Showing 2 ideas for tag "hydroxyurea"

Goal 2: Reduce Human Disease

Optimization of Existing Therapies for Sickle Cell Disease

How can the safety, dosing and benefits of existing therapies for sickle cell disease such as hydroxyurea, be optimized in order to increase its efficacy and improve patient adherence?

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Compelling Question (CQ)

Details on the impact of addressing this CQ or CC

Hydroxyurea is a widely available disease-modifying therapy for sickle cell disease (SCD), but its effectiveness is currently limited by inadequate utilization, and less than optimal response. Research is needed to improve adherence to this evidence-based therapy and emphasis needs to be placed on determining whether therapy with hydroxyurea can prevent or even reverse organ dysfunction. In addition, research identifying new adjunct therapies to blood transfusion and hydroxyurea, as well as disease-specific therapies for co-morbidities such as kidney disease, hypertension, obstructive lung disease, and pulmonary hypertension will be valuable in the management and treatment of SCD.

Name of idea submitter and other team members who worked on this idea Alice Kuaban on behalf of the American Society of Hematology (ASH)

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Goal 2: Reduce Human Disease

Impact of Hydroxyurea on Organ Function

Does hydroxyurea preserve organ function in patients with sickle cell disease?

Is this idea a Compelling Question (CQ) or Critical Challenge (CC)? Compelling Question (CQ)

Details on the impact of addressing this CQ or CC

Hydroxyurea is the only disease-modifying medication for sickle cell disease, and has been shown to be effective in reducing complications such as pain crisis and acute chest syndrome in some patients. Evidence suggests that hydroxyurea is underused, and, understandably, efforts are underway to encourage more people to take advantage of this treatment. Determining that it preserves organ function or reduces organ damage could encourage greater use or, if not, encourage the development of other medications that can.

Feasibility and challenges of addressing this CQ or CC

Given the greater understanding of sickle cell disease and improvements in life expectancy, it should be feasible to design a study that will supply sufficient data to answer this question.

Name of idea submitter and other team members who worked on this idea The Sickle Cell Association of New Jersey

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