There is currently no treatment in Africa, and many children die before age five.”
Dr Ifeyinwa Osunkwo, directs a sicklecell disease programme in Charlotte, North Carolina
Also, the research was done without a placebo control — a group of similar children not getting the drug. Oversight boards in the four test countries felt it would be unethical to deny the drug to any child, since it was known to work elsewhere, said Dr Leon Tshilolo, a paediatric haematologist at the Monkole Hospital Centre in Kinshasa, DR Congo, and the study’s lead author.
To compensate for the lack of a placebo group, the researchers watched children for two months before starting them on hydroxyurea. That established the baseline rates at which the children normally suffered pain crises, needed blood transfusions and got malaria or other infections.
The results “mean survival will be better even in very low-resource settings,” Dr Tshilolo said.
In 1998, the US Food and Drug Administration approved the drug for American adults with sickle-cell disease; paediatricians soon began giving it off-label to children, Ware said.
Trials proving it was safe in American children were not finished until 2016, and the FDA approved paediatric use last year, opening the way for a trial in children in Africa.
For years, many black Americans with sickle-cell disease were reluctant to enrol themselves or their children in drug trials, Dr Osunkwo said, because of the United States’ sordid history of medical experimentation on black patients — including the infamous Tuskegee Study, in which black men with syphilis were left untreated even after the invention of penicillin.
Also, she said, the drug is known to lower men’s sperm counts, break off women’s hair and turn fingernails dark gray. For safety reasons, it is not normally given to pregnant women even though they may suffer severe sickle-cell crises.
Dr Osunkwo said she slowly overcame patients’ reluctance by letting them help design the trials.
“And,” she added, “I would say, ‘Being dead is worse than having dark nails.’”