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使用去铁酮单一疗法使重型地中海贫血患者的机体铁储存水平维持在正常范围内长达4.5年。

Maintenance of normal range body iron store levels for up to 4.5 years in thalassemia major patients using deferiprone monotherapy.

作者信息

Kolnagou Annita, Kontoghiorghes George J

机构信息

Postgraduate Research Institute of Science, Technology, Environment and Medicine, Limassol, Cyprus.

出版信息

Hemoglobin. 2010 Jun;34(3):204-9. doi: 10.3109/03630269.2010.485890.

Abstract

New gold standard protocols are tested for the complete removal of iron overload in thalassemia using the International Committee on Chelation (ICOC) Maintaining Normal Body Iron combination protocol therapy of deferiprone (L1)/deferoxamine (DFO) and maintenance of normal range body iron store levels (NRBISL) using L1 monotherapy. Deferiprone (80-100 mg/kg/day) was administered for a mean of 21.3 months (range 7-91, total 171) in eight thalassemia major patients (four males and four females, 29-47 years) who have achieved serum ferritin (mean 108.5 microg/L, range 25-408), cardiac T2* (mean 31.5 ms, range 24-41) and liver T2* (30.1 ms, range 9.1-41) magnetic resonance imaging (MRI) relaxation times. At the end of the study normal range MRI T2* relaxation time was maintained in all eight patients with a cardiac mean of T2* 30.3 ms (range 23-37), liver T2* 28.8 ms (range 9.8-43) and serum ferritin with a mean of 173.7 microg/L (range 86-406). In one patient, this NRBISL was maintained for more than 4.5 years. No toxic side effects have been observed during the L1 monotherapy period.

摘要

采用国际螯合委员会(ICOC)的去铁酮(L1)/去铁胺(DFO)联合维持正常机体铁含量方案疗法以及使用L1单一疗法维持正常范围机体铁储存水平(NRBISL),对新的金标准方案进行了测试,以彻底清除地中海贫血患者的铁过载情况。对8例重型地中海贫血患者(4例男性和4例女性,年龄29 - 47岁)给予去铁酮(80 - 100 mg/kg/天),平均给药21.3个月(范围7 - 91个月,共171个月),这些患者的血清铁蛋白(平均108.5 μg/L,范围25 - 408 μg/L)、心脏T2*(平均31.5 ms,范围24 - 41 ms)和肝脏T2*(30.1 ms,范围9.1 - 41 ms)磁共振成像(MRI)弛豫时间均已达标。研究结束时,所有8例患者的MRI T2弛豫时间均维持在正常范围内,心脏平均T2为30.3 ms(范围23 - 37 ms),肝脏T2*为28.8 ms(范围9.8 - 43 ms),血清铁蛋白平均为173.7 μg/L(范围86 - 406 μg/L)。其中1例患者的这种NRBISL维持了超过4.5年。在L1单一疗法期间未观察到毒性副作用。

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