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Thalassemia Reports
Volume 9
Issue 1
10.4081/thal.2019.8101
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Article

Efficacy of Ruxolitinib as Inducer of Fetal Hemoglobin in Primary Erythroid Cultures from Sickle Cell and Beta-Thalassemia Patients

by
Alice Pecoraro
*,
Antonio Troia
,
Aurelio Maggio
and
Rosalba Di Marzo
Dipartimento di Oncologia ed Ematologia, U.O.C. Ematologia per le Malattie Rare del Sangue e degli Organi Ematopoietici, A.O. Ospedali Riuniti Villa Sofia-Cervello, Via Trabucco 180, 90146 Palermo, Italy
*
Author to whom correspondence should be addressed.
Thalass. Rep. 2019, 9(1), 8101; https://doi.org/10.4081/thal.2019.8101
Submission received: 7 February 2019 / Revised: 8 February 2019 / Accepted: 9 February 2019 / Published: 18 April 2019

Abstract

High levels of HbF may ameliorate the clinical course of β-thalassaemia and SCD. Hydroxyurea (HU) is the only HbF inducer approved for the treatment of patients. However not all patients respond to the treatment, for this reason it is noteworthy to identify new HbF inducers. Ruxolitinib is a JAK inhibitor that decreases the phosphorilation of STAT proteins. In particular STAT3 is a repressor of gamma-globin gene. The decrease of STAT3 phosphorilation could derepress gamma-globin gene and reactivate its trascription. In this study we evaluated the efficacy of ruxolitinib as inducer of HbF production. The analyses were performed in cultured erythroid progenitors from 16 beta-thalassemia intermedia (TI) and 4 sickle cell disease (SCD) patients. The use of quantitative RT-PCR technique allowed us to determine the increase of gamma-globin mRNA expression in human erythroid cultured cells treated with ruxolitinib. The results of our study demonstrated an increase in vitro of gamma-globin mRNA expression in almost all patients. These data suggest that ruxolitinib could be a good candidate to be used in vivo for the treatment of hemoglobinopathies.
Keywords: fetal hemoglobin; sickle cell; beta-thalassemia fetal hemoglobin; sickle cell; beta-thalassemia

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MDPI and ACS Style

Pecoraro, A.; Troia, A.; Maggio, A.; Di Marzo, R. Efficacy of Ruxolitinib as Inducer of Fetal Hemoglobin in Primary Erythroid Cultures from Sickle Cell and Beta-Thalassemia Patients. Thalass. Rep. 2019, 9, 8101. https://doi.org/10.4081/thal.2019.8101

AMA Style

Pecoraro A, Troia A, Maggio A, Di Marzo R. Efficacy of Ruxolitinib as Inducer of Fetal Hemoglobin in Primary Erythroid Cultures from Sickle Cell and Beta-Thalassemia Patients. Thalassemia Reports. 2019; 9(1):8101. https://doi.org/10.4081/thal.2019.8101

Chicago/Turabian Style

Pecoraro, Alice, Antonio Troia, Aurelio Maggio, and Rosalba Di Marzo. 2019. "Efficacy of Ruxolitinib as Inducer of Fetal Hemoglobin in Primary Erythroid Cultures from Sickle Cell and Beta-Thalassemia Patients" Thalassemia Reports 9, no. 1: 8101. https://doi.org/10.4081/thal.2019.8101

APA Style

Pecoraro, A., Troia, A., Maggio, A., & Di Marzo, R. (2019). Efficacy of Ruxolitinib as Inducer of Fetal Hemoglobin in Primary Erythroid Cultures from Sickle Cell and Beta-Thalassemia Patients. Thalassemia Reports, 9(1), 8101. https://doi.org/10.4081/thal.2019.8101

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