Olivieri N F, Brittenham G M
Hospital for Sick Children, Toronto, Ontario, Canada.
Blood. 1997 Feb 1;89(3):739-61.
Iron-chelating therapy with deferoxamine in patients with thalassemia major has dramatically altered the prognosis of this previously fatal disease. The successes achieved with deferoxamine, as well as the limitations of this treatment, have stimulated the design of alternative strategies of iron-chelating therapy, including orally active iron chelators. The development of the most promising of these, deferiprone, has progressed rapidly over the last 5 years; data from several trials have provided direct and supportive evidence for its short-term efficacy. At the same time, the toxicity of this agent mandates a careful evaluation of the balance between risk and benefit of deferiprone in patients with thalassemia, in most of whom long-term deferoxamine is safe and efficacious therapy.
对于重型地中海贫血患者，采用去铁胺进行铁螯合治疗已显著改变了这种既往致命疾病的预后。去铁胺取得的成功以及该治疗方法的局限性，促使人们设计铁螯合治疗的替代策略，包括口服活性铁螯合剂。其中最有前景的去铁酮在过去5年中进展迅速；多项试验的数据为其短期疗效提供了直接且有力的证据。与此同时，鉴于该药物的毒性，对于地中海贫血患者，必须仔细评估去铁酮风险与获益之间的平衡，而对于大多数患者而言，长期使用去铁胺是安全有效的治疗方法。
