To investigate whether a number of alternative treatments, delivered pragmatically by non-specialist staff could improve survival and quality of life for children with sickle cell disease living in resource-poor environments within Sub-Saharan Africa.
Rationale and Abstract
Sickle cell disease is a common but neglected genetic disease that has its greatest burden in sub-Saharan Africa. Without early diagnosis and appropriate treatment, aimed primarily at preventing the common causes of ill-health and death (which include bacterial diseases and malaria) the disease is associated with high mortality during childhood. Although implementation of early life screening and the prevention of infections with vaccines, penicillin and drugs for malaria, can lead to greatly improved survival, other complications of sickle cell disease can still lead to a reduced quality of life.
Participants will be randomly allocated to receive one of three different treatments and will be followed up over a maximum of 48 months. The first randomisation will be to hydroxyurea, prescribed pragmatically through a weight-band based dosing strategy aimed at delivering 25 +/-5mg/kg/day, with clinically based monitoring only, versus placebo control. The second and third randomisations will be to standard of care prophylaxis for malaria using suphadoxine-pyramethamine given monthly or alternative prophylaxis with dihydroartemisinin-piperoquine given weekly and standard of care prophylaxis for bacterial infections with penicillin V given twice daily until the age of 5years or alternative prophylaxis with cotrimoxazole given once daily throughout childhood.
- Study design: 2x2x2 factorial multi-centre randomised partially placebo-controlled trial
- Primary study design: Interventional
- Secondary study design: Randomised controlled trial
- Trial setting: Hospitals
- Trial type: Prevention
International Standard Randomised Controlled Trial Number: ISRCTN15724013