Pediatrics Commons^™

Instituting Robotic Pediatric Urologic Surgery In The Canadian Healthcare System: Evaluating The Feasibility And Outcomes Of Robot-Assisted Pyeloplasty And Ureteric Reimplantation, Noah Stern, Peter Wang, Sumit Dave

Paediatrics Publications

Introduction: Robotic pediatric urologic surgery has gained widespread adoption over the last decade. This article describes our experience in instituting the first pediatric urologic robotic surgery program in Canada. We evaluated the feasibility and safety of instituting pediatric robot-assisted urologic surgery and report our early outcomes for robot-assisted pyeloplasty (RAP) and ureteric reimplantation (RUR). Methods: We prospectively evaluated all patients undergoing RAP and RUR by a single surgeon from June 2013 to March 2019. Demographic and clinical data were prospectively collected and included sex, age, and preoperative grade of hydronephrosis or reflux. Descriptive statistics were performed, and comparisons were made …

Non-Steroidal Or Opioid Analgesia Use For Children With Musculoskeletal Injuries (The No Ouch Study): Statistical Analysis Plan., Anna Heath, Maryna Yaskina, Gareth Hopkin, Terry P Klassen, Christopher Mccabe, Martin Offringa, Petros Pechlivanoglou, Juan David Rios, Naveen Poonai, Samina Ali

Paediatrics Publications

BACKGROUND: Pediatric musculoskeletal injuries cause moderate to severe pain, which should ideally be addressed upon arrival to the emergency department (ED). Despite extensive research in ED-based pediatric pain treatment, recent studies confirm that pain management in this setting remains suboptimal. The No OUCH study consist of two complementary, randomized, placebo-controlled trials that will run simultaneously for patients presenting to the ED with an acute limb injury and a self-reported pain score of at least 5/10, measured via a verbal numerical rating scale (vNRS). Caregiver/parent choice will determine whether patients are randomized to the two-arm or three-arm trial. In the two-arm …

Quantification And Characterization Of Granulocyte Macrophage Colony-Stimulating Factor Activated Human Peripheral Blood Mononuclear Cells By Fluorine-19 Cellular Mri In An Immunocompromised Mouse Model, C Fink, M Smith, O C Sehl, J M Gaudet, T C Meagher, N A Sheikh, Jimmy Dikeakos, Michael Rieder, P J Foster, G A Dekaban

Paediatrics Publications

PURPOSE: The purpose of this study was to test fluorine-19 (19F) cellular magnetic resonance (MRI) as a non-invasive imaging modality to track therapeutic cell migration as a surrogate marker of immunotherapeutic effectiveness.

MATERIALS AND METHODS: Human peripheral blood mononuclear cell- (PBMC)-derived antigen presenting cell (APC) were labeled with a 19F-perfluorocarbon (PFC) and/or activated with granulocyte macrophage colony-stimulating factor (GM-CSF). Viability, phenotype and cell lineage characterization preceded 19F cellular MRI of PFC

RESULTS: A high proportion of PBMC incorporated PFC without affecting viability, phenotype or cell lineage composition. PFC

CONCLUSION: 19F cellular MRI is a non-invasive imaging technique capable of detecting …

Priority Outcomes In Critically Ill Children: A Patient And Parent Perspective, Nora Fayed, Saoirse Cameron, Douglas Fraser Md, Phd, Frcpc, Jill I Cameron, Samah Al-Harbi, Racquel Simpson, Maha Wakim, Lily Chiu, Karen Choong

Paediatrics Publications

BACKGROUND: Outcomes in pediatric critical care research are typically selected by the researcher.

OBJECTIVES: (1) To identify outcomes prioritized by patients and their families following a critical illness and (2) to determine the overlap between patient-centered and researcher-selected study outcomes.

