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DC Field | Value | Language |
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dc.contributor.author | Yasara, N. | |
dc.contributor.author | Wickramarathne, N. | |
dc.contributor.author | Mettananda, C. | |
dc.contributor.author | Silva, I. | |
dc.contributor.author | Hameed, N. | |
dc.contributor.author | Attanayaka, K. | |
dc.contributor.author | Rodrigo, R. | |
dc.contributor.author | Wickramasinghe, N. | |
dc.contributor.author | Perera, L. | |
dc.contributor.author | Manamperi, A. | |
dc.contributor.author | Premawardhena, A. | |
dc.contributor.author | Mettananda, S. | |
dc.date.accessioned | 2022-02-25T06:08:23Z | |
dc.date.available | 2022-02-25T06:08:23Z | |
dc.date.issued | 2022 | |
dc.identifier.citation | Scientific Reports.2022:12(1):2752. | en_US |
dc.identifier.issn | 2045-2322 | |
dc.identifier.uri | http://repository.kln.ac.lk/handle/123456789/24521 | |
dc.description | Indexed in MEDLINE. | en_US |
dc.description.abstract | Hydroxyurea is an antimetabolite drug that induces fetal haemoglobin in sickle cell disease. However, its clinical usefulness in β-thalassaemia is unproven. We conducted a randomised, double-blind, placebo-controlled clinical trial to evaluate the efficacy and safety of hydroxyurea in transfusion-dependent β-thalassaemia. Sixty patients were assigned 1:1 to oral hydroxyurea 10-20 mg/kg/day or placebo for 6 months by stratified block randomisation. Hydroxyurea treatment did not alter the blood transfusion volume overall. However, a significantly higher proportion of patients on hydroxyurea showed increases in fetal haemoglobin percentage (89% vs. 59%; p < 0.05) and reductions in erythropoietic stress as measured by soluble transferrin receptor concentration (79% vs. 40%; p < 0.05). Based on fetal haemoglobin induction (> 1.5%), 44% of patients were identified as hydroxyurea-responders. Hydroxyurea-responders, required significantly lower blood volume (77 ± SD27ml/kg) compared to hydroxyurea-non-responders (108 ± SD24ml/kg; p < 0.01) and placebo-receivers (102 ± 28ml/kg; p < 0.05). Response to hydroxyurea was significantly higher in patients with HbE β-thalassaemia genotype (50% vs. 0%; p < 0.01) and Xmn1 polymorphism of the γ-globin gene (67% vs. 27%; p < 0.05). We conclude that oral hydroxyurea increased fetal haemoglobin percentage and reduced erythropoietic stress of ineffective erythropoiesis in patients with transfusion-dependent β-thalassaemia. Hydroxyurea reduced the transfusion burden in approximately 40% of patients. Response to hydroxyurea was higher in patients with HbE β-thalassaemia genotype and Xmn1 polymorphism of the γ-globin gene. | en_US |
dc.language.iso | en | en_US |
dc.publisher | Nature Publishing Group | en_US |
dc.subject | β-thalassaemia | en_US |
dc.subject | Randomised | en_US |
dc.title | A randomised double-blind placebo-controlled clinical trial of oral hydroxyurea for transfusion-dependent β-thalassaemia | en_US |
dc.type | Article | en_US |
Appears in Collections: | Journal/Magazine Articles |
Files in This Item:
File | Description | Size | Format | |
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41598_2022_Article_6774.pdf | 1.39 MB | Adobe PDF | View/Open |
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