Hydroxyurea (HU) has been used to treat patients with non transfusion-dependent β-thalassemia major (β-TM) at the Thalassemia Research Center, Sari, Mazandaran Province, Islamic Republic of Iran since 1996. This study was performed to summarize and to share our experience. Medical records of all patients with β-thalassemia (β-thal) attending our center were reviewed in January 2013. Definition of β-TM was based on complete blood count (CBC), hemoglobin (Hb) electrophoresis, and for some patients, by the amplification refractory mutation system-restriction fragment length polymorphism (ARMS-RFLP) method. Patients who had not been transfused before, or had only occasionally had blood transfusions, were selected. Age at first blood transfusion, initial HU therapy and time of study was extracted from the records. The lowest Hb level before using HU and the last Hb value when on the HU regimen as well as the difference, were reported. Number of saved packed red cells was calculated according to duration of HU use and the usual needs of the patients. Hydroxyurea was discontinued before a planned pregnancy and during gestation and lactation periods. Hydroxyurea was discontinued for male patients willing to reproduce. A p value of <0.05 was considered statistically significant. It was consistent with 1856 patients/year, and 3542 units of blood were saved. We found HU to be effective and safe in treating patients with non transfusion-dependent β-TM. We strongly recommend HU therapy.
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http://dx.doi.org/10.3109/03630269.2013.869229 | DOI Listing |
Mediterr J Hematol Infect Dis
January 2025
Department of Diabetes and Endocrinology, Whittington Hospital, University College London, London, UK.
Background: Current guidelines for screening glucose dysregulation (GD) in patients with transfusion-dependent thalassemia (TDT) recommend an annual 2-hour oral glucose tolerance test (OGTT) starting at the age of 10 years.
Objective: Assessment of adherence to OGTT screening in patients with TDT.
Methods: A questionnaire was distributed to 18 Thalassemia Centers in 10 different countries, targeting factors influencing adherence to annual OGTT screening in specialized multidisciplinary pediatric and adult TDT units and identifying strategies to improve adherence to OGTT in TDT patients.
Mediterr J Hematol Infect Dis
January 2025
Department of Endocrinology, The 923rd Hospital of the Joint Logistics Support Force of the People's Liberation Army, Nanning, China.
Asian J Transfus Sci
December 2022
Cellular and Molecular Research Center, Birjand University of Medical Sciences, Birjand, Iran.
Background: Thalassemia is one of the most common congenital hemoglobinopathies globally. Regular red blood cell (RBC) transfusion is of paramount importance in the treatment of thalassemia patients. However, this practice increases the risk of alloimmunization.
View Article and Find Full Text PDFVox Sang
January 2025
Department of Transfusion Medicine, Postgraduate Institute of Medical Education and Research (PGIMER), Chandigarh, India.
Background And Objectives: Great variations may be observed in the haemoglobin (Hb) content of packed red blood cell (PRBC) units prepared by different methods. This study aimed to assess the Hb increment in thalassaemia major patients transfused with leucoreduced PRBCs (LPRBCs) prepared by two different methods: (i) standard leucoreduced PRBCs (SLPRBCs) and (ii) leucoreduced PRBCs prepared by a new method where leucoreduction of whole blood is done first (NLPRBCs).
Materials And Methods: This prospective, randomized, controlled trial included 80 adult thalassaemia major patients who were randomized into two groups of 40 each.
J Hematol Oncol
January 2025
Sylvester Comprehensive Cancer Center, University of Miami, Miami, FL, USA.
Background: Olutasidenib is a potent, selective, oral, small molecule inhibitor of mutant IDH1 (mIDH1) which induced durable remissions in high-risk, relapsed/refractory (R/R) mIDH1 AML patients in a phase 1/2 trial. We present a pooled analysis from multiple cohorts of the phase 1/2 trial of patients with R/R AML who received combination olutasidenib and azacitidine therapy.
Methods: Adult patients with mIDH1 AML received 150 mg olutasidenib twice daily plus standard-of-care azacitidine (OLU + AZA) and were evaluated for response and safety.
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