Sickle cell disease remains a major cause of childhood death and illness, most heavily in sub-Saharan Africa and relevant across the Indian subcontinent. A WHO-convened Paediatric Drug Optimization process reviewed therapies and set priorities for children.
Hydroxyurea (hydroxycarbamide) was confirmed as the leading near-term priority, with the central practical gap being age-appropriate soluble or dispersible formulations to make dosing feasible and equitable for young children; a formal target product profile was defined. Among investigational agents, pyruvate kinase activators and a decitabine-based regimen were flagged as promising for paediatric study. The process also warned against letting the promise of gene-therapy cure divert investment from scalable disease-modifying treatment.
The practical message is to keep hydroxyurea central to paediatric sickle cell care and to push for child-friendly formulations and access, rather than waiting for curative therapies that will reach few children in the near term.
- A WHO process set research and access priorities for sickle cell drugs in children.
- Hydroxyurea was confirmed as the leading near-term priority.
- Age-appropriate soluble or dispersible formulations were deemed essential for young children.
- Pyruvate kinase activators and a decitabine-based regimen were flagged as promising.
- The group cautioned against letting gene-therapy hype divert investment from scalable treatment.
Why it matters
It reaffirms a cheap, scalable disease-modifying drug as the priority for most children, against the pull of expensive cures.
Don't overread it
This is a priority-setting review, not a new efficacy trial; it signals where to invest, not a change to how an individual child is dosed today.
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