Determining the minimally clinically important difference for the pediatric liver transplant quality of life questionnaire.

PURPOSE: The Pediatric Liver Transplant Quality of Life (PeLTQL) questionnaire is a disease-specific patient reported outcome measure for pediatric liver transplant (LT) recipients. To-date, threshold values above which a change in PeLTQL score is considered meaningful to patients are unavailable. This study proposes the first values for the minimally clinically important difference (MCID) for the PeLTQL. METHODS: In this retrospective cohort study, anchor and distribution-based methods were used to estimate the MCID for the PeLTQL. Questionnaires completed between March 2013, and July 2022 were included if data from two sequential visits were available. An internal anchor question was used for anchor-based determination of the MCID. A final MCID estimate was ascertained from triangulation of all methods. RESULTS: PeLTQL data from 65 LT recipients (26 [40%] male, 17 [42%] biliary atresia, median age at LT 3.08 years [interquartile range 0.99-7.30]), and their caregivers were included for analysis. Median patient age at time of baseline PeLTQL completion was 13.84 (10.90-15.86) years. The MCID for self-PeLTQL total scores ranged from 4.53 to 8.46, and from 4.47 to 8.85 for proxy responses. By triangulation, the MCID of the PeLTQL total score was 6.45 and 6.78 for self and proxy responses respectively. CONCLUSION: A change in PeLTQL score of 6.5 or more points suggests a change in health status that is meaningful to the patient, providing the clinical team an opportunity to engage the patient's voice in reassessing current health status and management strategies.

Quality Indicators for Transition from Paediatric to Adult Care for Youth with Chronic Conditions: Protocol for an Online Modified Delphi Study.

BACKGROUND: The transition from paediatric to adult care poses a significant health system-level challenge impeding the delivery of quality health services for youth with chronic health conditions. In Canada and globally, the transition to adult care is regarded as a top priority in adolescent health in need of readily applicable, adaptable, and relevant national metrics to evaluate and benchmark transition success across disease populations and clinical care settings. Unfortunately, existing literature fails to account for the lack of engagement from youth and caregivers in developing indicators, and its applicability across chronic conditions, primary care involvement, and health equity considerations. OBJECTIVE: Our proposed study aims to establish a consensus-driven set of quality indicators for the transition to adult care that are universally applicable across physical, developmental, and/or mental health conditions, clinical care settings, and health jurisdictions. METHODS: Using an integrated knowledge translation (iKT) approach, a panel comprised of youth, caregivers, interdisciplinary healthcare providers, and health system leaders will be established to collaborate with our research team to ensure the study methodology, materials, and knowledge dissemination are suitable and reflect the perspectives of youth and their families. We will then conduct an iterative 3-round Online Modified Delphi (OMD) study (n= 160) to (1) compare and contrast the perspectives of youth, caregivers, health care providers, and health system leaders on quality indicators for transition, and (2) prioritize a key set of quality indicators for transition applicable across disease populations that are the most important, useful, and feasible in the Canadian context. Using the RAND/UCLA Appropriateness Method (RAM) multi-stage analytic approach, data from each panel and stakeholder group will be examined separately and compared to establish a key set of indicators endorsed by both panels. RESULTS: The study is funded by the Canadian Institutes of Health Research and Physicians Services Incorporated. CONCLUSIONS: This study will produce quality indicators to evaluate and inform action equitably to improve transition from paediatric to adult care for youth and their families in Canada.

Patient, caregiver and other knowledge user engagement in consensus-building healthcare initiatives: a scoping review protocol.

INTRODUCTION: Patient engagement and integrated knowledge translation (iKT) processes improve health outcomes and care experiences through meaningful partnerships in consensus-building initiatives and research. Consensus-building is essential for engaging a diverse group of experienced knowledge users in co-developing and supporting a solution where none readily exists or is less optimal. Patients and caregivers provide invaluable insights for building consensus in decision-making around healthcare, policy and research. However, despite emerging evidence, patient engagement remains sparse within consensus-building initiatives. Specifically, our research has identified a lack of opportunity for youth living with chronic health conditions and their caregivers to participate in developing consensus on indicators/benchmarks for transition into adult care. To bridge this gap and inform our consensus-building approach with youth/caregivers, this scoping review will synthesise the extent of the literature on patient and other knowledge user engagement in consensus-building healthcare initiatives. METHODS AND ANALYSIS: Following the scoping review methodology from Joanna Briggs Institute, published literature will be searched in MEDLINE, EMBASE, CINAHL and PsycINFO databases from inception to July 2023. Grey literature will be hand-searched. Two independent reviewers will determine the eligibility of articles in a two-stage process, with disagreements resolved by a third reviewer. Included studies must be consensus-building studies within the healthcare context that involve patient engagement strategies. Data from eligible studies will be extracted and charted on a standardised form. Abstracted data will be analysed quantitatively and descriptively, according to specific consensus methodologies, and patient engagement models and/or strategies. ETHICS AND DISSEMINATION: Ethics approval is not required for this scoping review protocol. The review process and findings will be shared with and informed by relevant knowledge users. Dissemination of findings will also include peer-reviewed publications and conference presentations. The results will offer new insights for supporting patient engagement in consensus-building healthcare initiatives. PROTOCOL REGISTRATION: https://osf.io/beqjr.

Severe acute hepatitis of unknown etiology in a large cohort of children.

BACKGROUND: We evaluated the proportion, clinical features, and outcomes of previously healthy children presenting to a large Canadian quaternary pediatric center with severe acute hepatitis of unknown etiology. METHODS: All patients with serum alanine aminotransferase (ALT) > 500 U/L or aspartate aminotransferase (AST) > 500 U/L between June 1, 2018, and May 31, 2022, at The Hospital for Sick Children, were identified. Subjects with only AST > 500 U/L were excluded. Clinical characteristics, investigations, and outcomes for patients without clear etiology for ALT > 500 U/L (severe acute hepatitis of unknown etiology) for our study period and from October 1 to May 31 of each year 2018-2021 were reviewed. RESULTS: Of 977 patients with ALT/AST> 500 U/L, 720 had only ALT > 500 U/L. We excluded age below 6 months (n = 99) or above 16 years (n = 66), known pre-existing liver conditions (n = 66), and ALT > 500 U/L in already admitted patients (n = 151). Among the remaining 338 children with ALT > 500 U/L at presentation, an etiology was identified in 303 subjects. 33 (9.8%) children [median age 6.1 y (range 0.5-15.5); 61% male] were confirmed as severe acute hepatitis of unknown etiology. Twenty patients (60.6%) were tested for blood adenovirus by PCR, and 1 (5%) was positive (serotype B7). Liver tissue specimens from 18 patients revealed no evidence of viral inclusions or adenovirus. Twelve (36.3%) presented with pediatric acute liver failure, with 8 (24.2%) requiring liver transplantation. There were no deaths. Hepatitis-associated aplastic anemia occurred in 5 (15%) patients. CONCLUSIONS: Of children presenting with severe acute hepatitis to a quaternary children's hospital over a 48-month period, 9.8% had unknown etiology with no change over time. Liver transplantation remains an important treatment strategy for those presenting with pediatric acute liver failure phenotype. The frequency of cases associated with human adenovirus infection was noncontributory.