised Trial ◽

Final Analysis ◽

Exercise Programme ◽

Randomised Trials ◽

Prognostic Variables ◽

New Treatment ◽

Randomised Controlled

Objectives: To assess whether it is feasible to elicit patients’ preferences for treatments and then to proceed with randomisation which may allocate those with preferences to their less preferred treatment; and to describe which prognostic variables were associated with such preferences within the context of a randomised trial of an exercise programme for back pain. Methods: The first 97 patients enrolled in a randomised controlled trial (RCT) for the treatment of back pain were asked about their preferences, health characteristics and other prognostic variables. Results: Fifty-eight (60%) patients preferred to be allocated to the exercise programme whilst 38 (39%) were indifferent; one patient preferred conventional general practitioner (GP) management. No patient refused randomisation. Comparing patients preferring the exercise programme with indifferent patients showed that the former had a higher belief in the effectiveness of the new treatment ( P > 0.01), tended to have worse back pain ( P = 0.09), had back pain for a shorter duration ( P = 0.04), and tended to have had more GP home visits ( P = 0.06). Conclusions: For many randomised trials preference may be an important prognostic variable. In such circumstances, preference should be taken into account in the final analysis. This study demonstrates it is sometimes feasible to randomise patients to their less preferred treatment, thus allowing more robust statistical comparisons between randomised groups. This modification may make RCTs more rigorous and improve their external validity.

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Web-based early intervention for children with motor difficulties aged 3–8 years old using multimodal rehabilitation (WECARE): protocol of a patient-centred pragmatic randomised trial of paediatric telerehabilitation to support families

BMJ Open ◽

10.1136/bmjopen-2020-046561 ◽

2021 ◽

Vol 11 (4) ◽

pp. e046561

Author(s):

Chantal Camden ◽

Jill G Zwicker ◽

Melanie Morin ◽

Tibor Schuster ◽

Melanie Couture ◽

...

Keyword(s):

Early Intervention ◽

Controlled Trial ◽

Randomised Trial ◽

Parental Knowledge ◽

Performance Measure ◽

Performance Goals ◽

Web Based ◽

Study Results ◽

Motor Difficulties ◽

Randomised Controlled

IntroductionMild motor difficulties in children are underdiagnosed despite being highly prevalent, leaving such children often underserved and at higher risk for secondary consequences such as cardiovascular disease and anxiety. Evidence suggests that early patient-oriented interventions, coaching parents and providing children with early stimulation should be provided, even in the absence of a diagnosis. Such interventions may be effectively delivered via telerehabilitation.Methods and analysisA family-centred, pragmatic randomised controlled trial will be carried out to evaluate the real-world effectiveness of a Web-based Early intervention for Children using multimodAl REhabilitation (WECARE). Families of children with motor difficulties, 3–8 years of age, living in Quebec, Canada, and receiving no public rehabilitation services (n=118) will be asked to determine up to 12 performance goals, evaluated using the Canadian Occupational Performance Measure (COPM, the primary outcome). Families will be randomised to receive either usual care or the WECARE intervention. The WECARE intervention will be delivered for 1 year via a web-based platform. Families will have access to videoconferences with an assigned rehabilitation therapist using a collaborative coaching approach, a private chat function, a forum open to all intervention arm participants and online resources pertaining to child development. Participants will be asked to re-evaluate the child’s COPM performance goals every 3 months up to 1 year post allocation. The COPM results will be analysed using a mixed Poisson regression model. Secondary outcomes include measures of the child’s functional ability, parental knowledge and skills and health-related quality of life, as well as qualitative outcomes pertaining to parental satisfaction and service delivery trajectories. Investigators and quantitative data analysts will be blinded to group allocation.Ethics and disseminationThe CIUSSS de l’Estrie—CHUS ethics committee approved this trial (2020-3429). Study results will be communicated via peer-reviewed journal publications, conference presentations and stakeholder-specific knowledge transfer activities.Trial registration numberNCT04254302.

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Methods for delivering the UK's multi-centre prison-based naloxone-on-release pilot randomised trial (N-ALIVE): Europe's largest prison-based randomised controlled trial

Drug and Alcohol Review ◽

10.1111/dar.12592 ◽

2017 ◽

Vol 37 (4) ◽

pp. 487-498 ◽

Cited By ~ 4

Author(s):

Angela Mary Meade ◽

Sheila Macdonald Bird ◽

John Strang ◽

Tracey Pepple ◽

Laura Lea Nichols ◽

...

Keyword(s):

Randomised Controlled Trial ◽

Controlled Trial ◽

Randomised Trial ◽

Randomised Controlled

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Clinically, what ought we to believe?

