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BJPsych Open Jun 2024One in 57 children are diagnosed with autism in the UK, and the estimated cost for supporting these children in education is substantial. Social Stories™ is a...
BACKGROUND
One in 57 children are diagnosed with autism in the UK, and the estimated cost for supporting these children in education is substantial. Social Stories™ is a promising and widely used intervention for supporting children with autism in schools and families. It is believed that Social Stories™ can provide meaningful social information to children that can improve social understanding and may reduce anxiety. However, no economic evaluation of Social Stories has been conducted.
AIMS
To assess the cost-effectiveness of Social Stories through Autism Spectrum Social Stories in Schools Trial 2, a multi-site, pragmatic, cluster-randomised controlled trial.
METHOD
Children with autism who were aged 4-11 years were recruited and randomised ( = 249). Costs measured from the societal perspective and quality-adjusted life-years (QALYs) measured by the EQ-5D-Y-3L proxy were collected at baseline and at 6-month follow-up for primary analysis. The incremental cost-effectiveness ratio was calculated, and the uncertainty around incremental cost-effectiveness ratios was captured by non-parametric bootstrapping. Sensitivity analyses were performed to evaluate the robustness of the primary findings.
RESULTS
Social Stories is likely to result in a small cost savings (-£191 per child, 95% CI -767.7 to 337.7) and maintain similar QALY improvements compared with usual care. The probability of Social Stories being a preferred option is 75% if society is willing to pay £20 000 per QALY gained. The sensitivity analysis results aligned with the main study outcomes.
CONCLUSIONS
Compared with usual care, Social Stories did not lead to an increase in costs and maintained similar QALY improvements for primary-aged children with autism.
PubMed: 38826027
DOI: 10.1192/bjo.2024.47 -
Heart, Lung & Circulation May 2024Physical activity (PA) and weight management are critical for cardiovascular disease (CVD) secondary prevention. However, PA adherence during or after cardiac...
An Australian Football Themed Health Behaviour Change Intervention for Men With Cardiovascular Disease is Feasible and Acceptable: Results From a Feasibility Randomised Trial.
BACKGROUND
Physical activity (PA) and weight management are critical for cardiovascular disease (CVD) secondary prevention. However, PA adherence during or after cardiac rehabilitation is low. Here, we assess the feasibility and acceptability of the Australian football-themed Aussie Fans in Training (Aussie-FIT) program and associated trial procedures when adapted for men with CVD.
METHOD
A pragmatic randomised control trial, with waitlist control arm, and follow-up measures at 3 and 6 months. Men with a CVD diagnosis and body mass index ≥25 kg/m were recruited from community and clinical settings, and randomised, following baseline measures of health and health behaviours. The intervention arm attended 12 face-to-face football-themed education and PA sessions. Feasibility (recruitment, retention, attendance, and adherence to trial procedures) was assessed via mixed methods.
RESULTS
A total of 74% (64/86) of participants expressing interest met the eligibility criteria. Of those, 49 men (mean age=61.4, standard deviation=9.5, mean body mass index=31.3, standard deviation=4.2) were randomised. Program attendance rates (87% attended ≥80% of sessions) and retention (92%) were high. Trial retention at the primary end point (3 months) was high (86%) and at the 6-month follow-ups reduced to 67%. Program and trial procedures were acceptable, except for the request to visit a pathologist for the blood draw.
CONCLUSIONS
Using a football theme and setting may be a feasible way to engage men with CVD in health behaviour change. Given the existing pilot evidence for men at risk of CVD, and that recruitment rates were under the target, trialling a program for men with or at risk of CVD is recommended.
PubMed: 38824056
DOI: 10.1016/j.hlc.2024.03.012 -
Drug and Alcohol Dependence Jul 2024To evaluate the effects of booster and no booster versions of web-based alcohol Personalised Normative Feedback (PNF) and whether descriptive norms mediated and/or... (Randomized Controlled Trial)
Randomized Controlled Trial
BACKGROUND
To evaluate the effects of booster and no booster versions of web-based alcohol Personalised Normative Feedback (PNF) and whether descriptive norms mediated and/or participant motivation moderated the effectiveness of the intervention in real world conditions (i.e. no financial incentives).
