How I treat non-transfusion-dependent ß-thalassemia.
Blood
; 142(11): 949-960, 2023 09 14.
Article
en En
| MEDLINE
| ID: mdl-37478396
ABSTRACT
The intricate interplay of anemia and iron overload under the pathophysiological umbrella of ineffective erythropoiesis in non-transfusion-dependent ß-thalassemia (NTDT) results in a complex variety of clinical phenotypes that are challenging to diagnose and manage. In this article, we use a clinical framework rooted in pathophysiology to present 4 common scenarios of patients with NTDT. Starting from practical considerations in the diagnosis of NTDT, we delineate our strategy for the longitudinal care of patients who exhibit different constellations of symptoms and complications. We highlight the use of transfusion therapy and novel agents, such as luspatercept, in the patient with anemia-related complications. We also describe our approach to chelation therapy in the patient with iron overload. Although tackling every specific complication of NTDT is beyond the scope of this article, we touch on the management of the various morbidities and multisystem manifestations of the disease.
Texto completo:
1
Bases de datos:
MEDLINE
Asunto principal:
Talasemia
/
Talasemia beta
/
Sobrecarga de Hierro
Tipo de estudio:
Diagnostic_studies
/
Etiology_studies
Idioma:
En
Revista:
Blood
Año:
2023
Tipo del documento:
Article
País de afiliación:
Mongolia