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Response-adaptive randomisation
Response-adaptive randomisation [Response-adaptive randomisation will be used to update randomisation probabilities as follows:
PMC10699085
Ineligible or unavailable domains
allergies
ADVERSE EVENTS, SECONDARY, ALLERGIES
A participant may be ineligible for a specific domain because of contraindications (e.g. allergies, intolerances, adverse events) or non-contraindications (e.g. lack of access, clinician discretion, site opted out of domain). Domain ineligibilities will be incorporated by including the If response-adaptive randomisatio...
PMC10699085
Scheduled updates and decision rules
The SNAP trial is designed to be perpetual and answer both clinician and consumer priority-driven research questions for
PMC10699085
Data source
SECONDARY
Allocation to randomised interventions remains concealed until the time it is revealed to site investigators. The assessment of primary and secondary outcomes by site investigators will not be blinded to knowledge of allocated intervention. All trial and site investigators will remain blinded to aggregated trial outcom...
PMC10699085
Posterior summaries
The primary endpoint is all-cause mortality at 90 days after platform entry. The primary contrast of interest (i.e. the The full list of endpoints, population summaries, and populations within the estimand framework can be found in the core protocol, domain-specific appendices, and the statistical appendix (see The pos...
PMC10699085
Decision rules
The posterior distribution of the odds ratio for the primary endpoint forms the basis of the decision rules for concluding
PMC10699085
Superiority
Clinically, the superiority of any intervention Similarly, the stopping decision for futility of the superiority objective will be made if, at a scheduled analysis, the posterior probability of superiority, for the adult subgroup, is less than
PMC10699085
Non-inferiority
Clinically, the non-inferiority of any intervention Similarly, the stopping decision for futility of the non-inferiority objective will be made if, at a scheduled analysis, the posterior probability of non-inferiority, for the adult subgroup, is less than
PMC10699085
Frequency of scheduled updates
Scheduled updates will be made each time that an additional 500 randomised participants, including both adults and children, have completed 90 days of follow-up from platform entry. Extensive simulations have led to this schedule that, along with prespecified decision rules, maintains a low probability of declaring a f...
PMC10699085
Introducing new interventions
If a new intervention is introduced to an active subdomain (a subdomain with at least two interventions), then randomisation will be fixed for the new intervention in order to guarantee a sufficient allocation to the new intervention. If upon introducing the new intervention to a subdomain indexed by
PMC10699085
Trial conclusions and reporting
RECRUITMENT
At each scheduled analysis, the SNAP trial analytic team will prepare an unblinded report for the data safety and monitoring committee. If stopping rules are satisfied, the data safety and monitoring committee will make recommendations to the global trial steering committee on the public disclosure of results and/or th...
PMC10699085
Changes to prespecified analyses
SECONDARY
The unblinded SNAP trial analytic team will monitor the primary and secondary model behaviour, including numerical stability and scientific appropriateness. Alternative models may be needed if there are unforeseen numerical, data, or modelling problems. Any alternatives will be implemented by the SNAP trial analytic te...
PMC10699085
Initial implementation
PMC10699085
Trial structure
DISEASE
The initial SNAP trial silo, domain, and subdomain structure at commencement is summarised in Table The primary endpoint is all-cause mortality at 90 days after platform entry.Each subdomain initially consists of two interventions only.The subdomains of each silo within the backbone antibiotic domain are unique.For the...
PMC10699085
Primary model
The model of the primary endpoint is a special case of (Note that the parameters in (
PMC10699085
Prior distributions and model hierarchy
The model hierarchy and prior structure is specified as detailed in the ‘
PMC10699085
Decision rule thresholds
The decision rule thresholds are set at 0.99 for each of the superiority,
PMC10699085
Current state
MAY, RECRUITMENT
The SNAP trial opened to recruitment on 16 February 2022. As of the date of the first scheduled analysis (31 May 2023), the trial has initiated all domains and treatments as described in Table
PMC10699085
Concluding remarks
We have provided a detailed description of the statistical principles of the SNAP trial. Rather than provide a comprehensive statistical analysis plan, our intention was to describe the trial design and structure using relatively formal language so that the different components of the trial could be referred to unambig...
