Hospital for Sick Children

Toward clinically actionable lupus biomarkers: a roadmap for research, validation, and translation

Abstract Despite advances in lupus biomarker research, clinical translation has remained limited, leading to delayed diagnosis, imprecise disease monitoring, and trial-and-error treatment approaches. This roadmap, developed by the Lupus Research Alliance’s Biomarker Roadmap Commi...

Preterm Risk Factors, Epigenetic Age, and Neurodevelopmental Outcomes

Importance Studies examining biological factors associated with neurodevelopment in the preterm population are limited. It was previously demonstrated that epigenetic age acceleration was associated with better neurodevelopmental outcomes among children born preterm. It is unclea...

The impact of non-medical determinants of health on patient-reported outcomes in pediatric lupus: A CARRA registry study

Objectives Social determinants of health (SDOH) and race are associated with disease activity, including lower achievement of the low lupus disease activity state (LLDAS), but it is unknown whether these factors are associated with patient-reported outcomes (PROs). Among a cohort...

Consented Universal Newborn Screening for Congenital Cytomegalovirus Infection Using Dried Blood Spot and Dried Saliva Spot Specimens: Implementation and Relative Programmatic Accuracy

The purpose of this research was to evaluate the implementation and relative diagnostic accuracy of dried blood spot (DBS) and dried saliva spot (DSS)-based newborn screening for congenital cytomegalovirus infection (cCMV). This prospective paired-sample pilot studied consecutive...

Interim Update on Reactive Infectious Mucocutaneous Eruption ( RIME )

ABSTRACT Reactive infectious mucocutaneous eruption (RIME), a term describing blistering skin and mucosal lesions, is a term coined by a group of Pediatric Dermatology Research Alliance (PeDRA) researchers and first published in 2021. Since that time, the literature has grown wit...

Managing growth and puberty concerns in individuals with Duchenne muscular dystrophy: Updated guidance and future directions

Long-term glucocorticoid therapy is standard of care for individuals with Duchenne muscular dystrophy (DMD) but often results in impaired growth and delayed or incomplete puberty, impacting their physical and psychosocial health. Although DMD endocrine care has evolved, leading t...

Identifying Cardiac Autoantibody Biomarkers that Predict the Severity of Chagas Cardiomyopathy

Chagas cardiomyopathy (ChCM) affects up to 30% of patients with chronic Chagas disease and lacks reliable biomarkers for severity and conduction abnormalities. We profiled cardiac autoantibodies using 2-dimensional gel electrophoresis and mass spectrometry, followed by enzyme-lin...

Shared patterns of human milk composition link mammary gland function to infant growth

The mammary gland produces nutrient-rich human milk (HM), yet how mammary functional state shapes HM composition and infant outcomes remains poorly understood. We used HM multi-omics - metabolomics, proteomics, micronutrients, macronutrients, and HM oligosaccharides - across 1,54...

Improving Discharge Instructions: Systematic Review of Language and Health Literacy Interventions.

CONTEXT: Effective pediatric inpatient discharge relies on clear, responsive communication to support safe transitions from hospital to home. Families who use a language other than English and those with lower health literacy experience disproportionate barriers during discharge,...

Rapid Whole Genome Sequencing Enhances Pediatric Cancer Diagnosis

Abstract Whole-genome sequencing (WGS) has been shown to improve diagnosis beyond standard-of-care genetic tests and guide precision treatment for childhood cancer. While WGS is now offered for children with suspected cancer in England, clinical workflows typically return results...

A global study of novel agents in paediatric and adolescent relapsed and refractory B-cell non-Hodgkin lymphoma (Glo-BNHL): protocol for an adaptive international platform clinical trial

The second Paediatric Strategy Forum of the international multi-stakeholder organisation, ACCELERATE (Nov-2017), concluded that an international approach to conducting clinical trials in relapsed and refractory paediatric B-cell non-Hodgkin lymphoma (r/r B-NHL) was critical and t...

Engineering neurovascular thrombosis: Light-based bioprinting for patient-specific modeling and women’s cerebrovascular health

Cerebral venous sinus thrombosis (CVST) and carotid web (CW) are distinct cerebrovascular disorders where vascular geometry, disturbed hemodynamics, and endothelial activation interact to drive thrombosis. Both conditions disproportionately affect women, yet remain understudied b...

Exclusive and Any Breastfeeding, Infant Formula Feeding, and Childhood Growth

OBJECTIVES: To understand the association between exclusive breastfeeding, any breastfeeding, and infant formula feeding and childhood growth. METHODS: A longitudinal cohort study was conducted among children from birth up to 10 years in Toronto, Canada. The primary exposure was...

The Childhood Liver Disease Research Network's prospective characterization of pediatric primary sclerosing cholangitis.

BACKGROUND: Primary sclerosing cholangitis (PSC) is a rare biliary fibrosing disease that leads to significant morbidity and the need for liver transplantation. The Childhood Liver Disease Research Network is conducting a prospective, observational longitudinal study to define th...

Consensus recommendations for neurofibromatosis type 1 management in Asia Pacific

Neurofibromatosis type 1 (NF1) is a complex multisystem disorder requiring coordinated multidisciplinary care. This study aimed to develop consensus recommendations for NF1 management in the Asia-Pacific region. A modified Delphi process involving two survey rounds and a virtual...

Maternal and Fetal Outcomes in Mice Following Exposure to Dolutegravir Alone or in Combination with Lamivudine or Tenofovir Disoproxil Fumarate/Emtricitabine

Abstract Background and Aims Dolutegravir (DTG)-based regimens are widely used in HIV treatment, including during pregnancy, but the contribution of the nucleoside reverse transcriptase inhibitor (NRTI) backbone on maternal and foetal outcomes remains unclear. We examined this in...

Efficacy and Safety of Risankizumab in Paediatric Patients With Psoriasis in the OptIMMize-1 Phase III Study.

BACKGROUND: Treatment options for paediatric patients with psoriasis are limited, and additional, more advanced therapies are needed. Risankizumab is an interleukin-23 inhibitor approved to treat moderate-to-severe psoriasis in adults. OBJECTIVES: To evaluate efficacy and safety...

Co-designing a financial navigator program in the neonatal neurodevelopmental follow-up clinic: a formative mixed-methods study

Families of infants requiring neonatal intensive care often experience substantial financial hardship due to income loss and out-of-pocket costs during hospitalization and after discharge. Hospitals currently lack dedicated, structured support to help families navigate these fina...

Adherence to treat-to-target recommendations in juvenile idiopathic arthritis in clinical practice

Abstract Objective To assess adherence to Treat-to-Target (T2T) recommendations for juvenile idiopathic arthritis (JIA) in clinical practice. Methods A nested cohort study in the multicenter international prospective UCAN JIA cohort was performed. Patient characteristics, clinica...

A phase 2 pharmacokinetic and pharmacodynamic dose-finding study of oral NEPA for chemotherapy-induced nausea and vomiting in pediatric patients with cancer

Chemotherapy-induced nausea and vomiting (CINV) remains a significant challenge in pediatric oncology, with limited data guiding optimal antiemetic regimens. This phase 2 study evaluated pharmacokinetics (PK), pharmacodynamics, efficacy, and safety of oral netupitant combined wit...