Goal 3: Advance Translational Research

Submitted by (@nhlbiforumadministrator)

Improving Community-Based Care for Sickle Cell Disease

Sickle cell treatment centers are located throughout the United States, primarily in urban areas, and play an invaluable role. However, there is a critical need to identify and educate primary care providers who can provide routine and preventive care, but will also know when to consult with/refer to hematologists and other appropriate providers when necessary.

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Goal 2: Reduce Human Disease

Submitted by (@lakshmanankrishnamurti)

What is the place of curative therapies in the management of Sickle Cell Disease

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 »

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112 net votes
141 up votes
29 down votes
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Goal 2: Reduce Human Disease

Submitted by (@juliewashko)

Assess the true impact of sickle cell trait on cardiovascular health across then age spectrum

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 »

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17 net votes
31 up votes
14 down votes
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Goal 2: Reduce Human Disease

Submitted by (@hulbertm)

Identification and validation of surrogate endpoints for long-term morbidity in Sickle Cell Disease

Research in sickle cell disease (SCD) has mostly focused on preventing or treating acute medical events, such as vaso-occlusive pain, acute chest syndrome, and, in pediatric patients, acute strokes. Chronic SCD complications such as chronic kidney disease or pulmonary hypertension, develop over decades, thus are poor choices for clinical trial endpoints. There is a great need to develop surrogate endpoints that predict ...more »

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13 net votes
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Goal 3: Advance Translational Research

Submitted by (@xuejunparsons)

Exploring Future Cardiovascular Medicine: Heart Precursors Directed from Human Embryonic Stem Cells for Myocardium Regeneration

Cardiovascular disease (CVD) is a major health problem and the leading cause of death in the Western world. Currently, there is no treatment option or compound drug of molecular entity that can change the prognosis of CVD.

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Goal 2: Reduce Human Disease

Submitted by (@jantin)

Transplantation across HLA barriers in aplastic anemia

Allogeneic stem cell transplantation is curative in aplastic anemia with much less intrinsic toxicity than transplantation in hematologic malignancies. The recent BMT-CTN trial demonstrated 97% survival at one year with little subsequent decline. However patients without matched related or unrelated donors have graft-rejection rates of up to 50%. Preliminary data from the Netherlands suggests that anti-thymocyte globulin ...more »

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110 net votes
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Goal 2: Reduce Human Disease

Submitted by (@sicklecellwarrior)

Mental health and wellness in 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 »

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25 net votes
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13 down votes
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