Case report: Success of allogeneic hematopoietic stem cell transplantation for refractory systemic-onset juvenile idiopathic arthritis.

Objectives: This study reports cases of systemic-onset juvenile idiopathic arthritis (sJIA) who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT) at our center and reviews published outcomes of allo-HSCT in sJIA. Methods: We present a case report of two patients with sJIA who underwent allo-HSCT at a tertiary pediatric hospital. Each patient's disease course and allo-HSCT protocol/outcome are described. Outcomes of published cases of allo-HSCT in sJIA were compared to our experience. Results: Two patients with sJIA had allo-HSCT. Both failed multiple lines of disease-modifying anti-rheumatic drugs and experienced severe disease/treatment-related complications. Despite post-HSCT complications, both recovered without sequelae. Five years post-HSCT, patient 1 is in complete remission (CR) and is off medications. Patient 2 was in CR until 11 months post-HSCT after which he developed three disease flares. At 4 years post-HSCT he is currently in CR on Adalimumab monotherapy. Engraftment was excellent with a donor chimerism of 100% for patient 1 and 93% for patient 2. In the literature, the outcome of allo-HSCT is reported in 13 sJIA patients. When merging those with our 2 patients, 1/15 patients died and 13/14 achieved CR, of which 12 are off medications (median [range] follow-up: 2.2 [0.2-7.0] years). Extended follow-up data on 11 of the 13 reported sJIA patients showed that an additional 3 patients flared at 3, 4, and 10 years post-HSCT. Conclusion: We report two patients with severe/refractory sJIA who underwent successful allo-HSCT and achieved CR. Allo-HSCT is a potential curative option for severe/refractory sJIA. It should be considered only after failure of conventional sJIA treatments and when an HLA-matched donor is available in order to lower transplant-related mortality. The outcomes of reported sJIA patients who received allo-HSCT are encouraging but long-term follow-up data are needed to better characterized the risk-benefit ratio of this procedure.

Paediatric inflammatory multisystem syndrome in Canada: population-based surveillance and role of SARS-CoV-2 linkage.

BACKGROUND: Paediatric inflammatory multisystem syndrome (PIMS) is a rare condition temporally associated with SARS-CoV-2 infection. Using national surveillance data, we compare presenting features and outcomes among children hospitalized with PIMS by SARS-CoV-2 linkage, and identify risk factors for intensive care (ICU). METHODS: Cases were reported to the Canadian Paediatric Surveillance Program by a network of >2800 pediatricians between March 2020 and May 2021. Patients with positive versus negative SARS-CoV-2 linkages were compared, with positive linkage defined as any positive molecular or serologic test or close contact with confirmed COVID-19. ICU risk factors were identified with multivariable modified Poisson regression. RESULTS: We identified 406 children hospitalized with PIMS, including 49.8% with positive SARS-CoV-2 linkages, 26.1% with negative linkages, and 24.1% with unknown linkages. The median age was 5.4 years (IQR 2.5-9.8), 60% were male, and 83% had no comorbidities. Compared to cases with negative linkages, children with positive linkages experienced more cardiac involvement (58.8% vs. 37.4%; p < 0.001), gastrointestinal symptoms (88.6% vs. 63.2%; p < 0.001), and shock (60.9% vs. 16.0%; p < 0.001). Children aged ≥6 years and those with positive linkages were more likely to require ICU. CONCLUSIONS: Although rare, 30% of PIMS hospitalizations required ICU or respiratory/hemodynamic support, particularly those with positive SARS-CoV-2 linkages. IMPACT: We describe 406 children hospitalized with paediatric inflammatory multisystem syndrome (PIMS) using nationwide surveillance data, the largest study of PIMS in Canada to date. Our surveillance case definition of PIMS did not require a history of SARS-CoV-2 exposure, and we therefore describe associations of SARS-CoV-2 linkages on clinical features and outcomes of children with PIMS. Children with positive SARS-CoV-2 linkages were older, had more gastrointestinal and cardiac involvement, and hyperinflammatory laboratory picture. Although PIMS is rare, one-third required admission to intensive care, with the greatest risk amongst those aged ≥6 years and those with a SARS-CoV-2 linkage.

Canadian Rheumatology Association Recommendations for the Screening, Monitoring, and Treatment of Juvenile Idiopathic Arthritis-Associated Uveitis.

