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At the Forefront of Sickle Cell Disease Research and Treatment
CHORI Scientists Invited to Present at NIH Consensus Development Panel

“The challenge here is that HU is a very effective medication for SCD but it isn’t as widely used it should be.”

Given that the comprehensive sickle cell and thalassemia research centers located at CHRCO are among the most prominent in the world, it should come as no surprise that CHORI scientist and hematology/oncology director, Elliot Vichinsky, MD, was invited to present at the NIH Consensus Development Conference on Hydroxyurea (HU) Treatment for Sickle Cell Disease (SCD). The 3-day panel in late February covered the most current research on HU treatment in pediatric, adolescent and adult populations, as well as the current barriers to treatment.

“The meeting brings together leading SCD clinical and research experts to present current information to a panel of unbiased, independent clinicians, researchers and public health experts about HU efficacy, effectiveness, potential short and long term harm, and barriers to its use," explains CHORI scientist Marsha Treadwell, PhD, a colleague of Dr. Vichinsky’s who will conduct the presentation.

"The panel then uses that information to build a consensus statement on how to address the challenges of HU treatment and how to move forward. The challenge here is that HU is a very effective medication for SCD but it isn’t as widely used it should be.”

Initially used as an anti-cancer drug, HU actually dilutes the number of “sickle” cells and increases the proportion of red blood cells in circulation that have normal structure and function. The result is that HU reduces many of the complications associated with SCD – such as painful events, acute chest syndrome and associated hospital admissions.

However, there has been very little widespread use of the drug, as the need for the NIH Consensus Panel indicates. Drs. Vichinsky and Treadwell’s presentation will address current barriers to treatment for children as well as potential solutions.

“The first barrier is the health care infrastructure itself, in which pediatricians have trouble implementing appropriate therapies for chronic illness,” explains Dr. Treadwell. “In general there seems to be a real challenge with translating research into practice.”

Dr. Treadwell refers here to studies that have shown that nationwide, less than half of children receive clearly indicated therapies to avoid serious adverse health outcomes. In addition to these larger healthcare issues, SCD treatment has its own barriers as well.
“Only a small proportion of people with SCD have access to comprehensive care such as what is offered at CHRCO, so it falls on the people in the community to care for patients.”
Often, these very busy primary care pediatricians lack both the time and the knowledge to properly educate and counsel families in the risks and benefits of therapies.

“It’s been shown that people can be accepting of and adherent with therapies that they see has posing some risk,” Dr. Treadwell says, “but it takes time. If you only have a few minutes to spend with them, people aren’t going to trust and accept it.”

In addition, treatment may be complicated by the lack of transportation, inability to finance co-payments associated with lab fees, or language issues.

Solutions to these barriers must be as equally multi-faceted as the barriers themselves. Drs. Vichinsky and Treadwell’s initial suggestions include the need for
coordination of local, state and federal healthcare agencies; research that more clearly defines who is actually caring for the majority of SCD patients so that the most effective strategies to target those caretakers can be developed; and revised and accessible Standard of Care guidelines for SCD.

With the help of the NIH Consensus Panel, many of these suggestions may in fact be implemented. In the mean time, as local patients already know, CHRCO is spearheading these issues, utilizing their comprehensive, multi-disciplinary program that offers a variety of approaches to thinking about how to translate research into practice.

“We’re at the forefront of translational research, and have been instrumental in offering our patients the newest therapies, creating and revising clinical care guidelines, and providing education to healthcare providers in our community,” says Dr. Treadwell.

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