Hydroxyurea - Pragmatic Reduction In Mortality and Economic burden

Can simple and affordable interventions improve the long-term survival and quality of life among children with sickle cell disease in sub-Saharan Africa?

What is this study about?

Sickle cell disease (SCD) is an inherited disorder caused by abnormal red blood cells which can lead to chronic anaemia, intermittent crises and progressive multi-organ deterioration. It is most commonly found in sub-Saharan Africa. Without treatment, SCD results in high rates of both disability and early death.  

Hydroxyurea has been shown to be safe and effective for the treatment of SCD when used in high-income countries, but the usual approach to treatment requires close laboratory monitoring to find the right dose, which is not practical or available in low-income settings. Therefore, H-PRIME will compare a high vs low dose of hydroxyurea, given pragmatically without extensive monitoring.

Since malaria and bacterial infections are widely considered to be the most common causes of early death among children with SCD in sub-Saharan Africa, H-PRIME also includes comparisons of two antimalarial and two antimicrobial regimens, in a 2x2x2 factorial design. This means that each participant is randomised in three independent comparisons:
  • In the first comparison, participants are randomly assigned to receive either hydroxyurea or a placebo control.
  • In the second comparison, participants are randomly assigned to receive either the current standard of care antimalarial treatment (suphadoxine-pyramethamine) or dihydroartemisinin-piperoquine.
  • In the third comparison, participants are randomly assigned to receive either the standard of care antimicrobial (penicillin) to prevent bacterial infections or cotrimoxazole.

Type of study

Randomised trial

Data Sharing

The datasets generated during from the trial will be available after publication under a controlled accessed approach on reasonable request to the trial principal investigator.

Who is funding the study?

The trial is supported by grant funding from the Joint Global Health Trials board (Medical Research Council UK), the UK Department for International Development (DFID), and the Wellcome Trust. 

When is it taking place?

The trial opened in January 2024 will run until July 2029.

Where is it taking place?

The trial is taking place across four sites in Eastern Uganda.

Who is included?

1,800 children aged 1 to 10 years with confirmed sickle cell disease.