Australasian Psychiatry ◽

10.1177/1039856218810160 ◽

2018 ◽

Vol 27 (1) ◽

pp. 72-74 ◽

Cited By ~ 1

Author(s):

John D Little ◽

Lorraine Davison ◽

Robert D Little

Keyword(s):

Randomised Controlled Trial ◽

Controlled Trial ◽

Relevant Information ◽

Evidence Base ◽

Social Interventions ◽

Hierarchy Of Evidence ◽

Differences Of Opinion ◽

The Status ◽

Randomised Controlled ◽

Clinical Strategy

Objective: To question the status of the randomised controlled trial (RCT) in the hierarchy of evidence. Conclusions: The RCT provides important and clinically relevant information, particularly in psychopharmacology. However, and as with other methodologies, RCTs too are flawed and automatic abdication to their conclusions, especially in complex social interventions, is unwise. A clinical example with conflicting and polarising views, each with their evidence base, is described alongside a suggested clinical strategy for resolving differences of opinion.

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Drooling Reduction Intervention randomised trial (DRI): comparing the efficacy and acceptability of hyoscine patches and glycopyrronium liquid on drooling in children with neurodisability

Archives of Disease in Childhood ◽

10.1136/archdischild-2017-313763 ◽

2017 ◽

Vol 103 (4) ◽

pp. 371-376 ◽

Cited By ~ 12

Author(s):

Jeremy R Parr ◽

Emma Todhunter ◽

Lindsay Pennington ◽

Deborah Stocken ◽

Jill Cadwgan ◽

...

Keyword(s):

Side Effects ◽

Controlled Trial ◽

Symptom Control ◽

Randomised Trial ◽

Frequency Scale ◽

Skin Patch ◽

Impact Scale ◽

Significant Difference ◽

Secondary Outcomes ◽

Randomised Controlled

ObjectiveInvestigate whether hyoscine patch or glycopyrronium liquid is more effective and acceptable to treat drooling in children with neurodisability.DesignMulticentre, single-blind, randomised controlled trial.SettingRecruitment through neurodisability teams; treatment by parents.ParticipantsNinety children with neurodisability who had never received medication for drooling (55 boys, 35 girls; median age 4 years). Exclusion criteria: medication contraindicated; in a trial that could affect drooling or management.InterventionChildren were randomised to receive a hyoscine skin patch or glycopyrronium liquid. Dose was increased over 4 weeks to achieve optimum symptom control with minimal side-effects; steady dose then continued to 12 weeks.Primary and secondary outcomesPrimary outcome: Drooling Impact Scale (DIS) score at week-4. Secondary outcomes: change in DIS scores over 12 weeks, Drooling Severity and Frequency Scale and Treatment Satisfaction Questionnaire for Medication; adverse events; children’s perception about treatment.ResultsBoth medications yielded clinically and statistically significant reductions in mean DIS at week-4 (25.0 (SD 22.2) for hyoscine and 26.6 (SD 16) for glycopyrronium). There was no significant difference in change in DIS scores between treatment groups. By week-12, 26/47 (55%) children starting treatment were receiving hyoscine compared with 31/38 (82%) on glycopyrronium. There was a 42% increased chance of being on treatment at week-12 for children randomised to glycopyrronium relative to hyoscine (1.42, 95% CI 1.04 to 1.95).ConclusionsHyoscine and glycopyrronium are clinically effective in treating drooling in children with neurodisability. Hyoscine produced more problematic side effects leading to a greater chance of treatment cessation.Trial registration numbersISRCTN75287237; EUDRACT: 2013-000863-94; Medicines and Healthcare Products Regulatory Agency: 17136/0264/001-0003

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Effects of injectable progestogen contraception versus the copper intrauterine device on HIV acquisition: sub-study of a pragmatic randomised controlled trial

Journal of Family Planning and Reproductive Health Care ◽

10.1136/jfprhc-2016-101607 ◽

2017 ◽

Vol 43 (3) ◽

pp. 175-180 ◽

Cited By ~ 10

Author(s):

G Justus Hofmeyr ◽

Mandisa Singata-Madliki ◽

Theresa A Lawrie ◽

Eduardo Bergel ◽

Marleen Temmerman

Keyword(s):

