Goal 3: Advance Translational Research

Submitted by (@coretta.jenerette)

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 »

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14 net votes
17 up votes
3 down 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
38 up votes
13 down votes
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Goal 2: Reduce Human Disease

Submitted by (@freddigoldman)

How can we more safely deliver stem cells to Sickle Cell patients

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 »

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51 net votes
67 up votes
16 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
16 up votes
3 down votes
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Goal 2: Reduce Human Disease

Submitted by (@smajumdar)

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 »

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23 net votes
28 up votes
5 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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