Validation of the brain injury associated visual impairment - impact questionnaire (BIVI-IQ).

PURPOSE: The Brain Injury associated Visual Impairment - Impact Questionnaire (BIVI-IQ) was developed to assess the impact of post-stroke visual impairment. The development of the questionnaire used robust methods involving stroke survivors and clinicians. The aim of this study was to assess the validity of the BIVI-IQ in a stroke population. METHODS: Stroke survivors with visual impairment were recruited from stroke units, outpatient clinics and non-healthcare settings. Participants were asked to complete questionnaire sets on three separate occasions; the BIVI-IQ at each visit with additional questionnaires at baseline and visit 2. Vision assessment and anchor questions from participants and clinicians were collected. The analysis included assessment of missing data, acceptability, Rasch model analysis, test-retest reliability, construct validity (NEI VFQ-25, EQ-5D-5L) and responsiveness to change. RESULTS: 316 stroke survivors completed at least one questionnaire of the 326 recruited. Mean age was 67 years and 64% were male. Adequate fit statistics to the Rasch model were reached (χ2 = 73.12, p = 0.02) with two items removed and thresholds of two adjusted, indicating validity and unidimensionality. Excellent test-retest reliability was demonstrated (ICC = 0.905) with a 3-month interval. Construct validity was demonstrated with a strong significant correlation to the NEI VFQ-25 (r = 0.837, p < 0.01). The BIVI-IQ also demonstrated responsiveness to change with significant differences identified between groups based on participant and clinician anchor questions (X2 = 23.29, p < 0.001; X2 = 24.56, p < 0.001). CONCLUSION: The BIVI-IQ has been shown to be valid and practical for 'everyday' use by clinicians and researchers to monitor vision-related quality of life in stroke survivors with visual impairment.

Development of a core outcome set for cutaneous squamous cell carcinoma trials: identification of core domains and outcomes.

BACKGROUND: The lack of uniformity in the outcomes reported in clinical studies of the treatment of cutaneous squamous cell carcinoma (cSCC) complicates efforts to compare treatment effectiveness across trials. OBJECTIVES: To develop a core outcome set (COS), a minimum set of agreed-upon outcomes to be measured in all clinical trials of a given disease or outcome, for the treatment of cSCC. METHODS: One hundred and nine outcomes were identified via a systematic literature review and interviews with 28 stakeholders. After consolidation of this long list, 55 candidate outcomes were rated by 19 physician and 10 patient stakeholders, in two rounds of Delphi exercises. Outcomes scored 'critically important' (score of 7, 8 or 9) by ≥ 70% of patients and ≥ 70% of physicians were provisionally included. At the consensus meeting, after discussion and voting of 44 international experts and patients, the provisional list was reduced to a final core set, for which consensus was achieved among all meeting participants. RESULTS: A core set of seven outcomes was finalized at the consensus meeting: (i) serious or persistent adverse events, (ii) patient-reported quality of life, (iii) complete response, (iv) partial response, (v) recurrence-free survival, (vi) progression-free survival and (vii) disease-specific survival. CONCLUSIONS: In order to increase the comparability of results across trials and to reduce selective reporting bias, cSCC researchers should consider reporting these core outcomes. Further work needs to be performed to identify the measures that should be reported for each of these outcomes.

Core outcome set for uncomplicated acute appendicitis in children and young people.

