Study protocol: developing, disseminating, and implementing a core outcome set for selective fetal growth restriction in monochorionic twin pregnancies

A Summary of the Meetings of the Development of a Core Outcome Set for Therapeutic Studies in Eosinophilic Esophagitis (COREOS) International Multidisciplinary Consensus

Call to action: long‐term neurodevelopment in monochorionic twins

Developing core outcome sets is essential to ensure that results of clinical trials are comparable and useful. A number of core outcome sets in pediatrics have been published, but a comprehensive in-depth understanding of core outcome sets in this field is lacking. To systematically identify core outcome sets in child health, collate the diseases to which core outcome sets have been applied, describe the methods used for development and stakeholder participation, and evaluate the methodological quality of existing core outcome sets. MEDLINE, SCOPUS, Cochrane Library, and CINAHL were searched using relevant search terms, such as clinical trials, core outcome, and children, along with relevant websites, such as Core Outcome Measures in Effectiveness Trials (COMET). Four researchers worked in teams of 2, performed literature screening and data extraction, and evaluated the methodological quality of core outcome sets using the Core Outcome Set-Standards for Development (COS-STAD). A total of 77 pediatric core outcome sets were identified, mainly developed by organizations or researchers in Europe, North America, and Australia and mostly from the UK (22 [29%]) and the US (22 [29%]). A total of 77 conditions were addressed; the most frequent International Classification of Diseases, 11th Revision category was diseases of the digestive system (14 [18%]). Most of the outcomes in pediatric core outcome sets were unordered (34 [44%]) or presented in custom classifications (29 [38%]). Core outcome sets used 1 or more of 8 development methods; the most frequent combination of methods was systematic review/literature review/scoping review, together with the Delphi approach and consensus for decision-making (10 [14%]). Among the 6 main types of stakeholders, clinical experts were the most frequently involved (74 [100%]), while industry representatives were rarely involved (4 [5%]). Only 6 core outcome sets (8%) met the 12 criteria of COS-STAD. Future quality of pediatric core outcome sets should be improved based on the standards proposed by the COMET initiative, while core outcome sets methodology and reporting standards should be extended to pediatric populations to help improve the quality of core outcome sets in child health. In addition, the COMET outcome taxonomy should also add items applicable to children.

881: The optimal timing of induction of labor in uncomplicated twin pregnancy

Monochorionicity refers to the condition in which twins share a single placenta. Selective fetal growth restriction (sFGR) in monochorionic (MC) twin pregnancy poses significant challenges due to its association with high risks of perinatal mortality and morbidity. Managing sFGR poses a particular challenge, as affected pregnancies require more frequent monitoring compared to uncomplicated singleton or twin pregnancies. Expectant management is typically applied for type I cases, which generally have favorable outcomes. In contrast, more severe cases (type II and III) may require invasive management, such as fetoscopic laser photocoagulation (FLP) or selective termination. However, how to best manage this complicated pregnancy remains debated, as no evidence-based guidance is currently available. This review aims to summarize the underlying pathophysiology, various diagnostic criteria, classification systems, and current management strategies for sFGR. These include the perinatal outcomes associated with expectant and invasive management in sFGR.

Development of consensus is required around a set of well-defined, relevant and feasible outcomes for all trials concerning particular obstetric and gynaecologic health conditions, such as preterm birth, incontinence, infertility and menstrual problems. With so many subspecialties involved, this is no easy task. Duplication of effort can be avoided by working with the Core Outcome Measures in Effectiveness Trials (COMET) Initiative, which is working towards core data sets for all medical specialties.3 Production of trustworthy core outcome sets will require engagement with patients, healthcare professionals, researchers, industry and regulators, and the employment of scientifically robust consensus methods.1 The data for these core outcome sets, once agreed upon, should be collected in trials and reported in publications as standard practice in the future. Journal editors now invite researchers to take the lead in beginning this work. What will we do as editors to support them and their colleagues? First, we are drawing wide attention to The CROWN Initiative by publishing this editorial in the journals listed below. We shall ensure that the global research community, which includes our many reviewers, is aware of the need for core outcome sets. Submissions which describe the development of core outcome sets, if deemed acceptable after peer review, will be effectively disseminated. Our collaboration is not for enforcing harmony at the expense of innovation. To quote from the COMET home page (www.comet-initiative.org): ‘The existence or use of a core outcome set does not imply that outcomes in a particular trial should be restricted to those in the relevant core outcome set. Rather, there is an expectation that the core outcomes will be collected and reported, making it easier for the results of trials to be compared, contrasted and combined as appropriate; while researchers continue to explore other outcomes as well’. We also expect that as new or superior ways of capturing outcomes emerge, core outcome sets will themselves need updating. Producing, disseminating and implementing core outcome sets will ensure that critical and important outcomes with good measurement properties are incorporated and reported. We believe this is the next important step in advancing the usefulness of research, in informing readers, including guideline and policy developers, who are involved in decision-making and in improving evidence-based practice. The CROWN Initiative is grateful to James Duffy (Trainee Scientific Editor, BJOG) and Louisa Waite (Assistant Editor, BJOG) for the drafting, revision and coordination required for the preparation of this article. The CROWN Initiative includes the following journals in alphabetical order (correct on 13th May 2014, up to date list available at www.crown-initiative.org): None to declare.

