Developing a core outcome set for the health outcomes for children and adults with congenital oesophageal atresia and/or tracheo-oesophageal fistula: OCELOT task group study protocol.

INTRODUCTION: Heterogeneity in reported outcomes of infants with oesophageal atresia (OA) with or without tracheo-oesophageal fistula (TOF) prevents effective data pooling. Core outcome sets (COS) have been developed for many conditions to standardise outcome reporting, facilitate meta-analysis and improve the relevance of research for patients and families. Our aim is to develop an internationally-agreed, comprehensive COS for OA-TOF, relevant from birth through to transition and adulthood. METHODS AND ANALYSIS: A long list of outcomes will be generated using (1) a systematic review of existing studies on OA-TOF and (2) qualitative research with children (patients), adults (patients) and families involving focus groups, semistructured interviews and self-reported outcome activity packs. A two-phase Delphi survey will then be completed by four key stakeholder groups: (1) patients (paediatric and adult); (2) families; (3) healthcare professionals; and (4) researchers. Phase I will include stakeholders individually rating the importance and relevance of each long-listed outcome using a 9-point Likert scale, with the option to suggest additional outcomes not already included. During phase II, stakeholders will review summarised results from phase I relative to their own initial score and then will be asked to rescore the outcome based on this information. Responses from phase II will be summarised using descriptive statistics and a predefined definition of consensus for inclusion or exclusion of outcomes. Following the Delphi process, stakeholder experts will be invited to review data at a consensus meeting and agree on a COS for OA-TOF. ETHICS AND DISSEMINATION: Ethical approval was sought through the Health Research Authority via the Integrated Research Application System, registration no. 297026. However, approval was deemed not to be required, so study sponsorship and oversight were provided by Alder Hey Children's NHS Foundation Trust. The study has been prospectively registered with the COMET Initiative. The study will be published in an open access forum.

Prevalence and early surgical outcome of congenital diaphragmatic hernia in the Netherlands: a population-based cohort study from the European Pediatric Surgical Audit.

BACKGROUND: Congenital diaphragmatic hernia (CDH) is a rare birth defect with substantial mortality. This study aims to generate a population-based overview of CDH care and outcomes in the Netherlands. Moreover, it assesses interhospital variations between the two Dutch CDH expert centres. METHODS: This study uses data from the Dutch branch of the European Pediatric Surgical Audit, a prospective clinical audit for congenital anomalies. Data of all patients with CDH treated between 2014 and 2021 were included for epidemiological analysis. For comparative analyses, patients presenting after the neonatal period or not treated in the two CDH expert centres were excluded. Identified interhospital variations were assessed using regression analysis. RESULTS: In the study period, 283 children with CDH were born, resulting in a national prevalence rate of 2.06/10 000 live births. The patient population, treatment and outcomes at 1 year were comparable between the hospitals, except for length of hospital stay. Regression analysis identified the treating hospital as the strongest significant predictor thereof. Other factors associated with longer length of stay include the presence of other malformations, intrathoracic liver position on prenatal ultrasound, extracorporeal membrane oxygenation treatment, patch repair, complicated postoperative course and discharge to home rather than to another care facility. CONCLUSION: Outcomes of CDH care throughout the Netherlands are comparable. However, the length of stay differed between the two hospitals, also when adjusting for other covariates. Further qualitative analysis to explain this interhospital variation is indicated. Our findings underscore the potential of clinical auditing as a quality measurement tool in rare conditions.

Bridging the Gap: A Systematic Review on Reporting Baseline Characteristics, Process, and Outcome Parameters in Rectosigmoid Hirschsprung's Disease.

The variation in standardized, well-defined parameters in Hirschsprung's disease (HSCR) research hinders overarching comparisons and complicates evaluations of care quality across healthcare settings. This review addresses the significant variability observed in these parameters as reported in recent publications. The goal is to compile a list of commonly described baseline characteristics, process and outcome measures, and to investigate disparities in their utilization and definitions. A systematic review of literature on the primary care process for HSCR was performed according to PRISMA guidelines. Relevant literature published between 2015 and 2021 was obtained by combining the search term "Hirschsprung's disease" with "treatment outcome," "complications," "mortality," "morbidity," and "survival" in Medline, Embase, and the Cochrane Library. We extracted study characteristics, reported process and outcome parameters, and patient and disease characteristics. We extracted 1,026 parameters from 200 publications and categorized these into patient characteristics (n = 226), treatment and care process characteristics (n = 199), and outcomes (n = 601). A total of 116 parameters were reported in more than 5% of publications. The most frequently reported characteristics were sex (88%), age at the time of surgery (66%), postoperative Hirschsprung-associated enterocolitis (64%), type of repair (57%), fecal incontinence (54%), and extent of aganglionosis (51%). This review underscores the pronounced variation in reported parameters within HSCR studies, highlighting the necessity for consistent, well-defined measures and reporting systems to foster improved data interpretability. Moreover, it advocates for the use of these findings in the development of a Core Indicator Set, complementing the recently developed Core Outcome Set. This will facilitate quality assessments across pediatric surgical centers throughout Europe.

Selection of Quality Indicators to Evaluate Quality of Care for Patients with Esophageal Atresia Using a Delphi Method.

