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目的探讨左卡尼汀在儿童重型β-地中海贫血异基因造血干细胞移植(allo-HSCT)过程中的应用价值。方法将41例拟行allo-HSCT的重型地贫患儿随机分为观察组25例及对照组16例,两组均采用环磷酰胺+白舒非+氟达拉宾+抗胸腺细胞球蛋白为主的预处理化疗方案,静脉予环孢素A(CsA)及口服吗替麦考酚酯(MMC)预防移植物抗宿主病(GVHD)。观察组移植前5 d(-5 d)至移植后20 d(+20 d)静脉应用左卡尼汀1 g,2次/d。监测两组-5 d~+31 d内血浆脑利钠肽前体(pro-BNP)水平及临床心功能不全相关指标。结果两组治疗后BNP均升高,+5~+15 d达峰值,+25 d后逐渐下降至正常范围;+6 d开始观察组pro-BNP显著低于对照组(P<0.05);观察组心功能不全事件发生率显著低于对照组(P=0.044)。结论左卡尼汀可有效降低重型地贫患儿在allo-HSCT过程中的心功能不全事件发生率,其机制可能通过为心肌提供足够能量及减少有害物质生成从而降低心肌损害程度。
Objective To investigate the value of levocarnitine in the treatment of allo-HSCT in children with β-thalassemia major. Methods Forty-one children with severe thalassemia major who were allo-HSCT were randomized into observation group (n = 25) and control group (n = 16). Both groups were treated with cyclophosphamide plus baisuflun + fludarabine + antithymocyte globulin Predominant chemotherapy regimen, intravenous cyclosporin A (CsA) and oral mycophenolate mofetil (MMC) for the prevention of graft versus host disease (GVHD). The observation group received intravenous levocarnitine 1 g twice a day for 5 days (-5 d) before transplantation and 20 d (+20 d) after transplantation. The levels of plasma brain natriuretic peptide (pro-BNP) and clinical dysfunction were monitored in the two groups from -5 d to + 31 d. Results After treatment, BNP levels in both groups increased from the 5th day to the 5th day, and gradually decreased to the normal range after 25 days. The level of pro-BNP in the observation group from the 6th day was significantly lower than that in the control group (P <0.05) The incidence of cardiac dysfunction group was significantly lower than the control group (P = 0.044). Conclusion L-carnitine can effectively reduce the incidence of cardiac dysfunction in allo-HSCT in children with severe thalamelthaemia. Its mechanism may reduce the degree of myocardial damage by providing sufficient energy to the myocardium and reducing the production of harmful substances.