A drug that protects children in affluent countries from painful and sometimes deadly attacks of sickle cells has proven safe in Africa, where the disease is far more common, scientists said Saturday.
According to experts, more research needs to be done, but knowing that hydroxyurea – a cheap, effective, and easy-to-take pill – can safely be given to African children can save millions of teens from excruciating pain and early deaths. 19659002] "I think this will be amazing," Dr. Ifeyinwa Osunkwo, who runs a sickle cell disease program in Charlotte, NC, but was not involved in the new study.
The disease, in which blood cells interweave into rigid semicircular forms, is caused by a genetic mutation that was supposed to have originated in Africa about 7,000 years ago.  Approximately 300,000 babies are born each year with the disease. 75 percent of them are in Africa and about 1 percent in the United States.
The disease is found throughout America and the Caribbean among descendants of Africans brought to this hemisphere by the slave trade. Sickle cell disease is also less common in southern Europe, the Middle East and India.
Malaria is also endemic until a few decades ago. People who inherit a copy of the sickle cell gene are partially protected against malaria, which is probably why the mutation has existed in Africa.
But children who inherit the gene from both parents are often susceptible to anemia infections and crises in which their blood cells clump together and bind capillaries in the brain, lungs, and other organs.
The pain is often so distressing that only opioids can help. The treatment may require blood transfusions or, in affluent countries, bone marrow transplants that kill themselves.
Many children die from strokes or organ damage without treatment.
Hydroxyurea has been used for decades in the United States and Europe. Some early animal studies have led researchers to fear that African children may become more susceptible to local infections, especially malaria.
The new study followed 600 children in Angola, Uganda, Kenya and DRC drugs for more than two years.
As with children in affluent countries, the daily intake of the drug also reduces the likelihood that they would die or require a blood transfusion because of their sickle cell disease. They had about half as much severe pain and less likely other infections.
In an unexpected twist, the investigators discovered that the children had about half as many malaria when using hydroxyurea as they had been before the trial . The reasons are unknown.
"With all the lack of malaria, malnutrition and vitamin deficiency in Africa, we could not assume it would work as well as it did," Dr. Russell E. Ware, director of hematology at Cincinnati Children's Hospital and co-author of the study, presented at a meeting of the American Society for Hematology and simultaneously published in the New England Journal of Medicine.
Hydroxyurea is already on the World Health Organization The organization's core list of medicines is available in generic form for about 50 cents per pill and can be stored at room temperature, said Drs. Would.
If this study raises the interest of buying millions of additional doses for use in Africa, the drug could probably be made much cheaper.
Although the study was quite large, it had some limitations.
Only evidence should be provided that the drug was safe for children 1 to 10 years of age. It was not meant to test different dosages to find the ideal ones, nor how many lab tests are needed to monitor children taking the drug, nor to determine the long-term effects.
The research will therefore require further work.
In addition, research was conducted without placebo control – a group of similar children who did not receive the drug. Regulators in the four test countries considered it unethical not to deny the drug to any child, as it was known that it should work elsewhere, said Drs. Leon Tshilolo, a pediatric hematologist at the Monkole Hospital Center in Kinshasa, Democratic Republic of the Congo. and the lead author of the study.
To compensate for the lack of a placebo group, researchers observed children two months before starting hydroxyurea. This set the baseline rates at which children usually suffer from pain crises, need blood transfusions and have suffered malaria or other infections.
The results "mean that survival is better, even in very resource-poor conditions," Dr. Tshilolo.
Hydroxyurea was originally developed to fight blood cancers such as leukemia, and individuals who ingest it must be monitored to ensure that this is not the case to dangerously lower their white blood cells and platelets.
However, the study used moderate daily doses and only about five percent of enrolled children had to lower their doses as their blood cell numbers decreased.
In 1998, the Food and Drug Administration approved the drug for American adults with sickle cell disease; Paediatricians soon began to give children outside the label . Would.
Trials proving that they were safe with American children were not completed until 2016, and the F.D.A. approved the pediatric application last year and opened the way for a process among children in Africa.
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Many black Americans with sickle cell disease have been reluctant to enroll themselves or their children in drug testing for years, Osunkwo said, because of America's dirty history of medical experiments on black patients – including the infamous Tuskegee study, in which black men remained untreated with syphilis even after the invention of penicillin.
She also said the drug is lower in sperm counts for men, break off women's hair and make fingernails dark gray. For safety reasons, it is usually not given to pregnant women, even if they can suffer severe sickle cell crises.
Dr. Osunkwo said she slowly overcame the patients' reluctance to help her plan the trials.
"And," she added, "I'd say, 'Dead is worse than having dark nails.'"
In Africa, 600 children enrolled were relatively easy, Dr. Tshilolo because Africans with sickle cell disease who had visited Europe had heard of Hydroxyurea and knew it worked.
Sperm counting is obviously not an issue in a children's process, he added. Normally, however, African men were willing to use the drug once it was stated that the decrease in sperm count was relatively small and recovered after the drug was discontinued.