New agent joins hydroxyurea as FDA-approved sickle cell treatment


Dr. Meier in clinic with young patient

By Dana LeBlanc, M.D., and Emily Riehm Meier, M.D., M.S.H.S., FAAP

Sickle cell disease (SCD) affects 70,000-100,000 Americans and millions worldwide. It is characterized by lifelong complications such as severe pain episodes, chronic anemia, stroke, organ damage and shortened life expectancy.

The Food and Drug Administration has approved therapies for use in children with SCD, and all pediatric health care professionals should be aware of their availability and requirements for safe use.

Vasoocclusive pain events (VOE) are the leading cause of health care utilization in the U.S. for people with SCD, with an average lifetime cost of care approaching half a million dollars per patient.

Acute chest syndrome (ACS) is the leading specific cause of death of people with SCD, and co-existing asthma can increase the severity and frequency of ACS. Transcranial Doppler (TCD) ultrasound screening should be performed annually in children with HbSS or HbSβ0thalassemia to identify those at highest risk of stroke.

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