Christine Duncan, MD

2009-12-05

Sickle cell disease (SCD) affects as many as 100,000 Americans, and a comprehensive understanding of SCD is essential for pediatric and adult hematologists. This common disease will be explored in depth in an Education Program Session today at 7:30 a.m. and again at 2:00 p.m. in rooms 393-396 of the Ernest N. Morial Convention Center. The session is designed for all hematologists who treat patients with sickle cell syndromes, including general adult and pediatric hematologists and those who practice in tertiary care centers.

Gene therapy offers promise as a curative therapy for SCD but is not yet a reality. Stem cell transplantation has successfully cured many severely affected patients, but it is limited by the availability of donors and by transplant-related toxicity. As a result, we are still searching for effective and tolerable alternative therapies for this common disease.

In today’s session, Drs. Kenneth Ataga of the University of North Carolina and Russell Ware of St. Jude Children’s Research Hospital will review existing and innovative therapies for SCD.

Dr. Ataga will describe treatments aimed at decreasing sickle hemoglobin polymerization directly, and therapies that target aspects of sickle cell pathobiology other than hemoglobin (e.g., vascular adhesion or cellular hydration).

Dr. Ware will then examine the use of hydroxyurea in SCD, a treatment widely used by clinicians since landmark clinical trial results were reported in the 1990s. While hydroxyurea cannot be considered a novel therapy, it is receiving increased attention, and there is renewed enthusiasm for the drug. The 2008 NIH consensus statement regarding the use of hydroxyurea in patients with SCD cites strong evidence for the efficacy of the drug in adult patients and compelling data for its use in children.

The NIH consensus statement that examined the use of hydroxyurea also addressed the need for patients with SCD to have a primary health care provider. The statement suggests that providers either specialize in the care of patients with SCD or maintain contact with a hematologist with expertise in SCD. This recommendation highlights the often overlapping arenas of SCD management and primary care, which Dr. Joshua Field from Washington University, St. Louis, will emphasize in his discussion of two commonly co-exisiting conditions that have complex interactions: SCD and asthma. As Dr. Field explains, "We do not fully understand if these are two separate disease processes or if the asthma is a manifestation of sickle cell disease. The diagnosis and treatment of patients with sickle cell and airway disease is not straightforward. The same rules used to diagnose a patient with asthma in the general population may not apply to a child with sickle cell disease. Sickle cell comorbidities may make it harder to diagnose and to treat a patient with asthma and SCD."

Dr. Duncan indicated no relevant conflicts of interest.

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