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Initiation Of New Study To Help Children With Sickle Cell Anaemia

SCA is an inherited disease in which the oxygen-carrying protein in the red blood cells is abnormal, giving the red cells a twisted (sickled) shape that can disrupt circulation, leading to pain, stroke, and other disabling and sometimes fatal complications. //

"Sickle cell anemia is a relentless disease that increasingly disables children as they grow," according to Dr.Ware, an expert in the field of Haematology.

The current treatment for children with SCA who have suffered strokes includes monthly blood transfusions to provide red cells that are not sickled. This ongoing treatment must later be combined with nightly injections of a drug (Desferal ?) that eliminates excessive iron buildup caused by regular blood transfusions.

Excessive iron damages internal organs--a condition called haemochromatosis. The nightly injections of Desferal, an iron chelator (molecules that bind to metals so they can be eliminated from the body), are painful and inconvenient, often prompting children to forego the injection, since iron buildup does not initially cause the child discomfort, but the injections always do.

In an attempt to increase the compliance level of the patients to the therapy, a national Phase III clinical trial to investigate whether a new combination treatment can prevent a secondary stroke in children with sickle cell anemia (SCA) and eliminate the need for nightly injections with a drug that reduces iron overload in these patients has been planned.

The study named Stroke With Transfusions Changing to Hydroxyurea (SWiTCH ) has been designed to determine if hydroxyurea can replace blood transfusions and if monthly blood withdrawal can substitute for daily injections to treat iron overload.

The goal of SWiTCH is to compare patient outcomes using blood transfusions with outcomes using hydroxyurea. Hydroxyurea will not completely eliminate the sickled red cells, but it is expected to stimulate production of eno ugh red cells carrying fetal hemoglobin to reduce SCA symptoms.

The study will also compare the efficacy of daily at-home injections of Desferal with the less painful and more convenient monthly removal of blood (phlebotomy) at a medical facility. Half of the children in SWiTCH will remain on the standard treatment (transfusions and chelator) while the other half will be treated with the alternative treatment (hydroxyurea and phlebotomy).

The study is supported by a grant of more than $18 million from the National Institutes of Health (National Heart, Lung, and Blood Institute) and includes 20 major pediatric sickle cell centers.

SWiTCH holds promise for greatly simplifying and improving the long-term care of children with the disease who have suffered strokes and finally give a better way to enhance the lives of children who are fighting it hard against the disease.


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