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Response to hydroxyurea therapy in β‐thalassemia
Author(s) -
Koren Ariel,
Levin Carina,
Dgany Orly,
Kransnov Tatyan,
Elhasid Ronit,
Zalman Lucia,
Palmor Haya,
Tamary Hannah
Publication year - 2008
Publication title -
american journal of hematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.456
H-Index - 105
eISSN - 1096-8652
pISSN - 0361-8609
DOI - 10.1002/ajh.21120
Subject(s) - transfusion therapy , medicine , thalassemia , beta thalassemia , blood transfusion , hemoglobinopathy , gastroenterology , hemolytic anemia
Abstract Although a relatively small number of previous studies suggest a modest response to hydroxyurea (HU) therapy in β‐thalassemia, more recent investigations have revealed that some transfusion‐dependent patients can become transfusion‐independent following HU therapy. Patients with Gγ XmnI polymorphism, several β‐globin mutations, and α‐thalassemia deletions were inconsistently reported to have significant responses to HU therapy. To better predict who may respond, we retrospectively evaluated the clinical response and the molecular background of 18 β‐thalassemia patients treated with HU for a mean of 46 months. The majority of transfusion‐dependent patients responded to HU therapy with 9 out of 11 (82%) becoming transfusion‐independent. Five thalassemia intermedia (TI) patients receiving occasional blood transfusion did not require any additional transfusions following therapy and two TI patients who had never received transfusions had a 2 g/dl increase in their hemoglobin level. The majority of β‐thalassemia major patients who became transfusion‐independent (7/9) were either homozygous (5) or heterozygous (2) for the XmnI polymorphism. No correlation was identified between response to therapy and the presence of specific β‐thalassemia mutations or α‐globin deletions. We conclude that further analysis of the degree of response of transfusion‐dependent β‐thalassemia patients to HU therapy, as well as, the impact of their genetic background on this response is required to identify patients likely to have significant response. Am. J. Hematol., 2008. © 2008 Wiley‐Liss, Inc.