365 results match your criteria: "Hospital for Sick Children and The University of Toronto[Affiliation]"
Crit Care Explor
January 2025
Division of Cardiovascular Critical Care Medicine, Department of Cardiology, Boston Children's Hospital, Boston, MA.
Background: Accurate assessment of oxygen delivery relative to oxygen demand is crucial in the care of a critically ill patient. The central venous oxygen saturation (Svo) enables an estimate of cardiac output yet obtaining these clinical data requires invasive procedures and repeated blood sampling. Interpretation remains subjective and vulnerable to error.
View Article and Find Full Text PDFLiver Int
February 2025
Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada.
Background: Alagille syndrome (ALGS) is a multisystem cholestatic disorder. Maralixibat is approved for the treatment of cholestatic pruritus in ALGS with limited data in adults.
Methods: Participants were included if they received ≥ 2 doses of maralixibat at age ≥ 16 years in one of the three previously published maralixibat ALGS clinical trials.
NPJ Genom Med
January 2025
Division of Immunology and Allergy, Department of Paediatrics, The Hospital for Sick Children and the University of Toronto, Toronto, ON, Canada.
Maturation of αβ lineage T cells in the thymus relies on the formation and cell surface expression of a pre-T cell receptor (TCR) complex, composed of TCRβ chain and pre-TCRα (pTCRα) chain heterodimers, giving rise to a diverse T cell repertoire. Genetic aberrations in key molecules involved in T cell development lead to profound T cell immunodeficiency. Definitive genetic diagnosis guides treatment choices and counseling.
View Article and Find Full Text PDFEarly Hum Dev
January 2025
Department of Pediatrics, Division of Neonatology, The Hospital for Sick Children and the University of Toronto, Ontario, Canada. Electronic address:
Objectives: To build an early, prognostic model for adverse outcome in infants with hypoxic ischemic encephalopathy (HIE) receiving therapeutic hypothermia (TH) based on brain magnetic resonance images (MRI), electrophysiological tests and clinical assessments were performed during the first 5 days of life.
Methods: Retrospective study of 182 neonates with HIE and managed with TH. The predominant pattern of HIE brain injury on MRI performed following cooling was scored by neuroradiologists.
Arthritis Care Res (Hoboken)
December 2024
Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
CJC Pediatr Congenit Heart Dis
August 2024
Safra Children's Hospital, Sheba Medical Center, and the Tel Aviv University, Tel Hashomer, Israel.
Background: Radiation reduction is an integral component in the management of a paediatric cardiac catheterization laboratory. Simple and easily implementable protocol changes and technical upgrades have been shown to significantly reduce radiation exposure.
Methods: Radiation exposures (2020-2022) at Safra Children's Hospital, Sheba Medical Center, Israel (unit A: n = 672) were retrospectively reviewed, including dose area product (DAP) (μGy m), DAP/kg, Air Kerma (mGy), and fluoroscopy time (minutes) for 16 procedural types.
J Pediatr
January 2025
Department of Pediatrics, The Hospital for Sick Children and the University of Toronto, Toronto, ON, Canada; Department of Pediatrics, BC Children's Hospital Research Institute and University of British Columbia, Vancouver, Canada. Electronic address:
Objective: To examine sex differences in neurodevelopmental outcomes and brain development from early life to 8 years in males and females born preterm.
Study Design: This was a prospective cohort study of infants born very preterm (24-32 weeks of gestation) and followed to 8 years with standardized measures of neurodevelopment. Brain magnetic resonance imaging scans were performed soon after birth, term-equivalent age, and 8 years.
J Cardiothorac Vasc Anesth
October 2024
Department of Anesthesiology and Pain Medicine, The Hospital for Sick Children and The University of Toronto, Ontario, Canada. Electronic address:
Objectives: Pulmonary arterial hypertension (PAH) is a devastating complication of pediatric congenital heart disease (CHD). A recent study has identified the protein high mobility group box-1 (HMGB1) as a diagnostic tool in adults with CHD-associated PAH. HMGB1 levels in adults with CHD-associated PAH correlated with mean pulmonary artery pressure and pulmonary vascular resistance, and HGMB1 levels fell in response to sildenafil therapy.
