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Five important updates in sickle cell disease

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National Sickle Cell Awareness Month — observed in September — draws attention to the collection of inherited blood disorders that affect up to 100,000 Americans.

In conjunction with Sickle Cell Awareness Month, HemOnc Today presents five updates in sickle cell disease treatment or research.

• Individuals with sickle cell disease demonstrated more than double the risk for leukemia compared with the general population, according to an analysis of population-based data from California. Read more.
• The FDA granted rare pediatric disease designation to GBT440 (Global Blood Therapeutics) — an oral, once-daily therapy for patients with sickle cell disease — for the treatment of sickle cell disease. Read more.
• Diffuse myocardial fibrosis, a common feature in sickle cell anemia, predicted the development of diastolic dysfunction. Read more.
• Patients with sickle cell anemia experienced acute myocardial infarction at earlier ages and also demonstrated increased risk for in-hospital mortality compared with patients who did not have sickle cell anemia. Read more.
• A multicenter study is underway to determine whether there is a safe level of exercise for children and adolescents with sickle cell anemia. The 5-year study — funded by a $2.7 million grant from NHLBI — will examine the effect of exercise intensity on inflammation in 140 children, half of whom have sickle cell anemia. Read more.