PMC10699085
Acknowledgements
ROBINSON
Thanks go to the SNAP trial participants and the SNAP Global Trial Steering Committee comprising collaborating members Asha Bowen, Matthew Cheng, Nick Daneman, Joshua Davis, Anna L. Goodman, George Heriot, Todd C. Lee, Roger Lewis, David Lye, Julie Marsh, Anna McGlothlin, Zoe McQuilten (Chair), Jocelyn Mora, Susan Morp...
PMC10699085
Authors’ contributions
RKM, AM, and JAM comprise the senior members of the core statistical team that conceived of the statistical modelling approach, simulations, and manuscript draft with input from all co-authors. All authors are members of the SNAP Statistical Subcommittee, SNAP Management Team, and/or SNAP analytic team who had substant...
PMC10699085
Funding
This work is supported by the Australian National Health and Medical Research Council (NHMRC) (1184238, 2014900), the Medical Research Future Fund (2017301), the Canadian Institutes of Health Research (451092), the Health Research Council of New Zealand (20/344), the Athlae Lyon/Starship Foundation Trust (ASF2144_WEBB)...
PMC10699085
Availability of data and materials
Not applicable.
PMC10699085
Declarations
PMC10699085
Ethics approval and consent to participate
The SNAP trial was initially approved by an Australian human research ethics committee (HREC Reference Number: HREC/74098/MH-2021). Ethical approval is sought from a properly constituted and accredited human research ethics committee (HREC) or institutional review board (IRB) for all trial sites. Where possible, a sing...
PMC10699085
Consent for publication
Not applicable.
PMC10699085
Competing interests
Infection
INFECTION
Anna McGlothlin is an employee of Berry Consultants LLC, in which capacity she is contracted as a consultant to numerous pharmaceutical and device companies on topics of statistical modelling and trial design, and reports grants and contracts unrelated to this work and consulting fees from Berry Consultants LLC. Todd C...
PMC10699085
References
PMC10699085
Purpose
gastrointestinal (GI) stress
We examined the effects of collagen peptides (CP) supplementation on exercise-induced gastrointestinal (GI) stress.
PMC9941265
Methods
In a randomized, crossover design, 20 volunteers (16 males:
PMC9941265
Results
There were no differences in heart rate, perceived exertion, thermal comfort, or core temperature during exercise in the CP and CON trials (all
PMC9941265
Conclusion
INFLAMMATION
Collagen peptides did not modify exercise-induced changes in inflammation, GI integrity or subjective GI symptoms but LPS was higher in CON 2 h post-exercise and thus future studies may be warranted.
PMC9941265
Keywords
PMC9941265
Introduction
intestinal barrier dysfunction, GI distress
PHYSIOLOGICAL STRESS, INTESTINAL BARRIER DYSFUNCTION, INFLAMMATION
The gastrointestinal (GI) barrier is comprised of simple columnar epithelial cells densely held together by tight junction proteins [Intestinal barrier dysfunction can result from psychological or physiological stress [In this regard, several dietary supplements have been tested for their effects on exercise-induced GI...
PMC9941265
Methods
PMC9941265
Participants
G*power 3.1.9.2 for Microsoft Windows [
PMC9941265
Experimental design
GI distress, GI, gastrointestinal; core temp, core temperature
BLIND
This study employed a randomized, double blind, placebo controlled, crossover design with two experimental treatment arms. Following preliminary testing, participants attended the lab for 3 main trials. On the first trial, non-exercise, no supplement, resting data was collected (REST). On the following 2 trials, the pr...