View Article and Find Full Text PDFHepatology
July 2024
Division of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, Children's Hospital of Philadelphia, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA.
Historically, the therapeutic options available to hepatologists managing cholestasis have been limited. Apart from bile acid--binding resins and the choleretic ursodeoxycholic acid, the medical management of cholestasis in children has been predominately focused on managing the complications of cholestasis, namely pruritus, malnutrition, fat-soluble vitamin deficiencies, and portal hypertension. As such, invasive surgical procedures such as biliary diversion and liver transplantation may become the only options for progressive and unremitting cases of cholestasis.
View Article and Find Full Text PDFJ Allergy Clin Immunol Glob
August 2024
Division of Immunology and Allergy, Department of Paediatrics, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada.
Background: Forkhead box protein N1 (FOXN1) transcription factor plays an essential role in the development of thymic epithelial cells, required for T-cell differentiation, maturation, and function. Biallelic pathogenic variants in cause severe combined immunodeficiency (SCID). More recently, heterozygous variants in identified by restricted gene panels, were also implicated with causing a less severe and variable immunodeficiency.
View Article and Find Full Text PDFLiver Int
August 2024
Division of Gastroenterology Hepatology and Nutrition, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada.
Background And Aims: Autoimmune hepatitis (AIH) in children presenting in acute liver failure (ALF) can be fatal and often requires liver transplantation (LTx). This individual patient data meta-analysis (IPD) aims to examine management and outcomes of this population, given the lack of large cohort studies on paediatric AIH first presenting as ALF (AIH-ALF).
Methods: A systematic review was conducted in accordance with the Preferred Reporting Items for Systematic Review and Meta-Analyses of IPD statement using PubMed and Excerpta Medica dataBASE, and included English studies published between 2000 and 2020.
Cardiol Young
May 2024
Congenital Heart Center, Division of Cardiovascular Surgery, Departments of Surgery and Pediatrics, University of Florida, Gainesville, Florida, United States of America.
In recent issues of the and the , Holzer and colleagues presented an Expert Consensus Document titled: "" This Expert Consensus Document is a massively important contribution to the community of paediatric and congenital cardiac care. This document was developed as an Expert Consensus Document by the Pediatric and Congenital Interventional Cardiovascular Society, the Association for European Paediatric and Congenital Cardiology, the Asia-Pacific Pediatric Cardiac Society, the Cardiac Society of Australia and New Zealand, the Society for Cardiovascular Angiography and Interventions, and the Latin American Society of Interventional Cardiology, as well as the Congenital Cardiac Anesthesia Society and the American Association of Physicists in Medicine.As perfectly stated in the Preamble of this Expert Consensus Document, "This expert consensus document is intended to inform practitioners, payors, hospital administrators and other parties as to the opinion of the aforementioned societies about best practices for cardiac catheterisation and transcatheter management of paediatric and adult patients with congenital heart disease, with added accommodations for resource-limited environments.
View Article and Find Full Text PDFN Engl J Med
May 2024
From Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial (H.F.), Nashville, and Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis (A.S.) - both in Tennessee; IRCCS, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome (F.L.); the Department of Pediatrics, Columbia University Irving Medical Center, New York-Presbyterian-Morgan Stanley Children's Hospital (M.B.), and the Department of Medicine, Division of Hematology-Oncology, Columbia University (M. Mapara) - both in New York; Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children's Hospital, San Antonio, TX (L.M., M.J.E.); the Hospital for Sick Children and the University of Toronto, Toronto (D.W.); Ann and Robert H. Lurie Children's Hospital of Chicago (R.I.L.) and the University of Illinois at Chicago (D.R.) - both in Chicago; Royal London Hospital, Barts Health NHS Trust, London (P.T.); Stanford University, Palo Alto (A.J.S.), and University of California San Francisco Benioff Children's Hospital, Oakland (M.C.W.) - both in California; the Biotherapy Department and Biotherapy Clinical Investigation Center (M.C.), Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Université Paris-Cité (M. de Montalembert), Paris; the University of Regensburg, Regensburg (S.C.), the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Duesseldorf (R.M.), and Gemeinschaftsklinikum Mittelrhein, Koblenz (S.L.) - all in Germany; Hôpital Universitaire des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels (L.D.); Boston University Chobanian and Avedisian School of Medicine (M.H.S.) and Vertex Pharmaceuticals (S.I., L.B., C.S., W.Z., F.X., W.E.H.), Boston, and CRISPR Therapeutics, Cambridge (P.K.M.) - all in Massachusetts; and the Division of Oncology, Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia (S.A.G.).