METHODS: An exploratory descriptive qualitative study nested within a longitudinal cohort study conducted in 2 pediatric intensive care units (PICUs). Participants were purposively sampled from the primary cohort to ensure adequate demographic representation. Qualitative descriptive approaches based on naturalistic observation were used to collect data and analyze results. Data were coded by using the International Classification of Functioning, Disability, and …

New Aspects Of The Epigenetics Of Pancreatic Carcinogenesis., Murat Toruner, Martin E. Fernandez-Zapico, Christopher Pin

Paediatrics Publications

Pancreatic cancer remains among the deadliest forms of cancer with a 5 year survival rate less than 10%. With increasing numbers being observed, there is an urgent need to elucidate the pathogenesis of pancreatic cancer. While both contribute to disease progression, neither genetic nor environmental factors completely explain susceptibility or pathogenesis. Defining the links between genetic and environmental events represents an opportunity to understand the pathogenesis of pancreatic cancer. Epigenetics, the study of mitotically heritable changes in genome function without a change in nucleotide sequence, is an emerging field of research in pancreatic cancer. The main epigenetic mechanisms include DNA …

A Pragmatic Randomized Controlled Trial Of Multi-Dose Oral Ondansetron For Pediatric Gastroenteritis (The Dose-Age Study): Statistical Analysis Plan., Anna Heath, Juan David Rios, Sarah Williamson-Urquhart, Petros Pechlivanoglou, Martin Offringa, Christopher Mccabe, Gareth Hopkin, Amy C Plint, Andrew Dixon, Darcy Beer, Serge Gouin, Gary Joubert, Terry P Klassen, Stephen B Freedman

Paediatrics Publications

BACKGROUND: Acute gastroenteritis is a leading cause of emergency department visits and hospitalizations among children in North America. Oral-rehydration therapy is recommended for children with mild-to-moderate dehydration, but children who present with vomiting are frequently offered intravenous rehydration in the emergency department (ED). Recent studies have demonstrated that the anti-emetic ondansetron can reduce vomiting, intravenous rehydration, and hospitalization when administered in the ED to children with dehydration. However, there is little evidence of additional benefit from prescribing ondansetron beyond the initial ED dose. Moreover, repeat dosing may increase the frequency of diarrhea. Despite the lack of evidence and potential adverse …

Medium-Term Complications Associated With Coronary Artery Aneurysms After Kawasaki Disease: A Study From The International Kawasaki Disease Registry., Brian W Mccrindle, Cedric Manlhiot, Jane W Newburger, Ashraf S Harahsheh, Therese M Giglia, Frederic Dallaire, Kevin Friedman, Tisiana Low, Kyle Runeckles, Mathew Mathew, Andrew S Mackie, Nadine F Choueiter, Pei-Ni Jone, Shelby Kutty, Anji T Yetman, Geetha Raghuveer, Elfriede Pahl, Kambiz Norozi, Kimberly E Mchugh, Jennifer S Li, Sarah D De Ferranti, Nagib Dahdah

Paediatrics Publications

Background Coronary artery aneurysms (CAAs) may occur after Kawasaki disease (KD) and lead to important morbidity and mortality. As CAA in patients with KD are rare and heterogeneous lesions, prognostication and risk stratification are difficult. We sought to derive the cumulative risk and associated factors for cardiovascular complications in patients with CAAs after KD. Methods and Results A 34-institution international registry of 1651 patients with KD who had CAAs (maximum CAA

Consensus Guidelines For Management Of Hyperammonaemia In Paediatric Patients Receiving Continuous Kidney Replacement Therapy., Rupesh Raina, Jirair K Bedoyan, Uta Lichter-Konecki, Philippe Jouvet, Stefano Picca, Nicholas Ah Mew, Marcel C Machado, Ronith Chakraborty, Meghana Vemuganti, Manpreet K Grewal, Timothy Bunchman, Sidharth Kumar Sethi, Vinod Krishnappa, Mignon Mcculloch, Khalid Alhasan, Arvind Bagga, Rajit K Basu, Franz Schaefer, Guido Filler, Bradley A Warady

Paediatrics Publications

Hyperammonaemia in children can lead to grave consequences in the form of cerebral oedema, severe neurological impairment and even death. In infants and children, common causes of hyperammonaemia include urea cycle disorders or organic acidaemias. Few studies have assessed the role of extracorporeal therapies in the management of hyperammonaemia in neonates and children. Moreover, consensus guidelines are lacking for the use of non-kidney replacement therapy (NKRT) and kidney replacement therapies (KRTs, including peritoneal dialysis, continuous KRT, haemodialysis and hybrid therapy) to manage hyperammonaemia in neonates and children. Prompt treatment with KRT and/or NKRT, the choice of which depends on the …