Background The concept of core outcome sets (COS) has been introduced in China for about 10 years. In recent years, some Chinese researchers also committed to developing COS, though the majority of COS are ongoing. However, there were more than 500 published COS for research in the COMET database by 2020. The extent of availability of COS for the top 25 diseases with the highest burden in China is unknown. In addition, the uptake of COS in clinical trials for these diseases is unknown, along with the knowledge, perceptions, and views of the clinical trialist community in China on the use of COS in relation to choosing outcomes for their research. Methods The main burden of disease in China will be identified. Then we will search the COMET database to identify if there are ongoing or completed relevant COS research A COS published since 2012 would be preferred to one published before 2012 for the analysis of COS uptake if one meets the eligibility criteria. We will extract scopes of published eligible COS, including condition, population, interventions, and core outcomes. Then we will search the Chinese Clinical Trial Registry using disease names for each disease that has a published COS. We will assess the overlap in scope between clinical trials and COS. Then we will conduct an online survey and semi-structured interviews to identify the knowledge and perceptions of COS among primary investigators of included clinical trials. Discussion This research will fill in gaps between COS and the burden of disease in China. Understanding clinical trialists’knowledge and perceptions of COS may help dissemination and application of COS in the future. Trial registration This research is registered in Core Outcome Measures in Effectiveness: https://www.comet-initiative.org/Studies/Details/2563.

Introduction: Inherited ichthyosis comprises a group of rare keratinization disorders caused by abnormal epidermal barrier function. Ichthyosis is yet incurable and current treatments mainly focus on alleviating symptoms such as scaling, erythema and pruritus. Recent developments show promising results for interventions based on the immune-phenotype like biologicals or pathogenesis-based therapies such as gene therapy. However, the lack of uniform reporting and variety of treatment outcomes may complicate performing and comparing efficacy studies. The core outcome set for inherited ichthyosis (COSII) aims to develop a core outcome set (COS), i.e., the minimum of outcomes that should be measured and reported in observational and interventional studies, including a minimum set of baseline characteristics. Methods: The COSII project will follow the guidelines from the Core Outcome Measures in Effectiveness Trials (COMET) initiative, including the Core Outcome Set-Standards for Development (COS-STAD) recommendations and the Core Outcome Set Standardised Protocol (COS-STAP) checklist. The COS development methodology, including this protocol, follows the guidance of the CHORD COUSIN Collaboration ‘C3’. The first stage of this project involves identifying a possible list of outcomes through performing a scoping literature review and conducting interviews with patient(s) (representatives). This list will be presented to five different stakeholder groups: healthcare professionals, researchers, patient(s) (representatives), industry representatives, and regulators. All stakeholders will rate the importance of each outcome in a three-round eDelphi survey. Ultimately, a virtual consensus meeting will be convened to finalize the COS. Ethical approval was obtained prior to the start of this project from the Medical Ethics Committee Board at Maastricht University Medical Centre (METC 2022-3192). Informed consent will be asked prior to enrolment in the eDelphi. This study is registered with the COMET. The results will be distributed via a peer-reviewed journal, communicated to all relevant parties and showcased at national and international conferences. Conclusion: This will be the first COS for inherited ichthyosis research in accordance with the Core Outcome Measures in Effectiveness Trials initiative. The development of a COS aims to improve the consistency of reporting and the heterogeneity of outcomes in ichthyosis research.