Neurology
April 2024
From Pediatrics (T.S., S.U., R.E.G., S.P.M.), BC Children's Hospital Research Institute and the University of British Columbia, Vancouver; Pediatrics (T.S., T.G., V.C., L.G.L., S.P.M.), The Hospital for Sick Children and University of Toronto; Diagnostic Imaging (H.B.), The Hospital for Sick Children and the University of Toronto, Ontario; Pediatrics (A.S., R.E.G.), BC Women's Hospital and the University of British Columbia, Vancouver; and Pediatrics (E.N.K.), Mount Sinai Hospital, The Hospital for Sick Children and the University of Toronto, Ontario, Canada.
Background And Objectives: We examined associations of white matter injury (WMI) and periventricular hemorrhagic infarction (PVHI) volume and location with 18-month neurodevelopment in very preterm infants.
Methods: A total of 254 infants born <32 weeks' gestational age were prospectively recruited across 3 tertiary neonatal intensive care units (NICUs). Infants underwent early-life (median 33.
Hepatol Commun
March 2024
Development & Stem Cell Biology program, Peter Gilligan Centre for Research and Learning, The Hospital for Sick Children, Toronto, Ontario, Canada.
Background: Ductular reactivity is central to the pathophysiology of cholangiopathies. Mechanisms underlying the reactive phenotype activation by exogenous inflammatory mediators and bile acids are poorly understood.
Methods: Using human extrahepatic cholangiocyte organoids (ECOs) we developed an injury model emulating the cholestatic microenvironment with exposure to inflammatory mediators and various pathogenic bile acids.
Hepatology
June 2024
Department of Paediatrics, Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada.
Background And Aims: Alagille syndrome (ALGS) is characterized by chronic cholestasis with associated pruritus and extrahepatic anomalies. Maralixibat, an ileal bile acid transporter inhibitor, is an approved pharmacologic therapy for cholestatic pruritus in ALGS. Since long-term placebo-controlled studies are not feasible or ethical in children with rare diseases, a novel approach was taken comparing 6-year outcomes from maralixibat trials with an aligned and harmonized natural history cohort from the G lobal AL agille A lliance (GALA) study.
View Article and Find Full Text PDFCurr Opin Pediatr
April 2024
Division of Neonatology, The Hospital for Sick Children and the University of Toronto, Toronto, Canada.
Purpose Of Review: Neurological problems are common in infants admitted to the neonatal intensive care unit (NICU). Various neuroimaging modalities are available for neonatal brain imaging and are selected based on presenting problem, timing and patient stability.
Recent Findings: Neuroimaging findings, taken together with clinical factors and serial neurological examination can be used to predict future neurodevelopmental outcomes.
JHEP Rep
August 2023
Albireo Pharma, Inc., Boston, MA, USA.
Background & Aims: PEDFIC 2, an ongoing, open-label, 72-week study, evaluates odevixibat, an ileal bile acid transporter inhibitor, in patients with progressive familial intrahepatic cholestasis.
Methods: PEDFIC 2 enrolled and dosed 69 patients across two cohorts; all received odevixibat 120 μg/kg per day. Cohort 1 comprised children from PEDFIC 1, and cohort 2 comprised new patients (any age).
J Rheumatol
October 2023
J. Scheck, BS, N. Rosenwasser, MD, G. Casselman, BS, A. Liau, BS, Y. Shao, BS, C. Yang, BS, Y. Zhao, MD, PhD, Division of Pediatric Rheumatology, Seattle Children's Hospital, University of Washington, Seattle, Washington, USA;
Objective: Prospective comparative effectiveness research (CER) in chronic nonbacterial osteomyelitis (CNO) is lacking. Our objectives were to (1) determine the use and safety of each consensus treatment plan (CTP) regimen for CNO, (2) assess the feasibility of using the Chronic Nonbacterial Osteomyelitis International Registry (CHOIR) data for CER, and (3) develop and validate a CNO clinical disease activity score (CDAS) using CHOIR.
