Galanello R, Campus S
Dipartimento di Scienze Biomediche e Biotecnologie, Ospedale Regionale Microcitemie, Azienda Sanitaria Locale 8, Università di Cagliari, Cagliari, Italia.
Acta Haematol. 2009;122(2-3):155-64. doi: 10.1159/000243800. Epub 2009 Nov 10.
Iron overload is one of the major causes of morbidity in patients with thalassemia major. Deferiprone (DFP), an orally active iron chelator, emerged from an extensive search for new drugs to treat iron overload. Comparative studies have shown that at comparable doses the efficacy of DFP in removing body iron is similar to that of desferoxamine (DFO). In retrospective and prospective studies, DFP monotherapy was significantly more effective than DFO in the treatment of myocardial siderosis in thalassemia major. DFP can be used in combination with DFO in the management of severe iron overload. This chelation regimen is tolerable and attractive for patients unable to comply with standard DFO infusions or with inadequate response to DFP monotherapy. DFP has a well-known long-term safety profile. Agranulocytosis is the most serious side effect associated with its use, occurring in about 1% of the patients. More common but less serious side effects are gastrointestinal symptoms, arthralgia, zinc deficiency, and fluctuating transaminase levels.
铁过载是重型地中海贫血患者发病的主要原因之一。去铁酮（DFP）是一种口服活性铁螯合剂，是在广泛寻找治疗铁过载的新药过程中出现的。比较研究表明，在同等剂量下，DFP清除体内铁的疗效与去铁胺（DFO）相似。在回顾性和前瞻性研究中，DFP单药治疗在治疗重型地中海贫血的心肌铁沉着症方面比DFO显著更有效。DFP可与DFO联合用于治疗严重铁过载。这种螯合方案对于无法遵守标准DFO输注或对DFP单药治疗反应不足的患者来说是可耐受且有吸引力的。DFP具有众所周知的长期安全性。粒细胞缺乏症是与其使用相关的最严重副作用，约1%的患者会发生。更常见但不太严重的副作用是胃肠道症状、关节痛、锌缺乏和转氨酶水平波动。
