Goal 2: Reduce Human Disease

Submitted by (@nhlbiforumadministrator1)

Innovations in Red Cell Transfusion in Sickle Cell Disease

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 »

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29 net votes
47 up votes
18 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 (@rjjones)

Allogeneic transplantation as a safe and universally available therapeutic strategy for treating non-malignant blood diseases

Can new advances in allogeneic blood or marrow transplantation (BMT) make the procedure a safe and universally available therapeutic strategy for treating non-malignant blood and immune disorders such as sickle cell anemia, thalassemia, aplastic anemia, and severe combined immune deficiency?

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164 net votes
214 up votes
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Goal 2: Reduce Human Disease

Submitted by (@coretta.jenerette)

Symptom management in sickle cell

Symptom management is a significant challenge for individuals living with sickle cell. In most cases, sickle cell research in symptom management focuses on pain. Although important, many other symptoms such a fatigue, anxiety, and depression need to be identified and intervened on to improve the quality of life for individuals living with sickle cell disease.

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15 net votes
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4 down votes
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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 (@bsachais)

Apheresis Medicine in the Management of Sickle Cell Disease

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 »

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130 net votes
152 up votes
22 down votes
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