Veronica Ka Wai Lai

Background: Defining outcomes completely before conducting clinical trials helps to mitigate concerns about reporting biases; however, there is limited guidance to help investigators define outcomes completely. Methods: Building on a previous framework, we developed guidance for defining trial outcomes completely and consistently. We reviewed literature, developed preliminary guidance, and refined guidance iteratively through pilot testing. We randomly selected 225 randomized controlled trials (RCTs) from ClinicalTrials.gov, which evaluated 3,424 outcomes. Two raters followed a structured approach to define each outcome. When raters encountered outcomes they could not define, we refined our guidance. We continued this process until no further changes were needed. We finalized the guidance through multiple drafts and discussions among authors. Findings: In clinical trials, a completely defined outcome includes six elements: outcome domain, specific measurement, specific metric, cutoff (if applicable), variable type, and time point. We developed guidance for naming specific measurements for both subjective and objective outcomes. We expanded on prior work by describing more comprehensive categories for specific metrics. To be consistent with the estimand framework, we replaced “method of aggregation” with two distinct elements that describe values in individuals (rather than summary measures): “variable type” and “cutoff.” We further defined “cutoff” with three sub-elements, and we defined “variable type” as whether the value for each individual is continuous or categorical. Conclusions: Trialists and sponsors could use this framework and guidance to define outcomes in trial registrations, protocols, and results reports. When all six elements are defined, readers can better determine whether reported outcomes were prespecified and whether different trials assessed the same outcomes.

Defining outcomes completely before conducting clinical trials helps to mitigate reporting biases; however, there is limited guidance to help investigators define outcomes completely. We aimed to develop a structured approach for defining trial outcomes completely and consistently.We reviewed literature, developed preliminary rules for defining outcomes, and refined them iteratively. We randomly selected randomized controlled trials (RCTs) on ClinicalTrials.gov that registered before their start dates and posted results by January 4, 2024. The 225 included RCTs evaluated 3,424 outcomes. Two raters independently applied preliminary rules to define each outcome. When raters encountered outcomes they could not define, we refined the rules. We continued this process until no further changes were needed. We discussed and finalized our approach in a consensus meeting.We define an “outcome” as a value for each participant that will be used in analysis to generate study results. A complete outcome definition includes six elements: outcome domain, specific measurement, specific metric, cutoff, variable type, and timepoint. We developed rules for naming specific measurements for both subjective and objective outcomes. We expanded on prior work by developing more comprehensive categories for specific metrics. We introduced "cutoff" as a distinct element with three subelements. To clarify the boundary between outcome definitions and statistical methods, we replaced a previously described element, "method of aggregation," with "variable type," which refers to whether the value for each individual is continuous or categorical.Trialists and sponsors could use this approach alongside other guidelines to define outcomes in trial registrations, protocols, and result reports.

Defining outcomes completely before conducting clinical trials helps to mitigate reporting biases; however, there is limited guidance to help investigators define outcomes completely. We aimed to develop a structured approach for defining trial outcomes completely and consistently.We reviewed literature, developed preliminary rules for defining outcomes, and refined them iteratively. We randomly selected randomized controlled trials (RCTs) on ClinicalTrials.gov that registered before their start dates and posted results by January 4, 2024. The 225 included RCTs evaluated 3,424 outcomes. Two raters independently applied preliminary rules to define each outcome. When raters encountered outcomes they could not define, we refined the rules. We continued this process until no further changes were needed. We discussed and finalized our approach in a consensus meeting.We define an “outcome” as a value for each participant that will be used in analysis to generate study results. A complete outcome definition includes six elements: outcome domain, specific measurement, specific metric, cutoff, variable type, and timepoint. We developed rules for naming specific measurements for both subjective and objective outcomes. We expanded on prior work by developing more comprehensive categories for specific metrics. We introduced "cutoff" as a distinct element with three subelements. To clarify the boundary between outcome definitions and statistical methods, we replaced a previously described element, "method of aggregation," with "variable type," which refers to whether the value for each individual is continuous or categorical.Trialists and sponsors could use this approach alongside other guidelines to define outcomes in trial registrations, protocols, and result reports.

This study tested whether the Perceptual Assimilation Model of Second Language Speech Learning (PAM-L2) predicts second language (L2) Cantonese tone discrimination across different perceptual modes. Punjabi speakers of Cantonese completed the Cantonese tone assimilation and discrimination tasks. In the assimilation task, the Punjabi listeners assimilated the Cantonese tones as two-category (TC), single-category (SC), uncategorized-categorized without overlap (UC-no), and uncategorized-categorized with partial overlap (UC-po) pairs, yielding testable predictions for PAM-L2 in the discrimination task (TC = UC-no > UC-po > SC). In the discrimination task, the model-driven predictions were largely supported in the double-talker context but not in the single-talker and pure tone contexts. These results suggest that PAM-L2 applies to phonological but not non-phonological discrimination of L2 tones. Moreover, our findings indicate that the distinction between partial and complete overlap may not be necessary for UC pairs.

