低剂量肝素联合前列腺素E1预防重型β-地中海贫血患儿异基因造血干细胞移植后的肝静脉阻塞病

目的：研究低剂量肝素联合前列腺素E1(PGE1)预防重型β-地中海贫血患儿异基因造血干细胞移植后的肝静脉阻塞病(hepatic veno-occlusive disease, HVOD)的临床效果。方法：43例重型β-地中海贫血患儿接受异基因造血干细胞移植，研究组给予肝素钠每日100 IU/kg，24 h微量泵维持静脉注射，PGE1每日 7.2 μg/kg，输液泵持续静脉输注10 h以上;对照组给予PGE1每日7.2 μg/kg，输液泵持续静脉输注10 h以上，两组均用至移植后第30天，以预防HVOD。结果：研究组6例发生HVOD（6/23，26.1%），其中3例轻度、3例中度；对照组12例发生HVOD(12/20，60.0%)，其中3例轻度、3例中度、6例重度。研究组HVOD的发生率低于对照组，差异具有统计学意义(P<0.05)。经治疗后研究组5例治愈，1例死于其他并发症，对照组10例治愈，2例死亡。两种预防方案在使用过程中均未出现明显的药物毒副反应。结论：该研究所采用的低剂量肝素钠+PGE1预防HVOD的方案比单独采用PGE1更有效。［中国当代儿科杂志，2007，9（4）：343-346］

Efficacy of low-dose heparin and prostaglandin E1 in the prevention of hepatic veno-occlusive disease after allogenic hematopoietic stem cell transplantation in children with β-thalassemia major

OBJECTIVE: Hepatic veno-occlusive disease (HVOD) is one of the most serious complications after allogenic hematopoietic stem cell transplantation (allo-SCT). Endothelial injury, leading to deposition of coagulation factors in the terminal hepatic venules, is believed to the key event in the pathogenesis of HVOD. This study was designed to explore the efficacy of low-dose heparin and prostaglandin E1 (PGE1) in the prevention of HVOD after allo-SCT in children with beta-thalassemia major. METHODS: Forty-three children with beta-thalassemia major received allo-SCT. For the prevention of HVOD, 23 of the 43 patients received low-dose heparin (100 IU/kg.d) and also received PGE1 (7.2 microg/kg x d) by continuous intravenous infusion (study group) from the beginning of conditioning treatment to the 30th day after allo-SCT. Patients who received continuous infusions of PGE1 (7.2 microg/kg x d) alone were used as the control group (n=20). RESULTS: HVOD occurred in 6 patients (26.1%) in the study group (3 mild, 3 moderate). Twelve patients in the control group had HVOD (60.0%) (3 mild, 3 moderate, 6 severe)(P < 0.05). In the study group, 5 cases of HVOD were treated successfully and one died from other complications. Of the 12 cases of HVOD in the control group, 10 patients were treated successfully and two patients died from HVOD. There were no obvious drug adverse effects in the two groups. CONCLUSIONS: Low-dose heparin and PGE1 is more effective than PGE1 alone for the prevention of HVOD after allo-SCT.