THE VIRGINIAN-PILOT
Copyright (c) 1995, Landmark Communications, Inc.
DATE: Tuesday, January 31, 1995 TAG: 9501310446
SECTION: FRONT PAGE: A1 EDITION: FINAL
SOURCE: BY WARREN E. LEARY, NEW YORK TIMES
DATELINE: BETHESDA, MD. LENGTH: Medium: 79 lines
Calling an early halt to a national trial because of promising results, doctors Monday announced the first treatment for sickle cell anemia, which in the U.S. primarily affects black Americans. The drug attacks the underlying cause of the disease rather than simply combat its painful symptoms.
The National Institutes of Health said it was issuing a clinical alert to thousands of doctors nationwide informing them that the drug hydroxyurea should be considered for many adult patients with the crippling blood disease. The institutes had sponsored the trial of the drug at nearly two dozen centers to see if it could reduce the painful episodes characteristic of the disease.
``This is the first effective treatment for sickle cell disease and we are elated,'' said Dr. Samuel Charache of Johns Hopkins University Medical School. Charache, the principal investigator and coordinator of the planned five-year study, said, ``but we want to emphasize that hydroxyurea is a treatment for the disease and not a cure.''
Moreover, doctors cautioned that there could be adverse effects in some patients and that the drug should not be given to women who plan to have children nor, for now, to children.
Sickle cell anemia is an inherited blood disease most common among people whose ancestors came from Africa, the Middle East, the Mediterranean and India. In the United States, experts estimate, between 70,000 and 80,000 people have it.
It is estimated that 1 in 12 blacks carries the sickle cell trait, a genetic predisposition to the condition that can be passed on to their offspring. If both parents have the trait, their child has a 25 percent chance of having the disease, a 50 percent chance of having the trait, which is usually symptomless, and a 25 percent chance of having neither.
Severe cases of sickle cell disease are characterized by periodic painful episodes, or crises, that can require hospitalization. Until now, doctors could treat only the symptoms of the disease when a crisis occurred, by using narcotics and other pain killers, blood transfusions and other palliative therapies.
During the hydroxyurea trial, which was to have ended in May, doctors tested 299 adults with severe sickle cell disease, meaning a patient had at least three severe pain crises in the previous year. Half the participants took daily oral doses of hydroxyurea and the rest had a placebo, or dummy drug.
Researchers said hydroxyurea had reduced by 50 percent the number of pain episodes, hospitalizations, situations requiring blood transfusions and incidents of a life-threatening complication called acute chest syndrome, which is characterized by fever and severe chest pain.
The results were so dramatic, the researchers said, that the NIH stopped the study on Jan. 14 so that the placebo patients could be offered the drug.
Ruthie Abney, 40, a patient who participated in the trial at the Howard University Center for Sickle Cell Disease in Washington, said, ``When I started taking hydroxyurea, I noticed that my crises became less frequent and less severe and I generally felt better, with a higher level of energy.''
Abney, a secretary in a federal agency, said that before the treatments she had suffered up to nine crises a year that required hospitalization. Since she began taking the drug in 1992, Abney said, she had had only about five crises and was able to treat them at home with prescription pain medicine. ILLUSTRATION: Graphic
ADRIANA LIBREROS/Staff
HOW THE DISEASE DEVELOPS
SOURCE: Associated Press
[For complete graphic, please see microfilm]
Graphic
WHERE TO CALL
For more information, call the Sickle Cell Disease Association,
800-421-8453.
by CNB