Sickle Cell Disease Guideline Highlights Need to Treat Acute Episodes

AAFP Member Served as Panel's Co-chair

October 08, 2014 10:37 am Chris Crawford

Sickle cell disease (SCD) is a life-threatening genetic disorder that was first described in 1910 and that now affects nearly 100,000 people in the United States. SCD is associated with many acute and chronic complications that can require immediate medical attention.

[Sickle Cells Blocking Blood Flow]

AAFP member Barbara Yawn, M.D., M.Sc., M.S.P.H., of Rochester, Minn., recently co-chaired a panel that developed a clinical guideline(www.nhlbi.nih.gov) on managing SCD, a summary(jama.jamanetwork.com) of which was published online Sept. 10 by JAMA: The Journal of the American Medical Association.

Because SCD is a relatively uncommon condition, there are not enough SCD specialists around the country, Yawn told AAFP News. As a result, family physicians and other primary care physicians play an important role in expanding support to all individuals and families living with SCD, she said.

"As we learn better how to treat and manage SCD, people with SCD are living longer and will need physicians who can care for them not only in childhood and adolescence but also into adulthood," said Yawn.

Guideline Overview

The expert panel identified and reviewed a final subset of 1,575 original studies, breaking them down into a series of evidence tables that focus on various aspects of the disease and its treatment.

Story highlights
  • According to a newly released clinical guideline, the recommended oral treatment for sickle cell disease (SCD) is hydroxyurea, which can be started as early as at age 9 months.
  • A person with SCD who arrives in the office or emergency department complaining of sudden onset of severe pain requires immediate treatment with appropriate pain therapy, which may include opioids.
  • People with SCD (and all other chronic diseases) often have multiple problems, and acute care is appropriately given to those issues.

Prior to publication, the full guideline was reviewed by the National Heart, Lung and Blood Advisory Council (NHLBI), a separate panel of SCD experts, and the National Blood Disorders Program coordinating committee.

Yawn highlighted three key points from the guideline for AAFP News.

First, SCD can and should be treated to prevent the disease's complications, she said. The recommended oral treatment is hydroxyurea, which can be started as early as at age 9 months. There are potential side effects associated with the drug, Yawn added, but many of the medications physicians prescribe have potential side effects.

"To provide adequate care to all people with sickle cell disease in the United States, we need family physicians who have multiple affected patients to learn to initiate and monitor hydroxyurea therapy," Yawn said. "Experts in SCD can be contacted and consulted to help with co-management. Just as rural physicians have long helped monitor and support chemotherapy, they can co-manage people with SCD."

Secondly, when a person with SCD arrives in the office or emergency department complaining of sudden onset of severe pain, he or she requires immediate treatment with appropriate pain therapy, which may include opioids. Delaying the initiation of adequate pain therapy can lead to further complications, Yawn added.

"While drug overuse is of course a concern for all health professionals, inadequate treatment is also a major concern for those with acute pain crises in SCD," she said. "You may see no physical signs, but if the person has a diagnosis of SCD (usually confirmed in the medical records or a call to their personal physician) then treating is the first step. Treatment should begin within 30 minutes of admission to the emergency department.

"These episodes are called vaso-occlusive crises for good reason."

Finally, people with SCD (and all other chronic diseases) often have multiple problems, and acute care is appropriately given to those issues. But people with SCD require important preventive care measures, as well. These include, for example, administering the correct immunizations and following the U.S. Preventive Services Task Force recommendation(www.uspreventiveservicestaskforce.org) on screening for SCD in newborns, Yawn said.

"Unique to SCD, infants require daily prophylactic penicillin therapy due to the high risk of invasive pneumococcal disease, and transcranial doppler screening to evaluate for the risk of strokes that often begin in childhood," she said.

With the advent of universal newborn screening, Yawn noted, virtually every infant with SCD will be diagnosed at birth or soon afterward. Doing so provides the opportunity to begin penicillin prophylaxis early, assure appropriate immunizations and begin the discussion with parents about potential use of other therapies, such as hydroxyurea, she said. Early diagnosis also allows for early identification of local SCD specialists with whom family physicians can consult for support in co-management.

"Almost all people with SCD will have multiple pain or vaso-occlusive crises beginning as early as a few months of age," said Yawn. "We hope these guidelines will be able to give the family physician and his or her staff the immediate information they need to guide routine care such as immunizations and screening, as well as immediate care of complications."

Expert Panel Makeup

The group of experts on the SCD guideline panel included several adult and pediatric hematologists who specialize in SCD, blood bank and transfusion medicine specialists, a psychiatrist who specializes in support and care of SCD patients and families, a maternal fetal medicine specialist, an emergency department nurse with expertise in acute SCD crises management and the methodologists. The NHLBI staff member who worked with the panel, Joylene John-Sowah, M.D., is a family physician, as well, said Yawn.

This is the seventh guideline panel Yawn has participated on, she said, and she thinks it's important that family physicians continue to represent the family medicine viewpoint on any guideline that will eventually affect their practices.

"It is important to have a family physician on these panels who can not only help interpret the evidence and level of evidence but can also make sure that the recommendations are written in a format that is useful for primary care and encompasses the important care we provide in prevention and health maintenance, as well as care of complications," Yawn said.

More From The AAFP
American Family Physician: "Opportunities to Improve Outcomes in Sickle Cell Disease"
(July 15, 2006)

American Family Physician: "Approach to the Vaso-occlusive Crisis in Adults with Sickle Cell Disease"
(March 1, 2000)