PMC9941265
Preliminary testing
After collecting measures of height and body mass, participants completed a maximal aerobic capacity test (
PMC9941265
Main trials
BLOOD
Approximately ~ 10 h before each main trial participants ingested a telemetric pill (Core Body Temperature Sensor, HQ Inc, Palmetto, FL) to assess core temperature, as described previously [During the 70 min exercise and REST trials, participants completed a survey to assess GI symptoms and thermal sensation [At 45 min...
PMC9941265
Blood and urine collection
intestinal fatty acid
BLOOD
Blood samples were obtained from a branch of the basilica vein at the antecubital fossa using the venipuncture technique. At all 3 time points (pre, 10 min post, 2-h post), blood was drawn into a 10 ml vacutainer for serum and a 10 ml vacutainer lined with EDTA. Samples were centrifuged at 4000 rpm for 10 min to separa...
PMC9941265
Blood and urine analysis
Haemoglobin and haematocrit were measured with a Hemo Control analyser (Hemo Control, EKF diagnostic, Cardiff, UK). The coefficient of variation (CV) for this analysis was < 4%. Cortisol is a glucocorticoid hormone that increases in response to a stress, such as exercise and alkaline phosphatase (ALP) is suggested to p...
PMC9941265
Serum I-FABP and LPS
GI distress, ®, CusaBio
I-FABP is a surrogate marker of enterocyte damage and was analysed in serum with a Quantikine human ELISA kit (R&D Systems, Inc. Minneapolis, MS, USA), with inter-assay CV ≤ 10% across the assay working range of 3.6–1000 pg/mL. Systemic LPS concentrations are used as a surrogate marker of GI distress, with increases su...
PMC9941265
Urine lactulose and rhamnose
Urinary excretion of orally consumed lactulose and rhamnose is a well-established measure of intestinal permeability [Urine lactulose and rhamnose results obtained from LC–MS/MS analysis were adjusted for variations in renal function by dividing by urine creatinine. Urine creatinine was analysed using Roche 2nd generat...
PMC9941265
Cytokines
cytokines increase
Numerous studies have shown that cytokines increase in response to exercise [
PMC9941265
Dietary supplements
ADVERSE EFFECTS
The CP and CON supplements were provided by Rousselot BV (Ghent, Belgium). Each serving of CP was 100 ml and contained 10 g of collagen peptides derived from bovine hide. We chose a 10 g dose because testing by the manufacturer has shown this dose to be safe and not result in adverse effects. The CON contained no activ...
PMC9941265
Data analysis
EVENT
Statistical significance was set at P < 0.05 and data were analysed using IBM SPSS Statistics 24 for Windows (Surrey, UK) and displayed as mean ± SD. Normality was assessed by inspecting histograms, skewness and kurtosis and the Shapiro–Wilk test (P < 0.05 considered not normally distributed). A repeated measures analy...
PMC9941265
Results
All participants completed the testing and unless otherwise stated, data is reported for all 20 volunteers.
PMC9941265
Plasma volume
Plasma volume decreased in both conditions post-run (CP: −5.3 ± 8.0% vs. CON: −6.6 ± 5.5%; time effect;
PMC9941265
HR, RPE and thermal comfort
HR increased during exercise (time effect; Changes in heart rate (HR) (
PMC9941265
Lactulose and rhamnose permeability test
Lactulose and rhamnose showed time effects (Changes in lactulose (
PMC9941265
I-FABP
One participant’s data was excluded due to technical difficulties during the analysis. I-FABP did not show overall time (Changes in lipopolysaccharide (LPS) (
PMC9941265
Lipopolysaccharide and anti-lipopolysaccharide antibody
LPS showed time (
PMC9941265
Cytokines
As > 80% of samples were below the limit of detection for IL-1β, this data was not analysed. In the present study, IL-6 showed time, condition, and interaction effects (all P < 0.001). IL-6 increased POST and 2 H POST in the CON and CP trials vs. PRE (all IL-8, a pro-inflammatory cytokine, showed time (MCP-1, a chemoki...
