Goal 2: Reduce Human Disease

Identifying Chronic Pain and exploring the onset and severity among patients with thalassemia

While the primary pathophysiology of thalassemia is related to globin gene mutations and unbalanced globin chain expression, the downstream consequences are manifold. Chronic pain turns out to be one of the most important factors identified by patients with transfusion dependent thalassemia major in health-related quality of life surveys and patient-reported outcome measures. Even as therapies aimed and gene editing, transplant, and modifications of hematopoiesis move forward, understanding the etiology and severity of, and best management strategies for thalassemia chronic pain deserve active NHLBI support at this time.

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

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37 net votes
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Idea No. 693