Publications by authors named "Offringa M"
Objective: The objective of this scoping review is to develop a list of items for potential inclusion in the Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) reporting guidelines for network meta-analysis (NMA), scoping reviews (ScRs), and rapid reviews (RRs).
Introduction: The PRISMA extensions for NMA and ScRs were published in 2015 and 2018. However, since then, their methodologies and innovations, including automation, have evolved.
Background: To inform the development of a core outcome set (COS) for children and youth with mucopolysaccharidoses (MPS), we aimed to identify all outcomes and associated outcome measurement instruments that are reported in recent clinical trials and recommended as measurements in clinical management guidelines.
Methods: To identify English-language clinical trials and guidelines pertaining to MPS published between 2011 and mid-2021, we applied a comprehensive peer-reviewed search strategy to relevant databases and registers on May 16, 2021. Two reviewers independently screened retrieved citations and then full-text articles to determine eligibility for inclusion.
Objective: The objectives of this review are to identify barriers/facilitators to designing, maintaining, and utilizing rare disease patient registries (RDPRs); determine whether and how these differ among patient partners, other knowledge users (KUs), and researchers; and chart definitions of rare diseases and RDPRs.
Introduction: RDPRs are vital to improving the understanding of the natural histories and predictors of outcomes for rare diseases, assessing interventions, and identifying potential participants for clinical trials. Currently, however, the functionality of RDPRs is not fully optimized.
Article Synopsis
- - The study explored the use of remote ischemic conditioning (RIC) as a potential neuroprotective therapy for infants with neonatal encephalopathy (NE), assessing its safety and feasibility when combined with therapeutic hypothermia (TH).
- - In their randomized trial involving 32 neonates, researchers found that RIC was safe to administer, without causing any significant adverse effects or differences in seizure rates, brain injury, or mortality compared to a sham group.
- - This pilot study provides important safety data needed for future research on RIC's efficacy in improving neurodevelopmental outcomes in infants with hypoxic-ischemic encephalopathy (HIE).
Article Synopsis
- Adaptive designs (ADs) in clinical trials make the testing process more flexible and can save time and money.
- Many people in the medical field don’t fully understand how these designs work, so a review has been done to explain their use in research.
- The study looked at a lot of research reports from 2010 to 2020 and found that most ADs were used in trials for adults, especially for cancer treatments, with a smaller number for children.
Article Synopsis
- - The study aimed to evaluate the effectiveness of 12 patient-reported outcome measures (PROMs) for assessing depression severity in adolescents, given the importance of accurately measuring depression in clinical settings.
- - Researchers conducted a comprehensive review using multiple databases and followed specific guidelines to assess various measurement properties of the PROMs, ultimately narrowing down to 31 studies for appraisal.
- - The review concluded that while some PROMs show promise in measuring depression in adolescents, none can currently be recommended for consistent use due to a lack of robust evidence and methodological issues, advising caution for clinicians and researchers.
Article Synopsis
- Randomised controlled trials often use surrogate endpoints instead of direct outcomes to enhance efficiency and reduce costs, but this can lead to increased uncertainty regarding treatment effects and intervention harms.
- A new guideline, known as CONSORT-Surrogate, provides a checklist for reporting trials that utilize surrogate endpoints, ensuring better clarity and transparency in the results.
- The recommended checklist, tailored for all stakeholders involved in clinical trials, aims to improve the reporting quality of these studies and ultimately reduce wasted research efforts.
Article Synopsis
- Randomised controlled trials often use surrogate endpoints instead of direct outcomes to save time, cost, and ethical concerns, but this can create uncertainty about the actual treatment effects and potential harms.
- The SPIRIT-Surrogate is a new guideline to enhance the reporting standards for trial protocols that use surrogate endpoints, building on the existing SPIRIT checklist.
- This guideline includes nine modified items, encouraging all stakeholders to adopt it, which aims to improve trial design, transparency, and ultimately reduce wasted research efforts.
Purpose: Although comprehensive and widespread guidelines on how to conduct systematic reviews of outcome measurement instruments (OMIs) exist, for example from the COSMIN (COnsensus-based Standards for the selection of health Measurement INstruments) initiative, key information is often missing in published reports. This article describes the development of an extension of the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 guideline: PRISMA-COSMIN for OMIs 2024.
Methods: The development process followed the Enhancing the QUAlity and Transparency Of health Research (EQUATOR) guidelines and included a literature search, expert consultations, a Delphi study, a hybrid workgroup meeting, pilot testing, and an end-of-project meeting, with integrated patient/public involvement.
Purpose: Although comprehensive and widespread guidelines on how to conduct systematic reviews of outcome measurement instruments (OMIs) exist, for example from the COSMIN (COnsensus-based Standards for the selection of health Measurement INstruments) initiative, key information is often missing in published reports. This article describes the development of an extension of the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 guideline: PRISMA-COSMIN for OMIs 2024.
Methods: The development process followed the Enhancing the QUAlity and Transparency Of health Research (EQUATOR) guidelines and included a literature search, expert consultations, a Delphi study, a hybrid workgroup meeting, pilot testing, and an end-of-project meeting, with integrated patient/public involvement.
