Abstract:
:Our understanding of the molecular and pathophysiological mechanisms underlying the disease process in patients with thalassemia intermedia (TI) has substantially increased over the past decade. TI encompasses a wide clinical spectrum of beta-thalassemia phenotypes. Some TI patients are asymptomatic until adult life, whereas others are symptomatic from as young as 2 years. A number of clinical complications commonly associated with TI are rarely seen in thalassemia major, including extramedullary hematopoiesis, leg ulcers, gallstones, thrombosis, and pulmonary hypertension. There are a number of options currently available for managing patients with TI, including transfusion therapy, iron chelation therapy, modulation of fetal hemoglobin production, and hematopoietic stem cell transplantation. However, at present, there are no clear guidelines for an orchestrated optimal treatment plan.
journal_name
Hemoglobinjournal_title
Hemoglobinauthors
Cappellini MD,Musallam KM,Taher ATdoi
10.3109/03630260903351528subject
Has Abstractpub_date
2009-01-01 00:00:00pages
S145-59eissn
0363-0269issn
1532-432Xjournal_volume
33 Suppl 1pub_type
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