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New Guidance for Managing Sickle Cell Disease

High-quality evidence is limited

An expert panel has created a new evidence-based guideline for managing sickle cell disease (SCD), with a strong recommendation for the use of hydroxyurea and transfusion therapy for many individuals with the disorder, although high-quality evidence is limited, with few randomized clinical trials conducted for this disease, according to an article in the September 10 issue of JAMA.

Sickle cell disease is a life-threatening genetic disorder affecting nearly 100,000 individuals in the U.S.; most of those affected are of African ancestry or self-identify as black. The disease is associated with acute and chronic complications requiring immediate medical attention. Care for persons with SCD often lacks continuity. Primary care and emergency care health professionals need up-to-date clinical guidance regarding the care of persons with this disease, according to background information in the article.

In 2009, the National Heart, Lung, and Blood Institute convened an expert panel that developed the Evidence-Based Management of Sickle Cell Disease: Expert Panel Report 2014. Barbara P. Yawn, MD, MSc, MSPH, of the Olmsted Medical Center, Rochester, Minn., and colleagues produced a summary of this report and conducted a search of the medical literature to examine the strength of the quality of the evidence for the recommendations.

Among the “strong” recommendations:

  • For prevention: daily oral prophylactic penicillin up to the age of 5 years; annual transcranial Doppler examinations from the ages of 2 to 16 years in those with sickle cell anemia; and long-term transfusion therapy to prevent stroke in children with abnormal transcranial Doppler velocity (elevated speed of blood flow in the cerebral arteries).
  • For acute complications: rapid initiation of opioids for the treatment of severe pain associated with a vaso-occlusive crisis (i.e., the blockage of blood flow due to the abnormal “sickled” red blood cells getting stuck in the blood vessels) and the use of incentive spirometry (a method for encouraging deep breathing with the use of an instrument to provide feedback) in patients hospitalized for a vaso-occlusive crisis.
  • For chronic complications: the use of analgesics and physical therapy for the treatment of avascular necrosis (a condition in which a poor blood supply to an area of bone leads to bone death) and the use of angiotensin-converting enzyme (ACE) inhibitor therapy for microalbuminuria (a subtle increase in the urinary excretion of the protein albumin that cannot be detected by a conventional urinalysis) in adults with SCD.
  • For children and adults with proliferative sickle cell retinopathy: referral to expert specialists for consideration of laser photocoagulation and for echocardiography to evaluate signs of pulmonary hypertension.

The authors concluded: “The process of developing guidelines for the management of children, adolescents, and adults with SCD has been challenging because high-quality evidence is limited in virtually every area related to SCD management. The systematic review of the literature identified a very small number of randomized clinical trials in individuals with SCD, demonstrating the extensive knowledge gaps in SCD and care of affected individuals. The expert panel realizes that this summary report and the guidelines leave many uncertainties for health professionals caring for individuals with SCD and highlight the importance of collaboration between primary care health professionals and SCD experts. However, we hope that this summary report and the SCD guideline begins to facilitate improved and more accessible care for all affected individuals, and that the discrepancies in the existing data will trigger new research programs and processes to facilitate future guidelines.”

Michael R. DeBaun, MD, MPH, of the Vanderbilt University School of Medicine, Nashville, Tenn., commented on the new guideline in an accompanying editorial.

“Yawn and colleagues have undertaken a monumental effort to produce a practical, evidence-based guideline for SCD,” he wrote. “Many aspects of this guideline will help both individuals with the disease and clinicians. As would be expected, when the guideline is based on recommendations from randomized clinical trials, such as penicillin prophylaxis, transcranial Doppler screening, blood transfusion therapy prior to surgery, or hydroxyurea therapy for severe disease, these strong recommendations will be embraced by the SCD community. However, when recommendations are based on consensus panel expertise, practice variation will justifiably continue.”

Sources: JAMA; September 10, 2014; and Medical Xpress; September 9, 2014.

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