Key Points: PRO-Kid is a patient-reported outcome measure of the frequency and burden of symptoms. Higher PRO-Kid scores are associated with lower Pediatric Quality of Life Inventory scores. Background: Measuring the burden of symptoms that matter most to children and adolescents with CKD is essential for optimizing patient-centered care. We developed a novel CKD-specific patient-reported outcome measure (PRO-Kid) to assess both frequency and impact of symptoms in children. In this study, we further assessed the validity and internal consistency of PRO-Kid. Methods: In this multicenter study, children age 8–18 years with stages 3–5 CKD, including those on dialysis, were recruited from five pediatric centers. Children completed the 14-item PRO-Kid questionnaire and the validated Pediatric Quality of Life Inventory (PedsQL 4.0). We explored the dimensionality of the PRO-kid scale using exploratory and confirmatory factor analysis, to either establish that it is a unidimensional construct or identify evidence of subfactors. We then assessed internal consistency (Cronbach alpha) and construct validity (Pearson correlations). Results: In total, 100 children were included. The median eGFR was 27.4 ml/min per 1.73 m² (7.43–63.4), and 26 children (26%) were on dialysis. Both the PRO-Kid frequency and the impact scales were unidimensional. Cronbach alpha was high for both the PRO-Kid frequency and impact scales, 0.83 (95% confidence interval [CI], 0.78 to 0.88) and 0.84 (95% CI, 0.80 to 0.89), respectively, showing strong internal consistency. Pearson correlations between PRO-Kid and PedsQL scores were also strong: −0.78 (95% CI, −0.85 to −0.70) for the frequency score and −0.69 (95% CI, −0.78 to −0.56) for the impact score, reflecting the association between poorer quality of life and higher symptom burden. Conclusions: PRO-Kid is a novel patient-reported symptom burden tool for children age 8–18 years with CKD that correlates strongly in the expected direction with PedsQL, supporting its validity. Future work will evaluate changes in PRO-Kid score with progression of CKD and implementation of the tool into clinical care.

Objectives This study assessed the feasibility and preliminary efficacy of a 2-hour compression-only cardiopulmonary resuscitation and automated external defibrillator (CO-CPRAED) course in secondary school students. Design Prospective pre-post feasibility study. Setting and participants 128 students (12–15 years old) without prior basic life support (BLS) training at four secondary schools in Hong Kong. All students were followed up at 3 months after training. Interventions Emergency medicine-trained nurse and physicians taught the 2-hour CO-CPRAED course using the American Heart Association ‘CPR in School Training Kit’ programme. Students were trained in groups up to 40 students/session, with an instructor to student ratio not exceeding 1:10. To practise hands-on compressions, the manikin to student ratio was 1:1. For a simulated cardiac arrest, the manikin and AED to student ratio was 1:10. Primary and secondary outcomes CPR and AED knowledge, attitude statements towards bystander CPR and AED, quality of BLS performance skills during training and at 3 months. Results Some students (46%) knew how deep to push on an adult chest when doing CO-CPR before training. The course was associated with an increase in knowledge score (pretraining 55%, post-training 93%; adjusted mean difference (MD) 38%, 95% CI 33% to 43%; pConclusions The results demonstrate the feasibility of scaling up the number of secondary schools trained in a brief CO-CPRAED course within the local school curriculum.

Background Preoperative education may help participants to psychologically prepare themselves for surgery, but the outcomes of such preparation have rarely been assessed in patients requiring postoperative care in the intensive care unit (ICU) as well as in family members. Objective To assess the effect of a preoperative multifaceted education intervention on patient and family satisfaction levels in the ICU and measures of perioperative patients’ anxiety and depression. Trial design Single-centre, two-armed, parallel, superiority, randomised controlled trial. Healthcare professionals in ICU and outcome assessor were blinded to treatment allocation. Participants 100 elective coronary artery bypass grafting±valve surgery patients and their family members. Interventions Preoperative education comprising of a video and ICU tour in addition to standard care (treatment), versus standard care (control). Outcomes Patient and family satisfaction levels with ICU using validated PS-ICU23 and FS-ICU24 questionnaires (0–100), respectively; change in perioperative anxiety and depression scores between 1 day presurgery and 3 days postsurgery. Results Among 100 (50 treatment, 50 control) patients and 98 (49 treatment, 49 control) family members, 94 (48 treatment, 46 control) patients and 94 (47 treatment, 47 control) family members completed the trial. Preoperative education was associated with higher overall patient (mean difference (MD) 6.7, 95% CI 0.2 to 13.2) and family (MD 10.0, 95% CI 3.8 to 16.3) satisfaction scores. There was a weak association between preoperative education and a reduction in patient’s anxiety scores over time (MD −1.7, 95% CI −3.5 to 0.0). However, there was no evidence of a treatment effect on patient’s depression scores over time (MD −0.6, 95% CI −2.3 to 1.2). Conclusion Providing comprehensive preoperative information about ICU to elective cardiac surgical patients improved patient and family satisfaction levels and may decrease patients’ anxiety levels. Trial registration number ChiCTR-IOR-15006971.

The Critical Care Society of Southern Africa Consensus Statement on ICU Triage and Rationing (ConICTri)