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 »

Voting

112 net votes
141 up votes
29 down votes
Active

Goal 2: Reduce Human Disease

Submitted by (@nareg.roubinian)

Biology of Red Blood Cell Alloimmunization

What determines which individuals will develop RBC alloimmune responses resulting in clinically meaningful sequelae? This question encompasses: 1) the generation of alloantibodies that limit the availability of compatible blood or cause hemolytic disease of the fetus or newborn (HDFN); 2) the distinction between clinically significant and insignificant alloantibody responses, especially within alloantibody specificities ...more »

Voting

44 net votes
58 up votes
14 down votes
Active

Goal 3: Advance Translational Research

Submitted by (@nhlbiforumadministrator)

Bone Marrow Stem Cell Transplant in Peds sibling matched SCD

There is a need to improve accessibility of Bone Marrow Stem Cell Transplantation (BMSCT) for Sickle Cell Disease patients who are most likely to benefit from this treatment option. 1. Building a culture of trust between and among primary care providers, specialists, patients/families, and other stakeholders 2. Consensus building around BMSCT as an acceptable treatment alternative (as opposed to another research endeavor) ...more »

Voting

52 net votes
80 up votes
28 down votes
Active

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 »

Voting

23 net votes
28 up votes
5 down votes
Active

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.

Voting

9 net votes
9 up votes
0 down votes
Active

Goal 2: Reduce Human Disease

Submitted by (@nhlbiforumadministrator1)

Exercise effects on cellular level

What cellular and/or intracellular signaling events are activated in response to acute or chronic physical activity that lead to or explain improvements in health outcomes, prevention, or treatment of lung diseases?

Which metabolic signatures induced by exercise/physical activity would be predictive/ associated with poor lung disease prognosis?

Voting

18 net votes
32 up votes
14 down votes
Active