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重型β地中海贫血患者移植后的口服铁螯合疗法

Posttransplant oral iron-chelating therapy in patients with beta-thalassemia major.

作者信息

Yesilipek M Akif, Karasu Gulsun, Kazik Mediha, Uygun Vedat, Ozturk Zeynep

机构信息

Department of Pediatric Hematology-Oncology, Akdeniz University School of Medicine, Antalya, Turkey.

出版信息

Pediatr Hematol Oncol. 2010 Aug;27(5):374-9. doi: 10.3109/08880011003739463.

Abstract

Allogeneic hematopoetic stem cell transplantation (HSCT) is the only radical cure of beta-thalassemia. However, iron overload remains a cause of morbidity and mortality in posttransplant period. The authors present 7 patients as a preliminary report who underwent bone marrow transplant (BMT) and received oral chelating therapy (deferasirox) because of poor compliance to phlebotomy and desferrioxamine. The patients investigated mainly for possible side effects of deferasirox. No negative effect was seen in aspartate aminotransferase (AST), alanine aminotransferase (ALT), hemoglobin (Hb), and donor chimerism of the patients while serum ferritin levels significantly reduced (P = .018). Although serum creatinin significantly increased (P = .034), it was in normal limits in all patients. The authors believe that this report shows promising findings to plan further studies to clarify clinical safety and efficacy of deferasirox in posttransplant period.

摘要

异基因造血干细胞移植(HSCT)是β地中海贫血的唯一根治方法。然而,铁过载仍然是移植后时期发病和死亡的一个原因。作者报告了7例患者作为初步报告,这些患者接受了骨髓移植(BMT),并因对放血疗法和去铁胺依从性差而接受口服螯合疗法(地拉罗司)。主要对患者进行地拉罗司可能的副作用调查。患者的天冬氨酸转氨酶(AST)、丙氨酸转氨酶(ALT)、血红蛋白(Hb)和供体嵌合率未见负面影响,而血清铁蛋白水平显著降低(P = 0.018)。虽然血清肌酐显著升高(P = 0.034),但所有患者均在正常范围内。作者认为,本报告显示了有前景的发现,可为进一步研究以阐明地拉罗司在移植后时期的临床安全性和疗效提供依据。

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