Randomised Controlled Trial ◽

South African ◽

Pregnancy Termination ◽

Controlled Trial ◽

Intrauterine Device ◽

Randomised Trial ◽

Contraceptive Device ◽

Increased Risk ◽

Hiv Acquisition ◽

Randomised Controlled

BackgroundEvidence from observational studies suggests an increased risk of HIV acquisition among women using depot medroxyprogesterone acetate (DMPA) contraception.MethodsWithin the context of a South African programme to increase women's access to the intrauterine contraceptive device (IUD), we conducted a pragmatic, open-label, parallel-arm, randomised controlled trial (RCT) of the IUD versus injectable progestogen contraception (IPC) at two South African hospitals. The primary outcome was pregnancy; secondary outcomes included HIV acquisition. Consenting women attending termination of pregnancy services were randomised after pregnancy termination between July 2009 and November 2012. Condoms were promoted for the prevention of sexually transmitted infections. Voluntary HIV testing was offered at baseline and at 12 or more months later. Findings on HIV acquisition are reported in this article.ResultsHIV acquisition data were available for 1290 initially HIV-negative women who underwent a final study interview at a median of 20 months after randomisation to IPC or an IUD. Baseline group characteristics were comparable. In the IPC group, 545/656 (83%) of participants received DMPA, 96 (15%) received injectable norethisterone enanthate, 14 (2%) received the IUD and one received oral contraception. In the IUD group 609 (96%) received the IUD, 20 (3%) received IPC and 5 (1%) had missing data. According to intention-to-treat analysis, HIV acquisition occurred in 20/656 (3.0%) women in the IPC arm and 22/634 (3.5%) women in the IUD arm (IPC vs IUD, risk ratio 0.88; 95% confidence interval 0.48–1.59;p=0.7).ConclusionsThis sub-study was underpowered to rule out moderate differences in HIV risk, but confirms the feasibility of randomised trial methodology to address this question. Larger RCTs are needed to determine the relative risks of various contraceptive methods on HIV acquisition with greater precision.Trial registration numberPan African Clinical Trials Registry number PACTR201409000880157 (04-09-2014).

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Longitudinal Study of music Therapy’s Effectiveness for Premature infants and their caregivers (LongSTEP): protocol for an international randomised trial

BMJ Open ◽

10.1136/bmjopen-2018-025062 ◽

2019 ◽

Vol 9 (8) ◽

pp. e025062 ◽

Cited By ~ 5

Author(s):

Claire Ghetti ◽

Łucja Bieleninik ◽

Mari Hysing ◽

Ingrid Kvestad ◽

Jörg Assmus ◽

...

Keyword(s):

Preterm Infants ◽

Neonatal Intensive Care ◽

Infant Development ◽

Controlled Trial ◽

Randomised Trial ◽

Developmentally Appropriate ◽

Well Being ◽

Positive Effects ◽

Implementation And Dissemination ◽

Randomised Controlled

IntroductionPreterm birth has major medical, psychological and socioeconomic consequences worldwide. Music therapy (MT) has positive effects on physiological measures of preterm infants and maternal anxiety, but rigorous studies including long-term follow-up are missing. Drawing on caregivers’ inherent resources, this study emphasises caregiver involvement in MT to promote attuned, developmentally appropriate musical interactions that may be of mutual benefit to infant and parent. This study will determine whether MT, as delivered by a qualified music therapist during neonatal intensive care unit (NICU) hospitalisation and/or in home/municipal settings following discharge, is superior to standard care in improving bonding between primary caregivers and preterm infants, parent well-being and infant development.Methods and analysisDesign:international multicentre, assessor-blind, 2×2 factorial, pragmatic randomised controlled trial; informed by a completed feasibility study.Participants:250 preterm infants and their parents.Intervention:MT focusing on parental singing specifically tailored to infant responses, will be delivered during NICU and/or during a postdischarge 6-month period.Primary outcome:changes in mother–infant bonding at 6-month corrected age (CA), as measured by the Postpartum Bonding Questionnaire.Secondary outcomes: mother–infant bonding at discharge and at 12-month CA; child development over 24 months; and parental depression, anxiety and stress, and infant rehospitalisation, all over 12 months.Ethics and disseminationThe Regional Committees for Medical and Health Research Ethics approved the study (2018/994/REK Nord, 03 July 2018). Service users were involved in development of the study and will be involved in implementation and dissemination. Dissemination of findings will apply to local, national and international levels.Trial registration numberNCT03564184

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Endoscopic sutured gastroplasty in addition to lifestyle modification: short-term efficacy in a controlled randomised trial

Gut ◽

10.1136/gutjnl-2020-322026 ◽

2020 ◽

pp. gutjnl-2020-322026

Author(s):

Vincent Huberty ◽

Ivo Boskoski ◽

Vincenzo Bove ◽

Pauline Van Ouytsel ◽

Guido Costamagna ◽

...