METHODS
Pragmatic randomised controlled trial with 1-, 3-, and 6-month assessments. Brazilian college students reporting alcohol use in the last 12 months (N=931) were recruited from May/2020 to December/2022 and allocated to 1) No booster/single PNF(S-PNF); 2) Booster/multiple PNF(M-PNF); or 3) Assessment-only control. We applied Helmert coding [1: Any intervention (S-PNF or M-PNF) vs. Control; and 2: S-PNF vs. M-PNF].
PRIMARY OUTCOMES
typical number of drinks/week and maximum number of drinks/week; secondary outcomes: drinking frequency and number of consequences. Three-months assessment was the primary interval. Descriptive norms were tested as mediator. Interest, importance, and readiness to change were examined as moderators.
RESULTS
Compared to control, any intervention did not influence primary outcomes at 3-months or 6-months, but did at 1-month, when reduced typical drinking (IRR:0.77, 95%CI:0.66;0.90) and maximum number of drinks (IRR:0.69, 95%CI:0.58;0.82). There was an intervention effect on the consequences at 3-months. No differences were observed between S-PNF and M-PNF. No mediation effects were found at 3-months. At 6-months, there was an indirect effect on typical drinking through norms at 3-months (b=-0.82, 95%CI:-2.03;-0.12) and effects on maximum drinks through norms at 1-month (b=-0.54, 95%CI:-1.65;-0.02). No support for moderation was found.
CONCLUSIONS
Intervention reduced alcohol drinking at 1 month only and was not effective thereafter. Mechanisms of effect remain unclear.
Topics: Humans; Male; Female; Young Adult; Students; Alcohol Drinking in College; Universities; Adolescent; Internet-Based Intervention; Internet; Feedback, Psychological; Motivation; Alcohol Drinking; Brazil; Adult; Social Norms
PubMed: 38823192
DOI: 10.1016/j.drugalcdep.2024.111337 -
BMC Medical Research Methodology May 2024Inequities in health access and outcomes exist between Indigenous and non-Indigenous populations. Embedded pragmatic randomized, controlled trials (ePCTs) can test the...
BACKGROUND
Inequities in health access and outcomes exist between Indigenous and non-Indigenous populations. Embedded pragmatic randomized, controlled trials (ePCTs) can test the real-world effectiveness of health care interventions. Assessing readiness for ePCT, with tools such as the Readiness Assessment for Pragmatic Trials (RAPT) model, is an important component. Although equity must be explicitly incorporated in the design, testing, and widespread implementation of any health care intervention to achieve equity, RAPT does not explicitly consider equity. This study aimed to identify adaptions necessary for the application of the 'Readiness Assessment for Pragmatic Trials' (RAPT) tool in embedded pragmatic randomized, controlled trials (ePCTs) with Indigenous communities.
METHODS
We surveyed and interviewed participants (researchers with experience in research involving Indigenous communities) over three phases (July-December 2022) in this mixed-methods study to explore the appropriateness and recommended adaptions of current RAPT domains and to identify new domains that would be appropriate to include. We thematically analyzed responses and used an iterative process to modify RAPT.
RESULTS
The 21 participants identified that RAPT needed to be modified to strengthen readiness assessment in Indigenous research. In addition, five new domains were proposed to support Indigenous communities' power within the research processes: Indigenous Data Sovereignty; Acceptability - Indigenous Communities; Risk of Research; Research Team Experience; Established Partnership). We propose a modified tool, RAPT-Indigenous (RAPT-I) for use in research with Indigenous communities to increase the robustness and cultural appropriateness of readiness assessment for ePCT. In addition to producing a tool for use, it outlines a methodological approach to adopting research tools for use in and with Indigenous communities by drawing on the experience of researchers who are part of, and/or working with, Indigenous communities to undertake interventional research, as well as those with expertise in health equity, implementation science, and public health.
CONCLUSION
RAPT-I has the potential to provide a useful framework for readiness assessment prior to ePCT in Indigenous communities. RAPT-I also has potential use by bodies charged with critically reviewing proposed pragmatic research including funding and ethics review boards.
Topics: Humans; Indigenous Peoples; Pragmatic Clinical Trials as Topic; Health Services, Indigenous; Surveys and Questionnaires; Research Design; Health Services Accessibility; Randomized Controlled Trials as Topic
PubMed: 38822242
DOI: 10.1186/s12874-024-02244-z -
American Heart Journal May 2024Aortic valve replacement in asymptomatic severe aortic stenosis is controversial. The Early valve replacement in severe ASYmptomatic Aortic Stenosis (EASY-AS) trial aims...
