Optimization of Existing Therapies for Sickle Cell Disease
How can the safety, dosing and benefits of existing therapies for sickle cell disease such as hydroxyurea, be optimized in order to increase its efficacy and improve patient adherence?
How can the safety, dosing and benefits of existing therapies for sickle cell disease such as hydroxyurea, be optimized in order to increase its efficacy and improve patient adherence?
What are the optimal strategies for transfusion of blood products in pediatric and neonatal population?
For patients in the ICU with coagulopathy and associated World Health Organization (WHO) grade 3 or 4 bleeding, which hemostatic therapy -- plasma versus 4-factor prothrombin complex concentrates -- is preferred?
What are the optimal RBC transfusion thresholds for adult and pediatric cancer patients undergoing chemotherapy regimens that may improve functional status and quality of life?
Goal 3: Advance Translational Research
There is a need to develop “designer platelets” and “designer red cells,” as well as facilitate large-scale production of these products for therapeutic and diagnostic use.
What are optimal transfusion practices for acute coronary syndrome?
At what INR threshold does prophylactic plasma transfusion, in non-bleeding critically ill patients who are planned to undergo an invasive procedure, prevent bleeding complications and improve patient outcomes?
Does the use of viscoelastic testing to guide plasma transfusion improve outcomes when compared to traditional coagulation testing?
For surgical patients meeting criteria for the critical RBC administration threshold (CAT) due to an associated coagulation disorder, which hemostatic resuscitation strategy (goal-directed versus ratio-based) is superior?
Do different hemoglobin transfusion thresholds alter outcomes in children with ARDS? What is the optimal *minimum* transfusion threshold for children with ARDS? What patient-centered outcomes can be affected by transfusion strategies: ventilator free days, time to organ function recovery, duration of intensive care stay, survival?
Are there specific conditions where a liberal transfusion strategy results in lower 30-day mortality as compared to a restrictive transfusion strategy?