Health Impact:
SCD is an orphan disease currently lacking treatment options for acute pain beyond opioid analgesics and supportive care. This proposal may change clinical practice in that Arginine may decrease pain in children presenting to the ED with SCD pain crisis.
Study Summary:
This is a pivotal phase 3 clinical trial using arginine as a novel treatment of pain in children with SCD. Pain in sickle cell disease (SCD) is the leading cause of hospitalizations and ED visits for children with SCD. Arginine has properties that may improve blood flow during these acute occlusion of blood vessels.
Health Impact:
To provide evidence-based guidance on the identification of PE in children to promote timely diagnosis and treatment, while excluding unnecessary radiation testing.
Study Summary:
A prospective, observational, cohort study of children ages 4 to 17 years old who have sufficiently high probability of pulmonary embolism (PE). The goal is to measure the diagnostic accuracy of a prediction rule for exclusion of PE (the PERC-Peds rule) and the D-dimer (a lab test used for diagnostic purposes). No study has been performed to prospectively record the presenting complaints, signs, symptoms and comorbidities of children who raised the suspicion of pulmonary embolism (PE) in children. Clinicians must extrapolate what is known about PE in adults to children potentially delaying diagnosis and/or causing unnecessary radiologic testing.
Health Impact:
This research will optimize clinical decision making and use of emergent neuroimaging when evaluating children with headaches, leading to the safe reduction of unnecessary neuroimaging and accurate stratification of children at greater risk of having an emergent intracranial abnormality.
Study Summary:
This is a prospective observational cohort study of children ages 2 to 17 years old presenting to emergency departments with a headache. The primary aim of the study is to derive and internally validate a risk stratification model that identifies the specific risk of emergent intracranial abnormalities in these children based on clinically sensible and reliable variables. We will also determine whether the prevalence of emergent intracranial abnormalities and association between risk factors and emergent intracranial abnormalities differ based on age.