Keyword(s):

Weight Loss ◽

Lifestyle Modification ◽

Controlled Trial ◽

Randomised Trial ◽

Body Weight Loss ◽

Total Body Weight ◽

Control Group ◽

Short Term ◽

Randomised Controlled

ObjectiveEndoscopic suture gastroplasty (ESG) has been developed as an alternative treatment for moderately obese patients. We present our results of a short-term randomised controlled trial on a new suturing technique, the Endomina system (E-ESG, Endo Tools therapeutics, Belgium).DesignEligible patients (body mass index 30–40 kg/m2) were randomised in a 2:1 ratio to receive lifestyle modification plus E-ESG or lifestyle modification alone (control group); dietetic counselling and follow-up were identical. Endpoints included a mean excess weight loss (EWL) of more than 25% 12 months after E-ESG and a 15% EWL difference at 6 months between groups. At 6 months, a cross-over to E-ESG was offered to the control group. All patients were followed for a total of 12 months after E-ESG.ResultsOf the 71 patients included (five male, mean age 40 years), mean EWL at 6 months was significantly higher in the treatment (38.6%, n=45) than in the control group (13.4%, n=21; p<0.001). At 6 months, satiety tests demonstrated a higher decrease in mean volume (41% vs 2.5%, p<0.001), and mean quality of life (QoL) was also higher in the treatment group (52.8 vs 45.1 p<0.05). No procedure-related or device-related severe adverse events were observed. Twelve months follow-up after E-ESG showed a mean EWL of 45.1%, which translated into a total body weight loss of 11.8%.ConclusionsThis study demonstrates that E-ESG is safe and effective, providing a 25% better EWL at 6 months than lifestyle modification alone. This weight loss was maintained and resulted in a significant improvement in QoL up to 18 months after treatment.Trial registration numberNCT03255005.

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A Randomised Controlled Trial of the Effect of Esmarch Tourniquet Use on Blood Loss, Bruising and Quality of Life in Elective Varicose Vein Surgery

Phlebology The Journal of Venous Disease ◽

10.1177/026835550201700104 ◽

2002 ◽

Vol 17 (1) ◽

pp. 10-12

Author(s):

A. Fowler ◽

M. Stechman ◽

D. Mitchell

Keyword(s):

Quality Of Life ◽

Blood Loss ◽

Varicose Vein ◽

Controlled Trial ◽

Randomised Trial ◽

Varicose Vein Surgery ◽

Clinical Value ◽

Prospective Randomised Trial ◽

Randomised Controlled

Objective: To assess the effect of tourniquet use on bleeding, bruising and quality of life in varicose vein surgery. Method: A prospective randomised trial of 49 patients undergoing varicose vein surgery was carried out. All patients were having single leg surgery as a day case. Blood loss was assessed by weighing swabs. Bruising was measured at 10 days, and quality of life assessed with SF36 questionaires. Synthesis: In the tourniquet group blood loss was significantly reduced but not the area of bruising. The SF36 scores showed deterioration in the non-tourniquet group at 10 days but this was not present at 6 weeks. We saw no complications of tourniquet use but the study was limited by disappointing recruitment reducing the power. Conclusion: Tourniquet use may be of clinical value. A larger study is required to assess the significance.

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Chewing gum to treat postoperative nausea and emesis in female patients (CHEWY): rationale and design for a multicentre randomised trial

BMJ Open ◽

10.1136/bmjopen-2018-027505 ◽

2019 ◽

Vol 9 (6) ◽

pp. e027505

Author(s):

Jai Darvall ◽

Britta Sylvia von Ungern-Sternberg ◽

Sabine Braat ◽

David Story ◽

Andrew Davidson ◽

...

Keyword(s):

General Anaesthesia ◽

Postoperative Nausea ◽

Controlled Trial ◽

Randomised Trial ◽

Ethics Committees ◽

Complete Response ◽

Chewing Gum ◽

Female Patients ◽

Patient Dissatisfaction ◽

Randomised Controlled

IntroductionPostoperative nausea, retching and vomiting (PONV) remains one of the most common side effects of general anaesthesia, contributing significantly to patient dissatisfaction, cost and complications. Chewing gum has potential as a novel, drug-free alternative treatment. We aim to conduct a large, definitive randomised controlled trial of the efficacy and safety of peppermint-flavoured chewing gum to treat PONV in the postanaesthesia care unit (PACU). If chewing gum is shown to be as effective as ondansetron, this trial has the potential to significantly improve outcomes for tens of millions of surgical patients around the world each year.Methods and analysisThis is a prospective, multicentre, randomised controlled non-inferiority trial. 272 female patients aged ≥12 years having volatile anaesthetic-based general anaesthesia for breast or laparoscopic surgery will be randomised. Patients experiencing nausea, retching or vomiting in PACU will be randomised to 15 min of chewing gum or 4 mg intravenous ondansetron. The primary outcome (complete response) is cessation of PONV within 2 hours of administration, with no recurrence nor rescue medication requirement for 2 hours after administration.Ethics and disseminationThe Chewy Trial has been approved by the Human Research Ethics Committees at all sites. Dissemination will be via international and national anaesthesia conferences, and publication in the peer-reviewed literature.Trial registration numberACTRN12618000429257; Pre-results.

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