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Hydroxyurea May Be A Useful Alternative To Erythrocyte Transfusions For Children With Severe Beta Thalassaemia

New data suggest that hydroxyurea can elevate total foetal haemoglobin (HbF) production to levels that permit the discontinuation of regular transfusions for children with transfusion-dependent beta-thalassaemia major.

Although hydroxyurea has been shown to successfully elevate Hb production in patients with sickle cell disease and patients with beta-thalassaemia intermedia, the benefit to patients with beta-thalassaemia major remains controversial.

In a collaborative study by researchers from Algeria and France, Dr. M. Bradai and colleagues treated 7 children, 2 with beta-thalassmia intermedia and 5 with beta-thalassaemia major, with hydroxyurea. Most of the patients had suffered major complications from transfusions, including anaphylactic reactions, severe chills and fevers, multiple red cell alloimmunisation, lack of venous access, and haemochromatosis following transfusion with cutaneous, hepatic and gonadic manifestations.

Treatment with hydroxyurea was initiated 15 to 65 days (median of 34 days) after the last transfusion for each patient. Blood counts and biochemical parameters, including creatinine, AST, ALT, BUN, and alkaline phosphatase, were determined monthly. The alkali denaturation technique was used to calculate HbF levels.

Increased Hb levels were found in all patients in the first month after beginning hydroxyurea, and these levels remained increased at 19 months follow-up (range of 13 to 21 months). A comparison of first and last Hb values show that the mean Hb values increased from 4.5 g/dL to 7.9 g/dL in 5 children with thalassaemia major and 6.5 to 10.5 g/dL in 2 children with thalassaemia minor. Because of this increase, transfusions were stopped in 5 patients. One patient with thalassaemia major developed a pulmonary infection and was treated with 2 erythrocyte concentrates because her Hb fell to 4.7 g/dL.

Overall, the study reported good clinical and haematological profile and safety in the children after receiving hydroxyurea. Increases were noted in both the mean corpuscular volume (from 72 to 87.4 fl) and HbF (from 90.9 to 97.7), and significant decreases were found in mean circulating erthroblasts/100 leukocytes (from 149,400 to 13,400) (p=0.01). Although 2 patients reported nausea with the initiation of treatment, this resolved overtime.

The authors report that their study supports previous data that show the benefit of hydroxyurea in patients with beta-thalassaemia intermedia, and is the first to show a benefit in children with beta-thalassaemia major as well.

Based on these promising results, 7 more children with beta-thalassaemia have been treated with hydroxyurea. After only months of follow-up, 2 patients are no longer receiving transfusions and 2 patients are receiving transfusions less often (1 every 2 months instead of once a month).

The authors conclude that hydroxyurea may be a useful alternative to transfusions for some patients with severe beta-thalassaemia. This is particularly important, they emphasize, in countries like Algeria where blood supplies are limited.
Blood 2003 Apr 17;[epub ahead of print]. "Hydroxyurea can eliminate transfusion requirements in children with severe beta thalassemia"

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