Meerpohl Joerg J, Antes Gerd, Rücker Gerta, Fleeman Nigel, Motschall Edith, Niemeyer Charlotte M, Bassler Dirk
German Cochrane Centre, Institute of Medical Biometry & Medical Informatics and Pediatric Hematology & Oncology, Center for Pediatrics & Adolescent Medicine, University Medical Center Freiburg, Berliner Allee 29, Freiburg, Germany, 79110.
Cochrane Database Syst Rev. 2010 Nov 10(11):CD007461. doi: 10.1002/14651858.CD007461.pub2.
The myelodysplastic syndrome (MDS) comprises a diverse group of haematopoietic stem cell disorders. Due to symptomatic anaemia most patients require supportive therapy including repeated red blood cell (RBC) transfusions. In combination with increased iron absorption, this contributes to the accumulation of iron resulting in secondary iron overload and the risk of organ dysfunction and reduced life expectancy. Since the human body has no natural means of getting rid of excess iron, iron chelation therapy is usually recommended. However, whether the new oral chelator deferasirox leads to relevant benefit is unclear.
To assess the effectiveness and safety of oral deferasirox in people with myelodysplastic syndrome and iron overload.
We searched MEDLINE, EMBASE, The Cochrane Library, Biosis Previews, Web of Science, Derwent Drug File, XTOXLINE and three trial registries: Current Controlled Trials: www.controlled-trials.com, ClinicalTrials.gov: www.clinicaltrials.gov, ICTRP: www.who.int./ictrp/en/. Most recent searches of these databases: June 2010.
Randomised controlled trials comparing deferasirox with no therapy/placebo or with another iron chelating treatment schedule.
No studies eligible for inclusion in this review were identified.
No studies were included in this review. However, we identified one ongoing study comparing deferasirox with deferoxamine.
AUTHORS' CONCLUSIONS: We planned to report evidence from randomised clinical trials evaluating the effectiveness of deferasirox compared to either placebo/no treatment or other chelating regimens such as deferoxamine in people with myelodysplastic syndrome. However, no completed randomised trials addressing this question could be identified.One ongoing randomised study comparing deferasirox with placebo was identified and preliminary data will hopefully be available soon. These results will be important to inform physicians and patients on the advantages and disadvantages of this treatment option.
骨髓增生异常综合征（MDS）是一组异质性造血干细胞疾病。由于出现症状性贫血，大多数患者需要支持性治疗，包括反复输注红细胞（RBC）。这与铁吸收增加相结合，导致铁蓄积，进而引发继发性铁过载以及器官功能障碍风险和预期寿命缩短。由于人体没有自然排出过量铁的方法，通常建议进行铁螯合治疗。然而，新型口服螯合剂地拉罗司是否能带来相关益处尚不清楚。
评估口服地拉罗司治疗骨髓增生异常综合征伴铁过载患者的有效性和安全性。
我们检索了MEDLINE、EMBASE、Cochrane图书馆、生物学文摘数据库、科学引文索引、德温特药物文件、XTOXLINE以及三个试验注册库：当前对照试验（www.controlled-trials.com）、临床试验.gov（www.clinicaltrials.gov）、国际临床试验注册平台（www.who.int./ictrp/en/）。这些数据库的最新检索时间为2010年6月。
比较地拉罗司与无治疗/安慰剂或与另一种铁螯合治疗方案的随机对照试验。
未识别出符合本综述纳入标准的研究。
本综述未纳入任何研究。然而，我们识别出一项正在进行的比较地拉罗司与去铁胺的研究。
我们计划报告来自随机临床试验的证据，以评估地拉罗司与安慰剂/无治疗或其他螯合方案（如去铁胺）相比，在骨髓增生异常综合征患者中的有效性。然而，未识别出针对该问题的已完成随机试验。识别出一项正在进行的比较地拉罗司与安慰剂的随机研究，初步数据有望很快获得。这些结果对于告知医生和患者该治疗方案的优缺点将很重要。