Teaching Neuroimages: Reversible Neuroimaging Findings During Treatment Of Infantile Spasms With Vigabatrin, David Dongkyung Kim, Amit Kumar Sharma, Manas Sharma, Andrea Andrade

Paediatrics Publications

A 10-month-old boy with global developmental delay presented to clinic with a few months of infantile spasms occurring multiple times a day. His seizures continued despite vigabatrin (dosed at 133 mg/kg/d), levetiracetam, and steroid therapy. On vigabatrin, routine follow-up MRI showed abnormal signal change , which may occur in 30.9% of patients.1 Risk is associated with a high peak dose but not cumulative.2 These findings are largely asymptomatic although rarely patients can present with hyperkinetic disorders.2 The imaging findings resolved on 4-month follow-up after tapering vigabatrin. At 18 months of age, the patient continues to have 1 …

Perfusion And Metabolic Neuromonitoring During Ventricular Taps In Infants With Post-Hemorrhagic Ventricular Dilatation., Ajay Rajaram, Lawrence C M Yip, Daniel Milej, Marianne Suwalski, Matthew Kewin, Marcus Lo, Jeffrey J L Carson, Victor Han, Soume Bhattacharya, Mamadou Diop, Sandrine De Ribaupierre, Keith St Lawrence

Paediatrics Publications

Post-hemorrhagic ventricular dilatation (PHVD) is characterized by a build-up of cerebral spinal fluid (CSF) in the ventricles, which increases intracranial pressure and compresses brain tissue. Clinical interventions (i.e., ventricular taps, VT) work to mitigate these complications through CSF drainage; however, the timing of these procedures remains imprecise. This study presents Neonatal NeuroMonitor (NNeMo), a portable optical device that combines broadband near-infrared spectroscopy (B-NIRS) and diffuse correlation spectroscopy (DCS) to provide simultaneous assessments of cerebral blood flow (CBF), tissue saturation (S

Screening And Treatment Outcomes In Adults And Children With Type 1 Diabetes And Asymptomatic Celiac Disease: The Cd-Diet Study., Farid H Mahmud, Antoine B M Clarke, Kariym C Joachim, Esther Assor, Charlotte Mcdonald, Fred Saibil, Heather A Lochnan, Zubin Punthakee, Amish Parikh, Andrew Advani, Baiju R Shah, Bruce A Perkins, Caroline S Zuijdwijk, David R Mack, Dror Koltin, Emilia N De Melo, Eugene Hsieh, Geetha Mukerji, Jeremy Gilbert, Kevin Bax, Margaret L Lawson, Maria Cino, Melanie D Beaton, Navaaz A Saloojee, Olivia Lou, Patricia H Gallego, Premysl Bercik, Robyn L Houlden, Ronnie Aronson, Susan E Kirsch, William G Paterson, Margaret A Marcon

Paediatrics Publications

OBJECTIVE: To describe celiac disease (CD) screening rates and glycemic outcomes of a gluten-free diet (GFD) in patients with type 1 diabetes who are asymptomatic for CD.

RESEARCH DESIGN AND METHODS: Asymptomatic patients (8-45 years) were screened for CD. Biopsy-confirmed CD participants were randomized to GFD or gluten-containing diet (GCD) to assess changes in HbA

RESULTS: Adults had higher CD-seropositivity rates than children (6.8% [95% CI 4.9-8.2%,

CONCLUSIONS: CD is frequently observed in asymptomatic patients with type 1 diabetes, and clinical vigilance is warranted with initiation of a GFD.

