Goal 2: Reduce Human Disease

Impact of each VOC Crisis in patients with sickle cell disease

While the long term cumulative effects of frequency, duration and severity of VOC on mortality is known in SCD, there is little known about the impact of each individual crisis or the amount of damage during crisis versus background smoldering ischemia from the disease. Any effort in quantifying this for SCD in the absence of interventional agents initially, and then as a potential measurement of the benefit of drug ...more »

Submitted by (@tosinola)

Voting

3 net votes
3 up votes
0 down votes
Active

Goal 3: Advance Translational Research

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?

Submitted by (@sicklecellwarrior)

Voting

46 net votes
55 up votes
9 down votes
Active

Goal 2: Reduce Human Disease

Assess the true impact of sickle cell trait on cardiovascular health across then age spectrum

Sickle cell disease is now understood as a disease of inflammation in addition to abnormal red blood cells. It is likely persons with sickle cell trait are also negatively affected by the damage caused by inflammation. There is a significant racial disparity in hypertension, stroke, and chronic kidney disease. It remains unclear the degree to which sickle cell trait contributes to this disparity. It also remains unclear ...more »

Submitted by (@juliewashko)

Voting

17 net votes
31 up votes
14 down votes
Active

Goal 2: Reduce Human Disease

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 »

Submitted by (@lakshmanankrishnamurti)

Voting

112 net votes
141 up votes
29 down votes
Active

Goal 3: Advance Translational Research

Next generation of cellular blood products for clinical use

What advancements in our understanding of hematopoietic stem cell differentiation can be combined with practical developments in novel processing and storage technologies to develop the next generation of transfused blood therapeutics? There is a dearth of tools to evaluate the efficacy of transfused blood products, although this is the most commonly employed therapeutic procedure in the US. There is also no financial ...more »

Submitted by (@nhlbiforumadministrator)

Voting

-3 net votes
24 up votes
27 down votes
Active

Goal 3: Advance Translational Research

Novel Cell Apheresis Technologies to Treat Hematologic Diseases

Current FDA approved apheresis technology uses elutriation/centrifugation or filtration separation techniques to remove pathologic cellular and/or plasma elements. Currently these techniques are non-specific, limited by inefficient removal kinetics and often require considerable blood product exposure. Despite tremendous improvement in our understanding of the pathophysiology of a variety of disease, our ability to ...more »

Submitted by (@ewong0)

Voting

118 net votes
139 up votes
21 down votes
Active