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Common drug helps babies with sickle cell disease

CHICAGO (Reuters) A drug that eases symptoms of sickle cell disease in older children is safe and effective to use for babies with the blood disorder and should become the new standard of care, US researchers said last week.They said the generic drug hydroxyurea significantly reduced pain and other common complications in babies with the disease, including hospitalisations and the need for blood transfusions.“These results show that hydroxyurea has the potential to dramatically improve the quality of life for an entire generation of patients with sickle cell disease,” said Dr Winfred Wang of St Jude Children’s Research Hospital in Memphis, Tennessee, who led the six-year study published in the journal Lancet.Wang said in a statement the findings mean hydroxyurea should now be considered for treatment of all infants and toddlers with the disease, also called sickle cell anaemia, in hopes of preventing or delaying disease complications.In sickle cell disease, an inherited blood disorder, the body makes mutant, inflexible, sickle-shaped red blood cells. These deformed cells block small blood vessels, causing pain, strokes, organ dysfunction and premature death.It affects 70,000 Americans and three million to five million people globally. At one time, most people with the disease died in childhood, but new treatments allow people to live into middle age.Hydroxyurea is a cancer pill that has been shown to stop the severe pain and prevent the need for blood transfusions in adults and older children with sickle cell disease.But little has been known about how it works in babies.For the study, Wang and colleagues enrolled 193 children from nine to 19 months at 13 US medical centres and a coordinating centre between October 2003 and September 2007.The children were randomly assigned to receive hydroxyurea or a placebo for two years, and 167 completed the trial.“We found a decrease in chest syndrome and hospitalization among trial participants who received hydroxyurea,” Dr Zora Rogers of the University of Texas Southwestern Medical Center, who worked on the study, said in a statement. “We used to offer hydroxyurea as secondary prevention, but with these findings, it could become the primary preventive measure.”The researchers said the study will likely change how doctors treat very young children with sickle cell anaemia.“This medication reduces painful events, the major crisis patients fear about sickle cell disease,” Rogers said.It also helps prevent chest syndrome a life-threatening condition, similar to pneumonia, in which an infection or sickle cells become trapped in the lungs.“Patients may still experience painful crises, but the events are much less frequent and severe,” she said.Rogers said the next step is to make a standardized liquid form of the drug.