Developing an International Core Outcome Set for SSD Interventions

To review perinatal and neurodevelopmental outcome (NDO) following selective fetoscopic laser coagulation (SFLC), cord coagulation (CC) or expectant management of monochorionic diamniotic twin pregnancies complicated with selective intrauterine growth restriction (sIUGR) and absent or reverse end-diastolic flow (AREDF) in the umbilical arteries (UA). Single-centre retrospective observational study. 108 cases of sIUGR diagnosed before 26+6 weeks' gestation with AREDF in the UA. Survival rate and potential risk factors were analysed. NDO was assessed using parental questionnaires. Survival, gestational age at delivery and NDO. SFLC, CC and EM were performed in 13, 50 and 45 cases, respectively, with an overall survival of 23.1, 40 and 77.8% and intrauterine demise of the co-twin of 30.8, 10 and 6.7% respectively. Intrauterine demise of the sIUGR twin occurred in 76.9 and 17.8% following SFLC and EM, respectively. The discordance in EFW at diagnosis was higher and absent/negative a-wave in the ductus venosus (DV) was more prevalent in the surgical groups. NDO in survivors at follow up was abnormal in 0 and 18% in the smaller twin following SFLC and EM, respectively, and in 25, 24 and 21% in the larger twin following SFLC, CC and EM, respectively. SFLC yielded a poor result. EM seems a valid option when EFW discordance is <30% and a-wave in DV is positive. Otherwise, CC should be considered to protect the AGA co-twin. The long-term outcome of both small and large twins seems unaffected by the choice in primary prenatal management strategy. In type II sIUGR in MC twins, long-term neurodevelopment is normal in over 80% of the survivors.

"Report from ROMA: an update on the development of a core outcome set for aphasia research." Aphasiology, 32(sup1), pp. 241–242KEYWORDS: Aphasiacore outcome set Disclosure statementNo potential conflict of interest was reported by the authors.Background: A Core Outcome Set (COS) is a minimum set of outcomes that should be measured and reported in research trials of a specific health condition or population. The use of a COS does not preclude the measurement of study-specific outcomes, but rather represents the minimum outcomes that should be collected and reported. Benefits of COSs include: (1) increased transparency and reliability of research findings through the recommended reporting of a minimum set of outcomes; (2) production of compatible research data which can be efficiently synthesised in subsequent meta-analyses; and (3) reduced research wastage through the measurement of relevant outcomes which are more likely to inform treatment decision-making.This presentation provides an update on ongoing works to develop a COS for aphasia treatment research (Wallace, Worrall, Rose, & Le Dorze, 2016 Wallace, S. J., Worrall, L., Rose, T., & Le Dorze, G. (2016). Core outcomes in aphasia treatment research: an e-Delphi consensus study of international aphasia researchers. American Journal of Speech-Language Pathology, 25, S729–S742.[Crossref], [PubMed], [Web of Science ®] , [Google Scholar], 2017a Wallace, S. J., Worrall, L., Rose, T., & Le Dorze, G. (2017a). Using the international classification of functioning, disability, and health to identify outcome domains for a core outcome set for aphasia: A comparison of stakeholder perspectives. Disabil Rehabil, 1–10. doi:10.1080/09638288.2017.1400593[Taylor & Francis Online], [Web of Science ®] , [Google Scholar], 2017b Wallace, S. J., Worrall, L., Rose, T., & Le Dorze, G. (2017b). Which treatment outcomes are most important to aphasia clinicians and managers? An international e-Delphi consensus study. Aphasiology, 31, 643–673.[Taylor & Francis Online], [Web of Science ®] , [Google Scholar]; Wallace, Worrall, Rose, Le Dorze, et al., 2017 Wallace, S. J., Worrall, L., Rose, T., Le Dorze, G., Cruice, M., Isaksen, J., … Gauvreau, C. A. (2017). Which outcomes are most important to people with aphasia and their families? An international nominal group technique study framed within the ICF. Disabil Rehabil, 39, 1364–1379.[Taylor & Francis Online], [Web of Science ®] , [Google Scholar]). Research to date has identified essential outcome constructs as: language, communication, patient-reported satisfaction with treatment and impact of treatment, emotional wellbeing, and quality of life (Wallace, Worrall, Rose, & Le Dorze, 2017a Wallace, S. J., Worrall, L., Rose, T., & Le Dorze, G. (2017a). Using the international classification of functioning, disability, and health to identify outcome domains for a core outcome set for aphasia: A comparison of stakeholder perspectives. Disabil Rehabil, 1–10. doi:10.1080/09638288.2017.1400593[Taylor & Francis Online], [Web of Science ®] , [Google Scholar]). During the first ROMA consensus meeting, consensus was reached for measures of language (The Western Aphasia Battery-Revised (WAB-R); 74%), emotional wellbeing (General Health Questionnaire (GHQ)-12 (83% consensus); and quality of life (Stroke and Aphasia Quality of Life Scale (SAQOL-39); 96%).Aims: To provide an update on the ROMA (Improving Research Outcome Measurement in Aphasia) project to develop a COS for aphasia treatment research.Methods & Procedures: This presentation will provide an update on the ROMA consensus statement and current research exploring measures of communication for inclusion in the COS. Outcomes from the 2nd ROMA international consensus meeting will be discussed.Outcomes & Results: ROMA COS consensus statement. The ROMA consensus statement provides recommendations for a COS for aphasia treatment research. The consensus statement has been endorsed by the Collaboration of Aphasia Trialists, the British Aphasiology Society, The Royal College of Speech and Language Therapists, and the German Society for Aphasia Research and Therapy.Measuring communication as a core outcome in aphasia trials: Updates will be provided on current research examining the measurement of communication in aphasia trials. The outcomes from the 2nd ROMA international consensus meeting, which aims to develop a research agenda for the identification of a core measure of communication, will be discussed.Conclusions: How an outcome is defined and measured is critical in research design. Well-chosen outcome measures not only allow the detection of meaningful change within individual studies, but also facilitate synthesis across trials (Brady et al., 2014 Brady, M. C., Ali, M., Fyndanis, C., Kambanaros, M., Grohmann, K. K., Laska, A.-C., … Varlokosta, S. (2014). Time for a step change? Improving the efficiency, relevance, reliability, validity and transparency of aphasia rehabilitation research through core outcome measures, a common data set and improved reporting criteria. Aphasiology, 1–8. doi:10.1080/02687038.2014.930261[Taylor & Francis Online], [Web of Science ®] , [Google Scholar]). The development and implementation of a COS has the potential to improve the quality of aphasia treatment studies.