Methods: Consenting children or young adults with CNO were enrolled into CHOIR.
Arthritis Care Res (Hoboken)
December 2023
Seattle Children's Hospital and the University of Washington, Seattle.
Objective: To describe the selection, development, and implementation of quality measures (QMs) for juvenile idiopathic arthritis (JIA) by the Pediatric Rheumatology Care and Outcomes Improvement Network (PR-COIN), a multihospital learning health network using quality improvement methods and leveraging QMs to drive improved outcomes across a JIA population since 2011.
Methods: An American College of Rheumatology-endorsed multistakeholder process previously selected initial process QMs. Clinicians in PR-COIN and parents of children with JIA collaboratively selected outcome QMs.
Hepatology
December 2023
Department of Inflammation Biology, Institute of Liver Studies, King's College London, London, UK.
Background And Aims: Refractory pruritus and other complications of cholestasis are indications for liver transplantation (LT) in patients with Alagille syndrome (ALGS). We evaluated predictors of event-free survival and transplant-free survival in patients with ALGS treated with maralixibat (MRX), an ileal bile acid transporter inhibitor.
Approach And Results: We assessed patients with ALGS from 3 clinical trials of MRX with up to 6 years of follow-up.
Eye (Lond)
December 2023
Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada.
Purpose: To characterize the retinal phenotype in RNU4ATAC-associated Roifman syndrome.
Methods: Ten patients (including 8 males) with molecularly confirmed Roifman syndrome underwent detailed ophthalmologic evaluation including fundus imaging, fundus autofluorescence (FAF) imaging, spectral-domain optical coherence tomography (SD-OCT), and electroretinography (ERG). Six patients had follow-up eye exams.
J Dent Child (Chic)
September 2022
Dr. Stallaert is a staff pediatric dentist and a clinical instructor, Division of Pediatric Dentistry, The Hospital for Sick Children and the University of Toronto, Toronto, Canada;, Email:
Genet Med
August 2023
Division of Genetics, Children's Hospital of Philadelphia, Philadelphia, PA; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA. Electronic address:
Purpose: Bone morphogenic proteins (BMPs) regulate gene expression that is related to many critical developmental processes, including osteogenesis for which they are named. In addition, BMP2 is widely expressed in cells of mesenchymal origin, including bone, cartilage, skeletal and cardiac muscle, and adipose tissue. It also participates in neurodevelopment by inducing differentiation of neural stem cells.
View Article and Find Full Text PDFNeurology
May 2023
From the Department of Pediatrics (H.M.A.), King Abdullah bin Abdulaziz University Hospital, Princess Nourah Bint Abdulrahman University, Riyadh, Saudi Arabia; Department of Pediatrics (E.F.P.), Centre Hospitalier Universitaire Sainte-Justine and the University of Montreal, Quebec; Neurosciences and Mental Health Program (M.-A.T., J.P.V., E.W., C.D.H., E.W.Y.T.), Hospital for Sick Children Research Institute; Department of Paediatrics (C.I.S.C., D.K., E.W., L.L., C.D.H., E.W.Y.T.), Department of Radiology (E.W.), and Department of Psychology (E.M.), The Hospital for Sick Children and the University of Toronto, Ontario, Canada; and Epilepsia Helsinki (P.N.), Department of Clinical Neurophysiology, HUS Diagnostic Center, University of Helsinki and Helsinki University Hospital, Finland.
Background And Objectives: Seizures are common during neonatal encephalopathy (NE), but the contribution of seizure burden (SB) to outcomes remains controversial. This study aims to examine the relationship between electrographic SB and neurologic outcomes after NE.
Methods: This prospective cohort study recruited newborns ≥36 weeks postmenstrual age around 6 hours of life between August 2014 and November 2019 from a neonatal intensive care unit (NICU).