Goal 2: Reduce Human Disease

Development of Optimally Hemostatic, Systemically Safe, Platelet Mimetics or Substitutes

What are the knowledge and technological gaps in production, evaluation and clinical translation of donor-independent platelets for transfusions? Specific questions include: a) How can stem or progenitor cells be expanded to maximize platelet production?; b) What are the hemostatically relevant design and function requirements and evaluation metrics for ideal/optimal “biologic” and “synthetic” platelets? c) What preclinical ...more »

Submitted by (@dtriulzi)

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Goal 2: Reduce Human Disease

Will Novel Therapies Create Bleeding Remission HHT and Angiogenic Disorders

Chronic bleeding due to epistaxis and gastrointestinal bleeding telangiectasia in HHT leads to transfusion dependence, iron infusion dependence,

decreased quality of life and premature death.

 

Novel therapies such as pomolidamide, Avastin and pazopanib may be new promising therapies to lead to remission in bleeding and reduce the burden of disease

Submitted by (@mariannes.clancy)

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Goal 2: Reduce Human Disease

How should platelet (PLT) transfusions be used to treat active bleeding?

Multiple randomized controlled trials have been performed to evaluate the use of prophylactic PLT transfusions in non-bleeding, thrombocytopenic hematology-oncology patients. However no high-quality data exist to guide PLT transfusions in actively bleeding patients inclduing pediatric and adult medical and surgical patients. After hematology-oncology patients, cardiac surgery patients are the next largest group of PLT ...more »

Submitted by (@bldbuddy)

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Goal 2: Reduce Human Disease

What new methods of platelet preparation, processing, and storage are needed for hemostasis in various clinical conditions?

The limitations of 5-day 22˚ C storage significantly impacts platelet availability. It is critical that we develop new methods of collection, processing, storage to extend the storage time of platelets, and evaluate the use of whole blood. The attributes of these products must be understood to optimally alignment product attributes, clinical efficacy and safety with hemostatic needs in a variety of clinical states. Specifically, ...more »

Submitted by (@bldbuddy)

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Goal 3: Advance Translational Research

Novel Mechanism for Clinical Trials of New Pro-Hemostatic Agents in Hemophilia

There are new exciting novel pro-hemostatic therapeutics in early phase clinical trials for hemophilia and hemophilia inhibitor patients. Yet, it is difficult to design randomized trials to compare these agents, or compare them with standard treatment, given the small sample size and competing studies for such patients. It is critical to develop novel approaches to compare new agents in rare populations. For example, ...more »

Submitted by (@ragni01)

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Goal 2: Reduce Human Disease

Need to assess a new method of warfarin management vs. new oral anticoagulants in patients with atrial fibrillation

The two obstacles to warfarin therapy (keeping the INR in range and the associated hassles of frequent lab visits) can be eliminated by INR self testing and online "virtual clinic" monitoring and management (as demonstrated in six small studies. Achieving an INR percent time in range of approximately 75% to 80% is associated with a 50% or lower rate of thromboembolism and major bleeding. The studies of new oral anticoagulants ...more »

Submitted by (@bussey)

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Goal 2: Reduce Human Disease

Balancing Risks and Benefits: How Do Clinical Guidelines in Cardiovascular Medicine Promote the Health of an Individual?

Much of the hopes for precision medicine (as outlined Dr. Dr. Collins) are based on deriving large amounts of genomic, proteomic, epigenomic and metabolomic data on large cohorts of patients. It will take decades to build these cohorts and even more time to analyze them and derive specific conclusions on how these will help individualize treatments. However, there is a pressing need for how to individualize contemporary ...more »

Submitted by (@jalees)

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