Optimal management of β thalassaemia intermedia.
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.
Department of Internal Medicine, Division of Haematology & Oncology, American University of Beirut Medical Centre, Riad El Solh 1107 2020, Beirut, Lebanon. ataher@aub.edu.lb
Ali T
AT
Taher
Khaled M
KM
Musallam
Maria Domenica
MD
Cappellini
David J
DJ
Weatherall
D001803:Blood Transfusion; D005319:Fetal Hemoglobin / Q000096:biosynthesis; D005838:Genotype; D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use; D010641:Phenotype; D013156:Splenectomy; D017086:beta-Thalassemia / Q000235:genetics / Q000503:physiopathology / Q000628:therapy*
D007502:Iron Chelating Agents; D005319:Fetal Hemoglobin