Iron chelation therapy in the management of thalassemia: the Asian perspectives

Abstract

Worldwide, thalassemia is the most commonly inherited hemolytic anemia, and it is most prevalent in Asia and the Middle East. Iron overload represents a significant problem in patients with transfusion-dependent beta-thalassemia. Chelation therapy with deferoxamine has traditionally been the standard therapeutic option but its usage is tempered by suboptimal patient compliance due to the discomfort and demands associated with the administration regimen. Therefore, a great deal of attention has been focused on the development of oral chelating agents. Deferiprone, even though available for nearly two decades in Asia with recent encouraging data on cardiac iron removal and long-term efficacy, has serious adverse effects including agranulocytosis and neutropenia which has impeded it from routine clinical practice. A novel oral chelator; deferasirox is effective throughout a 24 h dosing period and both preclinical and clinical data indicate that it successfully removes both hepatic and cardiac iron. In Asia, optimal management of severe thalassemia patients and the availability and access to oral iron chelators still presents a major challenge in many countries. In this regard, the development and implementation of consensus guidelines for management of Asian patients with transfusion-dependent thalassemia will be a major step towards improving and maintaining the continuity of patient care.

Deferiprone versus deferoxamine in thalassemia intermedia: Results from a 5‐year long‐term Italian multicenter randomized clinical trial

“…However, an increased liver iron concentration (LIC) has been shown to be a crucial marker of increased morbidity in TI [2]. Therefore, despite their limited need for transfusions, TI patients may develop clinically relevant iron overload, and guidelines currently recommend initiating iron chelation therapy when either the LIC increases to >7 mg/g dry tissue or the ferritin level is higher than 500 lg/l [3,4]. Therefore, it is important to establish the efficacy, safety, and optimal doses for chelation regimens in this population.…”

Deferiprone (GPO‐L‐ONE®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand

“…In Thailand, where more than 12,000 new thalassemia patients are born each year; at least 50,000 surviving patients are expected to be transfusion-dependent thalassemia [16]. This group is composed mainly by severe Hb E/b thalassemia, b thalassemia major and severe form of nondeletional Hb H disease and few others.…”

“…This group is composed mainly by severe Hb E/b thalassemia, b thalassemia major and severe form of nondeletional Hb H disease and few others. However, this figure might not be limited to Thailand, other Southeast Asian countries where might have a limited research data on thalassemia and hemoglobinopathies also possess a similar scale of the problem since inherited globin gene carriers are highly prevalent in nearly every country [16]. Because of the large numbers of affected thalassemia patients who require blood transfusion, transfusional iron overload is undoubtedly a significant health care problem in several developing countries in Asia.…”

“…Because of the large numbers of affected thalassemia patients who require blood transfusion, transfusional iron overload is undoubtedly a significant health care problem in several developing countries in Asia. Moreover, it is also a major resource burden for the local governments to allocate their limited health care budget to purchase imported life-saving iron chelators for their thalassemia patients (reviewed in [16]). …”

“…Even though, this hepatic complication seems to be associated on other factors in particular hepatitis C infection besides deferiprone exposure alone [22], most clinicians in Asia who have limited experience with deferiprone stay reluctant to try on using this oral chelator in their iron overload patients who fail to DFO therapy [16]. Concerning the fact that infectious diseases e.g.…”