Siriraj-Thalassemia Research Program, Haematology/Oncology Division, Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, 10700, Thailand.
Department of Pediatrics, University of Malaya, Kuala lumpur, Malaysia.
Int J Hematol. 2009 Nov;90(4):435-445. doi: 10.1007/s12185-009-0432-0. Epub 2009 Oct 29.
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.
在全球范围内，地中海贫血是最常见的遗传性溶血性贫血，在亚洲和中东地区最为普遍。铁过载是输血依赖型β地中海贫血患者的一个严重问题。去铁胺螯合疗法一直是标准的治疗选择，但由于治疗方案带来的不适和需求，患者的依从性并不理想，因此其应用受到限制。因此，人们非常关注口服螯合剂的开发。地拉罗司虽然在亚洲已经使用了近 20 年，并且最近在心脏铁去除和长期疗效方面有令人鼓舞的数据，但它有严重的副作用，包括粒细胞缺乏症和中性粒细胞减少症，这阻碍了它在常规临床实践中的应用。一种新型口服螯合剂——地拉罗司，在 24 小时的给药期间都有效，并且临床前和临床数据表明，它能成功地去除肝脏和心脏中的铁。在亚洲，严重地中海贫血患者的最佳管理以及口服铁螯合剂的可及性和可获得性，在许多国家仍然是一个重大挑战。在这方面，制定和实施亚洲输血依赖型地中海贫血患者管理共识指南，将是朝着改善和维持患者治疗连续性迈出的重要一步。