BACKGROUND
Aortic valve replacement in asymptomatic severe aortic stenosis is controversial. The Early valve replacement in severe ASYmptomatic Aortic Stenosis (EASY-AS) trial aims to determine whether early aortic valve replacement improves clinical outcomes, quality of life and cost-effectiveness compared to a guideline recommended strategy of 'watchful waiting'.
METHODS
In a pragmatic international, open parallel group randomized controlled trial (NCT04204915), 2844 patients with severe aortic stenosis will be randomized 1:1 to either a strategy of early (surgical or transcatheter) aortic valve replacement or aortic valve replacement only if symptoms or impaired left ventricular function develop. Exclusion criteria include other severe valvular disease, planned cardiac surgery, ejection fraction <50%, previous aortic valve replacement or life expectancy <2 years. The primary outcome is a composite of cardiovascular mortality or heart failure hospitalization. The primary analysis will be undertaken when 663 primary events have accrued, providing 90% power to detect a reduction in the primary endpoint from 27.7% to 21.6% (hazard ratio 0.75). Secondary endpoints include disability-free survival, days alive and out of hospital, major adverse cardiovascular events and quality of life.
RESULTS
Recruitment commenced in March 2020 and is open in the UK, Australia, New Zealand and Serbia. Feasibility requirements were met in July 2022, and the main phase opened in October 2022, with additional international centers in set-up.
CONCLUSIONS
The EASY-AS trial will establish whether a strategy of early aortic valve replacement in asymptomatic patients with severe aortic stenosis reduces cardiovascular mortality or heart failure hospitalization and improves other important outcomes.
PubMed: 38821453
DOI: 10.1016/j.ahj.2024.05.013 -
RMD Open May 2024To compare the effectiveness of a strategy administering baricitinib versus one using TNF-inhibitors (TNFi) in patients with rheumatoid arthritis (RA) after conventional... (Randomized Controlled Trial)
Randomized Controlled Trial Comparative Study
PERFECTRA: a pragmatic, multicentre, real-life study comparing treat-to-target strategies with baricitinib versus TNF inhibitors in patients with active rheumatoid arthritis after failure on csDMARDs.
OBJECTIVE
To compare the effectiveness of a strategy administering baricitinib versus one using TNF-inhibitors (TNFi) in patients with rheumatoid arthritis (RA) after conventional synthetic disease-modifying antirheumatic drugs (csDMARDs) failure in a real-life treat-to-target (T2T) setting.
METHODS
Patients with biological and targeted synthetic DMARD (b/tsDMARD) naïve RA with disease duration ≤5 years without contraindications to b/tsDMARD were randomised to either TNFi or baricitinib when csDMARD failed to achieve disease control in a T2T setting. Changes in clinical and patient-reported outcome measures (PROMs) were assessed at 12-week intervals for 48 weeks. The primary endpoint was non-inferiority, with testing for superiority if non-inferiority is demonstrated, of baricitinib strategy in the number of patients achieving American College of Rheumatology 50 (ACR50) response at 12 weeks. Secondary endpoints included 28-joint count Disease Activity Score with C reactive protein (DAS28-CRP) <2.6, changes in PROMs and radiographic progression.
RESULTS
A total of 199 patients (TNFi, n=102; baricitinib, n=97) were studied. Both study groups were similar. Baricitinib was both non-inferior and superior in achieving ACR50 response at week 12 (42% vs 20%). Moreover, 75% of baricitinib patients achieved DAS28-CRP <2.6 at week 12 compared with 46% of TNFi patients. On secondary outcomes throughout the duration of the study, the baricitinib strategy demonstrated comparable or better outcomes than TNFi strategy. Although not powered for safety, no unexpected safety signals were seen in this relatively small group of patients.
CONCLUSION
Up to present, in a T2T setting, patients with RA failing csDMARDs have two main strategies to consider, Janus Kinases inhibitor versus bDMARDs (in clinical practice, predominantly TNFi). The PERFECTRA study suggested that starting with baricitinib was superior over TNFi in achieving response at 12 weeks and resulted in improved outcomes across all studied clinical measures and PROMs throughout the study duration in these patients.
Topics: Humans; Purines; Sulfonamides; Arthritis, Rheumatoid; Pyrazoles; Azetidines; Male; Female; Middle Aged; Antirheumatic Agents; Aged; Treatment Outcome; Tumor Necrosis Factor Inhibitors; Treatment Failure; Adult; Patient Reported Outcome Measures; Severity of Illness Index
PubMed: 38816210
DOI: 10.1136/rmdopen-2024-004291 -
Body Image Sep 2024This study evaluated Raising Confident Girls (RCG), delivered to mothers of Year 8 students (mean age 12.8-years) who were receiving the classroom-based Dove Confident...
