Corporate Symposia Schedule
Wednesday, May 2
7:30 pm – 9 pm
Have You Encountered Plasminogen Deficiency In Your Practice? An Overview On Management & New Therapeutic Options.
Congenital Plasminogen Deficiency is a rare genetic disease that can have devastating effects on multiple organ systems throughout the body. Congenital Plasminogen Deficiency predominantly affects infants and children, with the most severe manifestations being reported in the pediatric population. Fibrinous lesions accumulate on mucosal membrane surfaces, posing significant risk and organ-compromising effects. This symposium will cover historical management approaches for PLGD as well as clinical trial information and real-world evidence involving a new, systemic plasminogen replacement therapy, currently under FDA review.
- Gain greater understanding and awareness on Congenital Plasminogen Deficiency; Disease state knowledge.
- Develop an appreciation for the inherent challenges associated with historical management approaches.
- Become familiar with clinical trial and real world evidence for a new systemic therapy, currently under FDA review.
Supported by: Prometic Biotherapeutics, Inc.
Thursday, May 3
The presentation will feature a case of a pediatric patient who develops VOD post HSCT. The case study will take the audience through the risk factors, symptom presentation, differential diagnosis, and progression of the disease. The presentation will then provide education about recognizing and diagnosing VOD and clinical information about treatment with Defitelio for patients with VOD with renal or pulmonary dysfunction post HSCT.
Supported by: Jazz Pharmaceuticals
Gaucher disease is a rare, heritable lysosomal storage disorder. Approximately half of affected individuals experience symptoms in childhood but low recognition of this disorder may lead to significant delays in diagnosis and management. This will be a case-based symposium highlighting the pediatric presentation of Gaucher disease.
Supported by: Sanofi Genzyme
- Importance and Goals of Asparaginase Treatment in ALL
- Clinical Impact of Asparaginase Hypersensitivity
- Overview of hypersensitivity reactions (HSR) in the context of the immune cascade
- Considerations when Differentiating Infusion Reactions from HSRs
- Therapy Considerations after HSRs
- Case Builder Activity
Supported by: Jazz Pharmaceuticals
Friday, May 4
Fernando Acosta, Jr., MD and Marcela D. Torres, MD
The incidence of pediatric stroke is steadily increasing, with most recent rates up to 13 per 100,000 children per year - similar to that of childhood brain tumors. Stroke is one of the leading causes of death in children, and 20 percent of children who have had a stroke will have a recurrence.
The Cook Children's Comprehensive Stroke and Thrombosis Program, one of 16 such centers in the U.S., is led by Fernando Acosta Jr., M.D. (Neurology) and Marcela Torres, M.D. (Hematology) with additional support from neurosurgery, neuro-radiology, neuropsychology, a dedicated nurse practitioner, nurse, social worker and Child Life specialist. The team is committed to the early recognition, treatment and prevention of pediatric stroke through research, innovation and education. As members of the International Pediatric Stroke Society, the Cook Children's team works with a network of other pediatric hospitals to improve stroke care worldwide.
Cook Children's has recently employed the use of novel imaging techniques to guide therapy and interventions aimed at reducing the risk of future strokes. Intracranial vessel wall MRI (case study, figure 1) is an innovative diagnostic imaging technique that allows evaluation of intracranial vascular pathology by directly visualizing arterial vessel wall abnormalities with MR sequences. With this technique, it is possible to identify distinct pathologic findings within the intracranial vessel wall itself, ranging from arterial dissection to vasculitis. Understanding the pathology then allows the team to select therapy specific to the etiology, which is expected to improve outcome.
The team also utilizes perfusion weighted imaging, which consists of a variety of MRI techniques that give insight into the perfusion of the brain tissue by the blood. This imaging technique provides critical information regarding the mean transit time (time it takes blood to reach specific areas of the brain), cerebral blood volume (amount of blood reaching specific areas of the brain) and cerebral blood flow (constant amount of blood reaching specific areas of the brain). These factors can be used to predict areas in the brain that are at risk for injury or subsequent stroke. Through these advanced imaging techniques, the Cook Children's Comprehensive Stroke and Thrombosis Program aims to continue improving the recognition, treatment and outcome of pediatric stroke.Supported by: Cook Children’s Health Care System