U.O.C. Ematologia II con Talassemia, A.O. Villa Sofia-V. Cervello, Palermo Dipartimento di Scienze Statistiche e Matematiche S. Vianelli, Università di Palermo, Palermo U.O.S. Talassemia Pediatrica, A.O. Cardarelli, Napoli U.O.C. Medicina Trasfusionale, A.O. Villa Sofia-V. Cervello, Palermo, Italy.
Br J Haematol. 2011 Sep;154(5):545-55. doi: 10.1111/j.1365-2141.2011.08769.x. Epub 2011 Jun 28.
Blood transfusions may prevent and treat serious complications related to sickle-cell disease (SCD) when performed according to specific guidelines. However, blood transfusion requirements in SCD inevitably lead to increased body iron burden. An adequate chelation treatment may prevent complications and reduce morbidity and mortality. This review evaluates the effectiveness, safety and costs of chelation treatment. The included trials were examined according to the recommendations of the American College of Cardiology (ACC) and the American Heart Association (AHA). Overall, 14 trials and a total of 502 patients with SCD were included in this review. Deferoxamine alone (s.c. or i.v.), deferiprone alone or versus deferoxamine, deferasirox versus deferoxamine and combined treatment with deferoxamine plus deferiprone were included and evaluated in the analysis. Only two randomized clinical trials have been reported. The results of this analysis suggest that use of chelation treatment in SCD to date has been based on little efficacy and safety evidence, although it is widely recommended and practised. The cost/benefit ratio has not been fully explored. Further research with larger randomized clinical trials needs to be performed.
输血可以根据具体的指导方针预防和治疗与镰状细胞病（SCD）相关的严重并发症。然而，SCD 中的输血需求不可避免地导致体内铁负荷增加。适当的螯合治疗可以预防并发症，降低发病率和死亡率。本综述评估了螯合治疗的有效性、安全性和成本。根据美国心脏病学会（ACC）和美国心脏协会（AHA）的建议，对纳入的试验进行了检查。总的来说，这项综述纳入了 14 项试验和总共 502 名 SCD 患者。单独使用去铁胺（皮下或静脉）、单独使用地拉罗司或地拉罗司与去铁胺比较、地拉罗司与去铁胺比较以及联合使用去铁胺加地拉罗司的治疗方法都进行了分析。只有两项随机临床试验报告了结果。该分析结果表明，迄今为止，SCD 中螯合治疗的使用依据的是疗效和安全性证据不足，尽管它被广泛推荐和应用。尚未充分探讨成本效益比。需要进行更大规模的随机临床试验的进一步研究。