This study evaluated Raising Confident Girls (RCG), delivered to mothers of Year 8 students (mean age 12.8-years) who were receiving the classroom-based Dove Confident Me (DCM) program. RCG is an interactive, multi-session intervention designed to improve body image in mothers in order to enhance their capacity to parent and role model this to their daughters. A pragmatic non-randomised controlled trial involved delivery of the program to mothers (n = 69) over three, 2-hour seminars in evenings, compared with a comparison group (n = 51). The study took place at an independent all-girls secondary school in Australia. Multilevel mixed modelling analyses compared pre- and post-test scores on standardized scales. Mothers who participated in the program had significantly higher scores on primary outcome variables of body esteem and body appreciation compared to the comparison group at post-test. Further, participation significantly improved mother's knowledge, confidence, and skills for parenting, and improved role modeling of positive body image. Mothers were well engaged, with low attrition rates, and rated the program highly. The RCG program was effective and engaging for mothers, offering deeper insight into improving parental engagement in body image interventions delivered within the school context.
Topics: Humans; Female; Body Image; Parenting; Mothers; Adolescent; Child; Adult; Self Concept; Mother-Child Relations; Australia; Schools
PubMed: 38815453
DOI: 10.1016/j.bodyim.2024.101718 -
Annals of Medicine Dec 2024Real-world data on tofacitinib's effectiveness is limited and mainly retrospective or registry-based. We elected to conduct a pragmatic prospective study to assess the...
Tofacitinib is an effective treatment for moderate to severe ulcerative colitis, and intestinal ultrasound can discriminate response from non-response: a pragmatic prospective real-world study.
INTRODUCTION
Real-world data on tofacitinib's effectiveness is limited and mainly retrospective or registry-based. We elected to conduct a pragmatic prospective study to assess the efficacy of tofacitinib for moderate to severe ulcerative colitis (UC), aiming to evaluate the ability of intestinal ultrasound (IUS) to discriminate responders vs. non-responders in real-time.
METHODS
This pragmatic prospective clinical study included consecutive adult patients starting tofacitinib treatment for active moderate to severe UC. Patients were evaluated at baseline and after 8 weeks of tofacitinib (clinical, biomarker, endoscopy, and IUS). The primary outcome was clinical response defined by a decrease in the full Mayo score (fMS) of ≥3 at week 8. Next, we explored ultrasonographic parameters in the sigmoid colon as potential real-time classifiers to differentiate between responders and non-responders at week 8.
RESULTS
Overall, 30 adult patients started tofacitinib; the median age was 26.3 years (IQR 22.5-39.8), and 50% were female. Most patients (86.6%) had left-sided or extensive colitis, 96.7% had previously failed biologic therapy, and 60% (18/30) were on oral corticosteroids at the start of tofacitinib. At week 8, clinical response (a decrease in the fMS ≥ 3) and remission (fMS ≤ 2) rates were 40% (12/30) and 20% (6/30), respectively. Biomarker response (FC < 250µg/g) and biomarker normalization (FC ≤ 100µg/g) were achieved in 47.6% (10/21) and 38.1% (8/21) of patients, respectively. Endoscopic healing (endoscopic Mayo sub-score [EMS] ≤ 1) was achieved in 33.3% (10/30) of patients. Sigmoid bowel wall normalization as assessed by IUS (sBWT ≤ 3) was achieved in 18.2% (4/22). The best sBWT cut-off at week 8 to accurately classify endoscopic healing vs. no healing was a sBWT of 3.6 mm (AUC of 0.952 [95% CI: 0.868-1.036], < 0.001).
CONCLUSION
In this real-world pragmatic prospective study, tofacitinib was an effective treatment for moderate to severe UC, and IUS at week 8 accurately discriminated treatment response from non-response.
Topics: Humans; Piperidines; Colitis, Ulcerative; Female; Male; Pyrimidines; Prospective Studies; Adult; Ultrasonography; Treatment Outcome; Young Adult; Severity of Illness Index; Protein Kinase Inhibitors
PubMed: 38813808
DOI: 10.1080/07853890.2024.2358183 -
Journal of Pain Research 2024Despite its well-acknowledged analgesic potential, curcumin's low bioavailability has been recognized. Piperine, a substance naturally contained in pepper, has been...
