Our understanding of the molecular and pathophysiological mechanisms underlying the disease process in patients with β thalassaemia intermedia (TI) has substantially increased over the past decade. The hallmark of disease process in patients with TI includes ineffective erythropoiesis, chronic haemolytic anaemia, and iron overload. There are a number of options currently available for managing patients with TI including splenectomy, transfusion therapy, iron chelation therapy, modulation of fetal haemoglobin production, and several other agents targeting specific clinical complications. Limited studies assessed the efficacy and safety of these modalities; hence, there are currently no clear guidelines for managing patients with TI. Until solid evidence-based guidelines are available, individualised treatment should be entertained.

Optimal management of ß thalassaemia intermedia / A. T. Taher, K. M. Musallam, M. D. Cappellini, D. J. Weatherall. - In: BRITISH JOURNAL OF HAEMATOLOGY. - ISSN 0007-1048. - 152:5(2011 Mar), pp. 512-523.

Optimal management of ß thalassaemia intermedia

M. D. Cappellini
Penultimo
;
2011

Abstract

Our understanding of the molecular and pathophysiological mechanisms underlying the disease process in patients with β thalassaemia intermedia (TI) has substantially increased over the past decade. The hallmark of disease process in patients with TI includes ineffective erythropoiesis, chronic haemolytic anaemia, and iron overload. There are a number of options currently available for managing patients with TI including splenectomy, transfusion therapy, iron chelation therapy, modulation of fetal haemoglobin production, and several other agents targeting specific clinical complications. Limited studies assessed the efficacy and safety of these modalities; hence, there are currently no clear guidelines for managing patients with TI. Until solid evidence-based guidelines are available, individualised treatment should be entertained.
Fetal haemoglobin induction; Iron chelation; Splenectomy; Thalassaemia intermedia; Transfusion
Settore MED/09 - Medicina Interna
mar-2011
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/156449
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