TY - JOUR
T1 - Management of the aging beta-thalassemia transfusion-dependent population – The Italian experience
AU - Pinto, Valeria Maria
AU - Poggi, Maurizio
AU - Russo, Rodolfo
AU - Giusti, Andrea
AU - Forni, Gian Luca
PY - 2019
Y1 - 2019
N2 - Thalassemia is among the most common monogenic diseases worldwide. Stem cell transplantation can be curative but is reserved for young patients, as probably gene therapy will be in the future. Adult thalassemia patients are treated with transfusion therapy and iron chelation, and improvements in the safety of transfusion protocols, use of iron chelation, monitoring of iron overload, and management of comorbidities have substantially prolonged survival, increasing the proportion of adult patients in the thalassemic population. However, older patients are more likely to develop multiple disease-related morbidities, including osteoporosis, endocrine disorders, liver disease, renal dysfunction, and cancer. Thus, the main objective of this article is to describe new challenges posed by the increasing life expectancy of patients with thalassemia, focusing on data from Italy where there is a well-documented history of thalassemia management. It is hoped that the mortality and morbidity benefits already seen in patients with thalassemia will continue to improve with ongoing advances in the quality of treatment.
AB - Thalassemia is among the most common monogenic diseases worldwide. Stem cell transplantation can be curative but is reserved for young patients, as probably gene therapy will be in the future. Adult thalassemia patients are treated with transfusion therapy and iron chelation, and improvements in the safety of transfusion protocols, use of iron chelation, monitoring of iron overload, and management of comorbidities have substantially prolonged survival, increasing the proportion of adult patients in the thalassemic population. However, older patients are more likely to develop multiple disease-related morbidities, including osteoporosis, endocrine disorders, liver disease, renal dysfunction, and cancer. Thus, the main objective of this article is to describe new challenges posed by the increasing life expectancy of patients with thalassemia, focusing on data from Italy where there is a well-documented history of thalassemia management. It is hoped that the mortality and morbidity benefits already seen in patients with thalassemia will continue to improve with ongoing advances in the quality of treatment.
KW - Anemia
KW - Hemoglobinopathy
KW - Iron chelation
KW - Iron overload
KW - Thalassemia
KW - Transfusion
UR - http://www.scopus.com/inward/record.url?scp=85070419452&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85070419452&partnerID=8YFLogxK
U2 - 10.1016/j.blre.2019.100594
DO - 10.1016/j.blre.2019.100594
M3 - Review article
AN - SCOPUS:85070419452
VL - 38
JO - Blood Reviews
JF - Blood Reviews
SN - 0268-960X
M1 - 100594
ER -