Atrx Deletion In Neurons Leads To Sexually Dimorphic Dysregulation Of Mir-137 And Spatial Learning And Memory Deficits., Renee J. Tamming, Vanessa Dumeaux, Yan Jiang, Sarfraz Shafiq, Luana Langlois, Jacob Ellegood, Lily R. Qiu, Jason P. Lerch, Nathalie G. Bérubé

Paediatrics Publications

ATRX gene mutations have been identified in syndromic and non-syndromic intellectual disabilities in humans. ATRX is known to maintain genomic stability in neuroprogenitor cells, but its function in differentiated neurons and memory processes remains largely unresolved. Here, we show that the deletion of neuronal Atrx in mice leads to distinct hippocampal structural defects, fewer presynaptic vesicles, and an enlarged postsynaptic area at CA1 apical dendrite-axon junctions. We identify male-specific impairments in long-term contextual memory and in synaptic gene expression, linked to altered miR-137 levels. We show that ATRX directly binds to the miR-137 locus and that the enrichment of the …

Transient Hyponatremia Of Prematurity Caused By Mild Bartter Syndrome Type Ii: A Case Report., Subhrata Verma, Rahul Chanchlani, Victoria Mok Siu, Guido Filler

Paediatrics Publications

BACKGROUND: Bartter syndrome subtypes are a group of rare renal tubular diseases characterized by impaired salt reabsorption in the tubule, specifically the thick ascending limb of Henle's loop. Clinically, they are characterized by the association of hypokalemic metabolic alkalosis, hypercalciuria, nephrocalcinosis, increased levels of plasma renin and aldosterone, low blood pressure and vascular resistance to angiotensin II. Bartter syndrome type II is caused by mutations in the renal outer medullary potassium channel (ROMK) gene (KCNJ1), can present in the newborn period and typically requires lifelong therapy.

CASE PRESENTATION: We describe a case of a prematurely born female infant presenting with …

Effects Of A Postnatal Atrx Conditional Knockout In Neurons On Autism-Like Behaviours In Male And Female Mice., Nicole Martin-Kenny, Nathalie G Bérubé

Paediatrics Publications

BACKGROUND: Alpha-thalassemia/mental retardation, X-linked, or ATRX, is an autism susceptibility gene that encodes a chromatin remodeler. Mutations of ATRX result in the ATR-X intellectual disability syndrome and have been identified in autism spectrum disorder (ASD) patients. The mechanisms by which ATRX mutations lead to autism and autistic-like behaviours are not yet known. To address this question, we generated mice with postnatal Atrx inactivation in excitatory neurons of the forebrain and performed a battery of behavioural assays that assess autistic-like behaviours.

METHODS: Male and female mice with a postnatal conditional ablation of ATRX were generated using the Cre/lox system under the …

Fri0547 The Effect Of Corrected Inflammation, Oxidative Stress And Endothelial Dysfunction On Fmd Levels In Patients With Selected Chronic Diseases: A Quasi-Experimental Study., Mahmut Ilker Yilmaz, Micol Romano, Mustafa Kemal Basarali, Abdelbaset Elzagallaai, Murat Karaman, Zeynep Demir, Muhammet Fatih Demir, Fatih Akcay, Melik Seyrek, Nuri Haksever, David Piskin, Rolando Cimaz, Michael Rieder, Erkan Demirkaya

Paediatrics Publications

While the pathophysiology of chronic disorders varies there are three basic mechanisms - inflammation, oxidative stress and endothelial dysfunction - that are common in many chronic diseases. However, the failure of these mechanisms to work synchronously can lead to morbidity complicating the course of many chronic diseases. We analyzed data of 178 patients from cohorts with selected chronic diseases in this quasi-experimental study. Endothelial dysfunction was determined by flow-mediated dilatation (FMD) and asymmetric dimethylarginine (ADMA) levels. Serum ADMA, high sensitive C-reactive protein (hs-CRP), serum PTX3, malondialdehyde (MDA), Cu/Zn-superoxide dismutase (Cu/Zn-SOD), glutathione peroxidase (GSH-Px) levels and FMD were studied in baseline …

The Effect Of Corrected Inflammation, Oxidative Stress And Endothelial Dysfunction On Fmd Levels In Patients With Selected Chronic Diseases: A Quasi-Experimental Study., Mahmut Ilker Yilmaz, Micol Romano, Mustafa Kemal Basarali, Abdelbaset Elzagallaai, Murat Karaman, Zeynep Demir, Muhammet Fatih Demir, Fatih Akcay, Melik Seyrek, Nuri Haksever, David Piskin, Rolando Cimaz, Michael Rieder, Erkan Demirkaya