Prospective risk of intrauterine death of monochorionic-diamniotic twins

Developing a core outcome set for future infertility research: an international consensus development study

BackgroundThe COMET (Core Outcome Measures in Effectiveness Trials) Initiative promotes the development and application of core outcome sets (COS), including relevant studies in an online database. In order to keep the database current, an annual search of the literature is undertaken. This study aimed to update a previous systematic review, in order to identify any further studies where a COS has been developed. Furthermore, no prioritization for COS development has previously been undertaken, therefore this study also aimed to identify COS relevant to the world’s most prevalent health conditions.MethodsThe methods used in this updated review followed the same approach used in the original review and the previous update. A survey was also sent to the corresponding authors of COS identified for inclusion in this review, to ascertain what lessons they had learnt from developing their COS. Additionally, the COMET database was searched to identify COS that might be relevant to the conditions with the highest global prevalence.ResultsTwenty-five reports relating to 22 new studies were eligible for inclusion in the review. Further improvements were identified in relation to the description of the scope of the COS, use of the Delphi technique, and the inclusion of patient participants within the development process. Additionally, 33 published and ongoing COS were identified for 13 of the world’s most prevalent conditions.ConclusionThe development of a reporting guideline and minimum standards should contribute towards future improvements in development and reporting of COS. This study has also described a first approach to identifying gaps in existing COS, and to priority setting in this area. Important gaps have been identified, on the basis of global burden of disease, and the development and application of COS in these areas should be considered a priority.

Background: Velamentous cord insertion (VCI) has been proposed to be associated with some specific complications among monochorionic (MC) twin pregnancies. This meta-analysis and systematic review aims to determine the role of VCI in MC twin pregnancies.Methods: The PubMed, Embase and Web of Science databases and reference lists were searched for relevant studies. Outcomes of interest included twin-to-twin transfusion syndrome (TTTS), birthweight discordance (BWD) and selective intrauterine growth restriction (sIUGR). The methodological quality of the included studies was assessed by using the Newcastle-Ottawa Scale. The pooled results were calculated by means of a random or fixed effect model to obtain odds ratio with 95% confidential interval (CI). Subgroup analyses were utilized to detect the sources of heterogeneity.Results: Twenty studies were eligible for inclusion. The pooled result suggested a significant association between VCI and TTTS (OR, 1.542; 95% CI, 1.116–2.129) with a moderate level of heterogeneity (Q test: p = .024; I2 = 50.2%). Subgroup analysis reported single-center study, methodological quality and exclusion of laser-coagulated TTTS as the sources of heterogeneity. Another analysis revealed an increased risk of BWD among twin pregnancies with VCI (OR, 2.945; 95% CI, 2.176–3.984) with a low heterogeneity (Q test: p = .347; I2 = 10.5%). None of study level characteristics was found to be an influencing factor. Three studies reporting on sIUGR suggested a significant association between VCI and sIUGR.Conclusions: The meta-analysis and systematic review suggests an association between VCI and BWD and sIUGR. However, the association between VCI and TTTS may be overestimated and high-quality studies with a representative sample are needed in further research.