New hope for infants with sickle cell disease

St. Jude Children's Research Hospital (ALSAC)
Monday, 17 December 2001

St. Jude researchers find that drug effective in adults may also help very young children

(Memphis, Tennessee, December 17, 2001) A new study indicates that hydroxyurea, a drug proven to help prevent the excruciating pain crises of sickle cell disease in adults, is a potential treatment for infants with the genetic disorder. The research was published in the December issue of The Journal of Pediatrics.

Collaborating on the two-year study were four institutions, including St. Jude Children's Research Hospital in Memphis and Duke University Medical Center in Durham N.C.

The team of scientists, led by Winfred Wang, M.D., of St. Jude, determined that hydroxyurea therapy in children aged 6 to 24 months is feasible, is well-tolerated with minimal side effects, results in improved blood counts and may delay chronic organ damage from sickle cell disease.

"We are very encouraged by these findings to further investigate the value of hydroxyurea in infants with sickle cell disease," Wang said. "However, it is important to recognize that the clinical usefulness of hydroxyurea in children this age remains to be proven with further research. It is much too early to recommend routine administration of hydroxyurea to very young children."

Sickle cell disease is an inherited disorder of red blood cells that causes the cells to become sickle-shaped and hard, instead of round and soft. These abnormal blood cells clog blood vessels, causing episodes of severe pain, organ damage and eventually a premature death.

Sickle cell disease occurs primarily in African-Americans, but it also affects people of Hispanic and Middle Eastern descent. If both parents have the sickle cell trait there is a one-in-four chance that their child will be born with sickle cell disease. About 2,000 babies with sickle cell disease are born in the United States each year, making it one of the most prevalent genetic diseases. More than 70,000 Americans live with the disease.

The chemotherapy agent, hydroxyurea has been used for approximately 30 years in the treatment of certain kinds of cancers. However, the drug's ability to fight sickle cell disease has only been known since the mid-1990s.

Hydroxyurea works by causing the body to produce fetal hemoglobin, which reduces the severity of sickle cell disease by blocking the effects of sickle hemoglobin. Normally the body produces very little fetal hemoglobin after the age of six months.

In addition to debilitating pain, sickle cell disease often causes permanent damage to organs such as the kidney, brain and spleen. For example, most sickle cell patients more than 3 years old no longer have functioning spleens. Hydroxyurea may prove to be effective—not only in preventing pain—but also in delaying or preventing the organ damage associated with sickle cell disease.

St. Jude Children's Research Hospital, in Memphis, Tennessee, was founded by the late entertainer Danny Thomas. The hospital is an internationally recognized biomedical research center dedicated to finding cures for childhood catastrophic diseases. The hospital's work is supported through funds raised by the American Lebanese Syrian Associated Charities (ALSAC). ALSAC covers all costs not covered by insurance for medical treatment rendered at St. Jude Children's Research Hospital. Families without insurance are never asked to pay.

For more information, or to contact St. Jude Children's Research Hospital (ALSAC), see their website at: www.stjude.org