NIH-convened panel recommends expanded adoption of drug and transfusion treatments for individuals with sickle cell disease

Group produces first systematic evidence-informed report to assist clinicians in preventing and treating the many conditions affecting those living with sickle cell disease

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An expert panel has recommended expanded adoption of the drug hydroxyurea for the care of people with sickle cell disease, according to a report issued today. The report also suggests that clinicians give periodic blood transfusions to children with the disease to reduce stroke risk. According to the panel, both treatments are underutilized.

The National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health, convened the panel to review the evidence and make recommendations relating to care available to those with this inherited blood disorder. The report, Evidence-Based Management of Sickle Cell Disease, Expert Panel Report, 2014, gathers the latest sickle cell prevention and treatment information into one document.

Along with those recommendations, the report emphasizes the need for consistent, high-quality health maintenance for people with sickle cell disease. Examples of health maintenance recommendations include the regular use of penicillin through age 5 to reduce the chance of deadly infections, and the importance of pneumococcal vaccines for children and adults.

“Since the NHLBI’s earliest days, sickle cell disease has been an important part of its mission,” said Gary H. Gibbons, M.D., director of the NHLBI. “This report represents an important milestone as NHLBI continues to seek new ways to reduce the burden of this devastating condition.”

Sickle cell disease is relatively rare, affecting an estimated 70,000 to 100,000 Americans. African-Americans make up the majority of those with the disease, though others affected include people of Hispanic/Latino, Mediterranean, and Middle Eastern descent. The report aims to provide a clinical resource for primary care providers, nurses, specialists, emergency care personnel, and other members of the health care community, many of whom have limited experience treating people with this condition.

Sickle cell disease results from an abnormality in hemoglobin, the protein found in red blood cells responsible for moving oxygen throughout the body. People living with this condition carry two copies of an altered gene that produces sickle-shaped hemoglobin instead of normal adult hemoglobin, which looks more like a disk. Sickle hemoglobin causes the red blood cell to become stiff, misshapen and sticky, which can slow blood flow to tissues. This process can damage organs and cause pain throughout the body. Severe pain episodes often require emergency room visits.

The NHLBI formed a 12-member external expert panel to thoroughly examine evidence and review current practices in the management of sickle cell disease. In addition to experts in adult and pediatric hematology, the panel included representatives from the fields of family medicine, general internal medicine, psychiatry, transfusion medicine, emergency nursing as well as obstetrics and gynecology. George R. Buchanan, M.D., of the University of Texas Southwestern Medical Center in Dallas, and Barbara Yawn, M.D., of the University of Minnesota, co-chaired the panel.

The NHLBI plans to work with organizations such as the American Society of Hematology and the Sickle Cell Disease Association of America to help broadly disseminate the report so that it can be more quickly put into wider practice and improve consistency of care.

“We recognize that these clinical guidelines are not the end of the conversation,” said Dr. Gibbons. “The NHLBI remains committed to being a global leader in sickle cell research. The panel report provides guidance for our future research agenda to enhance the well-being of patients with sickle cell disease.”

Among the known information gaps is the lack of a widely available cure and the fact that some children with the condition are at high risk of stroke. Modified bone marrow transplants have cured some children and adults, but the procedure is only currently viable for a small number of people.

Part of the National Institutes of Health, the National Heart, Lung, and Blood Institute (NHLBI) plans, conducts, and supports research related to the causes, prevention, diagnosis, and treatment of heart, blood vessel, lung, and blood diseases; and sleep disorders. The Institute also administers national health education campaigns on women and heart disease, healthy weight for children, and other topics. NHLBI press releases and other materials are available online athttp://www.nhlbi.nih.gov.

About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.

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