Advances in the care of pediatric patients with sickle cell disease ( SCD) have resulted in improved survival to adulthood.However, adulthood is marked by rapid disease progression, impaired quality of life and premature mortality. Hematopoietic cell transplantation(HCT) from matched sibling donor has curative potential, but has been offered mainly to children. Refinements in the conditioning regimen, supportive care,... more »

Can better predictors of disease severity such as specific biomarkers and/or genetic polymorphisms be identified so as to help understand the course and progression of sickle cell disease in various patients?

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?

Most of the patients suffering from non-malignant hematologic conditions are cured of the original disease with Hematopoitec Stem Cell Transplant (HSCT) but still their survival is less compared to age matched general population, and additionally they suffer from unique complications of HSCT culminating into a variety of late physical, psychologic, financial, and social complications (“late effects”). Considerable improvements... more »

Despite advances in care, patients with sickle cell disease have significant morbidity and mortality. One challenge is the optimal use of simple vs exchange transfusion vs no transfusion when managing these patients. Simple transfusions lead to iron overload while exchange transfusions may expose patients to increase numbers of red blood cell units. The mechanism of benefit from transfusion (oxygen delivery vs marrow... more »

There is a need for more enhanced pain research in order to help improve sickle cell disease patient outcomes and quality of life.

Challenges that need to be overcome in blood transfusion, especially in SCD, include:
a. Adopting molecular genotyping as the standard in blood transfusion therapy.
b. Advancing new generation, anti-oxidant hemoglobin-based oxygen carriers (HBOCs) for use in emergencies such as trauma, stroke, acute hemolysis, and in transfusion in SCD and related disorders. In SCD, HBOCs have the capacity to not only serve as substitutes... more »

Newer therapies using gene correction, rather than gene addition, are needed for sickle cell disease. Even with this potential advantage, there needs to be a way to safely deliver gene corrected HSC to the sickle cell patient. Chemotherapy is poorly tolerated, and often is the reason patients do not choose the BMT option. What is the status of other less toxic non myeloablative approaches, and how can they best be... more »

There is a growing desire for the development of alternative treatments and natural therapies for the treatment of sickle cell disease.

Are neurocognitive developmental delays significantly present in children and adolescents living with sickle cell disease? What effect do these delays have on the overall morbidity associated with sickle cell disease?

What markers in sickle cell disease can predict hypoxia after acute chest syndrome or pneumonia?

Does the aggressive treatment of asthma prevent the developement of acute chest syndrome (ACS) and vaso-occlusive syndrome (VOS) in patients with sickle cell disease (SCD)?

Sickle cell disease is now understood as a disease of inflammation in addition to abnormal red blood cells. It is likely persons with sickle cell trait are also negatively affected by the damage caused by inflammation. There is a significant racial disparity in hypertension, stroke, and chronic kidney disease. It remains unclear the degree to which sickle cell trait contributes to this disparity. It also remains unclear... more »

Do SNPs account for any of the variability seen in the phenotypic expression of sickle cell disease? Can microarray analysis be used to map these SNPs, promoting refined care plans for those living with sickle cell disease?

A growing concern among the sickle cell community surrounds the lack of mental health and wellness services. Many in the community deal with anxiety and depression. It is well known how intricately connected mental and physical health are. So if we know that stress can trigger a psychological crisis which in turn triggers a physical pain crisis, why do we not automatically include mental health services within patient... more »