Analgesic Potential Comparison Between Piperine-Combined Curcumin Patch and Non-Piperine Curcumin Patch: A Pragmatic Trial on Post-Cleft Lip/Palate Surgery Pediatric Patients.
PURPOSE
Despite its well-acknowledged analgesic potential, curcumin's low bioavailability has been recognized. Piperine, a substance naturally contained in pepper, has been known for its effect on increasing curcumin bioavailability. To investigate the analgesic potential of curcumin and piperine addition to curcumin patch used as adjuvant therapy in the management of acute postoperative orofacial pain.
PATIENTS AND METHODS
This pragmatic trial recruited 75 patients that underwent oromaxillofacial surgery at Unpad Dental Hospital, Bandung, Indonesia. Research participants were randomly assigned to three different groups: the first group that did not receive any intervention other than the post-operative standard treatment (POST), the second group that received POST and non-piperine curcumin patch, and the third group that received POST and piperine-combined curcumin patch. Participants' pain intensity was evaluated by using the face, leg, activity, cry, and consolability (FLACC) pain scale and salivary prostaglandin-E (PGE) level for two-time points, which were eight hours apart. All data were gathered and analyzed to compare the within and between-group differences.
RESULTS
Within groups comparison of the FLACC scores for two evaluation points showed significant differences for all groups (p < 0.01). For salivary PGE2 analysis, a comparison of the non-piperine group to the piperine group also showed significant results. Yet, when all three groups were compared, regardless of the differences, the results were not statistically significant.
CONCLUSION
Despite of the proven efficacy of curcumin patch, the addition of piperine to the curcumin patch in the current study did not provide any significant effects. Further investigation is of importance.
PubMed: 38812820
DOI: 10.2147/JPR.S463159 -
Canadian Journal of Kidney Health and... 2024There is little evidence on the ideal frequency of routine blood work in maintenance dialysis patients to manage complications, including anemia, mineral bone disease...
INTRODUCTION
There is little evidence on the ideal frequency of routine blood work in maintenance dialysis patients to manage complications, including anemia, mineral bone disease (MBD), and hyperkalemia. Recent quality improvement studies from Ontario showed no negative impacts when decreasing the frequency from monthly to every 6 weeks in conventional in-center hemodialysis (ICHD) patients. In December 2020, Alberta Kidney Care-South (AKC-S) reduced the frequency of routine blood work from every 6 weeks to every 8 weeks for ICHD patients.
OBJECTIVE
We aimed to assess the impact of reducing blood work frequency on patient outcomes.
METHODS
We compared prevalent AKC-S ICHD patients in 2 cohorts: (1) retrospective control (October 31, 2019-October 31, 2020) and (2) prospective intervention (December 1, 2020-December 1, 2021). Primary outcomes were true frequency of routine blood work, odds of patients being within target for anemia and MBD, and proportion of lab values of hyperkalemia. Furthermore, we compared hospitalizations and mortality.
RESULTS
A total of 972 patients in Calgary's ICHD program were included, 787 in each period (with 602 patients overlapping both cohorts). The frequency of routine blood work decreased from every 39.5 days in the control period to every 54.2 days in the intervention period ( < .01). There was a reduction in the odds of phosphate values in targets ( = .02), and an increase in the odds of labs with hyperkalemia (>6.0 mmol/L) during the intervention period ( = .01). There was no significant change in the odds of being within the accepted targets during the intervention period compared with the control period for hemoglobin, Tsat, calcium, or parathyroid hormone (PTH). Fewer patients were hospitalized during the intervention period and the risk of death decreased as well, although additional factors such as the COVID-19 pandemic may have affected this. A cost-savings of $32 962 occurred from the reduced anemia and MBD blood work during the intervention period.
CONCLUSIONS
When ICHD units in Calgary reduced routine blood work frequency from every 6 weeks to 8 weeks, there were no negative impacts on hospitalizations or deaths. A slightly lower proportion of phosphate values were within target, and a 0.7% increase in potassium values greater than 6 mmol/L was demonstrated. Our study suggests that blood work frequency in ICHD dialysis patients may be further reduced to every 8 weeks safely. Ultimately, additional pragmatic trials are needed to identify the optimal frequency of routine blood work.
PubMed: 38812721
DOI: 10.1177/20543581241255784