Management of the aging beta-thalassemia transfusion-dependent population - The Italian experience.

Centro della Microcitemia, Anemie Congenite e Dismetabolismo del Ferro, European Reference Networks for rare diseases ERN-EuroBloodNet, Ospedale Galliera, Mura Delle Cappuccine 14, 16128 Genoa, Italy. Electronic address: valeria.pinto@galliera.it. Department of Endocrinology, Sant'Andrea Hospital, via di Grottarossa 1035-1039, 00189 Rome, Italy. Electronic address: mpoggi@ospedalesantandrea.it. Nephrology, Dialysis, and Transplant Unit, Ospedale Policlinico San Martino, Largo R. Benzi 10, 16132 Genoa, Italy. Electronic address: rodolfo.russo@hsanmartino.it. Rheumatology Unit, Department of Locomotor System, La Colletta Hospital - ASL3, Via del Giappone 5, 16011 Arenzano, Italy. Electronic address: andreagiusti6613@gmail.com. Centro della Microcitemia, Anemie Congenite e Dismetabolismo del Ferro, European Reference Networks for rare diseases ERN-EuroBloodNet, Ospedale Galliera, Mura Delle Cappuccine 14, 16128 Genoa, Italy. Electronic address: gianluca.forni@galliera.it.

Blood reviews. 2019;:100594
Full text from:

Abstract

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.

Methodological quality

Publication Type : Review

Metadata

MeSH terms : Iron Chelating Agents