New strategies boost healthy RBC production to target anemia across a range of blood disorders

Dec. 8, 2014

Emerging treatment approaches may reduce the burden of anemia associated with blood disorders by enhancing production of healthy red blood cells, according to data presented yesterday, December 7, at the 56th American Society of Hematology (ASH) Annual Meeting and Exposition in San Francisco.

Many blood diseases are linked to anemia caused by a deficiency of healthy red blood cells (RBCs). These deficiencies occur either when the body cannot maintain adequate RBC production or, instead of healthy adult RBCs, it produces immature cells that cannot transport oxygen throughout the body. Myelodysplastic syndromes (MDS), beta thalassemia, and sickle cell disease (SCD) are examples of these types of disorders and are associated with moderate to severe anemia.

Many people suffering from anemia are treated with erythropoiesis-stimulating agents (ESAs, which help the bone marrow produce RBCs), or hydroxyurea, a chemotherapy agent that reduces the number of unhealthy cells in the blood. However, patients who do not respond to these agents must rely on regular blood transfusions to maintain proper RBC levels. While effective, transfusions are expensive, time-consuming, and associated with unique complications such as iron overload.

New research presented yesterday suggests that a class of agents known as activin receptor fusion proteins may reduce the burden of anemia by encouraging healthy RBCs to mature and proliferate. Two treatments in this class offer similar mechanisms and are under investigation for use in patients with low-risk MDS and beta-thalassemia. A separate study suggests that a complementary approach using the amino acid L-glutamine may support healthy RBC growth and diminish the complications of anemia with minimal side effects. Finally, a long-term observational analysis characterizes the natural history of SCD, better illustrating the early mortality associated with the disease and identifying opportunities for intervention during the transition from pediatric to adult care.

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