NOHARM MTD is an extension of a previous study for children with Sickle Cell Anemia (SCA) who were enrolled in the NOHARM study. All children enrolled in NOHARM received hydroxyurea treatment at a fixed daily dose of 20 mg/kg/day. This dose was selected as a likely safe dose, but does not escalate hydroxyurea to maximum tolerated dose "MTD" as is commonly done in the US. Without this information, we cannot know whether hydroxyurea treatment at the MTD would be feasible (since it requires closer monitoring to avoid hematological toxicities), safe (since adverse events may be greater with MTD, risk of malaria may be altered by MTD, and risk of infections as a result of neutropenia could also be greater with MTD) or beneficial (MTD is associated with higher hemoglobin and fetal hemoglobin concentration).
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Single group
hydroxycarbamide
DrugHydroxyurea at optimized dose
Composite measure of organ damage
The primary study endpoint will be a composite measure of organ damage to the brain (defined as ≥2 silent cerebral infarcts on brain MRI or ≥170 cm/sec flow velocities by TCD) or the kidneys (confirmed micro- or macro-albuminuria), or bones (femoral head AVN, Mitchell Stage B or higher).
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Children's Hospital Medical Center, Cincinnati
Lead sponsor
Makerere University
Collaborator
Indiana University
Collaborator
Indian Institute of Science
Collaborator
Mulago Hospital, Uganda
Collaborator
