An independent panel convened this week by the NIH concluded
that the use of hydroxyurea for sickle cell patients should
be increased in adolescents and adults. Hydroxyurea
was approved by the U.S. Food and Drug Administration for
use in adults with sickle cell anemia in 1998, but provider
and patient concerns have hindered its use, depriving many
patients of its proven benefits. Research has shown
that sickle cell patients on this drug experience fewer pain
crises and hospital admissions, and the panel advocated increased
utilization of this drug with appropriate monitoring. Additionally,
the panel concluded that the risks of serious side effects
of hydroxyurea appear to be lower than previously expected. Furthermore,
these risks are acceptable when compared to the risks of
untreated sickle cell disease in adolescents and adults.
"The compelling benefits of hydroxyurea warrant increased
adoption of this drug as a frontline therapy in adults with
sickle cell disease," reported Dr. Otis Brawley, conference
panel chair, Professor of Hematology, Oncology, Medicine,
and Epidemiology at Emory University, and Chief Medical Officer
of the American Cancer Society.
For younger patients, however, safety and efficacy data
are limited but supportive of hydroxyurea treatment. Although
the panel was unable to definitively recommend broad pediatric
use of the drug at this time, it is hoped that results from
ongoing clinical trials will help to resolve remaining questions.
The pain and complications associated with sickle cell disease
can have a profound impact on patients' quality of
life, ability to work, and long-term health and well-being.
Sickle cell disease often causes episodes of severe pain,
and decreased life span due to infections, lung problems,
and stroke. Worldwide, millions suffer from sickle
cell disease, most commonly people whose families come from
Africa, South or Central America, Caribbean islands, Mediterranean
countries, India, and Saudi Arabia. In the U.S., this
inherited blood disorder affects 50,000 to 100,000 people. In
addition, approximately 2 million Americans carry the sickle
cell trait, which increases the public health burden as this
disorder is passed on to future generations.
Surveys indicate that a large proportion of patients with
sickle cell disease are ethnic minorities, poor, and from
underserved communities. For many, limited resources
and lack of culturally competent clinicians set the stage
for suboptimal care. Recurring pain crises associated
with the disease can severely limit individuals' ability
to sustain employment or educational efforts, aggravating
problems with insurance coverage and subsequent healthcare
costs.
"This disease illuminates the limitations of our current
healthcare system," Dr. Brawley noted. "The
best way to achieve optimal care for patients with sickle
cell disease is for them to be treated in clinics specializing
in the care of this disease." The panel recognized
that many patients lack a single healthcare provider to direct
their sickle cell management. Instead, there is heavy
reliance on emergency and acute care facilities to treat
pain. Dr. Brawley added, "all sickle cell patients
should have a principal healthcare provider, and that provider,
if not a hematologist, should be in frequent consultation
with one." Additionally, patients often "fall
through the cracks" when transitioning from pediatric
to adult care. Contributing to this problem is a
lack of providers armed with the knowledge, skills, and experience
to effectively manage adults with sickle cell disease.
In addition to identifying numerous potential barriers to
hydroxyurea treatment at the patient, provider, and systems
levels, the panel called for Medicare or Medicaid coverage
of sickle cell patients of all ages.
The panel's complete consensus statement will be available
later today at http://consensus.nih.gov/. The conference
was sponsored by the NIH Office of
Medical Applications of Research (OMAR) and the National
Heart, Lung, and Blood Institute, along with other NIH and
Department of Health and Human Services components. This
conference was conducted under the NIH Consensus Development
Program, which convenes conferences to assess the available
scientific evidence and develop objective statements on controversial
medical issues.
The 14-member conference panel included experts in the fields
of internal medicine, family practice, hematology, oncology,
pediatrics, obstetrics, nursing, pediatric nursing, social
work, pharmacology, pharmacokinetics, and pain research,
mental health, epidemiology, biostatistics, public health,
and health systems research, in addition to a public representative. A
complete listing of the panel members and their institutional
affiliations is included in the draft conference statement. Interviews
with panel members can be arranged by contacting Lisa Ahramjian
at 301-496-4999 or AhramjianL@od.nih.gov.
In addition to the material presented at the conference
by speakers and the comments and concerns of conference participants
presented during discussion periods, the panel considered
pertinent research from the published literature and the
results of a systematic review of the literature commissioned
by OMAR. The systematic review was prepared through
the Agency for Healthcare Research and Quality (AHRQ) Evidence-based
Practice Centers (EPC) program, by the Johns Hopkins Evidence-based
Practice Center. The EPCs develop evidence reports
and technology assessments based on rigorous, comprehensive
syntheses and analyses of the scientific literature, emphasizing
explicit and detailed documentation of methods, rationale,
and assumptions. The evidence report on Hydroxyurea
Treatment for Sickle Cell Disease is available at http://www.ahrq.gov/clinic/tp/hydscdtp.htm.
The panel's statement is an independent report and is not
a policy statement of the NIH or the federal government. The
NIH Consensus Development Program was established in 1977
as a mechanism to judge controversial topics in medicine
and public health in an unbiased, impartial manner. NIH
has conducted 118 consensus development conferences, and
29 state-of-the-science (formerly "technology assessment")
conferences, addressing a wide range of issues. A backgrounder
on the NIH Consensus Development Program process is available
at http://consensus.nih.gov/forthemedia.htm.
Note to Radio Editors:
An audio report of the conference results will be available
by visiting http://www.nih.gov/news/radio/index.htm
The Office of the Director, the central office at NIH, is
responsible for setting policy for NIH, which includes 27
Institutes and Centers. This involves planning, managing,
and coordinating the programs and activities of all NIH components. The
Office of the Director also includes program offices which
are responsible for stimulating specific areas of research
throughout NIH. Additional information is available
at http://www.nih.gov/icd/od.
The National Institutes of Health (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. It is the primary federal agency for conducting
and supporting basic, clinical and translational medical research,
and it investigates the causes, treatments, and cures for both
common and rare diseases. For more information about NIH and
its programs, visit www.nih.gov.