News Release

Wednesday, February 27, 2008

Panel Finds Hydroxyurea Treatment Is Underutilized for Sickle Cell Disease

Improved access to care and education about the treatment are deemed priorities

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 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

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

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

Note to Radio Editors:
An audio report of the conference results will be available by visiting

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