JAMA: An expert panel has created a new evidence-based guideline for
managing sickle cell disease (SCD), with a strong recommendation for the
use of the drug hydroxyurea and transfusion therapy for many
individuals with SCD, 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 United States; most of those affected
are of African ancestry or self-identify as black. This disease is
associated with many 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 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, M.D., M.Sc.,
M.S.P.H., 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
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 those children with abnormal transcranial
Doppler velocity (elevated speed of blood flow in the cerebral
arteries).
• To address acute complications: rapid initiation of opioids for
treatment of severe pain associated with a vasoocclusive crisis
(blockage of blood flow due to the abnormal “sickled” red blood cells
getting stuck in the blood vessels) and use of incentive spirometry (a
method of encouraging deep breathing with the use of an instrument to
provide feedback) in patients hospitalized for a vasoocclusive crisis.
• For chronic complications: use of analgesics and physical therapy
for treatment of avascular necrosis (a condition in which poor blood
supply to an area of bone leads to bone death) and use of
angiotensin-converting enzyme 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.
• Hydroxyurea therapy is strongly recommended for adults with 3 or
more severe vasoocclusive crises during any 12-month period, with SCD
pain or chronic anemia interfering with daily activities, or with severe
or recurrent episodes of acute chest syndrome (an SCD syndrome
associated with one or more symptoms that may include fever, cough,
sputum production or difficulty breathing.
A recommendation of moderate strength suggests offering treatment
with hydroxyurea without regard to the presence of symptoms for infants,
children, and adolescents.
The authors note that some of the strong recommendations appearing in
this guideline are supported by low- or very low-quality evidence, and
that evidence is lacking in many areas important to care for individuals
with SCD.
“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,” the authors
conclude.
(doi:10.1001/jama.2014.10517; Available pre-embargo to the media at http://media.jamanetwork.com)
Editor’s Note: Please see the article for additional information,
including other authors, author contributions and affiliations,
financial disclosures, funding and support, etc.
There will also be a digital news release available for this study,
including the JAMA Report video, embedded and downloadable video, audio
files, text, documents, and related links. This content will be
available at 3 p.m. CT Tuesday, September 9 at this link.
Editorial: The Challenge of Creating an Evidence-Based Guideline for Sickle Cell Disease
Michael R. DeBaun, M.D., M.P.H., of the Vanderbilt University School
of Medicine, Nashville, Tenn., comments on this guideline in an
accompanying editorial.
“Yawn and colleagues have undertaken a monumental effort to produce a
practical, evidence-based guideline for SCD. 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.”
(doi:10.1001/jama.2014.11103; Available pre-embargo to the media at http://media.jamanetwork.com)
Editor’s Note: The author has completed and submitted the ICMJE Form
for Disclosure of Potential Conflicts of Interest and none were
reported.