OBJECTIVE: Survival of neonates with esophageal atresia (EA) is relatively high and stable, resulting in increased attention to optimizing care and longer-term morbidity. This study aimed to reach consensus on a quality indicator set for benchmarking EA care between hospitals, regions, or countries in a European clinical audit. METHODS: Using an online Delphi method, a panel of EA health care professionals and patient representatives rated potential outcome, structure, and process indicators for EA care identified through systematic literature and guideline review on a nine-point Likert scale in three questionnaires. Items were included based on predefined criteria. In rounds 2 and 3, participants were asked to select the five to ten most essential of the included indicators. RESULTS: An international panel of 14 patient representatives and 71 multidisciplinary health care professionals representing 41 European hospitals completed all questionnaires (response rate: 81%), eventually including 22 baseline characteristics and 32 indicators. After ranking, 10 indicators were prioritized by both stakeholder groups. In addition, each stakeholder group highly prioritized one additional indicator. Following an additional online vote by the other group, these were both added to the final set. CONCLUSION: This study established a core indicator set of twenty-two baseline characteristics, eight outcome indicators, one structure indicator, and three process indicators for evaluating (quality of) EA care in Europe. These indicators, covering various aspects of EA care, will be implemented in the European Pediatric Surgical Audit to enable recognition of practice variation and focus EA care improvement initiatives.

Variability in the Reporting of Baseline Characteristics, Treatment, and Outcomes in Esophageal Atresia Publications: A Systematic Review.

INTRODUCTION: As survival rates of infants born with esophageal atresia (EA) have improved considerably, research interests are shifting from viability to morbidity and longer-term outcomes. This review aims to identify all parameters studied in recent EA research and determine variability in their reporting, utilization, and definition. MATERIALS AND METHODS: Following PRISMA guidelines, we performed a systematic review of literature regarding the main EA care process, published between 2015 and 2021, combining the search term "esophageal atresia" with "morbidity," "mortality," "survival," "outcome," or "complication." Described outcomes were extracted from included publications, along with study and baseline characteristics. RESULTS: From 209 publications that met the inclusion criteria, 731 studied parameters were extracted and categorized into patient characteristics (n = 128), treatment and care process characteristics (n = 338), and outcomes (n = 265). Ninety-two of these were reported in more than 5% of included publications. Most frequently reported characteristics were sex (85%), EA type (74%), and repair type (60%). Most frequently reported outcomes were anastomotic stricture (72%), anastomotic leakage (68%), and mortality (66%). CONCLUSION: This study demonstrates considerable heterogeneity of studied parameters in EA research, emphasizing the need for standardized reporting to compare results of EA research. Additionally, the identified items may help develop an informed, evidence-based consensus on outcome measurement in esophageal atresia research and standardized data collection in registries or clinical audits, thereby enabling benchmarking and comparing care between centers, regions, and countries.

Outcome of functional treatment with a Lucerne Cast in patients with hand fractures: a retrospective case series.

PURPOSE: The purpose of this study was to determine the clinical outcome of patients treated with a functional Lucerne Cast (LuCa) for different types of hand fractures. Static casting has traditionally been the preferred treatment for hand fractures. However, functional casting may lead to good functional outcomes, less stiffness, and earlier return to work. METHODS: A retrospective case series of all consecutive patients with a metacarpal or proximal phalangeal fracture treated with a LuCa between 2018 and 2019 was conducted. A total of 90 patients were included. Clinical data were collected directly from the patient's medical records. Functional outcome was assessed with the Michigan Hand Outcomes Questionnaire (MHQ). RESULTS: The median MHQ score was 95 (IQR 83-100) with a median follow-up of 23 months. Complications occurred in 7 of 90 patients (8%). Functional impairment occurred in two (2%) patients. Functional impairment was defined as stiffness, persisting rotational deformity, a boutonnière or swanneck deformity. Persisting pain or CRPS occurred in six (7%) patients. CONCLUSION: The LuCa shows to be effective in the functional treatment of both metacarpal and proximal phalangeal fractures with excellent patient-reported outcomes but at a relatively high rate of persisting pain.

Development and validation of a condition-specific quality of life instrument for adults with esophageal atresia: the SQEA questionnaire.

The importance of multidisciplinary long-term follow-up for adults born with esophageal atresia (EA) is increasingly recognized. Hence, a valid, condition-specific instrument to measure health-related quality of life (HRQoL) becomes imperative. This study aimed to develop and validate such an instrument for adults with EA. The Specific Quality of life in Esophageal atresia Adults (SQEA) questionnaire was developed through focus group-based item generation, pilot testing, item reduction and a multicenter, nationwide field test to evaluate the feasibility, reliability (internal and retest) and validity (structural, construct, criterion and convergent), in compliance with the consensus-based standards for the selection of health measurement instruments guidelines. After pilot testing (n = 42), items were reduced from 144 to 36 questions. After field testing (n = 447), three items were discarded based on item-response theory results. The final SQEA questionnaire (33 items) forms a unidimensional scale generating an unweighted total score. Feasibility, internal reliability (Cronbach's alpha 0.94) and test-retest agreement (intra-class coefficient 0.92) were good. Construct validity was discriminative for esophageal replacement (P < 0.001), dysphagia (P < 0.001) and airway obstruction (P = 0.029). Criterion validity showed a good correlation with dysphagia (area under the receiver operating characteristic 0.736). SQEA scores correlated well with other validated disease-specific HRQoL scales such as the GIQLI and SGRQ, but poorly with the more generic RAND-36. Overall, this first condition-specific instrument for EA adults showed satisfactory feasibility, reliability and validity. Additionally, it shows discriminative ability to detect disease burden. Therefore, the SQEA questionnaire is both a valid instrument to assess the HRQoL in EA adults and an interesting signaling tool, enabling clinicians to recognize more severely affected patients.