(@hulbertm)

Goal 2: Reduce Human Disease

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
16 up votes
3 down votes
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(@amy.sobota)

Goal 3: Advance Translational Research

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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12 net votes
14 up votes
2 down votes
Active
(@coretta.jenerette)

Goal 4: Develop Workforce and Resources

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 »

Voting

18 net votes
21 up votes
3 down votes
Active
(@coretta.jenerette)

Goal 2: Reduce Human Disease

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
19 up votes
4 down votes
Active
(@coretta.jenerette)

Goal 3: Advance Translational Research

Follow-up care for newborns diagnosed with sickle cell trait or disease

• There is a need to develop and support formal programs to provide follow-up care for newborns who test positive for the sickle cell trait or sickle cell disease upon screening. While newborn screening programs exist nationwide, healthcare providers report that often, screening is conducted only upon request (likely related to cost) and there is usually no follow-up afterwards. Interventions are also needed further... more »

Voting

14 net votes
17 up votes
3 down votes
Active
(@smajumdar)

Goal 2: Reduce Human Disease

A Chidren's Oncology Group (COG) for sickle cell disease (SCD)?

We have all witnessed the success of the National Cancer Institute (NCI) funded Children's Oncology Group - an organization that has made tremendous advancements in the care of children with cancer, very rare compared to sickle cell disease. COG has been able to not only create a database of the numerous studies, but has the unique ability to make "smaller" institutions feel important as is evident by patient enrollment.... more »

Voting

23 net votes
28 up votes
5 down votes
Active
(@nhlbiforumadministrator1)

Goal 1: Promote Human Health

Interaction of blood cells with their environment

It is beginning to be known that malignant cells interact with neighboring cells and the course of their malignancy is directed by those cells. It is also known that transplanted stem cells interact with nearby cells and acquire direction of maturation and cell type from those cells. In my experience in unpublished work, the nature of the materials that leukocytes and monocytes have encountered during their apheresis... more »

Voting

39 net votes
47 up votes
8 down votes
Active
(@sicklecellwarrior)

Goal 2: Reduce Human Disease

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 »

Voting

25 net votes
38 up votes
13 down votes
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