TY - JOUR
T1 - Management of age-associated medical complications in patients with β-thalassemia
AU - Motta, Irene
AU - Mancarella, Marta
AU - Marcon, Alessia
AU - Vicenzi, Marco
AU - Cappellini, Maria Domenica
PY - 2020
Y1 - 2020
N2 - 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.
AB - 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.
KW - Adrenal insufficiency
KW - anemia
KW - arrythmias
KW - bone disease
KW - cancer
KW - cardiovascular disease
KW - iron overload
KW - pulmonary hypertension
KW - renal disease
KW - thalassemia
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U2 - 10.1080/17474086.2020.1686354
DO - 10.1080/17474086.2020.1686354
M3 - Review article
C2 - 31661637
AN - SCOPUS:85074977467
VL - 13
SP - 85
EP - 94
JO - Expert Review of Hematology
JF - Expert Review of Hematology
SN - 1747-4086
IS - 1
ER -