VCU researchers pursue hope for adults with sickle cell disease

RICHMOND, Va. – A national sickle cell disease expert at the Virginia Commonwealth University Medical Center said the latest treatment for sickle cell disease, the drug hydroxyurea, can do more than just counteract painful episodes associated with the disease. It is also proving effective at prolonging life.

The director of the VCU Medical Center’s Adult Sickle Cell Center, Dr. Wally R. Smith, is seeing real results among his own patients.  Smith is part of the national research team that recently published study results in the Journal of the American Medical Association that show that hydroxyurea prolongs life, cutting observed mortality by as much as 40 percent.   

“Now that patients know that this drug prolongs life, they are more eager to take it,” said Smith, associate professor and chair of quality health care in VCU’s School of Medicine. “Although the drug has some side effects, there are reduced hospital stays, reduced number of crises and reduced levels of pain.”

Sickle cell disease is a hereditary condition that affects the body’s red blood cells. Normal red blood cells are soft, donut-shaped structures that pass easily through veins and arteries. Sickle cell patients have hard, crescent-shaped red blood cells that have difficulty passing through the body’s circulatory system. Pain results when the irregular shaped cells cause blockages, depriving tissues of oxygen.

In 1994, Smith and others first published groundbreaking research involving hydroxyurea, showing the drug cut in half the pain of adults with sickle cell disease.  As a result, the National Institutes of Health issued a clinical alert to all U.S. physicians suggesting that sickle cell patients eligible for hydroxyurea be given an opportunity to take it in order to decrease their pain and suffering. As a result, those patients who took the drug before and after 1994 have shown improved mortality rates.

Smith – principal investigator of the NIH sponsored Pain in Sickle Cell Epidemiology Study – is at the midpoint of an effort to better understand the different degrees of pain and hospital use among adults with sickle cell disease. The three-year, $1.4 million PiSCES analysis will ultimately enroll 350 patients in the Virginia cities of Richmond, Hampton and Norfolk. Study participants are required to maintain a diary to record the number and intensity of painful episodes they experience during a six-month period. They will also record if hospitalization was required or if the pain was managed at home.

“The PiSCES study is about learning why sickle cell patients experience different degrees of pain and using that information to design better treatments to alleviate that pain,” said Smith.

To help sickle cell disease patients better cope with pain, Smith has begun allowing some patients to use a pain pump to deliver morphine. The pump is a drug delivery system that is implanted within the spinal canal, much like the epidural pain blocker used during labor and delivery.

Smith is currently leading a multidisciplinary team focusing on a new investigational anti-sickling drug.

Approximately 77,000 Americans suffer from sickle cell disease. The majority of patients are African-American, but the disease can affect people of Spanish, Portuguese, Italian and Greek descent. About 2.5 million Americans have sickle cell trait, which is the gene that causes the disease.    

For more information about PiSCES, sickle cell studies or hydroxyurea, call the VCU Adult Sickle Cell Center, at 1-866-VCU-PISC.