قالب وردپرس درنا توس
Home / Health / "Nothing to Gangbusters": Treatment of sickle cell anemia is effective in Africa

"Nothing to Gangbusters": Treatment of sickle cell anemia is effective in Africa



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.

"There is currently no treatment in Africa, and many children die before the age of five," said Drs. Osunkwo, who has treated children in the United States and Nigeria. "We go from nothing to gangbusters."

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.

Image
Dr. Leon Tshilolo, a pediatric hematologist at the Monkole Hospital Center in Kinshasa, Democratic Republic of the Congo, with one patient.

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.

[ Like the Science Times page on Facebook. | Sign up for the Science Times newsletter.

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.


Source link