Untreated Anemia in Nontransfusion-dependent β-thalassemia: Time to Sound the Alarm

espite advances in management and the consequent improvement in survival, a diagnosis of β -thalassemia continues to confer considerable burden on patients, their families, and the healthcare system. This is primarily driven by the complexity of management, including reg- ular transfusion and iron chelation, the chronicity of the disease, need for comprehensive monitoring, and multidisciplinary care; complicated by poor response/tolerance, adherence, or access to treatment. curative therapies such as bone marrow transplantation and gene manipulation use of is impeded need for specialized and expertise, strict subject or donor selection, and high cost. The future outlook for disease control and prevention of morbidity and mortality when curative therapy is not or practical will fall to optimizing the use of conventional and supportive therapies, novel disease modifying agents, and/or their combination. hematopoiesis, endocrine and bone disease, leg ulcers, throm- bosis, pulmonary hypertension). In a retrospective cohort study following untreated NTDT patients for 10 years, a hemoglobin level g/dL was associated with a significantly worse mor-bidity-free survival than those with hemoglobin levels ≥10 g/ dL (~4-fold increase in hazard ratio on multivariate analysis).

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