NIH urges increased use of hydroxyurea in patients with sickle cell disease

Various factors are preventing patients from receiving a treatment that could alter the process of their disease.

Changes in the treatment of sickle cell disease are needed on various levels, according to the National Institutes of Health.

Since it received FDA approval in 1998, hydroxyurea (Hydrea, Bristol-Myers Squibb) has been available for the treatment of sickle cell disease in adults. However, a limited number of physicians specializing in sickle cell disease, along with concerns from healthcare providers and patients, have caused a drop in use of hydroxyurea.

In order to address the pressing issues surrounding sickle cell disease, the National Heart, Lung and Blood Institute and the Office of Medical Applications of Research of the NIH held a Consensus Development Conference from Feb. 25 to 27, 2008.

Insufficient data

The group discussed the efficacy of hydroxyurea, as reported in multiple clinical trials, in the treatment of three groups of patients with sickle cell disease: infants, preadolescents and adults.

Despite the benefits to adults and preadolescents, such as decreases in pain crises, blood transfusions, hospitalizations and acute chest syndrome, data regarding hydroxyurea’s efficacy in children is lacking. While the evidence in children does not contradict efficacious results reported in adults, it is simply more limited due to weaker designs and a smaller number of participants, according to the NIH group.

Treatment obstacles

According to the NIH, there are four levels of barriers preventing patients from receiving the care they deserve: patient, family, provider and system.

Given possible short and long-term risks associated with hydroxyurea, patients and their families fear adverse events such as cancer and birth defects, and are generally unaware that hydroxyurea is presently the only therapy that modifies the process of the disease. A lack of information regarding hydroxyurea in the treatment of sickle cell disease, along with a shortage of experts knowledgeable about hydroxyurea for the treatment of the disease remains a problem in the provider arena. A shortage of insurance and coverage, limited access to centers that offer comprehensive care and inadequate patient support from the government, industry and philanthropists continue to be important issues at the system level.

Potential solutions

Though the issues remain serious, the group has developed a series of solutions to the problems surrounding sickle cell disease and treatment with hydroxyurea, including:

• promoting comprehensive and family-centered care;
• using physicians and health educators to improve the physical and mental health of patients with sickle cell disease; and
• constructing advocacy programs, including peer and patient advocates.

The group also suggested improvements are needed at the federal, state and local levels to increase awareness of sickle cell disease and treatment with hydroxyurea, and to improve insurance coverage, such as extending Medicare and Medicaid.

Future research is also necessary to determine the efficacy of hydroxyurea in clinical and laboratory outcomes. The group suggested that studies evaluate the long-term outcomes and explore interventions that will lower the barriers to treatment. – by Stacey L. Adams

This document voices the concern of many sickle cell physicians and researchers regarding the sub-optimal adoption of hydroxyurea in the treatment of sickle cell disease. While it often takes a few years for positive results from clinical trials to find their way into routine medical practice, this situation appears to exceed that norm. The major trials demonstrating a clear benefit for hydroxyurea are now more than a decade old, yet for reasons that have been addressed (although not fully resolved) in this NIH consensus statement, there has been a failure on the part of the community of patients, providers and health care systems to implement this advance. Among other reasons, it is suggested that this failure may in part be due to the fragmentation and non-uniformity of quality of care available to patients with sickle cell disease in the United States currently. As hematologists, we need to step up to the challenge of implementing this, the only disease modifying therapy available in sickle cell disease.

– Nigel Key, MD

HemOnc Today Editorial Board member