OBJECTIVE: To develop Canadian recommendations for the screening, monitoring, and treatment of uveitis associated with juvenile idiopathic arthritis (JIA). METHODS: Recommendations were developed using the Grading of Recommendations Assessment, Development, and Evaluation (GRADE)-ADOLOPMENT approach. A working group of 14 pediatric rheumatologists, 6 ophthalmologists, 2 methodologists, and 3 caregiver/patient representatives reviewed recent American College of Rheumatology (ACR)/Arthritis Foundation (AF) recommendations and worked in pairs to develop evidence-to-decision (EtD) tables. A survey to assess agreement and recommendations requiring group discussion was completed. EtD tables were presented, discussed, and voted upon at a virtual meeting, to produce the final recommendations. A health equity framework was applied to all aspects of the adolopment process including the EtD tables, survey responses, and virtual meeting discussion. RESULTS: The survey identified that 7 of the 19 recommendations required rigorous discussion. Seventy-five percent of working group members attended the virtual meeting to discuss controversial topics as they pertained to the Canadian environment, including timing to first eye exam, frequency of screening, escalation criteria for systemic and biologic therapy, and the role of nonbiologic therapies. Equity issues related to access to care and advanced therapeutics across Canadian provinces and territories were highlighted. Following the virtual meeting, 5 recommendations were adapted, 2 recommendations were removed, and 1 was developed de novo. CONCLUSION: Recommendations for JIA-associated uveitis were adapted to the Canadian context by a working group of pediatric rheumatologists, ophthalmologists with expertise in the management of uveitis, and parent/patient input, taking into consideration cost, equity, and access.

Resource use and disease severity of children hospitalized for COVID-19 versus multisystem inflammatory syndrome in children (MIS-C) in Canada.

Background: Direct comparisons of paediatric hospitalizations for acute coronavirus disease 2019 (COVID-19) and multisystem inflammatory syndrome in children (MIS-C) can inform health system planning. We describe the absolute and relative hospital burden of acute paediatric COVID-19 and MIS-C in Canada. Methods: This national prospective study was conducted via the Canadian Paediatric Surveillance Program from March 2020-May 2021. Children younger than 18 years old and hospitalized for acute COVID-19 or MIS-C were included in the analysis. Outcomes included supplemental oxygen (low-flow oxygen or high-flow nasal cannula), ventilation (non-invasive or conventional mechanical), vasopressors, paediatric intensive care unit (PICU) admission, or death. Adjusted risk differences (aRD) and 95% confidence intervals (CI) were calculated to identify factors associated with each diagnosis. Results: Overall, we identified 330 children hospitalized for acute COVID-19 (including five deaths) and 208 hospitalized for MIS-C (including zero deaths); PICU admission was required for 49.5% of MIS-C hospitalizations versus 18.2% of acute COVID-19 hospitalizations (aRD 20.3; 95% CI, 9.9-30.8). Resource use differed by age, with children younger than one year hospitalized more often for acute COVID-19 (aRD 43.4% versus MIS-C; 95% CI, 37.7-49.1) and more children 5-11 years hospitalized for MIS-C (aRD 38.9% vs. acute COVID-19; 95% CI, 31.0-46.9). Conclusion: While there were more hospitalizations and deaths from acute paediatric COVID-19, MIS-C cases were more severe, requiring more intensive care and vasopressor support. Our findings suggest that both acute COVID-19 and MIS-C should be considered when assessing the overall burden of severe acute respiratory syndrome coronavirus 2 in hospitalized children.

Pernio as the clinical presentation of celiac disease: A case report.

We present the case of a 12-year-old girl with severe pernio as the sole clinical presentation of celiac disease (CD), without associated gastrointestinal symptoms. Lesions greatly improved once a gluten free diet was initiated. At 5-year follow-up, she remains in clinical remission throughout the year with no pharmacological treatment, without skin lesions flare-up in the winter months.

What we call what we do affects how we do it: a new nomenclature for simulation research in medical education.

Rapid technological advances and concern for patient safety have increased the focus on simulation as a pedagogical tool for educating health care providers. To date, simulation research scholarship has focused on two areas; evaluating instructional designs of simulation programs, and the integration of simulation into a broader educational context. However, these two categories of research currently exist under a single label-Simulation-Based Medical Education. In this paper we argue that introducing a more refined nomenclature within which to frame simulation research is necessary for researchers, to appropriately design research studies and describe their findings, and for end-point users (such as program directors and educators), to more appropriately understand and utilize this evidence.

Rethinking programme evaluation in health professions education: beyond 'did it work?'.