PMC9941265
Discussion
chronic exercise-induced gastrointestinal, endotoxemia, GI distress, acute GI stress, inflammatory bowel diseases, GI injury, intestinal injury
INFLAMMATORY BOWEL DISEASES, ENDOTOXEMIA
The main finding of this study is that supplementation with CP (10 g/day for 8 days) before high intensity running exercise did not alter markers of GI integrity as measured by our primary outcome I-FABP. Collagen peptides also had no effect on GI permeability (L/R). However, at 2-h post-exercise, serum LPS remained lo...
PMC9941265
Conclusion
endotoxemia, GI injury
INFLAMMATION, ENDOTOXEMIA
This study demonstrates that supplementation with CP does not alter exercise-induced changes in GI injury, permeability, and inflammation, or modify commonly reported GI symptoms. Although CP had no effect on our primary outcome measure I-FABP, a novel and intriguing finding was the greater LPS levels in the CON vs. no...
PMC9941265
Funding
Funding for the study was provided by Rousselot BV.
PMC9941265
Availability of data and material
Not available.
PMC9941265
Code availability
Not applicable.
PMC9941265
Declarations
PMC9941265
Conflict of interest
The funders supplied the supplements used in this study. Janne Prawitt and Nicolina Virgilio are employees of Rousselot BV.
PMC9941265
Ethical statement
All the study procedures were in accordance with the ethical standards of the institutional research ethics committee and with the 1964 Helsinki Declaration and its later amendments or comparable ethical standards.
PMC9941265
References
PMC9941265
2. Materials and Methods
PMC9862599
2.1. Patient Population and Distribution
bruxism, infection, diabetes
INFECTION, PERIODONTAL DISEASE, HYPERTENSION, OSTEOPOROSIS, DIABETES
A total of 70 patients were selected and included in this study, 38 women and 32 men, whose ages ranged from 23 to 68 years old. The present clinical study was submitted and approved by the Ethics Committee of the Paulista University (UNIP, São Paulo, Brazil) under the number 5,728,743. All procedures were performed by...
PMC9862599
2.2. Implant Macrogeometries and Surface Treatment
In the present study, two implant macrogeometries of Morse taper connection manufactured in Grade-4 titanium (Implacil De Bortoli, São Paulo, Brazil) were used, as described in the following: a DuoCone implant, which presents a regular conical macrogeometry with progressive trapezoidal threads and a cervical plane area...
PMC9862599
2.3. Experimental Design
The patients were divided into two groups (
PMC9862599
2.4. Surgical Procedure
M-D, allergic, pain
Standard routine surgical procedures were applied. As a preoperative medication, all patients received Amoxicillin (875 mg orally twice daily or Clindamycin 600 mg if allergic to penicillin) for 5 days, with an initial dose (2 tablets) administered 2 h before surgery. The same surgeon, a specialist in implantology with...
PMC9862599
2.5. Statistical Analysis
REGRESSION
Descriptive statistics (Mean, Median, SD, 95% CI, box plots) were generated for the insertion torque (ITV) and ISQ values for the DC and MAE groups. Furthermore, normality and equal variance steps were applied to determine the homogeneity of data distribution and to select the most appropriate parametric or non-paramet...
