A team of international researchers has found that dose escalation of hydroxyurea treatment for children in Uganda with sickle cell anemia is more effective and has similar side effects than a lower fixed dose of the same drug.
The study, known as NOHARM MTD (Novel use Of Hydroxyurea in an African Region with Malaria – Maximum Tolerated Dose), focused on children in Uganda, but the results could impact use of hydroxyurea worldwide, including the United States and Europe.
This clinical research milestone removes a major barrier to broadly expand the use of hydroxyurea in low-resource regions like sub-Saharan Africa, according to the physicians who led the study at Makerere University in Kampala, Uganda, Cincinnati Children’s Hospital Medical Center, and the Indiana University (IU) School of Medicine.
Sickle cell anemia is a life-threatening blood disorder that distorts red blood cells into a sickle or crescent shape and leads to anemia, recurrent pain, organ damage, and early death.
According to lead study co-investigator Chandy John, MD, a physician scientist at the Ryan White Center for Pediatric Infectious Diseases and Global Health at the IU School of Medicine, the optimal dosing and monitoring plan for hydroxyurea was unknown when the study started.
Particularly for low-resource countries like Uganda, determining the hydroxyurea optimal dosing and monitoring plan was crucial.
John said the new study confirms that the dose escalation regimen is better than fixed dose, but expanding hydroxyurea treatment will require affordable drug costs, education of healthcare providers, and an increased drug supply. In addition, newborn screening for sickle cell anemia is needed to help identify those who will benefit from treatment as early as possible.
“Our data make it clear that children on a higher daily dose had substantially better clinical outcomes, with fewer adverse events,” John said.
“The study shows clearly that the optimized dosing strategy for hydroxyurea, though it requires more effort than a fixed-dose treatment regimen, results in far better outcomes for children with sickle cell anemia,” Ware said. (ANI)
