Goal 2: Reduce Human Disease

Combination Iron Chelator Trials in Thalassemia and other transfusion-dependent anemias

Three chelators are presently available in the US and much of the world: parenteral deferoxamine, and oral deferasirox, as well as oral deferiprone. Monotherapy is unsuccessful in a significant minority of patients, due to side effects or inadequate response at tolerable doses. Taking a page from enormously successful strategies for combination oral therapy in hypertension, led by NHLBI and others over the past four decades, it makes most sense to foster organized clinical trials of combination agents, as the drugs may have additive therapeutic effect, but tend not to have overlapping side effects.

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NHLBI should lead the way for these trials, which will be of enormous benefit to reduce morbidity and mortality in thalassemia, a terribly common problem worldwide, as well as in the rare, transfusion-dependent congenital anemia.

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Idea No. 690