Big win for sickle cell drug in African children means access is now the question

The largest pediatric clinical trial of hydroxyurea ever conducted in Africa found the drug — long the standard of care for sickle cell disease patients in developed countries — is also safe and effective in low-resource countries.

After a median of 2.5 years of treatment, the 600 children enrolled into the study and treated with hydroxyurea experienced less pain and anemia, lower rates of blood transfusions, and fewer deaths compared to the pre-treatment screening phase of the trial, said lead investigator Dr. Léon Tshilolo, of the Centre Hospitalier Monkole in Kinshasa, Democratic Republic of the Congo.

Previous studies had suggested hydroxyurea might increase the risk of malaria and other infections by suppressing the immune system. Importantly, this large study found the opposite: Episodes of malaria and infection decreased.

“Because of its positive impact on anemia, clinical events, and quality of life, hydroxyurea can now be considered for routine care of children with sickle cell anemia in Africa,” said Tshilolo. 

But improving patient access to the drug across Africa is a problem not yet solved. “The current cost of treatment is beyond the daily wage of most families living in sub-Saharan Africa,” Tshilolo said. “We hope that treatment will be made available to more patients through outside financial support, as is the case with treatment for HIV infection in several African countries.”