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Innovative therapeutic approaches are needed to alleviate the burden of life-limiting, rare, and chronic conditions affecting children, adolescents, and young adults (CAYA). This includes a need for improved access to both clinical research and to non-approved or off-label therapies, together with, ultimately, more therapies achieving regulatory approval in Canada. The single patient study (SPS), also known as an open label individual patient (OLIP) study, was introduced by Health Canada to open access to non-marketed drugs where a clinical trial is not readily available, but the drug is considered too investigational to be managed on a standard Special Access Program. SPS is designed for patients who have a serious or life-threatening condition and have exhausted available treatment options. Our report summarizes this relatively new development in the Canadian regulatory environment and highlights the opportunities and challenges as identified by regulators, pharmaceutical representatives, academic researchers, and patient/parent advocates. [ABSTRACT FROM AUTHOR]

While paediatric care providers are often the first point of contact for children or youth experiencing mental health challenges, they may lack the resources (e.g. access to a multidisciplinary team) or training to adequately identify or manage such problems. This joint statement describes the key roles and competencies required to assess and address child and youth mental health problems, and the factors that optimize outcomes in this age group. Evidence-informed guidance on screening for and discussing mental health concerns with young people and families is provided. Preventive and therapeutic interventions with demonstrated efficacy in community care settings are discussed. This foundational statement also focuses on the changes to medical education, health systems, and health policy that are needed to improve clinical practice and advocacy efforts in Canada, including appropriate remuneration models, stepped-care approaches, targeted government funding, and professional training and education. [ABSTRACT FROM AUTHOR]

Anxiety disorders are the most common mental health concerns affecting Canadian children and adolescents. The Canadian Paediatric Society has developed two position statements that summarize current evidence regarding the diagnosis and management of anxiety disorders. Both statements offer evidence-informed guidance to support paediatric health care providers (HCPs) making decisions around the care of children and adolescents with these conditions. The objectives of Part 1, which focuses on assessment and diagnosis, are to: (1) review the epidemiology and clinical characteristics of anxiety disorders and (2) describe a process for assessment of anxiety disorders. Specific topics are reviewed, including prevalence, differential diagnosis, co-occurring conditions, and the process of assessment. Approaches are offered for standardized screening, history-taking, and observation. Associated features and indicators that distinguish anxiety disorders from developmentally appropriate fears, worries, and anxious feelings are considered. Note that when the word 'parent' (singular or plural) is used, it includes any primary caregiver and every configuration of family. [ABSTRACT FROM AUTHOR]

Aims To quantify thoracic lymphatic burden in paediatric Fontan patients using MRI and correlate with clinical status. Methods and results Paediatric Fontan patients (<18-years-old) with clinical cardiac MRI that had routine lymphatic 3D T2 fast spin echo (FSE) imaging performed from May 2017 to October 2019 were included. 'Lymphatic burden' was quantified by thresholding-based segmentation of the 3D T2 FSE maximum intensity projection image and indexed to body surface area, performed by two independent readers blinded to patient status. There were 48 patients (27 males) with median age at MRI of 12.9 (9.4–14.7) years, time from Fontan surgery to MRI of 9.1 (5.9–10.4) years, and follow-up time post-Fontan surgery of 9.4 (6.6–11.0) years. Intraclass correlation coefficient between two observers for lymphatic burden was 0.96 (0.94–0.98). Greater lymphatic burden correlated with post-Fontan operation hospital length of stay and duration of chest tube drainage (r s = 0.416, P = 0.004 and r s = 0.439, P = 0.002). Median lymphatic burden was greater in patients with chylous effusions immediately post-Fontan (178 (118–393) vs. 113 (46–190) mL/m2, P = 0.028), and in patients with composite adverse Fontan status (n = 13) defined by heart failure (n = 3), transplant assessment (n = 2), recurrent effusions (n = 6), Fontan thrombus (n = 2), and/or PLE (n = 6) post-Fontan (435 (137–822) vs. 114 (51–178) mL/m2, P = 0.003). Lymphatic burden > 600 mL/m2 was associated with late adverse Fontan status with sensitivity of 57% and specificity of 95%. Conclusion Quantification of MR lymphatic burden is a reliable tool to assess the lymphatics post-Fontan and is associated with clinical status. [ABSTRACT FROM AUTHOR]