There is cur­rently no treat­ment in Africa, and many chil­dren die be­fore age five.”

The East African - - OUTLOOK -

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.’”

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