Paediatrics Publications

While the pathophysiology of chronic disorders varies there are three basic mechanisms - inflammation, oxidative stress and endothelial dysfunction - that are common in many chronic diseases. However, the failure of these mechanisms to work synchronously can lead to morbidity complicating the course of many chronic diseases. We analyzed data of 178 patients from cohorts with selected chronic diseases in this quasi-experimental study. Endothelial dysfunction was determined by flow-mediated dilatation (FMD) and asymmetric dimethylarginine (ADMA) levels. Serum ADMA, high sensitive C-reactive protein (hs-CRP), serum PTX3, malondialdehyde (MDA), Cu/Zn-superoxide dismutase (Cu/Zn-SOD), glutathione peroxidase (GSH-Px) levels and FMD were studied in baseline …

Prevalence And Clinical Features Of Inflammatory Bowel Diseases Associated With Monogenic Variants, Identified By Whole-Exome Sequencing In 1000 Children At A Single Center, Eileen Crowley, Neil Warner, Jie Pan, Sam Khalouei, Abdul Elkadri, Karoline Fiedler, Justin Foong, Andrei L Turinsky, Dana Bronte-Tinkew, Shiqi Zhang

Paediatrics Publications

BACKGROUND & AIMS: A proportion of infants and young children with inflammatory bowel diseases (IBDs) have subtypes associated with a single gene variant (monogenic IBD). We aimed to determine the prevalence of monogenic disease in a cohort of pediatric patients with IBD.

METHODS: We performed whole-exome sequencing analyses of blood samples from an unselected cohort of 1005 children with IBD, aged 0-18 years (median age at diagnosis, 11.96 years) at a single center in Canada and their family members (2305 samples total). Variants believed to cause IBD were validated using Sanger sequencing. Biopsies from patients were analyzed by immunofluorescence and …

Randomized Phase 2 Trial And Open-Label Extension Of Domagrozumab In Duchenne Muscular Dystrophy., Kathryn R. Wagner, Hoda Z. Abdel-Hamid, Jean K. Mah, Craig Campbell, Michela Guglieri, Francesco Muntoni, Yasuhiro Takeshima, Craig M. Mcdonald, Anna Kostera-Pruszczyk, Peter Karachunski, Russell J. Butterfield, Eugenio Mercuri, Chiara Fiorillo, Enrico S. Bertini, Cuixia Tian, Jeffery Statland, Alesia B. Sadosky, Vivek S. Purohit, Sarah P. Sherlock, Jeffrey P. Palmer, Michael Binks, Lawrence Charnas, Shannon Marraffino, Brenda L. Wong

Paediatrics Publications

We report results from a phase 2, randomized, double-blind, 2-period trial (48 weeks each) of domagrozumab and its open-label extension in patients with Duchenne muscular dystrophy (DMD). Of 120 ambulatory boys (aged 6 to <16 >years) with DMD, 80 were treated with multiple ascending doses (5, 20, and 40 mg/kg) of domagrozumab and 40 treated with placebo. The primary endpoints were safety and mean change in 4-stair climb (4SC) time at week 49. Secondary endpoints included other functional tests, pharmacokinetics, and pharmacodynamics. Mean (SD) age was 8.4 (1.7) and 9.3 (2.3) years in domagrozumab- and placebo-treated patients, respectively. Difference in mean …

Authorizing Medical Cannabis For Children., Michael Rieder

Paediatrics Publications

No abstract provided.

Determining A Bayesian Predictive Power Stopping Rule For Futility In A Non-Inferiority Trial With Binary Outcomes., Anna Heath, Martin Offringa, Petros Pechlivanoglou, Juan David Rios, Terry P Klassen, Naveen Poonai, Eleanor Pullenayegum

Paediatrics Publications

Background/Aims: Non-inferiority trials investigate whether a novel intervention, which typically has other benefits (i.e., cheaper or safer), has similar clinical effectiveness to currently available treatments. In situations where interim evidence in a non-inferiority trial suggests that the novel treatment is truly inferior, ethical concerns with continuing randomisation to the "inferior" intervention are raised. Thus, if interim data indicate that concluding non-inferiority at the end of the trial is unlikely, stopping for futility should be considered. To date, limited examples are available to guide the development of stopping rules for non-inferiority trials.