BACKGROUND: Research studies to inform clinical practice and policy in children and young people with appendicitis are hampered by inconsistent selection and reporting of outcomes. The aim of this study was to develop a core outcome set for reporting all studies of uncomplicated acute appendicitis in children and young people. METHODS: Systematic literature reviews, qualitative interviews with parents and patients treated for uncomplicated acute appendicitis, and a Study-Specific Advisory Group informed a long list of outcomes. Outcomes were then prioritized by stakeholders based in the UK (patients, parents, and paediatric and general surgeons) in an online three-round Delphi consensus process, followed by face-to-face consensus meetings. RESULTS: A long list of 40 items was scored by 147 key stakeholders in the first Delphi round, of whom 90 completed the two subsequent Delphi rounds. The final core outcome set comprises 14 outcomes: intra-abdominal abscess, reoperation (including interventional radiology procedure), readmission to hospital, bowel obstruction, wound infection, antibiotic failure, wound complication, negative appendicectomy, recurrent appendicitis, death, patient stress/psychological distress, length of hospital stay, time away from full activity and child's quality of life. CONCLUSION: A core outcome set comprising 14 outcomes across five key domains has been developed for reporting studies in children and young people with uncomplicated acute appendicitis. Further work is required to determine how and when to measure these outcomes.

ANTECEDENTES: Los estudios de investigación que sirvan de base para la práctica clínica y la política en niños y adultos jóvenes con apendicitis se ven obstaculizados por inconsistencias en la selección y descripción de los resultados. El objetivo de este estudio fue desarrollar un conjunto central de resultados para todos los estudios de apendicitis aguda no complicada en niños y adultos jóvenes. MÉTODOS: Para establecer una lista de resultados se efectuaron revisiones sistemáticas de la literatura, entrevistas cualitativas con padres y pacientes tratados por apendicitis aguda no complicada, y consulta con un Grupo de Asesoramiento Específico para el Estudio. Seguidamente, los resultados se priorizaron de acuerdo con los intereses de las partes interesadas (pacientes, padres, y cirujanos pediátricos y generales) en el Reino Unido a través de un proceso de consenso Delphi de tres rondas en Internet, seguido de reuniones personales de consenso. RESULTADOS: Un total de 147 participantes puntuaron una larga lista de 40 ítems en la primera ronda Delphi, de los cuales 90 completaron las dos rondas Delphi subsiguientes. El conjunto final incluye 14 resultados: absceso intra-abdominal, reoperación (incluyendo procedimientos radiológicos intervencionistas), reingreso, obstrucción intestinal, infección de herida, otras complicaciones de la herida, fracaso del tratamiento con antibióticos, apendicectomía blanca, apendicitis recidivante, muerte, estrés del paciente/sufrimiento psicológico, duración de la estancia hospitalaria, tiempo alejado de todas sus actividades y calidad de vida. CONCLUSIÓN: Se ha desarrollado un conjunto central de resultados que incluye 14 resultados en cinco dominios clave para la descripción de estudios en niños y adultos jóvenes con apendicitis aguda no complicada. Se requieren más trabajos para determinar cómo y cuándo conviene medir estos resultados.

Core outcome sets for prevention and treatment of postpartum haemorrhage: an international Delphi consensus study.

OBJECTIVE: To develop core outcome sets (COS) for studies evaluating interventions for (1) prevention and (2) treatment of postpartum haemorrhage (PPH), and recommendations on how to report the COS. DESIGN: A two-round Delphi survey and face-to-face meeting. POPULATION: Healthcare professionals and women's representatives. METHODS: Outcomes were identified from systematic reviews of PPH studies and stakeholder consultation. Participants scored each outcome in the Delphi on a Likert scale between 1 (not important) and 9 (critically important). Results were discussed at the face-to-face meeting to agree the final COS. Consensus at the meeting was defined as ≥ 70% of participants scoring the outcome as critically important (7-9). Lectures, discussion and voting were used to agree how to report COS outcomes. MAIN OUTCOME MEASURES: Outcomes from systematic reviews and consultations. RESULTS: Both Delphi rounds were completed by 152/205 (74%) participants for prevention and 143/197 (73%) for treatment. For prevention of PPH, nine core outcomes were selected: blood loss, shock, maternal death, use of additional uterotonics, blood transfusion, transfer for higher level of care, women's sense of wellbeing, acceptability and satisfaction with the intervention, breastfeeding, and adverse effects. For treatment of PPH, 12 core outcomes were selected: blood loss, shock, coagulopathy, hysterectomy, organ dysfunction, maternal death, blood transfusion, use of additional haemostatic intervention, transfer for higher level of care, women's sense of wellbeing, acceptability and satisfaction with the intervention, breastfeeding, and adverse effects. Recommendations were developed on how to report these outcomes where possible. CONCLUSIONS: These COS will help standardise outcome reporting in PPH trials. TWEETABLE ABSTRACT: Core outcome sets for PPH: nine core outcomes for PPH prevention and 12 core outcomes for PPH treatment.

