Developing a class of MSMs to estimate the effect of time-varying treatments on different types of recurrent time-to-event outcomes
Pragmatic trials and comparative effectiveness research often require causal inference methods to establish cause-effect relationships between interventions and outcomes. With treatments that can change over time, it is crucial to account for time-dependent confounders to accurately estimate treatment effects. Statistical…
Natural History of Enteroviral Sepsis Using Real World Data
Enteroviral (EV) sepsis with hepatic necrosis and coagulopathy (HNC) or myocarditis is a serious and life-threatening infection. This serious infection primarily affects neonates and is sometimes referred to as neonatal enterovirus sepsis syndrome (NES). The current incidence of NES is…
Health Services and Clinical Characteristics that Precede Diagnosis of Primary Hyperoxaluria Type 1
Primary hyperoxaluria (PH) encompasses 3 genetically distinct, autosomal-recessive, inborn errors of glyoxylate metabolism characterized by the over-production of oxalate, a highly insoluble metabolic endproduct that is eliminated mainly by the kidney. All 3 forms of PH exhibit some extent of…
Hemolytic Disease of the Fetus and Newborn (HDFN) Epidemiology Study
The Hemolytic Disease of the Fetus and Newborn (HDFN) Epidemiology Study aims to analyze the epidemiology and clinical outcomes of HDFN using data from the PEDSnet database. HDFN, caused by maternal IgG alloantibodies that destroy fetal and neonatal erythrocytes, remains…
Clinical Characteristics of Adolescents Hospitalized for Eating Disorders 2010-2020
This study will gather more data and characteristics of pediatric patients with eating disorders. The intention is to use PEDSnet data to gather demographic (age, sex, insurance status) as well as clinical (cardiac complications such as arrhythmias, bradycardia) from patients…
Implementing Minoritized Community Engagement with Pediatric Dermatology
Dermatology care is nearly non-existent for American Indian and Alaska Native (AIAN) youth. The Indian Health Service (IHS) currently employs a single dermatologist for more than 2 million patients, which suggests that AIAN youth face substantial barriers to accessing dermatology….
A Learning Health Systems Approach to Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis in Children
Anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV) is an understudied, chronic inflammatory disease in children for which only half of children reach remission by 6-months and approximately 7% do not survive past young adulthood. Due to its rarity, there are no large…
Barriers in the access to expensive genetic therapies for spinal muscular atrophy
Although the inherited neuromuscular disease spinal muscular atrophy is considered a rare disease, it has been on the forefront of genetic therapies research, with three therapies receiving FDA approval since 2016. Spinal muscular atrophy is an autosomal recessive loss of…
P50 Projects – CHOP Pediatric Center for Excellence in Nephrology (PCEN)
With this renewal, the Pediatric Center of Excellence in Nephrology at the Children’s Hospital of Philadelphia (CHOP PCEN) will continue to facilitate extensive collaborative research around the causes, diagnoses, and treatment of childhood kidney diseases. Increasing efficiency and effectiveness, the…
Computational Phenotype of Acute and Long-term Kidney Disease in Children with Septic Shock
This study aims to expand understanding of how crystalloid fluid impacts sepsis survivors’ lives beyond 30 days. Aim 1: Determine the most effective crystalloid fluid choice to minimize sepsis-induced AKI and CKD in PRoMPT BOLUS trial participants Aim 2: Measure…
NEST R1: Developing Capacity for Post Marketing Surveillance of Craniomaxillofacial Distractors
The National Evaluation System for health Technology Coordinating Center (NESTcc) is tasked to be a catalyst for the timely, reliable, and cost-effective development of real-world evidence (RWE) to enhance regulatory and clinical decision-making for medical devices. Real world data (RWD)…
Growth for Robust Ongoing Health and Wellness (GROWTH)
This study aims to improve the nutritional outcomes of infants with orofacial clefts, who face higher risks of malnutrition and growth faltering. A review of 833 infants treated at Seattle Children’s Hospital (2010-2020) showed that underweight rates were significantly higher…
Semantic Data Quality Standards for Multi-Center Clinical Research Studies and Networks
To address missed gaps in data quality, the purpose of this study is to create actionable guidelines for testing and reporting data quality, building on current practice and expanding it to address fitness for use and alignment with clinical meaning….
PEDSnet: Advancing Pediatric Patient-Centered Outcomes Research in PCORnet
PEDSnet’s guiding purpose is to improve child health through knowledge production that is efficient (produced faster at less cost) and meaningful (useful to patients, caregivers, clinicians, and health system leaders). Led by the Children’s Hospital of Philadelphia (CHOP), PEDSnet is…
Growth for Robust Ongoing Health and Wellness (GROWTH)
This study aims to improve the nutritional outcomes of infants with orofacial clefts, who face higher risks of malnutrition and growth faltering. A review of 833 infants treated at Seattle Children’s Hospital (2010-2020) showed that underweight rates were significantly higher…
Detection of and Characteristics of Disease Progression in Children with CKD
The purpose of this study was to inform the design of a possible clinical trial in children with chronic kidney disease. Our goals were to use the PEDSnet EHR database to: 1. Identify a population of children ages 6 months…
Determine the rate of clotting in central venous catheters (CVCs)
The purpose of this study is to assess the feasibility of conducting a study of central venous catheter-associated thrombosis in children using PEDSnet data. Aim 1. Identify patients with evidence of CVC placement based on procedure or device data. –…
Developing toolkit to control for missing data
Distributed health data networks (DHDNs) that leverage electronic health records (EHRs) (e.g., eMerge, pSCANNER, PEDSnet) have gained substantial interests in recent years, as they a) eliminate the need to create, maintain, and secure access to central data repositories, b) minimize…
NEST R2: Characterization and Utilization of Therapeutic Cardiac Devices in Children with Congenital Heart Disease
Utilizing the Electronic Health Record (EHR) to capture the use of implantable devices during congenital cardiac catheterization procedures across multiple institutions has not been previously possible. In this Test Case sponsored by the National Evaluation System for Health Technology Coordinating…
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