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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16 up votes
3 down votes
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Goal 3: Advance Translational Research

Submitted by (@amy.sobota)

Implementation Science to Improve Care in Sickle Cell Disease

There are approximately 100,000 individuals living with sickle cell disease in the US, however study after study has shown that many lack access to the few existing evidence based interventions such as hydroxyurea. We need to investigate novel ways to increase acess to hematology care and disease modifying therapies.

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14 up votes
2 down votes
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Goal 2: Reduce Human Disease

Submitted by (@gko000)

Role of epigenetic mechanisms in cardiovascular disease

Are epigenetic changes the cause or the consequence of changes in cell function that contribute to cardiovascular disease? If they are the cause, what are the mechanisms that lead to changes and how do they impact disease pathogenesis? If the consequence, do they play any role in disease pathogenesis? What methods can be used to test if epigenetic changes play a causal role in disease pathogenesis? Thus far studies ...more »

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21 net votes
28 up votes
7 down votes
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Goal 4: Develop Workforce and Resources

Submitted by (@coretta.jenerette)

Sickle cell education for healthcare providers

Although sickle cell was first described more than 100 years ago and more than 100,000 individuals in the US are living with sickle cell disease, healthcare providers still lack basic knowledge of the key components in providing care for individuals with sickle cell. This often leads to poor health outcomes including stigmatization of patients with sickle cell seeking care. Evidenced-based curriculum should be available ...more »

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18 net votes
21 up votes
3 down votes
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Goal 3: Advance Translational Research

Submitted by (@sicklecellwarrior)

Dissemination & Implementation of new treatments and therapies in sickle cell disease

Are current advances in gene editing, new drug therapies and less restrictive BMT criteria being explained and rolled out to the sickle cell community in an effective and timely manner? When can people living with sickle cell disease experience a better quality of life on more permanent based on the treatments we already have?

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46 net votes
55 up votes
9 down votes
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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
137 up votes
27 down votes
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