Efficacy of Ursodeoxycholic Acid with 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.

This study was to explore more efficient methods of prevention of VOD in patients with thalassemia. We explore the efficacy of VOD prophylaxis with ursodeoxycholic acid, lipo-prostaglandin E1 and low-dose unfractionated heparin in allogeneic heamatopoietic stem cell transplantation(HSCT) for patients with thalassemia. Methods:Fifty-eight children with β-thalassemia major received allo-SCT for 61 times. For the prevention of VOD, all patients received low-dose heparin(100-150IU/kg · d) and also received PGE1(1-1.5 · d) by continuous intravenous infusion from the beginning of conditioning treatment to the 20th day after allo-SCT. Patients who received ursodeoxycholic acid (12mg/kg.d,oral,from the beginning of conditioning treatment to the 90th day after allo-SCT)were used as the study group (n=33).Patients who did not received ursodeoxycholic acid were used as the control group (n=28).Results:VOD occurred in 3 patients(8.8%) in the study group. Ten patients in the control group had VOD(37.0%).There were no obvious drug adverse effects in the two groups.

Ursodeoxycholic acid with low-dose heparin and PGE1(study group) is more effective than low-dose heparin and PGE1 only (control group) for the prevention of VOD after allo-SCT for children with β-thalassemia major.

No relevant conflicts of interest to declare.