New treatment guidelines for sickle cell disease

Expand treatment of sickle cell disease with 2 “underused” preventive therapies to reduce painful flare-ups, an expert panel convened by the National Heart, Lung, and Blood Institute urges in new management guidelines.

Preventive maintenance with hydroxyurea and regular monthly blood transfusions reduces painful exacerbations that occur when sickle-shaped blood cells block blood vessels, the guidelines note, although neither therapy cures the disease. Daily oral prophylactic penicillin is strongly recommended for children aged up to 5 years.

Hydroxyurea, which increases fetal hemoglobin, can reduce occlusive crises in the short run and mortality in the long run. The guidelines recommend offering the therapy for children aged as young as 9 months whether or not they have symptoms. The drug takes 3 months to start working.

Blood transfusions halt production of abnormal blood cells and increase the blood count, decreasing anemia. They can lower the risk of stroke but may cause complications including increased blood viscosity and iron overload. The guidelines recommend annual transcranial Doppler ultrasound examination for children aged 2 to 16 years and long-term transfusion therapy to prevent stroke in children with abnormal results.

The guidelines also urge that children in severe acute pain receive rapid treatment with opioids when necessary. Early, aggressive treatment can prevent acute pain from becoming chronic, the panel notes.

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The expert panel, which spent 5 years reviewing evidence, found its task challenging “because high-quality evidence is limited in virtually every area related to [sickle cell disease] management”-including bone marrow transplants, which have the potential to cure the disease but are deemed experimental. The panel emphasizes the importance of further research as well as expanding the availability of care for all sickle cell patients through greater collaboration between primary care professionals and experts in the disease.

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