Dexamethasone and haemorrhage risk in paediatric tonsillectomy: a systematic review and meta-analysis.

Summary In children undergoing tonsillectomy, dexamethasone is recommended to reduce the risk of postoperative nausea and vomiting while non-steroidal anti-inflammatory drugs (NSAIDs) are used for pain relief. We aimed to determine whether children who receive dexamethasone or dexamethasone with NSAID are more likely to experience haemorrhage post-tonsillectomy. Randomized and non-randomized studies in which children undergoing tonsillectomy received dexamethasone or dexamethasone and NSAID were sought within bibliographic databases and selected tertiary sources. The risk of bias assessment and evaluation of haemorrhage rate data collection and reporting were assessed using the Cochrane Risk of Bias Tool and McHarm tool. Synthesis methods comprised pooled estimate of the effect of dexamethasone on the risk of haemorrhage rate using the Peto odds ratio (OR) method. The pooled estimate for haemorrhage rate in children who received dexamethasone was 6.2%, OR 1.41 (95% confidence interval 0.89-2.25, P=0.15). There was risk of bias and inconsistent data collection and reporting rates of haemorrhage in many of the included studies. Clinical heterogeneity was observed between studies. The pooled analysis did not demonstrate a statistically significant increase in the risk of post-tonsillectomy haemorrhage with dexamethasone with/without NSAID use in children. However, the majority of the included studies were not designed to investigate this endpoint, and thus large studies which are specifically designed to collect data on haemorrhage rate are needed.

COS-Speech: protocol to develop a core outcome set for dysarthria after stroke for use in clinical practice and research.

BACKGROUND: Dysarthria after stroke is when speech intelligibility is impaired, and this occurs in half of all stroke survivors. Dysarthria often leads to social isolation, poor psychological well-being and can prevent return to work and social lives. Currently, a variety of outcome measures are used in clinical research and practice when monitoring recovery for people who have dysarthria. When research studies use different measures, it is impossible to compare results from trials and delays our understanding of effective clinical treatments. The aim of this study is to develop a core outcome set (COS) to agree what aspects of speech recovery should be measured for dysarthria after stroke (COS-Speech) in research and clinical practice. METHODS: The COS-Speech study will include five steps: (1) development of a long list of possible outcome domains of speech that should be measured to guide the survey; (2) recruitment to the COS-Speech study of three key stakeholder groups in the UK and Australia: stroke survivors, communication researchers and speech and language therapists/pathologists; (3) two rounds of the Delphi survey process; (4) a consensus meeting to agree the speech outcomes to be measured and a follow-up consensus meeting to match existing instruments/measures (from parallel systematic review) to the agreed COS-Speech; (5) dissemination of COS-Speech. DISCUSSION: There is currently no COS for dysarthria after stroke for research trials or clinical practice. The findings from this research study will be a minimum COS, for use in all dysarthria research studies and clinical practice looking at post-stroke recovery of speech. These findings will be widely disseminated using professional and patient networks, research and clinical forums as well as using a variety of academic papers, videos, accessible writing such as blogs and links on social media. TRIAL REGISTRATION: COS-Speech is registered with the Core Outcome Measures in Effectiveness Trials (COMET) database, October 2021 https://www.comet-initiative.org/Studies/Details/1959 . In addition, "A systematic review of the psychometric properties and clinical utility of instruments measuring dysarthria after stroke" will inform the consensus meeting to match measures to COS-Speech. The protocol for the systematic reviews registered with the International Prospective Register of Systematic Reviews. PROSPERO registration number: CRD42022302998 .

