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WHO moves to expand hydroxyurea access for children with sickle-cell disease

The World Health Organization has begun a coordinated effort to expand access to hydroxyurea and comprehensive care for children with sickle-cell disease, following its first global clinical guideline dedicated to paediatric management.

WHO estimates that sickle-cell disease contributed to about 81,100 deaths among children under five in 2021. Roughly 80% of cases occur in sub-Saharan Africa, with substantial burden in South Asia and other regions.

What the guideline recommends

The May 2026 guideline contains 15 recommendations across seven priority areas. It strongly recommends hydroxyurea for children and adolescents aged nine months to 19 years with sickle-cell anaemia, regardless of clinical severity, alongside appropriate monitoring and wider supportive care.

Hydroxyurea can reduce painful crises, acute chest syndrome, hospitalisation and other complications. It is not a cure, and access must include diagnosis, dosing, laboratory monitoring, vaccination, infection prevention and education for families.

From guidance to supply

WHO issued a target product profile for a child-friendly formulation in July and has opened a prequalification expression of interest covering paediatric products and 500-milligram capsules. Prequalification can help procurement agencies identify products meeting quality standards, but it does not by itself guarantee affordability or delivery to every clinic.

Newborn screening remains scarce in many high-burden settings, so some children die without a diagnosis. Expanding treatment therefore requires stronger primary care, reliable medicine supplies and referral systems—not only a written guideline.

Sources: WHO access initiative; WHO paediatric sickle-cell guideline.

Official artwork for the initiative. Image: WHO.

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