Scientific Literature

Management of Age-associated Medical Complications in Patients with β-Thalassemia

Motta, I., Mancarella, M., Marcon, A., Vicenzi, M., & Cappellini, M. D. Expert review of hematology, 13(1), 85-94. (2020).

Introduction: β-Thalassemia syndromes are among the most common monogenic disorders worldwide. Clinically, on the basis of the severity of the phenotype, β-thalassemias are classified into two groups: transfusion-dependent thalassemia (TDT) and non-transfusion-dependent thalassemia (NTDT). In the last few decades, considerable advances in understanding the pathophysiology of β-thalassemia have significantly improve d patient management, which has led to an increase in the life span of these subjects. However, new complications associated with aging are emerging, and β-thalassemias are becoming a growing concern for the health care systems.

Areas covered: The present review focused on the age-related complications in adults with β-thalassemia. Among the cardiovascular diseases, which remain a major cause of morbidity, pulmonary hypertension and arrhythmias are exhibiting increased prevalence. Adrenal insufficiency and bone disease are emerging as endocrinological complications that require proper treatment. Moreover, age-related complications observed in the general population, including cancers and renal disease, should not be neglected.

Expert opinion: The present study reviews the management of above-stated complications in adults with β-thalassemia based on the experience of a referral center. It is noteworthy that clinical trials in this context are limited, and the expert opinion offered in the present report stems mainly from direct clinical experience.

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