CONTEXT: For nearly 40 years, outcome-based models have dominated programme evaluation in health professions education. However, there is increasing recognition that these models cannot address the complexities of the health professions context and studies employing alternative evaluation approaches that are appearing in the literature. A similar paradigm shift occurred over 50 years ago in the broader discipline of programme evaluation. Understanding the development of contemporary paradigms within this field provides important insights to support the evolution of programme evaluation in the health professions. METHODS: In this discussion paper, we review the historical roots of programme evaluation as a discipline, demonstrating parallels with the dominant approach to evaluation in the health professions. In tracing the evolution of contemporary paradigms within this field, we demonstrate how their aim is not only to judge a programme's merit or worth, but also to generate information for curriculum designers seeking to adapt programmes to evolving contexts, and researchers seeking to generate knowledge to inform the work of others. DISCUSSION: From this evolution, we distil seven essential elements of educational programmes that should be evaluated to achieve the stated goals. Our formulation is not a prescriptive method for conducting programme evaluation; rather, we use these elements as a guide for the development of a holistic 'programme of evaluation' that involves multiple stakeholders, uses a combination of available models and methods, and occurs throughout the life of a programme. Thus, these elements provide a roadmap for the programme evaluation process, which allows evaluators to move beyond asking whether a programme worked, to establishing how it worked, why it worked and what else happened. By engaging in this process, evaluators will generate a sound understanding of the relationships among programmes, the contexts in which they operate, and the outcomes that result from them.

Vaccination coverage in children with juvenile idiopathic arthritis followed at a paediatric tertiary care centre.

OBJECTIVE: To evaluate the vaccination coverage rate of patients with JIA followed at a paediatric tertiary care centre and to determine the coverage rate for individual vaccines required as per the Quebec Immunization Protocol. METHODS: Consecutive JIA patients coming for their scheduled visit were included if they were between 2 and 18 years of age and if they had an available written immunization record. Descriptive statistics were used to evaluate the proportion of children with complete vaccination status according to the Quebec Immunization Protocol at 2.5, 10.5 years and at their last clinic visit. RESULTS: A total of 200 patients were included. Complete vaccination according to schedule was identified in only 52% of patients at 2.5 years, 68% at 10.5 years and 61% at their last clinic visit. The vaccination coverage rate for individual vaccines was good overall with the exception of low measles, mumps and rubella vaccine coverage at 2.5 years (58%). CONCLUSION: Despite overall good vaccination coverage rate for individual vaccines, only 61% of our cohort had a complete vaccination status at their last clinic visit. Measures to optimize vaccination coverage, such as catch-up vaccination, should be implemented when possible.

The application of the challenge point framework in medical education.

OBJECTIVES: The current paper describes a model of learning that has been used to produce efficient learning, thus yielding greater retention of information and superior performance under stress. In this paper, the model is applied to the learning of technical skills. STRUCTURE: After a brief review of the learning-performance paradox and other relevant literature from the field of movement science, the benefits of challenge and adversity for learning are discussed in the context of a framework for learning known as the challenge point framework (CPF). The framework is based on laboratory and field studies of methods that have been shown to consistently enhance learning, and is used to model and generate insight into the relationships between practice protocols and the learning that results from them. APPLICATION: The practical application of the CPF to simulation-based medical education and training is described. Firstly, a simple conceptual model that utilises three key elements to adjust the functional difficulty of the tasks to be learned is outlined. Secondly, a number of assessment strategies that may be necessary to ensure that the trainee remains in the optimal learning zone are proposed. Thirdly, a practical example is used to demonstrate how to utilise this conceptual model to design simulation environments suitable for teaching an endotracheal intubation task to beginners and more advanced trainees.

Evaluating pain education programs: an integrated approach.

Evaluation of educational programs and assessment of learning are essential to maintain high-standard health science education, which includes pain education. Current models of program evaluations applied to the education of the health professions, such as the Kirkpatrick model, are mainly outcome based. More recently, efforts have been made to examine other process-based models such as the Context Input Process Product model. The present article proposes an approach that integrates both outcome- and process-based models with models of clinical performance assessment to provide a deeper understanding of a program function. Because assessment instruments are a critical part of program evaluation, it is suggested that standardization and rigour should be used in their selection, development and adaptation. The present article suggests an alternative to currently used models in pain education evaluation.

Solitary tumefactive demyelinating lesions in children.

Solitary tumefactive demyelination is rare in children, and the diagnosis is often conferred after brain biopsy. The authors report 3 children with solitary tumefactive demyelination and provide clinical and paraclinical clues to aid the clinician in reaching a diagnosis using a noninvasive approach.