Purpose: Although comprehensive and widespread guidelines on how to conduct systematic reviews of outcome measurement instruments (OMIs) exist, for example from the COSMIN (COnsensus-based Standards for the selection of health Measurement INstruments) initiative, key information is often missing in published reports. This article describes the development of an extension of the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 guideline: PRISMA-COSMIN for OMIs 2024.
Methods: The development process followed the Enhancing the QUAlity and Transparency Of health Research (EQUATOR) guidelines and included a literature search, expert consultations, a Delphi study, a hybrid workgroup meeting, pilot testing, and an end-of-project meeting, with integrated patient/public involvement.
Purpose: Systematic reviews evaluating and comparing the measurement properties of outcome measurement instruments (OMIs) play an important role in OMI selection. Earlier overviews of review quality (2007, 2014) evidenced substantial concerns with regards to alignment to scientific standards. This overview aimed to investigate whether the quality of recent systematic reviews of OMIs lives up to the current scientific standards.
View Article and Find Full Text PDFBackground And Objective: Although comprehensive and widespread guidelines on how to conduct systematic reviews of outcome measurement instruments (OMIs) exist, for example from the COSMIN (COnsensus-based Standards for the selection of health Measurement INstruments) initiative, key information is often missing in published reports. This article describes the development of an extension of the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 guideline: PRISMA-COSMIN for OMIs 2024.
Methods: The development process followed the Enhancing the QUAlity and Transparency Of health Research (EQUATOR) guidelines and included a literature search, expert consultations, a Delphi study, a hybrid workgroup meeting, pilot testing, and an end-of-project meeting, with integrated patient/public involvement.
To identify priority areas to improve the design, conduct, and reporting of pediatric clinical trials, the international expert network, Standards for Research (StaR) in Child Health, was assembled and published the first 6 Standards in Pediatrics in 2012. After a recent review summarizing the 247 publications by StaR Child Health authors that highlight research practices that add value and reduce research "waste," the current review assesses the progress in key child health trial methods areas: consent and recruitment, containing risk of bias, roles of data monitoring committees, appropriate sample size calculations, outcome selection and measurement, and age groups for pediatric trials. Although meaningful change has occurred within the child health research ecosystem, measurable progress is still disappointingly slow.
View Article and Find Full Text PDFBackground And Objectives: Variability in outcome reporting in necrotizing enterocolitis (NEC) treatment trials hinders conducting meta-analyses and implementing novel treatments. We aimed to develop a core outcome set (COS) for NEC treatment trials including outcome measures most relevant to patients and physicians, from NEC diagnosis to adulthood.
Methods: Clinicians and/or researchers from low-middle- and high-income countries were approached based on their scientific contributions to NEC literature, and patients and parents through local organizations.
Background: Bronchopulmonary dysplasia (BPD) remains an important complication of prematurity. Pulmonary inflammation plays a central role in the pathogenesis of BPD, explaining the rationale for investigating postnatal corticosteroids. Multiple systematic reviews (SRs) have summarised the evidence from numerous randomised controlled trials (RCTs) investigating different aspects of administrating postnatal corticosteroids.
View Article and Find Full Text PDFBackground: In recent years, projects to develop reporting guidelines have attempted to integrate the perspectives of patients and public members. Best practices for patient and public involvement (PPI) in such projects have not yet been established. We recently developed an extension of PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses), to be used for systematic reviews of outcome measurement instruments (OMIs): PRISMA-COSMIN (COnsensus-based Standards for the selection of health Measurement INstruments) for OMIs 2024.
View Article and Find Full Text PDFBackground: Despite the critical importance of clinical trials to provide evidence about the effects of intervention for children and youth, a paucity of published high-quality pediatric clinical trials persists. Sub-optimal reporting of key trial elements necessary to critically appraise and synthesize findings is prevalent. To harmonize and provide guidance for reporting in pediatric controlled clinical trial protocols and reports, reporting guideline extensions to the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT) and Consolidated Standards of Reporting Trials (CONSORT) guidelines specific to pediatrics are being developed: SPIRIT-Children (SPIRIT-C) and CONSORT-Children (CONSORT-C).
View Article and Find Full Text PDFBackground: Generating rigorous evidence to inform care for rare diseases requires reliable, sustainable, and longitudinal measurement of priority outcomes. Having developed a core outcome set for pediatric medium-chain acyl-CoA dehydrogenase (MCAD) deficiency, we aimed to assess the feasibility of prospective measurement of these core outcomes during routine metabolic clinic visits.
Methods: We used existing cohort data abstracted from charts of 124 children diagnosed with MCAD deficiency who participated in a Canadian study which collected data from birth to a maximum of 11 years of age to investigate the frequency of clinic visits and quality of metabolic chart data for selected outcomes.
Article Synopsis
- Open inguinal hernia repair is the traditional treatment for children, but laparoscopic methods are becoming more popular, though there's no clear consensus on which is better due to varying study outcomes.
- A core outcome set (COS) will be developed to standardize the measurement and reporting of outcomes across studies, addressing the current inconsistencies.
- The project involves identifying relevant outcome domains, conducting a Delphi study to prioritize outcomes, and holding an expert meeting to finalize the COS and its implementation strategies.