Methods: We used a Bayesian predictive power approach to …

A Systematic Review For The Management Of The Genetically Defined Il-1-Mediated Autoinflammatory Diseases, Caps, Traps, Mkd And Dira, R. Berard, M. Romano, Zs Arici, D. Piskin, O. Jones, K. Durrant, R. Goldbach-Mansky, M. Gattorno, E. Demirkaya

Paediatrics Publications

No abstract provided.

The Impact Of Covid-19 On Canadian Urology Residents, Ernest Pang Chan, Leandra Stringer, Peter Z.T. Wang, Sumit Dave, Jeffrey D. Campbell

Paediatrics Publications

No abstract provided.

Multi-Dose Oral Ondansetron For Pediatric Gastroenteritis: Study Protocol For The Multi-Dose Oral Ondansetron For Pediatric Acute Gastroenteritis (Dose-Age) Pragmatic Randomized Controlled Trial, Stephen B Freedman, Sarah Williamson-Urquhart, Anna Heath, Petros Pechlivanoglou, Gareth Hopkin, Serge Gouin, Amy C Plint, Andrew Dixon, Darcy Beer, Gary Joubert, Christopher Mccabe, Yaron Finkelstein, Terry P Klassen

Paediatrics Publications

BACKGROUND: There are limited treatment options that clinicians can provide to children presenting to emergency departments with vomiting secondary to acute gastroenteritis. Based on evidence of effectiveness and safety, clinicians now routinely administer ondansetron in the emergency department to promote oral rehydration therapy success. However, clinicians are also increasingly providing multiple doses of ondansetron for home use, creating unquantified cost and health system resource use implications without any evidence to support this expanding practice.

METHODS/DESIGN: DOSE-AGE is a randomized, placebo-controlled, double-blinded, six-center, pragmatic clinical trial being conducted in six Canadian pediatric emergency departments (EDs). In September 2019 the study began …

Histone Deacetylase 1 And 2 Drive Differentiation And Fusion Of Progenitor Cells In Human Placental Trophoblasts, Gargi Jaju Bhattad, Mariyan J. Jeyarajah, Megan G. Mcgill, Vanessa Dumeaux, Hiroaki Okae, Takahiro Arima, Patrick Lajoie, Nathalie G. Bérubé, Stephen J. Renaud

Paediatrics Publications

Cell fusion occurs when several cells combine to form a multinuclear aggregate (syncytium). In human placenta, a syncytialized trophoblast (syncytiotrophoblast) layer forms the primary interface between maternal and fetal tissue, facilitates nutrient and gas exchange, and produces hormones vital for pregnancy. Syncytiotrophoblast development occurs by differentiation of underlying progenitor cells called cytotrophoblasts, which then fuse into the syncytiotrophoblast layer. Differentiation is associated with chromatin remodeling and specific changes in gene expression mediated, at least in part, by histone acetylation. However, the epigenetic regulation of human cytotrophoblast differentiation and fusion is poorly understood. In this study, we found that human syncytiotrophoblast …

Late Referrals Of Pediatric Patients With Elevated Blood Pressure, Guido Filler, Laura Torres-Canchala

Paediatrics Publications

Editorial Commentary

Comparative Analysis Of American Heart Association And European Society Of Hypertension Ambulatory Blood Pressure Thresholds For Diagnosing Hypertension In Children., Ajaya Sharma, Luis Altamirano-Diaz, Michael Grattan, Guido Filler, Ajay P Sharma

Paediatrics Publications

Introduction: The influence of using 24-hour ambulatory blood pressure (ABP) thresholds recommended by the American Heart Association (AHA) (24-hour mean ABP >95th percentile and ABP load >25%) or the European Society of Hypertension (ESH) (mean 24-hour ABP >95th percentile or >130/80 mm Hg if mean ABP 95th percentile exceeds 130/80 mm Hg) on the diagnosis of pediatric hypertension has been understudied.