PMC9862599
3. Results
All seventy patients who received 100 implants (
PMC9862599
3.1. Insertion Torque Values
The 2-way ANOVA test (implant, arch) revealed statistically significant differences for implant macrogeometry (
PMC9862599
3.2. ISQ Values
M-D
The evaluation of the average ISQ between the values measured in both directions (B-L and M-D) for both groups (Regarding the comparisons of the diameter of the implants installed, regardless of the arch, few statistical differences were detected intragroup and intergroup in the 3 periods analyzed (
PMC9862599
3.3. B-L ISQ Values of Mandible and Maxilla
In the mandible for the DC group, the B-L ISQ values over time were statistically different (one-way ANOVA repeated measures, In the mandible for the MAE group, the B-L ISQ values over time were statistically different (Friedman´s repeated measures on ranks,
PMC9862599
3.4. M-D ISQ Values of Mandible and Maxilla
M-D
In the mandible for the DC group, the M-D ISQ values over time were statistically different (one-way ANOVA repeated measures, Over time, the M-D ISQ values in the mandible for the MAE group were statistically different (one-way ANOVA repeated measures,
PMC9862599
3.5. Multiple Linear Regression for Dependent Variable
The implant macrogeometry (DC and MAE groups), implant position (maxilla and mandible), diameter (3.5 and 4.0 mm), length (9, 11, and 13 mm), and periods (0, 30, and 45 days) were considered as independent variables. The
PMC9862599
3.6. Correlations between ITV and ISQ over Time
The correlation coefficients for each implant macrogeometry/arch according to the period are shown in
PMC9862599
4. Discussion
REGRESSION
Adequate primary stability in the bone bed is essential for achieving osseointegration [Previous studies showed that conical implants have higher primary stability values than cylindrical implants [In the multiple linear regression considering the insertion torque as a dependent variable, statistical differences were d...
PMC9862599
5. Conclusions
Modifications of the implant’s macrogeometry, by adding a healing chamber to its design (MAE group), produced a significant reduction in the insertion torque of the implants compared with the DC group, which presented a conventional implant design (without healing chambers). However, this reduction in ITV did not decre...
PMC9862599
Author Contributions
Conceptualization, S.A.G., G.V.d.O.F., A.S. and A.M.M.M.; Data curation, S.A.G., G.C.C., R.G.M. and R.M.C.; Formal analysis, S.A.G.; Investigation, S.A.G., G.C.C., R.G.M. and R.M.C.; Methodology, S.A.G., A.S. and A.M.M.M.; Resources, A.M.M.M.; Software, G.V.d.O.F.; Supervision, A.M.M.M.; Validation, A.S.; Writing—origi...
PMC9862599
Institutional Review Board Statement
The present clinical study was submitted and approved by the Ethics Committee of the Paulista University (UNIP, São Paulo, Brazil) under the number 5,728,743.
PMC9862599
Informed Consent Statement
All selected participants were informed about the conditions and the type of study that would be carried out, and they completed and signed the informed consent according to the agreement of the Declaration of Helsinki of 1994.
PMC9862599
Data Availability Statement
All data generated or analyzed during this study are included in this published article.
PMC9862599
Conflicts of Interest
The authors declare no conflict of interest.
PMC9862599
References
M-D ISQ values measured for MAE
Representative image of both tapered implants with different macrogeometries used in the present study.The CONSORT 2010 flow diagram.Box-plots graph (1st, 2nd, Median, 3rd, 4th quartiles) of ITV for DC and MAE groups within each arch. Outlier values are indicated in orange.Graph of the data distribution and statistical...
PMC9862599
Background
Retrospective cohorts have suggested that levosimendan may facilitate the weaning of veno-arterial extracorporeal membrane oxygenation (VA-ECMO). We therefore studied this clinical question by emulating a randomized trial with observational data.
PMC9906922
Methods
postcardiotomy cardiogenic shock
SECONDARY
All patients with refractory postcardiotomy cardiogenic shock and assisted with VA-ECMO, admitted to a surgical intensive care unit at La Pitié-Salpêtrière Hospital between 2016 and 2019, were eligible. To avoid immortal-time bias, we emulated a target trial sequentially comparing levosimendan administration versus no ...
PMC9906922
Results
thirty-nine
Two hundred and thirty-nine patients were included in the study allowing building a nested trials cohort of 1434 copies of patients. No association of levosimendan treatment and VA-ECMO weaning was found (HR = 0.91, [0.57; 1.45],
PMC9906922
Conclusions
postcardiotomy cardiogenic shock
Using the emulated target trial framework, this study did not find any association of levosimendan treatment and ECMO weaning success after postcardiotomy cardiogenic shock. However, the population of interest remains heterogeneous and subgroups might benefit from levosimendan.