Secondary analysis of data from a core outcome set for burns demonstrated the need for involvement of lower income countries.

OBJECTIVE: To compare the views of participants from different income-status countries on outcome selection for a burn care Core Outcome Set (COS). METHODS: A retrospective analysis of data collected during a two round Delphi survey to prioritise the most important outcomes in burn care research. RESULTS: There was considerable agreement between participants from low- and middle-income countries (LMICs) and high-income countries (HICs) across outcomes. The groups agreed on 91% of 88 outcomes in round 1 and 92% of 100 in round 2. In cases of discordance, the consensus of participants from LMICs was to include the outcome and for participants from HICs to exclude. There was also considerable agreement between the groups for the top-ten ranking outcomes. Discordance in outcome prioritisation gives an insight into the different values clinicians from LMICs place on outcomes compared to those from HICs. Limitations of the study were that outcome rankings from international patients were not available. Healthcare professionals from LMICs were not involved in the final consensus meeting. CONCLUSION: COS developers should consider the need for a COS to be global at protocol stage. Global COS should include equal representation from both LMICs and HICs at all stages of development.

Frequency and reasons for outcome reporting bias in clinical trials: interviews with trialists.

OBJECTIVES: To provide information on the frequency and reasons for outcome reporting bias in clinical trials. DESIGN: Trial protocols were compared with subsequent publication(s) to identify any discrepancies in the outcomes reported, and telephone interviews were conducted with the respective trialists to investigate more extensively the reporting of the research and the issue of unreported outcomes. PARTICIPANTS: Chief investigators, or lead or coauthors of trials, were identified from two sources: trials published since 2002 covered in Cochrane systematic reviews where at least one trial analysed was suspected of being at risk of outcome reporting bias (issue 4, 2006; issue 1, 2007, and issue 2, 2007 of the Cochrane library); and a random sample of trial reports indexed on PubMed between August 2007 and July 2008. SETTING: Australia, Canada, Germany, the Netherlands, New Zealand, the United Kingdom, and the United States. MAIN OUTCOME MEASURES: Frequency of incomplete outcome reporting-signified by outcomes that were specified in a trial's protocol but not fully reported in subsequent publications-and trialists' reasons for incomplete reporting of outcomes. RESULTS: 268 trials were identified for inclusion (183 from the cohort of Cochrane systematic reviews and 85 from PubMed). Initially, 161 respective investigators responded to our requests for interview, 130 (81%) of whom agreed to be interviewed. However, failure to achieve subsequent contact, obtain a copy of the study protocol, or both meant that final interviews were conducted with 59 (37%) of the 161 trialists. Sixteen trial investigators failed to report analysed outcomes at the time of the primary publication, 17 trialists collected outcome data that were subsequently not analysed, and five trialists did not measure a prespecified outcome over the course of the trial. In almost all trials in which prespecified outcomes had been analysed but not reported (15/16, 94%), this under-reporting resulted in bias. In nearly a quarter of trials in which prespecified outcomes had been measured but not analysed (4/17, 24%), the "direction" of the main findings influenced the investigators' decision not to analyse the remaining data collected. In 14 (67%) of the 21 randomly selected PubMed trials, there was at least one unreported efficacy or harm outcome. More than a quarter (6/21, 29%) of these trials were found to have displayed outcome reporting bias. CONCLUSION: The prevalence of incomplete outcome reporting is high. Trialists seemed generally unaware of the implications for the evidence base of not reporting all outcomes and protocol changes. A general lack of consensus regarding the choice of outcomes in particular clinical settings was evident and affects trial design, conduct, analysis, and reporting.