Methods: In a cross-sectional, retrospective study of 159 children from a tertiary care outpatient clinic, we classified office blood pressure (OBP) as normotension or hypertension based on the OBP thresholds recommended by the American Academy of Pediatrics (AAP) and …

Seizure Freedom Improves Health-Related Quality Of Life After Epilepsy Surgery In Children., Puneet Jain, Mary Lou Smith, Kathy Speechley, Mark Ferro, Mary Connolly, Rajesh Ramachandrannair, Salah Almubarak, Andrea Andrade, Elysa Widjaja

Paediatrics Publications

AIM: To determine whether epilepsy surgery improved health-related quality of life (HRQoL) and whether seizure freedom after surgery mediated the improvement in HRQoL.

METHOD: This multicenter cohort study compared HRQoL after epilepsy surgery to pharmacological management in children with drug-resistant epilepsy (DRE). HRQoL was measured using the Quality of Life in Childhood Epilepsy (QOLCE) questionnaire at baseline and 1-year follow-up. The mediator between treatment type and HRQoL was seizure freedom.

RESULTS: Two hundred and thirty-seven patients were recruited (surgery group: n=147 [92 males, 45 females]; pharmacological group: n=90 [53 males, 37 females]). Mean age at seizure onset was 6 years …

Standards Of Care For Children In Emergency Departments: Executive Summary., Rodrick K Lim, Baljit Cheema, Catherine Cartman, Ffion Davies, Marianne Gausche-Hill, Felix Hay

Paediatrics Publications

No abstract provided.

Standards Of Care For Children In Emergency Departments: International Federation Of Emergency Medicine Agenda For The Care Of Children., Camilo E Gutierrez, Marianne Gausche-Hill, Rodrick K Lim

Paediatrics Publications

No abstract provided.

Evaluation Of The Quality Of Clinical Data Collection For A Pan-Canadian Cohort Of Children Affected By Inherited Metabolic Diseases: Lessons Learned From The Canadian Inherited Metabolic Diseases Research Network., Kylie Tingley, Monica Lamoureux, Michael Pugliese, Michael T. Geraghty, Jonathan B. Kronick, Beth K Potter, Doug Coyle, Kumanan Wilson, Michael Kowalski, Valerie Austin, Catherine Brunel-Guitton, Daniela Buhas, Alicia K J Chan, Sarah Dyack, Annette Feigenbaum, Alette Giezen, Sharan Goobie, Cheryl R Greenberg, Shailly Jain Ghai, Michal Inbar-Feigenberg, N Karp, Mariya Kozenko, Erica Langley, Matthew Lines, Julian Little, Jennifer Mackenzie, Bruno Maranda, Saadet Mercimek-Andrews, Connie Mohan, Aizeddin Mhanni, Grant Mitchell, John J Mitchell, Laura Nagy, Melanie P. Napier, Amy Pender, Murray Potter, Chitra Prasad, Suzanne Ratko, Ramona Salvarinova, Andreas Schulze, Komudi Siriwardena, Neal Sondheimer, Rebecca Sparkes, Sylvia Stockler-Ipsiroglu, Yannis Trakadis, Lesley Turner, Clara Van Karnebeek, Hilary Vallance, Anthony Vandersteen, Jagdeep Walia, Ashley Wilson, Brenda J. Wilson, Andrea C. Yu, Nataliya Yuskiv, Pranesh Chakraborty

Paediatrics Publications

BACKGROUND: The Canadian Inherited Metabolic Diseases Research Network (CIMDRN) is a pan-Canadian practice-based research network of 14 Hereditary Metabolic Disease Treatment Centres and over 50 investigators. CIMDRN aims to develop evidence to improve health outcomes for children with inherited metabolic diseases (IMD). We describe the development of our clinical data collection platform, discuss our data quality management plan, and present the findings to date from our data quality assessment, highlighting key lessons that can serve as a resource for future clinical research initiatives relating to rare diseases.

METHODS: At participating centres, children born from 2006 to 2015 who were diagnosed …