PMC9906922
Supplementary Information
The online version contains supplementary material available at 10.1186/s13054-023-04328-6.
PMC9906922
Introduction
cardiogenic shock
CARDIOGENIC SHOCK
Veno-arterial extracorporeal membrane oxygenation (VA-ECMO) is the most widely used temporary mechanical circulatory support to restore adequate blood perfusion in patients with refractory cardiogenic shock [
PMC9906922
Materials and methods
PMC9906922
Regulations and ethics
CERAR
This is a single-center cohort study with retrospective data collection. Patients included in this study received standard of care without any change related to the research. Patient data were handled in accordance with the French regulations (MR004 reference methodology). The study protocol was approved by the Researc...
PMC9906922
Patient population
We enrolled all patients admitted to the surgical intensive care unit (ICU) at the Cardiology Institute of La Pitié-Salpêtrière University Hospital (Paris, France) between January 2016 and December 2019. The main inclusion criterion was the presence of mechanical circulatory support with VA-ECMO in the setting of cardi...
PMC9906922
ECMO implantation
low cardiac output
CARDIOGENIC SHOCK
ECMO was implanted either in the operating room, as a relay of cardiopulmonary bypass when patients presented low cardiac output refractory to adrenergic agonists, or postoperatively in ICU when patients presented a refractory cardiogenic shock. Implantation was always performed by cardiac surgeons. The initial ECMO fl...
PMC9906922
ECMO weaning
Patients were weaned from VA-ECMO according to a local protocol previously reported [
PMC9906922
Levosimendan administration
ADVERSE EFFECTS
All patients in the levosimendan group received a continuous infusion of 0.2 µg/kg/min over 24 h (i.e., 20 mg for a patient of 70 kg) administered as a single dose without an initial bolus of levosimendan according to local standard protocol. Administrated levosimendan was diluted in 0.9% NaCl at a concentration of 0.2...
PMC9906922
Outcomes definition
death
EVENTS, SECONDARY
The primary outcome was time to successful weaning from VA-ECMO within 30 days. Successful ECMO weaning was defined as follows: ECMO removal within 30 days in a patient being alive and without one of the following events occurring in the 30 days after ECMO removal: death, or need for repeated ECMO support or another me...
PMC9906922
Data collection
chronic kidney disease
DIABETES MELLITUS, LEFT HEART FAILURE, ACUTE HEART FAILURE, HYPERTENSION
Baseline covariates and follow-up of patients were collected retrospectively by medical record consultation and computer extraction of biological data. The following covariates were collected at baseline: age, height, weight (with calculation of body surface area according to Boyd's formula), sex, Simplified Acute Phys...
PMC9906922
Target trial emulation framework
death
In this study, we aimed to evaluate whether levosimendan administration in patients with VA-ECMO support shortened the time to successful ECMO weaning and reduced mortality at 30-day and at 1-year. We thus emulated a hypothetical target trial in which patients were randomly assigned to levosimendan administration or no...
PMC9906922
Description of the study populations
For each analytical population, patient selection process was represented by a flow diagram, and characteristics at inclusion were described as percentage for categorical variables, median with interquartile range (IQR), or mean with standard deviation (SD) for continuous variables, as appropriate. Characteristics were...
PMC9906922
Primary outcome analysis
lactatemia, death, chronic kidney disease
CHRONIC HEART FAILURE, DIABETES MELLITUS, REGRESSION, HYPERTENSION, ACUTE HEART FAILURE
The time to successful weaning from VA-ECMO was assessed for each copy of patient. Follow-up started at beginning of ECMO support and ended at first ECMO removal or death. The 30-day period after ECMO removal was only used to qualify ECMO weaning success but not included in the calculation of the time-to-event. Patient...
PMC9906922