Indian Journal of Human Genetics
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ORIGINAL ARTICLE
Year : 2013  |  Volume : 19  |  Issue : 2  |  Page : 251-258

Comparison of in-vitro and in-vivo response to fetal hemoglobin production and γ-mRNA expression by hydroxyurea in Hemoglobinopathies


1 National Institute of Immunohematology, Hematogenetics, 13th Floor, K.E.M. Hospital Campus, Parel, Mumbai, India
2 Department of Hematology, 10th Floor, K.E.M. Hospital, Parel, Mumbai, India
3 Department of Pediatrics, Nanavati Hospital, Vileparle (W), Mumbai, India

Correspondence Address:
Roshan Colah
National Institute of Immunohematology, 13th Floor, N. M. S. Bldg, K.E.M. Hospital Campus, Parel, Mumbai - 400 012
India
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Source of Support: None, Conflict of Interest: None


PMID: 24019630

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Background: Hydroxyurea, which induces Fetal hemoglobin (HbF) synthesis, is the only drug widely used in different hemoglobinopathies; however, the response is very variable. We compared the efficacy of hydroxyurea in-vitro in erythroid cultures and in-vivo in the same patients with different hemoglobinopathies to induce HbF production and enhance γ-messenger RNA expression. Materials and Methods: A total of 24-patients with different Hemoglobinopathies were given hydroxyurea and their response was studied in-vivo and in-vitro on mononuclear cells collected from them simultaneously. Results: A total of 57.7% of patients (responders) showed no further crisis or transfusion requirements after hydroxyurea therapy with a mean increase in fetal cells (F-cells) of 63.8 ± 59.1% and γ-mRNA expression of 205.5 ± 120.8%. In-vitro results also showed a mean increase in F-cells of 27.2 ± 24.7% and γ-mRNA expression of 119.6% ± 65.4% among the treated cells. Nearly 19.0% of the partial-responders reduced their transfusion requirements by 50% with a mean increase in F-cells of 61.2 ± 25.0% and 28.4 ± 25.3% and γ-mRNA-expression of 21.0% ± 1.4% and 80.0% ± 14.1% in-vivo and in-vitro respectively. The non-responders (15.3%) showed no change in their clinical status and there was no significant increase in F-cells levels and γ-mRNA expression in-vivo or in-vitro. Conclusion: Thus, this method may help to predict the in-vivo response to hydroxyurea therapy; however, a much larger study is required.


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