Sickle cell disease (SCD) is a life-threatening genetic disorder that affects approximately 100,000 people in the United States, with African Americans being more commonly affected than others. SCD is associated with many acute and chronic complications that require immediate medical attention. Sickled cells can block blood flow, which can lead to organ damage and pain and increase the risk of infection and strokes, among other problems. In JAMA, an expert panel issued guidelines for managing SCD after reviewing more than 12,000 scientific articles in an effort to provide a blueprint for caring for these patients.

Prevention & Treatment

A key guideline recommendation is to give children oral penicillin daily until the age of 5 and to vaccinate them against pneumococcal disease, says Barbara P. Yawn, MD, MSc, MSPH, who co-chaired the expert panel that was convened by the National Heart, Lung, and Blood Institute to develop the guidelines. “This is necessary to reduce the risk of pneumonia and other infections,” she says. In addition, the guidelines recommend that children between the ages of 2 and 16 have an annual transcranial Doppler exam to measure blood flow in the brain. If findings are abnormal, long-term transfusion therapy to prevent stroke is recommended.

Manage-Sickle-Cell-Callout

“In acute pain or vaso-occlusive crises (VOCs), pain treatment—often with opioids—needs to begin immediately,” Dr. Yawn say. “Appropriate therapy should be started and clinicians can worry about drug seeking behaviors later.” Incentive spirometry is also advised for those hospitalized with VOCs. The panel stresses greater use of hydroxyurea and long-term blood transfusions, two disease-modifying therapies that are effective but largely underused. The guidelines note that hydroxyurea should be used in adults who have three or more severe blood flow crises in a year to prevent formation of sickle-shaped red blood cells. “Hydroxyurea can also be used in infants, children, and teens, regardless of whether or not they currently have symptoms,” Dr. Yawn says. “This oral therapy is appropriate for almost all patients who have SCD.” She notes that clinicians should pay special attention to the transfusion recommendations because this therapy is complex and is not appropriate for all complications or all patients.

More Research Needed

The guidelines were challenging to develop because there currently is not enough research to guide SCD monitoring or therapy, according to Dr. Yawn. “People with the disease are living longer, but patient access to SCD clinics is not ideal,” she says. “To improve care, we need greater collaboration between primary care physicians or clinicians and SCD experts.” She also hopes that discrepancies in the existing data will trigger new research programs and processes to facilitate future guidelines. The recommendations will likely change over time as more evidence becomes available.

References

Yawn BP, Buchanan GR, Afenyi-Annan AN, et al. Management of sickle cell disease: summary of the 2014 evidence-based report by the expert panel members. JAMA. 2014;312:1033-1048. Available at: http://jama.jamanetwork.com/article.aspx?articleid=1902235.

Lanzkron S, Strouse JJ, Wilson R, et al. Systematic review: hydroxyurea for the treatment of adults with sickle cell disease. Ann Intern Med. 2008;148:939-955.

Segal JB, Strouse JJ, Beach MC, et al. Hydroxyurea for the treatment of sickle cell disease. Evid Rep Technol Assess (Full Rep). 2008; 165:1-95.

Sheth S, Licursi M, Bhatia M. Sickle cell disease: time for a closer look at treatment options? Br J Haematol. 2013;162:455-464.