FDA

Study Funding Source: FDA

PKDnet: ARPKD Learning Network

Autosomal recessive polycystic kidney disease (ARPKD) is a rare disease, affecting ~1,500 children and young adults in the U.S. In severely affected patients, ARPKD can present in the neonatal period with massively enlarged kidneys and pulmonary hypoplasia, but the clinical spectrum of ARPKD is highly variable. We now recognize that just ~50% of patients present […]

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 Center (NESTcc), we aimed to use the amalgamated EHR of a consortium of pediatric hospitals […]

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 not well described. The reported case fatality rate among infants varies across the literature from […]

NEST R2: AventaMed – Typmpanostomy Tube Insertion Treatment Episodes: Practice Variation and Outcomes

This study will use real-world data (RWD) from electronic health records (EHR) and health plan claims to examine the outcomes following the insertion of tympanostomy tubes – the device of interest –among children. Tympanostomy tube insertion (TTI) accounts for nearly 1 in 4 pediatric surgeries in the United States, making it the most common invasive […]

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